President Trump signs “right to try” legislation into law

The issue as to whether the new, so-called “right-to-try” legislation is a good idea or not is contentious and your sitemaster has no interest in getting into that discussion. What is a fact is that it has now been signed into law.

To quote the Associated Press, in theory:

The measure would give people diagnosed with life-threatening conditions who have exhausted treatment options the ability to gain access to unproven drugs without first getting permission from the FDA.

However, what the advocates for this initiative do not seem to grasp is that there are many, very good reasons why drug developers may be at best “unwilling” and sometimes absolutely unable to provide development-stage drugs to such patients. This is particularly the case for “start-up” developers of early stage, complex molecules like biologic agents, when the costs of the drug being made in small amounts to conduct clinical trials may be extremely high — and very few individual patients would have the financial resources to be able to pay for the drug even at cost (always assuming that they can find a physician who is willing to administer it and help them to get it).

The “New” Prostate Cancer InfoLink would, in general, advise prostate cancer patients who are in such desperate circumstances to do at least two other things before they act on the basis of the new “right to try” legislation:

  • See if the drug in question is available through a well structured clinical trial.
  • Apply for treatment with the drug through the existing FDA expanded access initiative (which will commonly allow a patient to get many investigational drugs free of charge).

We are going to need to wait and see whether the existence of “right to try” legislation

  • Actually leads to a meaningful number of people receiving treatments that they might not have been able to get any other way and obtain meaningful clinically beneficial outcomes as a consequence and/or
  • Actually leads to a meaningful number of patients suffering from or dying as a consequence of side effects of treatment with drugs about which we have very limited information (particularly if those drugs are used to treat conditions about which we have little to no information relevant to the clinical use of the drug in question).

We are also going to have to wait and see what happens when drug manufacturers refuse to comply with this piece of legislation. As far as your sitemaster is aware, there is no legal obligation for a drug developer to provide an experimental drug to a patient who asks for it (even if they are willing and able to pay the relevant production costs). No doubt someone will want to ensure that this one ends up on the docket at the Supreme Court sooner or later!

2 Responses

  1. I agree, Mike, with your assessment and take it further. This won’t change anything. 94% of compassionate care applications are approved by the FDA. But they get declined by the pharmacautical manufacturer because they don’t want the legal risk. These drugs are also declined by insurance carriers. And as far as to whether or not a person could benefit, no one knows, because prior to a clinical trial the drug has not been tested for benefit versus risk.

    With 89% of drugs failing to meet the hypothetical claims in level 1 phase III trials, the chances are more likely that there is not any benefit at all.

  2. I think another unspoken part about this legislation will apply to providers. I know most well-intentioned oncologists simply won’t recommend trying an unproven drug. And most probably won’t administer them either outside of controlled trials. And their hospital IRB’s, clinics, and attorneys won’t be in a hurry to either.

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