Elsevier

The Lancet

Volume 358, Issue 9294, 17 November 2001, Pages 1676-1681
The Lancet

Articles
Priority-setting decisions for new cancer drugs: a qualitative case study

https://doi.org/10.1016/S0140-6736(01)06714-9Get rights and content

Summary

Background

Frameworks for legitimate and fair priority setting emphasise the importance of the rationales for priority setting decisions. However, priority setting rationales, in particular for new cancer drugs, are not well described. We describe the rationales used by a committee making funding decisions for new cancer drugs.

Methods

We did a qualitative case study of a priority setting committee (Cancer Care Ontario Policy Advisory Committee for the New Drug Funding Program) by analysing documents, interviewing committee members, and observing committee meetings.

Findings

We identified and described decisions and rationales related to 14 drugs in eight disease conditions over 3 years. Our main findings were that: priority setting existed in relation to resource mobilisation; clinical benefit was the primary factor in decisions; in the context of an expanding budget, rationales changed; rationales could change as costs for individual treatments increased; when all options were reasonable, groups funded a range of options and let patients decide; and priority setting rationales involve clusters of factors, not simple trade-offs.

Interpretation

Observing priority-setting decisions and their rationales in actual practice reveals lessons not contained in theoretical accounts.

Introduction

A goal of priority setting is justice, which involves legitimate authorities using fair processes. Frameworks for legitimate and fair priority setting emphasise the importance of rationales for particular priority setting decisions.1, 2 However, priority-setting rationales are not well described.

Priority-setting rationales are important in both primarily private (eg, USA) and public (eg, UK, Canada) health-care systems. In primarily public systems, rationales are more often open to public deliberation, whereas in primarily private systems, in which there is no democratic political mechanism for health-care priority setting, rationales are often implicit.

Surveys of the public have explored rationales as hypothetical “trade-offs” such as lifesaving technologies versus community services,3 withholding of life-prolonging medical care from critically ill elderly people,4 equity versus cost-effectiveness or good outcomes,5, 6 “do-no-harm” principle versus maximising outcomes,7 helping the worst-off versus maximising outcomes,8 and personal treatment preferences versus abstract measures of utility.9 However, survey methods frame inquiries rigidly, precluding deeper insights into inherently complex issues that are not as neat and discrete in reality as they seem on a survey instrument. For example, representing priority setting as a trade-off between equity and efficiency oversimplifies a very nuanced decision. Moreover, public opinion regarding hypothetical scenarios does not permit generalisations to actual decision making. Only a few empirical studies have examined actual priority setting,10, 11, 12, 13, 14, 15 and these studies tend to focus narrowly on discrete technologies.

Cancer is the leading cause of death in Canada (27·2% of all deaths).16 Because the cost of new cancer drugs is rising dramatically,17 priority setting for new cancer drugs is critical. The National Institute of Clinical Excellence (NICE), on behalf of the UK's National Health Service, conducts appraisals of new technologies, including new cancer drugs, and recommends which should be made available to patients.18 However, NICE's priority-setting rationales (eg, docetaxel and paclitaxel for breast cancer) are limited to factors related to evidence of clinical outcomes.19 To our knowledge, only one study has explored rationales in priority setting for cancer. Foy and colleagues20 described a collaboration between a specialist cancer hospital and six regional health authorities in the UK with respect to funding new cancer drugs. They reported that funding decisions were based on evidence thresholds determined from information on effectiveness; the evidence thresholds were affected by political pressures, financial constraints, and the value placed on some clinical outcomes. The limitation of this study is that only four selected cases were examined in detail and, though the factors affecting decision making were described, the specific rationales for each decision were not. In a previous paper, we described a model of priority setting for new cancer drugs as a diamond having six inter-related facets, one being the rationales used in priority-setting decisions.21 To our knowledge, there is no in-depth description or analysis of rationales for priority setting decisions regarding new cancer drugs.

The purpose of this paper is to examine priority setting for new cancer drugs; specifically, we aim to provide an overview of decisions and rationales used, and describe how the rationales are assembled.

Section snippets

Design and setting

We have done a qualitative study of priority-setting decisions and rationales for new cancer drugs made by Cancer Care Ontario, a provincial disease management organisation responsible for cancer care in the province of Ontario, Canada. The analysis presented here was part of a larger qualitative case study of priority setting for new technologies in cancer and cardiac care.21

In this paper, we have focused on the Cancer Care Ontario Policy Advisory Committee for the New Drug Funding Program,

Results

During the study period, the Cancer Care Ontario Policy Advisory Committee considered 14 drugs for eight diseases. These decisions and their rationales are summarised in the appendix (available from the The Lancet offices and the authors at www.utoronto.ca/jcb/Research/prioritysetting/lancetaddendum2001.htm). Panel 1 summarises what we have identified from our study of these decisions regarding rationales for priority setting of new cancer drugs.

In the first few meetings, the committee members

Discussion

We have provided a detailed description of priority-setting decisions and rationales pertaining to new cancer drugs in the context of a provincial cancer organisation and drawn six important lessons from this description. The rationales used by the decision makers were shaped by the specific institution and process in which they worked. The primary limitation of this study is that the findings may not be fully generalisable. However, generalisability is not a goal of qualitative research.

References (30)

  • C Ham

    Priority setting in the NHS: reports from six districts

    BMJ

    (1993)
  • T Hope et al.

    Rationing and the health authority

    BMJ

    (1998)
  • HJ Aaron et al.

    The painful prescription: rationing hospital care

    (1984)
  • J Elster
  • R Zussman

    Intensive care: medical ethics and the medical profession

    (1992)
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