Good afternoon. My name is Gary Persinger, Vice President for Health Services Research at the National Pharmaceutical Council (NPC). On behalf of NPC, I would like to thank you for providing this opportunity to comment on comparative effectiveness research (CER) and the activities of the Coordinating Council. 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 CER priorities to focus on 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. The 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 Federal Coordinating Council to consider several key questions as it assesses the CER-related research conducted by Federal agencies and departments and develops recommendations for selection of the highest priority research.
- First, what strategies can be employed 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, how can CER be employed optimally in a manner that preserves incentives for the continuous innovation of health care technologies in areas of unmet need?
- Third, how can CER at a broad population level 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, what research should be conducted 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? This research should include, but not be 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, what support is required 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 be a part of this critical dialogue and stands ready to assist the Coordinating Council as it moves forward in developing recommendations to ensure coordination and best use of resources for CER. Thank you.