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Critics argue that the law could have negative effects on the clinical trial process.
The Senate today passed the Right to Try Act near-unanimously, joining 37 individual states that have already passed such legislation in the past 3 years. The bill, intended to change the protocol by which terminally ill patients may receive access to as-yet approved medications provided they have completed Phase 1 trials.
A debate on the extremely contentious topic took the stage days ago at MedCity CONVERGE in Philadelphia. Vying for the legislation was Christina Sandefur of the Goldwater Institute, which is behind the creation of the right-to-try laws. On the opposite side was Beth Roxland, JD, of New York University Langone Medical School. Both women are attorneys, and Pete Loftus of The Wall Street Journal moderated.
The two took feverish notes while the other was speaking, occasionally punctuated with sharp and suspect glances. Though conducted at a small regional conference, it took on the tenor and seriousness of a televised national debate between right and left, in which loaded, abstract concepts like “freedom” and “responsibility” clash against cited statistics.
It can take more than a decade and billions of dollars, Sandefur argued, for life-saving medications to make it from development and into the hands of patients. She called it an “inequitable” system, given that chemotherapy itself is a dangerous course of treatment, and yet traditional clinical trials allow healthy people to take potentially dangerous untested medicines. Dying patients, on the other hand, are not afforded such freedom, she argued.
“We’re really not talking about a medical or scientific issue,” she stressed, “We’re talking about who decides what risk is acceptable for a patient to take. Should it be the government, or should it be the patient and their doctor?” The FDA’s current compromise, expanded access through “compassionate use,” she cast as a convoluted and unfair system of government-issued permission slips. Throughout the debate, she characterized that process as one of bureaucratic begging.
Right-to-try, she said, allows patients and providers to work directly with their providers and pharmaceutical makers. She pointed to Ebrahim Delpassand, a Texas radiologist frequently cited in arguments for right-to-try as perhaps the most prolific user of what the bill grants. He has testified before Congress about how it granted him the ability to treat dozens of neuroendocrine patients with LU-177 octreotate. Sandefur said the law allowed Delpassand to “go back to being a doctor.”
“What it does is it reasserts a patient’s fundamental right, it says that a dying patient that’s out of all options should be able to make those choices for themselves, and shouldn’t have to beg the federal government for a compassionate use exception,” she said.
Roxland was formerly the chief medical ethicist for Johnson and Johnson as well as the State of New York, and representing multiple entities has given her multiple perspectives on the situation. She ceded that the ability to take care of one’s self is a fundamental right, but that right-to-try was a different animal.
“We’re talking about research, here,” she said. Indeed, Roxland stressed that clinical trials do take a very long time to conduct, but that the legislation at hand did nothing to remedy that, and might actually exacerbate the problem.
“I want to make sure we’re not conflating research, clinical trials, and medicine with the specifics of Right-to-try,” she said. She characterized the legislation as an attempt to move the FDA away from “compassionate use,” but that the FDA statistically has approved 99.7% of applications from patients to take advantage of expanded access, and in emergency cases the turnaround was typically 24 hours.
“I think there’s many ideas on the table that would actually encourage early access. It’s already effective now, and could be done better with certain kinds of incentives, including repurposing our ideas of real-world data to actually get drugs to the market faster. Right-to-try doesn’t only not do that, but it makes a lot of things worse, and harder,” she struck. She pushed back strongly against the idea that the FDA was the problem at hand.
Roxland pointed out that the state level laws have only been on the books for 3 years, at their earliest, and that the Delpassand story was anecdotal and was the only real use case that advocates for the legislation often provide.
These laws don’t inherently grant anybody an automatic right of access, she believes, because patients still need to request the drugs from the companies. She also finds the messaging of the right-to-try laws problematic, because so much of the current situation has more to do with the drug makers granting expanded access than the FDA allowing it. In the last decade, she says, the FDA granted over 10,000 protocols for early access, and many of which were for entire cohorts rather than single patients.
She believes there to be already existing viable ways for patients to get such treatments, and argued that debating right-to-try laws and characterizing the FDA as a burden may dissuade some patients from using already-viable means for accessing the treatments they seek. Roxland also expressed grave concerned that allowing patients to bypass the clinical trial process, the very system of testing and approving pharmaceuticals as the United States currently knows it could be “destroyed."
The two found rare common ground, however, on an FDA criticism. Sandefur noted that the FDA has not put in writing what exactly would happen if an adverse event occurred to a patient that they granted compassionate use to. Without that clarity, she said, companies would likely be more hesitant to release drugs to such patients for use.
Roxland expressed agreement on that front. In respect to such criticisms, the Senate form of the legislation passed today requires the FDA to receive notification of adverse events.