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.