Decision-making processes regarding cancer technologies: a review.

• Author: Stafinski, Tania
• Position: Canada

The issue of timely access to high quality cancer care has heightened in Canada, fueled in part by incidence rates reaching levels where cancer now touches, in some way, the lives of most Canadians. (1) Thus, deliberations over how best to maximize the health of cancer patients are often emotionally and politically charged. Two major issues have been highlighted through the media and the political process: (1) timely access to required services (i.e., waiting times for radiation, surgery and specialist consultation), (2) limits placed on equitable access to services such as diagnostic interventions (e.g., MRI and PET) and (3) cancer drug treatments. As cancer agencies across the country look for ways to provide timely access to technologies that deliver high quality care, they do so facing unlimited demands, limited budgets, and intense scrutiny by both those affected and the media. There seems to be a growing lack of public confidence in decisions about which technologies to publicly fund, particularly in the case of promising, often high cost innovations championed by physicians, patients and manufacturers. As a result, policy-makers charged with the task of ensuring prudent and principled use of scarce resources have become demonized, since the essence of priority-setting means that access to some will be denied.

It has become increasingly clear that approaches to priority-setting require a blending of two decisional domains: effectiveness and efficiency (the evidence-based paradigm) and equity/fairness (the values/ethics based paradigm). What remains unclear are ways of marrying the two so that coverage decisions may be deemed legitimate and fair by all stakeholders.

Over the past three decades, an international body of work focusing on resource allocation and technology funding decision-making has emerged in response to the increasing pressures faced by governments to adopt new and innovative technologies into their health care systems. (2) It contains research which draws from many disciplines including: clinical medicine; epidemiology; political, social, behavioral and management sciences; economics; and ethics. This work examines how decisions are actually made (i.e., descriptive processes), how they should be made (i.e., normative processes), and tools for aiding or informing these processes. (3) While the majority of this work has taken place outside of the cancer context, it addresses issues of a similar nature and complexity. This paper provides an overview of this work to inform both scholars (in ethics, law and health services research) and decision-makers in Canadian health care systems.

Descriptive Processes

Canadian research describing how funding recommendations or decisions for new cancer technologies are actually made is limited to a few case studies in Ontario. (4) Factors shaping decisions were identified through a combination of document analysis, interviews of Cancer Care Ontario's Policy Advisory Committee, and observations of their meetings. These factors included: benefit to patients and its magnitude, quality of evidence (i.e., the degree of certainty of the benefit), existence of alternatives, treatment duration, total population of patients affected, total cost to the system, pressure from physician and patient groups, and historical precedent.

Cost-effectiveness was not used, but the committee discussed the concept of value-for-money. Although the committee had initially considered developing a list of funding recommendations, ranking them, comparing the costs against a known fixed budget, and then drawing a line at the end of the resources, it abandoned the idea when members failed to agree upon potential priority measures. (5)

A similar study was conducted in the United Kingdom, where a specialist cancer hospital and a consortium of six regional health authorities set priorities for funding new drugs. (6) Decisions were based on evidence thresholds determined from evidence of clinical effectiveness. Of 16 proposed new drugs, only the seven that had so-called 'Category I' evidence (proven effectiveness over and above existing treatments) were funded. Key challenges reported in the study included: 1) a lack of comparative data on validated, relevant health and economic outcomes in new drug trials, which precluded their use in cost-effectiveness analyses, 2) disagreements over values placed on such outcomes, and 3) the absence of criteria used to determine what constituted satisfactory evidence of effectiveness. This, in turn, led to a call for the establishment of an agreed-to set of criteria before any future negotiations. Lastly, pressures from some health authorities to raise the threshold because of their individual financial constraints and from clinicians to lower the threshold to reflect their patients' expectations were reported. (7)

Most of the remaining literature on processes for making technology funding decisions have come from experiences of national systems in the U.K., Canada and the U.S., and have reported on decisions across diseases or conditions (as opposed to just cancer). (8) The National Institute for Health and Clinical Excellence [NICE] in the U.K. provides funding recommendations to the National Health Service [NHS] in England and Wales on the use of new and established health technologies. (9) Guidance is based on an appraisal of the technology's health benefits (impact on quality of life and survival) and associated costs to the NHS. The appraisal, itself, comprises evidence from a technology assessment (typically a systematic review of clinical effectiveness data and an economic evaluation) and input from patient representatives, caregivers and health professionals, and manufacturers. Such information, along with other prescribed factors (including the government's priorities, clinical need, benefit-cost balances, potentially available resources and their effective use, and encouraging innovation that will benefit patients) are considered by an Appraisals Committee...