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Rare Disease Patients: Placebo Controls, Sham Surgeries Not Appropriate For Gene Therapy Trials

 
• By Derrick Gingery

Advocates also worry that long follow-up times could prevent participation in future clinical trials.

placebo syringe
A rare disease group shared focus group data indicating they would not participate in gene therapy trials with placebo controls. • Source: Shutterstock

Rare disease patients and advocates believe gene therapies should not follow the traditional development pathway because it is unethical and could limit future research participation.

Clinical trials with placebo controls or so-called sham surgeries, i.e. a procedure intended to mimic the active arm, could hinder recruitment, numerous speakers said during a Patient-Focused Drug Development Meeting on gene therapy last month

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