Comparative Effectiveness Research

The health care system should guarantee that all individuals have access to the quality care they need. There must be a high level of evidence for doctors and patients to choose which care is appropriate, for whom, and under what circumstances. This is key to patient-centered care.  There are two necessary components to this evidence: the first is high quality clinical research of new interventions and the second, and equally necessary component, is research of interventions in the real life settings all doctors and patients face.  Comparative Effective Research, CER, is a term to describe this second component.  It provides an opportunity to conduct research to find answers, in settings that reflect the situations of the average person, adding value beyond what we obtain from the highly controlled setting of traditional clinical trials.

What is Comparative Effectiveness Research?
Comparative effectiveness research (CER) is a rigorous evaluation of the different options available for treating a particular medical condition.  It may compare similar treatments, such as competing drugs, or it may analyze very different interventions, such as surgery and drug therapy.   It may also compare the effectiveness of how and when health care is delivered, such as different intervals of follow-up, or schedules of dosing.

Comparative Effectiveness Research can use a range of research tools and methods.  It can be:

• A systematic review of existing studies and evidence,
• Studies using available data,
• Modeling to simulate effects of interventions on different populations, or
• A head-to-head clinical trial comparing one treatment to another.

Why is Comparative Effectiveness Research important?
CER is important research because it evaluates interventions in real-life settings, the kind all doctors and patients actually face.  Most clinical research is conducted in a very controlled setting.

CER is an important political issue because it can be used to inform coverage and other policy decisions.  When there are different options, CER can help with these decisions.  What benefit can we get from X, and how much does it cost us compared to Z, which might cost a lot less and work just as well?

Some examples of possible CER questions in breast cancer:

• Evaluate the methodologies for the clinical validation of biomarkers, including the use of adaptive design and Bayesian approaches, to determine the optimal methods;
• Compare the effectiveness of using Magnetic Resonance Imaging (MRI) or mammography in young patients and women who have dense breasts to improve health outcomes (i.e., survival, local recurrence, mortality);
• Compare different approaches to management of DCIS.

For Comparative Effectiveness Research to improve health care, however, it must meet the following criteria:

Quality – CER must be held to the highest standards of quality. This research must employ rigorous methods that can provide reliable answers to specific questions.  These may include experimental designs, observational studies like registries, systematic reviews and other methods.  Incorporating new technologies to better understand the utility of biomarkers and the interplay of co-morbidities will help achieve the promises of biomedical research progress on an individual level.  Great care needs to be taken to ensure that there are clear standards of quality so the investment in CER delivers value to the public.

Transparency and Accountability – Doctors, patients and policy makers must be able to trust the results of CER.  While quality is vital to that goal, transparency and accountability are also key. The processes for setting priorities, defining criteria and reporting results must be transparent and easily accessible to all. Methods and data must be shared so that they can be publicly critiqued and widely used in a practical manner. Moreover, trained lay consumer advocates must be meaningfully involved in all aspects of decision-making that affect CER.

Independence – CER infrastructure must be sheltered from political pressure. The usefulness and value of CER lie in its independent assessment of different interventions, the results of which can be used by all the different stakeholders in decision-making. The process for selection of topics to be studied must be objective, and the results must be credible. The entire research process must be insulated from political pressures and conflicts of interest generated by both government and private-sector stakeholders.ii

Integrity – CER must be conducted with integrity. High quality methods, accurate and detailed record keeping, and honest publication of the results, regardless of the outcome, must be emphasized. All contributors to CER must publicly disclose all relevant relationships and conflicts of interest. Institutional guidelines and procedures must be in place to define and address conflicts.

Comparative Effectiveness Research must deliver value to the individual and society by strengthening the evidence base, enabling better decision-making, improving health outcomes, more fairly allocating healthcare resources, and containing the currently unsustainable health care costs.