Health experts offer recommendations to combat shortages of lifesaving drugs

A group of prominent health care experts—including bioethicists, pharmacists, policymakers, and cancer specialists—has proposed concrete steps for preventing and managing a nightmare scenario that is becoming all too common: shortages of lifesaving drugs.

In a consensus statement published in the journal Pediatrics, the experts say they sought to move away from the current strategy of reaction to shortages once they have occurred and focus instead on prevention. Using the example of shortages of chemotherapy drugs used for treating children with cancer—therapies proven to have high survival rates for the most common childhood cancers—the group developed "a comprehensive blueprint for action" they say is critical for managing and preventing future drug shortages.

"Although our recommendations were developed with pediatric oncology in mind, and serve as a blueprint for preventing children with cancer from lacking access to essential lifesaving medications, we believe that they apply more broadly across medicine to include pediatrics and adult medicine in general," says Yoram Unguru, a pediatric hematologist/oncologist at the Herman and Walter Samuelson Children's Hospital at Sinai in Baltimore and a faculty member at the Johns Hopkins Berman Institute of Bioethics.

Unguru, a coauthor of the consensus statement, was the principal organizer of meetings that led to its creation, bringing together representatives from the Food and Drug Administration, leadership of the Children's Oncology Group and the American Society of Pediatric Hematology/Oncology, patient advocacy groups, legal scholars, and clinicians.

The statement includes six recommendations:

• Support current measures (and develop innovative new ones) to prevent future drug shortages at the national level

• Optimize and efficiently use supplies to reduce the likelihood and mitigate the effects of future shortages

• Develop explicit policies that give equal priority during a drug shortage to evidence-based use of chemotherapy agents whether patients are receiving treatment within or outside a clinical trial

• Create an improved, centralized clearinghouse for sharing information about drug availability and shortages

• Explore voluntary sharing of drugs at the state, regional, and national levels

• Develop a strategy for ongoing stakeholder engagement regarding managing drug shortages, with specific emphasis on patients and patient advocacy groups

"This statement is significant both for the consensus found by such a diverse group of experts, and for being the first to take seriously the ethical rationale to prevent shortages in the first place," says Matthew DeCamp, a professor at the Berman Institute and the Johns Hopkins School of Medicine, and the lead author of the statement.

The authors acknowledge that giving equal priority to patients regardless of whether they are participants in research "may be controversial" due to the sentiment that research participants should receiver high-priority access to drugs because of their contribution to medical knowledge and future patients. However, the statement's authors explain that "concerns over undue inducement, public perception, and the imperative to use drugs for indications for which evidence of benefit exists outweigh arguments for giving priority access to research participants."

For each recommendation, the statement also includes potential barriers to its implementation. A centralized information source of drug supply information, for instance, faces the risk that such information will encourage hoarding of existing supplies, including so-called "gray market" suppliers that sell scarce drugs for inflated prices. The statement calls for new policies for both reporting and avoiding these markets, though acknowledges the temptation of health care providers to use them in a shortage, when their patients are in need.

The authors also note the marketplace economy as an obstacle to implementing their recommendations and preventing drug shortages. Drug manufacturers do not like to disclose manufacturing problems that lead to shortages, nor are competitive health care institutions accustomed to cooperating to share resources. Nonetheless, the statement calls for an exploration of "ways to facilitate interinstitutional and interstate transfer of drugs, especially during shortages," as well as the ethical obligation to patients inside vs. outside a health care institution when there is a drug shortage.

"The reasons for drug shortages are complex, but we must not lose sight of the fact that without access to these lifesaving drugs, children and adults with cancer will almost certainly die," Unguru says. "It is untenable for this situation to continue any longer. We have a clear moral obligation to act to address this critical issue."