TMC PULSE

29 t m c » p u l s e | m ay 2 0 1 9 Must-Have Medicines for Kids with Cancer Clinical experts argue for an essential medicines list for pediatric cancer patients to address chronic shortages I magine that a child is diagnosed with cancer. Now, imagine that the lifesaving chemotherapy drugs needed to treat that child are in short supply—and for simple, fixable reasons. It's a reality playing out in hospitals across the United States and researchers at Baylor College of Medicine have a solution. In a recent position paper published in the journal JAMA Pediatrics, Stacey Berg, M.D., and Brooke Bernhardt, Pharm.D., called for the development of an essential medicines list for pediatric oncology. Modeled after the World Health Organization's own Essential Medicines Lists—recommenda- tions that cover the gamut of conditions for both children and adults—theirs is a straightforward call to address what has become an increasingly frequent obstacle to care. "One of the things that was really appalling when we were working on the paper was how many drugs we use all the time that turned out to be in short supply some time in recent history— I was shocked," said Berg, a professor and associ- ate dean for research assurances at Baylor College of Medicine. The reason these drugs are growing scarce is complex, she said. "For some drugs, the issue is difficulty manu- facturing them, or difficulty with supply or with the FDA [U.S. Food and Drug Administration] inspections of the plants. But really, I think the bottom line is economics—not at the level of individual drug companies, but at the level of what we, as a society, say is worth a lot of money," explained Berg, who is also co-director of Texas Children's Cancer Center's Developmental Therapeutics Program. "Old, generic drugs—even though they are essential to treatment of all kinds of health issues—they don't make a lot of money for people." But when it comes to pediatric oncology, old, generic drugs are the cornerstones of treatment. "One of the reasons that we use old drugs in pediatric oncology is because many of them work great," Berg said. "Adult oncology tends to use many newer, more expensive drugs because nothing that they've ever had worked, whereas in pediatric oncology, there have been increasing cure rates for children with cancer since the early '70s. Many of our treatments today are still based on those treatments, and it's not because the field hasn't advanced, it's because the treatments are very good and you don't want to go away from things that work so well." New, cutting-edge drugs are continuously being added to therapies to improve outcomes and, as Berg and Bernhardt pointed out, they make manufacturers a nice sum of money and are rarely in short supply. "In general, you don't hear about shortages of new drugs for very long," Berg said. "What you hear about is the shortages of old drugs that don't have patent protection anymore." Bernhardt, an assistant professor of pediatrics in the hematology and oncology department at Baylor, illustrated the issue with numbers. "The new drugs that can charge $70,000 and up a dose, of course they're going to make those drugs," she said. "The drugs that are $2.15 a vial, that's a little bit more challenging." Inferior outcomes Baylor and Texas Children's have set procedures in place to ensure these shortages don't affect patient outcomes, but it's no easy feat. ➟ In general, you don't hear about shortages of new drugs for very long. What you hear about is the shortages of old drugs that don't have patent protection anymore. — STACEY BERG, M.D. Professor and associate dean for research assurances at Baylor College of Medicine B y A l e x a n d r a B e c k e r Stacey Berg, M.D., and Brooke Bernhardt, Pharm.D., want to model an essential medicines list for pediatric oncology after the World Health Organization's own Essential Medicines Lists.

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