Laura Bray was informed by doctors that her 9-year-old daughter’s leukemia was “treatable,” which she considered fortunate.

Abby had a 90% chance of overcoming the blood cancer if she committed to a three-year treatment plan. However, there was a significant hurdle.

Bray learned that the primary drug required to start Abby’s chemotherapy back in 2018 was in short supply. She faced the difficult task of communicating this to her daughter.

“I didn’t have answers for her when she asked if she would die without her medication,” Bray, 47, from Tampa, Florida, recounted. “I could only reassure her that, ‘We will do our best to find it. Sometimes, remarkable things happen when you try.’”

Ultimately, the family managed to locate the necessary medication. Later, they faced challenges finding two other drugs Abby required. Like many families nationwide, the Brays became acutely aware of the critical shortages of medications for children with cancer.

Experts have noted that shortages of various pediatric cancer drugs have persisted for decades. Advocacy organizations are striving to align manufacturers, wholesalers, and hospitals to create a stable supply chain.

Bray, who established the nonprofit Angels for Change to tackle these drug supply issues, emphasized that all parties involved should prioritize the needs of children.

“Ultimately, it’s about someone’s life,” she stated.

Which pediatric cancer medications are in short supply and the reasons behind it?

According to the Food and Drug Administration (FDA), numerous pediatric cancer medications are currently unavailable, and some have been discontinued altogether.

The most common one affected is injectable methotrexate sodium, a chemotherapy drug frequently used for treating acute lymphoblastic leukemia, brain tumors, non-Hodgkin lymphoma, and osteosarcoma, as noted by St. Jude Children’s Research Hospital. The FDA’s shortage list also includes leucovorin, a medication that helps alleviate methotrexate’s side effects.

These medications, particularly sterile injectables, are vulnerable to shortages due to the limited number of manufacturers, explained Prashant Yadav, an expert in medical supply chains and a professor at INSEAD, a prestigious business school in France.

Creating generic sterile injectables is complex and requires rigorous quality control. Because these drugs are relatively inexpensive, manufacturers often exit the production arena, seeking more profitable options, Yadav said.

The few companies still producing these drugs are situated in the U.S., China, and India, where FDA officials encounter difficulties in imposing and maintaining the stringent quality standards expected of U.S. manufacturers.

If the FDA identifies a quality concern, which Yadav indicates occurs frequently, it halts production until the issue is addressed. A shortage can have widespread repercussions as other manufacturers strive to meet the heightened demand.

“A single glitch from one company can send (the drug) into a nationwide shortage,” noted Dr. Gwen Nichols, executive vice president and chief medical officer of The Leukemia and Lymphoma Society. “This situation is frightening for families, and it places immense pressure on doctors who have to tell families, ‘I can’t find any treatment options.’”

Resolving a quality issue and resuming production can take months, with the knock-on effects of these supply interruptions potentially lingering for years, according to Yadav.

Yadav termed this situation “heartbreaking,” recognizing that advancements in biological medicines are impressive, yet pediatric oncology medications remain elusive.

How healthcare providers cope with shortages of cancer drugs

Hospital personnel have adapted to the constant challenges posed by pediatric cancer drug shortages.

They often reach out to other hospitals to check for available stock that can be shipped or may use medications intended for adults, adjusting the doses for children.

If those options are exhausted, doctors may resort to alternative treatments. However, these substitutes are typically less effective than the preferred drugs that are in short supply, Dr. Gwen Nichols, chief medical officer of The Leukemia & Lymphoma Society, explained.

“It’s an unfortunate waste to resort to second-rate treatments when it involves a child,” she said. “This system is ineffective, irrational, and poses risks that should not manifest.”

While there are strategies to lessen delays, experts suggest that a portion of the issue stems from the lack of coordination among hospitals, wholesalers, and drug manufacturers.

Instead of operating in isolation, Yadav advised that these entities should collaborate by improving communication and sharing crucial information about product availability and the potential for drug shortages.

Alternative Approaches to Cancer Drug Shortages

Experts suggest that improving the profitability of cancer medications is key to resolving the core issues behind current drug shortages in cancer treatment. By making these drugs more lucrative, manufacturers may be encouraged to venture into the pediatric cancer market.

This could involve raising the prices of generic medications, a strategy referred to by Yadav as a “market resiliency premium.” However, he stressed that this additional cost should not fall on patients; instead, it should be absorbed by wholesalers, hospitals, and insurance providers.

The government may also play a role by offering incentives to new drug manufacturers, attracting more companies that can boost the overall supply and provide alternatives if problems arise in the supply chain.

“This is a crucial part of the solution,” remarked Brooke Bernhardt, the chief pharmaceutical officer at St. Jude Children’s Research Hospital. “We need to ensure drugs are available from the very start.”

To further alleviate shortages, Yadav recommended establishing a rotating stockpile. Wholesalers or manufacturers could maintain a reserve of medications for up to six months, serving as a cushion during times of limited supply.

This stockpile would help ensure that treatment progress remains uninterrupted, relieving providers from the stress of sourcing medications, as was seen during Abby’s treatment.

Since her daughter rang the bell to signal the end of cancer treatment in February 2021, Bray has been working actively to combat the problem of drug shortages for pediatric cancers through her nonprofit organization, Angels for Change.

“I understand what it’s like to be a parent or caregiver watching a child struggle to survive,” Bray stated. “You want to give them everything they need to fight.”