Comparative effectiveness research: A new approach to clinical research

Since the early 1990s, a popular procedure for relieving the pain of spinal column fractures in patients with osteoporosis was vertebroplasty – the injection of acrylic cement into the fractured areas of bone to relieve pain.  The procedure cost around $5,000 and was widely practiced.  Then in 2009, the medical community was surprised when a randomized clinical trial showed that the procedure was no better than placebo in improving pain in patients with osteoporosis.  Over the course of the last several years, studies like this trial have been drawing attention and prompting health policy experts to raise questions about whether spending on interventions with no proven benefit and potential adverse risks is justified.  These studies have also led to a reassessment of the priorities of medical research and shifted the paradigms of clinical investigation.

Traditional medical research seeks to determine whether a specified treatment works or does not work.  But a newly evolving approach, called comparative effectiveness research (CER), raises questions about the comparative efficacy of various interventions.  In other words, if traditional studies ask whether drug A works or not, CER asks how drug A compares to drug B and drug C in terms of efficacy, cost-effectiveness, side effect profile, etc.  CER does not only look at therapeutic interventions but also studies the relative effectiveness of preventive measures as well as diagnostic tests. CER is not an entirely new concept, but has evolved and taken center stage in the quest for improving the quality and cost of health care.   In 2009, as a part of the American Recovery and Reinvestment Act, the Obama administration allocated a $1.1 Billion budget to CER in collaboration with the Department of Health and Human Services and the Agency for Healthcare Research and Quality.  The goal of CER is to inform everyday patient care decisions that physicians face.  Furthermore, unlike traditional research approaches, which pay less attention to patient heterogeneity and extrapolate data from the ‘average’ patient, CER recognizes that patient populations are diverse and seeks to examine how different subpopulations respond to various interventions.  The ultimate aim is to provide more personalized care based on a patient’s demographic profile, past medical history, and other factors.  

At the forefront of CER is the Patient-Centered Outcomes Research Institute (PCORI), a non-profit organization created as part of the Patient Protection and Affordable Care Act.  This agency is commissioned to conduct CER and set priorities for research areas.  Last week, PCORI put forth the First Draft on National Priorities for Research and Research Agenda.  This agenda proposes the following five research priorities:

1. Focus on the comparative assessment of different options for prevention, diagnosis, and treatment of disease.
2. Study approaches that seek to improve the performance of health systems.
3. Devise effective methodologies for making new medical discoveries readily accessible to stakeholders including providers and patients.
4. Investigate ways to reduce disparities in health outcomes and access to health services among sub-populations of patients.
5. Place greater emphasis on studying patient-centered outcomes.  While traditional research is more focused on objective measures of health such as test results, PCORI promotes utilizing CER to study outcomes that are important to patients, such as quality of life.

One of the most interesting aspects of PCORI’s recent agenda is its focus on engaging stakeholders in the development of this agenda.  In fact, last week’s draft invites health care providers, patients, caregivers, and the general public to provide constructive comments and contribute to further development of research priorities.  To facilitate this process, PCORI has set up an online survey at http://www.pcori.org/provide-input and is putting together a National Patient and Stakeholder Dialogue on February 27th in Washington, DC. Dr. Joe Selby, the executive director of PCORI, believes this is a unique part of their agenda, “The novel part of what we are doing is taking substantial guidance from patients,” he said in a recent interview with the Washington Post.

Reaction to CER and PCORI’s recent agenda has been mixed.  Despite the potential advantages, conservative think tanks, such as the Heritage Foundation worry that this approach will limit choices for consumers and potentially result in health care rationing.  They also raise concerns that CER will streamline patient care and disregard variations in patient characteristics and preferences.  Finally, they state that the new agenda lacks specific goals.  Dr. Selby argues that this initial draft was intentionally written more broadly to allow it to further develop through feedback.  He asserts that the final draft of the agenda will have greater detail and focus more on specific disease priorities.    

Despite the controversy, it is clear that with more than $1 billion injected into CER, medical research is undergoing a transformation.  Greater emphasis will be placed on making comparisons across different preventive, diagnostic, and therapeutic approaches to patient care and patient-centered outcomes will become a priority. Researchers and policy makers will also begin to further scrutinize the cost-effectiveness of interventions like vertebroplasty.  It remains to be seen how much the evidence that emerges from this new approach to research will affect policy and clinical decisions.  Nonetheless, the idea of clinical investigation that is patient-centered, places greater emphasis on reducing costs, and focuses on improving health outcomes seems promising in an era where health care expenses are soaring and myriad treatment options are becoming available virtually everyday.

References
Kallmes DF et al.  A randomized trial of vertebroplasty for osteoporotic spinal fractures. New England Journal of Medicine, 2009 Aug; 361: 569-579.

Ramsey SD et al.  How comparative effectiveness research can help advance 'personalized medicine' in cancer treatment.  Health Affairs; 2011 Dec; 30(12): 2259-68.