Systematic integration of patient experience into regulatory decisions
We continue our review of the report on the Accelerating Rare Disease Cures (ARC) initiative. In the section on the patient-centered approach, the U.S. Food and Drug Administration (FDA) describes how the agency is formalizing the translation of patient experience into data suitable for use in planning clinical trials and assessing the clinical significance of results in rare diseases.
A key element of this approach is the Patient-Focused Drug Development (PFDD) program. Under the PFDD approach, patient experience is considered an input parameter for drug development. It includes information on:
During the reporting period, the Center for Drug Evaluation and Research (CDER) division held 10 externally-led PFDD meetings on rare diseases. This format allows patient organizations and the clinical community to structuredly formulate and convey to the regulator an understanding of the disease burden and therapeutic priorities for specific nosologies.
Additionally, patient listening sessions are held—sessions for the organized collection of patient descriptions of symptoms, the impact of the disease on quality of life, and treatment expectations.
ARC places special emphasis on methodology. The program includes seminars on methods for collecting and presenting information; patient experience data for further regulatory use, with a focus on the quality, interpretation, and applicability of such data in decision-making.
Thus, this ARC block describes the formation of a sustainable framework for integrating patient experience:
from its structured collection (PFDD meetings and listening sessions) —
to methodological understanding —
and then to the use of these data in the selection of clinically significant endpoints and discussion of trial design in rare diseases.
The beginning of the series on the ARC program can be read at the link in the previous post (https://t.me/occ_cis/261)
A key element of this approach is the Patient-Focused Drug Development (PFDD) program. Under the PFDD approach, patient experience is considered an input parameter for drug development. It includes information on:
- What symptoms and functional limitations are most significant for patients;
- What changes are perceived as clinically important;
- How does the disease affect daily life?
- What trade-offs between the benefits and risks of therapy are acceptable.
During the reporting period, the Center for Drug Evaluation and Research (CDER) division held 10 externally-led PFDD meetings on rare diseases. This format allows patient organizations and the clinical community to structuredly formulate and convey to the regulator an understanding of the disease burden and therapeutic priorities for specific nosologies.
Additionally, patient listening sessions are held—sessions for the organized collection of patient descriptions of symptoms, the impact of the disease on quality of life, and treatment expectations.
ARC places special emphasis on methodology. The program includes seminars on methods for collecting and presenting information; patient experience data for further regulatory use, with a focus on the quality, interpretation, and applicability of such data in decision-making.
Thus, this ARC block describes the formation of a sustainable framework for integrating patient experience:
from its structured collection (PFDD meetings and listening sessions) —
to methodological understanding —
and then to the use of these data in the selection of clinically significant endpoints and discussion of trial design in rare diseases.
The beginning of the series on the ARC program can be read at the link in the previous post (https://t.me/occ_cis/261)
