Dr. Siddhartha Mukherjee’s, author of the
book, The Emperor of all Maladies: A Biography of Cancer in the PBS Documentary
said, “Medicine being the most humane
science…sometimes scientists are caught doing inhumane things which is denying
experimental drugs. However, putting a
drug on the market too early has had historically devastating events.”
What
Mukherjee is referring to is the consequences of releasing an experimental drug
or treatment via Accerlated Access before Phase 3 clinical trials have been
undertaken. An example of this is the
STAMP treatment for breast cancer in the 1980s and 1990s.
In 1983, Dr.
Peters and Dr. Frei started breast cancer clinical trials on a new therapy
previously shown to have some success at treating childhood leukemia, known as
the Solid Tumor Autologous Marrow Program (STAMP). STAMP involved extracting bone marrow from
the participant and freezing it, then wiping out their cancer with extremely high
doses of chemotherapy, and injecting the bone marrow back into the person so
that the high-dose chemotherapy wouldn’t kill them. Promising success in Phase 1 and 2 clinical
trials with breast cancer patients encouraged women who could not participate
in the trials to seek accelerated approval of the treatment. Soon 30,000 some women received access to the
experimental therapy between 1985 and 1998 at the cost of millions of dollars. The study could not recruit people for the
Phase 3 clinical trials because no one wanted to be in the control group. About
1 in 5 women died from the treatment and it was later shown that the higher
dose of chemotherapy was not any more successful than the lower doses in improving
women’s survival. And in fact there
was a higher chance of death due to the bone marrow implants. Those who did survive the procedure suffered
long term cognitive problems such as short term memory loss.
Some of you might still be asking yourselves,
so what’s the big deal? The women at
least got a chance to try something? The big deal is that
an experimental treatment that has not gone through the full round of required
clinical trials carries a higher risk of actually shortening the person’s life
if not killing them. Perhaps someone who
is near the tail end of their disease is willing to take this risk, but what if
you are someone who is early on in their disease? By foregoing clinical trials you risk
shortened lifespan, death, and the right to try other treatments that might
become available. That’s right, clinical
trials have strict inclusion and exclusion criteria. Meaning, in order for the researchers to know
without a doubt that their treatment is having an effect, they cannot allow
anyone into the trial that has taken other experimental drugs. Moreover, what if a drug is approved for
Accelerated Access and a large number of people start taking the drug and
suddenly have major toxic reactions or die.
Even if this is unrelated to the treatment itself, such an event might
trigger the FDA to remove the treatment from the market altogether…that’s
it. A possible beneficial treatment to
some people with ALS will never reach them.
I’m not
saying we should not seek treatments through Accelerated Access period. I’m
simply saying there is a time and place.
The U.S. FDA Evaluation team is in place to insure consumer safety. If companies can satisfy their requirements
that show scientifically that their treatment warrants Accelerated Access based
on evidence that is safe and efficacious than this is a good thing. If not, we should consider other options for
treatments outside clinical trials such as Widespread and Group Expanded
Access, which is politically more kosher with the U.S. FDA and doesn’t hinder
the clinical trial process.
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