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Panel issues framework for allocation of scarce pediatric oncology drugs
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Pediatric oncology and bioethics experts have issued a framework for the allocation of scarce drugs to reduce waste and prioritize treatment across and within diseases, according to a commentary published in Journal of the National Cancer Institute.
Because there is a lack of formal and concrete guidance for allocation of drugs in shortage, this framework is intended to support reasoned decision-making and minimize biases that occur when health care rationing decisions are made, according to the members of the Allocation Task Force, which was convened as a directive of the Children’s Oncology Group.
Yoram Unguru, MD, MA, MS
“Physicians and administrators faced with having to decide which of two children with cancer receives a scarce lifesaving treatment need guidance and should not feel that they are on their own without a roadmap,” Yoram Unguru, MD, MA, MS, assistant professor in the school of medicine and a pediatric hematologist/oncologist at The Herman and Walter Samuelson Children’s Hospital at Sinai and the Berman Institute of Bioethics at Johns Hopkins University, said in a press release. “The context of allocation is always complex, but it is unethical to leave these challenges unaddressed. It is our hope that this framework will be helpful and spur further substantive action on this critical issue.”
The framework consists of a two-step allocation process: mitigation and allocation.
Mitigation
The task force members wrote that “the first response to a shortage of chemotherapy and supportive care agents should be to maximize efficiency and minimize waste.”
Mitigation strategies the task force identified to achieve these goals included:
- Verify if a scarce drug is listed on the drug shortage websites of the FDA or the American Society of Health System Pharmacists;
- Determine the supply and the anticipated length of time the shortage will continue;
- Anticipate the drug needs of current and future (estimated) patients;
- Contact the manufacturer to see if larger or smaller vial sizes of the drug are available if a certain drug brand or strength is not available;
- Select alternate therapies with similar safety and efficacy profiles, when applicable;
- Put patients into cohorts so they may share vials that may otherwise have been meant for single use;
- Borrow or share drugs from other institutions;
- Administer the scarce drug out of sequence if the shortage is meant to be short-lived;
- Compound drugs if possible, or acquire it from a commercial compounding pharmacy or through FDA lending; and
- Consider extending drug usage beyond a labeled expiration date if stability and sterility profiles support it.
Additionally, the panel identified a list of stakeholder responsibilities during a drug shortage. These included that pharmacists should order drugs based strictly on a hospital’s needs; that manufacturers and distributors prevent drug hoarding by internally allocating drugs in a fair and transparent manner based on a hospital’s ordering history; that an institutional drug shortage committee arbitrate challenges to allocation decisions; that hospital administrators assure timely treatment; that patients or their advocates proactively engage in the allocation decision-making process; and that the FDA potentially secure an adequate supply of a drug from a non-U.S. supplier until the shortage has been alleviated.
Allocation
The task force suggested there are three critical factors when determining the allocation of short-supply drugs: curability, prognosis and the incremental importance of a specific drug to an individual child’s outcome.
The framework includes 10 considerations for allocation, such as that clinical trial participants should not receive priority for drug access; patient age, development, ability to pay, ethnicity, disability and immigration status should not play a role in allocating scarce drugs; and patients and families should be apprised of decisions regarding treatment.
“In formulating [these] recommendations, in addition to maximizing lives, [we] considered several overarching principles, including, but not limited to, fairness, maximizing benefit and minimizing harms,” Unguru and colleagues wrote. “All similarly situated people deserve the same opportunity to receive the scarce drug. As a corollary, fairness requires justifying decisions to treat some people differently.”
The framework also includes different considerations for representative diseases such as acute lymphoblastic leukemia, germ cell tumor, non-Hodgkin’s lymphoma, osteosarcoma, rhabdomyosarcoma and Wilms’ tumor.
“While health care rationing is inevitable, rationing life-saving chemotherapeutics and supportive agents raises many ethical challenges,” the task force concluded. “Physicians lack sound guidance in making ethically appropriate decisions for allocating scarce drugs. In the absence of a much-needed national advisory statement on how best to allocate scarce drugs and until policymakers and stakeholders can prevent future shortages, the guidance articulated here supports reasoned decision-making in the fact of an actual drug shortage and aims to minimize bias as might occur when individual clinicians or institutions are forced to make difficult, and at times tragic, rationing decisions for children with cancer.” – by Anthony SanFilippo
Disclosure: Unguru reports a consultant/advisory role with Biosciences. One other researcher reports consultant/advisory roles with Jazz and Merck.
Perspective
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Stephen P. Hunger, MD
Unguru and colleagues have tackled the difficult problem of developing an ethical framework for how essential chemotherapy and supportive care agents (CASCA) should be allocated in a time of scarcity. They summarize potential mitigation strategies and allocation considerations.
Until relatively recently, most U.S. physicians, pharmacists, members of the medical care team, and patients and their families never considered that there might be shortages of essential CASCA. We assumed that an FDA-approved drug would be available if and when it was prescribed for an individual patient. We knew that it might be expensive and require discussions with insurers to gain approval, but we all assumed that CASCA would be there when we needed them.
Unfortunately, as we have faced a variety of shortages of CASCA over the past 5 to 10 years, we have all learned that our assumptions were wrong.
So how should we all behave? When a scarcity occurs, the “knee jerk” reaction is to try and purchase and stockpile supplies of the CASCA so that we are sure that we will have a supply when we need it. As pointed out by Unguru and colleagues, this is both counterproductive — the shortage will be worse if everyone hoards the drug — and problematic ethically. The suggestions made regarding mitigation strategies are thoughtful and likely to provoke very little disagreement.
The harder task is how to allocate CASCA in the case of a true shortage, where not all patients can be treated on schedule with the given agent. The authors’ suggestions are well considered, but could be very challenging to implement.
How do we to tell Johnny and his family that Sally will get the vincristine and he won’t? Missing one or two doses of a chemotherapy drug probably has minimal effect in the context of a complex multiagent chemotherapy regimen, but it will not feel that way to the patient and their family. It will feel like they are being denied a dose of a drug that is essential to their cure.
What impact will that have on their psyche as they struggle with cancer therapy? If Johnny is not cured, will his family accept later that he probably would not have been cured even if he received all the doses of vincristine?
The Joint Commission requires hospitals to have an emergency operations plan that describes how a facility will respond to and recover from all hazards. Many of us have participated in hospital drills that practice response to a natural or man-made disaster.
This commentary challenges us to do the same regarding shortages of CASCA. As we do this, we must educate ourselves about how to make difficult decisions regarding drug allocation and, critically, develop tools to educate patients and their families about this process.
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