BB’s Comparative Effective Research 101

Many Americans would be surprised to know that a lot of medical care being delivered today is NOT based on sound scientific research. This is the ugly truth behind the fact that many patients today are being given sub-standard medical care and why there are wide practice variations across the country. This is where Comparative Effective Research (CER), long recognized as the bridge between science and the delivery of the best patient outcomes, steps in.   Just hearing the words Comparative Effective Research makes most Americans’ eyes glaze over. You are probably wondering “why should I care about it at all?”

All Americans should care about Comparative Effective Research (CER) because getting better patient outcomes is what healthcare is all about for us. Our elected officials and healthcare policymakers are making decisions about how much of our taxpayer money is being spent on medical research and what exactly they are spending it on. Taxpayers need to know if our government is delivering healthcare value for the CER they fund and if the research is addressing the needs of the American people. For example, is the National Institute of Health (NIH) getting extra money for brain cancer research because Vice-President Joe Biden pushed for it after his son died from brain cancer or because it is truly a national medical research priority?   Government-funded research that is politically or self-interest motivated does not deliver value to the taxpayer and should be replaced with research that does.

With the enactment of the American Recovery and Reinvestment Act (ARRA) in 2009, our government’s $1.1 billion allocation in Comparative Effective Research (CER) represented a 37 fold increase over the previous year. In the following year, Obamacare (PPACA) established the Patient-Centered Outcomes Research Institute (PCORI) to organize and direct this federal investment in Comparative Effectiveness Research.

What is Comparative Effectiveness Research (CER)?

Comparative effectiveness research (CER)  is simply “the direct comparison of existing health care interventions to determine which work best for which patients and which pose the greatest benefits and harms.”

Comparative Effectiveness ResearchFor any given disease or condition, certain medical interventions are found to produce the best patient outcomes (defined where the benefits outweigh the risks). The data from individual patients is combined with the data from many other patients to get a better handle on the effectiveness of a given medical intervention . When a new prescription drug is approved by the Food and Drug Administration (FDA), it only has to show that it works BETTER than a placebo (i.e., that it shows efficacy) and does not do more harm than good (i.e., is safe) for approval. The research to show efficacy are usually randomized clinical trials, under controlled conditions (patients and settings).  For drug evaluation, Comparative Effective Research (CER)  goes one step further and would ask whether one drug does a better job than others currently being sold for the treatment of the same disease or condition.

Comparative Effective Research versus efficacy questionsWhile all new (and expensive) drugs that are approved for sale are “efficacious”, they might actually be LESS “effective” than older, less expensive drugs. As you might have guessed, pharmaceutical companies do not like CER that reveals this inconvenient fact.

Limitations of Comparative Effective Research Data

Comparative Effective Research (CER) does not always deliver clear cut answers (“best” versus “worst”) and must often be conducted for particular subgroups of patients like the elderly, females, or a particular race. All people do not react to medical interventions in exactly the same way and therefore the results from CER are not all-encompassing (i.e., the results are not 100% applicable to all Americans). This is not a criticism of CER, but rather a simple statement of fact. Therefore, when evaluating the results of CER (or any clinical study) one must also ask “for whom do the findings apply and under what circumstances? For example, my eye caught NIH’s “2016 Research Highlights–Clinical Breakthroughs” entitled Blood Pressure Management for Seniors  and I made a hasty assumption based on the title. “Seniors” in the research mean 75 and older who had hypertension but NOT diabetes. While NIH might consider the research as a 2016 “highlight” (they after all funded it), the American Heart Association does not and sticks to its current blood pressure recommendations (based on other CER findings).

Another important caveat about CER is that not all CER studies are as scientifically rigorous as they should be (i.e. they make conclusions that are not supported by the results of the study).  Some CER studies are even faulty in design or tainted by financial self-interest which is why it is good to have an organization of experts (like NICE in the United Kingdom) to interpret the research and summarize the findings in guidelines.

In medicine, there are no guarantees of 100% success, but CER does help individual patients and their doctors narrow down the intervention choices. If I had a disease, I would want to know what is the best treatment found using CER data and start with it rather than a treatment that has been found to deliver “less than best” treatment. In other words, CER points me to treatments that have a higher chance of success (e.g, 80% ) versus a low chance of success (e.g., 20%).

Applying CER For “Best” Patient Outcomes

Applying CER information is a political minefield where financial self-interest rules over best patient outcomes and what is best for patients and taxpayers. Collecting CER information without applying it to “best” practices clinical guidelines, insurance coverage decisions, drug formulary placement, and reimbursement decisions is like a farmer growing a crop and waiting around for his potential customers to harvest it themselves.

While the Patient-Centered Outcomes Research Institute (PCORI) is limited by law in its ability to apply CER, others are leading the way.  Private for-profit health insurers, employers, and Medicare are applying the CER data for benefit coverage and reimbursement policies as part of the Core Quality Measure Collaborative.  For these payers, the information from CER equals best patient outcomes and best patient outcomes equals less health spending for the payers without sacrificing quality of care.

Comparative Effective Research equals lower health spending

Whether the government formalizes Comparative Effective Research into best practices guidelines or not, CER still makes its way into our healthcare system in many other ways–not necessarily the best ways.

Examples of Comparative Effective Research

The examples of Comparative Effective Research (CER) are too many to list but they can be found throughout the literature. For people with heart disease, taking a daily aspirin has been found to prevent future heart attacks and strokes. New cancer treatments are routinely compared to currently accepted treatments and those with better patient outcome are usually applied. Many of the measures found in the Core Quality Measure Collaborative are CER-based. Sometimes CER reveals that less is better such as when it was discovered that people with terminal lung cancer had a better quality of life and longer survival rates when chemotherapy was stopped early in the diagnosis.

Summary– Comparative Effective Research

Comparative Effective Research (CER) separates the best from the not so best medical interventions for a given disease or condition and a given set of patients. It answers the question: Does one medical intervention work BETTER than others and for whom?   As the foundation for weeding out low quality interventions, Comparative Effective Research has great promise to increase healthcare quality and improving healthcare value.

Additional Reading

EBM, HTA, and CER: Clearing the Confusion (very good) 
Initial National Priorities for Comparative Effectiveness Research, Institute of Medicine, 2009
The Pragmatist’s Guide to Comparative Effective Research, 2011 (very good) 
What is Comparative Effective Research? Agency for Healthcare Research and Quality (AHRQ)

 

 

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