FDA should help any new federal comparative effectiveness research agency
by establishing written guidelines about when information is sufficiently rigorous to lead to action by other government entities.
That’s the view of American Enterprise Institute (AEI) resident fellow Scott Gottlieb, a former FDA deputy commissioner,
and regulatory attorney Coleen Klasmeier (Sidley Austin). The two say in an AEI Outlook article that FDA’s “substantial clinical experience” standard is appropriate
for judging the value of comparative research findings.
Gottlieb and Klasmier say that the substantial clinical
experience standard encompasses a broader range of clinical data than the “substantial evidence” standard and
thus is best suited for use with comparative effectiveness research, which will usually rely on epidemiological data, reviews
of databases, and registries.
With so much policy interest in establishing a formal framework for comparative effectiveness
research, the two say, FDA could play an important public health role by more clearly establishing how a substantial clinical
experience definition could assist in substantiating comparative effectiveness research and deciding when sponsors should
share the information with consumers and doctors and when government agencies should use the information for clinical and
policy decision-making. “Adopting a uniform definition for substantiation that is consistent across FDA and the proposed
comparative effectiveness research agency would also allow FDA to play its traditional role in defining standards for making
recommendations on the basis of research,” they write.
Gottlieb and Klasmeier say that it is unrealistic
to believe that over the long run federally-generated comparative effectiveness research results won’t be used by agencies
such as Medicare to inform access and pricing decisions. But, they say, that makes the need for a uniform standard all the
more important.
