Objectives: Our objective was to gather perspectives from payers on how
comparative effectiveness research (CER) in the United States and relative effectiveness
(RE) research in Europe will impact evidentiary standards for access decisions of new
drugs by 2020.Methods: We conducted semi-structured interviews with fourteen senior
officials representing public and private payers, health technology assessment groups, and
pricing and reimbursement bodies in the United States and Europe. An online survey
assessed current use of CER/RE evidence and potential trends that might influence its use
for decision making by 2020. A semi-structured interview elicited payers' definitions of
CER/RE and was structured around four hypothetical cases resembling drugs expected to be
more common or poised to create policy challenges by 2020. Topics included acceptance of
study designs and analytic methods associated with CER/RE. A systematic content review was
done to extract relevant information.Results: According to key informants, randomization will remain an essential
component for assessing comparative or relative effectiveness. They anticipate greater use
of policy levers such as conditional reimbursement or prior authorization to manage
diffusion of new drugs. Case studies provided important insights into situations when
certain types of CER evidence may be acceptable (e.g., observational data when differences
between drugs are largely convenience).Conclusions: Industry perceptions that CER/RE will change payers'
evidentiary requirements in the future are consistent with our findings. Growing
investment in payers' own data and increased reliance on policy tools to control diffusion
of new drugs may also influence the type of evidence industry will be required to produce
by 2020.