TOP - August 2012, Vol 5, No 5 published on August 22, 2012 in Drug Updates & News
Cristi Radford, MS, CGC

The drug shortage crisis is easing, but an actual solution to the problem is still elusive, according to participants in a press briefing that addressed the issue at the 2012 Annual Meeting of the American Society of Clinical Oncology (ASCO) held in Chicago, Illinois.

Richard Schilsky, MD, chair of ASCO’s government relations committee and an oncologist at the University of Chicago, indicated, “Patient care has been threatened in many cases. But the good news is that the frequency of drug shortages is beginning to decline.”

In the past 2 years, 22 oncology drugs were, or still are, in short supply, mostly generic injectables. “They are irreplaceable when there are no acceptable substitutes,” Schilsky pointed out. “We are not exactly sure when a generic drug will suddenly go out of supply, and this creates a tremendous amount of uncertainty, anxiety, and difficulty in planning.”

Sandra Kweder, MD, deputy director of the Office of New Drugs at the US Food and Drug Admin istration (FDA), said she was primarily concerned about the patients. “This is about people being able to get treatments they need, when they need them. This situation is not acceptable.”

FDA Hypervigilant

Kweder said that prevention of shortages is the FDA’s “absolute priority,” and a priority of Congress and the administration.

In the past 6 months, more than 150 drug shortages have been averted through early notification by manufacturers. “These drugs have not appeared on the list of shortages,” she noted. “But things can change quickly. Early notification is the key….Our staff is vigilant, and we work under extraordinary conditions to pull out the stops every day to prevent shortages and address those that are occurring.”

However, the early notification system is voluntary, and the FDA cannot mandate that companies up their production, she acknowledged. “One thing the FDA cannot do is tell a company they must make a drug.”

But it can work with companies to “ferret out problems” and resolve manufacturing and drug quality issues, which account for most shortages. “These issues are not minor,” she said.

The FDA helps companies ramp up production for new or “relaunched” products and uses “regulatory discretion” to help companies import drugs, she stated.

Thanks to such efforts, she noted that cytarabine, doxorubicin, liposomal doxorubicin, mitomycin, daunorubicin, and 5-fluorouracil are currently meeting demands. Methotrexate continues to be carefully monitored, although a resolution is expected soon.

User Fee Legislation, Economic Incentives

“ASCO believes a permanent solution will require congressional legislation,” Schilsky said.

Progress has been made. On May 30, the Prescription Drug User Fee Act (PDUFA) reauthorization legislation passed the United States Senate as the FDA Safety and Innovation Act (S 3187), and the companion bill passed the United States House of Rep resentatives as the FDA Reform Act (HR 5651). The next step was to reconcile the differences between the 2 versions, both of which contained a proposed user fee program for generic drugs and biosimilars and language on drug shortages. The program will allow the FDA to collect user fees from industry to help fund the agency’s timely review of applications. On June 18, the United States Congress released a reconciled version of the reauthorization bill, which was presented again to the House of Rep - resentatives and Senate. The House passed the final version on June 20 and the Senate passed it on June 26. President Barack Obama signed the bill on July 9, 2012.

The PDUFA legislation will bring in an additional $1.5 billion to the FDA, which is expected to reduce the review time for a new drug application from the current 30 months to 10 months or less. “This is a huge step forward in getting manufacturers into the game,” said Schilsky. “Industry is very willing to pay these fees because they believe it will enable a more timely review of their applications, and a faster time getting their drugs to market.”

The FDA “user fee” legislation that was passed by Congress, however, does not contain an enforcement mechanism that carries penalties for not reporting. “ASCO has concerns that if there is no teeth in that legislation, companies may decide not to report,” Schilsky said. “On the Hill, there is not much receptivity to putting that in the legislation.”

“We also feel that Congress should consider making economic incentives for companies to stay in the market for these lower-cost drugs,” he added. “And that the provisions addressing drug shortages also apply to biologics, which at the moment are branded but will eventually be generic.”

Would economic incentives en courage production of generics? The fact that virtually all shortages so far have been generics “speaks to the economic and business model,” Schilsky acknowledged. “These drugs have the smallest profit margins, but it’s hard to connect the dots on this.…The generic drug industry is thriving. Yes, they have manufacturing problems and yes, periodically plants go down, but it’s not like the economics of the situation is driving them out of business. Economic issues may be contributing to the shortage, but not at such a fundamental level that companies are folding their tents and going away.”

Could New Drugs Be Affected?

While the shortages to date have been almost restricted to generics, it appears possible that even supplies of the newest drugs may fall short of the demand.

News of the FDA’s approval of pertuzumab (Perjeta) for HER2-positive metastatic breast cancer was welcomed, but in its announcement, Genentech, the manufacturer, disclosed that production issues could affect the drug’s long-term supply.

“We expect to meet demand for Perjeta following today’s FDA ap - proval,” said spokesperson Patrick Y. Yang, PhD, head of technical operations. “We recently identified a cell growth issue that might affect our future supply of the medicine. We take this very seriously and are working with the FDA to ensure a consistent manufacturing process that maintains drug supply for the people who need it.”

In her FDA announcement of the drug’s approval, Janet Woodcock, MD, director of the FDA’s Center for Drug Evaluation and Research, said, “Given the need for additional treatments for metastatic breast cancer, we made the decision to approve this drug today and not to delay its availability to patients pending resolution of the production issues relating to future supply.”

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Last modified: May 21, 2015