In the age of “precision medicine,” “21st
Century Cures,” and “breakthrough” therapies, it should come as no surprise that legislation called “Right-to-Try” would pass in both the US House on Wednesday evening and the Senate earlier.
The bill will now move back to the Senate for another vote, after 35 House Democrats and all but two Republicans voted for it Wednesday evening.
Earlier in the day, Republicans said on the House floor that the bill will “ease the process” or create an alternative “pathway” for accessing experimental treatments after Phase I trials. But questions remain on how many will gain access if the bill passes. Few have received access so far because of similar legislation at the state level.
One of the biggest unknowns is how many companies developing products that must win US Food and Drug Administration (FDA) approval (and did not allow access to such therapies under state “Right-to-Try” legislation) will opt to use what has been proposed under the federal version of the bill?
Representatives on Wednesday made it sound like the flood gates of cures would finally be opened for those with terminal illnesses. The comments followed a Wall Street Journal
editorial earlier this week calling for the bill’s swift passage, saying it “hedges against a future FDA that could be hostile to patient choice,” setting the stage for FDA's return to the Mesozoic Era of not addressing efficacy.
And perhaps the federal bill will do what the state legislation has largely done: Raise the profile of FDA’s expanded access process
, where companies developing products alongside the agency that approves them can seek (and receive over 99% of the time) permission in as little as 24 hours to grant access to experimental treatments.
As generalizing about clinical trials can be difficult, at the end of the day, it will be up to the companies to decide. And as those in the clinical research field have explained, companies raising and spending billions to bring products to the US market faster need to conduct trials in a controlled way to ensure what patients get is safe and effective.
Hopefully the legislation will not do the opposite of what had seemed at the outset like an honorable goal: to help people with terminal illnesses and better ensure they do not spend their final months seeking unsafe treatments from companies or doctors who have no intention of ever marketing that experimental treatment or figuring out if they actually work.