The Role of Medical Affairs in a Successful Commercial Launch A FirstWord Dossier ExpertViews Report

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1 NOVEMBER 2015 The Role of Medical Affairs in a A FirstWord Dossier ExpertViews Report

2 Published Copyright 2015 Doctor s Guide Publishing Limited All rights reserved. No part of this publication may be reproduced or used in any form or by any means graphic, electronic or mechanical, including photocopying, recording, taping or storage in information retrieval systems without the express permission of the publisher. This report contains information from numerous sources that Doctor s Guide Publishing Limited believes to be reliable but for which accuracy cannot be guaranteed. Doctor s Guide Publishing Limited does not accept responsibility for any loss incurred by any person who acts or who fails to act as a result of information published in this document. Any views and opinions expressed by third parties and reproduced in this document are not necessarily the views and opinions of Doctor s Guide Publishing Limited. Any views and opinions expressed by individuals and reproduced in this document are not necessarily the views and opinions of their employers. Cover image: JumalaSika ltd AdobeStock PhotoDisc

3 Contents Executive summary... 1 Methodology and objectives...3 Contributors...4 Introduction...5 The key elements of medical affairs... 7 The medical affairs role builds from early development to pre-launch... 9 When is medical affairs first involved with a potential new medicine?...9 Involvement from concept...9 Strategic involvement from early clinical trials...10 Increased activity during Phase III...12 Planning for product launch...14 Working with the commercial team...14 How can the medical function be more influential?...16 Evidence generation beyond the registration package...18 Early engagement is vital for market access...18 Aligning market access with medical affairs...19 Generating the right evidence...20 Establishing what evidence will be required for reimbursement...20 Payer requirements vary Planning the medical affairs study programme...24 Medical affairs studies add value to the R&D studies...24 Understanding the data gaps...24 i

4 Deciding on a study programme...25 Being prepared for Phase IIIb and Phase IV...26 Planning local studies...28 Early real-world studies...28 Developing a communications strategy...30 Strategic partnerships and customer engagement...34 KOL identification and mapping...34 Medical provides expertise and credibility...36 Advisory boards...38 MSLs can help to identify local needs...40 Medical affairs as the bridge between R&D and commercial...42 Medical affairs activity increases from regulatory filing...44 Internal training...45 MSL training...45 Sales & marketing...46 The changing MSL role following regulatory approval Launch and post-launch...48 The medical affairs role broadens after initial launch...48 Product launch is an evolving process...50 Challenges remain for medical affairs...53 Financial resources and planning...53 Medical affairs still viewed as gatekeepers...55 Key findings...58 ii

5 Executive summary The launch of a new medicine is traditionally a crucial time for any pharmaceutical company. Historically, regulatory approval was enough to ensure market access, but the market environment has changed significantly. Payers and health technology assessment agencies now play a central role in healthcare decision making and, with the emphasis on value, companies need to differentiate their medicine over existing therapies in a more restrictive market environment. A deep understanding of patient needs and payer requirements is therefore more important than ever. As the main communication channel between a pharmaceutical company and the medical community, the medical affairs team has a critical role to play in a successful commercial launch. The early involvement of medical affairs can provide valuable insights into the market environment, disease area, unmet medical need and what patients want from a new medicine. Medical and scientific expertise combined with this customer knowledge results in the medical affairs team having the ability to look at the whole picture, including how the treatment of a disease is likely to change over time, what the competition is doing and what will be required for market access if a new product candidate is approved. So, while the R&D team is focused on the registration dossier, the medical affairs team is able to identify potential gaps in the data requirements for reimbursement and to plan studies to generate the evidence needed to fill those gaps. Medical affairs teams are uniquely placed to understand and communicate complex, comprehensive scientific information to external stakeholders, including medical practitioners, scientific researchers, patient advocates and government authorities. Internally, the medical affairs function provides a vital link between the R&D team and the commercial team, which can facilitate the transition of a new product from development to commercial launch. The medical affairs team has an important role in educating colleagues about the disease and the new drug and is likely to have an advantage over the commercial team in terms of market knowledge, having been working in the field for some time. 1

6 In some companies, however, medical affairs still has some way to go in establishing its position as a true partner in the launch process, rather than being seen as simply playing a supportive role to other teams. At the same time, people within medical affairs need to see themselves in a partnership role rather than as a service function. Our experts note that medical affairs teams can often be swamped with administrative tasks, leaving little time for the more strategic role that could provide greater value to their company. 2

7 Methodology and objectives This FirstWord Dossier ExpertViews report provides insights into the evolving structure and organisation of medical affairs teams as internal partners and key participants in cross-functional teams. The information for this report was gathered from multiple sources, including primary research from in-depth telephone interviews with senior medical affairs professionals from within the pharmaceutical industry, supported by secondary research, mainly from publically available sources of information. Research objectives: To understand when the medical affairs team first becomes involved with a potential new product candidate and why it is important to be involved at that stage; To understand the role of the medical affairs team during the development phases of a new product and through commercial launch; To learn about some of the challenges that the medical affairs team faces in managing support for a new product launch; To understand how medical affairs is positioned as a vital link between R&D and commercial and the key role the team plays in the transfer of knowledge; To gain insight into medical affairs best practices. 3

8 Contributors The contributors were selected on the basis of their knowledge, experience and expertise in medical affairs. Interviews were conducted with the following individuals from a cross-section of pharmaceutical and biotechnology companies: Global Medical Affairs Director Top 10 Pharma Company Global Medical Affairs Lead Top 10 Pharma Company Franchise Head, Medical Communications Top 10 Pharma Company Executive Medical Director, Medical Affairs Top 10 Pharma Company Former Vice President, Head of Medical Affairs Europe and Canada Top 10 Pharma Company Vice President of Medical Affairs, US Medium-sized Pharma Company Julian Jenkins, Medical EVP, Global Head of R&D and Medical Affairs, Finox Biotech Dr Swapneel Anaokar, Medical Director, BluMed (Medical Affairs Consultancy) 4

9 Introduction The medical affairs role is an increasingly valuable asset to the pharmaceutical industry as relationships between companies and external stakeholders become more complicated. 1 The medical affairs team is the main communication channel between a pharmaceutical company and the medical and scientific community and, as such, has a critical role to play in any successful commercial launch. 2 Following changes in the pharmaceutical industry, including more stringent regulations and governmental scrutiny, the medical affairs function has grown beyond its original brief to take on a more pivotal role in managing the strategic scientific components involved in bringing innovative therapeutic products to market. 3 A successful product launch has traditionally created sustained value for a pharmaceutical company. The healthcare market has become more difficult in recent years, however, and regulatory approval is no longer a guarantee of market access, but simply the first hurdle. Pharmaceutical companies are having to adapt to a more restrictive market environment where the emphasis is on value. A recent study indicated that between 60 percent and 90 percent of new drugs launched in Europe faced access restrictions by payers and governments due to a lack of either perceived differentiation or cost-effectiveness in comparison with existing therapies. 4 At the same time, product launches are becoming smaller and more numerous and it is more important than ever to stand out from the crowd. In 2014, the FDA approved 41 novel new drugs, either as new molecular entities under new drug applications (NDAs) or as new therapeutic biologics under Biologics License Applications (BLAs). This was the highest number of new drug approvals for more than a decade and compared with an average of 25 per year from 2005 to Morgon, P. (September 2014). Medical Affairs: From Info Gatekeeper to Value Driver. Retrieved from 2 McGuire, R. (2013). Pharma Product Launch: A Busy Time for Medical Affairs Teams. Retrieved from 3 Joyce, D. (Apri, 2013). Medical Affairs: The Catalyst for Effective Medical Communications. Retrieved from 4 Kunst et al., (June 2013). A new pharma launch paradigm: From one size fits all to a tailored product approach. Retrieved from 5

10 Among these, around 41 percent (17) were first-in-class approvals that may have, for example, unique mechanisms of action. They included targeted drugs to treat relatively small patient populations for serious and life-threatening conditions, such as Keytruda (pembrolizumab; Merck & Co.) for unresectable metastatic melanoma and Lynparza (olaparib; AstraZeneca) for advanced ovarian cancer. The Center for Drug Evaluation and Research (CDER) also noted that 41 percent of new drugs were approved to treat orphan diseases that affect 200,000 or fewer Americans, which is significant due to the previous lack of drugs available to treat these conditions. 5 This move was repeated in Europe, with the European Medicines Agency (EMA) recommending marketing authorisation for 17 new drugs intended for the treatment of rare diseases in 2014, from a total of 41 new active substances. The EMA noted that this was the highest number of orphan designated medicines recommended for marketing authorisation in a year. 6 The trend towards the development of targeted therapies has increased over the last few years. These personalised medicines are an attractive proposition, partly because they can command premium prices. 7 They are also characterised by large amounts of complex medical and scientific information that support regulatory submission. Because we are learning more about how to target specific areas that cause disease, the understanding of disease states is becoming more complex, particularly in oncology. 8 An expert interviewed for this report highlights that in order to be involved in launch planning, you need expertise in the disease area and the science to be able to understand and communicate the data; this is one area in which medical affairs plays a critical role. 5 US FDA CDER (January 2015). Novel New Drugs Summary Retrieved from 6 EMA (April 2015). Annual Report Retrieved from 7 Evers et al., (March 2013). Managing for success in biomarker R&D: Challenges and opportunities. Retrieved from mckinsey%20on%20personalized%20medicine%20march% ashx 8 FirstWord (October 2015). Tomorrow s MSLs. Retrieved from 6

11 The key elements of medical affairs Experts interviewed for this report broadly identified three main elements of the medical affairs function, which continue throughout the lifecycle of a drug: Evidence generation; Communication; and Strategic partnerships or customer engagement. Medical affairs evidence generation is informed by the insights gained during early discussions with stakeholders, and is additional to the formal clinical studies required for registration. Communication can be viewed as an umbrella term that includes both written and verbal communication to internal and external stakeholders and is undertaken throughout the lifecycle of a product. The development of partnerships with external stakeholders begins in the early phases of development and continues for the life of the product, albeit with a different emphasis at different stages of the lifecycle. The typical things that any medical affairs organisation is doing can be summarised by three elements. The first one is evidence generation, or data generation. The second area is around communication and the third area is around strategic partnerships or customer engagement. Global Medical Affairs Director The dissemination and communication of data, market access, reimbursement, planning for Phase IIIb and Phase IV studies [and] engagement with medical experts. Those are the things that are our core. Franchise Head, Medical Communications 7

12 Figure 1: Medical affairs key launch activities Data generation and analysis Training internal colleagues Planning Phase IIIb/IV studies Medical affairs Continuing medical education Engagement with strategic partners/msl activities Dissemination and communication of data Source: FirstWord research 8

13 The medical affairs role builds from early development to pre-launch When is medical affairs first involved with a potential new medicine? The timing of initial involvement with a potential new drug therapy varies significantly between companies. In some organisations, the medical affairs team is involved at a strategic level from as early as proof of concept. In others, the team becomes involved later in the process, when the product candidate enters Phase II or Phase III trials. Involvement from concept The early involvement of medical affairs can provide valuable input into identifying unmet medical need to ensure development of the right products. While involvement at this stage may not be typical in pharma companies, not undertaking early market research could potentially result in the development of an expensive mistake due to a lack of understanding of the unmet needs or competitive environment. Market research should guide study design so that the new drug can truly satisfy unmet medical need. 9 Indeed, the EMA advocates very early dialogue between patient groups, pharmaceutical companies, regulators and payers on areas of unmet medical need to help ensure that new medicines coming to market respond to patients needs. 10 As the main channel of communication between the drug company and its customers, the medical affairs team can be better placed to understand the market than the R&D (research and development) department and is able to provide value in terms of customer knowledge, including what patients want from a new medicine. My experience has been from concept, to identify an unmet need in clinical care and suggest that we should develop a new treatment for that. So it s before there s even a target for a drug. Global Head of R&D and Medical Affairs 9 FirstWord (May 2015). Building Strong Cross-functional Medical Affairs Teams. Retrieved from 10 EMA (April 2015). Annual Report Retrieved from 9

14 Having established that unmet need exists, the medical affairs team can assist with market knowledge, such as who the main competitors are and what additional value a new therapy could provide. Basic scientists would then look at that particular need and try and find out as much as possible in terms of, What s the current treatment? What s the disease pathology? What do people know about potential candidate targets or treatments, to address that unmet need? From a medical affairs perspective, you re in there, trying to identify the needs but the discovery people will be doing most of the work in this early phase, critically reviewing the science and, where appropriate, validating the science often by replicating key experiments. Global Head of R&D and Medical Affairs This early strategic involvement can happen several years before a potential product even enters human trials. Strategic involvement from early clinical trials While involvement of the medical affairs team is not universally applied during the early phases of clinical development, there is an increasing realisation of the importance of medical affairs input into the clinical trial programme from Phase II. A medical affairs lead notes that while most work has traditionally been done from Phase III onwards, this limits the role of medical affairs to mainly disseminating data. Earlier involvement enables medical affairs to look at the bigger picture and to project the evidence requirements for market access in the future. There is an attempt to get medical affairs involvement early on, before Phase III. The reason being, if you get involved when you re in Phase III, all you can do is use the data as it comes out of trials. You haven t been able to influence the study design. Global Medical Affairs Lead In this scenario, the medical affairs role is based around communicating the evidence, building confidence and having scientific discussions. For medical affairs to have a more strategic role, involvement needs to be early enough to feed information back to the R&D team at the planning stage. 10

15 But as medical affairs role expands, you re talking to experts and you find out what they are looking for. So you should be then able to put that back into the R&D plans so that the evidence which is being generated is appropriate. But you can t really do that if you re only starting to be involved in Phase III. So there is an attempt to try and get involvement from Phase I onwards. Global Medical Affairs Lead In some companies, the medical affairs team becomes involved with a product candidate when Phase II studies are underway. At this time, it is likely to be at a strategic level, as part of a cross-functional team. Dialogue with medical affairs can help the team to gain a better understanding of the marketplace ahead of the Phase III programme, as well as identify which additional studies are likely to be needed for market access. From Phase II, when you get your POC [proof-of-concept] study and you re designing your Phase III programme there should be some interaction with medical affairs, to understand and to advise on what needs to be done when it comes to the clinical and payer landscape in order to facilitate and optimise market access. Former Vice President, Head of Medical Affairs Europe and Canada We typically get involved as early as Phase II, in a very limited capacity, one person at a time. We may assign an MSL [medical science liaison] lead to keep an eye on the therapeutic area, on the KOL network and attending some of the key congresses. Vice President of Medical Affairs, US We are involved as early as Phase II to start thinking about strategy, including launch and post-launch activities, such as Phase IIIb and Phase IV trials, as well as activities required for market access in various regions and countries. But we do that as part of a cross-functional product team. Medical affairs sits on that team but we don t have an in-place medical affairs team at that point. Executive Medical Director 11

16 Increased activity during Phase III The role of medical affairs tends to increase during the Phase III programme. At this time, the medical affairs team would be put in place. An executive medical director interviewed for this report notes that the team would expand at the time when the Phase III pivotal trial results become available. At the beginning of Phase III, which is sometimes referred to as Phase IIIa, medical affairs would have involvement in multiple areas. These are likely to include: Building familiarity with the science and mechanism of action of the new medicine; Working with market access and HEOR [health economics outcomes research] to define the data required for the value proposition; Developing the Phase IIIb/IV study plan; Medical education activities around the disease pathophysiology and Phase II data; Expanding the KOL network and building a continuing engagement plan; and Working more closely with the commercial team. As you start the Phase III programme, as you notice the sites in the US that are involved in the clinical trial, this very small team will start to work closely with some commercial counterparts. There is a build up towards getting more familiar with the new medicine, more familiar with the therapeutic area if needed, and then more familiar with the KOL network. Vice President of Medical Affairs, US While Phase III is going on, we would be more carefully defining and then refining our Phase IIIb/IV plan. What we would like and it depends on the product and the disease area and the company s desire to spend money at risk or not but in general the trigger for launching the Phase IIIb plan would be positive results out of Phase III. So you would need to have at least the high-level concept and the protocol synopses for your Phase IIIb studies in place. A second thing you would be doing would be working with your market access/heor colleagues to really define the data that s required for the value proposition and to obtain reimbursement. If it s a new mechanism of action that isn t in the marketplace, then as you re going into Phase III you re probably wanting to build some of the excitement around the 12

17 science related to your mechanism of action. So a big component would be some medical education programmes, sharing some of the Phase II data at congresses, perhaps some medical education about unmet need within the pathophysiology of the disease that points towards the importance of your mechanism of action. You would also be building your medical expert engagement plan. You might already have in place a global steering committee and/or global advisory board for your programme even before you started Phase III, but you would continue to engage with them around the launch. Then you might, as you were moving forward in Phase III, be starting to develop regional and country thought leaders around your product and the mechanism of action. Executive Medical Director Figure 2: Medical affairs input from concept to Phase III Involvement from concept Identify unmet medical need Input market knowledge Suggest a new treatment is developed Phase I/ Phase II Involvement may be as part of a cross-functional team Input clinical and market knowledge into Phase III trial design Attend key congresses Start to plan Phase IIIb/IV studies Phase III Build familiarity with the new drug Work with market access and health economics teams to define data required Develop and refine Phase IIIb/IV study plan Share Phase II data at congresses Medical education around disease pathophysiology and mechanism of action Increase contact with KOL [key opinion leader] network and build continuing engagement plan Work more closely with commercial team Source: FirstWord research 13

18 Planning for product launch The medical affairs role in launch planning is likely to vary between companies. One medical director suggests that to be involved in launch planning you need to be expert in the disease area and in the science to understand the medicine; you also need to be able to understand the clinical data and to be able to communicate that effectively. As the pharmaceutical industry moves towards the development of differentiated medicines that treat smaller patient populations, the science is becoming more complex and the medical affairs team has a crucial role in understanding and communicating this to healthcare professionals (HCPs) and payers. It takes time to build this expertise, however, so the medical affairs team should be involved early in the launch planning process. One of the questions you should ask is, when any organisation sets up a launch planning process, should they recognise more that there is greater need for medical leadership within that, especially the way the world is going? The world is now headed more towards differentiated medicines which treat smaller populations of patients. The science tends to be more innovative, so doctors often are not as familiar with it. It s very important that when doctors prescribe medicines they fully understand the science and they fully understand how best to use the medicine and how best to interpret the prescribing information. That is where the medical function is absolutely critical. Sometimes those elements tend to be overlooked in launch planning. The emphasis often tends to be still very commercial. Working with the commercial team Medical Director In some companies, the medical affairs team will work in partnership with commercial colleagues as part of a cross-functional collaboration. In this scenario, the global medical affairs and commercial teams may work together to review and evaluate country-led launch plans. An executive medical director highlights that launch planning is a country-level activity, where the global commercial and medical affairs teams play a central role in reviewing and evaluating these plans. 14

19 Country by country we re involved in evaluating, contributing to and testing the individual country launch plans. Launches are done at a local and a country level so each country develops their launch plan. That s done by local commercial and local medical. Those launch plans are then in-depth reviewed and challenged at the global level by global commercial, global medical and sometimes development as well. For many of the major countries, global medical usually has a very big role in the actual launch meeting. So in addition to helping them with their launch plans, we usually attend and participate in those meetings as well. Executive Medical Director In other companies, medical affairs may play more of a supporting role to the commercial team. There is an argument, however, that medical affairs could play a leadership role in some aspects of launch planning and the strategic placement of a new product. Indeed, a recent study suggests a systematic approach to product launch is required, beginning with identifying an unmet medical need in a disease area and then developing deep customer insight as a basis for truly differentiated product positioning. 11 The expertise to undertake these critical tasks lies within the medical affairs team, which can convey the needs of key decision-makers and thus guide commercial strategies through internal education. Companies need to ask, Are there elements which medical needs to lead and not commercial? The commercial forecast and those kinds of things commercial needs to lead on. But everything else [in the strategic plan for the product] is around, how is this disease going to evolve over time? How would physician behaviour change in terms of how they treat these patients? Who else is launching in that field? What additional evidence do you need? How does that influence your Phase IIIb studies? The disease area knowledge and knowledge of how the medicines are used, what other medicines are there, how do doctors treat patients? That expertise lies with the medical organisation. You could easily have a medic who s leading that aspect of launch planning. Medical Director 11 Ahlawat et al., (March 2014). The Secret of Successful Drug Launches. Retrieved from 15

20 Importantly, the medical affairs role in launch planning should not be considered secondary to the commercial role. One expert highlights that launch planning should ideally be a partnership between the medical affairs and commercial teams. The medical function needs to be engaged at the same time as the commercial function. It shouldn t be the commercial function sets up something, they run with it for a year and then they feel the need for medical so they bring them in later. It should happen at the same time. The bottom line is medical and commercial need to be partners [with] equal onus on medical leadership. How can the medical function be more influential? Medical Director How well medical affairs can work with commercial during launch planning often depends on the individuals involved. A medical director notes that this can work both ways. Some medics want more ownership responsibility, whereas others are happy to play a supportive role. At the same time some commercial leads will only go to medical affairs when necessary, whereas others can be more engaging. Some commercial leaders can be a bit more engaging and they respect that they re equal partners in this and they involve the medic more and let them have the leadership position as well. Sometimes there needs to be a bit more uniformity around that. A launch planning activity is not just a commercially led activity, it s a joint medical/commercial activity. So the leadership profile of the medical function needs to be raised and a bit more accountability given to medics. That varies across organisations [but] it is critical for the medical function to be successful. Medical Director In terms of strategy, launch planning involves prioritisation and compromise, and decisions are often made on a purely commercial basis. When you re in an international role and you re launch planning, you ve got the whole world in front of you from Latin America all the way to Australasia and in between across Europe and the US. It s often impossible to meet everybody s needs. If you try to connect with affiliate countries across the globe, everybody will highlight some different needs in order to make the drug successful. In terms of strategy for launch planning one of the challenges is making some trade-offs. 16

21 You can t do it all, so you ve either got to focus on what you can do now or there needs to be a phasing plan. You might still want to do it all but it won t happen all at the same time. Then how do you make those trade-offs? How do you prioritise? Medical Director The real challenge for medical affairs is in influencing decisions that are not purely based on future sales figures, but may make a real difference in terms of unmet medical need and patient access to the medicine. This, the medical director continues, is the responsibility of the pharma industry when launching medicines. In 99.9 percent of the time, those trade-offs are made by commercial based on the sales strategy for the market. Where the challenge often lies for the medic is that sometimes you need to look beyond sales. There might be a very high unmet patient need and if you generate some data it s genuinely going to help doctors make some important decisions on how to use your medicine in patients. Doing that study may not have high returns in terms of sales numbers, but it makes a genuine difference to how that medicine might be used. This might not always make the best business sense, but it s part of the responsibility of launching medicines in the pharma industry. I think the challenge for the medical function is to be able to negotiate to not make all decisions 100 percent based on what makes business sense. Of course that s an important driver, but there are other things as well when you re talking about patients, disease and drugs. Medical Director 17

22 Evidence generation beyond the registration package Key insights Medical plays an important role in establishing What is the right evidence? There are two levels of evidence: clinical studies for regulators and additional studies for market access Medical studies should provide additional evidence to Phase III The first step is to gather insights such as unmet need and payer requirements Ideally the protocol for Phase IIIb will be in place during Phase IIIa Specific local needs should be considered but there will be compromises Source: FirstWord research and analysis Early engagement is vital for market access Historically, if a pharmaceutical product gained regulatory approval, it could be launched on the market. Nowadays, however, regulatory approval is only the first hurdle towards market access, and additional studies beyond the pivotal Phase III trials are often required to demonstrate value to payers and obtain reimbursement status. 12 A medical director notes that not only is the medical role evolving, but the commercial role is changing as well. Until recently, commercialisation was about positioning and having a trained sales force engaging with doctors. I think we re beyond that now. The medical role becomes more important as the emphasis shifts towards, What s the right evidence which meets customer needs and how best can it be interpreted and communicated? Medical Director 12 FirstWord (May 2015). Building Strong Cross-functional Medical Affairs Teams. Retrieved from 18

23 Aligning market access with medical affairs Market access is critical to the success of any pharmaceutical company, and most have a dedicated market access organisation. This may be a stand-alone function or it may come under the commercial umbrella. As the market environment has become more challenging and payers increasingly require evidence of the value of a new medicine, there is an argument that market access should be more closely aligned to medical affairs. Indeed, a global medical director notes that some organisations are beginning to merge market access with medical affairs. He goes on to explain that market access can have differing interpretations depending on your point of view. If you put on a commercial hat it really means how can you get the best price for your medicine which will then drive your forecast and profits? That s a very simple commercial way of looking at it. Then if you put on a medical hat it really means what data do the payers need to see on your medicine to feel comfortable enough to pay for it? What are the health economic arguments? How are you going to generate the data? When are you going to generate that evidence? How do different payers needs vary? Are the data going to suit everyone s needs? More importantly, once you have the data, how do you interpret and communicate that in a way which convinces the payers? Clearly that then becomes a market access definition which slants more towards evidence generation and evidence communication, which is medical ownership. Medical Director If the definition of market access is aligned with evidence generation, interpretation and communication to payers, the argument is strengthened for this coming under the umbrella of medical affairs rather than commercial. You could easily argue that market access could sit under medical leadership so that you have the right data generated and communicated appropriately. I can see that already happening in some organisations where market access, health outcomes and health economics tend to sit under a common umbrella [and] that s moving gradually closer to medical than to commercial. Medical Director 19

24 Generating the right evidence The medical affairs team has the ability to take a holistic view of the market environment and project what might happen in the future in terms of the disease area and treatment landscape. The clinical team leads usually tend to focus more on how to design a trial based on what regulators might want and how to get the drug registered. But there s a much bigger picture there with regard to, what s going to happen in five years time when you actually launch the medicine? How would the disease area or the treatment paradigm have changed? What would be payers expectations? What might be the challenges that doctors face which vary across the countries and regions? Medical Director Having taken a broad view, the medical affairs team can ensure that the company is made aware of the evidence requirements of payers, HCPs and sometimes patients, and that these are either addressed through the Phase III programme or a strategy is put in place for additional studies in Phase IIIb/IV to help generate these data. If the wider picture is not considered, this medical director cautions, the company could register a medicine but then not get access because payers are unwilling to pay for it or the data is not solid enough to meet the needs of all customers. I think sometimes those elements can get overlooked by the R&D physician, because they may not be close enough to some of the commercial realities or customer needs or what happens in the markets at a global level. That s where medical can partner the clinical lead and supplement that knowledge, working with the external medical community in the countries. Medical Director Establishing what evidence will be required for reimbursement For one global medical affairs lead, getting the resources in place early enough in the product lifecycle so that everything is ready as soon as a product is approved is a challenge. He suggests that nobody s going to think about reimbursement until you ve got the licence. While everyone else is focused on regulatory approval, he suggests that medical affairs has an important role to play at both global and country levels to establish what evidence is going to be required for reimbursement and to ensure this is in place at the right time. 20

25 Licensing is only a small part of the journey of the medicine in the current environment. You need somebody to go and make the case after the licence, to get it recommended or or included in the formulary for it to be reimbursed in most of the Western countries for funding. Global Medical Affairs Lead He highlights that health economics and medical affairs should be closely aligned, and that this may be as part of a cross-functional team. Ultimately health economics relies on the medical data, so you can t do that without understanding the data, understanding the clinical practice, understanding how the physicians are going to assess it. So it is important that it [the health economics team] works very closely with medical affairs. Global Medical Affairs Lead Payer requirements vary Addressing the needs of payers, in terms of generating the appropriate evidence of the value of a new medicine compared with the current standard of care, needs to be considered several years before a potential product launch. You might have the best science and you might have the best dataset, you might try to differentiate yourself, but if the payers don t see any health economic value in the medicine, they won t fund it. If you don t address that three or four years ahead of launch, then it s going to hit you hard one year before launch when you get into payer negotiations. Medical Director One of the problems with evidence generation, however, is that payer requirements vary. While regulators have clear guidelines on how to develop medicines for different disease areas, including what kind of studies they want to see and the data they would like be generated from them in terms of specific endpoints, there is no central repository that companies can interrogate to ascertain the needs of payers. For this reason, market access involves talking to payers to establish what they need and then negotiating with them to find some common ground. Since additional studies to fill the gaps in the evidence after Phase III are likely to be led by medical affairs, there is a case for this to be a medical-led function. 21

26 When it comes to payers, you ve got different payers in different countries with different needs. Some might ask for more, some might ask for less. You don t have a common place where it s regulated so you know exactly what needs to be done. You need to go out and have conversations and learn from them, and also negotiate with them to find some kind of common ground, because it s impossible to do 10 studies to meet the needs of 10 different payers. Sometimes you ve got to make trade-offs. I think those kinds of things can be medically led rather than commercially led. I think medical could drive that generating that in-depth knowledge very early in the process, three to four years before launch on asking, apart from what regulators might want, what would payers want that is additional or different? Then, what can we do about it? Where we can t do much about it for other reasons, then play a role in making decisions on trade-offs. Right now a lot of that is commercially led. Medical Director By way of an example, one expert highlights the UK s National Institute of Health and Care Excellence (NICE) and the Federal Joint Committee (G-BA) in Germany as having influential roles over what happens in these pharmaceutical markets. He suggests that an efficient medical affairs organisation should be having discussions with these agencies as early as Phase II, when the clinical development team is thinking about the design of the Phase III programme and the trial protocols. These discussions are particularly important in the current climate, when companies are focusing on specialised, innovative therapies. They ll be looking at what unmet medical need this new medicine addresses. Is the disease serious or life threatening? Are there therapeutic options available for the disease in question? What are the current standards of care? Is there a market potential and a clinical need for this medicine? Is the risk-benefit favourable versus existing options? How will the organisation present the value proposition of this new medicine? Certainly the efficacy, safety and quality are the three historical hurdles. But it s more about, where is the value of this new medicine in terms of cost effectiveness and other health related outcomes? Former Vice President, Head of Medical Affairs Europe and Canada 22

27 What medical affairs should be doing, he suggests, is having appropriate conversations with experts in the countries, particularly through the medical affairs networks, which then enable them to provide input into the clinical programme in collaboration with the R&D team. Medical advisors or MSLs should understand the medical strategy of any assets that are in development (and those being commercialised) and through training should understand the disease and treatment area of the disease area of interest. Medical affairs, through MSLs or medical advisers, should be having appropriate conversations in the environment so they are able to provide input to the global or regional medical affairs or clinical team on what design features or end-points these clinical protocols should include, and what feedback they re getting from the significant stakeholders. In addition, there should be understanding either from the conversations or working closely with market access colleagues as to what the disease treatment paradigms are, what the burden of illness is and what are the drivers for determining cost effectiveness or deriving the value of the medicines from the research. Local markets will consider these depending on particular HTA requirements. This means, are protocols going to include any patient reported outcomes? How are they going to apply the standard of care or consider the most appropriate comparator, for example? What kind of endpoints do you need to think about when they look at cost utilisation or other quality of life indicators? These kind of conversations should be happening very early on to incorporate them into the Phase III programme, so when companies file for registration, the dossier includes clinical as well as other health outcomes data to the various authorities, be they regulatory or health technology assessment related authorities. Former Vice President, Head of Medical Affairs Europe and Canada 23

28 Planning the medical affairs study programme Medical affairs studies add value to the R&D studies The pivotal trials remain the domain of the R&D teams and are geared towards the data required for registration. Medical affairs teams become involved in ascertaining which additional studies are required to generate the evidence required by payers and HCPs in order to gain market access. After the R&D trials have been agreed upon and decided, medical is designing and executing studies that provide additional evidence. Typically, the R&D-run trials should result in a registration package. Medical trials should provide additional value, beyond the endpoints required by health authorities. Global Medical Affairs Director R&D are very much involved with the development group with the Phase II and Phase III. When you have Phase IIIb and certainly Phase IV, it tends to be more the medical affairs team that then take the lead. Global Head of R&D and Medical Affairs There are two levels of evidence. The regulators have a very clear process on what they would like to see in clinical studies to give a licence for the medicine to be marketed. That s quite finite and the R&D organisation tends to work towards that. But then there is another piece which is a little more ambiguous, which is what payers might want to pay for the medicine. In western markets, the payers and the funding mechanisms are fairly predictbale, but if you look at emerging markets it s not as clear and very often the patients pay for it [the medicinal product] out of pocket. Medical Director Understanding the data gaps The first step for the medical affairs teams involves gaining insights into unmet need and ascertaining where the gaps are in the data. This involves talking to external stakeholders, synthesising existing evidence and bringing those insights back into the company early enough in the development process to be able to generate the required data. 24

29 The first piece is gathering all the insights which you need to inform your strategy, to inform your clinical development. What s the unmet need at the patient level or at the physician level, at the payer/regulatory level? What are the gaps based on the standard of care? That comes through external dialogue with the medical and payer community. Often the R&D organisation is looking for deep insight into those needs, many of which might be around what additional evidence these customers require. Medical has a key role to play engaging with external payers or physicians to understand and be able to alert organisations well in advance so that there is sufficient time to generate that data. Then you have the right body of evidence by the time you launch a medicine, and not just the parts which satisfy the regulators. Such insight-generating activities which positively influence evidence planning and design need to be driven and led by medical rather than by commercial and are very much part of launch planning. Medical Director Discussions to understand the market environment and market access requirements for innovative medicines should be ongoing much earlier in the development process to enable insights to be incorporated into the clinical trial programme. There should be early discussions to understand the environment, the clinical landscape and what needs to be done when it comes to optimising market access. That often doesn t happen until very late, particularly when one looks at what the access needs are across regions or in particular countries, where HTAs [health technology assessments] are having a greater role in the access to medicines, particularly to innovative medicines. Former Vice President, Head of Medical Affairs Europe and Canada Deciding on a study programme Once the gaps in the data have been identified, decisions are made on what type of studies should be undertaken to best fill those gaps. The team also needs to be aware of the competition and where the product candidate is likely to fit into the treatment armamentarium, assuming it eventually gains market access. Studies run by the medical affairs team prior to launch include formal randomised Phase IIIb clinical trials, which are designed to provide additional data to the Phase IIIa R&D studies. Medical affairs studies can form part of a global strategy for a product candidate, or they can be specific to the needs of a particular country market. 25

30 Then, having understood that [the gaps in the data], how do you decide what you are going to invest in? What kinds of studies do you need to now do which will help address the unmet need of the customers with the kind of data they [HTAs and payers] are looking for from medicine? You must be one step ahead of the competition as well. Very often you might need to conduct some head-to-head studies versus standard of care or other drugs which might be in the pipeline of competitor companies. Evidence generation is always a challenge, because there is no perfect study. Very often it s difficult to do head-to-head studies, to understand what the competitors are doing and how the standard of care is evolving. With the dataset that you are generating, where is your medicine going to fit in the treatment paradigm assuming it gets access? Medical Director It [the medical affairs study programme] can be part of global strategy. For some disease areas, health authorities may demand a certain set of endpoints related to efficacy and safety in comparison with a standard of care. But economically it might be more valuable to do a different type of study with a different endpoint and against a different competitor, because that s mostly used across the world. The type of evidence coming from a country could be your local market has different products that they use in a certain disease area [so] for competitiveness you want to have those other products [as comparators]. I think an ideal medical affairs organisation is where the countries get input into the global strategy and then global finds the most common denominator to design their studies. Global Medical Affairs Director Being prepared for Phase IIIb and Phase IV The protocol for the Phase IIIb studies can be designed during the Phase IIIa programme so that, in an ideal world, the Phase IIIb programme is ready at the time the results of the Phase IIIa trials become available. Typically you can design your Phase IIIb protocol after you ve designed your Phase IIIa studies. In the timeline you negotiate with health authorities based on your Phase II data and then you agree on the Phase III programme. Once that is crystallised, the 26

31 commercial teams can look at, What data do we get from this package if we have registration? What is the ideal need to market the product? What type of data package do we need and by when do we need this? That is your Phase IIIb plan. Global Medical Affairs Director If the Phase III studies are positive, you would then be able to trigger the start of your Phase IIIb programme and put the resources required towards that. Executive Medical Director In the pre-launch period following regulatory submission, a vice president of medical affairs notes, the intensity of the involvement of medical affairs in preparation for the lifecycle of the medicine, assuming it is approved, increases. This includes engaging in Phase IIIb studies and considering the Phase IV studies that may be undertaken after launch. Medical affairs is intensely involved, talking to the investigators that have worked on the clinical trials and understanding some of the experience in the clinical trials so that we can build upon this in our other lifecycle, Phase IIIb and Phase IV programmes. For example, we may engage in a Phase IIIb study. We may start thinking about a Phase IV study that may help in building the clinical experience database, because the Phase III trials involved too little of a certain population, and we may want to look at different populations that may be affected with the same disease. So we re very intensely preparing for the rest of the lifecycle of that medicine, and filling that gap in between the submission and the approval is critical. It s critical because we don t know yet the label that we re going to get but we have an overview of what data we re pursuing and what the medicine will look like. Vice President of Medical Affairs, US You might be thinking about follow-on studies Phase IV studies. So when the drug is in the market, what studies could you do which will then help to establish its place in the market? Where you have approval, you re trying to build the value and awareness of how to use that drug properly. You might be involved with the Phase IIIb trials. So you have your primary indication but you then may want to start to look at other areas in which this particular drug might be useful? Global Head of R&D and Medical Affairs 27

32 Planning local studies As product launch approaches, local country-level studies will begin to be considered in addition to the global study programme. The global medical affairs team will be involved, providing information to the country teams about the types of studies that would be likely to fill gaps in the data and therefore be useful to the company, as well as in reviewing study proposals. In addition to developing our global Phase IIIb/IV plans, one of the things we do as we re getting closer to launch is work with the countries and the regions to develop their local Phase IV plans, because they may have very specific local country needs or regional needs for studies. All of those plans have to be reviewed and approved at the global level. Each of those studies ultimately are reviewed and approved by global medical affairs. Local studies have to be approved at the country level, then at the regional level and then they go to a global medical affairs study team for approval. The global medical affairs team review is for scientific quality, to make sure that the study makes sense, that they re not just doing a local study or an IIT [investigator-initiated trial] because they want to gain favour with their local KOL [and that] there s scientific value to the study. Executive Medical Director As one medical affairs lead notes, it will not be feasible to satisfy all the requests for future studies, so a list of priorities will help to identify which studies should be funded. How do you decide this is a study you can spend money on? It s having a priority list when it comes to a recommendation for future studies, either externally or internally, deciding that you need to fill the gap [in evidence] because of a country s requirements. Global Medical Affairs Lead Early real-world studies In addition to the formal Phase IIIb/IV studies, medical affairs may be involved with some real-world studies in order to generate additional evidence outside the Phase IIIa programme. Our experts note that real-world studies are useful because they are conducted on patients that would not necessarily have met the strict entry criteria of the Phase IIIa clinical studies; for example, they may have comorbidities, be in a different age group, be over or under weight, or have any number of other complications. 28

33 These are screened out of your Phase III study. But when you come to use the drug in routine clinical practice, you give it to these patients and these are the patients who are likely to give you most trouble. So there s a lot to be said for a real-world study where you say, Okay, it performed very well in the Phase III trial with everybody in ideal circumstances. How does it perform under different circumstances found in real clinical practice? To answer this question, the real-world study is a valid approach to consider. Global Head of R&D and Medical Affairs Conducting real-world studies can provide useful information, even if you are not able to directly study your own product candidate prior to regulatory approval. Additional studies in the real world can provide insight into the currently approved therapies and how the novel product candidate may offer some improvements for patients, perhaps in terms of fewer side-effects, for example, or a better fit to the logistics of administration. It s difficult to do a real-world study on the drug itself prior to its being approved and registered for use. However, what you can do is to study that particular therapeutic area so you understand what the different types of patients are in that therapy area and how each of them behaves with current treatments. So you can get some insights into how the drug is likely to perform because you can build that up from existing practice. For example, supposing you find that your particular treatment has less of a particular side effect than your comparator drug. What you will then want to know is, how much of a problem is that side effect in the real world? So you may study that general population treated with the product that s already on the market and establish just how much of an impact that side effect nausea for example has on routine clinical care at present. Because you can then argue that nausea s a big issue when you re using this particular drug and the product we ve got now actually has no nausea therefore we d anticipate that this will have added benefit compared to the original drug. You may need to do some further follow-on studies to demonstrate that, but at least you ve got some circumstantial evidence that there may be a genuine benefit in what this product is going to be offering to the patients. Global Head of R&D and Medical Affairs 29

34 Developing a communications strategy Key insights Communication will involve multiple channels, both internally and externally The plan will consider the projected timeline of when data are expected from clinical trials Medical affairs should identify the appropriate outlets for communicating trial data Medical affairs should ensure a consistent and compliant communications strategy across the company Medical affairs has a significant role in developing the value dossier for payers Source: FirstWord research and analysis A comprehensive communications strategy with internal teams and external stakeholders evolves over time. Once data are available, medical affairs has a key role in identifying the communication outlets. Our experts note that there are multiple types of communication, both internal and external, and it is likely that a multi-channel approach will be utilised. Channels include peer-reviewed publications, conferences, seminars, symposia, continuous medical education and individual conversations with stakeholders. Communication encompasses communicating internally [and] externally about the drug, undertaking congress activities, preparing for medical information inquiries [and] preparing for requests from investigators to do more studies. Global Medical Affairs Lead Internally, according to a global medical affairs lead, companies need to make their own teams aware of the data. This is likely to include medical affairs, market access and commercial colleagues at country level, and companies need to have the 30

35 infrastructure in place to be able to share the relevant information. Most companies have it, he notes, and this may be some form of focused medical education centres at a global level. External communication prior to approval is limited, and direct communication with HCPs about the drug increases after launch. That s when you can really clearly communicate things externally. Before you have a licence, your communication is not really that much on data, because you don t really have the data. All you can do is answer questions and try understand what is required, and start to build that plan of communication [from medical affairs] that is more on the new evidence. Global Medical Affairs Lead Once you ve done the studies, how are you going to interpret the results? How are you going to communicate them? Communication could be at different levels. It could be in conferences, seminars, in symposia or some medical information setup, or even through individual dialogue with physicians. Then there are multiple layers of activities under these, but at a much higher level you ve got medics being the experts, having all the insights, understanding unmet need, finding solutions through evidence generation and then communicating that. Medical Director Medical needs to identify what the communication outlets are. Typically, you have your GCP [good clinical practice] requirement of having your studies published, but just a simple publication is not enough. You also need to identify congresses and societies [and] you should think about education. It can be done through accredited continuous medical education, pharma company stand-alone meetings, symposia there are many communication activities that you could choose from. I think it s the medical affairs organisation taking the lead in, What are the educational and communication efforts about a product? How do we want to talk about the product? Those types of things need to come from the medical organisation. Global Medical Affairs Director The timing of different types of communication is also likely to form part of the strategic plan. A medical communications expert notes that while the medical affairs team would support the publication of information when the compounds are preclinical, the more customer-facing types of communication typically begin from 31

36 Phase II. Once a decision has been made to fully develop the products, the team may organise medical education and communication activities regarding disease awareness and unmet needs or physician and patient education. Direct interactions between the medical communications team and HCPs in terms of medical education, meetings or advisory boards would begin from Phase II. The strategic communications plan evolves over time. For example, if we start working on a product in Phase II, the communications plan would involve several aspects [and] various stakeholders. You identify your key target audience and then you have other audiences. So you really plan when the data s going to be communicated and to whom. Franchise Head, Medical Communications Our experts note that the medical affairs team will meet with the thought leaders (or KOLs) who form part of a steering committee and will advise on the communication plans. The plan will consider the projected timeline of when data are expected from clinical trials and ascertain the appropriate outlets for communicating these data. Based upon when studies are completing, when certain data become available, when your anticipated timelines are for your pivotal studies, you map that out in data availability. The publication of clinical data should be done as these become available. Typically, the first product dissemination of data, especially if it s major data, is at a major congress for whatever the therapy area may be, followed shortly or coinciding with a publication in a peer-reviewed medical journal. You also have to plan what forms of dissemination you re going to have. You need to take a multi-channel approach. So maybe earlier on it would be more publications heavy and then you may have online medical education programmes or similar things. Depending upon what the product is and what the market is for a therapy area in terms of the competitive environment, part of the communications plan could be raising disease state awareness [and] unmet needs. Also in terms of competition, being able to position our product versus the competitor product. Those are all things that are carefully considered when a product s in development. Franchise Head, Medical Communications External communication plans would include such things as publications [and] being involved with presentations at international scientific meetings. The medical affairs group probably have more to do with communication than R&D in the later 32

37 stages. But in the earlier stages, particularly with the pivotal Phase III trials and Phase II trials, there is probably more coming from the R&D group than the medical affairs group. It should be a joint activity but it tends to be that Phase II pretty much is run by R&D, Phase III is a combination of the two, Phase IV it s medical affairs, as a general principle. Global Head of R&D and Medical Affairs One of our experts highlights the medical affairs role in ensuring a consistent communications strategy across the company. All the presentations on our medicines or clinical development programmes are basically vetted by us, so that whatever is presented is consistent with the medical platform and strategy. For example, if we decide that hospitalisation is a key focus of the medical strategy because our data supports a reduction in hospitalisation, then that medical strategy is consistent with the commercial and access strategy saying that hospitalisation is a key feature a hard end point of our effectiveness. So everybody s got to work in harmony towards hospitalisation even on the access side, hospitalisation will be presented as a key component of that medicine and the managed market MSL would be expected to focus on hospitalisation. All presentations even those to PBMs [pharmacy benefit managers], third party payers and others are vetted by the medical affairs team to make sure it s consistent with the medical platform and strategy. Vice President of Medical Affairs, US Our experts highlight that the medical affairs team would also have a significant input into the value dossier that is provided to payers to support market access and reimbursement. One of the most important things that goes to payers is the value dossier. It s really a compendium of all the clinical data for your product and isn t necessarily something that gets published, but this is a document that is critical in regard to market access and reimbursement for your product. The medical communications group would be involved in various aspects of the preparation of the document. Franchise Head, Medical Communications 33

38 Specific materials include the market access dossier, so that you identify the value of the new product and are able to communicate that in a meaningful way to the payers and others of interest. [Also] it ll be looking at things like a monograph, so you have a detailed overview of all of the studies and knowledge about that particular product, which is helpful when you re dealing with pharmacies, so that they understand what the drug s about. Global Head of R&D and Medical Affairs Strategic partnerships and customer engagement Key insights KOL mapping may be led by medical affairs or a cross-functional team with R&D KOLs may be ranked according to global, regional, country and city level Medical affairs advisory boards may stay in place through Phase III and launch MSLs play an important role in identifying local needs Being experts in their field enables MSLs to have credible scientific exchanges with stakeholders Source: FirstWord research and analysis KOL identification and mapping The role of medical affairs in KOL identification and mapping is likely to vary between companies. In some companies, this activity is led by medical affairs. In others, it may be a collaboration between medical affairs and R&D as part of a cross-functional team. The level of involvement of medical affairs in this team is likely to change over time, with R&D taking more of a leading role during the early phases of development. 34

39 In large pharma companies, KOL identification may be a two-stage process, whereby the global medical affairs team will identify the leading experts globally and regional or country-based teams will conduct a similar exercise at a local level. In the early stages it would be my organisation [the global medical affairs team] doing the KOL mapping, to understand what is the top tier in a particular area, the second tier, who they influence, where they sit, which are the centres of excellence. Then later on, after the Phase III trial results are available or just before, we might get the MSLs involved to do basically the same exercise but at a different scale. Vice President of Medical Affairs, US The discovery team has already identified the leading experts to talk to them at a very early stage in the product. When you get into the clinical development stages, you ll have a principle investigator for the study. So that will usually be chosen by the R&D people [but] usually you d involved medical affairs in that choice. As things move on, you start to get more and more engagement with the external environment, directed by the medical affairs group rather than the development group. But it s not a sudden one drops, another one takes over. It s a gradual transition where at the beginning, it s the development group leading the integration of the external community. But as time goes by, the medical affairs team gradually takes on that role. Global Head of R&D and Medical Affairs In a large global pharma company, KOL mapping exercises may involve a ranking system, whereby the leading experts are ranked at global, regional, country or city levels. We do what we call thought leader or KOL mapping exercises where we have hard metrics in terms of their influence, [considering] what boards they sit on, what committees, how much they have published, who they are advisors for. We can rank them globally [or] by region even down to country or city level. A global medical affairs team may work with the first 50 for example. But say you re a country Italy for example. You re going to rank your top 50 Italian thought leaders and you re going to work with those. So depending on where you work and the organisation, rankings are relative to what level of thought leaders you work with. Franchise Head, Medical Communications 35

40 Our experts note that the different expertise of KOLs is also considered and that its relevance is likely to vary according to the phase of product development and the specific reason for engagement. If you execute your development programme from Phase I to Phase III, you need different types of people to work with. Earlier on you may want to use the top experts, not the highest recruiters, but maybe people with access to a special type of patient. Then if you go to Phase III, [there are] typically more patients, so you want to go to a different group of investigators. What medical is doing is identifying who [the company] ideally wants to work with and for what reason. Some can be excellent investigators or advisers. Others might be excellent authors, because they ve done many studies with the company, or they are of high scientific calibre. Understanding who the company engages with and for what reason is the remit of medical affairs. There are changes in different stages of development. Medical affairs is typically not involved much in Phase I, because these are very early studies that have a high chance of failure. But starting from Phase II, especially the IIb Phase, I think medical affairs should be at the table. Then understanding who to work with is very important. I think for countries it would also affect their resource planning. So if you have a country with a certain population of medical experts that you need to work with, that determines how large your MSL team is, for example. Medical provides expertise and credibility Global Medical Affairs Director Medical affairs is emerging as the face of the pharmaceutical industry as companies put more resources into medical affairs and MSL engagement with external stakeholders. 13 Medical affairs teams are increasingly required to provide complex and comprehensive information to a wide range of external stakeholders, including medical practitioners, scientific researchers, patient advocates and government authorities. 14 Being an expert in their field enables an MSL or other medical affairs team member to have a credible scientific exchange with these stakeholders. You could look at it holistically, using evidence as a platform. Medics are there in the companies for their insights, knowledge, expertise, and credibility, which 13 FirstWord (October 2015). Tomorrow s MSLs. Retrieved from 14 Morgon, P. (September 2014). Medical Affairs: From Info Gatekeeper to Value Driver. Retrieved from 36

41 is unique to them. It s really about building that expertise, having that knowledge, being an expert in your field. You re leading all the external engagement, so whether you re talking to payers or physicians outside the company it s the medical credibility which helps build trust. Most external engagements tend to be medically led, whether it s advisory boards or talking to HCPs about potential studies and other projects they might be involved with. Medical Director The majority of our external stakeholders would be doctors and specialists in the field. However, we may also talk to other healthcare professionals that are associated with the patient management for this particular disease, and that could include the pharmacists, nurses or allied healthcare professionals. The patient groups are new stakeholders. The patient consultation is becoming the next blueprint for successful drug development. I think as we understand more of the features of the medicines we re developing we will involve patient groups and associations. It also depends which kind of disease area you re focusing on. In orphan and rare disease areas it s absolutely essential that the entire development be conducted with and in partnership with patients and patient associations. That s becoming more of a norm, where the patients are involved even in the design of the Phase III studies. Therefore, as we move along the continuum in development you continue to engage patient associations and organisations. Patient organisations are not necessarily organisations that medical affairs would reach out to in terms of the data or the study design, but more in terms of what the important features of the medicine are that are really critical for patients, whether it s compliance, showing side effects, long or short term outcome and how patients feel, function and live. Vice President of Medical Affairs, US We work with the top thought leaders but another important thing that we do is identify what we consider the next generation of thought leaders. They re commonly referred to as up and comers, or rising stars who are younger, not so established. We work with a number that we put into that category as well. Probably 80 to 90 percent are physicians, but there s a select few nurses who we consider global thought leaders for the nurse community. We work with thought leaders who are experts in compensation market access. Franchise Head, Medical Communications 37

42 Advisory boards Advisory boards provide companies with valuable expert insights on various topics, from general questions such as whether the company should pursue a particular therapy area to more specific questions related to the clinical development programme or the competitive market for a new product candidate. A recent study highlighted that an advisory board can be a valuable asset for the provision of non-binding but informed guidance to pharma companies on various aspects of their business. 15 The main purpose of the advisory board is to benefit the company. I think a lot of external experts very much appreciate being involved in these activities because it s quite an exciting for them to engage with pivotal research at an early stage. Medical affairs normally help organise advisory boards throughout development the earlier you start the better but, the purpose of the advisory boards would change over time. Initially advisory boards would be trying to look at the unmet need, in the early discovery stage. Then your advisory boards in Phase II and Phase III would be more focused on, How do you design the study? Then when you get into the end of Phase III, you d be then looking at, How do you communicate the results to external bodies and external experts? Global Head of R&D and Medical Affairs Experts interviewed for this report note that advisory boards are likely to be run by the R&D department in the early phases of development, while the medical affairs team typically runs advisory boards from the end of Phase II onwards. For example, during the development of a particular drug, experts may be recruited for an advisory board that would provide input through Phase III development and remain in place right through to product launch. Medical affairs advisory boards typically happen at end of Phase II, beginning of Phase III. Development has advisory boards from preclinical, proof of concept. In general, the purpose of an advisory board is to have specific questions answered. It could be general advice on whether or not we should pursue this therapy area. We present the data and ask for expert opinion. An advisory can 15 Best Practices LLC (May 2015). New Study Presents Insights to Advisory Board Effectiveness in the Pharmaceutical Sector. Retrieved from 38

43 be held to give advice on how to design a development programme or a study protocol. It could be more commercially focused, in terms of here s our data, how do we compare versus our competitors? Where do you see our strengths, our weaknesses? You can have an advisory board for a number of reasons. Franchise Head, Medical Communications We ve tried to have a model in place where we have a global advisory board that will help us during Phase III, then stay in place through launch and through the early lifecycle of the drug, so that they are involved right from the beginning all the way through to the success of the product. The people are usually chosen in a collaboration between medical and R&D, and also with input from our key regions and countries. For a given therapeutic area, those are the very top global thought leaders, but if there s a reason to have specific people in specific countries, we will usually seek input from the various regions and countries around who we might want in those regions. Executive Medical Director Once the [clinical trial] data are available, the advisory board can discuss the data and where we go from there, what other disease-related activities might be of interest [and] what other things we need to clarify. We bring the advisors together to help us answer these questions. Vice President of Medical Affairs, US Advisory boards can also be held with payers and health technology assessment (HTA) agencies in order to ascertain varying regional and local requirements. We have interactions with multiple payers. The payer environment, like the regulatory environment, varies very much by region and even by country. So for example, for regulatory purposes, R&D has meetings with the main regulatory body. In Europe, [this is the] EMA, but the EMA doesn t control pricing and reimbursement in all of its countries. So you have to meet with the payers or the health technology assessors within each country. We won t necessarily meet with every country but we may have a payer advisory board and then we also may have individual consultations with specific countries and payers to understand their needs. So it s very common for us to have a meeting with NICE to get advice from NICE on health technology, 39

44 etc. It would be extremely common for us to meet with the GBA the reimbursement body in Germany because they have very specific requirements for how they evaluate new products. In addition to thought leader meetings, we ll often have one or often more payer advisory boards or small group meetings to share with them, if not product-specific information, the potential product profile for a product we re developing, and what would be seen as value added. Sometimes those are done with multiple payers around one table and sometimes they re done with just one or two. So in the US, you might do it [run an advisory board] with just a couple of the very large payers. Whereas you might do it with a group of payers from varying countries. The model depends on the specific need. The payer advisory boards formally report into the commercial organisation but it would be a collaborative, cross-functional effort with patient access and medical affairs. MSLs can help to identify local needs Executive Medical Director Working within compliance restrictions, MSLs can engage with experts locally and help to identify clinical and patient unmet medical needs. This can help to build the company s internal knowledge base and expertise, and thus help to build on the medical strategy in a particular disease area. MSLs are relatively restricted, because during development the product is still off label. The role of the MSL pre-launch would be mainly to identify the needs from experts, such as What data do they need? How are patients being treated? This helps to build the internal knowledge base on a certain disease area. I think the customers typical MSLs are engaging with might already have a clue of what type of product the company is developing. So if they have any questions, the MSL can already answer those unsolicited requests. If the expert has a keen interest in this, because he or she thought about this a couple of years ago, then the MSL might be able to connect that expert to activities for the company, [for example] as an adviser or an investigator. Global Medical Affairs Director 40

45 MSLs can be extremely valuable for organisations, especially when it comes to specialty areas; they can have appropriate conversations about strategic areas of interest in disease and research including pipeline discussions, and can through their conversations with them [HCPs] give some medical insights on the key and most important areas of interest from their particular markets. But they can also work with clinical research, medical affairs and market access locally in those countries. In Germany and the UK particularly, you often find many companies have strong representation from market access. Working alongside these internal and external stakeholders, MSLs can understand what kind of data needs should be acquired in Phase III to enable reimbursement approval (through data generation for HTA submissions), as well as regulatory approval, in the countries. It s really trying to facilitate access to medicines post regulatory approval that is going to be critical, and medical affairs should play an increasing key role in that. Former Vice President, Head of Medical Affairs Europe and Canada The knowledge that MSLs bring back to the company from external experts can help the local medical affairs team to make decisions about whether to participate in a certain clinical trials programme. While this information is used locally, however, it may not always be taken back to the global medical affairs team, unless this has been specifically requested. If they [KOLs] say, In this country we do not see this patient type, or recruitment for a specific trial is going to be extremely difficult, then the country might decide to prioritise some studies over others. These insights are definitely shared on local level [but] not all knowledge goes to global, unless global is asking for these insights. If, for example, you go through strategic planning, you may want to know what is happening in a certain country and then the questions will be asked, so ultimately they will roll up into global. Global Medical Affairs Director 41

46 Medical affairs as the bridge between R&D and commercial Key insights Medical affairs is the interface between R&D and commercial Early involvement can facilitate the transition between development and launch Launch planning is commercially driven but medical plays a leading role in some aspects Medical is uniquely positioned to look at the bigger picture beyond registration and sales figures The team can make the company aware of additional evidence requirements for market access Source: FirstWord research and analysis Operationally, the medical affairs function can be seen as a vital link between the R&D team and the commercial team. Our experts note that medical affairs often plays a supporting role to both the development and commercial teams, but ideally should contribute to both. The view that medical affairs should be full partners and be seen as an internal bridge between R&D and commercial is echoed in a recent white paper. 16 The early involvement of medical affairs can enable the transition of a product from development to commercial launch. As our experts highlight, the medical affairs team plays a central role in understanding and communicating the clinical data and the science around the mechanism of action and the disease. The team is also likely to have an advantage over their commercial colleagues in terms of market knowledge, as they have already been working in the field for some time. 16 Cegedim (February 2014). Medical Affairs White Paper. Retrieved from 42