Testimony on Comparative Effectiveness Research

Amy M. Miller, Personalized Medicine Coa

On April 3, 2009, public testimony on comparative effectiveness research was given at a meeting of the National Advisory Council for Healthcare Research and Quality. The testimony represents the views of the presenter and not necessarily those of the Agency for Healthcare Research and Quality (AHRQ) or the Department of Health and Human Services (HHS).

PMC Position Statement on CER
Presented to AHRQ via E-mail
by Amy M. Miller, Ph.D. Public Policy Director

The Personalized Medicine Coalition (PMC) is pleased to have the opportunity to present our perspective on comparative effectiveness research to AHRQ. PMC represents all stakeholder communities in personalized medicine, which we define as the tailoring of medical treatment to the individual biomarkers of each patient. We see personalized medicine as the future of healthcare and we support healthcare based on sound science.

As you consider priorities for comparative effectiveness research, we urge you to incorporate personalized medicine concepts and technologies throughout the effort. We have outlined some examples of how this might be done:

Define evidence levels for the demonstration of effectiveness.
When comparing the standard of care approach with the personalized medicine approach, federal groups often find that there is not enough empirical evidence to recommend personalized medicine, even when FDA changes the label of a drug or when professional associations change the recommended course of care for a particular condition. Empirical evidence is traditionally accrued over many years and thus tends to favor older treatments even when observation suggests that a new one is more beneficial. We urge AHRQ to ensure that comparative research recognizes targeted approaches to medicine and perhaps more importantly, define what levels of evidence and what type of evidence are needed to deem an intervention effective.

Sequence every research participant in comparative drug studies.
Individual variations in drug metabolism and sensitivity genes for over 30 classes of therapeutics have been identified by researchers. Based on the groundbreaking mapping of the human genome, we can now indicate if a drug is going to work for an individual based on genotype in some cases. Therefore, studies that compare the effectiveness of drugs should take these factors into consideration by sequencing participant DNA.

Consider the full range of relevant research questions.
To support the timely and appropriate adoption of high-value personalized medicine technologies, research priorities should include both medical interventions and the broader health system factors affecting the adoption and use of these interventions. The scope of CER proposals must focus on the totality of the healthcare delivery system, including approaches to care management, delivery, and benefit design, to empower clinicians and patients to make more appropriate decisions that support personalized, evidence-based care.

Designate a permanent, personalized healthcare standing committee within AHRQ.
During our comments, we have outlined a few examples of personalized medicine but there are many more. Some personalized approaches reduce the need for hospitalization while others prevent disease. A committee on personalized healthcare would assist AHRQ in incorporating personalized approaches into all comparative effectiveness work.

Outline a process for decision reconsideration.
The science of personalized medicine is particularly fast-moving. Comparative approaches such as meta-analyses and systematic literature reviews reward "old medicine"—the one-size-fits all, trial-and-error methods that are expensive and inefficient. Therefore, we urge AHRQ to outline a plan by which a comparative-effectiveness finding will be reconsidered, such as when FDA changes the label of a therapeutic, or when a professional organization changes the suggested standard of care.

Thank you again for inviting our participation in such an important discussion. We urge you to continue public engagement efforts at every step of this process.

The PMC looks forward to working with AHRQ and others on personalizing the evidence base and improving the quality of healthcare in our country.

Current as of April 2009
Internet Citation: Testimony on Comparative Effectiveness Research: Amy M. Miller, Personalized Medicine Coa. April 2009. Agency for Healthcare Research and Quality, Rockville, MD. http://www.ahrq.gov/news/newsroom/speech/pmc.html