A new law recently passed by Congress and signed by President Trump has been making headlines over the past few months. This “Right to Try” law allows terminally ill patients to request an investigational drug that has completed Phase I trials directly from a pharmaceutical company. A physician must support the request and forty states already had a similar law. At first glance this sounds like a great idea and law. However, is it?
Let’s take a step back to what is called the Expanded Access or Compassionate Use program currently in place at the FDA. Compassionate Use also allows a patient to request usage of a medicine not yet FDA-approved, but in clinical trials. The request goes straight through the FDA, which, if it approves the request, provides guidance on how to administer the drug and what information it already knows about its safety and potential side effects. While the FDA has been criticized as being slow and not granting many requests, data has shown that the process typically only took 24 to 48 hours and that over 90% of requests were actually approved by the FDA. Once the request clears the FDA, it is up to the pharmaceutical company to make its decision.
So what is the difference under the Right to Try law? Under this new law, the FDA is out of the process. Patients, with their physician’s support, can request the drug directly from the pharmaceutical company. The FDA’s knowledge of other clinical trials using this drug, or safety and administering suggestions will not be shared.
The concern is that this process could ultimately hurt patients. Not only is FDA information not shared, but Right to Try could open the door to companies more concerned about selling or promoting their drug than with patient safety. As it is not clear under this new law who will pay for the drug or the administration of that drug, images of the so-called “snake oil” salesman comes to mind. Could a company charge an exorbitant price for an unproven drug that could do more harm than good? It really isn’t clear.
Of course, as with the Compassionate Use program, under the Right to Try law pharmaceutical companies can say no to the request. There are a variety of reasons they may do so, including the concern that providing the drug outside the context of a clinical trial could impact the trial results. Ultimately, they can say no for any reason.
If you are a patient in need of a new drug, which appears promising in early clinical trials, what should you do? There is nothing preventing you from making your request under both the Right to Try law and the Compassionate Use program. Given the FDA guidance which can be provided under Compassionate Use, it may be worthwhile to start there. If you proceed under the Right to Try law, have some frank conversations with your doctor about what is known about the investigational drug. What are the potential side effects? What clinical trial data already exists? What costs may you incur?
Hopefully, these requests made under the Right to Try law are documented so we can see if concerns about patient safety are warranted and craft regulations to implement the law in a way that keeps patient safety in mind.
For more information, see a recording of our Facebook Live “Right to Try” presentation with Holly Lynch Fernandez Lynch from the University of Pennsylvania.