Choosing cancer's youngest victims?

Every parent’s biggest fear is that her child will get sick.  Finding out that the bruises on her son’s shins are not caused by rough and tumble backyard play, but by leukemia, or that her daughter’s abdominal pain isn’t indigestion, but a neuroblastoma, is her worst nightmare. You’d think it really could not get any worse than hearing that your child has cancer.

Except, it could get worse.  As in: “We think we can treat your child’s cancer, but we can’t get the medication to do so.”  Shortages of common cancer drugs such as vincristine, methotrexate, and doxorubicin are becoming a major problem for doctors who treat children with cancer. 

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Two pediatric patients with cancer require an intravenous oncology drug, but due to a drug shortage, the physician can only treat one of them. How does a doctor explain this to desperate parents, and how does the doctor decide which child to treat?

Shortages of essential drugs, including critical chemotherapy drugs, have become both commonplace and dangerous. Drug shortages detract significant time and financial resources, leading to adverse patient outcomes, delayed clinical trials, and significant ethical challenges. The unspeakable outcome, a child’s death due to a lack of critical medicine, looms.

When a drug becomes scarce, physicians have several options: change the regimen, lower the dose, substitute a similar yet different drug, wait until supply returns, withhold chemotherapy from a different patient, or consider buying drugs at very high prices on the “gray market”, unauthorized secondary drug distributors who profit handsomely from the shortages.

The principal cause of the drug shortages can be linked directly to how pharmaceutical products are developed, marketed, and protected by patents. Pharmaceuticals companies spend a great deal of time and money (in some cases, billions of dollars) on the research, development and testing of a new drug to ensure that the drug action is safe, effective, and fulfills its intended use in order to achieve FDA approval.  The pharmaceutical company may hold a patent for a new drug for 20 years from the time the clinical trials have begun. During the period of market exclusivity, a drug company will often charge a high price for the drug, allowing it to recoup the investment it made developing and marketing the drug, and to maximize profit for its shareholders.  

Once the patent expires, however, a drug can be manufactured and marketed in generic form by any drug company, as long as the drug has the “identical” action and composition. In many cases, generics are sold at much lower prices than their branded predecessors because the generic manufacturers hope to quickly grow sales and capture market share from the original manufacturer. Since generic drugs are not protected by patents, multiple manufacturers can enter the market and compete with each other, further driving the prices and profits down.

Medicare legislation strictly limits price increases of generic drugs, even the ones currently in short supply. Drugs used for cancer often serve small niche markets and have the added drawbacks of short shelf life and complicated handling requirements. The sad but simple truth is that the market for many of these specialized drugs is too small and specialized to be attractive for generic drug companies, who would rather churn out tons of high profit, quick turnover meds.

Although the focus groups like The 2014 Working Group on Chemotherapy Drug Shortages in Pediatric Oncology provide a “comprehensive blueprint for action” which aims to prevent all drug shortages with several recommendations for how it would prevent shortages, optimize drug supplies, and improve information, glaringly lacking, was any specific recommendation that addresses the problem of pricing.  

Other experts (B Chabner, NEJM 2012) have called for relaxing government price controls, encouraging importation of generic drugs from European manufacturers, and rewarding good manufacturing practices with expedited consideration of new generic drug applications.

Drug shortages affect the most vulnerable patients: very sick children and the parents desperate to find them treatment at any cost. The media are full of stories about parents going bankrupt seeking treatment and medication. Shortages also tend to favor children whose parents have the financial resources to gain better access to medications, a morally unacceptable situation.

While the FDA Safety and Innovation Act signed into law on in 2012 lays out a strategic plan to inform physicians about drug shortages, this problem cannot be solved by simply listing drugs in short supply. It requires a major change in the way these drugs, both branded and generic, are developed, manufactured, and marketed.

Physicians, pharmaceutical industry executives and insurance companies need to figure out a way to incentivize companies to produce needed drugs in the right quantities at the right time, and according to good manufacturing standards.  

We put a man on the moon, and we can ship watercress from California to Maine overnight. We should be able to get children the chemotherapy medications they need.

Thompson is a radiation oncologist and patient advocate.  She received a master’s degree in Public Health in Health Care Policy and Management from Harvard University and received her MD from Johns Hopkins University School of Medicine, specializing in Surgical Gynecology. 

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