Admittedly I wasn’t watching the State of the Union address last night… I was playing Fortnite, but such is life when we live in the era of recorded TV.
The president mentioned “Right-to-Try” in his address to the nation and members of Congress. From CNBC:
We also believe that patients with terminal conditions should have access to experimental treatments that could potentially save their lives…People who are terminally ill should not have to go from country to country to seek a cure — I want to give them a chance right here at home. It is time for the Congress to give these wonderful Americans the “right to try.”
Sounds good, but what is Right to Try?
On the surface it is a bipartisan effort to lift the barriers that exist for terminally ill patients when trying to receive experimental medication before the medication has received FDA approval. A few FAQ’s from RightToTry.org:
- Right To Try is legislation that allows terminally ill patients to access investigational treatments that have passed basic safety testing (Phase I) with the FDA, but are not yet available on pharmacy shelves.
- The only treatments available under Right To Try laws are those that have already passed the FDA’s basic safety testing and remain within the FDA’s approval process.These are no different than the treatments currently available to the 3% of patients who are lucky enough to be accepted into clinical trials.
- Fewer than 3 percent of terminally ill patients gain access to investigational treatments through clinical trials. Right to Try was designed to help the other 97 percent.
So far several states have Right to Try laws, but there is nothing on the federal level in the US, which is what the President was alluding to last night.
There are however, some naysayers. From STAT:
Opponents of the legislation point out that 38 states already have similar laws on the books. They also note that the FDA itself has a pathway for approving individual patients’ requests for access to experimental therapies. The FDA approves about 99 percent of those requests, often within a short time frame. Advocates, however, say they need the federal legislation to ensure the FDA and other federal agencies don’t interfere with the state laws…At an October hearing, FDA Commissioner Scott Gottlieb warned that Johnson’s bill, as written, would broaden access to experimental therapies so much that it might weaken FDA’s drug approval process.
Where does the bill stand now?
The Senate unanimously passed a right-to-try bill from Sen. Ron Johnson (R-Wis.) last August, but it has since stalled in the House.
So this is a pretty interesting thing, since well… I fall into the category of terminally ill. It’s a pretty exclusive club.
It’s kind of a hard thing to be against this bill. No one wants to be seen as denying a very sick person a chance at living, but I’ll play the devil’s advocate here.
Undermining the FDA approval process and the path for drug development is no small thing. As much as I love to believe the FDA is an annoying agency that functions way too slowly, it does a pretty good job of achieving its mission. In a way, it keeps us safe from ourselves.
I have been in clinical trials that have “failed” or have been discontinued for lack of efficacy. The FDA holds strong to study endpoints for a reason, and the placebo effect, as I’ve said many times before, is more than a reality that we see in medicine. We want to believe we are getting better, but are we?
Beyond that, I’m not sure I totally see an incentive for drug developers to jump on board with this… or rather I’m not sure I see the incentive is 100% there. Sure it’s a good idea to get a potentially life saving drug in the hands of as many sick people as possible, but how is the developer going to do that? Who is paying for it? What risk is involved?
What happens if this drug has an enormous unforeseen safety consequence in the hands of one of these Right to Try patients? How does that impact the drug on a large scale and its availability to the rest of the patient population?
If this law is passed, what force is pushing these companies to disseminate medications beyond already existing compassionate use programs? How is that extreme cost justified without reimbursement?
On the other side of the coin, I could be all for this. Something I struggle with is the idea that there are medications out there slowly progressing through clinical trials that seem to be helping people with CF in a huge way. What about the people who aren’t able to get into these trials for falling below inclusion parameters (In CF: FEV1, strains of bacteria, erratic health, etc.) or the people who are stuck on placebo?
Being given the placebo in a trial that ends up being successful is like a cruel twist of fate.
In talking with a friend about this subject, she mentioned that this, on the surface, seems very similar to the open-label portion of a clinical trial – the part where all participants receive live drug for a set period of time (usually between the end of the phase and the hopeful approval date so participants don’t have to discontinue the medication if it works). We’re also entering the world of personalized medicine. It may be impossible to do enormous studies that enroll dozens, or even hundreds of patients. Is Right to Try appropriate for someone who requires a personalized medication? What about the person who has such a rare combination of CF mutations? How is that person benefiting from genetically pooled trials? Or what about the person who has run out of antibiotic options and won’t be around much longer (or be able to keep his FEV1 above a certain level) to see the next anti-infective advance to a trial he can enroll in (or get FDA approval)?
To that end I have another friend who is going through a compassionate use program where the road to the medication seems longer than ever. Despite my friend being one of the “99% of people who received FDA approval for compassionate use” they have yet to receive the medication. The fact of the matter is that the road to new medications is long, too long.
Ultimately I think I support Right to Try, but I could be swayed against the law if it truly does undermine the FDA’s approval process. My only caveat is that the approval process must be sped up… and maybe it is, albeit slowly – we are seeing huge numbers of medications getting approved. That’s a funny thought, though… the FDA approval process is slowly speeding up.
I understand that science takes time, and I’m not sure legislating science really kicks it into high gear, but we can certainly work on removing some of the red tape that exists.
What do you think about Right to Try?