NPC Submits Testimony on Draft CER Definition, Criteria and Framework

On behalf of the National Pharmaceutical Council (NPC), thank you for the opportunity to comment on the draft comparative effectiveness research (CER) definition, prioritization criteria, and the proposed strategic framework that was developed by the Federal Coordinating Council on Comparative Effectiveness Research (FCCCER). The definition, prioritization criteria, and the strategic framework for CER can play an important role in improving patient health outcomes, and we congratulate the FCCCER on this critical effort.

About the National Pharmaceutical Council

Briefly, the National Pharmaceutical Council sponsors and conducts scientific analyses on the appropriate use of pharmaceuticals and the clinical and economic value of improved health outcomes through pharmaceutical innovation. CER and its foundation of high quality scientific evidence are important areas of focus for NPC.  It is our goal to ensure that sound evidence is recognized by independent experts, considered appropriately by private and public payers, reflected adequately in benefit designs, and incorporated into clinical practice.  NPC was established in 1953 and is supported by the nation’s major research-based pharmaceutical companies.

Draft Definition of CER

When the $1.1 billion in federal funding was first allocated for CER, there were many unanswered questions regarding which projects would be prioritized, what kind of strategic framework would be developed, and how the research would be conducted and disseminated. To address those questions, NPC and other healthcare stakeholders provided recommendations in previous testimony that priorities for CER should:

  • Focus on conditions with the greatest impact on morbidity and cost, such as chronic conditions like cardiovascular disease, chronic respiratory diseases, cancer, diabetes, arthritis, and serious mental health conditions.
  • Include all major therapeutic options used to treat those conditions such as drugs, medical and surgical procedures, diagnostics, and medical devices. 
  • Take into account the needs of patient subgroups who may respond differently to therapeutic options based on demographic characteristics, genetic variation, and coexisting medical conditions; and
  • Express clear support for the development of new CER methodologies, such as analysis of non-randomized studies of treatment effects using secondary databases, practice-based clinical practice improvement studies, more accurate modeling and simulation techniques, and methodologies that ensure optimal interpretation and application of CER in a variety of patient care settings.

NPC is pleased that the draft definition of CER encompasses these important concepts in a broad and thoughtful manner.

Draft Prioritization Criteria

The general nature of the prioritization criteria allows for considerable flexibility in their interpretation. While they represent a good foundation, clarification is needed to ensure that CER funding decisions will be made in the best possible manner and result in useful information that improves clinical decision making for health care providers and patients.

In particular, NPC is concerned about the reference to the “time necessary for research,” and whether this would preclude lengthy or more in-depth projects from consideration. Proposed prioritization of research topics and studies, their associated research time frames, final study outcomes, and related information should be made transparent to all stakeholders and should be disseminated in a timely manner  To maximize this potential, the FCCCER should prioritize the funding of an  assessment of strategies to ensure the continuous evaluation of new evidence related to specific health care technologies – for example, how best to determine when a health technology assessment should be revised based on new clinical information. 

The criteria also suggest that CER “lays the foundation for future CER or generates additional investment.” CER not only lays the foundation for future CER, but also the foundation for future innovation.  How the agenda and conduct of CER develops has the potential to influence incentives for innovation and we would recommend that the study of this important question be an explicit interest of publicly funded CER. 

Additional Factors for Consideration in Priority Setting Under the Strategic Framework

Moving forward, it also will be important to consider other key factors in the selection of the highest priority research.

  • First, it will be important to conduct research to define rigorous, high quality, and validated CER methodologies that are focused on providing timely, accurate and balanced information in order to assist clinical decision making.
  • These questions include, but are not limited to, defining how best to address the full range of health effects of a new technology including quality of life, functionality, and productivity, as well as how best to appropriately characterize the strengths, weaknesses, and limitations of various underlying health technology assessment analytic techniques.
  • In order to minimize the likelihood for inaccurate or inappropriate interpretation of CER, we suggest the inclusion of a transparent and readily accessible description of the strengths, weaknesses, limitations, and potential for generalizability of the findings of CER utilizing varied experimental and non-experimental research designs.
  • Second, and consistent with our comment on the prioritization of the study of the impact of CER on innovation, the strategic framework should implicitly assume that innovative technology is an external input to the CER framework. It should be encompassed within and considered integral to the framework.
  • Third, the agenda for CER should be driven by the condition and the “key unanswered questions” in the context of that condition. Answering these questions may require comparisons between different types of technologies, processes, or procedures that may be considered to treat the condition; for example, the framework should reflect the need for comparisons of drug vs. surgery, drug and diagnostic vs. procedure, procedure vs. surgery, or other combinations.
  • Fourth, comparisons should also include delivery system architecture options, insurance plan designs, methods for primary/secondary prevention, and approaches to provider incentives to effect improvements in health.

The National Pharmaceutical Council appreciates the opportunity to take part in this critical dialogue and stands ready to assist FCCCER as it moves forward with the development of the CER definition and criteria. Thank you.