NPC Testifies on Priorities for Comparative Effectiveness Research

Good afternoon. My name is Dr. Les Paul, Vice President for Clinical and Scientific Affairs at the National Pharmaceutical Council (NPC). On behalf of NPC, I would like to thank you for inviting us to comment today on the setting of national priorities for comparative effectiveness research (CER). This is a critical discussion focused on providing health care decision makers with timely, balanced, and high quality clinical evidence to help inform their decisions and improve patient health outcomes.


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.


Chronic Diseases Afford Greatest Impact

It is clear today that health care costs are rising at an unsustainable rate, making it reasonable for the Institute of Medicine (IOM) to recommend that the prioritization of CER be biased toward medical conditions with the greatest impact on morbidity and cost. These include chronic conditions such as cardiovascular disease, chronic respiratory diseases, cancer, diabetes, arthritis, and serious mental health conditions. CER should not be limited to the drugs used to treat those conditions, but rather, it should be extended to all relevant health care services including medical and surgical procedures, diagnostics, and medical devices. In addition, this research should include alternative health care delivery methods and insurance benefit designs. Proposed prioritization of research topics and studies in these areas of medicine, 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.


Key Additional Factors for Consideration

It also will be important for the IOM to consider other key factors in the selection of the highest priority research.

  • First, it should conduct 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.
  • Second, it is necessary to study how best to employ CER in a manner that preserves incentives for the continuous innovation of health care technologies in areas of unmet need.
  • Third, it should be a priority to study how CER at a broad population level can be balanced with the goals and rapid scientific advancements in the area of personalized and stratified medicine in order to encourage the development of targeted therapies for subpopulations.
  • Fourth, 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.
  • Lastly, there should be 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.

The National Pharmaceutical Council welcomes the opportunity to bea part of this critical dialogue and stands ready to assist the Committee as it moves forward with its CER prioritization recommendations.

Thank you.