I have a lot of political hot buttons and, while I try not to write about them all, this is one that is not particularly partisan and drives me absolutely bonkers. Yesterday at his now daily coronavirus press conference President Trump touted the possibility of people using the Right To Try legislation to access prescription treatments not approved by the Food and Drug Association (FDA). Drafted and lobbied for by the libertarian Goldwater Institute think-tank and pushed by several people with ALS (pALS) and pharma companies, Right To Try was signed into law in 2018. I was against Right To Try (RTT) before it was law, and nothing I have seen in the past two years has changed my mind. RTT was, and is, nothing more than a red herring that distracts people and politicians from the very real issues facing those of us with life-threatening illness and no options. I, more than most, understand the desperation that goes with fighting a terminal illness that has no effective treatment. I share the frustration of seeing one drug after another look promising in mouse trials and flame out in humans, doing nothing or even accelerating death. And I also chafe at FDA trials that take too long and have participation requirements that are too restrictive. Unfortunately RTT solves none of these problems. Right To Try legislation allows a person with a life-threatening illness to request of a company making a treatment that has passed FDA Phase 1 (human safety) trials access to that treatment. Proponents of RTT argue that the government, in this case the FDA, should not stand in the way of a person with informed consent trying to save their own life. The FDA drug approval process consists of three major phases, and phases 2 and 3 can take 3-10 years to complete after a drug is proven safe. The average pALS has only 2-5 years to live. The criteria for participating in a Phase 2 or 3 trial are so restrictive that finding enough qualified and willing participants with relatively rare diseases can take years. And God knows none of us want to spend precious months in a trial only to find we have been receiving a placebo. Given this situation, RTT sounds like a good idea. Bypass the FDA and go straight to the company to get an unapproved drug that just might lengthen your life. But here are the problems:
1. The drug manufacturer does not have to provide the drug to anybody.
2. Your doctor does not have to be involved.
3. The drug manufacturer does not have to collect any data from RTT people using their drug.
4. If the manufacturer does collect data, it does not have to send that data to the FDA.
5. If the FDA receives any data from RTT patients, it is not required to use that data when making decisions to move a drug forward or approve it.
That first point, a company does not have to provide the drug when someone requests, is a big one, and has led to skeptics of the bill derisively calling it “Right To Ask”. Pharmaceutical companies invest enormous amounts of money in developing drugs, and their priority is getting those drugs through the FDA approval process and to the market as quickly as possible. Evaluating RTT requests, expanding production, developing pricing (RTT patients have to pay for treatment out-of-pocket), setting up legal protection and distribution are distractions companies just do not want. Also, no company wants to risk having an RTT drug recipient, who is not under an established trial or treatment protocol and may not even be under medical supervision, having a negative response and trashing the drug on social media before it is even approved. Simply put, betting $100 million of research and development on someone’s Twitter feed is not fiscally responsible. The last three points are related: RTT does not require that any science be done. This is my biggest problem with the bill. Any one person not only does not speed a potential treatment to market, but likely slows the approval process down and increases costs due to diversion of company resources. In my mind, this is selfish and unconscionable. No one person’s life is worth more than anyone else’s, which is why I have taken the stand with my family to not use RTT unless everyone could access it. If my RTT application and use delays approval of a life-saving ALS treatment by just one-half of a business day, I have caused the death of 6 other Americans and dozens of pALS worldwide. I cannot be these people’s executioner. So why do I call RTT a red herring? Because with the bill’s passage, politicians can be satisfied they have addressed the cries for help of people with terminal illness when in fact nothing has changed. RTT has been stunningly unsuccessful. In the two years since becoming federal law, Right To Try has benefitted exactly three people. By contrast, the FDA has for years had a program called Expanded Access, also known as compassionate use. Although similar to RTT, Expanded Access requires a request to the FDA by your physician and approval and oversight from a member of that physician’s Institutional Review Board. It also requires reporting any adverse effects to the FDA. In a similar two-year period, the FDA received 1332 Expanded Access requests and approved 1309 – an approval rate of over 99%. All Americans have an interest in streamlining and accelerating FDA approval processes, both to speed access to new treatments and to reduce the cost of treatments. RTT does neither of these things and gives the FDA and Washington D. C. politicians an excuse to not address the current problems, all while helping virtually no one.