It’s Critical to Get Comparative Effectiveness Research Right

Tucked inside the economic stimulus bill signed into law by President Obama is $1.1 billion to fund comparative effectiveness research (CER) evaluating the effectiveness of competing medical treatments, technologies, drugs and devices.  The comparative effectiveness provisions in this bill also take steps toward the creation of an entity that would oversee this research, and there is already significant debate about the composition of such a council and its powers.

It’s critical to the health care system for policy makers to get this right by establishing an open and transparent process for the prioritization of CER topics for study to ensure that sound evidence leads to quality patient care. To determine what kind of framework might – or might not – be feasible for a CER entity in the U.S., policy makers can look to a number of CER and health technology agencies around the world.  One example is the United Kingdom’s National Institute for Health and Clinical Excellence (NICE), the organization that provides guidance to the U.K’s National Health Service on the clinical and economic effects of various medical treatments.  There are a number of lessons to be learned from what NICE does, according to a recent study of NICE commissioned by the National Pharmaceutical Council and written by University of York Professor Michael Drummond, along with Corinna Sorenson, Panos Kanavos and Alistair McGuire of the London School of Economics and Political Science.

NICE has a standardized system for determining the services it will –or will not – cover.  First, NICE receives suggested topics for assessments and guidelines from the U.K.’s Department of Health, clinicians, and other healthcare stakeholders, although priorities are set by the government in the U.K. Following systematic reviews of existing literature and economic modeling, along with input from a “wide range of stakeholders,” NICE prepares guidances and disseminates the information to the appropriate healthcare decision-makers. NICE is not without controversy, having come under fire from patients for its preliminary decision not to cover certain new drugs for the treatment of kidney cancer.

In their critical examination of NICE’s practices, the study authors suggest that, in order to make the most impact, any comparative effectiveness research “should consider all health technologies, not just drugs.” By reviewing all aspects of the healthcare delivery system, CER studies are more likely to improve health outcomes.

Second, CER should involve all major stakeholders, in a transparent manner, in the development of assessments, guidance, and commentary on the results of studies.  Third, CER should focus on making high quality assessments and should take a broad array of factors into account instead of focusing solely on clinical outcomes. Additionally, the National Pharmaceutical Council would point out that evidence should encourage and facilitate good decision-making by healthcare professionals and patients, recognizing and supporting the physician and the patient as the center of the decision-making process.

Given the complexity of the issues and the implications for our health care system, it’s important to learn from the U.K. and other countries that have tackled these issues. To this end, the National Pharmaceutical Council is undertaking a second study focusing on CER organizations in Scotland, Australia, Canada, Sweden, and the Netherlands that will be completed this spring.

As the CER process moves forward in the United States, there are several critical questions that policy makers must answer first: 1) How to deal with the role of rapidly advancing science in personalized medicine and the needs of patient subgroups who may respond differently to therapeutic options based on age, genetic variation, and other conditions; 2) How to rapidly adopt healthcare technology, which has been long proven to be effective in the management of chronic disease; 3) How to address the requirement that new CER be rigorous, transparent, and conducted with proven methodology; and 4) How to ensure that CER helps to incent healthcare technology innovation focused on improving health outcomes, quality of life, and productivity.

It’s clear that the debate over comparative effectiveness research will not be settled overnight, given the wide range of issues for policy makers and stakeholders to consider. While there is much to be learned from international CER systems, we need to maintain the strengths of the unique U.S. healthcare system while preserving quality innovation and patient focus.