Patient involvement in development, review and approval of drugs is an increasing focus for health authorities and other stakeholders. Patients, as true research partners, are increasingly involved in defining the research agenda to address unmet medical needs, advising on clinical trial designs and study implementation and fostering generation and review of patient-relevant evidence for regulatory decision and market access. Patients, regulators, HTA bodies, payers, and health-care providers are increasingly demanding patients’ experience and/or preference data to comprehensively ascertain the benefit-risk or value of a given medicinal product or to support treatment decisions in the increasingly complex clinical treatment options.
New guidelines are being developed to inform generation of patient relevant experience (e.g., FDA PFDD guidances), and inclusion of such evidence as part of the benefit-risk assessment (ASCO and ESMO clinical benefit models, patients involvement in scientific advices in Europe). Overall, however, there is not enough transparency on how best to involve patients in clinical research and which patient-relevant evidence could be used to inform stakeholders decision making.
This session will therefore look at the opportunities and challenges of incorporating the patient-relevant evidence into oncology drug development and approval process from the perspective of patients, health care providers, health authorities, HTA bodies, payers and industry and will address the following topics:
- Examine the different type of patient-relevant evidence for patient-focused drug development
- Exemplary models for interactions between patients’ communities, sponsors and decision-makers
- Examples of qualitative and quantitative patient-experience data and its contribution to development, review, approval and access to new drugs
The target is a multidisciplinary audience of Patient Associations, Academia Representatives, EU and US Regulatory Bodies (EMA, FDA, National Agencies), Pharmaceutical Industry, and HTAs.