Comparative effectiveness and relative efficacy: Expectations for drugs in the US and EU in 2020

By William Chin, PhD, Project Manager, EUCRAF

A paper entitled “The future of comparative effectiveness and relative efficacy of drugs: An international perspective” was published in the March 2015 issue of the Journal of Comparative Effectiveness Research. In this article, Messner DA et al. from Center for Medical Technology Policy addressed the question of how the evidence requirement for comparative effectiveness research (CER) in the US and relative effectiveness (RE) in the EU will evolve in the next 5 years.

Citing the definition from the Institute of Medicine in the US, the authors defined CER as ‘the generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care.

Likewise in the EU, the European Commission Working Group on Relative Effectiveness has defined relative efficacy or effectiveness as comparisons of benefits and harms of alternative interventions made either under ideal conditions (efficacy) or usual circumstances of practice (effectiveness).

The authors reported that the main driver for the change in the future US scenarios is the degree of healthcare integration, specifically the degree to which healthcare integration shifts risk acceptance for patient care from payers to provider organizations. The healthcare integration is defined as “the extent to which hospitals, multispecialty care delivery and other services, and coverage become integrated into a comprehensive system for delivering care to members”.

On the other hand, pan-European initiatives related to the expectations and decision-making of regulatory and HTA bodies, and the degree to which alignment can be achieved between them, were identified as the underlying drivers of change in the EU.

Furthermore, the authors implied that the FDA is not seen as a key driver for change in the USA as there are currently no formal FDA policy changes anticipated to impact expectations for CER. In addition, the FDA is assumed not to coordinate scientific advice for drug developers with payers.

On the contrary, the launch of parallel scientific advice by the EMA and HTA bodies is seen as major milestone in the EU and EMA is likely to require postauthorization efficacy studies including effectiveness studies.

The authors envisaged that there will not be a drastic change in the near future scenarios for FDA with regard to policies that recognize the evidence needs of payers. However, in Europe, the EMA will become more involved in commissioning post efficacy/effectiveness studies, and will be more favorable towards adaptive licensing.


1. J Comp Eff Res. 2015 Mar 2:1-9. The future of comparative effectiveness and relative efficacy of drugs: an international perspective. Messner DA, Towse A, Mohr P, Garau M.