How Should Therapeutic Decisions about Expensive Drugs Be Made in Imperfect Environments?

Abstract

Clinicians must inevitably make therapeutic decisions under nonideal conditions. They practice in circumstances that involve incomplete evidence. They deliver care in health care systems that are complex and poorly coordinated. Each of the patients that they take care of is unique while research offers evidence regarding relatively homogeneous populations of patients. Under these circumstances, many partiesmedical scientists, reviewing agencies, insurers, and accountable care organizations-can and should contribute to optimizing the development, approval, funding, and prescription of therapiesparticularly expensive and marginally beneficial therapies. In aggregate, they should aspire to achieve a pattern of fair, cost-effective therapeutic decisions to ensure a sustainable health care system. Here we offer some suggestions regarding decisions that physicians might pursue to facilitate fair and cost-effective patient care. CaseDr. C sits on a committee as part of his tertiary care center's accountable care organization (ACO) that is considering whether a new biologic, Expensivimab, should be included in the organization's bundled treatment plan for its patients. Expensivimab is a new humanized antibody that targets an apoptotic receptor. One study suggests that it increases tumor-free survival in late stage non-small cell lung cancer (NSCLC) by a median of six months relative to a drug approved several years ago. Another suggests that it increases overall survival by a month-and-a half relative to the same drug. Yet no research on the comparative effectiveness of Expensivimab relative to other interventions for NSCLC exists, and even data on the risks of Expensivimab relative to the older drug is scant.Furthermore, assessing Expensivimab's cost-benefit ratio is difficult. Although Expensivimab costs $750,000 per patient, the United States Food and Drug Administration (FDA) did not request any data on trial participants' perceptions of their quality of life. Hence, no assessment of quality-adjusted life years (QALYs) or disability-

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