Navy Officer Fights for Right to Try Unproven Drugs Before DyingBy
Terminally ill patients want easier access to novel therapies
‘This bill makes it a lot easier to say yes:’ patient advocate
Matt Bellina was a 30-year-old officer in the U.S. Navy flying out of the Pacific Northwest to protect the country when he started showing symptoms of the deadly Lou Gehrig’s disease in 2012. Now he’s taken on a new fight: a law making it easier for terminally ill patients to get access to experimental drugs.
He’s getting closer to his goal, with the House now the last stop for the effort known as Right to Try after the Senate unanimously passed similar legislation in August.
“I need to know before I die that if my children find themselves in this unenviable position, that this nation that I proudly served will respect their liberties and their right to make their own decisions about their medical treatments,” Bellina, a father of three boys, said Tuesday at a health subcommittee hearing at the House Energy and Commerce Committee.
On the surface, Right to Try is a no-brainer. President Donald Trump and Vice President Mike Pence have voiced support, as have many Democrats, and a federal law would reinforce legislation already passed in 37 states and under consideration in the remaining 13.
But it’s more complicated than that.
Critics including drugmakers, researchers and ethicists say tending the urgent needs of one person, no matter how compelling, can lead to greater harm and slow development of medicines for thousands of others. They say there’s already a system in place, called compassionate use, that gives access to experimental drugs after an independent reviewer and the U.S. Food and Drug Administration have signed off. Under the Right to Try bills, the entire process takes place between the patient, the doctor and the company, leaving out all others.
The FDA commissioner said at the House hearing Tuesday that legislation should be used narrowly, for patients expected to die within months -- which isn’t what people with progressive diseases like Bellina’s are pushing for.
And a federal law isn’t going to improve the approval rate of requests for compassionate use, which already stands at 99 percent, FDA Commissioner Scott Gottlieb said. The main hurdle to get to the treatments isn’t the regulator, said Gottlieb, who was treated himself for cancer with a drug therapy that wasn’t approved for his condition.
“There is a perception that there are certain companies and products that aren’t necessarily being offered under the current construct, and the Right to Try legislation might provide more of an opportunity for companies to offer products in a different setting,” the commissioner said. “The biggest obstacle to offering drugs through expanded access is supply constraints.”
Neither the FDA nor lawmakers can compel companies to make their experimental products available, not under the existing compassionate use framework and not under Right to Try bills. To critics like Ira Loss, an analyst who has been tracking the FDA for four decades at Washington Analysis, that renders any law useless.
“This is an unfortunate effort that will only lead to disappointment in the end for those who try to take advantage of it,” Loss said in a phone interview. “It’s not necessary in my view.”
PhRMA, the pharmaceutical industry’s trade association, hasn’t taken a formal position on the matter in the past and declined to comment after the hearing.
For drugmakers, Right to Try can be fraught with risks: Patients who are already terminally ill may suffer complications or die after taking their experimental medicine, potentially jeopardizing clinical studies for other patients. Consequences could be devastating for biotechnology startups, the center of some of the most daring medical innovation, as they have a limited supply of their novel therapies.
Opponents favor improving existing programs such as compassionate use. The FDA took such steps Tuesday, with measures that may make manufacturers more willing to provide drugs. The agency will allow companies not to publicly report side effects and serious adverse reactions for compassionate-use patients if there’s no evidence suggesting the drugs caused the problem. The goal is to ease concerns that complications arising from compassionate use put development of the medicines on hold -- and scare away investors.
Very Few Treatments
There’s no timetable scheduled yet for a House vote on the two Right to Try proposals under consideration. The two bills -- one for drugs and the other for both medicines and medical devices -- allow terminally ill patients to use unapproved products without the permission of the FDA and without any ramifications for the manufacturer if anything goes wrong.
For patients like Bellina, a national law is necessary. His disease, formally known as amyotrophic lateral sclerosis, attacks nerve cells in the brain and progressively takes away the patient’s control of voluntary muscles -- the brain itself isn’t affected. Most die from respiratory failure within three to five years. There are very few treatments, but several companies are experimenting with therapies. Bellina hopes that removing barriers for drugmakers to make their products available to people like him will eventually help the quest for a cure.
“It’s not that they are heartless and don’t want to, but they are afraid,” he said by email after the hearing. “The company could always say no, but this bill makes it a lot easier to say yes.”
— With assistance by Cynthia Koons