2 min read

FDA Guidance Series on Patient-Focused Drug Development (PFDD) to Enhance Product Development and Regulatory Decision Making

Regulatory Consulting Strategic Consulting Regulatory Submissions CMC

As the industry continues to adopt a patient-centric approach, the use of real-world data is becoming increasingly essential in informing drug development and regulatory decision-making. To develop patient-focused strategies, researchers must gain a deeper understanding of the patient experience, including their needs, concerns, and perspectives on the impact of disease and existing therapies.

To support this approach, FDA has developed four methodological guidance documents on patient-focused drug development (PFDD) to address how stakeholders can systematically collect, submit, and meaningfully use patient experience data.

1) Collecting Comprehensive and Representative Input

Examines various sampling methods suitable for collecting patient input; offers a general overview of the correlation between potential research questions and methodologies when selecting the target population to gather input from.

2) Methods to Identify What is Important to Patients

Discusses methods for obtaining data from individuals identified in Guidance 1, including the impacts of disease and issues important to patients; examines best practices for conducting qualitative research (i.e., conducting interviews and surveys).

3) Selecting, Developing, or Modifying Fit-for-Purpose Clinical Outcomes Assessments

Explores strategies for selecting, modifying, developing, and validating clinical outcome assessments (COA) to measure outcomes of importance to patients in clinical trials.

4) Incorporating COAs into Endpoints for Regulatory Decision Making

Examines methods, standards, and technologies to collect, capture, store, and evaluate clinical outcome assessment (COA) data, and explores methods to better incorporate COAs into endpoints for regulatory decision-making.


Impacts of Patient-Focused Drug Development (PFDD)

Integrating the perspectives of patients, caregivers, and patient advocates in drug development and evaluation enables sponsors to better inform clinical trial design, trial endpoint development and selection, and regulatory reviews. Sponsors can guide their research efforts and target products that have potential therapeutic effects across the specific functional or symptomatic aspects that are most meaningful to patients. By adopting PFDD, developers can help to reduce patient burden while also improving patient outcomes.


Executing Patient-Focused Drug Development (PFDD) for Successful Clinical Development

Learn the fundamentals of understanding and collecting patient experience data, the impact of patients, caregivers, and patient advocates on new research and in regulatory decisions with agencies, and key advantages of PFDD.

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Conclusion

The integration of Patient-Focused Drug Development (PFDD) into CDER’s regulatory framework sparks a fundamental shift towards a more patient-centered approach to drug development and approval. By valuing patients’ experiences and insights, CDER not only enhances the credibility of the drug approval process, but also promises to deliver treatments that are more closely aligned with the needs and preferences of those that need help.

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