EFPIA Position on Transparency of Patient Evidence in Regulatory Decision Making and Product Information

The Pharmaceutical Industry is increasingly including the views and perspectives of patients in all stages of drug development. The EMA has also recognised the importance of this by including patients representatives in its committees and decision-making processes. Both Industry and Regulators are now taking this a step further by leveraging scientific methodologies that enable a more structured and representative patient input so that patient preferences and outcomes that are meaningful to patients can be systematically considered and captured during product development. A significant indicator of this is the Reflection Paper on Patient-Focused Drug Development proposed by both EMA and FDA and endorsed by the ICH Assembly in November 2020.

Currently no clear guidance exists on how robust patient evidence can be generated and there is limited information available on how this evidence has been used during the regulatory assessment/for decision-making, and the impact it has had. To encourage and optimise implementation of a more evidence driven patient centric approach and provide useful information to patients, prescribers and other stakeholders, greater transparency is required.

EFPIA propose that transparency on how patient evidence has been considered in the regulatory decision as an explicit goal in the EMA plans for the development of patient centric drug development. Guidance should be provided on the criteria to ensure the patient evidence generated is adequately robust to be included in the regulatory decision-making process, in the regulatory review/decision documents (e.g. EPAR) and, where appropriate, in the product information.