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   From: yourdime@outlook.com   
      
   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   
      
   --- SoupGate-Win32 v1.05   
    * Origin: you cannot sedate... all the things you hate (1:229/2)