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ASCO to Congress: PDUFA Drug Shortage Provision Needs Teeth

By Mari Serebrov
Washington Editor

If Congress wants to curtail drug shortages, it needs to put some teeth in a PDUFA V provision that requires companies to notify the FDA six months in advance of events that could lead to a shortage, according to a panel at the annual meeting of the American Society of Clinical Oncology (ASCO).

While both the House and Senate passed versions of the user fee package that call for early notification, neither grants the FDA any tools to enforce it. Richard Schilsky, chairman of ASCO's government relations committee, doesn't hold out much hope for getting Congress to back up its bark with a bite when a conference committee reconciles the two bills. (See BioWorld Today, May 25, 2012, and May 31, 2012.)

There's not a great deal of receptiveness among lawmakers for putting teeth into the measure, Schilsky said at an ASCO media briefing Monday.

While drug shortages have threatened many therapeutic areas, "the crisis has been particularly bad in cancer care," Schilsky said, as many of the drugs affected by shortages are chemotherapy drugs that have no acceptable substitutes.

Over the past two years, 22 cancer drugs have been in short supply, ASCO President Michael Link said, noting that the shortages have hit the most essential mainstays of cancer treatment.

While the majority of those standard-of-care drugs are generics, the shortages also are holding up clinical trials that could advance cancer treatments that could potentially save more lives. Most cancer trials use at least one of the chemo drugs in shortage, either in control arms or in combination with an investigational therapy. Sponsors can't begin enrollment in a trial unless they have clear access to all the drugs included in the trial. Because the sterile, injectable, standard drugs aren't always available, trials have been delayed and enrollment has declined. (See BioWorld Today, Sept. 27, 2011, and Dec. 1, 2011.)

The frequency of shortages may be starting to decline, Schilsky said, but the unpredictability remains. The uncertain availability of cancer drugs creates anxiety for patients and treatment challenges for providers.

To end the uncertainty, ASCO is pushing for permanent solutions, including enforcement of the early notification requirement and expansion of that requirement to biologics. As written, the PDUFA bills give the FDA the discretion to require early notification from makers of biologics, but they don't mandate it, Schilsky said.

Early notification is a key tool to preventing shortages, Sandra Kweder, deputy director of the FDA's Office of New Drugs, said during the briefing. Since an executive order was issued Oct. 31 urging early notification, more than 150 shortages have been averted and the number of new shortages has declined slightly, she noted.

While many factors contribute to drug shortages, ASCO is pushing Congress to consider incentives to encourage generic drugmakers to keep making specific drugs, even when prices drop to low profit margins. Such incentives could also encourage companies to develop contingency plans.

Kweder attributed the majority of shortages to quality manufacturing problems that sometimes stem from the business model used by generic producers. Innovator drug companies follow a business model with periods of idle capacity for maintenance.

"There's precious little of that" in the model used by generic companies that produce multiple drugs at a single plant, she said.

If that plant has to shut down, even temporarily, it could trigger a shortage for a number of generic drugs, Kweder added.

On the bright side, the large generic makers are beginning to build new plants. Modernizing those facilities will help prevent the problems that have plagued the industry, Kweder said. But since it takes two to five years to bring a new plant online and many plants need to be updated, the uncertainty of specific drug availability may continue to be the norm for many cancer patients and providers.