APHON Pediatric Chemotherapy-Biotherapy Renewal

The causes of drug shortages are multifactorial. The U.S. Food and Drug Administration (2019) reports that economic forces are the root cause of drug shortages in the United States. These economic factors include the falling prices of the drugs, declining revenues from sales and the minimal contribution of certain drugs to the company’s overall revenue. A manufacturer has little incentive to invest considerable time and money to produce a drug that will not bring sufficient income to the company to justify that investment. Another contributing factor is the limited availability of raw materials. In 2018, 88% of pharmaceutical ingredients came from non- U.S. sources. Obtaining pharmaceutical ingredients from sources overseas may be cost effective for pharmaceutical manufacturers, but it may also hinder the manufacturer’s timely response to an increase in demand. Additional causes include quality-control problems in manufacturing that create production delays; a limited number of manufacturing companies; and manufacturers’ business decisions, restricted distribution methods, inventory practices, and to a lesser degree, regulatory issues. Impact Drug shortages have a high impact on health, and in pediatric oncology, they have a particularly serious impact. As Bunnell and colleagues (2020, p. 2) note, “Childhood cancer treatment relies on the use of sterile injectable generic agents, which make up the majority of scarce medications and which manufacturers have limited economic incentives to produce.” Pediatric hematologist/oncologist Yoram Unguru reinforced this point in a 2020 APHON webinar, A Dearth of Lifesaving Medications: Scarcity and Shortage in Childhood Cancer : “Costly chemotherapy agents with limited efficacy are rarely, if ever, in short supply, while inexpensive, older, curative drugs are.” Clinical trials have led to a dramatic improvement in childhood cancer survival over the past 5 decades, but drug shortages may negatively affect enrollment in those trials. Furthermore, many of the scarcest drugs have served as the backbone of childhood cancer clinical trials that have led to proven, lifesaving regimens. No adequate substitutes or alternative drugs are available to treat these pediatric patients during a shortage. In 2019, a critical shortage of the drug vincristine had a significant impact on the childhood cancer population because of its widespread use in the treatment of many different childhood cancers. In the case of acute lymphoblastic leukemia (the most common childhood cancer, which accounts for nearly one-quarter of all children with cancer), a shortage of a critically important drug like vincristine means that the current 90% 5-year event-free survival rate for 3,000 U.S. children affected each year may be compromised. Manufacturing of medical devices has also had an impact on drug shortages. For example, Puerto Rico is responsible for $40 billion of the pharmaceuticals market, more than any other state or territory. More than 100 companies that produce pharmaceuticals or medical devices have manufacturing sites in Puerto Rico. Puerto Rico was devastated by Hurricane Maria in 2017. However, the shortage of sterile normal saline that occurred during that time was not actually a