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Medicare, Medicaid, And Pharmaceuticals: The Price Of Innovation


November 20th, 2014

Editor’s note: This post is part of a series of several posts stemming from presentations given at “The Law of Medicare and Medicaid at Fifty,” a conference held at Yale Law School on November 6 and 7.

Through much of the last half century, Medicare and Medicaid (MM) have not for the most part supported research intended to lead to new drugs. For their role in drug development, we need to look to infrastructure and incentives. The record of the National Institutes of Health (NIH) illustrates the potential of both for pharmaceutical innovation. The current budget of NIH, the big elephant in the zoo of the federal biomedical enterprise, is $30 billion, but apart from a dozen small programs devoted to targeted drug development, most of these billions are not aimed directly at pharmaceutical innovation (See page 234).

Yet the NIH investment in biomedicine has indirectly fueled drug development in the private sector to a huge degree. It has paid for the training of biomedical scientists and clinicians, many of whom went on to staff the drug industry, especially its laboratories. NIH-sponsored research has also generated basic knowledge and technologies and it has encouraged universities to spin out their potentially useful findings into the industry by allowing for the patenting and licensing of the findings.

Like NIH, MM has helped fuel drug development indirectly by supporting selected experimental cancer treatments, medical education, and some clinical research and training. But investment in these activities has been small and their impact on drug development apparently very limited. In contrast to NIH, the MM stimulus to drug innovation has resided not in the production of new scientists or the patented uses of new knowledge, but principally in markets and pricing.

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Continuity Of Care For Chronic Conditions: Threats, Opportunities, And Policy


November 18th, 2014

Continuity of care is a bedrock principle of the patient-doctor relationship and is believed to be a fundamental attribute of high-quality medical care. Mounting evidence suggests that continuity of care for patients with chronic conditions prevents hospitalizations, reduces health care costs, and may prolong life in some populations.

Because patients are most likely to have longitudinal relationships with their pediatricians, family physicians, and internists, taken together, these primary care doctors are integral to translating continuity into meaningful care coordination. However, within the rapidly shifting landscape of health care delivery in the United States, continuity of care is simultaneously threatened and promoted by emerging care models.

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Battle Lines Drawn Over Biosimilar Application And Patent Disclosure Process


November 17th, 2014

The Biosimilars Price Competition and Innovation Act of 2009 (BPCIA) introduced a long-awaited, and highly-supported, abbreviated route to market for “biosimilar” and “interchangeable” biologic products. The goal was to create incentives for development and reduce health care costs in the same way that previous legislation had accomplished in the generic arena over three decades ago.

However, widespread criticism of BPCIA provisions to facilitate interactions between biosimilar applicants and reference biologic sponsors regarding application submission and patent status was swift and unrelenting. Attorneys practicing in the realm of Hatch-Waxman litigation for generic drugs balked at the drastic differences between the familiar public process grounded in required patent disclosures, FDA Orange Book listings, and generic applicant certifications and the private, iterative process set forth in the BPCIA. Legal scholars, industry representatives, and practitioners alike projected that a courtroom battle regarding real-world operation of the provisions was imminent.

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Health Affairs Twitter Chat With PCORI


November 12th, 2014

Health Affairs recently partnered with the Patient Centered Outcomes Research Institute (PCORI) to produce three videos about ways patients and practitioners are incorporating patient engagement in health care decisions. The videos are hosted and reported by journalist John Dimsdale.

Twitter Chat On Patient Engagement

To further the conversation, Health Affairs and PCORI will host a Twitter chat with Sue Sheridan, director of patient engagement for PCORI, on Monday, November 17 at 2 p.m. ET on the topic of patient engagement in research.

Monday, November 17
2-3 p.m. ET
Join the conversation using #PatientHC!

Join in the conversation with the hashtag #PatientHC and follow us @Health_Affairs. Have a question about patient engagement in research? Send it along using #PatientHC.

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Addressing The Threat Of Antibiotic Resistance: Policy Solutions To Fix A Broken Pipeline


November 6th, 2014

Recently, the White House released a major new national strategy to combat antibiotic resistance. As efforts begin to translate that unprecedented announcement into action, it is critical that any strategy to address resistance contain a plan to ensure an adequate antibiotic development pipeline. The overall number of antibiotics reaching the market has declined over time, with 29 and 23 new antibiotics approved in the U.S. in the 1980s and 1990s, respectively, but only nine between 2000 and 2010.

Meanwhile, the evolution of drug-resistance has outpaced the development of new antibiotics. Doctors routinely encounter patients with infections that do not respond to currently available treatments. Some life-threatening infections, such as those caused by carbapenem-resistant Enterobacteriaceae, or CRE, are resistant to nearly all available therapies. The Centers for Disease Control and Prevention estimates that in the United States at least two million people are sickened by resistant bacteria each year and 23,000 die as a result.

Current State of Antibiotic Development

In order to better understand the pipeline and evaluate policies to spur antibiotic development, the Pew Charitable Trusts has identified antibacterial drugs in clinical development (Phases 1-3) for the U.S. market. Published on our website and updated quarterly, this resource provides policymakers, the medical community, and industry stakeholders with an up-to-date picture of drugs in development.

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Lessons from Ebola: The Infectious Disease Era, And The Need To Prepare, Will Never Be Over


October 28th, 2014

With the wall-to-wall news coverage of Ebola recently, it’s hard for many to distinguish fact from fiction and to really understand the risk the disease poses and how prepared we are to fight it.

Fighting infectious diseases requires constant vigilance. Along with Ebola, health officials around the globe are closely watching other emerging threats: MERS-CoV, pandemic flu strains, Marburg, Chikungunya and Enterovirus D68. The best defense to all of these threats is a good offense — detecting, treating and containing as quickly and effectively as possible.

And yet, we have consistently degraded our ability to respond to these new, emerging and re-emerging threats by underfunding and undercutting existing capabilities and expecting the country to ramp up overnight when new threats emerge.

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How Engaging Patients Can Improve Care And Health Outcomes


September 26th, 2014

Patients and caregivers are gaining momentum as powerful new resources in efforts to improve the health care system. They are increasingly becoming active partners in their own care, as well as seeking to make the health care delivery system more responsive to their needs and easier to navigate. And they are increasingly engaging as collaborators in planning and conducting research, and disseminating its results, with the goal of producing evidence that can help patients and those who care for them make better-informed decisions about the clinical choices they face.

It is this last trend that led the Patient-Centered Outcomes Research Institute (PCORI) to support Health Affairs in developing a series of videos illustrating some of the ways that patients are bringing their unique experiences and community connections to efforts to improve care for themselves and others. This includes stories of how patients are becoming partners in research designed to address the outcomes important to them, taking account of their own concerns and circumstances.

Seen through this lens, being a research partner goes well beyond being the subject of a trial. Rather, it means helping to guide researchers in formulating the questions to be studied, making the right clinical comparisons, looking at appropriate populations, and focusing on the outcomes important to patients. This should greatly increase the chance that the research findings will produce relevant results that can have a real-world impact — something we plan to evaluate carefully over time.

Meaningful patient engagement is at the heart of PCORI’s approach to research, and several of the patients featured in the videos have in fact partnered with researchers in just this way in patient-centered outcomes research (PCOR) studies we fund. They recognize that PCOR, a form of comparative clinical effectiveness research that focuses on issues of concern to patients, is a vital building block for developing truly patient-centered care and health policy, more effective treatments, and better outcomes.

In the following sections, we highlight the projects mentioned in the videos to give you an idea of how patients and community members are partnering in research projects.

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Global Health Update: High Bed Occupancy Rates And Increased Mortality In Denmark


September 24th, 2014

High levels of bed occupancy are associated with increased inpatient and thirty-day hospital mortality in Denmark, according to research recently published in the July issue of Health Affairs.

Authors Flemming Madsen, Steen Ladelund, and Allan Linneberg received considerable media attention in Denmark for their research findings. For one major Television channel, it topped Germany’s victory in the World Cup finals.

In another story from the Danish newspaper, Information, Councillor Ulla Astman, Chairman of the North Denmark Regional Council and second highest ranking politician, who runs all of the Danish public hospitals, reportly stated that “we have to live with it [the increased mortality],” since they cannot afford to reduce bed occupancy.

“Or die with it,” said lead author Madsen, a pulmonary physician and director of the Allergy and Lung Clinic in Helsingør, Denmark, at the July 9 Health Affairs briefing, “Using Big Data To Transform Care.” Madsen, who left his position as director of the Department of Internal Medicine at Frederiksberg Hospital in Copenhagen to pursue this research, believes that Astman’s statement explains why they have a bed shortage problem and supports his argument that bed shortage is a result of planning.

“It is dangerous to focus on productivity without looking at the consequences,” says Madsen.

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Should We Be Done With Describing Health Disparities?


September 17th, 2014

A recent Health Affairs podcast featured a conversation with AcademyHealth president and CEO Lisa Simpson on health disparities along with Darrell Gaskin, the lead of one of the panel sessions at the 2014 National Health Policy Conference (NHPC), “Community Health and Disparity: Moving Beyond Description.” The conversation endorses interventions rather than descriptions as the future direction of health disparities research.

But should we be done with describing health disparities? In a paper we recently published online in the International Journal for Equity in Health, we show that the answer is: Not entirely.

In this paper, using large, publicly available data, 2008, 2009, and 2010 Behavioral Risk Factor Surveillance System (BRFSS) Selected Metropolitan/Micropolitan Area Risk Trends (SMART) and 2008, 2009, and 2010 United States Birth Records from the National Vital Statistics System, we reported education-, sex-, and race-related disparities in four health outcomes (poor/fair health, poor physical health days, poor mental health days, and low birthweight) in each of the selected 93 counties in the United States representing about 30 percent of the U.S. population.

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Advancing Innovation To Eliminate Health Disparities


September 4th, 2014

The advent of population health management, community-based care coordination and mobile health technologies provide a promising opportunity to address longstanding and persistent health disparities. Separately each adds a new dimension to research and analysis, and to individual and community-level public health prevention and access to quality care. Together, providers, payers and researchers alike can acquire a richer understanding of contextual, environmental, and behavioral factors that contribute to disparate outcomes in health.

Existing innovations in data capture, epidemiologic profiling, clinical translation, and workforce development have yet to be taken to scale or appropriately deployed in a manner that would benefit vulnerable populations. Meaningful use technologies, for example, appear to be stuck in the proverbial pipeline with resistance in uptake and limited distribution of incentives. Meaning access and application in poor and disparate communities where they are more often subjects of research and not partners in innovation is far off.

What public health, and community-based and clinically focused interventions need is a fresh look at how health disparities are measured and the processes for application of solutions to needy populations.

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The Winding Path To Effective Bundled Payment


August 28th, 2014

Tom Williams and Jill Yegian’s excellent blog post makes a great companion to our recent paper on the evaluation of the Integrated Healthcare Association (IHA) Bundled Payment Demonstration. Williams and Yegian offer lessons from their experience implementing a demonstration project that failed to meet its original objectives. This type of analysis is essential.

It’s not unusual for a demonstration to fall short of its original objectives. Learning from such cases is part of the innovation process. This is especially worthwhile for bundled payment, which has many potential benefits for patients, providers, and payers.

None of the barriers encountered in IHA’s demonstration signal a “death sentence” for bundled payment. However, the demonstration clearly shows that bundled payment is difficult to implement.

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Rescue Me: The Challenge Of Compassionate Use In The Social Media Era


August 27th, 2014

The Development of Brincidofovir and its Possible Use to Treat Josh Hardy

Last March 4, seven-year old Josh Hardy lay critically ill in the intensive care unit at St Jude Children’s Hospital in Memphis, Tennessee with a life-threatening adenovirus infection. His weakened immune system was unable to control the infection, a complication of a bone marrow stem cell transplant he needed as a result of treatments for several different cancers since he was 9 months old.

His physicians tried to treat the adenovirus with an anti-viral agent, Vistide (IV cidofovir), but had to stop due to dialysis-dependent renal failure. They were aware of another anti-viral in Phase 3 clinical development, brincidofovir, an oral compound chemically related to Vistide. In earlier clinical testing brincidofovir had shown the potential for enhanced antiviral potency and a more favorable safety profile.

Chimerix (where one of us, Moch, was CEO), a 55 person North Carolina-based biopharmaceutical company, had previously made brincidofovir available to more than 430 critically ill patients in an expanded access program for the treatment of serious or life-threatening DNA viral infections, including adenovirus as well as herpes viruses (such as cytomegalovirus) and polyomaviruses.  This program started in 2009 as a series of individual physician-sponsored emergency INDs — investigational new drug exemptions issued in physician-certified compassionate use situations.

The program evolved via word of mouth to the extent that brincidofovir was made available under emergency INDs for more than 215 patients. During 2011, Chimerix received funding from Health and Human Service’s Biomedical Advanced Research and Development Authority (BARDA) to provide brincidofovir to an additional 215 critically ill patients for the purpose of gaining insights into the potential use of brincidofovir as a medical countermeasure against smallpox. (Department of Health and Human Services Contract No. HHSO100201100013C.  For further information on brincidofovir Study CMX001-350, see ClinicalTrials.gov Identifier NCT01143181.)

When BARDA funding ended in 2012, Chimerix stopped accepting new requests for compassionate use under its expanded access program. Although requests continued from physicians around the world, the company decided that it was in the best interests of the greatest number of patients to devote its limited human and financial resources to the clinical development pathway necessary for FDA approval of brincidofovir.

This post examines the implications of the ultimately successful campaign waged by Josh Hardy’s family to obtain access to brincidofovir for their son. We discuss several issues raised by the Hardy case, including the overarching question of whether it is fair for social media or influence of any form to play a role in determining which patients get access to experimental treatments; whether rescuing individual patients in need can be reconciled with an evidence-based regulatory approval process for new therapies; and whether there is a duty to “rescue” terminally ill patients by paying for access to experimental therapies.

Finally, we propose a new framework for regulating access to experimental treatments.

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An Evolutionary Approach To Advancing Quality Measurement


August 8th, 2014

The Medicare Payment Advisory Commission’s June report, like many current discussions on measuring quality in health care, focuses on the need for measures of overuse and outcomes.  The National Committee for Quality Assurance (NCQA) agrees and is committed to developing better measures for these important priorities.

NCQA’s Healthcare Effectiveness Data and Information Set (HEDIS), a tool used by more than 90 percent of America’s health plans to measure performance, includes a readmissions outcome measure, intermediate outcome measures like blood pressure and blood sugar control for diabetics, and measures of relative resource use.

MedPAC suggests focusing on important resource use outcomes, including preventable admissions, emergency department visits, mortality, and readmissions, as well as healthy days at home. These are important for helping us understand the costs of care.

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Seeing Clinician Slack As A Strategic Investment


August 1st, 2014

The French filmmaker Jean Renoir said, “the foundation of all civilization is loitering,” expressing the view that transformative value is created when people have time to step back and imagine a better way. Most businesses today seem to take a contrary position. Organizations in health care and beyond have spent a generation attacking slack, removing inefficiencies within processes and budgets. The narrow operating margins of health systems have led many to turn to companies such as Toyota or General Electric (GE) to learn about lean or Six Sigma techniques.

Subsequently, frontline clinicians are easy targets for attacks on slack. They are among the most expensive personnel within health systems and their productivity drives profitability. Working at the top of one’s license is set as a goal — reflecting the view that anything that can be delegated to a less expensive resource should be, and that everyone should be adding directly measurable peak value at all times.

A problem in translating lessons derived from general management experience is that even when conceptually appealing, they rarely meet medicine’s evidentiary standards defined by randomized trials or carefully controlled observations with homogenous populations, standardized interventions, and explicit outcomes. Instead, management lessons often take the form of stories – and perhaps only those selected to support a particular point.

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Individual Patient Expanded Access: Developing Principles For A Structural And Regulatory Framework


July 31st, 2014

Editor’s note: In addition to Meaghan George, Sara Bencic and Darshak Sanghavi also coauthored this post. 

Individual patient expanded access, sometimes termed “compassionate use,” refers to situations where access to a drug still in the development process is granted to patients on a case-by-case basis outside of a clinical trial, prior to completion of mandated clinical trials and approval by the Food and Drug Administration (FDA). This typically involves filing a single patient or emergency investigational new drug (IND) request with the Food and Drug Administration and voluntary release of the drug by the manufacturer.

Generally, the following criteria must be met: there is reasonable expectation of meaningful benefit despite the absence of definitive clinical trial data, the patient has a serious or life-threatening condition, there are no comparable or satisfactory treatment alternatives, and there are no suitable clinical trials for the drug available to the patient. This form of expanded access, which is the focus of this paper, is different from the situation in which a drug is discharged to a large group of needy patients in the interval between successful phase 3 trials and presumed FDA approval, a strategy often termed a “treatment” IND or protocol, which was initially used in the 1980s for releasing zidovudine to patients with acquired immune deficiency syndrome.

A Call to Action: The Importance of Expanded Access Programs

The Engelberg Center for Health Care Reform at the Brookings Institution recently invited senior leaders from several pharmaceutical companies, two bioethicists, a senior FDA representative, and a patient advocate to share experiences and discuss organizational strategies related to expanded access (see acknowledgements). A driving factor for this meeting was a recent flurry of highly public cases of desperate patients seeking access to experimental drugs, which lead to social media campaigns and media coverage.

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Lessons Learned: Bringing Big Data Analytics To Health Care


July 14th, 2014

Big data offers breakthrough possibilities for new research and discoveries, better patient care, and greater efficiency in health and health care, as detailed in the July issue of Health Affairs. As with any new tool or technique, there is a learning curve.

Over the last few years, we, along with our colleagues at Booz Allen, have worked on over 30 big data projects with federal health agencies and other departments, including the National Institutes of Health (NIH), Centers for Disease Control (CDC), Federal Drug Administration (FDA), and the Veterans Administration (VA), along with private sector health organizations such as hospitals and delivery systems and pharmaceutical manufacturers.

While many of the lessons learned from these projects may be obvious, such as the need for disciplined project management, we also have seen organizations struggle with pitfalls and roadblocks that were unexpected in taking full advantage of big data’s potential.

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Recent Health Policy Brief: E-Cigarettes And Federal Regulation


July 11th, 2014

The latest Health Policy Brief from Health Affairs and the Robert Wood Johnson Foundation (RWJF) describes federal policy makers’ recent efforts to propose rules for e-cigarette regulation. E-cigarettes, virtually non-existent ten years ago, have skyrocketed in popularity, including among people who claim to use e-cigarettes as a tool to help them quit smoking altogether.

The 2009 Family Smoking Prevention and Tobacco Control Act gave the Food and Drug Administration (FDA) authority to oversee the manufacture, marketing, distribution, and sale of regulated tobacco products such as cigarettes, tobacco in cigarettes, roll-your-own, and smokeless tobacco. But it left unregulated other tobacco products such as cigars, pipe and hookah tobacco, nicotine gels, and e-cigarettes.

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Interview: IOM President Harvey Fineberg Reflects On Lessons Learned


July 10th, 2014

On June 16, 2014, I spoke with Dr. Harvey V. Fineberg, as he wrapped up his second six-year term as president of the Institute of Medicine (IOM). We discussed how requests for work come to the IOM, the attributes of IOM reports that make them effective, and how the IOM maintains a strong voice in a crowded field.

Dr. Fineberg shared lessons learned from his analysis of events surrounding the Swine Flu immunization effort of 1976, and how, today, those lessons help him guide the IOM’s thinking about program assessment. We also discussed Dr. Fineberg’s work to bring together the various arms of the National Academies of Sciences to improve health.

You can listen to the full podcast at Health Affairs. These are a few of my favorite quotations taken from our discussion:

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The Era Of Big Data And Its Implications For Big Pharma


July 10th, 2014

Editor’s note: For more on the topic of big data, check out the July issue of Health Affairs In addition to Marc Berger, Kirsten Axelsen and Prasun Subedi also coauthored this post. 

Health care research is on the cusp of an era of “Big Data” — one that promises to transform the way in which we understand and practice medicine.

The Big Data paradigm has developed from two different points of origin. First, significant efforts to digitize and synthesize existing data sources (e.g., electronic health records) have been driven by policy and practice economics. Second, a wide range of novel ways to capture both clinical and biological data points (e.g., wearable health devices, genomics) have emerged.

The era of Big Data holds great possibility to improve our ability to predict which health care interventions are most effective, for which patients, and at what cost.

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ACAView: New Findings On The Effect Of Coverage Expansion Since January 2014


July 9th, 2014

Editor’s note: In addition to Josh Gray, Iyue Sung also coauthored this post. 

Together, athenahealth and the Robert Wood Johnson Foundation (RWJF) have undertaken a new joint venture called ACAView, as part of the foundation’s Reform by the Numbers project, a source for timely and unique data on the impact of health reform.

The goal of ACAView is to provide current, non-partisan measurement and analysis on how coverage expansion under the Affordable Care Act (ACA) is affecting the day-to-day practice of medicine. athenahealth provides a single-instance, cloud-based software platform to a national provider base.

Any information that our clients enter using our software is immediately aggregated into centrally hosted databases, providing us with timely visibility into patient characteristics, clinical activities, and practice economics at medical groups around the country.

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