Senator Kay Hagan Encourages FDA to Consider Faster Approval Pathway for Orphan Disease Treatments

A recent article from Bloomberg noted that, “Congressional talks to renew the fees drugmakers pay to fund Food and Drug Administration (FDA) reviews include proposals to fast-track evaluations of medicines for conditions with no approved cures.”

Plans by Senator Kay Hagan, a North Carolina Democrat, and the Biotechnology Industry Organization (BIO) in Washington would let manufacturers bypass customary clinical trial requirements before bringing products to market, according to drafts of both proposals.

The proposals are factoring in Congressional talks to reauthorize the fee system pharmaceutical companies use to fund FDA reviews, known as the Prescription Drug User Fee Act (PDUFA).  Hagan and BIO’s proposal may be wrapped into the PDUFA renewal. FDA and the drug industry have agreed to a 6 percent fee increase as part of a plan that would run through fiscal 2017. Drugmakers would pay $712.8 million in fiscal 2013 under the deal Congress must approve before Sept. 30, 2012.

While the FDA occasionally grants exemptions, “the experience of many biotechnology companies is that the criteria for utilizing this type of regulatory flexibility are unclear and unpredictable,” according to the biotech industry group’s document, a copy of which was obtained by Bloomberg.

Diseases that may be targeted are rare disorders affecting fewer than 200,000 patients in the U.S. About 360 drugs exist for 7,000 such conditions, according to the National Organization for Rare Disorders in Danbury, Connecticut.

Faster Reviews

Hagan’s plan, based on the biotech group’s proposal, would expand on an FDA accelerated-approval program used mainly for AIDS and cancer treatments, according to a draft proposal.

As the article from Bloomberg explained, :Manufacturers under accelerated approval can conduct shorter trials on patients based on a measured effect of a drug instead of an actual clinical outcome. For some cancer medicines, tumor shrinkage is considered a sufficient sign of survival to justify approving a product. The agency requires companies to prove the anticipated benefit once the treatment is cleared.”

Hagan would allow agency approvals based on the second of three phases of clinical trials, when data cannot be “ethically, feasibly or practicably generated,” according to the draft.

Tools In Place

The FDA already has many tools in place to expedite approval of promising drugs, said Karen Riley, an agency spokeswoman, in an e-mail. The regulator also allows patients access to unapproved medicines through clinical trials and physician-submitted applications, she said.

The FDA has approved 85 rare-disease treatments since 2006, 75 percent of which were based on more-limited trials, John Jenkins, director of the agency’s Office of New Drugs, told a conference in Washington last month.

The FDA’s “orphan drug” program offers tax breaks and a seven-year market monopoly for rare illnesses that affect fewer than 200,000 patients. “I’m worried that a policy where we define flexibility will lock us in,” Jenkins said at the rare disease conference, discussing unspecified calls for faster reviews.

‘Exceptional Promise’

FDA officials discussed the possibility of expediting approvals for treatments showing “exceptional promise” in an innovation report the agency released Oct. 5.

The report said the agency plans to release guidelines for an expedited pathway for high-risk breast cancer treatments. The President’s Council on Jobs and Competitiveness backed a progressive approval system in its interim report last month.

The faster trials envisioned won’t compromise patient safety, said Marc Dunoyer, head of Glaxo’s rare disease unit, in an interview.  “I’m trying to bring earlier access to patients, it doesn’t decrease the work,” Dunoyer said. “You don’t want to compromise on safety and effectiveness.”

The European Union has a program of expedited reviews for rare diseases, emergency situations and debilitating or life- threatening diseases.   “I think it is impossible to have all the response upfront that you want on a drug,” Dunoyer said. “Regulators, companies, physicians need to agree that at one point in time you won’t know everything.”