Is Right-to-Try Legislation a Bust? Time for a Second Opinion.

In 2001, twenty-one-year-old Abigail Burroughs was dying of cancer. After all conventional treatment methods failed to improve her condition, Abigail’s oncologist pleaded with the Food and Drug Administration to allow her to try Erbitux. At the time, Erbitux had not fully passed the FDA’s drug approval process. Abigail was denied access and lost her battle to cancer shortly after. The FDA eventually approved Erbitux in 2004 to treat the same cancer which cost Abigail her life.

Heartbreaking stories like Abigail’s (and thousands of others) provoked a country-wide movement to allow terminally ill patients the right to try experimental treatments to prolong their lives. This movement became the impetus for right-to-try legislation. Right-to-try legislation grants patients with terminal illnesses access to potentially lifesaving drugs before the FDA fully approves them. By side-stepping the FDA’s formal approval process, decisions to try unproven, although potentially beneficial, treatments are left to patients, physicians, and drug producers.

The movement spread quickly, becoming “law of the land” for many states. By 2017, forty-one states passed right-to-try laws. Then, in May 2018, President Trump signed right-to-try legislation into national law, allowing terminal patients across the country to access experimental medication without requiring permission from the FDA.

But legislation which curbs government power is rarely met without resistance. In route to becoming national law, right-to-try endured considerable criticism. It was called a “cruel joke,” a “sham,” and “false hope,” among other defamations. Others claimed the law was “tailored to undermine public health” and that it “wasn’t worth passing.”

National right-to-try legislation passed nearly a year and a half ago. Have its critics quieted? Hardly. Much of the right-to-try critics’ rhetoric remains the same. Now, however, they have added “failure” to their list smears.

A recent piece published in the Los Angeles Times claims the law has been a “bust” and “a cynical ploy aimed at emasculating the Food and Drug Administration in a way that would undermine public health and harm all patients.” Others echo similar sentiments.

Why is right-to-try a bust? The column’s author and many other critics hold that, because noticeably few terminally ill patients have used the right-to-try process, it has failed those it intended to help. In his own words:

Right-to-try advocates claim that two patients have benefited from federal law thus far. But the claim doesn’t hold water. In one case involving an anonymous patient suffering from glioblastoma… turns out that both the FDA and the institutional review board of UC Irvine [where treatment occurred] were involved in oversight. According to Caplan and two colleagues who reviewed the case in January, providing the patient with the drug, Gliovec, took longer than it would have under existing FDA procedures.

It seems the Los Angeles Times columnist is confused over what right-to-try legislation offers. The law provides terminally ill patients the option to try experimental medication without requiring the FDA’s approval. Drug providers may decide to involve the FDA for a variety of reasons. However, this is entirely different from requiring the FDA’s approval to administer treatment. Similarly, UC Irvine is where the patient received treatment. Involving the university’s review board hardly seems to undermine right-to-try legislation.

The column’s author also prematurely concludes that treatment under right-to-try “took longer than it did under existing FDA procedures.” By existing FDA procedures, the author means the FDA’s Expanded Access Program, which also grants terminally ill patients access to experimental treatments. The author is correct that the FDA often quickly approves applications for expanded access. He fails to mention how involved and time-consuming applying for expanded access can be. Right-to-try allows those seeking treatment to avoid this application process, saving them precious time. As the lead medical investigator caring for the patient explained in a statement, “It was believed that (Right to Try) offered a more expedited path to treatment, which UCI began after meeting regulatory and compliance requirements of state and federal Right to Try laws.”

The author continues:

A second case involves Matthew Bellina, a victim of amyotrophic lateral sclerosis or Lou Gehrig’s disease, who was treated with a drug called NurOwn by manufacturer Brainstorm under the right-to-try law. But the drug hasn’t been shown to be significantly more effective than a placebo, and Brainstorm says it’s not giving the drug to any more patients under right-to-try but would continue testing the drug via an FDA-approved Phase 3 trial, which it called “the best and most credible pathway” to establishing the safety and efficacy of NurOwn for ALS.

The author omits that the reason Brainstorm is no longer offering NurOwn is because of their financial limitations, not because of the right-to-try process. His claim that NurOwn is no more effective than a placebo is incorrect. Recently, the FDA approved NurOwn for fast-track status, allowing it to advance to full approval more quickly due to demonstrated effectiveness.

We should also recall the inspiring story of Natalie Harp, who gained access to experimental treatment under right-to-try laws to treat bone cancer. Several terminally ill patients also benefited from state-level right-to-try legislation before it became national. It is difficult to see how any of these stories constitute a “failure.”

I do agree with the author, however, that right-to-try is underused. Fortunately, efforts to expand its role in helping those with terminal illnesses are underway. However, until criticism of right-to-try is addressed, misconceptions and false allegations will continue to undermine efforts to help the terminally ill. It’s a cause worth defending, and I’ll gladly continue to do so.

Republished from Independent.org.