One fear they have is that an adverse event, like an injury or death — even if it is not directly due to the medication — will derail a company’s ability to push a drug forward for FDA approval, something they argue would ultimately undermine efforts to develop drugs that can help other families.
I personally feel that right-to-try is a political maneuver not based in reality. Making medicine is a dangerous and expensive business. Your right to try something ends at someone else’s right to not accept risk or cost they don’t need to. This example is exactly what I expected to happen with reputable companies. I also believe we will see much less reputable companies cause actual irreparable harm. Imagine that Theranos made “medicines” and could gather data for VC meetings by using right-to-try users as subjects. I’m waiting for that wave of opinion pieces on the horizon.