“works best, for whom, and under what circum- the collection of data via claims data or electronic
stances.” While research comparing the effectiveness medical records. CER expands the notion of evi-
of interventions has been conducted for decades, the dence, and challenges our discipline to play a part
term “comparative effectiveness research,” or CER, in defining the most appropriate evidence to answer
is relatively new. a specific question.
The 2009 American Recovery and Reinvestment CER encompasses the range of health care, from
Act (ARRA), or “Stimulus Bill,” provided $1.1 bil- screening to diagnosis to treatment. It addresses
lion to support CER, among other directives to decisions at both the patient and public health levels.
health care–related activities. ARRA divided the One intent of the ARRA legislation was to ensure
CER funding into $300 million for AHRQ, $300 that subpopulations (e.g., children) were considered.
million for the National Institutes of Health (NIH), The vision was to include stakeholders in the prior-
and $400 million for the Office of the Secretary of ity setting. To that end, both a public hearing and
Health and Human Services (HHS). According to Internet survey were used to gather public input for
the law, the funding is to be used to evaluate the the IOM committee deliberations. See the report at
relative effectiveness of different health care services
www.iom.edu/CMS/3809/63608/71025.aspx.
and treatment options and encourage the develop- CER is also patient-centered. It is about provid-
ment and use of clinical registries, clinical data net- ing information to patients and their families, care-
works, and other forms of electronic data to gener- givers, and health-care providers about what treat-
ate outcomes data. ment works best and under what circumstances. In
The law also provided $1.5 million to support particular, CER outcomes are those that matter to
an Institute of Medicine (IOM) study. The result- patients, and CER studies are long-term. Evidence
ing IOM committee (of which I was a part) drew is worth nothing if it does not answer the questions
together people from a host of disciplines, includ- that are important to patients and if it is not trans-
ing statistics, to make recommendations to the sec- lated into usable information and disseminated.
retary of HHS on national priorities for CER. The As Lynne Billard, a past president of the ASA,
committee (see
www.iom.edu/CMS/3809/63608. said in her 1996 address:
aspx) defined CER as “the generation and synthesis
of evidence that compares the benefits and harms It is up to us as an association to chart
of alternative methods to prevent, diagnose, treat, a course that focuses on the unique
and monitor a clinical condition, or to improve the strengths inherent to statistics and its
delivery of care.” The purpose of CER is to assist boundless opportunities to play pivotal
consumers, clinicians, purchasers, and policymakers and indispensable roles in resolving
in making informed decisions that improve health contemporary issues, a course that
care at both the individual and population levels. guarantees the success of our profession and
Several components of this definition merit of statistical science. “Do we count?” We
emphasis. First, CER is about effectiveness, not effi- like to think we do. The crucial question
cacy. Efficacy examines how a treatment performs is: “Do others think that we count?” That
under ideal circumstances, generally via an internal- answer and our response to it will fashion
ly valid—but limited in generalizability—random- our future.
ized controlled trial. Effectiveness examines how a
treatment works in the real world, for example in To me, this is still the key question. Evidence-
a variety of different settings, for different types of based decisionmaking is a contemporary chal-
patients, such as those with comorbid conditions lenge in which statistics and statisticians can, and
and so on. must, count.
Methodologically, CER expands beyond what is As always, I welcome your comments.
traditionally thought of as evidence-based medicine Thank you.
methodology, the latter being synthesis, sometimes
meta-analysis, of existing data. CER includes the
generation of new evidence, for example the fielding
of a clinical trial or an observational cohort study, or
4 AMsTAT NEWs OCTObER 2009
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