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Drug shortages frustrate pediatric oncologists, patients

CCH Newsletter - Spring 2012


By Sara Farris

Recent news stories have unveiled a growing concern among pediatric oncologists and health care institutions. Many predict we’re facing a nationwide drug shortage issue.

Most recently, the injectable form of preservative-free methotrexate, commonly used to treat pediatric patients with leukemia and osteosarcoma, was at a critical supply level.

The bigger drug shortage picture

Drug shortages are an increasingly frequent and serious problem affecting health care organizations.  

"A number of contributing factors are causing these shortages, such as raw material unavailability, manufacturing difficulties and regulatory issues, voluntary recalls related to manufacturing problems, changes in medication formulation, and industry consolidations and economic decisions," says Wendy Heck, Pharm.D., manager of drug information and drug use policy at MD Anderson.

Joel Lajeunesse

Regardless of the catalyst, drug shortages create great frustration for everyone involved, including purchasing agents, pharmacists, nurses, physicians and patients. Fortunately, MD Anderson is usually able to work around drug shortages due to its larger volume of stocked pharmaceutical agents.

"We meet weekly to review current drug shortages. If a shortage does reach MD Anderson, our team works diligently to develop a management plan to minimize the impact on our patients," says Joel Lajeunesse, vice president and head of the Division of Pharmacy. "For now, we have sufficient supplies of methotrexate for our patients."   

Managing methotrexate supply

Pediatric oncologists at MD Anderson Children's Cancer Hospital work closely with pharmacists to manage the supply of methotrexate for pediatric patients. The most recent concern involves the preservative-free form of injectable methotrexate. It’s commonly used for intrathecal, or spinal cord, injections because it lowers the risk for neurotoxicities, such as paralysis.

The shortage was alleviated when the U.S. Food and Drug Administration allowed the temporary import of methotrexate and another scarce drug from countries abroad. Although helpful, many feel this is a band-aid approach to a larger issue.

"We've been dealing with drug shortages in pediatric oncology for a while now, whether it stems from lack of development or discontinuation of production," says Patrick Zweidler-McKay, M.D., Ph.D., section chief of leukemia at the Children's Cancer Hospital.

"It's not as profitable for companies to make drugs for rarer cancers, such as childhood cancer. However, this situation involves vital standard-of-care drugs, and we can't get a consistent supply of them. Something must be done for our patients' sake."

Advocating for a solution

Advocacy groups, health care professionals and patients are uniting to bring a voice to the specific needs related to childhood cancer. 

Last March, families congregated on Capitol Hill as part of a special day hosted by the Children’s Oncology Group. Then, in September, Eugenie Kleinerman, M.D., head of the Children’s Cancer Hospital, and other leaders in pediatric oncology spoke before a Congressional panel on the need for drug development for childhood cancer.

Val Marshall

Val Marshall is one of thousands of parents who have lobbied on Capitol Hill with the Children’s Oncology Group. Her son, Addison, has spent the last three years undergoing treatment at the Children’s Cancer Hospital for acute lymphocytic leukemia, the most common cancer in children.

Much of Addison’s treatment, both initially and after a relapse, has consisted of methotrexate, including intrathecal administration. He knows firsthand the feeling of fear that comes with a pending drug shortage.

“It’s scary because you think if you can’t get the drug when you need it, the cancer will come back,” says the 18-year-old.

Val plans to return to Capitol Hill in June to continue meeting with legislators and raising awareness about the needs of young cancer patients like her son.

“This has been a band-aid solution, and band-aids are better than open wounds,” she says. “But it’s time we find a more long-term solution, and that’s why I’m going back to Washington.”

© 2015 The University of Texas MD Anderson Cancer Center