The New Role of Medical Affairs in Defining and Driving Product Success

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1 The New Role of Medical Affairs in Defining and Driving Product Success Suma Ramadas, PhD, and Susan Suponcic, PhD Syneos Health Consulting 2018 Syneos Health. All rights reserved.

2 The Organizational Role of Medical Affairs in Early Product Development to Define the Holistic Evidence Strategy What Crosstalk? Pharmaceutical-related headlines today are filled with references to access and reimbursement decisions related to value-based care and value-based pricing. While regulatory approval remains necessary for marketing authorization, meeting regulatory requirements for safety and efficacy is no longer sufficient to show the value of a new therapy. Value is being defined and assessed by an ever-increasing and varied group of stakeholders across the healthcare ecosystem. Measures such as cost-effectiveness and patient-reported outcomes and quality of life now can be critical to a drug s reimbursement, uptake and utilization. Therefore, successful new product development and commercialization require that the right evidence to substantiate value is available at the right time to the right stakeholders. Given that evidence generation, and subsequently, value substantiation, is both costly and time consuming, planning should begin in early development (Phases I-IIb). Table of Contents Early Development and the Clinical Development Plan... 3 Early Development and the Clinical Development Plan The Clinical Development (and Operations) function typically guides early compound development to demonstrate safety and efficacy for regulatory approval and marketing authorization. However, structuring clinical trials to meet only regulatory requirements does not provide an adequate measure of a drug s value for the key stakeholders necessary for its adoption and utilization. In particular, double-blind placebo-controlled trials remain a regulatory standard, but do not always address the needs of reimbursement authorities, health technology assessment (HTA) authorities and prescribers seeking information about safety, efficacy and clinically meaningful and economically beneficial value relative to current and future standards of care (SoC). Although regulatory approval and marketing authorization are often viewed as the hallmark of success, in today s complex healthcare environment, substantiating value to a breadth of stakeholders is the key to ultimately getting innovative therapies to patients in need. Statistically significant evidence toward a clinically meaningful endpoint Comparative safety and efficacy. Reduced administration burden and/or complications. Improvement in relevant QoL measures. Regulators Patients HTA HCPs Payers Fill an unmet medical need regarding safety and efficacy Demonstrate a cost or social benefit Case Study... 4 The Role of Medical Affairs Today... 5 Definitions of Commonly Used Terms... 5 Crosstalk: Everyone Is Talking Evidence, but What Do They Really Mean?... 6 The Strategic Medical Foundation: Right Evidence at the Right Time... 8 Medical Messages... 8 Innovation compared to other available therapies While generating registration-enabling safety and efficacy evidence is necessary, the traditional Compound Development Plan is limited. With a focus on regulatory requirements, the plan may not adequately consider or incorporate the views of broader, highly relevant groups of external stakeholders such as physicians, payers and HTA professionals. As a consequence, the evidence generation outlined in the Compound Development Plan typically does not address these very different evidence requirements of nonregulatory external stakeholders, who all play influential roles in the treatment paradigm and resulting patient journey. Therefore, companies should recognize the potential for evidence gaps and proactively plan in early development for holistic evidence generation and value substantiation throughout the product life cycle Syneos Health. All rights reserved Syneos Health. All rights reserved. 3

3 Early Development and the Clinical Development Plan (continued) A critical first step in this effort is creating a stakeholder map of decision makers and those influencing a new product s reimbursement, uptake and utilization. The map identifies stakeholders, their relationships to one another and their evidentiary needs and substantiation standards or requirements. Depending on the disease community, this diverse group may include primary care physicians, multiple disease or treatment specialists, empowered and increasingly sophisticated patients/caregivers, advocacy groups, as well as purchasing groups and payers, among others. Pharmaceutical and device companies can identify these key stakeholders through surveying and engaging the disease community early in the drug development process. It is important to note that this engagement with nonregulatory stakeholders at such an early stage in the drug development process is restricted to scientific and nonpromotional discussions. The Role of Medical Affairs Today Historically, Medical Affairs has been and often still is viewed by pharmaceutical and device companies as a reactive support function to disseminate medical information and provide scientific expertise. However, pharmaceutical and device companies, particularly those innovating and paving their way to scientific leadership, are realizing that Medical Affairs can be a strategic function for scientific engagement early in drug development and throughout commercialization. Medical Affairs provides a powerful means of harnessing two key activities: disseminating clinical and medical data while gathering insights from the community to bring back into the organization. By using different channels that range from scientific publications and congress meetings to advisory boards and individual engagements, this two-way scientific exchange becomes the catalyst for the organization to proactively understand and incorporate unmet medical needs. As a function, Medical Affairs typically is resourced by individuals medically qualified or scientifically trained, who are fluent in the language of their professional counterparts and now key customers. As such, the Medical Affairs team is well-positioned to have meaningful conversations and scientific exchange with physicians, healthcare providers and, in unique situations, with patients. The Medical Affairs team is qualified to assess and inform medical and clinical landscapes and is able to tailor scientific discussions to the needs of the customer by distinguishing between what is relevant in the day-to-day clinical setting and what is required in a laboratory or registration-study setting. Medical Affairs can gather insights to identify clinically meaningful differentiators by engaging with the external community years ahead of launch. These insights can inform clinical strategy and a holistic evidence generation plan that will produce and enhance value substantiation. As the product progresses through its development cycle, the evidence generation priorities and opportunities of the product will evolve. At the prelaunch phase, generating registration data will be prioritized, whereas during launch, generating real world and economic evidence will become more prominent. Definitions of Commonly Used Terms Regulatory requirements: what is needed, at the minimum, for regulatory approval (registration, market/drug approval, etc.). Clinically relevant evidence that is used to satisfy the requirements of regulators (FDA, EMA, etc.). Unmet needs: The unmet medical, clinical, societal, and/or economical needs in a particular disease area. Regulators, payers, HTAs, HCPs, patients and other members of the disease community will each define unmet need differently (e.g., what is clinically meaningful versus what is economically beneficial). HTA (Health Technology Assessment): A multidisciplinary field of policy analysis. It encompasses the medical, social, ethical and economic implications of development, diffusion and use of health technology, including pharmaceuticals. Payer: any commercial, government or individual responsible for funding and/or reimbursing the cost of healthcare including pharmaceuticals, medical services, devices, or ancillary care Syneos Health. All rights reserved Syneos Health. All rights reserved. 5

4 Crosstalk: Everyone Is Talking Evidence, but What Do They Really Mean? The origin of the word evidence is from the Latin evidentia, which originates from evident, obvious to the eye or mind. However, as internal functions seek to address the evidentiary needs of external stakeholders, the variety of standards and the differentiated requirements of each is not always evident (Table 1). Table 1. Crosstalk. CLINICAL MEDICAL AFFAIRS How Evidence Is Defined Defined in terms of superiority or noninferiority to a placebo or indicated comparator; measured as a statistically significant difference Inclusion/exclusion criteria optimized for regulatory success Clinically or medically meaningful and differentiated from other therapeutics Typically defined relative to a treatment standard of care Clinical/medical benefits Key Stakeholders Regulator bodies (e.g., FDA, EMA) Healthcare providers Patients/caregivers (U.S.) Patient advocacy groups Societies IDNs While Clinical is focused on meeting the safety and efficacy requirements of regulators, Market Access is focused on demonstrating innovation and cost effectiveness to HTA bodies based on their requirements. In contrast, Medical Affairs is focused on addressing unmet needs of HCPs and patients by disseminating clinically and medically meaningful data. Outcomes Research is focused on providing to its stakeholders real world evidence of longer-term clinically meaningful or patient-relevant outcomes. It is easy to see how these internal stakeholders may talk past each other when discussing evidentiary requirements and priorities across stakeholder groups. Yet, to develop and deliver a commercially compelling and viable product, companies need to move beyond this crosstalk. Truth be told, this is no small feat, particularly if functions do not understand each other s perspectives when discussing evidence generation priorities. There are time and resource implications when each function considers evidence only from its own perspective. There is also the potential for communicating inconsistent and incompatible messages to stakeholders. Alignment with regard to evidence generation is essential for rationalizing time and resource allocation and ensuring consistent messaging to all stakeholders. Case Study The commercial division of a large pharmaceutical company wanted to focus its commercial positioning and messaging on the speed of onset of action of its product compared with its competitors. Based on external advice, the company decided to have a crossfunctional exercise to determine how to most effectively use the evidence to position the product. However, it was only after six weeks when a member of the biostatistics team understood the needs of the commercial team and indicated that the endpoint was not measured in this way. In the study, all measurements were taken every two weeks. The speed of onset occurring earlier than two weeks was not even included as an endpoint in the pivotal clinical trial (Table 2). From a regulatory perspective, the product gained approval, as it had met the necessary clinical endpoints required in the study. However, by not addressing the evidentiary needs of the commercial team, the company was unable to move ahead with its original commercial positioning and messaging strategy. This could have placed its product ahead of the major competitor and avoided the need to rethink the entire strategy. MARKET ACCESS Typically defined relative to a standard of care or existing reimbursed treatment alternative Value substantiated Payers (e.g., IDNs, MCOs, ACOs, CMS) HTA bodies (ex-u.s.) Table 2. Stakeholder Evidentiary Needs by Function. The table illustrates the evidentiary needs that were not addressed in the pivotal clinical trial for the product following a six-week crossfunctional exercise. OUTCOMES RESEARCH HE/HTA satisfaction including cost-effectiveness and budget impact Innovation Real world evidence Long-term clinical outcomes Patients/caregivers (U.S.) Payers HCPs Regulatory bodies (FDA and EMA) Function CLINICAL/ REGULATORY MARKET ACCESS Stakeholder Evidentiary Needs Fixed time points for measuring endpoints, consistent with regulatory requirements (measure at 10 days) Payer perspective: value for money and cost offsets Incremental physician visits Concomitant medications Included in Trial as Endpoint YES NO (magnitude of difference may not be substantial over duration of trial) COMMERCIAL Commercial viability of product Internal stakeholders Some of the abovementioned stakeholders FDA: Food and Drug Administration; EMA: European Medicines Agency; IDNs: Intergrated Delivery Networks; MCOs: Managed Care Organizations; ACOs: Accountable Care Organizations; CMS: Centers for Medicare & Medicaid Services MEDICAL AFFAIRS Needs of prescribing community NO COMMERCIAL Marketplace (patient and physician) gaps/unmet needs: Speed of onset of action looking at weakness or gap in competitor profile NO Syneos Health. All rights reserved Syneos Health. All rights reserved. 7

5 The Strategic Medical Foundation: Right Evidence at the Right Time Crossfunctional alignment on the common and differentiated evidence that will be meaningful to all stakeholders can help companies optimize the way they execute and deliver science and ultimately deliver and explain value. Having the right evidence at the right time can optimally position both the science and the product in the evolving value-based health ecosystem. Toward this end, Medical Affairs plays a significant role in aligning the organization and the evidence generation plan because clinical/medical benefit is the common component of evidence necessary for adoption by all stakeholders. The ability of all functions to align and agree on clinical/medical benefit and how it meets stakeholder needs is essential for moving forward. Therefore, a unifying document, which we call the Strategic Medical Foundation, can be utilized to achieve crossfunctional alignment, rationalize resources and ensure consistent messaging to all stakeholders. The Strategic Medical Foundation, which we propose should be owned and maintained by Medical Affairs, integrates and contextualizes all key clinical and medical attributes and their evidence substantiation needs. In addition, once aligned, it ensures that consistent messages about these attributes can be communicated by internal functions to the external community. Medical messages are used by pharmaceutical and device companies to contextualize evidence generated for a product and ensure all internal stakeholders within the company communicate the evidence in a consistent and substantiated manner to all relevant external stakeholders. While the word message usually brings to mind something commercial in nature, a medical message is intended to focus on the evidence and how it is consistently communicated to external stakeholders Syneos Health. All rights reserved Syneos Health. All rights reserved. 9

6 About Syneos Health Consulting Syneos Health Consulting is an industry-leading consulting firm specializing in the biopharmaceutical industry and part of Syneos Health, a 22,000-employee biopharmaceutical solutions organization designed to help the biopharmaceutical industry shorten the distance from lab to life. We provide services across a comprehensive range of key areas, including commercial strategy and planning, medical affairs, risk and program management and pricing and market access. Recognized by Forbes magazine as one of America s Best Management Consulting Firms for two years running, our industry focus and depth of functional expertise, combined with strong scientific and market knowledge, uniquely position us to tackle highly complex business and market challenges to develop actionable strategies for our clients. Contact us: Suma Ramadas, PhD Managing Director Medical Affairs Syneos Health Consulting suma.ramadas@syneoshealth.com Susan Suponcic, PhD Managing Director, Practice Area LeadPricing & Market Access Syneos Health Consulting susan.suponcic@syneoshealth.com Additional Contributors Keith Morris Lisa Arbogast Steven Gomez Corporate Headquarters 3201 Beechleaf Court Suite 600 Raleigh, NC Phone: Fax: Toll-Free: About Syneos Health Syneos Health (Nasdaq:SYNH) is the only fully integrated biopharmaceutical solutions organization. Our company, including a Contract Research Organization (CRO) and Contract Commercial Organization (CCO), is purpose-built to accelerate customer performance to address modern market realities. Created through the merger of two industry leading companies INC Research and inventiv Health we bring together more than 21,000 clinical and commercial minds with the ability to support customers in more than 110 countries. Together we share insights, use the latest technologies and apply advanced business practices to speed our customers delivery of important therapies to patients. To learn more about how we are shortening the distance from lab to life visit SyneosHealth.com Syneos Health. All rights reserved.