By Micahlyn Whitt-Flicker, Copywriter, Managed Markets at Palio, email@example.com
CER and the real world
Most of us are familiar with the basic principles of study design for randomized control trials (RCTs). While RCTs continue to be seen as the gold standard for providing unbiased clinical evidence for pharmacological and other interventions, by design, these studies omit specific variables that exist in real-world settings. For advocates of CER, these real-world variables provide the foundation needed to glean important insights into the utilization and effectiveness of interventions. Ultimately, it’s believed that these insights and outcomes can lead to the development and support of more effective, patient-centered interventions.
In a recent article in the American Journal of Managed Care, the head of Medco’s personalized medicine research department commented on the potential for CER. He said, “If we do it right, we can learn a lot about how drugs work and for who, in what I believe is a much more realistic real-world environment.”
Of course, doing it right begins with the implementation of sound study design.
To CER or not to CER
There are many methods for conducting comparative effectiveness research (CER) and even more opinions and debates about which of these methods are most effective in producing unbiased outcomes with meaningful applications versus the gold standard RCTs. Retrospective observational studies and prospective observational studies are often cited as an important foundation for robust and comprehensive CER outcomes. These methods also provide an interesting contrast of advantages and disadvantages to RCTs.
RCTs continue to be viewed as the most accurate form of study for determining whether a new drug should be approved for marketing. These studies are believed to most effectively control for diagnostic and prognostic factors that affect treatment decisions. In contrast, CER that seeks to demonstrate the effectiveness of the real-world treatment and other intervention practices of patients and physicians are particularly appropriate for observational studies. For example, large observational studies are often essential when treatment effects differ across types of patients, and when analyses of subgroups are needed to understand which patients are most likely to benefit. Let’s take a closer look at these distinctions.
CER by design
Retrospective observational studies typically employ existing secondary data sources in which both exposure to an intervention and the outcomes of that exposure have already occurred. Researchers conduct comprehensive reviews of current literature, large established databases, (including electronic health records), and results from RCTs. Systematic retrospective observational studies are often utilized in CER initiatives as a means for summarizing a body of evidence, identifying information gaps, and generating ideas for new research in areas of unmet clinical need.
A prospective observational study is another form of observational study in which outcomes occur after the creation of the study protocol and analysis plan and study commencement. Patient exposure to interventions being studied may have been recorded prior to the study initiation. Prospective observational studies such as those conducted over a 20-year period by the United Kingdom Prospective Diabetes Study Group (UKPDS) have resulted in providing influential long-term data that have informed treatment guidelines as well as research and development of new interventions.
Observational studies have also been employed to examine multiple treatment paradigms simultaneously. For example, the wide range of approaches for treating hearing loss including cochlear implants, assisted listening and electric acoustic devices, and habilitation and rehabilitation methods. A prospective observational study of patients being treated for hearing loss would permit researchers to examine treatment preferences as well as the risks and benefits of the different treatments. In general, observational studies are considered to be best used to evaluate the real-world applicability of evidence derived largely through randomized trials; to study patients and conditions not typically included or studied in randomized trials; to better understand current treatment practices and how patients are assessed in order to design an appropriate clinical trial.
Did CER help to shape perceptions about heart disease in the United States?
Retrospective observational studies have been particularly useful in identifying and reporting on the inequity of important disease-state research results; notably, previously underrepresented patient experiences. During the 1980s, for example, several retrospective reviews of major disease-state research identified a stark absence in the number of females as subjects in research, resulting in the recognition of an alarming void of information across the spectrum of healthcare for the female population. Subsequent retrospective studies demonstrated significant lack of research data for other underrepresented populations, such as nonwhite and female populations in cardiovascular diseases. With this in mind, it’s interesting to consider that in 1948 the American Heart Association’s (AHA) campaign to raise awareness of heart disease in the United Stated was called “Walking Man.” Today, the “Go Red For Woman” campaign is a hallmark of the AHA’s awareness initiatives.
Room for debate
Researchers cite several advantages of observational studies versus RCTs. For example, larger numbers of subjects can be observed at more affordable costs. And this, in turn, allows researchers to examine a broader range of patient experiences such as those for meaningful subgroups and comparison populations. Another noted benefit of CER is longer follow-up periods, which make it possible to examine long-term risks and benefits.
As we can see, observational research methods can provide many viable options for identifying key outcomes data for many types of interventions. However, one of the primary criticisms of CER, specifically observational research, is that there is too much room for bias and inaccurate reporting. Some believe that because patient characteristics influence real-world clinical decisions, there will always be uncertainty about whether the subject (the patient), or the intervention itself, causes the observed outcomes.
While the impact and application of CER methodology and study design continue to be hotly debated, at the end of the day, or at least the end of this blog, most seem to agree that it will continue to play an important role in the future of healthcare, informing everything from practice to policy. The general opinion held is that in order to arrive at the best possible decisions about patient care, we need research that has met the highest possible standards in its conduct and reporting. This includes outcomes from RTCs as well as a comprehensive range of high-quality comparative effectiveness research.
Next on Managed Markets Mondays: Do well-designed, industry-supported CER studies have a role in managed care decision-making?
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