As Congress renegotiates the FDA Reauthorization Act, a must-pass piece of legislation that lets the Food and Drug Administration collect fees from pharmaceutical companies that submit their products for FDA review, Sen. Ron Johnson (R-Wis.) has threatened to hold up the bill unless the Senate adds a “right-to-try” amendment to the House version currently on the Senate calendar. Despite its benevolent intention to help patients without approved treatment options gain access to drugs in development, right-to-try legislation threatens the integrity of clinical trials, which remain the safest way for patients to try experimental drugs.
The money collected through the FDA Reauthorization Act funds approximately 70 percent of the Center for Drug Evaluation and Research, the FDA division that regulates prescription and over-the-counter drugs. This bill must pass in order for the agency to continue approving drugs for safety and efficacy. If the user-fee bill is not reauthorized by Sept. 30, thousands of FDA employees could lose their jobs.
A right-to-try amendment would tack onto this legislation a way for terminally ill patients to use investigational drugs outside of clinical trials and without FDA oversight. But, existing state right-to-try laws do not require that drug companies give patients exercising their “right” experimental drugs, and the policy allows companies to charge patients for them, even if they don’t work or could be fatal. The federal policy would be no different. Ironically, adding a right-to-try amendment to a bill that funds a large part of the FDA’s operations would theoretically allow patients to use drugs that aren’t yet shown to be safe or effective — the very reason the agency exists and requires user fees.
Although right-to-try language hasn’t yet been added to the FDA Reauthorization Act, an amendment called the Enhanced Clinical Trial Design Act has been added to the House version of the bill currently scheduled for consideration by the Senate. This positive bipartisan amendment, sponsored by Sen. Orrin Hatch (R-Utah), calls for a public meeting with the FDA and the National Institutes of Health to address potential barriers for patients who wish to participate in clinical trials. No more than three months after the meeting, the Department of Health and Human Services secretary must provide a publicly available report of the topics discussed at the meeting. Three months after that publication is available, the FDA would be required to release new draft guidance regarding eligibility criteria for clinical trials.
The Enhanced Clinical Trial Design Act is step toward removing barriers patients face when seeking clinical trial access, something that right to try does not address. But the amendment could be removed from the FDA Reauthorization Act as it moves through the legislative process — and right to try could be added instead.
Right to try and the Enhanced Clinical Trial Design Act represent a tension between individual health and population health. Patients without approved treatment options for their illnesses may not have the luxury of time to wait for new drugs to be approved by the FDA, and might — I stress the “might” — benefit from drugs that are being tested in clinical trials. Yet that diverts patients from participating in clinical trials, which may slow drugs’ paths toward regulatory approval. And slowing regulatory approval affects a much larger number of patients, since approval of a drug allows all patients who can afford it to benefit from it.
The clinical trial process exists for a reason: to measure the effectiveness and safety of new drugs before they are released into the market. While our health care systems must try to help individual patients who want access to potentially lifesaving treatments, they must also protect the public’s health and the interests of future patients. This requires a delicate balancing act. By circumventing clinical trials, right-to-try legislation disrupts a system that exists for good reason.
Patients who participate in clinical trials enter into a “contract” with the scientists conducting the study. The participant allows scientists to use his or her body to advance science for the benefit of other patients. In return, the participant hopes to reap a personal benefit — a cure, or at least improved health — from taking part in the trial. In addition to learning about the effectiveness of a new drug, clinical trials also capture data about adverse events, data that are crucial to deciding whether the drug is both effective and safe.
Not everyone who wants to take part in a clinical trial can do so. Those who are very sick are often excluded from trials, and the cost of traveling to the site of a trial may be too high. In addition, researchers are sometimes accused of cherry-picking patients for clinical trials, choosing the healthiest ones so they have the “cleanest” data possible. An unintended outcome of such selectivity is that a newly approved drug may not work as well in the general population among individuals with comorbidities and complications the same way it did among trial participants.
Expanding the population of people who can join clinical trials is important. Not only are the sickest individuals often denied spots in a clinical trial, but people of color, women, those who live in rural communities, and those without adequate insurance are vastly underrepresented in clinical trial populations. Their absence greatly diminishes the utility of the data gathered from these trials.
What happens to those who have an urgent medical need but who are shut out from accessing an experimental drug through a clinical trial? They aren’t condemned to die without trying to save their own lives. Such individuals can get pre-approval access to an investigational drug, also known as expanded access or compassionate use. But we don’t need new legislation for that. The FDA and drug companies have worked together since 1987 to give thousands of patients each year access to experimental drugs outside of clinical trials.
Useful new legislation would provide incentives to pharmaceutical companies that are reluctant to provide their drugs for legitimate pre-approval access requests. This would reassure companies that providing their investigational drugs to patients outside of clinical trials is not a risky proposition when done through the FDA’s existing program. The benefit of this program is that it can provide instruction from FDA scientists on matters like dosage and dose scheduling for physicians administering experimental drugs and thereby reduces the risk of adverse events. Right to try offers no such safeguards.
Scott Gottlieb, the new FDA commissioner, has expressed support for accelerated trial designs to bring drugs to market faster. Keeping the Enhanced Clinical Trial Design Act attached to the FDA Reauthorization Act is the first step toward addressing barriers to clinical trial participation; though we don’t yet know what the results the public meeting will produce, it opens a larger conversation on real solutions to help patients, like adaptive trial designs that allow sick patients the ability to participate in research. Right-to-try legislation won’t do that. To help those who still don’t qualify for a clinical trial, the FDA must strengthen its program for evaluating requests to use experimental drugs outside of clinical trials, and must develop a robust system to gather results from these attempts.
We need the FDA to ensure the effectiveness and safety of new drugs, and right to try undermines this process. And the agency certainly can’t work effectively if it is cut out of the process altogether.
Kelly McBride Folkers is a research associate in the Division of Medical Ethics at the NYU School of Medicine.