The FDA recently released updated guidance, titled “Rare Diseases: Considerations for the Development of Drugs and Biological Products,” emphasizing the importance of engaging patient and caregiver populations in the development of drugs for rare diseases. This final version builds upon draft guidance issued in February 2019, offering recommendations for sponsors developing drugs for rare diseases. The guidance emphasizes the involvement of patients, caregivers, and advocates throughout the development process, advocating for direct communication between sponsors and patients to navigate the FDA’s strict confidentiality requirements.
The guidance outlines various avenues for engagement, ranging from providing consultation on scientific issues, collaborating with industry sponsors during clinical investigations, and contributing to patient-focused drug development initiatives. The updated guidance includes a streamlined approval pathway with modified nonclinical data requirements for drugs intended to treat severely debilitating or life-threatening rare diseases. Furthermore, it encourages the study of drugs in relevant pediatric populations and proposes strategies for addressing challenges in using clinical endpoints, such as leveraging natural history or registry-based studies and exploring early development work on biomarkers.
Read the guidance here.
Last Updated on February 14, 2024 by Aimed Alliance
