[1]pic thx to british red cross flickr creative commons
On Sunday, California Governor Jerry Brown vetoed a bill [2] that would have expanded access to experimental drugs — bucking a trend that is sweeping the country. These “Right to Try” laws, so far enacted by 24 states, allow people who are battling fatal illnesses to gain access to unapproved medicines. They also directly defy federal regulations.
Under federal law, if terminally ill patients are not eligible to obtain an experimental therapy through a clinical trial, they can apply to the Food and Drug Administration for “compassionate use.” Although the agency approves the vast majority of such requests – more than 99 percent [3] of nearly 1,900 application last year, critics complain the program is arbitrary and cumbersome. And so the state laws seek to cut the FDA out of the process of green-lighting experimental drugs.
“The process is so complex that not enough people are able to get a promising drug they need,” said Starlee Coleman of the Goldwater Institute, a Libertarian think tank that helped draft state laws nationwide, including in Massachusetts [4]. The Goldwater Institute, which is based in Arizona, also worked with three federal lawmakers to introduce a national version of the legislation [5] in the House last July.
These efforts, however, appear misguided.
The laws threaten to unravel decades of patient protections, since the FDA is precluded from providing its traditional oversight. And the laws could even make it more difficult to obtain experimental drugs because of requirements not found in the existing federal program. In some states, for instance, more than one physician diagnosis is needed for drug access. Certain state laws also demand that written consent forms carry added stipulations that may need legal review.
Insurers, meanwhile, are not required to pay for treatment, yet most of the states failed to create funding to help patients cover medical expenses. Plus, there are unanswered legal questions about whether state laws trump federal regulations.
“If the intent of Right to Try is to streamline access and lower costs, it’s not really working,” said David Vulcano, who’s in charge of clinical research at the Hospital Corporation of America, which operates 280 hospitals and surgery centers nationwide.
It’s not even clear if anyone has really been helped by the measures. Coleman could point to only one doctor who she believed was providing treatment under a state Right to Try law, but she would not provide details.
“The laws may have started a national conversation about right to access,” said Alison Bateman-House, a bioethicist and public health researcher at the New York University Langone Medical Center. “But there’s no evidence that anyone has benefited.”
The frustration with the FDA is also misplaced. Compassionate use applications certainly take time and effort. And given the momentum behind these laws, one could be excused for thinking the desire to marginalize the FDA is warranted. But between 2010 and 2014, the agency turned down only 33 requests and approved 5,995 [6]. And in dire circumstances, approvals were often made in a matter of days.
This is a good track record, yes? So, what accounts for the logjams that spark patient anger and a growing number of social media campaigns to win experimental medicines?
The FDA lays the blame on the drug makers.
Here’s why: Even before patients apply to the FDA for compassionate use, they must first persuade drug makers to provide their medicines at cost, meaning for no more than the price of manufacturing. So there is little financial incentive for companies to comply with a request. In fact, doing so can jeopardize their bottom lines — either because a drug may be in short supply or, worse, someone who receives an experimental treatment experiences a side effect, which can damage the chances of a company winning FDA approval.
“It can slow down or even ruin the drug development process,” said Kenneth Moch, head of Euclidian Life Science Advisors and former chief executive at Chimerix, which caused an outcry last year [7] when the company initially denied an experimental drug to a young boy who was dying of cancer.
The Right to Try laws do not make such concerns disappear. Instead, drug makers that make their experimental medicines available under a state law may face a quandary, because doing so could put them in direct violation of the very agency they need to approve their products. The House bill under consideration is designed to alleviate this concern. But alienating the FDA is not a smart strategy.
Indeed, all 50 states could pass such a law, but desperate patients are unlikely to be helped. To actually put experimental drugs into the hands of patients, other solutions are needed. One might be for the FDA and drug makers to work more closely in devising workarounds so that a patient can receive an experimental drug without the risk of undermining the integrity of ongoing clinical trials.
Finding new ways to help terminally ill patients is a worthy goal. But marginalizing the FDA and removing a needed degree of oversight is not a remedy.
Editor’s note: This first appeared yesterday as the weekly Pharmalot column [8] in The Boston Globe.