End to children's cancer drug shortage is unclear

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By LINDA A. JOHNSON, Associated Press

TRENTON, N.J. (AP) — A severe shortage of a childhood cancer drug should ease before hospitals run out of it in a couple weeks, a top federal regulator said Tuesday. But the companies that make the drug are giving few details about how they will find a long-term solution to end the problem.

Valerie Jensen, associate director of the Food and Drug Administration's drug shortage program, said her team is working with the three makers of preservative-free methotrexate, which is used to treat the most common childhood cancer, acute lymphoblastic leukemia, or ALL.

The drug, which cures up to 90 percent of children with ALL, has been in very short supply in recent weeks because a leading maker of the drug shut down some of its factories late last year.

Hospitals have warned the FDA that treatments for children with ALL could be stalled if new shipments of the drug aren't received within a couple weeks. That could lessen the chances of children being cured of the disease.

"We understand from all three companies that they will be starting to ship by the end of the month," Jensen told The Associated Press. She noted that federal regulations bar the FDA from discussing plans of specific companies, as that's considered proprietary information.

The three manufacturers of the drug — Mylan Inc., Hospira Inc. and Sandoz Inc. — weren't specific about how they plan to resolve the shortage of the cancer medicine:

—Mylan says it's working on increasing manufacturing capacity, which includes getting approval for that from the FDA. The company has an emergency supply of small vials of methotrexate, and plans to ship larger vials at the end of the month.

—Hospira temporarily boosted production to address the shortage issue, but then ran out of the active ingredient. It is still producing some of the drug, but is trying to get more of the active ingredient.

—Sandoz is aiming to ship some of the drug in late February. The company did not provide any details.

APP Pharmaceuticals LLC, which makes a form of methotrexate with preservative, said it is working with the FDA to determine whether it could get approval for a preservative-free formula. The preservative-free version is needed by both children and adults when methotrexate is given in very high doses intravenously or by injection in spinal fluid. Preservatives in the drug can cause potentially fatal respiratory and organ damage or, when put into spinal fluid, paralysis.

Dr. Peter Adamson, chairman of the Children's Oncology Group, a network of 200-plus North American hospitals treating children with cancer, said FDA officials "have been reassuring in discussions that this is not going to be a prolonged shortage."

Still, he said: "Until the drug is actually delivered, we can't be sure."

Until late last year, five drugmakers in the U.S. manufactured the generic injected cancer medicine methotrexate, which is crucial for treating children and adults with ALL as well as children with other, less-common cancers.

Four of those companies made a preservative-free version. So, when one of the biggest makers of the preservative-free methotrexate, Ben Venue Laboratories Inc., recently shut down its four factories in Bedford, Ohio, possibly for a year, due to serious quality problems, the on-again, off-again methotrexate shortage that began in late 2008 quickly turned into a crisis. That's because the other companies cannot quickly pick up the slack.

Methotrexate is just one of 283 drugs currently in short supply. Those include 27 new shortages reported this year and 215 that began in 2010 or 2011 and remain unresolved, according to Erin R. Fox, manager of the University of Utah Drug Information Service, which tracks national drug shortages.

Multiple hospitals and cancer specialists contacted by The Associated Press say they still have just enough methotrexate to treat current patients. But a January survey of 204 oncologists around the country found at least 40 percent believed that one or more patients in the past year had either died prematurely or suffered a tumor recurrence because of the cancer drug shortages.

But the methotrexate shortage has gotten particular attention because it's frightening parents. The drug has been a mainstay of childhood cancer treatment for 40 years and it's highly effective.

In addition, for ALL, there is no good alternative treatment for spinal fluid injections. For the other cancers, the fallback drugs will not work as well, but it's unclear how much worse children getting that second-best option will fare.

Meanwhile, several members of Congress have been introducing bills aimed at addressing various causes for the shortage and price gouging that's been reported by marketers outside hospitals' normal distribution channel.

Sen. Amy Klobuchar, D-Minn., for a year has been trying to win passage for her bill requiring all drug manufacturers to notify the FDA in advance when they anticipate shortages. Currently, that's only required if there is only one manufacturer of a particular drug, and there are no penalties for not doing so.

On Tuesday, she proposed adding her bill as an amendment to a pending transportation funding bill.

"In the Senate, it's so hard to get an individual bill to pass," Klobuchar said, adding that she'll keep trying this strategy until it works.

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