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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

Editor’s note: Mary Barton also coauthored this post. 

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

Editor’s note: In addition to Roy Rosin, this post is also coauthored by Evan Fieldston and David Asch. 

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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New Health Affairs July Issue: The Impact Of Big Data On Health Care


July 8th, 2014

Health Affairs explores the promise of big data in improving health care effectiveness and efficiency in its July issue. Many articles examine the potential of approaches such as predictive analytics and address the unavoidable privacy implications of collecting, storing, and interpreting massive amounts of health information.

Big data can yield big savings, if they are used in the right ways.

David W. Bates of the Brigham and Women’s Hospital and coauthors analyze six use cases with strong opportunities for cost savings: high-cost patients; readmissions; triage; decompensation (when a patient’s condition worsens); adverse events; and treatment optimization when a disease affects multiple organ systems.

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Health Affairs Event Reminder: Using Big Data To Transform Care


July 7th, 2014

The application of big data to transform health care delivery, health research, and health policy is underway, and its potential is limitless.  The July 2014 issue of Health Affairs, “Using Big Data To Transform Care,” examines this new era for research and patient care from every angle.

You are invited to join Health Affairs Editor-in-Chief Alan Weil on Wednesday, July 9, for an event at the National Press Club, when the issue will be unveiled and authors will present their work.

WHEN:
Wednesday, July 9, 2014
9:00 a.m. – 12:30 p.m.

WHERE:
National Press Club
529 14th Street NW
Washington, DC, 13th Floor

REGISTER NOW

Twitter: Follow Live Tweets from the briefing @HA_Events, and join the conversation with #HA_BigData.

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New Drug And Device Approval: What Is Sufficient Evidence?


July 1st, 2014

Editor’s note: In addition to Jonathan Darrow, this post is also coauthored by Aaron Kesselheim. 

The federal Food, Drug, and Cosmetic Act gives the Food and Drug Administration (FDA) the authority to evaluate all prescription drugs and high-risk medical devices before they can be marketed to physicians and patients to ensure that they are safe and effective.

However, there is growing pressure to lessen the traditional standards for defining “safe and effective” for particularly promising therapies and accelerate patient access to these products.

A recent national health policy conference in Washington, D.C., explored the nature of the evidence needed for the regulatory approval of new therapeutics and the implications for patient care. The conference was organized by the Program On Regulation, Therapeutics, And Law (PORTAL) at Brigham and Women’s Hospital/Harvard Medical School, the National Center for Health Research, and the American Academy for the Advancement of Science (AAAS).

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Health Affairs Briefing: Using Big Data To Transform Care


June 23rd, 2014

The application of big data to transform health care delivery, health research, and health policy is underway, and its potential is limitless. The July 2014 issue of Health Affairs, “Using Big Data To Transform Care,” examines this new era for research and patient care from every angle.

You are invited to join Health Affairs Editor-in-Chief Alan Weil on Wednesday, July 9, for an event at the National Press Club, when the issue will be unveiled and authors will present their work.

WHEN:
Wednesday, July 9, 2014
9:00 a.m. – 12:30 p.m.

WHERE:
National Press Club
529 14th Street NW
Washington, DC, 13th Floor

REGISTER NOW

Twitter: Follow Live Tweets from the briefing @HA_Events, and join the conversation with #HA_BigData.

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Treated Prevalence Versus Spending Per Case: Responding To Starr And Coauthors


June 17th, 2014

I was surprised but pleased to see the Martha Starr, Laura Dominiak, and Ana Aizcorbe article in the May issue of Health Affairs replicating earlier work of Charles Roehrig and David Rousseau. These papers attempt to understand the role that treated disease prevalence and spending per case treated assume in accounting for the growth in average per capita healthcare spending. (Treated disease prevalence can increase when either a disease becomes more common or is diagnosed more frequently.) Starr and coauthors, like Roehrig and Rousseau, conclude that spending per case treated accounts for more of the growth in per capita spending than treated prevalence.

In our own work, my colleagues and I have addressed a different question. Our line of research focuses on changes in total health care spending – as opposed to per capita spending — over time. We have updated this work now through 2011 and our major conclusion remains the same; the rise in treated disease prevalence accounts for a slightly larger share of the growth in total healthcare spending than spending per case treated.

The three research teams employ different methods and use different spending measures. (In an earlier appendix, we described in detail the differences between our approach and that of Roehrig and Rousseau.) For example, our expenditures include home health and dental services, which are excluded in the paper by Starr and colleagues.

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The End Of The Blockbuster? Implications For Pricing Of New Drugs


June 16th, 2014

Editor’s note: In addition to Elias Dayoub, Anupam Jena and Darius Lakdawalla also coauthored this post. 

The productivity slowdown in drug innovation has been identified and widely discussed in recent years. In addition, the stagnation in the number of novel drugs (new molecular entities and new biologics) approved by the U.S. Food and Drug Administration (FDA) has also been well-documented.  Less well-appreciated, however, is the decline in the “applicability” of new drug approvals.  Each new drug treats fewer disease indications than drugs approved in earlier years.

Measuring the applicability of new drugs serves a dual purpose in helping better understand drug innovation. For one, assessing the number of indications for new drugs adds more nuance to assessments of growth or slowdown in the development pipeline. The relevant question is not necessarily how many new drugs are launched, but how many patients and diseases can be newly treated.  By accounting for the number of indications a new drug treats, one can better assess the number of patients the new therapy may benefit as well as the potential impact the drug approval will have on pharmaceutical spending.

The second important reason to measure pharmaceutical applicability is that it sheds light on how the costs of drug development translate into drug prices. If a drug has broad applicability to a larger number of indications, a drug developer can reasonably anticipate a greater number of patients will take the new medication. Conversely, if the drug treats only one indication, a developer expects fewer patients will use their drug.

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Health Affairs Web First: How Do Health Policy Researchers Use Social Media?


June 6th, 2014

As the United States moves forward with health reform, conveying complex information to the public becomes increasingly important. Social media represent an expanding opportunity for health policy researchers to communicate with the public and policy makers – but its use among these researchers appears to be low, according to a new study released today as a Web First by Health Affairs.

Authors David Grande, Sarah Gollust, Maximilian Pany, Jame Seymour, Adeline Goss, Austin Kilaru, and Zachary Meisel surveyed a sample of 325 health policy researchers who had registered for the 2013 Academy Health Annual Research Meeting.

The survey found small minorities using social media: 14 percent of participants reported tweeting, and 21 percent noted blogging about their research in the past year. Survey participants expressed reluctance to use social media, fearing it is incompatible with research, creates professional risks, and is not respected by their peers or their academic institutions.

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Exhibit Of The Month: Racial Disparities In Clinical Studies


May 27th, 2014

Editor’s note: This post is part of an ongoing “Exhibit of the Month” series. Readers who’d like to highlight other noteworthy exhibits from the same issue are encouraged to make their pitch in the comments section below.

This month’s exhibit, published in the May issue of Health Affairs, illustrates losses to follow-up among black men in clinical studies due to incarceration. Their findings, based on certain National Heart, Lung and Blood Institute cohort studies, raise concerns regarding the generalizability of clinical research.

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Health Policy Research And Disparities: A Health Affairs Conversation With Lisa Simpson And Darrell Gaskin


May 22nd, 2014

Earlier this year, AcademyHealth held its 2014 National Health Policy Conference; Health Affairs was a media partner for the NHPC. In a new installment of our Health Affairs Conversations Podcast series, we talk about the conference, as well as the challenges and opportunities facing the health services and health policy research communities, with AcademyHealth president and CEO Lisa Simpson. Before taking the helm of AcademyHealth, Dr. Simpson was director of the Child Policy Research Center at Cincinnati Children’s Hospital Medical Center and professor of pediatrics in the Department of Pediatrics, University of Cincinnati. She served as the Deputy Director of the Agency for Healthcare Research and Quality from 1996 to 2002.

We also take a close look at one of the NHPC sessions: “Community Health and Disparity: Moving Beyond Description.” (The disparities session is freely available to all readers.) Darrel Gaskin, who led the panel discussion, joins us as well. He is Deputy Director of the Johns Hopkins Center for Health Disparities Solutions and Vice Chair of AcademyHealth’s Board of Directors

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Patient-Centered Medical Home Evaluations: Let’s Keep Them All In Context


May 21st, 2014

Editor’s note: This post is authored by eleven participating leaders of the Pennsylvania Chronic Care Initiative; the full list is included at the end.

Since the American Academy of Pediatrics (AAP), American Academy of Family Physicians (AAFP), American College of Physicians (ACP) and American Osteopathic Association (AOA) issued their Joint Principles of the Patient-Centered Medical Home in 2007, there has been an explosion in medical home transformation activity in the United States. According to National Committee for Quality Assurance (NCQA), which offers one of multiple medical home recognition programs in the U.S., 5739 practices representing 27,820 clinicians had received NCQA medical home recognition status as of May 2013.

Medical homes have represented a promising approach to improving care for patients, reducing avoidable utilization, and improving professional satisfaction for clinicians. Extensive research by Ed Wagner and colleagues provided an evidence-supported basis for optimism, but only over the last several years has the model been implemented broadly.

Taking concepts tested in small application to broader context is challenging, as experience has shown. The state of Pennsylvania, along with its community of primary care practices and insurers, has been committed to testing and adopting the medical home model. A key catalyzing effort was the Pennsylvania Chronic Care Initiative.

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New Health Policy Brief: Breakthrough Therapy Designation


May 16th, 2014

The latest Health Policy Brief from Health Affairs and the Robert Wood Johnson Foundation describes a new expedited drug development pathway designed to speed up the Food and Drug Administration’s (FDA’s) premarketing approval process for drugs and devices that treat serious or life-threatening conditions.

Created under a 2012 law, the Food and Drug Administration Safety and Innovation Act (FDASIA), a drug may be designated a “breakthrough therapy” if it shows far more promise over comparable treatments already on the market. At that point, the FDA will initiate a special rapid approval process.

The pharmaceutical industry has responded positively to this law, and as of last month the FDA has received 178 requests for this designation. The law, whose full impact will not be known for several years, carries significant implications for approaches to clinical development, patient access to new drugs, and the drug regulations process itself.

Topics covered in this brief include:

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