The FDA is developing a series of four methodological patient-focused drug development (PFDD) guidance documents to address, in a stepwise manner, how stakeholders can collect and submit patient experience data and other relevant information from patients and caregivers for medical product development and regulatory decision-making.
In today’s episode, we discuss the process with the FDA publishing a timeline of planned public workshops, draft guidance documents and final guidance documents for 4 documents, originally planned to finish in end 2021 and its objective to explore the factors that need to be considered when developing COA-based endpoints.
Stay tuned as we also talk about the following points:
- What’s in the 4 guidelines
- What factors should be considered when constructing COA-based endpoints?
- Effect of disease type (e.g., acute, chronic) on endpoint selection – acute symptoms resolve within weeks
- Treatment objective (e.g., cure, symptom management)
- Population
- Endpoint
- Intercurrent Events – What can affect your measurements interpretation
- Population-level summary – What Is the Final Way All Data Are Summarized and Analyzed?
References:
- FDA Patient focused drug development guidance
- PFDD Guidance 4 workshop discussion document
- Public Workshop (Dec 2019)
- Meeting slides
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Rachael Lawrance
Director and Functional Lead – Statistics | Adelphi Values, Patient-Centered Outcomes UK
Rachael leads a team of statisticians at Adelphi Values Patient-Centered Outcomes group, involved in working with clients in all aspects of strategy, analysis and interpretation of PRO data in clinical programmes. Prior to Adelphi, Rachael worked at AstraZeneca for 16 years, including 6 years as a late phase clinical trial statistician on oncology projects. Rachael has particular interest in the topic of estimands in oncology, leading the PRO task force within the Estimands on Oncology PSI/EFSPI Working Group and co-chairs the ISOQoL Statistical SIG workstream on estimands. Rachael is also a Director on the Board for PSI, leading the scientific programme of events for PSI.
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