Federal ‘Right to Try’ Bill: wrong on the law, and wrong for patients

On June 16, Jordan McLinn, a seven-year old boy who suffers from Duchenne muscular dystrophy, joined other patients, their advocates, and members of Congress at a rally on the Capitol lawn in support of a federal “right to try” bill. At first blush, the Trickett Wendler Right to Try Act (S. 2912) would grant McLinn and other dying patients an automatic right to pre-approval, or “compassionate,” access to experimental drugs that appear promising in early testing but that have not yet completed clinical trials and been approved by the FDA. Unfortunately, the new federal bill, like similar state laws, is both legally ineffective and ethically troubling. We urge Congress not to spend further time on this hollow bill and encourage lawmakers to work to find real solutions to desperate situations for patients.

As of June 23, 31 states had enacted these laws, and eight more are considering similar legislation. McLinn lives in Indiana, which passed a right to try law more than a year ago. Yet here he was in Washington, still fighting for early access to unapproved drugs.

{mosads}He is not the only one. While every right to try law says that terminally ill patients have the right to ask for access to experimental medications, the laws do not, and in fact cannot, require anyone or any entity — including drug companies — to acknowledge this “right” by mandating that they provide any medications. It is therefore not surprising that there has not been a single substantiated case of any patient receiving access to experimental therapies solely pursuant to a right to try law in the more than two years since the first law was enacted. The legislation only appears to be helping desperately ill people but in fact creates no new rights for them. Yet, as the rally attests, patients, understandably, believe this illusion.

Rather than help, right to try laws can actually harm patients. The Trickett Wendler bill reinforces state stipulations that permit access to experimental drugs after they have passed only Phase 1 trials. At this point in drug development, there is scant evidence about basic safety of the new substance and virtually no data that proves its efficacy in treating the targeted disease. Yet the right to try movement suggests that these early interventions are a magic pill, and that cures are being withheld from dying people solely because of government red-tape or for financial reasons. Even if there were such impediments, the implication that a drug that passes Phase 1 is a cure — and a cure that would not pose substantial risk of pain, injury, or earlier death — is unsupportable.

The federal bill ostensibly further facilitates access allowed by state laws. Put aside the fact that the legislation does not mandate insurance companies to cover costs associated with the experimental therapy or similarly compel any other entity likewise bound by federal regulation of drug development. And disregard the several potential constitutional challenges to the bill as currently written — including its provision restraining all branches of the federal government from taking any action whatsoever on an early access provision. The Trickett Wendler bill still does very little to give teeth to state laws or make them ethical. Instead, it focuses mainly on creating a more hospitable legal and regulatory environment for the companies and clinicians both by its elimination of patients’ right to sue them for injuries arising from an early access grant and its legally questionable attempts to interfere with the FDA’s performance of its statutory oversight duties.

One of the greatest harms caused by this and all right to try legislation is that patients and their advocates believe that these laws give them new rights that may help save their lives. They don’t, nor can they, and the false promise that they hold out is shameful.

We hope that Congress, in partnership with patients, advocates, and the many others committed to bringing treatments and cures to patients as quickly as possible, will not act in vain by spending more time on ineffective right to try legislation. This precious time should be spent on the meritorious (and challenging) task of proposing real solutions to improve aspects of drug development and health care delivery that make the need so vast.

Kearns is a research associate at NYU Langone Medical Center’s Division of Medical Ethics. Roxland is an associate at the Division. Both are members of NYULMC’s Working Group on Compassionate Use and Pre-Approval Access.