FDA

In a previous post, we discussed the FDA’s July 2024 Draft Guidance entitled "Addressing Misinformation About Medical Devices and Prescription Drugs: Questions and Answers" (the "Draft Guidance"). The Draft Guidance suggests a purportedly voluntary pathway for drug and device sponsors, manufacturers, distributors, and other stakeholders legally responsible for the labeling of approved/cleared medical products ("firms") to respond to internet-based posts and comments in a manner consistent with existing product labeling and advertising laws.

You may remember the internet postings during the height of the COVID pandemic touting hydroxychloroquine, a drug approved by the Food and Drug Administration (FDA) for the treatment of malaria and rheumatologic disease, as a prophylactic or even a cure-all for COVID-19. The postings, many initiated by celebrities such as Elon Musk and Dr. Oz and by social media “influencers,” referenced a now discredited, nonrandomized, 36-patient study on YouTube. COVID misinformation campaigns like this, trumpeting off-label use of an approved drug, took a deadly turn, as some infected patients elected to reject physician-recommended treatments or vaccination based upon the belief, encouraged by social media, that hydroxychloroquine and similar unproven treatments such as ingesting borax, were the ultimate answer.

Our last gene therapy article highlighted several novel cures for rare, life-threatening illnesses using gene therapy technology. Many more treatments based on gene therapy are in the clinical trial phase. Despite the remarkable results thus far, significant challenges lie ahead. As with any new treatment modality, scientists must be cautious in their approach, carefully studying the potential side effects in the short and long term. Unlike conventional treatments, gene therapy research raises unique economic, distribution, and manufacturing concerns. Below, we will discuss the medical and social hurdles posed by gene therapy research and commercialization, and the nascent regulatory response underway to help move this new frontier of medicine forward in a safe and ethical manner.