Which Orphans Will Find a Home? The Rule of Rescue in Resource Allocation for Rare Diseases

EMILY A. LARGENT & STEVEN D. PEARSON

Hastings Center Report, Volume 42, Issue 1, pages 27–34, January-February 2012

Abstract

The rule of rescue describes the moral impulse to save identifiable lives in immediate danger at any expense. Think of the extremes taken to rescue a small child who has fallen down a well, a woman pinned beneath the rubble of an earthquake, or a submarine crew trapped on the ocean floor. No effort is deemed too great. Yet should this same moral instinct to rescue, regardless of cost, be applied in the emergency room, the hospital, or the community clinic?

In health care, the desire to save lives at any cost must be reconciled with the reality of resource scarcity. As one example, the estimated cost for prophylactic Factor VIII to treat one patient with hemophilia for one year is $300,000. Costs of this magnitude have been accepted by public and private insurers in the developed world, even though, in principle, these sums could provide greater overall health benefit if allocated to pay for the unmet health care needs of many other patients. Looking forward, however, broad application of the rule of rescue will be increasingly untenable. But the moral instinct will remain: the desire to help those weakest among us, especially when their small numbers allow us to see them as unique individuals. What, then, is the ethical framework that can guide coverage and reimbursement decisions for orphan drugs into the future?

Article

I call this … the rule of rescue. Our moral response to the imminence of death demands that we rescue the doomed. We throw a rope to the drowning, rush into burning buildings to snatch the entrapped, dispatch teams to search for the snowbound. This rescue morality spills over into medical care, where our ropes are artificial hearts, our rush is the mobile critical care unit, our teams the transplant services. —Albert Jonsen

The rule of rescue describes the moral impulse to save identifiable lives in immediate danger at any expense. Think of the extremes taken to rescue a small child who has fallen down a well, a woman pinned beneath the rubble of an earthquake, or a submarine crew trapped on the ocean floor. No effort is deemed too great, and seemingly unlimited resources are expended to save these individual lives. To do less would be deemed unconscionable. Yet should this same moral instinct to rescue, regardless of cost, be applied in the emergency room, the hospital, or the community clinic? In health care, the desire to save lives at any cost must be reconciled with the reality of resource scarcity.

The treatment of patients with rare, or “orphan,” diseases raises persistent ethical dilemmas of this nature. In America, an orphan disease or condition is defined by law as one that affects fewer than 200,000 people. Six to seven thousand different rare diseases have been identified  and approximately five new rare diseases are described in the medical literature every week. Orphan diseases are mostly chronic, and many are life-threatening or significantly life-altering. Familiar examples include Huntington disease, amyotrophic lateral sclerosis (commonly known as Lou Gehrig disease), and cystic fibrosis. Drugs developed to treat orphan diseases are known as orphan drugs and are usually extremely expensive. As one example, the estimated cost for prophylactic Factor VIII to treat one patient with hemophilia for one year is $300,000.

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Read Dr. Doris T. Zallen’s , response to the report
Professor of Science and Technology Studies at Virginia Tech