Childhood cancers have seen remarkable improvements in prognosis over the past few decades, with survival rates in countries such as the US at around 85%.1 Although prognosis varies widely between the type of childhood cancer, stage of disease at diagnosis, and even cytogenetic analysis, perhaps surprisingly, the increase in survival is largely not due to new, innovative therapies, nor surgical techniques. 

For example, for acute lymphoblastic leukemia (ALL), the most common type of childhood cancer, the drugs used have been around for several decades. Approximately 90% of children with ALL have at least a 10-year survival,2 which is typically achieved by gradually tweaking timings and dosages of drugs for a complex treatment regimen that typically lasts between 2 to 3 years. 

Despite the vital need for these drugs, they are often in short supply. A new paper published in JAMA Pediatrics discusses these drug shortages and makes the case for government intervention to ensure uninterrupted supply.3

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“Cancer isn’t treated with a single drug; we use cocktails. The ability to cure a child’s cancer is dependent on the armory of this cocktail. If we are missing one drug, we may not be able to cure this cancer,” said Yoram Unguru, MD, a pediatric oncologist at The Herman and Walter Samuelson Children’s Hospital at Sinai and an assistant professor at Johns Hopkins School of Medicine in Baltimore, Maryland. Dr Unguru is also an author of the JAMA Pediatrics paper.

In a 2015 national survey, two-thirds of principal investigator or pharmacist members of the Children’s Oncology Group indicated that drug shortages affected patient care.4

“When you have a little bit of drug, we are forced to choose what deserving child in front of you gets this limited supply. There isn’t a patient or parent [who] isn’t shocked by this. These are preventable shortages,” said Dr Unguru.

The paper includes a list of vital drugs for childhood cancer treatment, composed of not only chemotherapy agents, but also other drugs which are termed critical supportive drugs, such as painkillers and anesthetics. Considering the crucial nature of these agents, why do supplies so frequently run low?

“These are old drugs, off-patent — they are reimbursed at relatively low rates. There are very few manufacturers of these drugs nationally; [there are] only 3 companies that make over 70% of all the sterile injectables in the US. Very few companies want to do it. Root drivers of the shortages are economic factors and business decisions. You don’t see shortages of blockbuster, highly profitable drugs,” said Dr Unguru.

Dr Unguru wants the government to intervene to ensure uninterrupted supply of these drugs. But how feasible is it to involve government to incentivize companies to ensure there are no shortages of these cheap, low-profit drugs?