Pediatric Oncology Groups Endorse Ethical Framework for Rationing Scarce Drugs


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Yoram Unguru, MD, MS, MA

What we need is a policy shift to prevent these shortages from ever occurring. The fact that children with cancer cannot obtain the lifesaving drugs needed to cure their diseases is a national disgrace.

—Yoram Unguru, MD, MS, MA

The Children’s Oncology Group (COG) and the American Society of Pediatric Hematology/Oncology (ASPHO) have endorsed formal clinical recommendations for rationing chemotherapy and supportive care agents during times of shortage. The guidance is needed because persistent shortages of many standard therapies for cancer, such as methotrexate and vincristine, have put oncologists in the difficult position of choosing which patients to treat first with scarce supplies.

“The vast majority of treatment centers don’t have a formal process to deal with drug shortages, and decisions often are made in the heat of the moment by physicians who are faced with making a tragic choice and who have to decide which of two kids gets the scarce drug,” said Yoram Unguru, MD, MS, MA, who led a COG task force that wrote the recommendations, published online January 29 in the Journal of the National Cancer Institute.1

Applicability of Recommendations

The goal of the task force was to develop a process that COG member institutions could put into place to base rationing decisions on an ethical framework. The guidance aims to remove the burden from the treating physician as well as the potential for biased decisions. 

“These types of decisions are too consequential,” said Dr. Unguru, a pediatric oncologist at The Herman & Walter Samuelson Children’s Hospital at Sinai, Baltimore, and a faculty member at the Berman Institute of Bioethics at Johns Hopkins University. “The bedside physician should never be in the position of having to decide between competing interests.”

The recommendations advise hospitals and practices to form drug-shortage committees and adopt a clear and public drug-allocation method that maximizes the number of children’s lives saved, based on evidence of a drug’s importance in improving the chances of survival. The paper describes several actual examples of rationing situations.

The guidelines are limited to pediatric oncology. The task force did not address drug allocation among children and adults with cancer nor in patients with other diseases commonly treated with chemotherapy agents, such as rheumatoid arthritis. However, the ethical framework could be adapted for these situations, Dr. Unguru explained.

“Although we make it clear that our recommendations apply to children with cancer, the principles and the decision-making process we rely upon are very applicable across other diseases and to adults as well,” Dr. Unguru said.

Drug-Shortage Committees and Appeal Process

Institutions that have not already formed a multidisciplinary drug-shortage committee should establish one now, the guidelines state. The committee should include physicians, nurses, psychosocial clinicians, pharmacists, administrators, ethicists, and patient and family representatives. A process for appealing the committee’s decisions should also be established, the guidelines note.

The guidelines recommend ways to mitigate a shortage by maximizing efficiency and minimizing waste and include steps for prioritizing drugs for patients across and within pediatric cancers. To mitigate shortages, the task force recommends 11 steps, including the following 3: (1) Hospitals should not hoard drugs by ordering more supply than typically is needed; (2) Hospitals that lack one or more drugs required by a patient should attempt to secure a supply from another institution or refer the patient to another institution, rather than delay treatment or select an inferior regimen; (3) Permission to administer an expired drug should be sought when evidence supports the safety of doing so.

Some COG institutions have reduced drug waste by bringing children together for treatment at the same time, because agents like methotrexate often come in vials containing much more drug than is needed for one patient, Dr. Unguru revealed. Before drug shortages became more common, the remaining drug in the vial was thrown away.

In making allocation decisions, the task force makes the following recommendations:

  • Hospitals should make allocation decisions transparent and public.
  • Decisions should be supported by evidenced-based recommendations whenever possible.
  • Clinical trial participants should not receive priority over nonparticipants, to avoid any appearance of coercion. 
  • “Curability” and prognosis should be emphasized. 
  • Consider the incremental importance of a particular drug to a given patient’s outcome.
  • Give consideration to patients with less curable diseases for whom the scarce drug offers the chance of cure.
  • Consider the phase of therapy (see article for specifics).
  • Amount of drug required should be considered; a patient with a curable disease who requires a small amount of a drug should receive priority over those with difficult-to-treat diseases that require larger amounts of the drug.
  • In a clinical trial, use of a scarce drug within the standard-of-care backbone should receive priority over investigational use of the same drug.
  • Patient age, development, ability to pay, race, ethnicity, disability, immigration status, and weight or body surface area should not be considered in drug-allocation decisions.
  • Patients and families must be informed of decisions about their treatment, preferably through a letter from the institution explaining the allocation decision-making process.

The seven-member task force included experts in pediatric oncology, nursing, psychiatry, research ethics, palliative care, pharmacy, pharmacology, and bioethics. A parent advocate represented the interests of children and their parents. The paper went through roughly 10 internal revisions prior to review by an independent advisory committee, COG and ASPHO leadership, and publication peer review, Dr. Unguru said.

‘A National Disgrace’

Although drug shortages appear to have peaked in 2012 and lessened slightly since, the American Society of Health-System Pharmacists and the U.S. Food and Drug Administration currently list about 150 therapeutics in shortage, including many cancer drugs. Reasons for shortages include safety issues, manufacturing delays, and business decisions by drug makers to discontinue products that do not earn high profits. 

“This is largely a U.S. problem,” Dr. Unguru said. “Other countries occasionally experience shortages, but they don’t last as long, and they certainly don’t involve as many drugs.”

The rationing guidelines won’t resolve the shortage problem, Dr. Unguru admitted. “The solution is to change the way our health-care system works—What we need is a policy shift to prevent these shortages from ever occurring. The fact that children with cancer cannot obtain the lifesaving drugs needed to cure their diseases is a national disgrace,” he said. “Right now, we’re trying to put the fire out, but we really need to prevent the fire from starting in the first place.” ■

Disclosure: Dr. Unguru reported no potential conflicts of interest.

Reference

1. Unguru Y, Fernandez CV, Bernhardt B, et al: An ethical framework for allocating scarce life-saving chemotherapy and supportive care drugs for childhood cancer. J Natl Cancer Inst 108(6):djv392, 2016.



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