Right to Try-Walking the Tightrope

tightrope-walker-1314832Yesterday, the president, signed the Right to Try Bill into law. But, what does that really mean for the majority of patients suffering from TERMINAL illness?

I’ll try to break it down as best as possible from what I know and understand. These are MY opinions only, for your own certainty, I suggest reading articles that contain pros and cons.

The FDA already has a “Compassionate Use” program. They approve approximately 99% of requests. The new bill implies that the hold up is the fault of the FDA, this is incorrect; per the article noted below, the time from a complete application to approval is about 4 days, in some instance verbal approval is given immediately over the phone. The ACTUAL culprit is the drug companies themselves. Here is the link to the FDA requirements-here

How does the Right to Try law differ-

First of all it bypasses the FDA and by association the FDA safety guidelines. It allows for drugs which have only passed a Phase I trial to be given out to patients. A Phase I trial is very small, it’s basically a “it doesn’t kill you outright” kind of phase. There is no data on efficacy, no data on possible side effects, no concrete optimal dosage. The FDA will not oversee the Right to Try “program”.

How are they similar-

Both have some safe guards for patients, but the Right to Try law sets a much lower bar.

In my opinion-what are some of the pros to The Right to Try Law

  1. Definitely if a person is near death and qualifies, this could be their only option, a hail Mary if  you will.
  2. There is the chance that an experimental drug that hasn’t made it through trials yet miraculously cures a patient.
  3. It gives people who can afford it a chance.

In my opinion- what are some of the cons to The Right to Try Law

  1. It sets the lowest bar for safety, only a Phase I trial.
  2. There is no regulation by the FDA, that means a drug company does not have to file side effects/death etc. statistics except for once a year.
  3. Very few drugs make it through the Phase II trials because efficacy is either not proven or the side effects are severe. Therefore, ineffective drugs can be given to patients or worse, drug companies will knowingly provide drugs that have not had safety proven and reap the rewards of a dying person’s hope.
  4. Just because a patient has a right to request the experimental drug, the drug company is under no obligation to actually provide the medication AND insurance companies are under no obligation to cover an experimental medication, which means there is almost a 100% chance that the patient would need to cover the costs, which could mount into the hundreds of thousands of dollars, and get no actual benefit.
  5. I can easily see a scenario, where a company decides to forgo costly trials and just make the medicine available to those who can afford it, saving millions in trial costs, but creating a severe loss to the population that cannot afford an experimental drug, one that might have eventually made it to market and been covered by insurance.

What are some of the reasons a drug company would say no?

  1. A  drug company would most likely say no if there is an ongoing trial due to insufficient supply of a drug for the trial if it is given to patients not enrolled. Also, a patient who has reached the point where there are no other options and is given the medication may die while taking the medication. This would probably be a natural course and in no way connected to the drug, but this would look VERY bad to investors and make them leery to continue investing in a trial.
  2. Even if a drug company has completed a Phase II, there is still the matter of a Phase III, if the drug is given and again the patient dies, the Phase III may never get completed and a drug that may be perfectly safe won’t get approved by the FDA and insurances will never cover it.

What are some reasons a drug company might say yes?

  1. If a drug has gone through a Phase III and is simply waiting for approval from the FDA, which appears imminent, then yes, I can see a drug company releasing some of the medication to a patient- optimal dosing will have been worked out, in addition there will be documentation of safety and efficacy, so that if the patient does not survive it can reasonably be assured that it was not from the medication. In such a case, I can even see the drug company providing the medication for free for a period of time, while awaiting for FDA approval as it is possible any participant in the Phase III trial would have been offered the same.
  2. A drug company may also say yes if it wants to gather data and bypass the stringent rules of the FDA. It could do a Phase I and then instead of doing all the work to get to a Phase II and possible failure, provide the medication to the sickest of patients and if there is no improvement in any of them, scrap a Phase II and not only save a ton of money, but MAKE money.

Here is an article that really goes into it all in depth.

In my opinion, what does this law mean for our children. Unfortunately, nothing.

In order to get an unapproved medicine, patients will have to have a “life-threatening illness” and they can’t be eligible for clinical trials. They’ll also need to have exhausted all available treatment options.”

Even with this low bar, I don’t believe Rett children/adults will be approved. First, we do not know the criteria for Trofinetide, so it may very well be that they would be eligible for clinical trials. There are other promising treatments in the works (Anavex comes to mind) and until the criteria is set for a trial how can anyone know if they are eligible? I can think of some children and adults for whom this may hold true, that they are not eligible for clinical trials; but, then we are left with these hurdles-

  1. Will the drug company supply the medication and take a chance that the patient survives and actually improves if there is a current trial going on.
  2. If there is not currently a trial, will the drug company take the chance of investors interpreting a death as a failure of the medication and pull out of funding for future trials.
  3. Will the drug company have enough medication TO supply it to a patient not in a trial.
  4. Even if a drug company is reasonable assured of efficacy, has enough medication to provide to a patient, how will a patient afford an experimental drug? Someone suggested crowdfunding, and yes, perhaps in a rare instance this is a viable option, but if a thousand people with an incurable disease all start to fundraise for a medication that they have to take the rest of their lives, we’re talking millions of dollars per patient.

Is there some hope in this law, of course-if a patient can afford it. Do I think that this law should exist? Actually, yes. I think a patient SHOULD have the right to at least try IF they are willing to sign away any right to sue a pharmaceutical company for unseen side effects, since that is not the focus of a Phase I trial. Do I see the real potential for abuses by pharmaceutical companies, yes, I do.

In the end, each parent/patient should do their own research.




This entry was posted in Anavex, cure, FDA, fund-raising for rett syndrome, hope, Rett Research, Right to Try Law, Trofinetide, Trofinetide Phase III, white house and tagged , , , , . Bookmark the permalink.

3 Responses to Right to Try-Walking the Tightrope

  1. james says:

    Isn’t Right to Try Act mainly aims to accelerate use of gene therapy and not for more conventional drugs such as Trofinetide and Anavex?

    Why? Because gene therapy is mainly ‘one shot’. All the therapy is applied in just hours if not in minutes. Therefore if Phase I is successful generally it means that Phase II and III will also be.

    The most important and crucial example for Rett patients and families is Avexis and it’s gene therapy for SMA. It is proven to be successful in Phase I only and before even Phase III started Novartis bought Avexis. And the latest news is:

    “Novartis’ brand-new gene therapy player AveXis will build $55M plant, hire 200”

    The demand for the gene therapy is immense it seems.

    And for us if Avexis AVXS-201 is successful in phase I (not a cure but important improvements) than the Right to Try Act will be more meaningful?

    The real problem will be the cost it seems!

    • melelllan says:

      Certainly, gene therapy would be the most cost effective, but again what is to prevent a company from selling the drug to patients that can afford it, without moving forward with a Phase II and then Phase III? This would save then MILLIONS. And since gene therapy is in its infancy, not all diseases have someone working on them, so initially more conventional drugs would be the subject, imo.

      • james says:

        From my understanding, only a single Phase is enough to understand if gene therapy is working or not. Unlike conventional drugs, where duration of the therapy is crucial and double-blinded participation is necessary to compare placebo and actual people who receive the drug, gene therapy’s effectiveness is not depending on how long the drug is being given. That’s the reason Phase I-II-III trials for the SMA is all the same and it took only 2 years for the drug to reach from Phase I to Phase III. So if gene therapy is successful in only for Phase I, than that’s it. Phase II and Phase III are just for FDA necessity.

        For our children I believe that both gene therapy and Trofinetide is necessary, as for now neither of them can ‘cure’ Rett syndrome. If gene therapy is successful and can reduce symptoms up to 50% -%60 (the graphs says so) than take Trofinetide afterwards.

        So I believe and really hope that in a couple of years we can enter the ‘improvement phase’ of Rett syndrome, if not the ‘cure’, with symptoms minimised and talking simple sentences and walking independently become the norm.

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