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.
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