A Streamlined Pathway to Drug Approval
My son's case shows that an emphasis on patient experience data could foster drug development breakthroughs
Randomized, placebo-controlled clinical trials have long been the gold standard for drug approval. Patients are assigned to either drug or placebo and the comparison between the two groups can provide meaningful data to allow the Food and Drug Association (FDA), pharmaceutical companies, physicians, and patients to understand whether the perceived drug effect (or safety signal) results from the drug or is the result of the placebo effect. While this approach to drug approval is undoubtedly good and often best, is it optimal for all patients? Specifically, is it optimal for children (and families) who have a rare debilitating disease that lacks reasonable treatment options?