WASHINGTON, Dec 11 (Reuters) – More patients with serious diseases could get experimental drugs under revised rules proposed on Monday by the U.S. Food and Drug Administration that also detail how manufacturers can charge for them.
The FDA said the changes spell out more clearly which patients are eligible for special access to the unapproved products, as well as the fees they may have to pay.
“When people are dying of a disease and they’ve run out of therapies, not everyone wants to take an investigational drug under those circumstances.
But some people definitely want to take that risk and we want to give them that choice,” Dr. Janet Woodcock, FDA’s deputy commissioner for operations, told reporters.
Experimental, unapproved medicines have been available to some patients under certain circumstances since the 1970s, the agency said. In the past, thousands of patients with HIV and AIDS, cancer and heart problems have been able to use some drugs prior to approval.
But Woodcock said the current confusion about the regulations made them “one of the best kept secrets around” for needy patients. She could not estimate how many more people might now seek experimental drugs but said the new details should help.
Patient groups have long been pushing for the FDA to expand access for desperate patients.
In 2003, two advocacy groups sued the FDA seeking patient access to new cancer drugs that were shown to be safe in initial tests but had not yet received approval. The case was sent back to lower courts for review in May.
The new rules, which are open for 90 days of public comment, detail access for small groups of patients as well as individuals with serious but not immediately life-threatening conditions, the FDA said. It also clarifies how companies should calculate their fees.
Under the proposal, companies could charge consumers for what it cost in materials and labor to make the medicine as well as shipping, handling and other administrative expenses, the FDA said. Patients participating in clinical trials also could have to pay.
Woodcock said the rules aimed to help academic researchers and small companies offer experimental drugs when they otherwise could not by allowing them to recoup their costs. Larger drugmakers usually do not charge patients and the FDA expects that to continue, she said.
Current, vague rules kept many manufacturers from helping such needy patients, she said, adding the revisions have “nothing to do with profits” and will be strictly monitored.
She also cautioned against company attempts to commercialize their products with such early use, adding they are expected to participate while continuing to develop them for future regulatory approval.
Woodcock would not comment on how the new rules could impact the pending lawsuit. Representatives for the Abigail Alliance, one of the groups that filed the suit, could not be immediately reached for comment.