FDA's Patient-Focused Drug Development – Part 2


In part 1 of our FDA's Patient-Focused Drug Development blog, we discussed the origin of FDA’s patient-focused drug development from Prescription Drug User Fee Act (PDUFA) V and VI and the 21st Century Cures Act (Cures Act) to the development of a guidance that will allow for the direct collection of patient experience data to guide regulatory decision-making. Plan for Issuance of Patient‐Focused Drug Development Guidance from May 2017 describes FDA’s plan for development of a guidance under the Cures Act.


  • The results of FDA sponsored patient-focused drug development meetings determined that:
    • Patients are better equipped to contribute an understanding of the benefit-risk ratio due to their intimate knowledge of living with their disease.
    • Clinical trial design and product labeling may not consider patient preferences.
  • Methods for collection of patient described disease impacts and benefit-risk assessments need to be developed.
  • After the patient identified criteria are collected, they need to be translated into a validated tool to be utilized in clinical trial design to reflect patient preferences.

The Cures Act provisions (c)(1) through (4) of Section 3002 describe ways that stakeholders can collect information about disease and treatment impacts to patients and their preferences. The Patient-Focused Drug Development Plan describes how the FDA will implement these provisions:

  • The FDA plans to issue four guidance documents describing the process of moving from collecting patient-driven benefit-risk assessments and disease impacts to development of validated tools for use in regulatory decision-making.
    • The first guidance draft involves methods of collecting patient and caregiver responses for disease and treatment impact and is expected to be released by the end of the second quarter of 2018.
    • The second guidance draft will describe development of a comprehensive description of the most significant impacts to patients and what matters most and will be published by the end of the second quarter of 2019.
    • The third guidance draft will specify methods to identify impacts to patients to improve the collection of patient input in clinical trials and will be released by the end of the second quarter of 2020.
    • The fourth guidance draft will assess clinical outcome measures and may revise or append the extant 2009 Guidance to Industry on Patient-Reported Outcome Measures. The draft will be published by the end of the second quarter of 2020.
  • Public workshops will be held prior to each guidance document draft to gather public input.

These four draft guidances are designed to integrate patient preferences into the drug development process. The movement toward patient-focused drug development recognizes patient preferences, disease and therapy impacts, risk-benefit considerations from the patients’ point of view, and what matters most to the patient may affect patient satisfaction and well-being. Understanding patients’ experiences and integrating patient preferences into the drug development process should improve clinical trials best practices and reduce study impacts to the patient.

- The Clinical Pathways Team

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