The History of the "Right-to-Try" and the FDA

On May 30, 2018, President Donald Trump signed Senate Bill 204 into law. Senate Bill 204, better known as the “Right-to-Try” Bill, allowed patients with terminal illnesses access to drugs not yet approved by the FDA, but have passed Phase 1 testing. Several states had already passed bills similar to this, but with the passage at federal level, it covers every state. This bill is a victory for patients and doctors everywhere, giving them more options in their treatments. To understand this victory, the process of drug approval by the FDA needs to be detailed.

Ever since FDR signed the “Food, Drug, and Cosmetic” Act into law in 1938, the FDA has, among other duties, conducted pre-market reviews of any and all drugs intended for medicinal use. Any drugs that do not pass these pre-market tests cannot be sold on market or given to patients. To say these tests are difficult to pass is an understatement. There are several phases through which a drug must be tested and pass in order for the drug to be offered for sale to consumers.

(I am simplifying the process a bit here, as there are exceptions to rules, and nuances to the whole affair, but what is detailed below as a good estimation of the journey a drug would have to go through to make it to consumers.)

To start this process, a company has to file an IND, or Investigational New Drug. There are three different kinds of INDs, but the Investigator IND is the default path, with the other kinds of paths for emergency situations. After this, the initial testing begins. This consists of very basic observations in patients, and if it shows promise, the trial is expanded. This is sometimes referred to a Phase 0 and Phase 1 respectively, even though Phase 0 is often combined with Phase 1 in most descriptions of the process.

If successful, the drug then proceeds to Phase 2 and 3. With each progression in phase, the trials expand to more and more patients and more intensive observation to detail to catalog the main and side effects of the drug. By the time the drug hits Phase 3, there could be as many as 3,000 patients involved in the trial.

After this, the evidence and information from the trials are piled together and presented to the FDA. A typical page count is around 100,000 pages. The company then files an NDA, or New Drug Application. The FDA reviews this information, and either accepts the NDA, and the drug goes to market, or they deny the NDA. The denied company can file an appeal hearing, but if this too is rejected, they are out of luck and are sent home empty-handed.

As one might expect, many drugs filed for INDs do not go on to have their NDAs accepted. Only about 10% of drugs survive the whole process. The time and money it takes to carry a drug through the entire process varies from drug to drug. However, 10 years time and 1 billion dollars spent is not unusual.

The FDA outdoes themselves in proving the effectivity of drugs, as you can see. There does exist a major flaw with this system, however. Namely, that the process is all-or-nothing. If a drug passes through Phases 1 and 2, but falls just short of passing through Phase 3, then the drug fails the whole process. This becomes a problem when we consider the plight of patients with terminal illnesses. There might be a drug in testing that has the potential to cure their disease, but if the FDA considers the drug to be dangerous, then they can’t acquire the drug.

(Technically, it is not true that the patients have no way to get access to experimental drugs to save their lives, as the FDA has a “compassionate use” pathway that patients can apply for. This pathway allows patients to apply use drugs currently in trial, but not yet approved for commercial sale. This program is mostly an afterthought, however, as many drug trials are not open to Compassionate Use pathways, and many doctors do not even file applications for the pathway simply because of the long process involved in doing so.)

This means that if you have a terminal illness, and an experimental drug exists that could cure you, if you can’t get in a trial for the drug, you can’t access it. If the FDA’s standards for the drug, which are very high as the approval process shows, are not met, terminal patients are left out to dry.

Not only was this the de facto system, it has also been defended in court. Abigail Burroughs was a college student diagnosed with head and neck cancer. Her father created the “Abigail Alliance for BetterAccess to Developmental Drugs” to sue the FDA to get access to an experimental drug that could treat Abigail’s cancer. In Abigail Alliance v. Von Eschenbach, AA argued that patients a constitutional right to life-saving experimental drugs before the FDA approves them for general use. AA proposed that once drugs have passed through Phase 1 testing, patients with terminal illnesses can apply for them.

The US Court of Appeals for the District of Columbia heard the case, and ruled in favor of Abigail Alliance. The FDA requested a rehearing of the case. The court reheated the case “en banc” and ruled in favor of Von Eschenbach. The Supreme Court declined to hear the case, and thus, the case was resolved with the FDA on top.

Patients continued to be without access until 2014 when Colorado passed a “Right-to-Try” bill. This bill allowed terminally ill patients to access experimental drugs that had passed through Phase 1. In addition, permission from the FDA is not required to obtain the drugs. Other states introduced similar laws, with 41 states in total passing laws similar to the first bill in Colorado.

This brings us back to the beginning, with the federal “Right-To-Try” bill. However, there is an important question. Why so long? Ever since the FDA’s drug mandate in 1938, patients with no chance at life had been denied access to the drugs that could save them? I don’t mean to be hyperbolic, but how much blood does the FDA have on their hands?

Even accepting the current framework of the FDA, how could one argue that terminally ill patients don’t have a right to try treatments, even if the government has not approved them?

Saving them from any potential harm to themselves is clearly pointless. A worst-case scenario gives the same result as doing nothing at all.

The FDA may have had good intentions in keeping access to experimental drugs limited. It may have thought it was doing what was best for the patients. But what do you say to Abigail Burroughs? What about every other patient who could have lived, but were effectively sentenced to endure their fate? The history of your “Right-to-Choose” is one of warning. A warning for the results of government control over the pharmaceutical industry and its potential to save lives.