A 25-year-old living with Duchenne muscular dystrophy has written an opinion piece urging for a more patient-centred approach to FDA drug approval processes. The author, not expected to live much longer according to medical statistics, argues that the next FDA commissioner should empower patients and doctors to decide whether experimental treatments are worth trying, rather than having a regulator in Washington make that determination unilaterally. This matters because the FDA commissioner's decisions directly affect the 30 million Americans living with rare diseases, potentially determining how many treatment options are available and how quickly they can access them.

The author acknowledges the importance of scientific evidence and clinical trials, but argues that when a patient understands the risks, their physician understands the risks, and there is genuine evidence a treatment could help, the FDA should shift from a paternalistic to a partnership approach. They cite the Right to Try Act (signed in 2018), which allows terminally ill patients to try experimental treatments when approved options are exhausted, as important precedent. Senators have proposed Right to Try 2.0 to extend this patient-centred philosophy to rare-disease treatments and individualised medicines. The author also highlights practical barriers to access, noting that requirements like travelling long distances for multi-year clinical trials create additional hardship for patients whose bodies are already working against them.