Sep. 12, 2016

Engelhard: Are 'right-to-try' laws good medicine or partisan politics?

The 11-year-old asked whether Pence would advocate for a national “right-to-try” law similar to the one passed in his state of Indiana in 2015. Right-to-try laws allow terminally ill patients to take experimental drugs before final Food and Drug Administration (FDA) approval. The Indiana-governor-turned-running-mate promised the lad that he would continue to support all right-to-try laws, apparently agreeing with similar language embedded earlier in the 2016 Republican platform.

Although more than half of states have passed right-to-try laws, some have resisted the trend, saying they make false claims, conflict with existing federal regulations, and have the potential to exploit patients. Gov. Jerry Brown (D) of California vetoed a right-to-try bill last October, disappointing many who had hoped for less-regulated access to unapproved therapies for the dying.

Why are these kinds of laws so controversial and so politically charged?

At the most basic level, right-to-try laws challenge federal regulations. Under federal law, if terminally ill patients are not eligible for an experimental therapy through a clinical trial, they can apply to the FDA for a “compassionate use” waiver. Officially known as the “Expanded Access Program,” the agency approves almost all requests — over 99 percent of nearly 1,900 applications last year — but the appeal can be cumbersome, time-consuming and take months for the FDA to act.

Tired of the FDA application rigmarole and frustrated by the wait for federal action, desperately ill patients and their families took to the halls of state legislative chambers to lobby for passage of right-to-try laws, a model first championed by the libertarian-leaning Goldwater Institute. The right-to-try movement quickly became a symbol of states’ rights and a response to an overly bureaucratic and bloated federal system. To date, nearly 30 states have enacted these laws.

However, despite the highly emotional appeal of right-to-try these laws appear to be lackluster in their performance. A survey conducted last year by Modern Healthcare found no evidence of any patient receiving an experimental therapy as a result of state right-to-try measures.

Few, in any, states have created the infrastructure to help doctors, who are required to make the request on behalf of their patients, obtain the unapproved medicines. The Goldwater Institute claims it is working with states to build the necessary regulatory structure to enable implementation of right-to-try, but as of now, there appears to be little to show for its actions other than anecdote.

Even if states do step up with ways to accommodate patients and their physicians who want to invoke right-to-try mechanisms, there are very real cost barriers. Health insurers do not typically cover treatments without FDA approval, and state right-to-try laws do not guarantee access to drugs or provide a funding mechanism. As a result, patients requesting an experimental treatment under their states’ right-to-try laws may find drugs unavailable and the cost prohibitive. In a Colorado case, ALS patients seeking an experimental stem cell therapy were told that the unproven procedure was still under active scientific investigation and would cost up to $100,000 per patient.

The scientific and the bioethics communities also find fault with state right-to-try laws. Scientists worry that state laws could suppress clinical trial participation, the gold standard of patient-oriented research. Since the 1960s, the FDA has mandated that medications go through three stages of rigorous clinical investigation before doctors can prescribe them. State right-to-try laws allow requests for any therapeutic agent that has completed only the first phase of the FDA’s clinical trial process. Since Phase 1 trials establish safety but not efficacy, clinical researchers are concerned that less savvy patients seeking experimental drugs may falsely believe that access alone connotes efficacy. This wrong perception, in turn, could dissuade patients from participating in clinical trials where they could be assigned a placebo.

Likewise, bioethics experts fear that right-to-try laws make false promises to despairing patients. The laws purport to provide information regarding the best and worst outcomes of potential treatments — an unfounded claim since outcomes of unapproved therapies are generally unknown. By conflating experimentation with treatment, medical ethicists fear vulnerable patients may be unable to give true informed consent.

Is there a middle ground in this federal-state law debate? Perhaps. As we head into the fall presidential campaign, it turns out the politics surrounding state right-to-try laws may have put enough pressure on the FDA to streamline the application process for their compassionate use program. In June, the agency announced changes to “reduce the procedural burdens on physicians and patients,” and promised that the time required to fill out applications for putting experimental drugs into the hands of terminally ill patients would be reduced to 45 minutes and approved within a matter of days.

Although cost issues remain, the FDA program continues to offer standardized regulatory oversight and consumer protections that some state right-to-try laws do not, such as posting on the FDA website which medications are available from drugmakers for compassionate use and ensuring the continued right to hospice and home health after undertaking experimental treatments.

In the meantime, advocates of state right-to-try laws are continuing the crusade to expand their use. GOP lawmakers have introduced legislation in both chambers that would prevent federal action to block state right-to-try laws and politicians on the campaign trail are embracing them. Whether this is good public policy, ideological snake oil or partisan politics exploiting terminally ill patients is the question we should all be asking our elected representatives.

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Associate Professor of Public Health Sciences and Public Policy
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