Child cancer drugs increasingly scarce

When Maryland Gov. Larry Hogan was diagnosed with non-Hodgkin lymphoma in 2015, his doctors sprang into action. Over 18 weeks and through multiple rounds of multi-agent chemotherapy, the governor's disease was quickly driven into remission.

As a pediatric hematologist/oncologist, I strive to provide my patients with the same level of care Governor Hogan received. Unfortunately, due to shortages of old and off-patent drugs used to cure approximately 85 percent of all children with cancer in the U.S., increasingly, it is getting hard to do so. Typically, we only get one chance to cure disease. If that opportunity is missed, it is rare that we are able to cure childhood cancer.


U.S. drug shortages have become the new normal. According to the FDA, shortages are occurring with greater frequency and lasting longer, causing a significant public health impact. The lack of available lifesaving chemotherapeutics and supportive care agents represents a true national crisis.

Most experts agree that the primary driver for drug shortages is economic. We don't see shortages of six-figure chemotherapeutics that may prolong life by a few months, but all too frequently we see shortages of decades-old proven and lifesaving medications that cost dollars per dose. Where is the logic?


Medications used to treat, and to cure, most childhood cancers are largely older drugs developed many decades ago. For example, acute lymphoblastic leukemia (ALL) is the most common childhood cancer, accounting for nearly one-quarter of all pediatric cancers with a survival rate of about 90 percent. Most drugs used to treat and cure ALL have been in use for more than 50 years. Over the past decade, eight of the 10 drugs most commonly used in the treatment of ALL have temporarily been unavailable.

At the height of the shortages, a survey of medical oncologists published in the New England Journal of Medicine found that 83 percent of oncologists were unable to prescribe their preferred chemotherapeutic, 77 percent had to alter treatment by choosing a different treatment regimen or substitute different drugs during the course of treatment, and 43 percent had to delay the start of treatment. Imagine being diagnosed with cancer, yet unable to start treatment because of a lack of some lifesaving medication. Two recent studies of childhood cancer specialists found that 66 percent of pediatric oncologists reported that patients' clinical care was compromised by drug shortages.

Access to essential medicines is considered a basic human right. As defined by the World Health Organization, essential medicines satisfy the priority health care needs of the population and are both clinically effective and cost effective. They are to be available at all times with assured quality and at an affordable price. The U.S. has neither adopted — nor does it have — an essential medicines list.

The current WHO Essential Medicines List for Children (EMLc) includes 18 chemotherapy and four supportive care agents. Within the past 2.5 years, 63 percent of these essential medicines for children with cancer have been or are currently in short supply in the U.S. (in fact, right now, 22 percent of these essential medicines are scarce here). During this same timeframe, another nine chemotherapy agents not included on the EMLc have been or are currently in short supply in the U.S.

Ironically, the U.S., a leader in medical breakthroughs and one of the most resource-rich countries in history, is also a leader when it comes to shortages of lifesaving chemotherapy and supportive care agents.

Experts have proposed meaningful solutions aimed to prevent and mitigate drug shortages, which have largely remained overlooked. Twice over the past five years, the FDA considered establishing an essential medicines list to address drug shortages. While an important first step, solving the U.S. drug shortage crisis requires more than just this list. Ultimately, reconciling drug shortages requires greater government involvement.

Congress must grant federal authorities the ability to ensure that patients in need have access to medications. Possible ways to achieve this include offering pharmaceutical manufacturers subsidies to maintain production of critically important medications, providing incentives for high quality manufacturing standards and assuring equitable reimbursement practices. To guarantee adequate supply and access, government must also purchase and produce essential medicines. Ideally, this can be achieved through collaboration with motivated industry partners.

Collectively, we must act to ensure that children with cancer, among society's most vulnerable people, are able to access basic and lifesaving medications like the most powerful among us.


Dr. Yoram Unguru ( is a pediatric hematologist/oncologist at the Children's Hospital at Sinai and chair of the Sinai Hospital Ethics Committee. He is also core faculty at the Johns Hopkins Berman Institute of Bioethics.