What's Happening to the 'Right to Try'?

One of the accomplishments of the first Trump administration, one that 
President Trump regularly hyped himself, was the right to try. That is the 
ability of people “diagnosed with a life-threatening disease or condition” 
to try medicines still in the trial phase, not approved by the Food and 
Drug Administration (FDA). In those cases, where all approved and 
conventional treatments have failed, it gives hope where it otherwise 
would not be.

So, what happened to that?

The right to try is still the law of the land and is racking up success 
stories. The Goldwater Institute reports on a Naval aviator who was 
diagnosed with Lou Gehrig’s Disease and was helped significantly by an 
experimental treatment. The pilot, Matt Bellina, “was one of the first 
beneficiaries of the federal law that carries his and a handful of other 
patient advocates’ names. In 2019, he began receiving an investigational 
treatment under the law, and within weeks, he experienced improvements in 
his breathing and physical strength. Matt, a husband and father of young 
sons, has now significantly outlived his expected prognosis of just two to 
five years.”

It is impossible to know what would have happened without the right to 
try, but we know it was good. Matt is still alive.

Of course, the same cannot be said for everyone who exercises the right to 
try. Life, as we all know, is fragile, and nothing is certain. Illnesses 
for which there are known treatments still claim lives, as no treatment 
has a 100 percent success rate. Each individual is different.

One of the biggest problems with prescription drug price controls is that 
it severely limits the potential profit a company can make during the 
short life of their patent, thereby limiting what companies will spend 
resources to research treatments for. There are plenty of ailments that 
impact a small number of people, and the potential for profit is the only 
thing motivating treatment development. Without that, there will be fewer 
treatments sought and only for ailments that impact enough people. That 
makes right to try all the more important, and the research in gene 
therapy a wildly important component of it.

On the issue of gene therapy, a promising field of development for chronic 
conditions, the right to try has come up against a roadblock. Tony 
LoSasso, professor of economics at DePaul University, wrote in The Wall 
Street Journal of a gene therapy called Elevidys, “a gene therapy approved 
by the Food and Drug Administration for a form of Duchenne muscular 
dystrophy. This devastating progressive disease robs children of their 
ability to walk, breathe and live independently. In 2023 the drug was 
approved based on encouraging results in a small trial, as is typical in 
the world of rare-disease research.”

Unfortunately, families with children suffering from Duchenne have been on 
an additional and unnecessary rollercoaster as the FDA abruptly halted and 
resumed shipments for ambulatory patients in recent weeks. Non-ambulant 
patients are still waiting for access to Elevidys to be reinstated.

The Washington Post reported the therapy sought “accelerated approval, 
designed by the FDA for drugs that treat patients suffering from severe 
diseases with few effective therapies.” Now, many sufferers of Duchenne 
will have one less option in a field that is very few. What about the 
right to try?

As a parent, I can tell you I finally understand what my parents were 
talking about when they said there was nothing they would not do to try to 
help me and my siblings. Imagine the government getting out of the way, 
then getting back in it.

Horrible. Speaking of horrible, a friend who knows I have an interest in 
this topic reached out to me to inform me of what they’d come across – a 
LinkedIn post in which the “Chief, Oncology Branch 1” at the FDA appears 
to have written in a comment that a drug to treat cancer – melanoma (skin 
cancer, specifically – stated that “the BLA clinical team thought the 
applicant had provided adequate evidence to support contribution of effect 
of the RPI to nivolumab but leadership did not agree.”

What does this mean? It sure seemed like it means FDA leadership under FDA 
Commissioner Martin Makary, for whatever reason, overruled researchers and 
clinicians on a drug called Replimune, which is described as “a viral-
based treatment from Replimune Group intended for patients with advanced 
skin cancer.” If that sounds a little complicated, that’s because it is. 
It’s also wildly interesting and important, and you can read all about it 
from the man, Sean Khozin, who the oncologist was replying to in the now-
deleted comment.

Derek Hunter is the host of the Derek Hunter Show on WMAL in Washington, 
DC, and has a free daily podcast (subscribe!) and author of the book, 
Outrage, INC., which exposes how liberals use fear and hatred to 
manipulate the masses, and host of the weekly “Week in F*cking Review” 
podcast where the news is spoken about the way it deserves to be. Follow 
him on Twitter at @DerekAHunter. 

https://townhall.com/columnists/derekhunter/2025/08/03/whats-happening-to-
the-right-to-try-n2661313