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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Even in a rare disease with no approved treatments, the agency shows once again that without clear efficacy data, FDA has few options for approval.
The group will be called the Office of Therapeutic Products, but needs more people to handle its increasing workload.
Development standards for a one-time gene therapy should be different from a drug taken regularly, CBER Director Peter Marks says.