US Navy officer fights for right to try unproven drugs before dying

YouTube image of Matt Bellina, a terminally ill US Navy officer. WASHINGTON, Oct 5 — Matt Bellina was a 30-year-old officer in the US 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 US 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.

Limited use

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 per cent, 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 […]