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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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What’s Past Is Prologue: Making The Case For PET Beta-Amyloid Imaging Coverage


April 9th, 2014

Editor’s note: This post is published in conjunction with the April issue of Health Affairs, which features a series of articles on Alzheimer’s disease.

In September of 2013, CMS issued its final decision memo that concluded positron emission tomography- amyloid beta (PET Aβ) imaging is “not reasonable or necessary”, finding “insufficient evidence” that use of this diagnostic tool would improve health outcomes for patients with dementia or neurodegenerative disease. As such, PET Aβ imaging to help diagnose Alzheimer’s disease (AD) is not a covered service for Medicare beneficiaries except for those enrolled in CMS-approved clinical trials.

CMS’ final decision underscores the emerging new paradigm for coverage decision-making, requiring innovators not only to demonstrate to FDA’s satisfaction that their products are effective, but also to prove to CMS and other payors that their use will improve clinical outcomes. This paradigm will increase confidence in the value and health benefit of new technologies, although it will make the path to coverage more difficult and uncertain for diagnostic developers.

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Accelerating Medicines Partnership: A New Public-Private Collaboration For Drug Discovery


April 8th, 2014

Editor’s note: This post is also coauthored by Yongtian Tan and is published in conjunction with the April issue of Health Affairs, which explores the many subjects raised by Alzheimer’s disease including a new public-private research collaboration designed to produce improved treatments.

Earlier this year, the National Institutes of Health joined forces with ten major pharmaceutical companies and several nonprofit disease interest groups to create the Accelerating Medicines Partnership (AMP). With an integrated governance structure consisting of representatives from all partners, the AMP venture aims to combine public-private expertise and pooled resources to reduce the time and cost of  developing biomarkers for therapeutic targets.

The initial capitalization is reported to be $230 million.  The AMP is the first national cross-sector partnership of its size and scale, and is the latest initiative in the drug development market to embrace open data exchange, encouraging collaboration over competition as pathways for promoting innovation.

The AMP management chose to focus on four diseases—Alzheimer’s disease, type 2 diabetes mellitus, rheumatoid arthritis, and systemic lupus erythematosus—in which there was solid knowledge about the underlying pathophysiology, a sufficient level of potential therapeutic targets open to pursuit, and a lack of substantial individual manufacturer commitment.  The latter criterion explains why more prevalent diseases such as cancer did not make the list.

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Health Policy Brief: Biosimilars


October 10th, 2013

A new Health Policy Brief from Health Affairs and the Robert Wood Johnson Foundation explains key elements of the Biologics Price Competition and Innovation Act (BPCIA), a provision of the Affordable Care Act. The BPCIA authorizes the Food and Drug Administration (FDA) to develop an accelerated approval process for biosimilars. Biosimilars are follow-on versions of original therapies derived from a biological source, including vaccines, antitoxins, and blood products—commonly referred to as biologics.

The FDA released draft guidelines for an accelerated approval process for biosimilars in February 2012, but the widespread introduction of biosimilar drugs is likely several years away. This policy brief discusses the opportunities and challenges of producing and introducing biosimilars into the US marketplace.

Topics covered in this brief include:

What’s the background? The brief explains the evolution of the 1984 Hatch-Waxman Act, which provides the regulatory foundation for modern generic drugs. However, Hatch-Waxman does not apply to biosimilars, and, with demand for biologics expanding rapidly in the global marketplace, the Obama administration needed to establish a new regulatory review process in the United States for this type of drug. The United States can look to the work of the European Medicines Agency, which has an approval process in place. An article in the October 2013 issue of Health Affairs, by Francis Megerlin and colleagues, describes the European experience.

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System Transformation Intensifies Demand for Evidence: The Trust Conundrum


September 10th, 2013

Although we have witnessed historic lows in the rate of growth of healthcare spending, the implementation of the Affordable Care Act and secular trends have created fundamentally new dynamics in how providers, clinicians, and patients are exposed to the cost of care. New payment and delivery models being tested by the Center for Medicare & Medicaid Innovation are shifting the financial risk for managing population health to providers and clinicians. In addition, state health insurance exchanges are poised to introduce new insurance products targeted to meet the needs of price-sensitive consumers. In Silver Plans, designed to have lower premiums, the out-of-pocket payments for brand named pharmaceuticals could be $70 per prescription.

At the same time, specialty pharmaceuticals are the fastest growing sector in healthcare. ExpressScript’s Drug Trend Report estimates 22 percent growth in spending for specialty products in 2014. These dynamics have resulted in heightened demand for evidence on the safety, effectiveness and value of pharmaceuticals by health plans, clinicians, and patients. Further, these decision-makers desire real-world evidence demonstrating durable patient benefits and cost savings from use of pharmaceuticals.

Simple enough. Clear demands to an industry with billions invested in research. What’s the problem? Trust. The current level of trust in biopharmaceutical and medical products industry-funded research and credibility of industry-sponsored study results is extremely low. Some of this is a result of highly publicized fines and corporate integrity agreements (CIAs) levied for off-label marketing, inappropriate financial arrangements with providers/researchers, and lack of transparency about trial results. The resulting loss of trust in industry and its research weakens the role of a key player in meeting the demands for more evidence.

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Misguided Genetic Exceptionalism


August 3rd, 2012

While society should be careful about its uses of genetic testing and its efforts to modify the genome, some people overreact when dealing with DNA. Consider two recent examples reported in the media. A genetic cancer test. In a fascinating series on genetics in medicine, Gina Kolata reported in the New York Times on a genetic test […]

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Your Genome Belongs To You


June 8th, 2012

Just four years ago, only two people in the world had their genome sequenced:  James D. Watson (co-discoverer of the structure of DNA) and J. Craig Venter (former President of the firm that mounted a private-sector rival to the Human Genome Project).  There are now many thousands of such people.  At genome meetings, scientists are […]

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Get A Grippe: Lessons Learned From The Controversy Over Publication Of Pandemic Flu Research


May 8th, 2012

If one were to try and identify what issue has most roiled the biomedical community in the past few months it is surely the effort to censor two papers describing genetic modifications of the H5N1 flu virus. Background.  Last December, the U.S. National Science Advisory Board for Biosecurity (NSABB) was asked by the U.S. National […]

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Adolescents And Young Adults: Bringing A Neglected Group Into Cancer Research


January 13th, 2012

“A child is not a small adult,” but an adolescent is not a large child.  Adult oncologists, reluctant to care for cancer patients under the age of 16, believe that adolescent and young adult (AYA) cancer patients should be within their purview.  We believe younger cancer patients are a special group needing special attention, even […]

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Medicare Part D drug pricing


April 8th, 2010

Reducing Medicare Part D drug prices requires careful tools, not simply the power to negotiate

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The Case For A Follow-On Biologics Global Health Exclusivity Incentive


July 14th, 2009

Global health issues, especially those affecting the world’s poor, rarely gain anywhere near the attention that the U.S. public and policymakers give to domestic concerns.  However, in one small corner of the current health reform discussion, there is a golden opportunity not only to reduce U.S. health care costs but also to improve the health […]

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Should FDA Regulate Nanomedicine Differently?


June 20th, 2008

Editor’s Note: In an interview published this week, Health Affairs Contributing Editor Barbara Culliton asks Food and Drug Administration (FDA) Associate Commissioner For Science, Norris Alderson, about his agency’s regulation of nanomedicine and the potential for health care cost savings. Here’s an excerpt of their conversation: Barbara Culliton: Nanomedicine is the “next big thing” in […]

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COMPARATIVE EFFECTIVENESS INFORMATION: Would The U.S. Use It In A NICE Way?


June 12th, 2007

What happens when a government agency in charge of assessing the effectiveness of medical interventions crunches numbers and tells pharmaceutical companies their drugs are just too expensive? Sometimes, the government gets a better deal. Twice last week, the much-feared National Institute for Health and Clinical Excellence (NICE) in England and Wales was a factor in […]

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BIOTECH: The Business of Biotechnology and Biotechnology-Bashing


March 15th, 2007

Biotechnology firms constitute a subsector of the larger pharmaceutical industry (“drug companies with needles,” we call them), but to date have been spared from the blood sport of American health punditry, pharma-bashing. While drug firms routinely are castigated for their sins, real and imagined, biotech firms have been appreciated as innovative and entrepreneurial startups (rather […]

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BIOTECH: The Myth of Value-Based Pricing – So Far


February 20th, 2007

At last policymakers and readers are being set straight that the prices of drugs are not related to the immense costs of research and development but to “what the market will bear,” as James Robinson put it in his Health Affairs Blog post last fall. Never mind that pharmaceutical executives told Congress and everyone else […]

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BIOTECH: A Road Toward Value-Based Pricing


October 25th, 2006

In his post, Jamie Robinson has raised the specter of an upside-down world of setting prices for biomedical innovations based on cost. Before we examine his serious admonition to focus on value in pricing new biotechnology drugs, let’s walk down the other trail: the argument that drugs should be pricy because they cost so darn […]

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BIOTECH: Evaluating Interventions With No Close Substitutes


October 24th, 2006

What is the appropriate price for a biotechnology product? Jamie Robinson’s thoughtful post touches all the right bases in arguing that discussing the role of value in pricing is far preferable to discussing the cost of research, development and production.

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BIOTECH: Value-Based Pricing In Biotechnology


October 23rd, 2006

The biotechnology industry has grounds for complaint. The research pipeline is disgorging breathtaking new treatments for cancer, rheumatoid arthritis, multiple sclerosis, and other once-intractable diseases. But instead of praise, or in addition to praise, the industry finds itself subjected to ever-louder criticism of its prices and earnings. America again seems to demand the best health […]

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