Patient-focused Drug Development in EU: From EPAR to Smpc?

Description: The patient perspective is increasingly sought across the product life cycle. But what role does PED currently play in regulatory, reimbursement and prescription decisions? What considerations are shaping a path to more explicit guidance?

Global regulators have placed an emphasis on developing regulatory guidance to better incorporate patient perspective in medicines development. This includes Patient-Focused Drug Development (PFDD) Guidelines in the US, new EMA reflection paper and ICH E22 work in progress. This focus is combined with the growing consideration of patient’s perspective as part of the wider EU General Pharmaceutical Legislation (GPL) revision. They all explicitly embed patient voice in medicine’s development.

Patient Experience Data (PED) are incorporated by companies as an integral part of medicines development from the early stages of clinical development. But how is PED being captured in health authority assessment reports (e.g. EPARs) and documents available to healthcare professionals and/or patients, since this is the key to ensure relevance in clinical decisions? Analysis of the labelling presence of explicit PED language in EU SmPCs and PILs shows we are still far from having patients voice actually informing prescription decisions. The goal of all patient-focused drug development is to address patients’ needs. For PED to be impactful for the patient, these data also need to be considered relevant to aspects surrounding the decision making for a medicine i.e., part of the benefit/risk assessment informing Regulator decisions or within the effectiveness review by payers and HTA.

This session will bring together Patients, Regulators, HTA/Payers and Industry to discuss EMA’s Reflection paper, current landscape of PED in EPAR and SmPC and explore questions such as:

- How can we strengthen the use of PED (beyond clinical development) to support Regulatory & access discussions?

- What role can transparency of PED in publicly available documents play to accelerate its clinical relevance?