ith Congress back from its December recess, the House of Representatives is facing mounting pressure from multiple quarters, including Vice President Mike Pence and conservative groups funded by Charles and David Koch, to pass so-called “right-to-try” legislation that would create a dangerous, uncharted pathway for patients to access experimental medications. Last summer, the Senate passed such legislation — which my organization, Public Citizen, and other patient safety advocates have dubbed the “False Hope Act.” In the House, however, the bill has so far has encountered surprisingly strong headwinds.
As a physician who has cared for critically ill patients, I understand the desire to access experimental medical products from those who have exhausted available treatment options. But as a former senior official at the U.S. Office for Human Research Protections and now as director of a research-based advocacy group that for more than four decades has worked to strengthen patient safety, I also have a deep appreciation of the essential role that federal requirements play in protecting vulnerable patients from harm and exploitation that may result from exposure to unproven experimental drugs.
Under federal law, the Food and Drug Administration oversees the use of all experimental drugs, biological products, and medical devices in the U.S. The agency’s long-standing expanded access program, also known as compassionate use, allows patients to gain access to such products, provided that each patient’s doctor believes such access is appropriate and that the manufacturer of the product agrees to provide it for that use.
To protect patients, the FDA and an ethics committee called an institutional review board must approve each use of an experimental medical product under the expanded access program. As conditions of approval, there must be sufficient evidence of the safety and effectiveness of the experimental product to support its use in a particular patient, and the probable risk to the patient from the product must not be greater than the probable risk from the patient’s disease or condition. The program further protects patients by requiring a robust informed consent process that is similar to the consent process for a clinical trial, as well as monitoring and reporting of serious adverse events.
The right-to-try movement took off in 2014 when states, at the behest of the libertarian Goldwater Institute, began to adopt laws that purportedly allowed terminally ill patients to receive experimental drugs without FDA oversight or compliance with the requirements of the expanded access program. While pushing state adoption of these laws, the Goldwater Institute asserted that terminally ill patients had a fundamental right to access experimental medical products. However, the U.S. Court of Appeals for the District of Columbia had correctly ruled in 2007 that patients have no right “to a potentially toxic drug with no proven benefit.”
Fortunately, the various state right-to-try laws were effectively moot because they were preempted by FDA laws and regulations. However, unlike state right-to-try laws, the federal false-hope legislation passed by the Senate which is now being considered by the House would effectively undermine the FDA’s safety rules regarding the use of experimental drugs outside the context of a clinical trial, putting countless patients at risk.
Proponents of the federal legislation posit that the FDA is preventing thousands, if not millions, of seriously ill patients from accessing a wide array of miracle cures. But this is a misleading narrative. First, the FDA grants more than 99 percent of all expanded access program requests and, in urgent circumstances, can respond to such requests within one or two days. The real hurdle to access to experimental drugs are pharmaceutical companies, which for a variety of often legitimate reasons frequently choose not to make their experimental drugs available to patients outside the context of well-designed and monitored clinical trials. Congress cannot force companies to do otherwise.
Moreover, under the proposed federal legislation, patients would be allowed access to experimental drugs so long as a single Phase 1 clinical trial of the product — the most preliminary of testing in humans — had been completed and development of the product in other clinical trials was ongoing. But data from a single Phase 1 trial would fail to provide sufficient evidence for allowing widespread use of an experimental drug outside the context of a clinical trial without any FDA oversight. Phase 1 clinical trials often enroll only healthy volunteers, typically involve testing of a single dose of the experimental drug, provide no meaningful data on a drug’s effectiveness, and yield only limited preliminary data on its safety. Indeed, a large proportion of experimental drugs that progress beyond Phase 1 testing are ultimately found to be unsafe or ineffective upon further clinical testing and are not approved by the FDA.
Thus, the right-to-try legislation now being considered by the House would expose countless vulnerable patients — many of whom would harbor false hopes of likely benefit — to drugs that have little probability of benefit but substantial risks of serious harm, including dying earlier and more painfully than they otherwise would have, without appropriate safeguards.
A 2017 review by the Government Accountability Office found that the FDA recently has taken step to streamline the expanded access program and improve patient access to experimental drugs. Patients would be better served if Congress developed legislation to further enhance, rather than undermine, the FDA’s expanded access program.
Michael A. Carome, M.D., is director of Public Citizen’s Health Research Group.