Federal Right to Try bills remove crucial patient safeguards
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In its deregulatory zeal, the Trump administration has endorsed federal legislation, called the Right to Try Act, that could harm terminally ill patients seeking access to experimental drugs by eliminating FDA oversight of the process. It is an endorsement of seriously misguided policy.

Removing FDA review would not only dismantle patient safeguards, it would deny patients the expertise and, importantly, the objectivity of the agency. Proponents of Right to Try legislation should instead work to improve the FDA’s Expanded Access Program, which has been effectively overseeing pre-approval access to medicines for decades. To do otherwise is to put politics above patient welfare. 

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The administration recently voiced support for “Right to Try” (RTT) advocates, after bills were introduced in the House and Senate that would put legislative force behind 33 state RTT laws. These laws claim to give terminally ill patients “compassionate” access to experimental drugs.

However, the laws do not operate as advertised: They do not provide access to unapproved drugs but merely the option of asking for such drugs from manufacturers, which are fully free to refuse. These laws, then, deceive by offering false hope of a right to access. They also discourage enrollment in clinical trials, which are central to establishing the safety and efficacy of products before they come on the market. 

If companies cannot enroll trials because potential participants are choosing right to try access instead, the development of an agent can be delayed or even derailed; subsequent market access to that drug for all appropriate patients could be jeopardized. Unsurprisingly, then, companies are reluctant to provide access to experimental products.

The federal bills were crafted to strengthen state laws, and, of special importance to proponents, to prevent the FDA from penalizing companies if bad outcomes result from the use of an unapproved drug. But the bills wouldn’t merely indemnify industry; they would entrench all the state law provisions, including those that are patient-hostile. Some states allow insurers to deny hospice or home healthcare benefits if a patient uses an unapproved drug. Some let them cancel coverage altogether. Equally alarming: Every law allows access to a product after just Phase 1 testing, in which a drug is tested on only a small number of people, thus producing virtually no information on how dying patients would tolerate it.

The Goldwater Institute, a political organization dedicated to “defending liberty,” spearheaded the Right to Try movement. It believes that patients, not government, should decide whether to try experimental drugs. But patients cannot make informed decisions if they don’t have adequate information. Phase 1 trials offer limited safety and zero efficacy data. How can a patient weigh risks and benefits without such information? And how can a physician in good conscience prescribe a treatment without knowing how it will work in a sick patient?

This is what FDA is for. It has the expertise to weigh the risks and benefits of even post-Phase 1 drugs for use in terminally ill patients. And it is not the roadblock to access that RTT proponents make it out to be: The agency approves more than 99 percent of requests for pre-approval access. And far from rubber-stamping applications, the agency found that 11 percent of requests required some adjustment to the proposed treatment protocols. These statistics clearly point to an established program that works.

At the same time the federal bills are eliminating patient safeguards, they are shielding from liability every actor in the chain of access, from manufacturer to prescriber. And they state that an adverse outcome due to the use of a product won’t impact approval of the product. It’s absurd that if an experimental drug killed patients, FDA reviewers would be powerless to respond in any manner.

Even with this liability shield, PhRMA, the trade association for pharmaceutical companies, opposes the legislation. Responding to President Trump’s support of the Right to Try Act, a spokesperson said, “Any legislation should protect … FDA oversight of expanded access to maintain the best interests of patients.”

The FDA is not perfect. It could better reassure companies that adverse events wouldn’t automatically threaten drug development. And it could establish a funding mechanism to help patients cover costs associated with pre-approval access. These would improve its Expanded Access Program without stripping it of its crucial patient oversight. Instead of promoting legislation that strives to remove patient protections in service to an ideological goal of small government, RTT advocates should promote programs that have been shown to work and are devoted to patient safety.

Kearns is a Senior Research Associate at NYU School of Medicine’s Division of Medical Ethics. Neveloff Dubler is a Consultant for Ethics at the New York City Health & Hospitals Corporation. Both are members of the Division of Medical Ethics’ Working Group on Compassionate Use and Pre-Approval Access.


The views expressed by this author are their own and are not the views of The Hill.