Lessons from Abroad

Comparative effectiveness research is a relatively new term in U.S. health policy discourse, but the concept has been embraced for years by many other countries. Some of them, like the United Kingdom, have single-payer systems, whereas others have insurance-based ones—for example, Germany (with a predominantly public insurance system) and the Netherlands (where private insurers play a larger role). Two reviews published earlier this year in the Milbank Quarterly and the Euro Observer assessed how comparative effectiveness research is used in Australia, France, Germany, the Netherlands, Sweden, and the United Kingdom.¹ They concluded that health technology assessment (HTA) systems have played central, if not transformative, roles in contributing to evidence-based decision-making and in identifying interventions that provide the most value for money.

For each of the countries studied, the hub of comparative effectiveness research is an HTA agency, although the scope of its assessments varies from country to country. Australia's Pharmaceutical Benefits Scheme (PBS), for example, concentrates on pharmaceuticals only, whereas the French Haute Autorité de Santé (HAS) and the United Kingdom's National Institute for Health and Clinical Excellence (NICE) assess a wide range of other measures—the latter, for instance, evaluating treatments, diagnostic tests, procedures, medical devices, and public health programs. The agencies also differ in the extent and methods of considering the costs of interventions: while NICE has been charged from its inception with taking cost into account, other HTAs have begun to do so only recently.

HTA agencies make their assessments based on evidence synthesized from literature reviews and data from manufacturers. While much work is intramural, all HTA agencies also outsource some work to academics. In addition, the agencies draw on the methodological work of the Cochrane Collaboration, an international, not-for-profit organization that plays an important role in the field of evidence-based health care.

HTA agencies each occupy a different place in their particular country's health care system and, as a result, their work has different degrees of impact, especially regarding practice guidelines and coverage decisions. While NICE is at arm's length from government, it is highly integrated in the health care system and issues guidance directly to the National Health Service. Much of its work is binding, legally entitling patients to interventions it has approved. However, guidance on, for example, disease management strategies and health promotion initiatives is primarily advisory. The German Institut für Qualität und Wirtschaftlichkeit im Gesundheitswesen (IQWiG) and the French HAS are equally at arm's length from insurers and government; however, their assessments have no direct impact on patient access or entitlement. Rather, other bodies or government departments draw on their data and decide how to use them in coverage decisions.

Evaluating Medications

All HTA agencies evaluate the effectiveness of medications, which typically takes between six months and two years, depending on the methodology. All countries prefer randomized clinical trial data because they are generally regarded as the most valid source of evidence. While the distinction is not clear-cut, Denmark, France, and the Netherlands draw primarily on evidence from manufacturers' submissions and study reports, whereas Sweden, Germany, and the United Kingdom focus more on systematic reviews and analyses from clinical studies, although industry submissions are also considered in some cases, and the United Kingdom considers economic analyses. France, the Netherlands, and the United Kingdom have created fast-track systems for highly innovative drugs that typically rely to a larger extent on manufacturers' data. While the emphasis is on the market-entry phase of new drugs, countries like the Netherlands and Sweden also reevaluate drugs to identify those with insufficient value for money.

Value for money is typically considered by relating an intervention's effectiveness data to its price, usually expressed in cost per quality-adjusted life year, or QALY. For example, the Netherlands would generally not regard interventions costing more than $30,000 per QALY as cost-effective, whereas the threshold is around $45,000 in the United Kingdom, and $65,000 in Sweden. It is not uncommon for opponents of QALY-based health technology assessment to suggest that such thresholds are...