October 3, 2019
By Naomi Lopez
A diagnosis of glioblastoma can seem like a death sentence: It’s an aggressive form of brain cancer with a five-year survival rate of only about 5 percent. But now, patients with this aggressive form of brain cancer may have access to a treatment previously unavailable to them—thanks to the federal Right to Try law, a policy created by the Goldwater Institute: Epitopoietic Research Corporation (ERC) has become the first drug manufacturer to announce a formal program to treat patients under Right to Try. This is not only great news for terminal patients—it also points to a more positive future for treatment innovations.
ERC is the manufacturer of ERC1671 (also known as Gliovac), a vaccine to treat patients with glioblastoma. According to the
ERC website, the vaccine uses “freshly extracted tumor cells” to “stimulate[]
the patient’s immune system to recognize and reject cancer cells.” ERC has
announced that this treatment is being made available, at cost in compliance
with the federal law, to patients who meet the Right to Try criteria, as well
as treatment protocols. (You can read the full version of ERC’s recently
released Right to Try policy here.) According to an ERC spokesman, the company is prepared to treat
all patients who are eligible over the next year under the Right to Try law. ERC has treated
about six patients this year.
Under
Right to Try, patients who are terminal or facing life-threatening illness and
who have exhausted other treatment options may, with their physician’s
approval, seek investigational treatments that are safe enough to be used in
clinical trials but remain under clinical evaluation for final U.S. Food &
Drug Administration (FDA) approval. Signed into law in
May 2018 following a bipartisan grassroots
effort across the United States, the federal
Right to Try Act marks a significant step forward—and does not sacrifice
existing safety protocols—because the FDA’s existing “compassionate use” program bars untold numbers of terminal patients
at the door, thanks to an overly complex application process that can take a physician dozens of hours
to navigate and complete. Right to Try works in tandem
with the FDA’s drug approval process while providing some additional liability
protections and clearly states when and how the FDA can use adverse event data
is why there is now an uptick in treatments offered through this pathway.
ERC’s
program is the first-known Right to Try program in the country, but there will
likely be many more in the next couple of years. Take, for example, Beacon of Hope. Former pharmaceutical executive Richard Garr, who is now CEO of
Beacon of Hope, has announced that his organization will be running a Right to
Try program on behalf of an ALS treatment manufacturer. ALS, commonly known as
Lou Gehrig’s disease, is a progressive neurodegenerative disease which destroys
the patient’s nerves in the brain and spinal cord, robbing the patient of
muscle and motor functions including walking, speaking, swallowing and,
eventually, breathing. According to Garr, the ALS
Right to Try program is expected to treat between 200 and 300 patients over the
next year.
Right to Try marks a
new day in moving toward the goal of bringing the right treatment, to the right
patient, and at the right time. The treatments allowed
under the Right to Try Act are the same
treatments available to patients with the same disease who are lucky enough to
qualify for a clinical trial. By removing the federal red tape, Right to Try is
starting to make more treatments accessible for those patients whose lives hang
in the balance.
Right to Try’s most
important contribution is that it recognizes and protects the fundamental principle
of patient autonomy. Indeed, the law explicitly intends to “expand[] the scope of individual
liberty and agency among patients, in limited circumstances.” It does this by
recognizing that these deeply personal decisions belong in the hands of
patients and their physicians.
Reforms are speeding
up access, recognizing that these important decisions rightfully belong with
patients whose doctors are recommending these treatments. These types of
reforms did not start with Right to Try—nor will they end here. We are in the midst of a rapid period of medical innovation that
holds the promise of delivering personalized treatments and curing diseases
that, just a few short years ago, were death sentences. And there is a growing
realization that today’s FDA, which was established in its current form in the
1960s, is not keeping pace with the innovations now taking place.
Sadly, many glioblastoma
patients are now gone, having run out of time and treatment options. But Right
to Try, coupled with the growing recognition that there are treatments in the
clinical pipeline that can save lives, is an important first step in getting
the right treatment to patients when their lives hang in the balance.
Naomi Lopez is the director of healthcare policy at the Goldwater Institute.
