Giving Patients One More Shot

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American medicine is producing new treatments for everything from cancer to blindness, and one question is whether government drug approvals can keep pace. One response is to allow some patients access to drugs still in the pipeline, and a “right to try” bill in Congress is an important moment for personal freedom, with a word or two about the finer details.

President Trump said in his State of the Union address that “patients with terminal conditions, and terminal illness, should have access to experimental treatment immediately that could potentially save their lives.” He called on Congress to “give these wonderful, incredible Americans the right to try.”

A right-to-try bill sponsored by Wisconsin Senator

Ron Johnson

passed the Senate last year. The bill essentially says: Government will not get in the way of a patient with a life-threatening disease who wants to take a flyer on a treatment not approved by the Food and Drug Administration. The drug must have cleared a phase of FDA trials for safety but not efficacy.

FDA currently runs a program known as “expanded access,” in which a patient’s physician can ask FDA to sign off on treatment with investigational drugs. FDA approves 99% of requests, but there are only about 1,000 a year, and some medical experts say an application is filed only if the approval is all but assured.

The process was a bureaucratic nightmare for patients who don’t have the luxury of time, and in 2016 the FDA streamlined the application. FDA Commissioner

Scott Gottlieb

testified last fall that the form now takes about 45 minutes. Only one attachment is required, down from as many as eight. Emergency requests are “usually granted immediately over the phone.”

The Johnson bill doesn’t change FDA’s program but says an agency permission slip isn’t required. Mr. Gottlieb has been willing to work with Congress on a right-to-try law and laid out some ideas for technical corrections. This is a notable departure from past FDA leaders who viewed the idea as a threat to agency control.

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Another problem is that drug companies are often reluctant to furnish treatments. Companies worry that an adverse outcome—side effects, death—could be used against them in the approval process. The Johnson bill tries to address this by stipulating that results in right-to-try cases cannot delay or adversely influence an FDA review.

Manufacturers also fear siphoning patients from clinical trials, which for rare diseases are hard to enroll. Disease in some patients will have progressed too far, among dozens of other disqualifying criteria. FDA can help by updating its processes that have been hostile toward smaller trials that rely on, say, statistical methods to compare patients against a disease’s typical path. Under Mr. Gottlieb the agency has awarded grants for “natural history studies” that will try to understand better how some rare diseases progress.

Still, a right to try does not compel companies to participate. Companies are allowed to charge for the direct costs of providing the treatment, and patients could try to find financing through crowdsourcing or private foundations. For patients and companies, right to try is an insurance policy against a future FDA that could clamp down on innovation.

The House has sat on the bill for months, and House Energy and Commerce Chairman

Greg Walden

has offered bromides about giving patients “false hope,” which is paternalism usually exhibited by the left. Right to try is not a miracle drug. But at minimum it would move the decision over treatment and risk closer to the patient facing a tough diagnosis or death.



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