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PCORI’s Research Will Answer Patients’ Real-World Questions



March 25th, 2014

As a physician, I know the challenge of helping patients determine which health care options might work best for them given their personal situation and preferences. Too often they — and their clinicians — must make choices about preventing, diagnosing and treating diseases and health conditions without adequate information. The Patient-Centered Outcomes Research Institute (PCORI) was created to help solve this problem — to help patients and those who care for them make better-informed health decisions.

Established by Congress through the Patient Protection and Affordable Care Act as an independent research institute, PCORI is designed to answer real-world questions about what works best for patients based on their particular circumstances and concerns. We do this primarily by funding comparative clinical effectiveness research (CER), studies that compare multiple care options. But more research by itself won’t improve clinical decision-making. Patients and those who care for them must be able to easily find relevant evidence they can trust. That’s why our mandate is not just to fund high-quality CER and evidence synthesis but to share the results in ways that are meaningful to patients, clinicians and others. We’re also charged with improving the methods used in conducting those studies and enhancing our nation’s capacity to do such research.

We will be evaluated ultimately on whether the research we fund can change clinical practice and help reduce the variations and disparities that stand between patients and better outcomes. We’re confident that the work we’re funding brings us and the audiences we serve closer to that goal.

Recently, some questions have been raised in health policy circles about our holistic approach to PCORI’s work. That view holds that direct comparisons of health care options — especially those involving high-priced interventions — should be the dominant if not sole focus of PCORI’s research funding approach as a path to limiting the use of expensive, less-effective options.

We agree that discovering new knowledge on how therapies compare with one another is a critical mandate of PCORI and is essential to improving the quality and effectiveness of care.  However, ensuring that patients and those who care for them have timely access to and can use this knowledge, so that they can effectively apply it to improve their decisions, is also very important. That is the reasoning behind our integrated approach path that addresses the gaps in available evidence, and also studies how best to make the evidence available and usable.

Much of our research portfolio is in fact dedicated to the research that our critics desire. We’re funding studies to answer questions about common, serious conditions like cardiovascular disease, cancer, and mental illness, which affect millions of Americans.  And the specific topics we’re addressing are recommended and vetted by researchers and the patient and stakeholder communities to whom we’re responsible.

This work will be bolstered by a new series of large pragmatic studies that will compare outcomes between two or more approaches to addressing high priority clinical issues in real-world settings. We also are establishing a national clinical research infrastructure to make it easier and less expensive to conduct these kinds of large studies in diverse patient populations.

By focusing on outcomes and data from real-world settings, we’re more likely to obtain relevant answers to the many important questions patients have.  Consider patients newly diagnosed with cancer. They want to know not just survival rates but the impact of different therapies on their quality of life or ability to work. Such outcomes matter to patients, but research often fails to address them.

That’s why we’ve made engagement a cornerstone of our research. Every research study we fund must include a plan to engage patients, their clinicians and others across the health care community to ensure the research focuses on practical questions.

Because engagement of this depth is new to many researchers and patients alike, we’re developing methods to improve our ability to incorporate patients’ perspectives in research. Patient-centered research methods that are transparent and scientifically sound will enhance the credibility and usefulness of the studies we fund.

This integrated approach will provide patients with the right information in the right place at the right time. It recognizes that engagement, methods, infrastructure, and effective dissemination make patient-centered research timely and useful.

We’ve invested more than $464 million in such research to date. About 62 percent of this research funding has focused on CER, with the rest spread across infrastructure (18%), methods (11%), and communication and dissemination research (8%). We expect to commit $1 billion over the next two years to expand this work.

With a foundation for patient-centered research in place, we’re confident our work will provide patients, caregivers, clinicians and others throughout the health care community with the information they need and help to improve health and well-being for all of us.

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3 Responses to “PCORI’s Research Will Answer Patients’ Real-World Questions”

  1. Joe Selby Says:

    Dr. Hadler is right. Comparative effectiveness studies are often done in situations where expected differences are small or where the expectation is that there is no difference – difficult territory to operate in, but nonetheless important. We also agree that comparative effectiveness research is complementary to and usually follows on the establishment of efficacy and even effectiveness. Three points may offer some comfort. First, a major problem when differences are small is that it’s hard to discern true differences from apparent differences that are due to selection bias (confounding), especially in non-randomized studies. That’s why nearly half of all PCORI studies and nearly all of the larger studies we’re funding are randomized trials. Second, although differences for some outcomes may be small or nonexistent, they may be larger for others, like side effects or quality of life. That’s why PCORI emphasizes considering a broader range of outcomes than in typical efficacy studies. And third, “marginal” effects often result from studying heterogeneous populations that contain subgroups who benefit differentially and subgroups who don’t – so the average effect is “marginal.” PCORI emphasizes and expects funded research to pay special attention to possible differences in relative effectiveness within a study population, and to seek to identify those patients who benefit a lot from making a particular choice and those in whom the choice doesn’t matter or is reversed. This is the patient-centered approach: learning what works better for whom, for the outcomes that matter most to them.

  2. Nortin Hadler Says:

    I applaud the premise behind CER and therefore the rationale for PCORI. However, I have grave concerns that the effort is largely doomed by methodological challenges. I expressed these reservations on THCB shortly after the creation of PCORI and nothing has assuaged my concerns to date:

    http://www.thehealthcareblog.com/the_health_care_blog/2010/01/comparative-effectiveness-research-and-kindred-delusions.html#more

    In essence, I don’t think any degree of data torturing can compensate for the marginal efficacy of so many targets for CER. If the efficacy in a carefully selected subset of patients is marginal (NNT>50 or 100 or…), you run the risk of comparative ineffectiveness research. CER only makes sense if it can be anchored in a relevant example of efficacy in some subset.

    Examples of marginal efficacy are legion such as oral hypoglycemics, statins, many cancer screening protocols, and much more including highly marketed fads such as the stenting of STEMIs:

    http://thehealthcareblog.com/blog/2013/10/13/the-end-of-the-coronary-angioplasty-era/

    and anti-coagulants for non-valvular A-fib:

    http://blogs.scientificamerican.com/guest-blog/2014/01/30/the-a-fib-marketing-blitz-be-still-my-heart/

    Of course, this concern would not pertain to comparative cost research, but that is not a PCORI agenda.

  3. Gerald Rogan Says:

    In the real world, some invasive procedures are done without medical necessity, such as cardiac stent placements and angiograms. See cardia examples at a hospital in Maryland (2008) and a hospital in California. (2002). Peer review provided by the FBI.

    The common unmet need was failure to perform peer review by the hospital medical staff for problem cases and to vaidate medical necessity. Peer review is shown to interefere with profits short term, and its absence causes financial loss, long term.

    We have no measure for the performance of effective peer review. The lack of this measure is unacceptable.

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