A key engine of healthcare reform is poised to accelerate, with the potential to improve clinical decision-making and care quality, curtail inappropriate utilization of ineffective treatments, and lower costs. Comparative-effectiveness research (CER), until now, has received relatively meager funding and has occupied a relatively low profile among policymakers, clinicians, and the public.
With a $1.1 billion injection from the American Recovery and Reinvestment Act of 2009 (better known as the “stimulus”) and dedicated funding mandated by the Affordable Care Act, a new national center dedicated to CER—the Patient-Centered Outcomes Research Institute (PCORI)—has recently released a draft of its national priorities and is mobilizing a national research agenda for CER.1
Demonized by critics as a prelude to coverage denials, healthcare rationing, and intrusion upon physicians’ clinical autonomy, CER is reconstituting its reputation as a non-coercive yet powerful tool to reduce uncertainty about which healthcare options work best for which patients, and to encourage adoption of care practices that are truly effective.
“I practice on weekends as a pediatric hospitalist, and it is still far too common to encounter a case for which we don’t have good, evidence-based guidance—such as whether surgery or medical management is best for a neurologically impaired child presenting with aspiration and gastroesophageal reflux disease,” says Patrick Conway, MD, MSc, SFHM, chief medical officer of the Center for Medicare & Medicaid Services (CMS) and director of its Office of Clinical Standards and Quality.
SHM strongly supported the creation of PCORI during the health reform debate, and hospitalists have important opportunities to be part of the nation’s CER agenda, as well as key beneficiaries of its results, according to Dr. Conway.
Efficacy vs. Effectiveness
While the traditional “gold standard” of clinical research is the randomized controlled trial, its focus on efficacy typically involves comparing some treatment to no treatment at all (a placebo), and requires highly controlled, ideal conditions, often with narrow patient-inclusion criteria. All of those requirements sacrifice generalizability to patients whom physicians encounter daily in clinical settings who often have multiple chronic conditions and comorbidities and might require multiple therapies, Dr. Conway says.
CER studies larger, more representative patient populations treated in real-world clinical circumstances, explains SHM Research Committee chairman David Meltzer, MD, PhD, FHM, who is a member of the PCORI’s Methodology Committee. “One of the big initial tasks of the PCORI is to produce a translation table of what research study designs—randomized controlled trials, head-to-head, observational studies, and others—can best answer which kinds of questions,” he says.