RWE from pre-clinical to launch. RWE from pre-clinical to launch. Standard of care Unmet needs. Disease burden Budget impact.

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1 Real Real World World Data Data Across Across the the Product Product Lifecycle: Lifecycle: RWE from pre-clinical to launch RWE from pre-clinical to launch Standard of care Unmet needs Disease burden Budget impact Resource utilisation Patient pathway SVMPharma Ltd Landmark House Station Road Hook, Hampshire RG27 9HA

2 Real World Data across the product lifecycle: Generating RWE Patient level data access in the NHS: Generating positive outcomes from through pre-clinical Real World to launch Evidence Real world evidence (RWE) is becoming commonplace in support of HTA submissions and resubmissions in order to demonstrate efficacy and value for new drugs. Cost and time efficient evidence gathering across the product lifecycle has never been more important. Real world evidence opportunities across all phases of drug development are increasing through pre and post approval studies, providing opportunities for savings and efficiencies through strategic planning, data collection, and re-purposing of study materials generated pre-launch as well as postlaunch. We examine the RWE opportunities across the development lifecycle, and assess how regardless of the phase, real world data is generated and available to drive RWE insights. RWE: more than a post-launch tool Real world evidence collection currently primarily occurs at a level to indicate cost effectiveness and efficacy in real world patient populations. Being generated in cohorts of clinical patients, this data is therefore primarily captured retrospectively, post-launch to support HTA submissions. However Real world data is available from across the product lifecycle, and may be used to generate RWE to provide insights and answer questions that inform future research and development prospects. Because data types, sources and availability are more variable, proper study design is especially important in RWE. The choice of study depends on why the evidence is wanted, for example, to study long-term safety, study effectiveness in multiple care settings, conduct economic analyses, investigate unmet needs and interventional pathways. The research question to be answered will dictate the appropriate study type and origin of RWD. [2] 2

3 Evidence gathering across the product lifecycle Six areas throughout the product lifecycle that generate revenue growth and cost or productivity improvements: Optimising commercial spend effectiveness Brand growth via RWE-enabled marketing, improved promotion via physician-patient segments, and better forecasting via disease progression models Demonstrating ongoing safety and value Supports continued access and reimbursement through responding to queries and publications, as well avoiding label changes and protecting brand from generic entry Improving launch planning and tracking Accurate patient pool segmentation and rapid adjustment of messaging/resource allocation at launch Better initial pricing and market access Accelerates market access submissions, payment by use/indication for more effective price negotiations, and conditional access via coverage with evidence development Increasing productivity and cost savings Reduces duplicate primary research spending and doubles impact factor of publications Improving clinical development Improves trial enrolment, reduction in strategic design flaws, and better product profile design. Pre launch, RWE enables us to understand current treatment and the factors influencing their effectiveness. In response to this, pharmaceutical companies can better demonstrate where their brand has advantages over current management choices in areas such as impact on disease burden, standard of care, budget impact, unmet needs improved adherence, increased quality of life, reduced healthcare resource use, and patient preference. Typically companies have focused RWE on demonstrating value, market access and clinical development. The approach is shifting toward a systematic strategy across all above areas, bringing about a change in RWE value. By connecting different datasets, companies can deepen their understanding of patient journeys and market influences. Clinical trials Health economics and outcomes research has become a more integral part of traditional research and development, impacting clinical trial design and the management of economic endpoints within these trials. With major global shifts in healthcare services to control costs, a growing number of Phase III trials now include economic endpoints and analyses, alongside the clinical outcome measures, to demonstrate the cost-efficacy of new products. [1] RWE is regarded as having the ability to improve clinical development via enabling translational research, better understanding of drug pathways, higher value population and product profiles, and trial simulation and recruitment. Suggestions include trial simulation using RWE to ensure that trials likely to fail are not initiated (over 30% of trials fail for strategic rather than operational reasons). 3

4 Named patient programmes Despite only being available on limited access, it is a myth that data cannot be captured during a named patient program. There is no legislation prohibiting data capture, although some territories have specific guidance over what data can be provided. In fact, most managed access programs contain clinical data capture in some form as standard. Data capture in these incidents are incidental and does not mean the program is intended for research; patient cohort treatment remains priority. In a managed access program observational data is provided at the physician s and patient s discretion meaning there is no requirement for additional protocol or ethics review to collect data than already required for a managed access program. The intended use of data will dictate the context in which the data should be collected and may affect the mechanism used. Managed access programs provide opportunities for data capture, just the same as post-launch, as long as collection doesn t impact the patient or overburden the physician. Pharmaceutical companies are embedding named patient programs within the timeline of product development, and these largely untapped opportunities should be leveraged where possible to generate ancillary RWD that may inform future development prospects or support RWE. EU initiatives to increase use of RWE There are already many national and EU initiatives ongoing to strengthen RWE. Through good coordination and a cross-stakeholder collaborative approach we can address the challenges and realise the full potential of RWE in supporting product development, monitoring and decision-making at all stages of the product lifecycle. EU level initiatives to increase the utility of RWE include: Initiatives on patient registries (such as PARENT Joint Action, ENCR - European Network of Cancer Registries, and the EMA Initiative on Patient Registries) Initiatives on electronic health records (such as EH4CR, EMIF, EU-ADR Alliance, RD- Connect, epsos, EuroRec) Initiatives aimed at establishing methods and platforms to enable and facilitate data access, analysis and collaboration (such as IMI GetREAL, IMI PROTECT, IMI ADAPT SMART, ENCePP; the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance Initiatives on HTA (EUnetHTA JA3 aims to conduct pilots on post-launch evidence generation and to develop a tool to support permanent collaboration on post-launch evidence Approaches aimed at the use of social media Through better data sharing initiatives such as these, RWE is better placed to support access to novel products by providing information across the life cycle of a medicine from development through to HTA. For example RWE could support product development by providing information on the natural history of the disease and the unmet need, an understanding of resource utilisation and current standards of care, patient recruitment and potentially differential benefitrisk profiles in targeted subpopulations and better forecasting via disease progression models that would all benefit pre-launch applications. 4

5 RWE Preclinical Case study Recently a company who we will term X was looking to understand which molecule entities it wanted to carry forward to preclinical studies within disease areas it had no historical knowledge. Traditionally market insights would have been gained through a classical approach with the use of disease registries, market research and Key Opinion Leader insights. RWE through patient chart reviews is a new approach to gaining insight into preclinical studies and scoping. This example of patient chart reviews involving 25 patients provided key insights into: Patient clinical pathway from diagnosis to death Patient treatment and diagnostic pathway use of drug interventions and its outcomes Market sizing and potential Real world evidence opportunities across all phases of drug development are increasing through pre and post approval studies, meaning opportunities for savings and efficiencies can be realised through strategic planning, data collection, and re-purposing of study materials generated pre-launch as well as post-launch. SVMPharma is a RWE agency with experience of working on successful HTA resubmissions for NICE and SMC that can help gather the right RWE data and ease the RWE process to ensure your best chance of gaining positive HTA submissions. SVMPharma is an innovative strategic consultancy, specialising in Real World Evidence (RWE) for the pharmaceutical industry. SVMPharma generates RWE within UK and Europe through bespoke online Real World Treatment Evaluators, leading to successful health technology appraisal (HTA) submissions. Clinical trial programmes do not reflect real-world clinical practice and outcomes, RWE supplements and enhances clinical datasets. SVMPharma s specialist teams focus on delivering the outcomes that matter to your brand. SVMPharma also provides Global RWE, Patient Real World Outcomes, and Big Data Analytics. To find out more call +44 (0) Brooks, K. CRO Outlooks & Market Trends Available from Accessed July Page, M. J. Demonstrating effectiveness with Real World Evidence Available from Demonstrating-Effectiveness-RWE.pdf Accessed July Hughes, B., Kessler, M., & McDonell, A. Breaking new ground with RWE: How some pharmacos are poised to realize a $1 billion opportunity Available from ices%20tl/rwes_breaking_new_ground_d10.pdf 5