Expedited FDA Approval for Breakthrough Therapies: Promises and Pitfalls

By Michael Young

In 2012, the FDA introduced a novel pathway for expedited review of breakthrough therapies. The ‘breakthrough-drug’ designation was carved out by the Food and Drug Administration Safety and Innovation Act (FDASIA), which provided for expedited development and review of any drug “intended, alone or in combination with 1 or more other drugs, to treat a serious or life-threatening disease or condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”  This pathway added to a variety of other accelerated FDA approval programs aimed at improving access to innovative, safer, or more effective therapies for serious medical conditions.

In a recent New England Journal of Medicine article, Jonathan Darrow, Jerry Avorn and Aaron Kesselheim examine the clinical consequences and ethical dimensions of the breakthrough-drug category, and raise a variety of vital questions about its implications for patients.

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International Aid, Public Health, and Corruption

My wonderful HLS colleague Matthew Stephenson has just launched the Global Anticorruption Blog (GAB). As it happens, his first two posts may be of interest to BOH readers, especially those  may be of interest to readers interested in international aid for public health projects, of the sort supported by the Gates Foundation.  The first post argues that the extent of corruption in these projects is much larger than the Gates Foundation and others acknowledge.  The second post contends that one reason for lowballing of corruption estimates is political: these projects depend substantially on public funding, and political support for health aid may be undercut by candid assessments of the extent of the corruption and fraud problems. Both the posts and the blog are well worth a read.

Kevin Outterson on the Search for New Antibiotics

Kevin Outterson appeared on NPR’s “Here & Now” to discuss the growing problem of antibiotic resistance and possible ways to incentivize development of new antibiotics. From the interview:

On the misuse of antibiotics

“We should think of this as a global resource that needs to be conserved and taken care of. So antibiotics should never be used inappropriately. In the country right now, we have something on the order of 23 million people who are getting antibiotics for ear aches. Most of those situations would resolve on their own in a couple of days. We also give antibiotics many times for people just because they have some sort of a common cold — it’s estimated 18 million prescriptions a year — doesn’t help anyone who has the common cold. It’s a complete waste.”

On the rise of antibiotic-resistant bacteria

“It’s frightened people for more than a decade. You mentioned at the top the 23,000 Americans who are dying from resistant infections. The CDC said on top of that, there’s another 14,000 dying from a horrible disease, intestinal disease, called Clostridium difficile [C-diff] in the United States. Together, that’s larger than the number of people who die in this country each year from AIDS. And we’re not — as bad as things are now, the more troubling aspects, or what might happen in five or 10 years if some sort of a pathogen was resistant to everything we had got out to the population. It sounds like a Hollywood movie.”

You can listen to the full interview here.

Prioritizing Parks and Patients

By Nathaniel Counts

During the government shutdown in October 2013, a battle in part over the future of healthcare reform, a non-negligible amount of media attention focused on the shutdown of public parks.  Perhaps because the parks were the least expected casualty of the shutdown, or the most ludicrous – many are, after all, large outdoor spaces that functioned for millions of years before there were federal funds for them – Americans were frustrated or amused that they could not walk around outside some places because politicians in D.C. could not agree on a budget.

The healthcare reform debate pitted those who believed that everyone should have health insurance or that access to healthcare was a right against those who believed that health spending was already too high or that everyone does not have a right to access to healthcare.  In a world of infinite resources, where everyone could have complete access to healthcare without anyone having to give up anything of their own, it is difficult to imagine that anyone would say that there should not be universal access to healthcare, that some are not deserving of the service.  It would be strange to require a threshold public showing of effort to obtain health insurance through employment if there was no cost to giving the healthcare – if fairness is an issue, as it appears to be a concern for some, there are certainly other services that could be denied.  It is likely that for most the fairness concern only becomes salient in the face of resource constraints where these same funds could fund other programs or allow others to pursue their interests.

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Capturing Value in Advanced Medical Imaging

On December 12, a bipartisan bill entitled the Excellence in Diagnostic Imaging Utilization Act of 2013 (HR 3705) was introduced in the House of Representatives which would require clinicians to use electronic clinical decision support tools (CDS) before ordering advanced diagnostic imaging tests for Medicare patients.  Structured around appropriate use criteria  developed by professional medical societies, the tools would aim to increase the value of advanced imaging studies by informing and guiding practitioners’ decisions across a variety of clinical settings.

Such tools would provide active feedback on the appropriateness and evidence base of various imaging modalities, and would require physicians to furnish rationales for ordering tests that are inconsistent with appropriate use criteria.  The bill also envisions the creation of registries that document how diagnostic tests are used in order to facilitate research and to enable feedback to clinicians on metrics related to appropriate use criteria.  In a press release, the American College of Radiology lauded the proposed legislation, stating that it would “revolutionize the specialty of radiology.”

Mandating the use of electronic clinical decision support tools portends at least three key improvements in clinical workflows and healthcare quality more broadly.

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Only a Right to Health

By Nathaniel Counts

Human rights disaggregates otherwise related issues into separate rights.  We discuss rights to health, education, housing, association, etc., and, in countries where these rights are codified, we litigate each one separately in the courts.  We also know that each of these issues for which there is a corresponding right is, to some extent, a symptom of poverty.  In some cases it might not be possible to treat the symptoms without addressing the root cause.  For example, in 1966, the Coleman study on equality in education found that “[s]chools bring little influence to bear on a child’s achievement that is independent of his [or her] background and general social context.”  These findings have been contested, but it is likely that socioeconomic factors are a determinant of a child’s academic success, along with the educational experience itself.  If the socioeconomic background is the greater determinant, it may not make sense to use scarce government resources to fund school improvement rather than addressing poverty itself.  In a country with a right to education, school improvement could be litigated and potentially derail national efforts to address root causes.

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Medical Advice and the Limits of Therapeutic Influence

By Michael Young

It is estimated that 500,000 patients are discharged from U.S. hospitals against the recommendations of medical staff each year.  This category of discharges, dubbed discharges against medical advice (DAMA), encompasses cases in which patients request to be discharged in spite of countervailing medical counsel to remain hospitalized.  Despite safeguards that exist to ensure that patients are adequately informed and competent to make such decisions, these cases can be ethically challenging for practitioners who may struggle to balance their commitments to patient-centered care with their impulse to accomplish what is in their view best for a patient’s health.

Writing in the most recent issue of JAMA, Alfandre et al. contend that “the term ['discharge against medical advice'] is an anachronism that has outlived its usefulness in an era of patient-centered care.”  They argue that the concept and category of DAMA “sends the undesirable message that physicians discount patients’ values in clinical decision making.  Accepting an informed patient’s values and preferences, even when they do not appear to coincide with commonly accepted notions of good decisions about health, is always part of patient-centered care.”  The driving assumption here seems to be that if physicians genuinely include patients’ interests and values in their assessments, then the possibility of “discharge against medical advice” is ruled out ab initio, since any medical advice issued would necessarily encapsulate and reflect patients’ preferences.  They therefore propose that ”[f]or a profession accountable to the public and committed to patient-centered care, continued use of the discharged against medical advice designation is clinically and ethically problematic.”

While abandoning DAMA procedures may well augment patients’ sense of acceptance among medical providers and reduce deleterious effects on therapeutic relationships that may stem from having to sign DAMA forms, it leaves relatively unaddressed the broader question of how to mitigate health risks patients may experience following medically premature or unplanned discharge.  Alfandre and Schumann’s robust interpretation of patient-centeredness also raises the question of how to handle situations in which patients refuse medically appropriate discharge.  On this interpretation, can the ideal of patient-centered care be squared with concerns for optimizing the equity and efficiency of resource allocations more broadly?

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PAPER NOW AVAILABLE for 11/18 Health Law Workshop w/Aaron Kesselheim

Aaron Kesselheim is a physician and an Assistant Professor of Medicine at the Harvard Medical School. He will be speaking at the Petrie-Flom Health Law Workshop on Monday, November 18, at 5:00pm, on “Do March-In Rights Protect Public Interests in Medical Products Arising from Federally-Funded Research?”

For more details on the workshop, including information on how to obtain a copy of the paper, please visit the Petrie-Flom Center’s website.

HHIP: Evaluating Effective Charities – GiveWell cofounder Elie Hassenfeld: Tuesday, 8 PM

Harvard High Impact Philanthropy presents:

Evaluating Effective Charities with Elie Hassenfeld of GiveWell

How can you maximize the impact of your charitable giving?  What distinguishes the most effective causes and organizations? Elie Hassenfeld, co-founder and co-Executive Director of GiveWell, will describe how his organization is revolutionizing charity evaluation with completely transparent, rigorous analysis. Q&A to follow.

8 pm, Tuesday, Nov. 12; Sever 102

RSVP here

Elie Hassenfeld graduated from Columbia in 2004 and co-founded GiveWell in mid-2007 where he currently serves as co-Executive Director. GiveWell finds outstanding charity and publishes the full details of its analysis to help donors decide where to give. The Boston Globe has called GiveWell “The gold standard for giving” and its research has attracted attention from Peter Singer and other media. GiveWell has tracked over $10 million in donations to its recommendations as a direct result of its research.

11/21: Neil Flanzraich on “Responsibility and Integrity in the Pharmaceutical Industry”

Please join the Petrie-Flom Center for a lecture by Neil Flanzraich on responsible pricing strategy, access to care, clinical trial design, outsourcing, and other topics that raise thorny but crucial issues for pharmaceutical and biotechnology companies.  There will be substantial time for Q&A.

Mr. Flanzraich graduated from HLS in 1968, and was appointed by Dean Martha Minow as an Expert in Residence at the Harvard Innovation Lab (i-lab) in fall 2012. He is the Executive Chairman of Kirax Corporation and the Executive Chairman of ParinGenix, Inc., both of which are privately owned biotech companies. He previously served as the Vice Chairman and President of Ivax Corporation, an international pharmaceutical company, which was sold to Teva in 2006 for an enterprise value of $10 billion.

For more information, please visit our website.

EMTALA and Patient Hoarding

By Jeremy Kreisberg

It is clear that EMTALA aims to prevent hospitals from dumping patients that require emergency care until these patients are stabilized.  But whether EMTALA also prevents hospitals from hoarding emergency patients to their detriment is far less clear.  Recently, the Tenth Circuit confronted this question in Genova v. Banner Health, a case concerning a physician who claims that he was fired for complaining about overcrowding at his emergency room.  The physician sought protection under EMTALA’s whistleblower provisions, which prevent hospitals from taking an “adverse action” against an employee simply because that employee “report[ed] a violation of a requirement of [EMTALA].”  Thus, the question for the Tenth Circuit was whether a hospital violates EMTALA if it has an overcrowded emergency room and refuses to offer a transfer to its waiting patients.  Writing for a unanimous panel, Judge Gorsuch held that there was no such violation.

As a matter of statutory purpose, the Tenth Circuit had a persuasive case.  After all, EMTALA is clearly intended to prevent hospitals from dumping its emergency patients onto other hospitals to avoid the costs of providing uncompensated care.  So when Dr. Genova complained about “patient hoarding” rather than “patient dumping,” the court cleverly stated that “[h]is complaint wasn’t about an EMTALA violation but more nearly its inverse.”  Indeed, there are serious potential issues with finding that EMTALA requires any overcrowded hospital to transfer its patients.  Such a rule might allow hospitals, which are often overcrowded, to use resource constraints as an excuse for transferring patients that require expensive care.

But the Tenth Circuit rested much of its case on the statutory text.  And as a matter of statutory interpretation, this issue is far from clear.  EMTALA states that a hospital which receives a patient “must provide for an appropriate medical screening examination within the capability of the hospital’s emergency department.”  If a patient has an “emergency medical condition,” the hospital must provide either (A) “within the staff and facilities available at the hospital, for such further medical examination and such treatment as may be required to stabilize the medical condition,” or (B) “for transfer of the individual to another medical facility in accordance with subsection (c) of [EMTALA].”

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Racism in Transplant Denial? Or Too Few Hearts To Go Around?

Anthony Stokes, a fifteen year old kid from Decatur County, Georgia, is expected to die in a matter of months, according to his doctors at the Children’s Healthcare of Atlanta.  Maybe, they say, he will live for six months.  Who knows?  Anthony suffers from an enlarged, increasingly less functional heart.  His condition is not unusual, and a reasonably effective cure is at hand: a heart transplant. (Learn more about Anthony’s story here.)

However, Anthony has become the latest victim of a dysfunctional U.S. transplantation system, which tempts Americans with a transplant waiting list, but kicks them off if they become too sick or too old.  The problem is that there are too few organs to meet demand, and this perennial problem receives far too little attention from Congress.  Indeed, the U.S. transplantation list, coordinated by the United Network for Organ Sharing, UNOS, (a private organization that coordinates significant aspects of the U.S. transplant system) is so overcrowded that patients increasingly turn to black markets in India, China, Pakistan, South Africa, and other countries if they hope to survive.  (Learn more about that here.) Congressional hearings document Chinese prisoners dying and shortly thereafter Americans receiving organs.

Anthony’s family and some local organizations claim that racism is behind doctors refusing to place the boy on the transplant list.  Anthony is African American.  They ask, what is the harm in letting him on the list?

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Art Caplan on teen organ transplants

Art Caplan has a new opinion piece up at nbcnews.com. In “Ethicist: Teens have high failure rates after organ transplants — but cut them some slack,” Caplan responds to a recent study showing that teen organ recipients have much higher failture rates than recipients in other age groups:

Remember the long fight over whether Sarah Murnaghan, the little 10-year-old girl from suburban Philadelphia who was dying from cystic fibrosis, should have a shot at getting a transplant from lungs taken from an adult? The fight hinged in part on whether there was sufficient evidence to show that adult lungs would work as well in Sarah, who is still struggling to recover from two lung transplants, as they would in another adult where they would fit better. Some, including me, argued that the best way to allocate scarce lungs for Sarah or anyone else is to determine who is most likely to live if they get them.

That may seem a sensible ethical policy to use when there are not enough organs for all. But there is a new study out that calls into question the merits of an efficacy-only rationing policy.

Read the full article here.

ART, lesbians and justice in the distribution of health care

In the wake of our seemingly everlasting economic crisis, the Spanish health authorities have decided to exclude single women to access ART treatments – mainly artificial insemination- in the public health care system. “The lack of a male partner is not a medical problem”, has said Ana Mato, our Secretary of Health. Coming from a devout Catholic and extremely conservative politician, her remark, and ultimately, her Department’s policy, have been widely interpreted as another vindication of the idea that only traditional, i. e. heterosexual, families are suitable for rearing children. The spokeswomen of various feminist and lesbian NGOs have entered the public arena to denounce her lesbophobia.

The fact of the matter is that women in Spain, whether married to another woman or single, will still be authorized to be artificially inseminated (in some European countries such as France, Austria or Sweden, for instance, single women are excluded from medically assisted reproduction). Even the fertile, married heterosexual woman might still get artificial insemination – maybe she just wants to do things differently, for a change- although they will all have to bear the costs. The public health care system has, therefore, reconfigured ART as a pure medical remedy for a medical condition: infertility. The days of IA as an “alternative means of reproduction” for “alternative life-styles” are over. But with this new policy the demand made by economically disadvantaged lesbian couples willing to procreate finds an answer along the following lines: “go find a male”. A crude response if there is one. Continue reading

ACA Final Rule on Wellness Plans

On May 29th, HHS issued the final rule governing wellness incentives in group health plans. While the incentives themselves are not a surprise, the scope they are given is worthy of ongoing attention. Wellness incentives have been controversial because of their potential for intrusion into individual choice, their subtle (or not so subtle) coerciveness, their valorization of a particularly model of health, and the possibility that they will impose differential burdens and costs on people with disabilities or other disfavored groups. The final rule attempts to meet these objections in several helpful ways.

Nonetheless, the final rule still will allow programs that are differentially burdensome as a result of factors other than health status. It will also allow programs under which it is more difficult for some than for others to obtain rewards because of their states of health. In programs that give rewards for health outcomes, alternatives must be available for those who do not meet targets—but the reasonableness standard for these alternatives permits requirements that may be differentially burdensome so if they are medically appropriate and follow the recommendations of the patient’s personal physician. HHS supports wellness programs as engaging individuals in their health, as encouraging them in healthy behaviors and discouraging them in unhealthy behaviors, and as incentivizing people to make use of recommended health care services such as screenings. Continue reading

Politics and Science

By Joanna Sax

On April 29, Scott Burris blogged about a new bill that would allow Congress to set the scientific agenda, which would replace the traditional peer review process.  I echo his expressed concerns, but want to add more.  The idea that politicians, and not scientists, could determine the advancement of science is, frankly, a disaster.  In the past we have seen political leaders spar with scientists over many things – such as whether the earth is round, whether the earth is the center of the universe, etc.  If scientists did not or could not answer those questions, we might think we are walking on a flat earth.

Even now, there is a strong interaction between politics and science.  Evolution, a scientific theory with unequivocal consensus among the scientific community, still faces political opposition.  Recently, I’ve been thinking and writing in this area, that is, the interaction of politics and science.  Questions for scientific inquiry should be determined by scientists.  How we allocate and manage our resources requires, as others have argued, experts in many areas, including economics, management, and public policy.

To look at the interaction of science and politics, I conducted an empirical analysis comparing the type of information communicated to the public versus the consensus in the scientific community to determine whether politics is playing a role in scientific inquiry.  The study centers on the debate regarding the funding of embryonic stem cell research.  If you are interested in this area, please check out a recent draft here.  It’s an early draft and I welcome comments – you can email comments to me at jsax@cwsl.edu

Sex, People with Disabilities, Prostitution, and Universal Health Care: Reflections on “The Sessions”

[Cross-Posted at PrawfsBlawg]

One of my favorite initiatives at Harvard Law School, where I teach, is that faculty members get to offer an optional 10-12 student not-for-credit “First-Year Reading Groups” on a topic of interest to them that is related to law in some way but not too law-class like. I’ve taught a reading group on bioethics and law through film that pairs films with papers/topics in bioethics (e.g., A.I. with readings on personhood, Minority Report and neuroscience and law and predicting criminality, Dirty Pretty Things and organ sale and exploitation, The Constant Gardener with clinical trials in the developing world, Eternal Sunshine for the Spotless Mind and therapeutic forgetting and “cosmetic neurology” and many others…)

Next year I will add The Sessions, a film I found very enjoyable starring John Hawkes, Helen Hunt, and William H. Macy from last year that I also found very bioethically interesting. The film is based on a true story and follows Mark O’Brien, a poet who lives in an Iron Lung due to complications from Polio. After unsuccessfully proposing to his caretaker, and believing the end of his life may be nearing, he decides he wants to lose his virginity. He hires Cheryl Cohen-Greene, a professional sex surrogate, who will offer him a maximum of six sessions but makes clear to him this is therapy not romance. I will stop there to avoid ruining the film, but on to the bioethics…

There are fairly clear issues raised about commodification, exploitation, the difference between sex therapy and prostitution, that I have written about in various forms in various places. These are certainly interesting issues but familiar enough. What the film newly prompted me to think about, though, is actually universal health care. In particular, as I have written about indirectly in a couple of papers, what would some of the most prominent theories explaining why we need universal health care say about whether the state should pay for sex therapy (or perhaps even prostitution) for people with disabilities like Mark who find themselves otherwise unable to have sex?

For example, in his wonderful book Just Health, my colleague Norman Daniels, coming from a more Rawlsian tradition (i.e., a liberal tradition focused on promoting liberty and distributive justice through giving priority to the worst-off), grounds the state’s role in promoting health in the obligation, as a matter of political justice, to ensure access to the “normal opportunity range” to pursue the “array of life plans reasonable persons are likely to develop for themselves.” Although Daniels’ focus is on health care, it seems to me that sexual satisfaction is also part of that normal opportunity range and part of a life plan most of us would like to pursue.

Similarly, Martha Nussbaum in her great book Frontiers of Justice, writing from a more aretaic (i.e., Aristotelian, focusing on character and virtue) perspective, has argued that the state’s role is to enable human flourishing by raising people above the threshold level on a number of “capabilities.” Among these she mentions “bodily integrity,” as including “having opportunities for sexual satisfaction and for choice in matters of reproduction.” I have previously discussed how this kind of approach may justify funding reproductive technologies, but it seems to me as though it also fairly directly establishes an argument for funding Mark’s attempts to lose his virginity.

Now this is meant to be provocative, of course. And for some this is no doubt a reductio ad absurdum against universal health care. Fair enough. But for those who believe there is a moral case for funding universal health care, does the argument also lead to funding these kinds of sex therapies? Health is important, of course, but let’s be frank (and my parents can stop reading at this point) so is sexual satisfaction, and both seem to me essential parts of the normal opportunity range and/or human flourishing.

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Pool of Siloam in Tennessee? Brute luck in health care distribution

I had recently come across this piece in the NYT. The first thing that came to my mind is the biblical story about the pool of Siloam, a tale that, with all due respect to believers, illustrates the unfair distribution of health, but mostly the unfair distribution of health care (if we take as “health care” the healing powers of the pool). I have tried to adapt the story, as told in the Bible (John 5, 1-8) to the crude reality of the Tennessee of our days, in which, as reported by the NYT, many of its citizens lack access to basic health care services and have to trust in a sort of “phony miracle”. My paraphrasing experiment gave the following result:

“Now there is in Tennessee a program ran by the State Government, called TennCare, which has a hotline for applications. In these lay a multitude of invalids–blind, lame, and paralyzed [waiting for the opening of the line;] [for the Governor went down at certain seasons and opens the line: whoever gets in first after the opening of the line was helped for whatever disease he had.] One man was there who had been an invalid for thirty-eight years. When people from the Legal Aid Society in Nashville saw him lying there and knew that he had already been there a long time they told him, “Do you want to be helped?” The sick man answered him, “Sir, I have no one to put me into the pool when the line is open, and while I am trying to go through another connects before me.” The people from Legal Aid said to him, “Get up, take up your bed, and dial.” And at once the man was healed, and he took up his bed and walked…”

[The original King James version’ of the Gospel can be read here]

New Paper on Coercion and the Constitutionality of the Affordable Care Act

I have a new paper on the Supreme Court’s decision on the Affordable Care Act, just published in the European peer-reviewed philosophy journal Ethical Perspectives. It is available for free download here.  Here is the abstract:

While NFIB v. Sebelius largely upheld the Affordable Care Act (ACA), it did not do so as as to the proposed expansion of Medicaid. Seven of the nine U,S, Supreme Court Justices (all except Justices Ginsburg and Sotomayor) endorsed a ‘coercion’ argument that gave individual States a right of objection grounded in the Constitution’s Spending Clause, wherein individual states could refuse to expand Medicaid as demanded by the federal government without being directly penalized by a denial of federal funding. Two Justices in dissent focused on the lack of judicial administrability of such a standard, and suggested it would open up a Pandora’s box of future constitutional challenges without any clear rules.

In this article, part of a symposium on philosophical analysis of the Court’s decision published in the peer-reviewed journal Ethical Perspectives, I discuss what I see as a more fundamental question: by what theory is the Medicaid expansion coercive, and even if coercive, by what theory is it coercive in a problematic way that justifies constitutional redress?

The Court’s failure to address this issue stems, in part, from confusion over what it means for an offer to be coercive. In some sense, Justice Kagan seemed to recognize this issue in a question to Paul Clement, the lawyer for the challengers to the ACA, at oral argument: “Why is a big gift from the federal government a matter of coercion?” Kagan asked. “It’s just a boatload of federal money for you to take and spend on poor people’s health care,” Kagan added. “It doesn’t sound coercive to me, I have to tell you.” The exchange is all the more curious because, despite her scepticism, Kagan signed on to the Court’s holding that the Medicaid expansion was coercive.

I will examine this issue by first discussing whether Medicaid itself and the ACA’s expansion are coercive (as stand-alone offers). I will then examine whether the offer to change from the existing Medicaid program to the ACA’s Medicaid expansion was problematic. I will analyze these questions under the assumption that the Court is not committing a category error by treating States as the kinds of entities subject to this kind of coercion inquiry. In my conclusion, however, I briefly consider whether that assumption is warranted.

Reminder, Upcoming Conference on Universal Health Coverage in Low-Income Countries

A reminder that the Harvard University Program in Ethics and Health annual conference, Universal Health Coverage in Low-Income Countries: Ethical Issues, will be held in Boston on April 18-19, 2013.

AGENDA

http://peh.harvard.edu/events/2013/universal_coverage/agenda.html

REGISTRATION

No fee. Space limited. Registration required. Please register on our registration website.