CCCA Calls for Action to Address Drug Shortage Crisis

The number of drug shortages in the United States has risen to unprecedented levels – more than tripling since 2005 according to a recent analysis (Source: Premier Inc.). Several oncology drugs used to treat children with cancer are among those facing limited supply in the ongoing crisis.

Oncologists in 30 states have reported shortages of cytarabine, a chemotherapy drug used to treat the two most common types of childhood cancer – Acute Myeloid (AML) and Acute Lymphoblastic Leukemia (ALL). Other drugs in short supply in pediatric oncology treatment have included doxorubicin, vincristine, cisplatin, and nitrogen mustard.

As a result of the shortages, patient treatment has been delayed, clinical trials have been disrupted, and doctors have been forced to modify or seek alternative treatments for patients. Oncologists have noted that for certain drugs, there are no suitable substitutes. Shortages have forced some doctors to make difficult treatment decisions and prioritize which patients receive drugs that are in short supply.

Many factors have contributed to the growing drug shortages, such as industry consolidation, manufacturing delays or shortages in raw materials. In addition, many cancer drugs facing short supply are off-patent, made by generic drug companies and no longer yield substantial profit.

The ongoing drug shortage crisis has a significant and adverse impact on children with cancer:

• Treatment delays: Doctors have been forced to delay patient chemotherapy regimens until they receive an emergency supply of a drug. In some instances, doctors must provide alternative treatments that may be more toxic and less effective than the drug in short supply.
• Unavailability of potentially curative therapies: Certain types of childhood cancer are highly treatable and often curable. Acute Lymphoblastic Leukemia (ALL) has a cure rate of 85-90%. Currently, four drugs used to treat ALL are in short supply. Without access to these lifesaving therapies, some children will not receive treatment that could potentially save their lives.
• Clinical trials disruption: The absence of certain cancer drugs has also disrupted clinical trials. The scientific value of a trial can depend on uniform treatment of patients in one group, which is compared with another group receiving different therapy. If the required number of patients do not have access to a drug, the clinical trial cannot be completed.
• Increased costs on patients and providers: Hospitals have been forced to purchase needed therapies from secondary distributors, who often significantly mark up the cost of the drug. A recent analysis found that the shortages could cost hospitals $415 million this year alone (Source: FDA) . These costs may eventually be passed on to the patient.

CCCA urges Congress to take action to address drug shortages:

• S. 296 and H.R. 2245 have been introduced by Senator Amy Klobuchar (D-MN) and Representative Diana DeGette (D-CO) to address the growing drug shortage crisis. The proposed legislation would strengthen requirements that companies notify the Food and Drug Administration (FDA) early about impending shortages. CCCA supports the legislation as important immediate action to address to the drug shortage crisis.