Global Harmonization for Patient Focused Drug Development (PFDD): What Are the Arguable Challenges for Pragmatic Guideline Uptake and Implementation and How Should We Negotiate and Navigate Alignment?

ISSUE:

There is growing consensus that patient experience data (PED) and patient engagement (PE) is important to consider by regulatory authorities during drug development. However, there are a multitude of guidelines and frameworks published and emerging for patient focused drug development (PFDD) and ongoing challenges for harmonization. Further, an initial response to the guidelines by regulated industry challenges the clarity on how and/or when PE and PED collected during drug development will be considered by regulators as sufficiently robust for the purpose of benefit-risk decision making and/or inclusion in the regulatory documents and product information.

OVERVIEW:

Rebecca will open discussion with the imperative, benefits and state-of-the-art of PFDD. The panel will present their multi-stakeholder perspectives on the importance to ensure that the chief complaints of patients are explicitly factored into drug development especially for outcome measures and endpoints used in a clinical trial to establish safety and efficacy of a medical product.

Robyn will examine some tangible examples of PE and collecting PED in product development.

Challenges and barriers for PE and collecting PED during drug development such as “Time constraints”, “Lack of budgetary commitment”, “Challenges in finding/engaging patients to collect patient centric data” and “Lack of expertise and resources” will be argued by the Yvette and Rebecca.

Robyn will present perspectives from regulatory authorities for opportunities for a globally harmonized approach to PFDD that is methodologically sound and sustainable and for successful partnerships with regulated industry to support PFDD.

Yvette will highlight the key role of the PFDD message being driven by regulatory bodies to mitigate skepticism and provoke the knock-on impacts for value demonstration from a HTA/payer evidence needs perspective through to implementation science and to long-term overall improvement of standards of healthcare and health outcomes.

An interactive discussion (panelists, moderator, audience) for questions and comments will follow.