Individuals dying of cancer or other diseases should be able to try unproven new treatments that might save their lives without interference from the Food and Drug Administration. That’s the gist of state “right-to-try” laws that may soon get federal support. While these laws tap into human compassion, they actually undermine patient protection and the ability to determine how well these treatments work.
The right-to-try message has met with some success, owing in part to the work of the Goldwater Institute, a libertarian think tank that has developed model legislation and lobbied successfully for passage in more than 30 states — including Indiana, where Vice President Pence signed a right-to-try law in 2015 when he was governor of the state.
Pence met last month with a group of patients and advocates to signal his support for federal right-to-try legislation. The focus of a years-long advocacy campaign, this proposed law, now being considered by both the House and Senate, would prevent the federal government from blocking state-level right-to-try laws, which allow terminally ill patients to bypass the FDA’s existing pathways for accessing experimental therapies.
To date, there is no credible evidence that these laws have allowed a single patient to access an experimental therapy. That’s partly because clinical research is governed by federal law, which preempts state law. In addition, terminally ill patients already have two pathways for accessing experimental therapies — they can join a clinical trial, or a physician can apply to the FDA for so-called expanded access (compassionate use), which allows access to experimental therapies for treatment purposes.
Between January 2005 and December 2014, the FDA received 8,922 new applications for expanded access, and approved 99.3 percent of them. This review is not simply a rubber stamp — the agency often provides important feedback on dosing, safety monitoring, and informed consent documentation.
Supporters of right-to-try laws argue that the FDA’s application process is too complex and burdensome, discouraging patients and their physicians from applying. While that may have once been true, the agency has simplified its application and developed guidance for physicians and patients. In emergency situations, approvals can even be granted over the phone, if a formal application is submitted within 15 days.
However, the FDA cannot compel a pharmaceutical company to supply an experimental therapy to a patient, and many may decline because of concerns over liability, additional costs, limited supply, or fears that providing the drug via the right-to-try pathway would undermine their ongoing drug development program and jeopardize formal approval.
In addition to letting patients bypass FDA review in right-to-try states, the proposed federal bill exempts physicians and drug companies from any liability related to the prescription or dispensing of these unapproved therapies. It also prohibits the FDA from considering the outcomes for right-to-try patients in its review when deciding whether to approve or reject the therapy. The bill doesn’t offer any additional support or safeguards for patients. A therapy need only to have passed basic safety testing in a small number of healthy patients, which does not provide information on effectiveness. And the bill does not require insurance companies to cover the drugs or any associated costs.
In other words, this bill strips dying patients of the protections afforded by FDA oversight, sets no requirement that the drug show promise for treating their disease, impedes their ability to seek redress for any harm, and forces the FDA to ignore scientific data on the effects of the drug among right-to-try patients — data that would be useful for determining its safety and efficacy. The ethical problems posed by this bill are substantial, not the least of which are the false hope it offers desperate patients.
Efforts are currently underway to improve the expanded access process. Under the 21st Century Cures Act, companies will be required to publish details on their expanded access policies and procedures. This is an important step, as most companies do not currently do this, leaving doctors and patients with little guidance.
The best way to get new drugs to patients is to shorten the amount of time it takes to gain FDA approval. This requires us to think more broadly about how we can find efficiencies in the drug development process, such as through the use of innovative trial designs, better patient recruitment strategies, and new mobile technologies that can collect data more efficiently and at lower cost. The FDA is already tackling many of these issues as part of its obligations under 21st Century Cures.
Congress should ensure that the FDA has the resources to achieve these mandates, rather than undermine its ability to protect patients and the public health by passing the right-to-try proposal.
Elizabeth Richardson is a managing associate at Duke University’s Robert J. Margolis, MD, Center for Health Policy.