07/12/2022
Photo by Rodion Kutsaev on Unsplash
The US Food and Drug Administration (FDA) released a draft guidance “Patient-Focused Drug Development: Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcome Assessments” June 2022. The focus of the draft guidance is on selecting, modifying, developing, and validating clinical outcome assessments (COA), such as patient reported outcome (PRO), that are meaningful to patients, with an emphasis on methods to ensure high quality measures are utilized.
This draft guidance is the third in a series of patient focused FDA guidance. The first, “Patient-Focused Drug Development: Collecting Comprehensive and Representative Input” finalized in June 2020, focuses on best practices for the methods of data collection that ensure accurate and representative experience data, which includes patients’ experiences, perspectives, needs, and priorities. The second, “Patient-Focused Drug Development: Methods to Identify What Is Important to Patients” finalized in February 2022, focuses on best practices in approaches to identifying what matters most to patients, related to their experience with living with the disease or condition and the burdens of treatment. These guidance documents are aligned with the 21st Century Cures Act (Cures Act) of 2016 requirement for patient-focused drug development (PFDD).
A well designed COA measures outcomes that are meaningful to the study participant, is modifiable by an investigational product, and could show clinically meaningful differences between the study arms. Fit for purpose means “valid.” Considerations to ensure COA are fit for purpose are:
The use cases of the COA are clearly described.
Evidence supports the rationale for using and interpreting the COA.
Other considerations for COA include:
Understanding the disease or condition
Conceptualizing clinical benefits and risks
Evaluating existing and available COAs
Considerations for COAs in pediatrics
Using digital health technology to collect COA data
COA accessibility and universal design
Developing a Conceptual Framework
Developing evidence to support the COA is fit for use
Helpful appendices with examples cover the following four different types of COA:
Appendix A: Patient-reported outcome (PRO) Measures
Appendix B: Observer-reported outcome (ObsRO) Measures
Appendix C: Clinician-reported outcome (ClinRO) Measures
Appendix D: Performance outcome (PerfO) Measures
Using high quality, fit for purpose COA helps ensure it measures what matters most to patients with the condition or disease while providing valid results.
Comment on the draft guidance now through September 28, 2022 HERE.
Read about the origin of FDA’s patient-focused drug development programs in our blogs:
Read about the other patient centered guidance:
Clarified in ICH E8(R1), the General Considerations for Clinical Studies Guideline, study design should incorporate a patient centered approach with a consultation of stakeholders who are knowledgeable about living with the condition. Want to learn more? The interactive eLearning course is available for purchase HERE. Read our blog HERE for more information.
- The Clinical Pathways Team
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