Medical Examiner

“Right to Try” Is Merely “Thoughts and Prayers” for the Terminally Ill

The House is set to pass the meaningless legislation this week.

Pills and grave.
Photo illustration by Natalie Matthews-Ramo. Photos by Thinkstock.

The House of Representatives is expected to pass a federal “right to try” bill this week, a piece of legislation that will ostensibly allow terminally ill patients access to experimental medicines. It’s a favorite of President Trump, Vice President Pence, and the Koch brothers, but it shouldn’t pass because it won’t deliver on its promise. Rather, it would accomplish what it was intended to do the whole time—diminish the authority of the FDA, a move that four former FDA commissioners who served under Republican and Democratic administrations agree would endanger vulnerable patients.

The consumer advocacy organization Public Citizen has asserted that right to try laws offer patients “false hope.” While supporters of the bill may claim that there is no such thing as false hope, right-to-try laws are precisely that: They represent compassionate sentiment with no viable policy mechanism to help anyone. They’re basically the health care policy equivalent of the “thoughts and prayers” politicians proffer after instances of gun violence.

At first glance, “right to try” legislation seems like a commonsensical, compassionate policy. After all, everyone, regardless of where they fall on the political spectrum, can agree that it’s necessary to improve the lives of the dying. As opponents of this bill, we are often asked, “How can you possibly argue that dying patients shouldn’t have the right to save their own lives?” We don’t, because of course they should. We agree that terminally ill patients without approved treatment or clinical trial options should have every tool at their disposal to seek access to investigational products not yet approved by the FDA. Right to try, however, does not do anything to accomplish this. Instead, the House bill would allow terminally ill patients who have exhausted all other treatment options to try experimental drugs that have not yet been approved without getting permission from the FDA first. Under current regulations, the agency reviews these requests to ensure that the benefits outweigh the risks and, in some cases, the FDA provides the patient’s physician with valuable information on how to safely administer a drug for which there is minimal safety and efficacy data in human patients. The new legislation would provide a path to bypass this.

There are 38 states that have enacted versions of right to try laws since 2014, and all have been a grave disappointment. No right to try law has made an experimental drug available that was not already accessible through the FDA’s existing expanded access program. And the cruel sham of the “right to try” approach is that, while it targets the FDA, it’s actually drug companies that control who can get their investigational medicines. Even if the legislation completely eliminated the FDA tomorrow, that would not make it easier for a single dying person to get a drug, device, or vaccine that may save their life.

How, then, do companies currently decide whether to give their drugs to patients with no other possible treatment options? If they are tiny startups, they probably just say no, since they have no resources to divert away from their essential business development. Big companies have many reasons to say no, too, including limited supplies of experimental drugs and worries about what Wall Street will think if an adverse event in someone who asked for the treatment is made public. And because experimental medicines are not covered by insurance, only patients who are able to pay for them can take advantage of this “right” to try them—something the right to try law does nothing to fix.

Despite the current hurdles, thousands of Americans get access to investigational medicines from drug companies each year. Supporters of “right to try” continually distort data to claim that only around 1,000 Americans get access to investigational drugs per year. This wrongly conflates the number of requests to treat patients with experimental drugs that the FDA receives from doctors with the number of individual patients that receive access to an experimental product through these requests, though. While most expanded access applications are for individual patients, some are for larger groups accounting for hundreds—even thousands—of patients.

The only way to help dying patients get more access to experimental medicines is to work with the pharmaceutical industry to identify and adjust the push and pull factors that will make companies more willing to provide their products. And because we don’t want a situation in which only the wealthy or famous are able to get access, we also need to work with industry to ensure access to investigational treatments is just, fair, and equitable for all.

The first step toward improving expanded access is education. The FDA allows over 99 percent of expanded access requests it receives to proceed and has worked to simplify the process to apply; these are crucial facts that government officials should make known. The president, the secretary of health and human services, and the FDA commissioner should use their pulpits to educate patients, physicians, and pharmaceutical innovators about the current reality of how expanded access works, an area already rife with confusion.

Even if certain companies wish to participate in expanded access, some of them—particularly small ones—may be unable to provide their experimental products to patients. There are other factors at play, but FDA Commissioner Scott Gottlieb has stated that the biggest obstacle is supply constraints of available experimental medicines. Legislators and the industry must work together to make expanded access financially feasible for companies, perhaps by incentivizing pharma and biotech startups with tax breaks for providing their products-in-development to terminally ill patients who might benefit from them.

In addition to making expanded access appealing for industry, legislators must ensure that companies handle expanded access requests with justice and equity for all Americans. Right to try supporters have claimed that only the wealthy and well-connected can get access to experimental drugs through the current system. On the House floor on Tuesday, Texas Rep. Michael Burgess, the chairman of the House Energy and Commerce Subcommittee on Health, cited a news story from 2010 detailing how House Minority Leader Nancy Pelosi “pulled strings” to obtain an experimental cancer drug for a wealthy Democratic donor. Burgess is right to point out that expanded access requests often favor those who can game the system, particularly through successful social media campaigns that pressure companies to provide a drug. But the proposed law does nothing to fix these concerns. It doesn’t include a mechanism that requires companies to evaluate each expanded access request with the same considerations for every patient, which must be included in successful expanded access programs.

To safeguard equity, legislators should require that company employees tasked with making expanded access decisions anonymize certain patient information that may bias a decision on a request, including income, nationality, sex, race, and celebrity status. We know from experience that blinding expanded access requests aids in successfully getting experimental medicines to patients, thanks to our work on the Compassionate Use Advisory Committees (CompACs). The program deploys independently appointed physicians, bioethicists, and patient advocates to help pharmaceutical companies decide how to provide experimental medicines promptly and fairly to patients who may be helped by them. The first CompAC pilot project recommended approving 180 physician requests for individual patients to receive an investigational medicine —far more than any state right to try law has provided.

In our many years of experience with access to investigational medicines, we have found that right to try is nothing more than feel-good legislating at its finest. At best, right to try does not help patients. At worst, it could cause them serious harm and decrease patient access to investigational drugs. And at its heart, the right to try approach attacks the FDA while preserving the current pharmaceutical industry status quo. The sentiment behind right to try is commendable, but patients deserve a law that truly works for—not against—their interests.