FDA moves to speed access to compassionate-use drugs

The Food and Drug Administration has reduced an obstacle from its compassionate use policy, streamlining paperwork that physicians must submit to obtain experimental drugs for patients with life-threatening illnesses.

Doctors will now file an application for FDA approval that contains just 11 questions, 15 fewer than the old form. They should be able to complete this new version in 45 minutes, the FDA said. The new form is simpler because it was designed for individual patients, replacing an all-purpose format that had been used by doctors acting on behalf of individuals or groups of patients.

There had been concerns that doctors might have been deterred from applying for compassionate access, which is also known as expanded access, says FDA spokeswoman Sandy Walsh.

The policy is intended to help patients with incurable diseases who have tried all standard therapies and hope to extend their lives by taking experimental drugs not yet approved by the FDA, says Dr. Edward Kim, chair of the Department of Solid Tumor Oncology at the Carolinas HealthCare System's Levine Cancer Institute.

The FDA's old form was a "pretty laborious process," Kim said. When doctors are serving patients whose time is running out, every minute saved on paperwork can help, he said.

In streamlining its path to approval, the FDA has bolstered a larger movement to make experimental drugs more accessible. Currently, 20 states have "right to try" laws aimed at improving terminally ill patients' access to experimental treatments, according to the Regulatory Affairs Professionals Society.

Despite benefits for time-pressed physicians, the FDA's slimmed-down application form might not do much to speed those drugs to patients whose time is running out.

Doctors still must first obtain a letter of authorization from that drug's manufacturer. The FDA can't compel drugmakers to grant permission. Manufacturers might reject requests because they're worried about liability if the drug causes harm or they might consider the drug unsuited for a particular patient.

"There has been a tendency to focus on this FDA paperwork as the significant part of gaining access to drugs, but where most requests stop is with the company making the drug," says Mark Fleury, a policy analyst at the American Cancer Society Cancer Action Network.

After doctors get manufacturers' consent, they next submit an application to the FDA. The agency has approved 99 percent of the applications filed for the past six years, FDA figures show. Only 14 out of 1,430 applications were rejected in fiscal 2015.

Expanded access is intended for patients unable to get drugs that are being tested in clinical trials. Sometimes a patient is unsuited for the trial or there might not be a trial to enter at the time the patient needs the drug.

Investigational drugs aren't without risk. The FDA hasn't vouched for the safety of these drugs and they could cause unexpected and potentially deadly side effects, such as liver damage.

They may also be ineffective. Just because a drug is working for one patient in a clinical trial doesn't mean it will work for someone else.

But in situations like these, it's hard not to hope.

This story was created as part of a collaboration between NPR and Kaiser Health News. Copyright 2019 Kaiser Health News. To see more, visit Kaiser Health News.