Hope on the Horizon

By: Margaret Hamburg, M.D.

This week we were once again reminded that the critical issue of drug shortages isn’t about industry, it isn’t about government, or even about the drugs themselves. It is about getting people the treatments they trust, they need, and they rely on. One person who understands this all too well is Sara Stuckey from Lincoln, Illinois. Sara’s son, six-year old Nate, has been on the cancer drug methatrexate since he was diagnosed with leukemia back in 2009. The drug is the preferred treatment for the disease. Unfortunately, due to the shortage, Nate’s doctor informed Sara that he only had enough of the drug for Nate’s treatment this week. 

At a briefing Tuesday hosted by the FDA for representatives from industry, health professional and patient organizations, as well as the FDA team that has been working day and night to address this problem, Sara Stuckey shared her story.
 
Watch videos from the FDA Progress on Drug Shortages briefing.
 
Sara talked about Nate, the little boy, who is the “laid-back” one in her family. She explained how the family has learned to be flexible, working around Nate’s disease, compromised immune system, and chemotherapy treatment. For this family and many others, methotrexate is a life-saving drug. So, when Sara heard about a possible shortage of the drug, she talked to Nate’s clinic. They said there was just enough supply for a week but that there were alternatives. Still, Sara was left with many questions and concerns – the same questions any parent would be asking if their child faced a life-threatening illness.
Sara Stuckey

Sara Stuckey speaks at "FDA Progress on Drug Shortages," a briefing held on February 21, 2012, about her son, six-year old Nate.

View photos from the FDA Progress on Drug Shortages briefing.
 
The good news for Sara and Nate – and many other children and families – is that the shortage has been averted.
 
  • FDA has prioritized review of and approved a preservative-free methotrexate generic drug manufactured by APP Pharmaceuticals and expects that product to become available in March and continue indefinitely.
  • At Tuesday’s briefing, the CEO of Hospira, another company that manufactures the drug, said that supplies are being ensured for months to come. Hospira expedited release of additional supplies, resulting in 31,000 vials of new product – enough for more than one month’s worth of demand – being shipped to hundreds of U.S. hospitals and treatment centers on Tuesday.
  • And FDA is actively working with other manufacturers of methotrexate who have also stepped up to increase production in order to meet patient needs, including Mylan and Sandoz Pharmaceuticals.

To remedy the critical shortage of another cancer drug Doxil (liposomal doxorubicin), the FDA took proactive steps needed to increase available supply for patients in the U.S. For Doxil, there will be temporary importation of a replacement drug, Lipodox (liposomal doxorubicin), which is expected to end the shortage and fully meet patient needs in the coming weeks.

Still, we cannot stop there. At Tuesday’s briefing, we heard from representatives of the American Cancer Society, the American Society of Clinical Oncology, the Children’s Oncology Group, and APP, as well as Hospira. All spoke forcefully for working together to prevent future shortages from affecting the lives of both children and adults. It is enough for families to cope with disease; adding real or potential shortages to their list of worries is more than unfortunate.

Members of the FDA Drug Shortages team

Members of the FDA Drug Shortages team

This event reminded us of the human impact of drug shortages – as well as what can be done when we work together. We hope this can be a model for future efforts as we move forward, working with our partners in industry, with health professionals, and with patient advocates to find answers that will work for families like Nate’s.

While there is no simple solution to resolving drug shortages, we are doing all that we can to make sure patients have access to the critical medicines they need when they need them. I’d like to give a special thanks to the FDA Drug Shortages team and all the other staff throughout our agency for their hard work and leadership on this topic.

We are making progress. Hope is on the horizon. 

Margaret Hamburg, M.D., is Commissioner of the U. S. Food and Drug Administration.

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