Health Affairs

With ACA implementation now at hand — and with it, the formation of accountable care organizations (ACOs) — health services research (HSR) has an especially important role to play. As ACOs take steps that will substantially change health care delivery, the ability to measure and improve health system performance and acquire this data efficiently will be in greater demand. Is HSR up to the challenge?

As the “basic science” of the health care system, HSR focuses on access, cost, and quality. Health services researchers work to identify and assess vital signs of a well-functioning health care system and develop performance measures for examining system aspects. They also seek to improve the system by identifying gaps in quality and then testing and disseminating solutions to those problems. HSR’s goals and often its approaches are different from the development, testing, and translation of new drugs or other interventions.

But as we’ve seen all too frequently, the necessity of responding to ongoing change within the health system outpaces HSR’s ability to produce timely evidence. As a result, large scale changes are sometimes instituted on the basis of imperfect evidence (e.g., tying financial incentives to measures of physician or hospital performance before studies are completed). Or, as we’ve also seen, by the time a research project comes full cycle — proposed, funded, conducted, and published — external or internal forces impacting the health system may have rendered the original question moot. Within the VA, for example, studies regarding the effectiveness of telehealth interventions often have been overtaken by these technologies’ rapid dissemination throughout the system. For HSR to fulfill its mission of helping transform health care, it first must be able to transform itself.

Performance measurement — if done right — can be a core activity to move the health care system to higher value for the American public, while rewarding health professionals and health care institutions for doing the right thing for their patients. Yet, policy makers, private and public, have a duty to the public, patients, and providers to get it right — to measure and report accurately and meaningfully.

Harlan Krumholz and Peter Pronovost have been among the most important contributors to the development of performance measures for quality and safety of health care. At the same time, each has written powerful critiques of particular aspects of the current measurement enterprise with suggested improvements. I work mostly inside the Beltway in a world of policy makers who, despite good intentions, by their actions often display a lack of understanding of the challenges associated with measures, measurement, public reporting, and pay-for-performance. For example, the physician value-based modifier, which was mandated as part of the Affordable Care Act and now must be implemented by CMS, cannot produce a valid snapshot of an individual physician’s “value” but will be imposed nevertheless, unfortunately feeding those within the physician community who resist all efforts to improve accountability and transparency of performance.

With the encouragement of the Robert Wood Johnson Foundation, Harlan, Peter, and I joined in a collaborative endeavor to produce a comprehensive look at the state of play of performance measurement and public reporting — their conceptual underpinnings and limitations, successes and failures, and, perhaps most importantly, recommendations for major steps that are needed now to put the measurement enterprise on track to achieve its potential to improve the value of U.S. health care without doing harm.

Medicare is caught between two countervailing impulses: the desire of beneficiaries (and providers and the adult children of beneficiaries) to have a benefit package that covers more, rather than less, and the desire to restrain program spending due to its impact on the federal budget. This tension is heightened by the transition of the Baby Boomers from paying taxes into Medicare to receiving benefits.

The default is that Medicare covers acute care therapies, tests and procedures if there is a patient that wants to receive them and a provider who is willing to deliver them, whether there is evidence of any benefit to the patient or not. As I tell students in my Introduction to Health Policy Course, while Medicare sets payment rates (and is therefore like Marlon Brando in The Godfather: “I have an offer you can’t refuse”), when it comes to what is covered in the acute care setting, it is more like my Grandmother serving lunch (“whatever you would like, honey.”)

There are exceptions. Recently, the Medicare Evidence Development and Coverage Advisory Committee decided not to approve the payment of PET scans to aid in the diagnosis of Alzheimer’s disease. However, such a move is rare, and both provider and patient groups are protesting this decision.

There is a growing consensus that the regulatory system for research is in need of reform. Established 21 years ago by the Common Rule, it has functioned via a rigorous environment to assure that risk in research is dealt with and transparency maintained.

The trigger for these regulations is a definition of research as a “systematic investigation…designed to develop or contribute to “generalizable knowledge.” When this definition is satisfied, an intensive set of requirements ensues including review, approval, and continued oversight by an Institutional Review Board (IRB); reporting requirements;, the necessity for informed consent (often highly complex); and other administrative components. If projects are not “generalizable,” (e.g., local hospital programmatic or quality review), they fall strictly under healthcare system purview rather than under Common Rule regulatory oversight.

The current system has a strong moral imperative and has been critical to mitigating risk for research subjects and providing transparency. However, it is burdensome and fails to take into account the considerable progress made in both the research and clinical enterprises over the last few decades: in research, technological advances in generating data on routine care, and in healthcare, much more stringent oversight.

Medicare is poised to revise its “coverage with evidence development” (CED) policy, which has important implications for beneficiaries’ access to new medical technology as well as manufacturers’ reimbursement for their products.

For years, Medicare has employed CED, under which the program provides conditional coverage for new technology while it collects additional evidence on the technology’s effectiveness. The concept has great intuitive appeal in that it promises to provide access to promising technology for which the evidence base may be immature. As Medicare officials and other experts have argued, by linking coverage of new technologies to requirements that patients participate in registries or clinical trials, CED can help identify the circumstances in which patients are most likely to benefit and potentially accelerate access to innovations.

Though Medicare has experimented with conditional coverage policies since the 1990s, the formal CED designation and its characterization date to two guidances the Centers for Medicare and Medicaid Services (CMS) issued in 2005 and 2006. CMS has used CED in 19 cases over the years on diverse technologies, including lung volume reduction surgery, implantable cardioverter defibrillators (ICDs), and positron emission tomography (PET) for cancer. (See Table 1 at the end of the post, click twice to enlarge.) The CED policies have varied in their data collection requirements, with some featuring randomized controlled trials and others relying on patient registries or other data collection strategies

Proposal

The National Research Council’s Precision Medicine report found that it is imperative to create a new scientific base for biomedical research, clinical care, and public health that accurately reflects the genetic variations in diseases and in individual responses to therapies.

This proposal calls for using the nation’s rapidly expanding capabilities for computerized biomedical research to accomplish this goal as quickly as possible. Research databases and analyses for most diseases would be completed over the next three years (by the end of 2015).

Background

The US-led Human Genome Project was finished ten years ago (2003). In the past several years, key elements for moving forward on a Precision Medicine-type initiative have been coming together.

A goal of Twenty-First Century Healthcare is to establish and enhance the Learning Healthcare System (LHS). As discussed in numerous forums, journals, and social media, the LHS is viewed as critical to improving healthcare. Fundamentally, the LHS converts data about care and operations into knowledge that it translates into evidence-based clinical practice and health system change. In so doing, the LHS utilizes as vehicles health information technology, databases, the electronic healthcare record (EHR) and, importantly, a research infrastructure. The continuing narrow evidence base for clinical care combined with the need for substantial amounts of data to fill large evidentiary gaps, among other factors, have fostered the LHS concept.

To assure the utility and validity of data converted and then translated into improvements by the LHS, we need rigorous research approaches that are also efficient. Research is defined as “a systematic investigation…designed to develop or contribute to generalizable knowledge.” At present, evidentiary inputs to the LHS range from activities not generally considered research (e.g., programmatic and quality improvement evaluations) to various forms of research that are sufficiently rigorous, but for various reasons, can be difficult to employ and translate into the routine workings of the LHS.

For example, the randomized clinical trial (RCT) — considered the cornerstone, or “gold standard” methodology — provides the best data, but by its very nature, is separate from the workings of clinical care in a given healthcare system (HCS). Instead, the RCT functions in an alternate environment precisely controlled for the approach’s particulars. Further, the RCT is costly and time-consuming, and its rather narrow entry criteria may diminish its ability to be generalized to routine patients (e.g., those with comorbidities, or those outside of the entry criteria of age and severity).

Will pay for performance in health care backfire? That was the question addressed through the lens of behavioral economics by Steffie Woolhandler, Dan Ariely, and David Himmelstein in the most-read Health Affairs Blog post for 2012. Next on the most-read list were two posts, one by Diane Archer and the other by Archer and Theodore Marmor, contrasting Medicare and private insurance. They were followed by Rushika Fernandopulle’s rethink of primary care and Ken Kaufman’s post suggesting that the recent slowing in health care cost growth reflects more than just temporary effects from the economic slowdown.

Many thanks to our readers and authors for making 2012 a good year for Health Affairs Blog. We had 369 posts and over 50 million pageviews, more than a 60 percent increase from 2011. Since we had many good posts that were closely grouped in the readership numbers, we’ve expanded this year’s most-read list to a “top 15″ rather than a “top 10.” The full 2012 list is below.

A new Health Policy Brief from Health Affairs and the Robert Wood Johnson Foundation examines waste in US health care. Estimates are that more than a third of annual US health spending may be wasteful. A September 2012 Institute of Medicine report estimated that $765 billion a year was wasted through provision of unnecessary services, inefficiently delivered services, excessive prices and administrative costs, and missed prevention opportunities and fraud and abuse.

This policy brief discusses these and other types of waste in health care, ideas for eliminating waste, and the considerable hurdles that must be overcome to do so. It is the companion to a July 2012 Health Policy Brief, “Eliminating Fraud and Abuse.”

Tim Jost’s look at the 2012 election and the implementation of the Affordable Care Act in its aftermath tops the list of most-read Health Affairs Blog posts for November. Jost’s piece is joined on the list by election-related posts by Chas Roades, James Morone, and Jim Capretta.

Second on November’s top-ten list is a look at pay-for-performance through the lens of behavioral economics, by Steffie Woolhandler, Dan Ariely, and David Himmelstein. Other posts on the list discuss regulations issued under the Affordable Care Act; a new effort to promote outcomes measurement around the world; the VA Health System’s new “mega-database” for genomic medicine; and challenges for safety-net institutions in delivering accountable care.

The full list appears below.

Editor’s note: For more on PCORI and comparative effectiveness research, see the October 2012 issue of Health Affairs,”Current Challenges In Comparative Effectiveness Research.”

Earlier this year, the Patient-Centered Outcomes Research Institute (PCORI) satisfied one of its foundational requirements mandated in the Affordable Care Act. With public input, we established national research priorities to guide our work and support our mission of improving health by producing trustworthy information that will help patients make better-informed decisions about their care.

We are implementing those priorities through an ambitious research agenda, expecting to commit up to $96 million in contracts by the end of the year in response to funding announcements we issued in May. That’s on top of $31 million in funding we announced this past spring for our Pilot Projects Program.

Complementing that work, we are engaged with patients and the entire healthcare community to select specific research topics for targeted funding announcements. In particular, we are focused on conditions that occur frequently and impose heavy burdens on patients and their families, where outcomes can be substantially improved through new research.

To mark World Diabetes Day, I wanted to call readers’ attention to the thematic issue on diabetes published by Health Affairs in January 2012. Much of the issue dealt with the diabetes crisis in the United States, but several articles dealt with the global diabetes epidemic. For example, K.M. Venkat Narayan of Emory University and colleagues argued for four “policy paradigm shifts” in the global battle against diabetes:

conceptually integrating primary and secondary prevention along a clinical continuum; recognizing the central importance of early detection of prediabetes and undiagnosed diabetes in implementing cost-effective prevention and control; integrating community and clinical expertise, and resources, within organized and affordable service delivery systems; and sharing and adopting evidence-based policies at the global level.

The issue also included a look at a diabetes pay-for-performance program in Taiwan. Interested readers can visit the Health Affairs website to view our briefing on the January issue.

Editor’s note: Joe Selby’s post below responds to David Introcaso’s Health Affairs Blog post, also published today. For more on the concept of patient centeredness, comparative effectiveness research, and the Patient-Centered Outcomes Research Institute, see Health Affairs’ October issue, “Current Challenges In Comparative Effectiveness Research.“

The Health Affairs Blog post by David Introcaso, PhD, “PCORI’s Problem: Reasons Are Not Causes,” emphasizes the critical importance of the patient-provider relationship and communication in improving health outcomes. It also notes the challenges to changing practice through the creation and dissemination of new evidence, including the non-linear nature of the process of turning knowledge into action. But it argues that PCORI has missed the boat on these issues, specifically having failed to address them either in the $30 million in Pilot Project awards we issued earlier this year or in our current research priorities and funding announcements.

We genuinely appreciate the serious thought Dr. Introcaso has given to these matters and fully agree with him on their importance if we are to change practice and improve patient outcomes. And that’s why both our work to date and the work we continue to pursue focuses directly on these questions in a number of concrete ways – a point he seems somehow to have missed.

The significance we at PCORI place on communication between patients and those who care for them is evident throughout our work: in our foundational research roadmap; in our initial round of research support (the Pilot Projects Program); and in the scope and detail of our primary research funding announcements’ requirements.

Editor’s note: In another Health Affairs Blog post also published today, Joe Selby, the executive director of the Patient-Centered Outcomes Research Institute, responds to David Introcaso’s post below. For more on the concept of patient centeredness, comparative effectiveness research, and the Patient-Centered Outcomes Research Institute, see Health Affairs‘ October issue, “Current Challenges In Comparative Effectiveness Research.“

This past May, the Patient-Centered Outcomes Research Institute (PCORI) approved five research priority areas: “assessment of prevention, diagnosis and treatment options”; “improving health care systems”; “communication and dissemination”; “disparities”; and, “patient-centered outcomes research and methodological research”. In June PCORI announced 50 pilot project research awards totaling $30 million. PCORI is anticipated to spend $3 billion between now and 2019.

What does it mean to be “patient centered” and what does this then mean about ways of “improving “health care systems” and “communication and dissemination”? PCORI belies its “patient centered” mandate since it has not put a primacy on understanding and improving the interaction between the patient and the provider — the only way the quality of health care delivery is ultimately improved. None of the fifty PCORI pilot projects examine the quality of these interactions. Understanding and improving patient-provider interactions also explains how clinical evidence is produced or becomes meaningful.

Faced with the alarming implications of health care spending growth and suboptimal quality, many nations are turning to market-based reforms. The United Kingdom recently empowered primary care physician groups to choose among competing specialty care providers for their patients. In the United States, payers are introducing tiered copays, making patients pay more to visit what payers deem high-cost or low-quality providers. Even Sweden has introduced choice and competition in areas such as knee replacement and spinal surgery to curb the tide of rising costs.

The rationale behind these reforms is compelling and age-old: competition will unleash innovation, lower costs, and boost quality. But in health care, this logic hasn’t held, and largely for one simple reason: we do a terrible job of measuring, disclosing, and competing on the things patients actually care about: their survival, quality of life, and rates of complications after care.

Beneficial competition is rooted in informed choice. For competition to achieve its promise of incentivizing higher-value health care, patients and payers must first be able to determine and elect higher value options — providers who deliver better outcomes at the same or lower costs. Providers, meanwhile, must be able to benchmark their own performance against peers to find and adopt innovations that deliver improved outcomes for patients.

At Health Beat, Maggie Mahar has assembled a great collection of health policy blogging in a pre-election issue of the Health Wonk Review. Among the posts she highlights: A “contributing voices” Health Affairs Blog post by Joe Selby, the executive director of the Patient-Centered Outcomes Research Institute, and Rachael Fleurence, a scientist at the organization.

The authors describe the Patient-Centered Outcomes Research Institute’s two paths for choosing research to fund: an investigator-initiated path and a patient- and stakeholder-initiated path. As indicated by the title, they spend most of their time talking about the newer of the two paths, the patient- and stakeholder-initiated path.

“We recognize that our investigator-initiated process, even with the collaboration of stakeholders, could miss important questions that matter to patients and must be implemented judiciously. So we initiated a second path … that directly involves patients and other stakeholders in generating questions that address specific problems identified as having a significant impact on them and the health care system as a whole,” Selby and Fleurence write.

Video and speaker materials from the Health Affairs briefing on the journal’s October issue, “Current Challenges In Comparative Effectiveness Research,” are available on the Health Affairs website. The event is broken out by panel and by speaker, so you can view the entire briefing or just portions of particular interest.

Every day, patients, caregivers and clinicians must make any number of complex health care decisions. They might have to choose between several options for preventing, diagnosing or treating a disease or condition. They might need to decide between doing something specific and not doing anything at all. And often, they make these decisions without the evidence or information that answers the questions that matter most to patients.

The Patient-Centered Outcomes Research Institute (PCORI) was established to address this problem by funding comparative clinical effectiveness research to help patients make better-informed health and health care decisions, taking full and meaningful account of the concerns they have about the issues they face. Based on these principles, we developed our National Priorities for Research and Research Agenda, which guides our decisions about the kinds of research to fund.


To do this most effectively, we have implemented two complementary but equally critical paths: a broad, “investigator-initiated” funding process and a more directed “patient- and stakeholder-initiated” path. Together, they will help us build a robust portfolio of patient-centered outcomes research that will address the unmet needs of patients, caregivers, clinicians and other health care stakeholders.

As insurance exchanges emerge next year, and as states grapple with exactly what options health plans will provide, high-deductible health plans (HDHPs) are likely to be on the menu. But do such plans have the secret sauce to move participants from being passive health care consumers to savvy shoppers?

HDHPs have grown steadily in recent years. A 2012 report by America’s Health Insurance Plans estimates more than 13.5 million people are enrolled in high-deductible health plans with health savings accounts. Even in California — where benefit-rich HMO plans continue to have loyal followers — more than half of those who purchase insurance in the individual market report buying a plan with a deductible greater than $500.

To learn how HDHPs affect the health care marketplace and individual buyers, the RAND Corporation, with support from the California HealthCare Foundation and the Robert Wood Johnson Foundation, completed a large, five-year study of the effects of HDHPs on consumer use of, and spending on, health care. The analysis found that during the first year following enrollment, members of HDHPs had fewer hospitalizations than patients enrolled in traditional plans. They also had fewer episodes of care, fewer visits to specialists, and lower use of brand-name drugs — all of which reduced overall costs. However, there also was one troubling finding: Those in HDHPs cut back on preventive services along with other services. For example, cancer screenings in the first year of enrollment declined by 3 percent to 5 percent – even though such services were covered at 100 percent and not subject to deductibles.

The RAND study was summarized in the May issue of Health Affairs. The article suggested that growth of HDHPs to one-half of all employer-sponsored insurance (from 13 percent as of 2010) could save as much as $57 billion annually.

The October 2012 issue of Health Affairs provides an in-depth look at challenges in comparative effectiveness research. To surface issues around communication of research results, the issue includes a hypothetical comparative effectiveness case study of a fictional migraine drug and offers varying commentaries and analyses of the hypothetical case study from the Patient-Centered Outcomes Research Institute (PCORI), the Food and Drug Administration, the pharmaceutical industry, payers, and representatives of patient groups.

These and related articles raise questions about how the research might be applied to decision making across the health care system and the ways it could affect how pharmaceutical companies communicate to both health professionals and the public about competing treatments and options in the future.

Other studies examine additional issues related to comparative effectiveness research as well as topics of interest to the pharmaceutical and medical device industries, insurers, health care providers, and consumers.

The new Health Affairs volume will be discussed at a Thursday, October 11, briefing in Washington DC. The issue has funding support from the National Pharmaceutical Council.