National public insurance for drugs is often based on evidence of comparative effectiveness
and cost-effectiveness. This study describes how that evidence has been used across
3 jurisdictions (Australia, Canada, and Britain) that have been at the forefront of
evidence-based coverage internationally.
To describe how clinical and cost-effectiveness evidence is used in coverage decisions
both within and across jurisdictions and to identify common issues in the process
of evidence-based coverage.
Descriptive analysis of retrospective data from the Common Drug Review (CDR) of Canada,
National Institute for Health and Clinical Excellence (NICE) in Britain, and Pharmaceutical
Benefits Advisory Committee (PBAC) of Australia. All publicly available information
as of December 31, 2008, was gathered from each committee's Web site (data set begins
in January 2004 [CDR], February 2001 [NICE], and July 2005 [PBAC]).
Listing recommendations for each drug by disease indication.
NICE recommended 87.4% (174/199) of submissions for listing compared with a listing
rate of 49.6% (60/121) and 54.3% (153/282) for the CDR and PBAC, respectively. Significant
uncertainty around clinical effectiveness, typically resulting from inadequate study
design or the use of inappropriate comparators and unvalidated surrogate end points,
was identified as a key issue in coverage decisions. Recommendations varied considerably
across countries, possibly because of differences in the medications reviewed; different
agency processes, including the willingness to negotiate on price; and the approach
to "me too" drugs. The data suggest that the 3 agencies make recommendations that
are consistent with evidence on effectiveness and cost-effectiveness but that other
factors are often important.
NICE, PBAC, and CDR face common issues with respect to the quality and strength of
the experimental evidence in support of a clinically meaningful effect. However, comparative
effectiveness and cost-effectiveness, along with other relevant factors, can be used
by national agencies to support drug decision making. The results of the evaluation
process in different countries are influenced by the context, agency processes, ability
to engage in price negotiation, and perhaps differences in social values.