Blog Home

Archive for the 'Bioethics' Category




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.

Read the rest of this entry »

Ethical Dilemmas In Prison And Jail Health Care


March 10th, 2014

Editor’s note: This post is published in conjunction with the March issue of Health Affairs, which features a cluster of articles on jails and health.

Prison and jail health care, despite occasional pockets of inspiration, provided by programs affiliated with academic institutions, is an arena of endless ethical conflict in which health care providers must negotiate relentlessly with prison officials to provide necessary and decent care.  The “right to health care” articulated by the Supreme Court pre-ordained these ongoing tensions.  The court reasoned that to place persons in prison or jail, where they could not secure their own care, and then to fail to provide that care, could result in precisely the pain and suffering prohibited by the Eighth Amendment to the Constitution.

Good reasoning was followed by a deeply flawed articulation of the “right” that defines the medical care entitlement as care provided to inmates without “deliberate indifference to their serious medical needs.” By forging a standard which was, and remains, unique in medicine and health care delivery — designed to avoid intruding on state malpractice litigation regarding adequacy of practice and standards of care — the court guaranteed that dispute would surround delivery.  That first framing, which did not establish a right to “standard of care” or to care delivered according to a “community standard,” set the stage for endless ethical and legal conflict.

The Eighth Amendment’s deliberate indifference standard, forbidding cruel and unusual punishment, presents a relatively demanding standard for proving liabil­ity.  The Eighth Amendment, as interpreted by the federal courts, does not render prison officials or staff liable in federal cases for malpractice or accidents, nor does it resolve inter-professional disputes — or patient-professional disputes — about the best choice of treatment. It does require, however, that sufficient resources be made available to implement three basic rights: the right to access to care, the right to care that is ordered, and the right to a professional medical judgment.

Read the rest of this entry »

Workplace Wellness Programs: Continuing The Discussion With Ron Goetzel


October 30th, 2013

We imagine that reading a point-by-point reply to Professor Ron Goetzel’s lengthy rebuttal to our previous reply, which responded to a previous lengthy rebuttal by Professor Goetzel to our original article, would try the patience of even the most dedicated readers of Health Affairs. Therefore, beyond reminding readers of the original article’s scope, here we use Professor Goetzel’s last response as a touchstone to summarize our difficulties with the arguments by advocates of workplace wellness programs based on financial incentives. We ask weary readers who do not wish to continue with this exchange, but are interested in workplace wellness, to look at the evidence we provide in our appendices, and make their own judgments.

First, we must remind Professor Goetzel that we wrote our original article to address the assumptions underlying workplace wellness programs based on substantial financial incentives — those encouraged by the Affordable Care Act — and not any of the other types of programs. That article did not comment on the myriad other public health interventions that might take place at the worksite, although some of our arguments could be applied to those programs as well. Unfortunately, many of Professor Goetzel’s comments are directed at claims we never made about those other programs, and we find his continued advocacy for those programs in the guise of a response to our work distracting.

Second, Professor Goetzel’s most recent posting helpfully outlines ACA wellness regulations that were released after we published our article. Like other commentators, he is right to highlight the new, expansive “reasonable alternative standard,” because it may mean that many more employees can access alternatives to the program standards. Once again, however, he is more optimistic than we and other researchers are that such protections will be easily accessed by employees. We are skeptical that it will be nothing more than a simple matter for an employee to exempt themselves from the general program. Among other things, the employee needs to know about the possibility of an alternative and to feel that asking for an alternative will not harm her, and her employer must be willing to comply.

Read the rest of this entry »

Applying Comparative Effectiveness Research To Individuals: Problems And Approaches


October 29th, 2013

A Comparative Effectiveness Research (CER) study shows that surgery is better than medical treatment for a particular cardiac condition. My patient is 78 years old and has complicated diabetes. – does the study apply? Another patient 48 years old and otherwise healthy. Does it apply here?

Can the overall results of a CER study be applied to all patients in the target population? Are there substantial, undetected variations among patients in the results of CER? What is the extent of exceptions? These are important policy questions in applying results of CER to day-to-day decisions, clinical guidelines, performance measures and other facets of the modern healthcare system.

The “gold standard” approach to CER is the randomized (RCT), a scientific comparison of two or more clinical strategies, with the downsides that it is generally conducted in a special environment and usually has a rather narrow (and possibly unrepresentative) population spectrum. Two variants, the Practical (or Pragmatic) Clinical Trial (PCT) and the Large Simple Trial (LST) are inclusive of a wider spectrum of patients and more diverse clinical settings.

These approaches provide “average” results and for the most part it is thought that averages do apply to a large segment of the population at large for which they are intended. However, there are clearly differences in effect (heterogeneities of treatment effect – HTE’s) that manifest among CER study subjects and presumably to a greater extent in the intended population outside the study. Two approaches may be equivalent on the average but one may be better in a particular group, and differences may be less apparent when the study’s population base is narrow. A long list of factors contribute to these HTEs for CER and other trials – comorbidities, severity of illness, genetics, age, medication adherence, susceptibility to adverse events, ethnicity, site, economics and others.

Read the rest of this entry »

Inside the Gray Zone: Reflections on Gautham Suresh’s Narrative Matters Essay


October 21st, 2013

Note: This piece was written in collaboration with John. Lantos, director of the Bioethics Center at Children’s Mercy Hospital.

“I just caution you that we don’t know what we don’t know.”

These words came from one of my senior faculty members at Children’s Mercy Hospital in Kansas City, Mo. The context and tone of her warning made me clench my teeth. As a young neonatology fellow, I wanted to think that we did know.

But it turns out, the senior faculty member was right. Her words may have been the most important lesson I’ve learned during my neonatology fellowship. When it comes to the most premature of babies, there is far more uncertainty than certainty. I have learned to recognize this, and believe that it is the key to being a good neonatologist.

In the October Health Affairs Narrative Matters Essay, Dr. Gautham Suresh recounts the tension that he experienced when resuscitating an infant at the borderline of viability. The essay raises the issues of parental decision making, the high rate of premature birth in the United States, and the always touchy issue of resource allocation.

Read the rest of this entry »

Narrative Matters: A Doctor Faces Tough Decisions On Infant Resuscitation


October 16th, 2013

In the October Health Affairs Narrative Matters essay, a neonatologist must decide whether to revive a premature baby on the borderline of viability. Gautham Suresh’s article is freely available to all readers; or you can subscribe to iTunes and listen to him read it. Next week on Health Affairs Blog, another neonatologist will react to Dr. Suresh’s thoughts and describe her own experiences grappling with the difficult decisions that too often accompany the treatment of very premature babies.

Read the rest of this entry »

The Privacy Conundrum And Genomic Research: Re-Identification And Other Concerns


September 11th, 2013

No matter what the arena — finance, health care, or national security — questions surrounding the provision of personal data are always the same: how much benefit vs. how much risk? Who handles these data, and can those individuals be trusted? How do organizations guard against data misuse? What are the legal safeguards to protect privacy, and are they sufficient in an era when more data are shared more widely?

Nowhere is the privacy discussion more personal than in genomics, the very hardwiring of our existence. Genomic data are unique to individuals (or identical twins) and, except for occasional mutations, do not change over a lifetime, thereby rendering disclosures permanent. Genomic data also have special properties regarding privacy, especially as comprehensive whole genome sequencing becomes the major technique.

The benefits of amassing genomic data in sufficient case numbers for validity and making this knowledge available to an appropriately wide body of expert investigators are extensive. Research derived from genomic databases offers potentially large health payoffs. Genomics can help scientists predict who will develop a disease (e.g., Huntington’s Disease) and tailor treatments. It also holds the potential to bring about a paradigm shift in how we think about and classify disease; i.e., allowing us to move from the pathology-based approach begun in the late 19th century — which focuses on the progression of disease in a specific organto a biochemical-and genomics-based approach. This new approach is already being applied to a number of diseases, including certain cancers.

Read the rest of this entry »

Workplace Wellness Programs: Continuing The Discussion With Dinardo, Horwitz, And Kelly


August 21st, 2013

This is a response to the DiNardo, Horwitz, and Kelly Health Affairs Blog post in which the authors replied to my previous commentary, “Structuring Legal, Ethical, and Practical Workplace Health Incentives: A Reply to Horwitz, Kelly, And DiNardo.” In my prior post, I highlighted my disagreements with many of the points made by Horwitz et al. in their Health Affairs article entitled “Wellness Incentives in the Workplace: Cost Savings through Cost Shifting to Unhealthy Workers.” This post continues that dialogue.

I begin this commentary with some hesitation. I want to be clear that my intent in posting these blogs is not to “dig in my heels.” In fact, I fully understand, appreciate, and empathize with DiNardo et al.’s positions. They are rightfully concerned about protecting poor, minority, and disenfranchised workers whose rights may be threatened by unscrupulous employers who wish to place the onus on employees to “become healthy” or “else” — the “else” meaning paying a higher health insurance premium than their “healthy” counterparts. I appreciate that DiNardo et al. are protecting the interests of workers who, through no fault of their own, have become ill and are now faced with the prospect of paying more for health care coverage because of their illness.

Let me unequivocally state that paying more for health insurance because you are ill or have certain health risk factors is not the goal of workplace health promotion (wellness) advocates. Quite the opposite is true. Our intent is to keep workers healthy for as long as possible so that they can be spared the human and financial burden of paying for health care services that might otherwise have been avoided. The point of workplace wellness programs is to inspire people to improve their health behaviors and biometric measures so that they do not suffer from illnesses that are to a large degree attributable to lifestyle practices — e.g., lung cancer, type-2 diabetes, chronic obstructive pulmonary disease (COPD), and coronary heart disease (CHD).

In my previous post, I highlighted ways to structure incentive programs so that they are fair and contain provisions to guard against abuse. Here, I address additional challenges to workplace health promotion programs posed by DiNardo et al.

Read the rest of this entry »

Toward A Scientific Approach To Workplace Wellness: A Response To Ron Goetzel


July 1st, 2013

We thank Health Affairs for the opportunity to respond to Professor Ron Goetzel’s comments on our recent Health Affairs article, “Wellness Incentives in the Workplace: Cost Savings through Cost Shifting to Unhealthy Workers.” In many respects our article was quite limited in scope. We started by noting that companies are increasingly adopting wellness programs based on the idea that, with the help of financial incentives, employees will improve their health and employers will save money. We set out to explore the assumptions underlying this idea and found scarce high-quality evidence on the subject of workplace wellness incentives. What evidence we found offers, at best, limited support to justify these assumptions.

We hope that others will conduct the much needed research about whether financial incentives can change behavior, improve health, and control spending. We also hope that the body of causal research will be expanded to a wider variety of populations, such as the elderly, and other related issues, such as the effects on incentives of absenteeism, productivity, and long term health, or to programs that do not involve financial incentives or do not claim to control costs. These other effects and other types of programs are worthy of consideration, but we have not considered them in our article.

The Ethics Of Incentive Programs

Unfortunately, Professor Goetzel misunderstood the scope of our research. Contrary to Professor Goetzel’s assertions, we did not suggest that people should avoid making choices consistent with their health (and other goals), or that employers should not act in the best of interest of employees, employees’ health, or society at large. Still, we are skeptical that wellness programs of the sort encouraged by the Affordable Care Act (ACA) create, as Professor Goetzel suggests, a “win—win—win” situation: good for all workers, good for society, and consistent with the financial interests of employers, firms, and their owners. Rather remarkably, despite the potential for workplace wellness to become a large “social experiment”, we found scant reliable evidence that implementing wellness programs can easily save costs through health improvements without being discriminatory.

Read the rest of this entry »

Unpacking The Meaning Of ‘Rationing’: A Response To Dowd And Allison


June 27th, 2013

Bryan Dowd and Kirk Allison are to be thanked for their lengthy treatise on the word “rationing.” It is a term whose interpretation economists have left to politicians — not invariably models of erudition. Check the subject index of introductory textbooks or even intermediary textbooks in economics and rarely will you see there the term “rationing.”

Part of the lack of clarity on the term can be laid at the doorsteps of the profession that claims to know all about resource allocation but rarely ever takes up the subject of rationing in its teaching. Sadly, modern textbooks in economics are, by and large, just copies of one another — worse than me too drugs in which at least one molecule is changed. At some point, someone forgot to cover the term, so all other texts followed.

But another reason for the lack of clarity on the term reflects the fact that even economists cannot seem to agree on its meaning.

Read the rest of this entry »

Improving The Rhetoric Of Rationing: Part 1


June 24th, 2013

Note: Rationing of health care services is a contentious topic and in our view, current discussions of rationing often are unproductive or harmful. We suggest two reasons for that result. First the discussants are imprecise in their use of the term rationing – often for politically motivated reasons. Second, the discussants write about rationing as an activity that “we” will undertake, with significant consequences for “you.” It would be more helpful to discuss the prudent use of health care services in terms of the health plan that we would want for ourselves and our families.

In part 1 of this post below, we address the first problem by offering a more precise definition of rationing. In part 2 of this post [which will appear on Health Affairs Blog tomorrow], we address the second problem by describing the type of health plan we would like for ourselves and our families.

“Rationing,” and particularly “government rationing” is politically charged rhetoric. Merely mentioning the possibility of government rationing of health care services has a chilling effect on health policy debates. For example, some of the early criticisms of comparative effectiveness research (CER) were based on the possibility that the research would be used as a basis for coverage decisions in public insurance plans, especially Medicare.

Read the rest of this entry »

Seven Ways For Health Services Research To Lead Health System Change


May 30th, 2013

With ACA implementation now at hand — and with it, the formation of accountable care organizations (ACOs) — health services research (HSR) has an especially important role to play. As ACOs take steps that will substantially change health care delivery, the ability to measure and improve health system performance and acquire this data efficiently will be in greater demand. Is HSR up to the challenge?

As the “basic science” of the health care system, HSR focuses on access, cost, and quality. Health services researchers work to identify and assess vital signs of a well-functioning health care system and develop performance measures for examining system aspects. They also seek to improve the system by identifying gaps in quality and then testing and disseminating solutions to those problems. HSR’s goals and often its approaches are different from the development, testing, and translation of new drugs or other interventions.

But as we’ve seen all too frequently, the necessity of responding to ongoing change within the health system outpaces HSR’s ability to produce timely evidence. As a result, large scale changes are sometimes instituted on the basis of imperfect evidence (e.g., tying financial incentives to measures of physician or hospital performance before studies are completed). Or, as we’ve also seen, by the time a research project comes full cycle — proposed, funded, conducted, and published — external or internal forces impacting the health system may have rendered the original question moot. Within the VA, for example, studies regarding the effectiveness of telehealth interventions often have been overtaken by these technologies’ rapid dissemination throughout the system. For HSR to fulfill its mission of helping transform health care, it first must be able to transform itself.

Read the rest of this entry »

Reforming the Research Regulatory System


April 24th, 2013

There is a growing consensus that the regulatory system for research is in need of reform. Established 21 years ago by the Common Rule, it has functioned via a rigorous environment to assure that risk in research is dealt with and transparency maintained.

The trigger for these regulations is a definition of research as a “systematic investigation…designed to develop or contribute to “generalizable knowledge.” When this definition is satisfied, an intensive set of requirements ensues including review, approval, and continued oversight by an Institutional Review Board (IRB); reporting requirements;, the necessity for informed consent (often highly complex); and other administrative components. If projects are not “generalizable,” (e.g., local hospital programmatic or quality review), they fall strictly under healthcare system purview rather than under Common Rule regulatory oversight.

The current system has a strong moral imperative and has been critical to mitigating risk for research subjects and providing transparency. However, it is burdensome and fails to take into account the considerable progress made in both the research and clinical enterprises over the last few decades: in research, technological advances in generating data on routine care, and in healthcare, much more stringent oversight.

Read the rest of this entry »

Bringing Patients Into Health System Change


February 5th, 2013

Editor’s note: For more on the attitudes of Americans toward medical care and empowering patients to be active participants in their own care, see the February issue of Health Affairs, “New Era Of Patient Engagement.”

The attention now devoted to defining the proper role of patients and consumers in clinical decision-making is unusual, both in terms of who is addressing the issue and the intensity of the concern. Once, it was physicians and bioethicists who took the lead; now it is health policy analysts and health care administrators. Their aims go well beyond improving doctor-patient communication or promoting patient autonomy. The primary goal is to enlist patients in the effort to bring fundamental change to health care delivery.

The obligations conferred upon or assumed by patients have changed dramatically over the past half-century. Well into the 1950s, prevailing norms reflected Talcott Parsons’ formulation of the sick role. Patients were duty-bound to seek medical care when ill and follow physicians’ orders. In the 1970s and ‘80s, the new field of bioethics successfully challenged this paradigm, demanding that physicians obtain patients’ informed consent for all interventions, particularly in end-of-life decision-making.

Current expectations are different, less concerned with bioethical principles like autonomy and more committed to the idea that unless patients are genuine partners in medical decision making, altering the health care delivery system is not likely to succeed. The goal is not to advance patient rights but to transform patterns of care.

Read the rest of this entry »

A Cure For Patent Pathology? The Supreme Court Reviews The Patentability Of Human Genes


December 7th, 2012

“Are human genes patentable?” On November 30, 2012, the U.S. Supreme Court agreed to answer this single question in Association for Molecular Pathology v. Myriad Genetics. Of course, the petitioners, including health care providers, professional associations, and patients, worded the question to favor the answer they want: “No, human genes are not patentable.” For Myriad Genetics, the patent owner who would like its patent rights upheld, the question is better phrased as whether one can patent “isolated molecules of deoxyribonucleic acid that were identified and defined by human inventors.”

The practical stakes in the Court’s decision, which should come in the first half of 2013, are enormous. The U.S. Patent and Trademark Office (USPTO) has issued tens of thousands of patents on genetic sequences over the past few decades, and the U.S. Court of Appeals for the Federal Circuit, the appellate court entrusted with hearing virtually all U.S. patent appeals, has never declared such sequences to be non-patentable subject matter. For more than a century, patents have issued on isolated versions of naturally occurring substances other than DNA.

Many believe that gene patents are crucial to the modern biotechnology industry. On the other hand, many researchers and clinicians feel that gene patents, particularly human gene patents, are commonly unnecessary to spur innovation and in fact interfere significantly with scientific and technological progress, whether by slowing or diverting research, impeding the provision of diagnostic tests, or generally increasing costs for clinical and scientific work.

The patents at issue in Myriad make such concerns particularly poignant. These patents relate to BRCA1 and BRCA2, genes associated with a predisposition to breast cancer. The obvious public interest in ready access to cancer diagnosis helps account for the fact that petitioners’ lead lawyers are not the normal high-priced advocates for a private company accused of patent infringement. Instead, those lawyers work for the American Civil Liberties Union Foundation, an organization more commonly associated with battles for civil rights than rights in technological innovations.

Read the rest of this entry »

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 […]

Read the rest of this entry »

Reflections On AIDS 2012


August 1st, 2012

Editor’s note: The current issue of Health Affairs is a thematic volume focusing on the President’s Emergency Plan For AIDS Relief (PEPFAR). Last week’s 19th International AIDS Conference in Washington, D.C. convened 25,000 scholars, activists, practitioners, policy makers, and members of the general public; people living with HIV and people living without the virus; students, […]

Read the rest of this entry »

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 […]

Read the rest of this entry »

Health Care Economics 101 And The Supreme Court


May 23rd, 2012

Editor’s note: Kathryn Gilbert, a J.D. candidate at the University of Michigan Law School, is a coauthor of this post, in addition to Jill Horwitz and Helen Levy (photos and linked bios above). The case that will decide the fate of the most important piece of health care legislation in the past fifty years has, […]

Read the rest of this entry »

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 […]

Read the rest of this entry »

Click here to email us a new post.




This blog is protected by dr Dave\\\'s Spam Karma 2: 1288196 Spams eaten and counting...