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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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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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Washington Wakes Up To Socioeconomic Status


July 11th, 2014

John Mathewson, executive vice president of Health Care Services for Children with Special Needs (HSC) – a Medicaid managed care plan in D.C. for children on Supplemental Security Income (SSI) – recently spoke at the Association for Community Affiliated Plans (ACAP) CEO Summit before the July 4 Recess.

Mathewson described what he has dubbed The Kitten Paradox: When HSC examined environmental factors for children with asthma, it found that the presence of pets in the house was a common thread, not too far behind having a smoker around. Yet, it turns out the value a cat brings by protecting from mice or spawning a litter for sale outweighs any financial costs to the family associated with an ER visit, which are often free or carry a low copayment. Thus the paradox.

An awardee at the conference, Hennepin Health, catalogued the evidence showing that reliable housing can improve health outcomes, including improving mental health and lowering emergency room and inpatient hospital utilization.

The focus of these sessions was the social determinants of health, and a lot of these safety net health plan leaders’ heads were nodding throughout. The plans, which disproportionately serve Medicaid enrollees and thus ‘dual eligible’ seniors in Medicare, know something about the importance of social determinants that the health policy community – at least in Washington – is only now slowly waking up to.

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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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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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The Supreme Court And The Contraception Mandate: A Temporary Setback For Contraception Coverage


June 30th, 2014

Editor’s note: See Health Affairs Blog for more coverage of the Supreme Court’s Hobby Lobby decision.

Today, the United States Supreme Court ruled that for-profit companies may avoid providing contraception coverage to employees if the companies sincerely object on religious grounds. At its narrowest interpretation, this decision is a significant but remediable setback for women’s reproductive health. At its broadest (but least likely interpretation), the decision has the potential to wreak havoc on public health regulation.

The legal challenges to the Affordable Care Act’s (ACA’s) contraception mandate have been well described in three previous Health Affairs Blog posts. To briefly recap, though, the ACA requires preventive services to be covered without copayments or other cost sharing in most employer-supported health plans. To implement this requirement, the Department of Health and Human Services (HHS) issued regulations that include all FDA-approved contraceptives, and a company that does not provide no-cost coverage of contraception is subject to substantial penalties.

Three groups of employers are exempt from the mandate: small businesses with less than 50 employees, purely religious employers, and “grandfathered” plans that have not changed meaningfully since the ACA was passed. Additionally, religiously affiliated non-profits (such as universities and hospitals) received a special accommodation from HHS by which women can receive contraception from third-party insurers at no extra cost to employees or the organization if the organization objects to covering contraception and identifies an alternate insurer.

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The 2014 Culture of Health Prizes


June 25th, 2014

Today the Robert Wood Johnson Foundation awarded its 2014 Culture of Health Prize to six communities. These communities —  Brownsville, TexasBuncombe County, North CarolinaDurham County, North CarolinaSpokane County, WashingtonTaos Pueblo, New Mexico; and Williamson, West Virginia – were selected for the work they have done to place a high priority on health and bring partners together to drive local change.

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Correcting The Blind Spot In Accountability: The Role Of Pharmacy Care


June 25th, 2014

Editor’s note: In addition to William Shrank, this post is also coauthored by Andrew Sussman, Patrick Gilligan, and Troyen Brennan.

The Centers for Medicare and Medicaid Services (CMS) recently issued a Request for Information (RFI) to solicit suggestions about how to improve the Accountable Care Organization (ACO) programs. CMS stated that they seek recommendations about how the ACO program might evolve to “encourage greater care integration and financial accountability.”

The RFI explicitly stated that they seek information about how to better integrate Part D expenditures into ACO cost calculations to make pharmaceuticals part of the approach to care delivery and health care transformation.

The deadline for comments about encouraging Part D integration in ACOs has now passed. But the issue extends beyond ACOs. In addition, bundled payments and patient-centered medical home programs target hospitals and primary care providers to promote higher quality and lower cost care. All these programs have largely excluded prescription drug costs in their calculus, and offer no direct incentives for Part D plans to participate in and improve care.

Nonetheless, retail pharmacies and Part D plans have developed a number of strategies to participate. As CMS and policymakers reconsider ACO regulations to stimulate greater integration of prescription drug use in delivery system reform, we thought it important to offer a description of the marketplace response to payment reform activities at large.

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The Role Of Sales Representatives In Driving Physicians’ Off-Label Prescription Habits


June 19th, 2014

Off-label prescribing is widespread in Canada and the United States (U.S.). One in nine prescriptions for Canadian adults are for off-label uses with the highest percentages coming from anticonvulsants (66.6 percent), antipsychotics (43.8 percent), and antidepressants (33.4 percent). Overall, 79 percent of the off-label prescriptions lacked strong scientific evidence for their use.

For 160 drugs commonly prescribed to U.S. adults and children, 21 percent were for off-label indications totaling 150 million prescriptions. In this case, 73 percent had little to no scientific backing and once again psychoactive drugs such as gabapentin had the highest level of off-label use.

Moreover, doctors do not seem to know what are and are not approved FDA use for many of the drugs that they prescribe. Now an article published in the June issue of Health Affairs by Ian Larkin and colleagues points to active promotion by sales representatives as one reason for the widespread off-label use of antipsychotics and antidepressants in children.

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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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FDA Should Pass The Salt…Standards


June 16th, 2014

Editor’s note: In addition to Rep. Rosa DeLauro, Sen. Tom Harkin also coauthored this post.

For years, Americans have been inundated with messages about the dangers posed by the food we eat whether from fat, carbohydrates, or sugar. But another additive lurks in virtually every package and bag on the supermarket shelves, as well as practically every restaurant meal, and it is having a dire impact on our health.

That additive is sodium, and Americans are consuming far too much, with profoundly negative consequences on our health and well-being as a country. It is time for the U.S. Food and Drug Administration (FDA) to take serious and meaningful action to address this trend and issue federal standards to reduce the amount of salt in our foods.

Current dietary guidelines recommend that most individuals between the ages of 2 and 50 consume no more than 2,300 milligrams of sodium a day, or approximately one teaspoon of salt. For the roughly two-thirds of Americans in an at-risk population – seniors, African-Americans, those with high blood pressure, and individuals susceptible to hypertension, diabetes, or chronic kidney disease – only 1,500 milligrams is recommended.

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Roll-Out Of New TB Drug Must Be Handled With Care


June 13th, 2014

Janssen Pharmaceuticals, a subsidiary of Johnson and Johnson, has announced that it will make its breakthrough new tuberculosis (TB) drug, Sirturo, available at a discount in 130 developing countries. As the first new antibiotic to be approved to treat TB in over 40 years, Sirturo will be an important new weapon in the aging arsenal of medicines used to treat this deadly disease.

Sirturo’s approval was a breakthrough for global health and TB treatment. In 2012, the airborne disease killed about 1.3 million people, making it second only to HIV/AIDS in the ranks of infectious killers. While the number of people dying from TB each year is slowly falling, drug-resistant strains are proliferating. According to the World Health Organization, ninety two countries have reported cases of extensively drug resistant TB since it was first reported in 2006.

Tuberculosis Treatment Programs

Now that Sirturo is ready for prime time – and poised for international distribution – it’s critical that the roll-out is meticulously managed by health care systems in each and every country that plans to administer it. TB drug resistance usually arises from poorly managed tuberculosis treatment programs. Treatment is long – about six months for basic treatment, and up to two years for drug-resistant strains – and only adds to the challenge of successful treatment completion. With improper or inadequate treatment, virulent new forms can evolve.

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Agenda Overload In Primary Care: A Call To Protect The Real Capital Of Caring


May 15th, 2014

“When you go in to see a patient, you need to be focused. You need to have your agenda clearly in mind.”

As a physician educator offered this advice to hundreds of clinicians at a recent primary care update conference, heads nodded in agreement. It seemed like reasonable counsel given the wide range of complex goals of today’s primary care. But my heart sank as the address continued. Underlying the call for focus was a perspective that patients often bring up issues during a visit that distract us from priorities.

Both the sentiment expressed and the audience’s acquiescence indicate how far we have moved away from our patients’ agendas. I don’t think any of us in that convention center chose our careers thinking we would someday experience patients’ concerns as distractions in our work day. This incident was a sign to me that we need to chart a course back to the type of care that listens to and responds to patients.

The conference encapsulated a tension I had been feeling in my own practice for years. I was a family physician in what I consider an excellent, mission-driven community health center. We had motivated staff, interdisciplinary team care, electronic medical records, and many organizational efforts for quality improvement. Clinically, I found myself moving from room to room, pressured by time, trying to keep up with a multitude of health maintenance or quality metric items, and often feeling unable to engage with “yet another” issue that the patient in front of me wanted to talk about. While it was my goal and the goal of our practice to give compassionate, holistic care to patients, too often it seemed that the time and mental/emotional reserve to listen to patients were crowded out.

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Beyond Access: High Quality Care For All


April 30th, 2014

In medical school, I had an epiphany about my role as a doctor during my obstetrics-gynecology rotation at the county safety-net hospital in Fresno, California. I loved the thrill and satisfaction of delivering babies, but after about 10 births, it hit me that virtually all the women for whom I had delivered babies were teenage girls, as young as 13. They and their children would face uphill battles. As a clinician, I was too often a cog in a machine, fixing immediate needs but not addressing underlying problems to prevent poor health outcomes.

The Affordable Care Act tears down a fundamental barrier preventing vulnerable populations from accessing care by reducing the number of uninsured and underinsured Americans. But expanded access to coverage will not, by itself, guarantee high quality care for all. It would be a serious mistake to assume that insurance will eliminate disparities in health outcomes. In Chicago, a person with diabetes living in an African American neighborhood is five times more likely to have his or her leg amputated than a person living in a white neighborhood. Insurance alone won’t close that gap. Indeed, many who needlessly lose limbs are already insured.

I have spent a great deal of my career caring for inner city patients and working with other professionals to research the health of the public. If we really want to end disparities in the health care system, here are some things we should be doing:

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Hobby Lobby, Conestoga Wood, The ACA, And The Corporate Person: A Historical Myth Bedevils The Law


April 18th, 2014

On March 25, 2014, the Supreme Court of the United States heard arguments in two cases—Sebelius v. Hobby Lobby Stores, Inc. and Conestoga Wood Specialties v. Sebelius—challenging the validity of the Affordable Care Act’s (“ACA”) mandate that employer-sponsored health plans cover all FDA-approved contraceptives (the “Contraceptive Mandate”). In each case, closely held plaintiff corporations contend that the Contraceptive Mandate illegally infringed upon the corporations’ freedom to exercise religion.

The cases attracted attention because the Supreme Court had agreed to hear yet another challenge to the validity of the ACA’s provisions, but it has been less noticed that both cases, and others like them, implicate a fundamental question that the Supreme Court has never decided; on what basis, if any, is a corporation a “person” entitled to assert the constitutional and statutory rights of natural persons. Without denying the significance of the challenge to the ACA’s Contraceptive Mandate, the Supreme Court’s failure to define a principled corporate person theory has had—and continues to have—important and pervasive implications for the American legal system beyond the present cases.

Typically, legal concepts creating and regulating societal rights and obligations, like the corporate personhood concept, come into being incrementally in an extended evolutionary process. That evolutionary process is characterized by a dialectic give and take in which the principles justifying—or precluding—application of the concept in a variety of different factual scenarios are gradually clarified, defined and developed through a series of judicial decisions. The problem confronting the Supreme Court as it considers the Hobby Lobby and Conestoga Wood cases is that the concept of corporate personhood did not develop gradually or in an evolutionary process in which the meaning of the concept was developed and defined. Instead, the concept of the corporate person was imposed on the law ipse dixit, that is, by judicial fiat and without definition, in a series of late nineteenth century Supreme Court cases.

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Development Assistance For Global Health: Is The Funding Revolution Over?


April 17th, 2014

In many ways, the last twenty years have been somewhat of a “revolution” in global health, as marked by rising attention, growing funding, and the creation of new, large scale initiatives to address global health challenges in low and middle income countries.  Indeed, the 1990s brought a steady increase in global concern about health, largely centered on the HIV epidemic and due to civil society organizing to draw attention to the growing crisis, leading to the creation of the Millennium Development Goals, and soon thereafter, the GAVI Alliance, the Global Fund to Fight AIDS, Tuberculosis and Malaria (Global Fund), and the U.S. President’s Emergency Plan for AIDS Relief, and other efforts.

A key driver of increased funding has been donors – governments and multilateral agencies, non-governmental organizations (NGOs), and foundations.  And tracking their funding has become one of the critical measures of the global health response.

A new analysis from Dieleman et al., published as a Health Affairs Web First on April 8, provides a needed contribution to the literature on donor funding for health, including an understanding not just of where donor funding is going but of the relationship between aid, burden, and income.

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Embarking On A New Journey With Health Affairs


March 31st, 2014

I am delighted to be taking on the role of editor-in-chief of Health Affairs. This is a dynamic time in all aspects of health and health care: insurance coverage expansions, delivery system changes, and growing attention to population health.  Building upon thirty-three years of peer-reviewed scholarship, Health Affairs will continue to serve as the nation’s primary resource for the health policy community.

My goals for Health Affairs coalesce around a single theme: broadening the reach of the journal.

Health Affairs is strong in the core health policy community, but our scholarship is relevant to myriad actors in the one-sixth of the United States economy represented by health care.  My goal is to broaden our engagement with the worlds of law, finance, design, and many others.

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Health Policy Leader Alan Weil To Become New Health Affairs Editor-in-Chief


March 31st, 2014

Health Affairs and its publisher Project HOPE are pleased to announce that Alan Weil will become the journal’s new editor-in-chief on June 2, 2014.

Weil, a highly respected expert in health policy and current member of Health Affairs’ editorial board, will lead the journal after serving as the executive director of the National Academy for State Health Policy (NASHP) since 2004. His work with state policymakers of both political parties put Weil at the forefront of health reform policy, implementation, innovation, and practice. Prior to his leadership of NASHP, he served in both the public and private sectors. He directed the Urban Institute’s “Assessing New Federalism” project; served as the executive director of the Colorado Department of Health Care Policy and Financing and a health policy advisor to Colorado’s then-governor, Roy Romer; and was the assistant general counsel in the Massachusetts Department of Medical Security.

“We’re delighted to welcome Alan to the Project HOPE family,” said John P. Howe III, M.D., President and CEO of Project HOPE. “He comes to Health Affairs with more than 24 years of experience in health policy development and a stellar record of leadership and innovation in this field. I’m confident he will lead the journal’s talented staff on a new and successful path forward. I am extremely grateful to John Iglehart, the Founding Editor of Health Affairs for his stewardship of the journal for more than 25 years, ensuring its coveted rank as the leading health policy journal of our time.”

“Alan Weil’s extensive background in health and health care policy will serve him well in his new role as Health Affairs’ editor-in-chief,” noted John Iglehart, who currently leads the journal. “With his position on the front lines of health system change, he is an experienced leader who has deep familiarity with and longstanding connections to the health policy, research, and health care leadership communities. In particular, in his role as NASHP’s executive director, Alan worked on complex issues of critical importance to leaders in state and federal government and the private sector. This background will serve Health Affairs well as it continues to grow in influence both in the US and globally.”

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A March Madness Health Wonk Review


March 27th, 2014

Welcome to the “March Madness” edition of the Health Wonk Review. The NCAA college basketball tournament seemed like a natural theme for a health care policy blog post: huge amounts of money floating around in ways that only sometimes correlate with performance, and head-to-head match-ups that can yield results no one expected (though in the tournament those unexpected results produce quicker and more certain changes than is often the case in health care).

We considered illustrating each blog post with pictures of a college basketball team from the author’s home state celebrating a championship, but we thought better of that after seeing this cautionary tale. So let’s get to the great collection of posts from our Wonkers.

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The New Nutrition Facts Panel: Public Health Improvement Or Distraction?


March 19th, 2014

Last month, the United States Food and Drug Administration announced long awaited proposed changes to the Nutrition Facts Panel (NFP), the nutrition information found on the back of packaged foods and beverages. The NFP is required to be on all packaged foods, with significant regulations on what is presented and how the information can be presented. Initially mandated in the first Bush Administration, the NFP offers a clear and consistent manner of presenting nutrition information—at least for those with the time and nutrition knowledge to benefit from the information.

The key questions behind the proposed changes are: will they be successful in altering consumer behavior, how might they be improved, and what overall role might they play in obesity prevention?

The NFP is clearly a source from which those already motivated and knowledgeable can easily access information and a base on which to build future approaches to addressing obesity. Put differently, this information will only work if people actively, directly choose to turn the package over, engage in information, and push past the many impulses pulling them towards the less healthy foods. The compelling nature of unhealthy foods means that individuals have to be particularly motivated, or the nutrition information has to be particularly compelling.

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