Rare Disease Drug Development Should Engage Patients, FDA Says

By Jessica Karins / February 8, 2024 at 2:27 PM
Development of drugs for rare diseases should engage patient and caregiver populations and carefully consider which endpoints are most effective for evaluation of a drug’s efficacy, FDA says in a recent guidance. FDA notes that it is subject to strict confidentiality requirements, so it may not be able to share information about drug development programs with the public, making direct communication between sponsors and patients a better option. “This engagement can take many forms, such as providing solicited consultation on...


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