The House of Representatives passed a controversial bill Tuesday night that would allow patients with life-threatening illnesses to try experimental treatments while sidestepping federal regulators.
Senate Bill 204 – dubbed the Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act and named after patients – passed in the House with 250 votes in favor and 169 against and will go to President Trump, who has strongly supported so-called right-to-try legislation.
The newly-passed bill is a departure from an earlier right-to-try bill that the House passed in March. That version included more safeguards, including greater Food and Drug Administration oversight, stronger reporting requirements and a narrower definition of who is eligible for right-to-try – measures that critics said are stripped from the current version.
The Goldwater Institute, a Phoenix-based libertarian think tank backed by Charles and David Koch, heralded the bill as a win for patients.
“Millions of Americans who have been told they are out of options and that it’s time to get their affairs in order are closer to having the opportunity for one last treatment, without having to get permission from the federal government first,” read a statement from the group’s president, Victor Riches.
The Goldwater Institute did not respond to a request for additional comment.
Critics have pointed out that existing programs, such as the FDA’s expanded access program, already address the needs that right-to-try laws are meant to while providing necessary regulatory and institutional review board oversight. The danger of right-to-try legislation is that it potentially exposes patients to dangerous side effects while removing the FDA – along with its mission to protect public health and its ability to consult on important matters like dosing and route of administration – from the equation, critics say.
In a letter Monday to Reps. Paul Ryan, R-Wisconsin, and Nancy Pelosi, D-California, several dozen patient advocacy and medical research organizations pointed out that the FDA approves 99 percent of expanded access requests, while it is often drug companies that deny access. Friends of Cancer Research Chair Ellen Sigal wrote in an article earlier this year that the agency reviews most expanded access requests within 24 hours.
“FDA’s expanded access program, though imperfect, facilitates access to investigational therapies for over a thousand patients facing serious and life-threatening conditions each year,” read the letter, signed by the American Cancer Society, the National Organization for Rare Diseases and others.
For its part, in a February 2016 article, the Goldwater Institute criticized expanded access programs as “so rigged with bureaucracy and disincentives that it is bound to fail in most cases.” The same article noted that filling out the form for expanded access can take physicians 100 hours or more, but linked to an article on the Regulatory Affairs Professionals Society website about a new form that reduced the application time to as little as 45 minutes.
Still, under the right-to-try laws, drug companies have no obligation to provide access to investigational therapies. Janssen, a division of Johnson & Johnson, has expressed a preference for continuing to use expanded access programs. While calling the legislation “well-intentioned,” Merck & Co. was reported also to view it negatively.
Indeed, drug companies have little incentive to honor right-to-try requests when their main priority is getting patients into clinical trials, said Alison Bateman-House, a professor of public health at New York University and critic of right-to-try legislation, in an online interview. Right-to-try could hinder trial recruitment, especially if a company does not put a policy in place stating that patients eligible for clinical trials are ineligible for expanded access or right-to-try, she said.
Furthermore, she added, the policy could make the FDA look askance at an investigational drug. “Since you will eventually (hopefully) take that drug to the FDA for an approval decision, you don’t want to risk the agency raising its eyebrows at your decision to give the drug out without its oversight,” she said.
Consequently, the sorts of companies most likely to take advantage of right-to-try laws, Bateman-House said, are “sketchy” companies and those like small start-ups that don’t understand all the trade-offs of the program.
One way to potentially dampen enthusiasm for right-to-try would be to improve access to clinical trials, Bateman-House said in an earlier phone interview. Trials tend to take place at academic centers in large cities during business hours, creating barriers to entry for people who live far away and must take time off work and travel. Providing compensation to patients for their time, energy and gas mileage would thus help people participate, she said. Additionally, she pointed to a study by the American Society of Clinical Oncology and Friends of Cancer Research that recommended loosening some of the eligibility requirements in trials that often keep patients out, such as brain metastases and HIV infection.