Rare Disease Patients: Placebo Controls, Sham Surgeries Not Appropriate For Gene Therapy Trials
Advocates also worry that long follow-up times could prevent participation in future clinical trials.
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A recent externally-led PFDD meeting on kidney xenotransplantation, rather than a specific disease, allowed for more tailored patient comments on the procedure, which could impact development plans going forward.
A single-arm trial using a biomarker and animal model could be enough for an accelerated approval, especially in a pediatric rare disease where randomization and placebo controls may not be feasible, the CBER director said.
Statements on patient experience with the diseases treated by the drugs subject to price negotiation and how they impact their lives are requested; 10 meetings are planned for the fall.