Please join us for oursecond annual Health Law Year in P/Review event, co-sponsored by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School and the New England Journal of Medicine. The conference will be held in Wasserstein Hall, Milstein East C at Harvard Law School on Friday, January 31, 2014, from 8:30am to 5:00pm.
This year we will welcome experts discussing major developments over the past year and what to watch out for in areas including the Affordable Care Act, medical malpractice, FDA regulatory policy, abortion, contraception, intellectual property in the life sciences industry, public health policy, and human subjects research.
The full agenda is available on our website. Speakers are: Read More
Please join us for oursecond annual Health Law Year in P/Review event, co-sponsored by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School and the New England Journal of Medicine. The conference will be held in Wasserstein Hall, Milstein East C at Harvard Law School on Friday, January 31, 2014, from 8:30am to 5:00pm.
This year we will welcome experts discussing major developments over the past year and what to watch out for in areas including the Affordable Care Act, medical malpractice, FDA regulatory policy, abortion, contraception, intellectual property in the life sciences industry, public health policy, and human subjects research.
The full agenda is available on our website. Speakers are: Read More
Bonjour de Toulouse, where I’m visiting this month at the Institute for Advanced Study (IAST), which is hosted by the Université de Toulouse Capitole and physically (and in many senses conceptually) situated inside the Toulouse School of Economics. That setting is a particularly good one for this post on markets in human tissue.
In early October, I wrote about a rule that the Department of Health and Human Services has proposed that would amend the National Organ Transplant Act (NOTA) to criminalize compensation for providing peripheral blood stem cells (PBSCs) given through apheresis. Compensating those who allow their bone marrow to be aspirated — the other major source of hematopoietic stem cells (HSCs) — is already explicitly criminalized under the statute. At the end of the post, I promised to follow up with a second post on why I’m skeptical about this rule, as a matter of policy. This is that post.
But before discussing appropriate policy for addressing the problem of the 3,000 Americans who die each year for want of a bone marrow transplant, and for reasons I’ll explain near the end of my post, I want to say something about one particular person: Professor Nalini Ambady, a Stanford social psychologist who, about one year ago, began an international search for a compatible, willing HSC donor to treat her leukemia.
Because she is South Asian, and because few Asians and other racial and ethnic minorities are listed in the U.S. bone marrow registry — South Asians comprise only 2% of the U.S. registry — she had a difficult time finding a match. She nevertheless found 12 potential matches in the U.S. bone marrow registry, and apparently at one point had scheduled the transplant. In the end, however, half were ruled out as imperfect matches — and each of the remaining six matches declined to donate.
Having exhausted the U.S. registry, Ambady and her supporters sought a match in South Asia registries — but even fewer people are registered in that region than in the U.S. Of India’s 1.2 billion people, for example, only about 45,000 are registered. Nevertheless, a potential thirteenth match was found in an Indian registry. He matched at least 6 of 10 HLA markers; he would need a blood test to determine whether he was a complete match. Unfortunately, after a worrisome week of silence about whether he was indeed a complete match, Ambady and her family learned that the potential donor had backed out.
Weeks later, it happened with another potential match for Nalini, this time an 8/10 and so an even likelier match. This man wanted to watch someone else donate their bone marrow before making up his mind. A registry chief actually arranged this for him in early May. Somehow, it wasn’t persuasive enough. He, too, decided not to donate.
Professor Ambady died on October 28 after eight compatible or potentially compatible registered donors ultimately declined to donate.
Apparently, declining to donate after agreeing to be listed in a registry and being found to be a match is quite common. According to one registry chief, the attrition rate is 50% in the U.S., Europe, and India. Would a financial incentive have tipped any of these vaccillating prospective donors towards a decision to donate? Although we can’t say, this is an eminently answerable question. Unfortunately, the Department seems uninterested in learning whether a compensation scheme would save lives — or have ill or counterproductive effects — or not.
In its Notice, the Department lists the following policy reasons for expanding NOTA’s list of “organs,” the exchange of which for valuable consideration is prohibited, to include PBSCs: (1) preventing commodification; (2) curbing opportunities for coercion of PBSC providers and (3) exploitation of PBSC recipients; (4) encouraging altruistic donations, and (5) decreasing the likelihood of disease transmission resulting from paid donations. My responses to these arguments are after the jump. I end with some thoughts about alternative policy tools for increasing the supply of PBSCs in the absence of a legal compensation scheme.
Stem cells hold tremendous potential to advance health and medicine. Through replacement of damaged cells and organs or supporting intrinsic repair, stem cell offer promising treatments for debilitating diseases and conditions such as Parkinson’s disease, diabetes, and spinal cord injury. Currently, however, the evidence base to support the medical use of stem cells is still limited, with few clinical applications shown to be safe and effective. Despite the preliminary nature of clinical evidence, consumer demand for treatments using stem cells has risen, fueled by direct-to-consumer advertising of stem cell-based treatments. Clinics have been established throughout the world, both in newly industrialized nations such as China, India, and Mexico, as well as developed countries such as the United States and within Europe, that offer “stem cell therapies” for a wide range of diseases and conditions. Often provided at great expense and often promoted as established and effective, these treatments have generally not received stringent regulatory oversight, have not been tested through rigorous trials to determine safety and likely benefit, and the claims remain largely unsubstantiated by medical science. Complications from treatments have ranged from tumor formation to the death of patients. Some feel that the false claims and potential for harm to patients could significantly damage the real potential of research to produce valid stem cell therapies.
The Institute of Medicine and the National Academy of Sciences will co-host a workshop with the International Society for Stem Cell Research that will take a critical look at the practice of unproven stem cell treatments. Speakers will examine the evidence base of unsubstantiated treatment offerings and the associated research and clinical risks and concerns. Discussions will delve into legal hurdles for establishing standards and criteria to govern stem cell trials and treatments and explore a range of potential solutions for assuring the quality of unregulated therapies.
By Melissa Lopes, Fellow, Division of Medical Ethics, Harvard Medical School and Director of Research Policy and Compliance, Office of the Vice Provost for Research, Harvard University
The DISCUSS project is designed to develop consensus recommendations for the use of stored biospecimens (originally consented for research) to:
derive iPSC lines and
deposit the derived lines in a cell bank or repository for widespread distribution.
The project represents a formal collaboration of NIH, CIRM and the International Stem Cell Forum. The broadly stated aim of this collaboration is to evaluate consent protocols and ethical requirements in support of ongoing collaborations and the exchange of research materials.
The Petrie-Flom Center and Harvard Law School are delighted to announce our partnership with Duke University, Stanford University, and Oxford University Press to launch a new peer-reviewed, open access, online journal in 2014: Journal of Law and the Biosciences (JLB).
JLB will become the preeminent outlet to publish cutting-edge scholarship wherever law and the biosciences intersect. The journal will take a broad and interdisciplinary view of these areas, publishing articles on topics generally considered part of bioethics or neuroethics, such as the ethical, legal, and social implications of reproductive technologies, genetics, stem cell research, neuroscience, or human biological enhancement. At the same time, JLB will be a home for work that speaks directly to legal issues where the biosciences can be involved, such as food and drug regulation, biosciences patent law, scientific evidence, and criminal responsibility.
On October 2, in the midst of the government shutdown—either HHS somehow managed to convince itself that the rule was “necessary for the protection of life” or, more likely, it had already been scheduled for printing—HHS quietly published a Notice of Proposed Rulemaking. The proposed rule would effectively moot the recent Ninth Circuit case of Flynn v. Holderby criminalizing the compensation of bone marrow donors, even when the life-saving stem cells are extracted through a newer, minimal risk procedure. In this post, I’ll explain the medicine, the Ninth Circuit’s decision, and what HHS proposes to do in response. In my next post, I’ll have some reaction to HHS’s policy arguments, about which I’m skeptical, and perhaps a few thoughts about where plaintiffs might go from here.
Section 301 of the National Organ Transplant Act (NOTA) of 1984 criminalizes the transfer of “human organs” for “valuable consideration.” Reimbursement of reasonable out-of-pocket expenses associated with travel and lost wages are okay—as are, since the 2008 amendment of NOTA, paired living donor chains—but any other “valuable consideration” that might incentivize sources of organs is not. Under NOTA, as amended,
‘human organ’ means the human (including fetal) kidney, liver, heart, lung, pancreas, bone marrow, cornea, eye, bone, and skin or any subpart thereof and any other human organ (or any subpart thereof, including that derived from a fetus) specified by the Secretary of Health and Human Services by regulation
(emphases added). HHS has since added to this list “intestine, including the esophagus, stomach, small and/or large intestine, or any portion of the gastrointestinal tract.”
So-called “bone marrow transplants”—in reality, infusions of hematopoietic (blood) stem cells (HSCs)—are often life-saving procedures for those with, for example, leukemia or aplastic anemia. (See these sobering statistics compiled by the Institute for Justice, which represented the plaintiffs in Flynn.) When NOTA was passed, the only way to obtain HSCs was through bone marrow aspiration using a long needle thick enough to suck liquid marrow directly from the donor’s pelvic bone. HSCs are then harvested from the marrow. The procedure is done under general anesthesia and so the donor is subject to the usual risks of anesthesia. Although the donor can return to usual activities in two to seven days, discomfort may linger for up to two weeks. Read More
A sound scheme of public regulation seeks to find the best way to align the incentives of public regulators with the interests of the public at large. That task has proven to be particularly acute with the U.S. Food and Drug Administration, which possesses vast powers to regulate the manufacture and distribution of drugs and medical devices in the United States, which it all too often overuses.
That case examined the line between the regulation of pharmaceutical products on the one hand and the practice of medicine on the other. It is well established that the FDA has power over the former, but lacks power over the latter. That distinction is of no small consequence because it allows, for example, for the rise of an extensive market in “off-label use” by physicians of drugs for purposes for which they have not received FDA approval. This class of drug use is very large, and in cancer cases is thought to be more common than treatment using FDA-approved products.
The persistence of off-label drug use should caution people about allowing the FDA to exert extensive control over the drug licensing process. It could be that the thousands of physicians, hospitals, and patients that use drugs off-label have, time after time, all made some major mistake. Or it could just be possible that the accumulated level of institutional knowledge post-release demonstrates that the FDA has been too restrictive in its decisions as to whether a new drug should be allowed into the marketplace in the first instance. The FDA is heavily incentivized to be tough in its approval process lest it take heavy criticism for the all-too-visible injuries that may be caused by approved drugs. But in the social calculus, the individual losses that occur to people who are denied treatment by the heavy-handed actions of the FDA count every bit as much. Yet these diffuse losses, often undocumented, bring much less heat to the FDA, which accordingly is prepared to shrug them off as a cost of supplying people the “protection” that is so central to the FDA mandate.
Stem cells have been an endless source of fascination and controversy since Dolly the sheep was cloned in 1996. This month’s announcement of a cloned human embryo from a single skin cell [1] came on the heels of Sir John B. Gurdon and Dr. Shinya Yamanaka’s receipt of the 2012 Nobel for Physiology and Medicine for their work with induced pluripotent stem cells. Pluripotent stem cells can be embryonic or induced. Embryonic stem cells (ESCs) can generally be obtained from human embryos or by cloning embryos through somatic cell nuclear transfer (SCNT), as was done for Dolly. Gurdon and Yamanaka demonstrated that pluripotent cells may also be formed by reprogramming adult cells to an embryonic state, resulting in induced pluripotent stem (iPS) cells without having to use eggs or cloning, or destroy embryos. However derived, pluripotent cells are capable of differentiating into virtually any cell type in the human body. This imbues them with great promise for scientific breakthroughs and medical advances, but also raises serious ethical, legal and safety concerns about their use.
Less controversial are “multipotent” adult stem cells (ASCs) which do not involve embryos or raise as many safety concerns as pluripotent cells. ASCs are found throughout the body. Their ability to differentiate is more limited than pluripotent cells but is vast nonetheless. The NIH’s clinicaltrials.gov site lists some 4500 ASC trials as compared with 27 for embryonic stem cells and 21 for induced pluripotent stem cells. Recent announcements of new stem cell treatments usually involve ASCs, such as last month’s news that a toddler born without a trachea received a new one made from her own adult stem cells. It is therefore no surprise that ASCs have captured the attention of researchers, investors, physicians, patients and – increasingly – regulators, both here and abroad.
A growing number of physicians routinely offer treatments involving ASCs to their patients which can be performed in their offices. Autologous adult stem cells, used to treat a variety of conditions, are harvested from the patient, processed, and returned to the same patient. It is no surprise that moving ASCs from laboratories to physician offices raises complex questions of law. We consider one of the more pressing ones: to what extent can the FDA regulate a physician’s ability to treat a patient with that patient’s own stem cells? In the coming months, the D.C. Circuit Court of Appeals will hear oral arguments on this very issue in United States v. Regenerative Sciences.[2]
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
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