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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New regulatory pathways and incentives for sustainable antibiotics: Recent European & US Initiatives

Please find attached a ppt presentation on “New regulatory pathways and incentives for sustainable antibiotics: Recent European & US Initiatives” given on March 7, 2014 at the Broad Institute of MIT and Harvard.  The presentation was followed by a discussion moderated by US patent attorney Melissa Hunter-Ensor, Partner at Saul Ewing, Boston.

I started out by emphasizing increasing problems of antimicrobial resistance (AMR) on a global level, providing new statistics and facts. This was followed by a discussion of main reasons for these alarming developments, such as inappropriate use in agriculture and medicine, insufficient precautions, lack of education, climate change, travel behavior, insufficient collaboration and funding of R&D, scientific complexities, and the problem that incentives provided by the traditional innovation system model often fail in the case of antibiotics.

Next the presentation focused on a variety of solution models that could be discussed to fight AMR. These include both conservational and preventive approaches comprising use limitations, increased public awareness, and better hygiene, but also reactive push & pull strategies, such as increased investments, new collaborative models for R&D in antibiotics, prizes, “sui generis” IP-related incentives, regulatory responses and new pathways for approval.

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Capsule Endoscopy Instead of Colonoscopy? The FDA Approves the PillCam COLON

By Jonathan J. Darrow

In January, the Food and Drug Administration (FDA) approved the use of the PillCam COLON 2 as a minimally-invasive means of viewing the colon, a development that is sure to be welcomed by U.S. patients who currently undergo an estimated 14 million colonoscopies each year.  While the approval represents a major step forward, the PillCam is unlikely to supplant current procedures just yet.

The colon has traditionally been examined via optical colonoscopy, a procedure perceived by many to be uncomfortable and embarrassing that involves insertion through the rectum of a 5-6 foot long flexible tube as part of an examination that can take 30 to 60 minutes. Air must be pumped in through the rectum in a process called “insufflation.” Sedatives and pain medication are generally used to help relieve discomfort. In contrast, the PillCam COLON contains a power source, light source, and two tiny cameras encapsulated in an easy-to-swallow pill that produces no pain or even sensation as it moves through the colon. Reflecting the absence of discomfort, one report from a clinical researcher noted that a few patients have insisted on X-rays to confirm that the device had passed in their stool (FDA Consumer). The pill takes about 30,000 pictures before passing naturally from the body, which usually occurs before the end of its 10-hour battery life.

The safety record of capsule endoscopy, the category to which the PillCam COLON belongs, so far appears to compare favorably with the alternatives. Capsule endoscopy may be less likely to produce accidental colonic perforations or other serious complications, which occur in less than 1% of traditional colonoscopies despite the best efforts of the treating physician. Tears of the colon wall can in turn “rapidly progress to peritonitis and sepsis, carrying significant morbidity and mortality.” (Adam J. Hanson et al., Laparoscopic Repair of Colonoscopic Perforations: Indications and Guidelines, 11 J. Gastrointest. Surg. 655, 655 (2007)). Splenic injury or other serious complications also occur rarely with optical colonoscopies. Unlike “virtual colonoscopy,” which uses computed tomography (CT) to peer into the body, capsule endoscopy does not involve bombarding the body with radiation. A leading study published in the New England Journal of Medicine reported no serious adverse events among 320 subjects given the PillCam COLON, and concluded that use of the device was “a safe method of visualizing the colonic mucosa through colon fluids without the need for sedation or insufflation.” Continue reading

Disclosing Genetic Risks: Lessons from the Philosophy of Language

By Michael J. Young

Earlier this month, landmark findings were published in the American Journal of Psychiatry illuminating the effect of disclosing genetic risk for Alzheimer’s disease on older adults’ cognition and memory.  In a case-control study, researchers administered memory function tests to a group of known carriers of the apolipoprotein E4 allele (one of the best studied genetic risk factors for developing Alzheimer’s disease), half of whom were informed of their carrier status, and half of whom were not informed of their carrier status.  They reported “[s]ignificant genotype-by-disclosure interaction effects were observed on several memory rating scales and tests of immediate and delayed verbal recall. Older adults who knew their ε4+ genotype judged their memory more harshly and performed worse on an objective verbal memory test than did ε4+ adults who did not know. In contrast, older adults who knew their ε4− genotype judged their memory more positively than did ε4− adults who did not know…informing older adults that they have an APOE genotype associated with an increased risk of Alzheimer’s disease can have adverse consequences on their perception of their memory abilities and on their performance on objective memory tests. Similar consequences might be expected if other indices of an increased risk of Alzheimer’s disease are disclosed (e.g., neuroimaging or CSF biomarkers of preclinical Alzheimer’s disease.)”

These findings raise profound questions for practitioners who must make regular decisions concerning how to communicate health risk information to patients, and more fundamentally, about what principles and policies ought to govern which findings should be disclosed or disguised.

The study and its implications bear striking correlates to a set of key issues in philosophy of language relating to the meta-descriptive aspects of speech and discourse.  In a series of lectures later published as How We Do Things with Words, philosopher J.L. Austin persuasively argued against a longstanding philosophical tradition that had conceptualized language as a construct that exists solely to describe reality as it is or is imagined to be; Austin reasoned that language can serve not only to describe facts but can also serve to establish new facts by representing them as such.

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New paper on “Standardization, IPRs and Open Innovation in Synthetic Biology”

I am pleased to announce that we have today published the following paper:

Minssen, Timo and Wested, Jakob Blak, Standardization, IPRs and Open Innovation in Synthetic Biology (February 14, 2014). Available at SSRN.

This brief book contribution stems from a presentation given at the 2013 conference “Innovation, Competition, Collaboration” at Bucerius Law School, Hamburg, Germany. It is currently under review by Edward Elgar.  A longer journal-version will follow.

Abstract: 

An effective and just sharing of resources for innovation needs a supportive infrastructure. One such infrastructure of both historic and contemporary significance is the development of standards. Considering recent developments within the software and ICT industries, it seems fair to assume that the process of standardization may also have significant impact on the development and adoption of Synthetic Biology (SB). Within SB different standardization efforts have been made, but few have assumed dominance or authority. Standardization efforts within SB may differ within various technical areas, and also the basic processes of standard creation can be divided into various categories. The different technical areas and processes for standardization differ in their speed, handling of interests and ability to dodge possible IPR concerns.

Out of this notion arises i.a. the following questions: How comparable is engineering in SB to more traditional fields of engineering?; What type of standards have emerged and what bearing have IPRs on these?; and, How applicable are the approaches adopted by the standards-setting organizations in the information and communication technology (ICT) to biological standards? These and further legal issues related to IP, regulation, standardization, competition law & open innovation require a careful consideration of new user-generated models and solutions.

Before this background, our paper seeks to describe IP and standardization aspects of SB in order to discuss them in the context of the “open innovation” discourse. We concentrate on describing the technology and identifying areas of particular relevance. Ultimately we also sketch out open questions and potential solutions requiring further research. However, due to the limitation of this paper we do not aim to create elaborated theories or to propose solutions in more detail. Rather this paper, which will be complemented by more extensive follow-up studies, provides a first overview on the complex questions that we are currently dealing with.

To achieve this modest goal, section 1 commences with a brief introduction to the fascinating science of SB and a description of recent technological advances and applications. This will lead us to section 2, in which we will address standard setting efforts in SB, as well as the relevance and governance of various IPRs for specific SB standards. This provides the basis for section 3, in which we debate problematic issues and summarize our conclusions.

DC Circuit Upholds FDA Authority Over Stem Cells

Earlier this week, the D.C. Circuit upheld the FDA’s authority to regulate stem cells (for a good news report see here). The company in question, Regenerative Sciences, had received a warning letter from FDA, which the company challenged claiming that its use of stem cells as therapy was not prohibited by existing federal law and that the FDA lacked authority to regulate it. They lost before the district court and appealed to the D.C. Circuit.

In a unanimous decision (by judge Griffith for himself, Judge Srinivasan and Edwards) the D.C. Circuit affirmed this decision. Here are some key passages: Continue reading

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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Sleep versus Training: The NY Times on Surgical Work Hours

Pauline Chen at the NY Times Blog has an article on cut backs on the number of hours of training for young surgeons.

According to her story:

“For the past decade, in response to increasing pressure from politicians, unions and sleep experts, the Accreditation Council for Graduate Medical Education, the organization responsible for accrediting American medical and surgical training programs, has been working to cap the hours that residents work. In 2011 the council passed the strictest limits yet. To maintain their accreditation, residency training programs had to abide by a 22-page set of scheduling rules that limited all in-hospital work including any elective “moonlighting” jobs to 80 hours per week, mandated the number of hours “free of duty” after different “duty periods” (eight hours off after 16-hour duty periods and 14 hours off after 24-hour duty periods) and even specified the timing of “strategic napping” in no uncertain terms (after 16 hours of continuous duty and between the hours of 10 p.m. and 8 a.m.).

While most residency programs chafed under the exhaustively detailed regulations, surgical training programs had particular difficulty adopting the new mandates. For nearly a century, surgical residency had been a period of both intensive experience and increasing responsibility under the guidance of more experienced surgeons. More recent research has affirmed that approach, demonstrating the strong link between a surgeon’s operative skill, the number of operations performed and patient outcomes. With limits set on their time at the hospital, young surgeons-in-training had fewer opportunities to care for patients or scrub in on operations. While previous generations of trainees had the luxury of participating in at least one operation a day, new trainees had only enough time to be involved in two or maybe three operations each week.

Calculating the number of hours “lost” by cutting back on in-hospital time, surgical leaders estimated that young surgeons-to-be were now missing out on as much as a year’s worth of experience”

She relies heavily on an new Annals of Surgery Article, wherein, as Chen reports,

“Researchers sent questionnaires to the directors of subspecialty fellowship training programs and asked them to comment on the bedside and operating skills of the young surgeons enrolling in their programs. These fellowships are a kind of high-level and often prestigious surgical “gap year” where young surgeons who have completed the basic five-year surgery residency can delay independent practice to pursue an additional year or two of training.

The results were abysmal. Fewer than half of the young surgeons could operate or make clinical decisions on their own. Nearly a third of them were incapable of performing even the most basic operations like a gallbladder removal on their own. And a quarter were unable to recognize the early signs of complications.

Even in areas of surgery where the young surgeons had had supplementary online learning modules during their residency, they performed poorly, with more than half unable to perform basic maneuvers.”

While acknowledging that “some observers have criticized the study for being self-serving – most respondents also noted that the young surgeons’ deficiencies resolved after additional training under their auspices —” Chen’s article reads largely as an attack on these new residency work hour rules.

Since I have written on the subject of these residency work hour rules with sleep specialists earlier this year in the Journal of Law, Medicine, and Ethics, it may be unsurprising that I take a somewhat different view.

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Fox interviewed on NPR about FDA’s Ban on Direct-to-Consumer Genetic Testing

Bill of Health Contributor Dov Fox was interviewed today on NPR’s “Marketplace” about the FDA’s decision to ban all sales of direct-to-consumer genetic testing by 23andMe.

23andMe provides information about genetic health risks to people who buy at-home “DNA spit kits.” The company seeks to inform consumers about their susceptibility to more than 250 diseases. But the FDA now says the company hasn’t proved the tests are accurate enough, and the agency is worried Americans are relying on the results instead of visiting their doctor. The FDA exercised its jurisdiction under the Food Drug & Cosmetic Act to regulate the DNA spit kits as a genetic device used in the diagnosis or treatment of disease.

“This field of personalized medicine is really in its infancy,” said Fox, “and its terrifically exciting what we might learn one day, but its just not there yet. 23andMe hasn’t shown that their reports about your health from your genes alone are all that useful. They’re just not accurate at this time in the way that the FDA requires.”

Critics say regulators are standing in the way of consumers having the convenience of obtaining information about their own health at a reasonable price point without an expensive trip to the doctor.  According to Fox, it’s a powerful argument that is often made by genetic testing companies such as 23andMe. The problem is the public’s understanding of genetics is very low. “These tests,” he argued, “come not only with limited accuracy, but also without the benefit of genetic counseling.”

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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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Disruptive Innovation and the Rise of the Retail Clinic

By Michael Young

The Association of American Medical Colleges (AAMC) projects that by 2025 the United States will face a shortage of 130,600 physicians, representing a near 18-fold increase from the deficit of 7,400 physicians in 2008.  The widening gap between physician supply and demand has grown out of a complex interplay of legal, political, and social factors, including a progressively aging population, Congressionally mandated caps on the number of Medicare-funded residency slots and funding for graduate medical education, and waning interest among medical school graduates in pursuing careers in primary care.

These issues generate unprecedented opportunities for healthcare innovators and entrepreneurs to design solutions that can effectively address widening disparities between healthcare supply and demand, particularly within vulnerable and underserved areas.

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Is Turkey’s Health Systems Reform as Successful as It Sounds?

In July, the Lancet covered Turkey’s development and implementation of universal health coverage extensively in an article and in supplementary comments. The main article, written by those who are directly involved in the development of the health systems reform (including the former Health Minister), presents a success story. Within Turkey, however, the success of the reform has been disputed. Two points in particular are being repeated by the Turkish Medical Association (TTB), doctors, and journalists: the negative effects of the reform on (1) the quality of health care personnel and (2) privacy.

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Mental Health in the Intensive Care Unit: The Need for Early Intervention

By Michael J. Young

A prospective cohort study recently published in the New England Journal of Medicine adds to a growing body of research illuminating the neurocognitive sequelae of critical illness requiring intensive care.  Researchers reported that “one out of four patients [treated in the ICU] had cognitive impairment 12 months after critical illness that was similar in severity to that of patients with mild Alzheimer’s disease, and one out of three had impairment typically associated with moderate traumatic brain injury.”  They underscore the possible role of delirium – a syndrome of disturbed consciousness commonly seen in critically ill patients treated in the ICU – in contributing to downstream cognitive impairments.  More broadly, these findings prompt intensivists to rethink the relative priority assigned to the mental health needs of patients during ICU stays.

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HBR/NEJM online forum on health care innovation

For those of you who haven’t seen it yet, there’s a great ongoing online forum over at the joint Harvard Business Review and New England Journal of Medicine Insight Center on Leading Health Care Innovation.  It’s online at HBR here, and will feature an ongoing series of posts about innovation in high-value health care through November 15.  Short articles from scholars in various fields will focus on three main areas: Big Ideas (foundational principles of high-value health care); Managing Innovations (organization and delivery); and From the Front Lines (stories of specific case solutions from practitioners).

They’re looking to host a lively forum, so comments seem both quite welcome and unusually thoughtful so far.

 

Improving Outcomes by Empowering Patients: The Empirical Case for Patient-Centered Care

By Michael Young

As healthcare institutions work to meet the triple aim of reducing healthcare costs while improving quality of care and population-wide health, efforts to expand the role of patient involvement in clinical decision making have gained appreciable momentum.  Interest in increasing patient participation in clinical decision making and goal setting is premised in large part on the notion that greater patient involvement can improve health outcomes by ensuring that treatment plans align with patients’ interests, abilities and sociocultural contexts.  A recent study published in the Journal of Personality and Social Psychology adds to the growing body of empirical research supporting these efforts, suggesting that the very act of allowing patients to choose between treatment alternatives can augment the efficacy of treatment by enhancing patients’ sense of personal control.  By giving patients more opportunities to exercise autonomy at times when many are inclined to perceive their circumstances as well beyond their influence, healthcare providers can help to restore patients’ sense of self-governance and optimize patients’ perceptions of their situations and the medical care they receive.

The idea that empowering patients to choose between treatment options might itself contribute to better health outcomes invites clinicians to rethink how to approach formulating and conveying treatment plans to patients.  Whereas traditionally some clinicians might have conceptualized their roles as experts whose responsibility is to design therapeutic plans on behalf of their patients, the principle of patient-centered care encourages providers to regard patients as associates in developing shared plans to advance and sustain health.  This paradigm of care and the distinctive stance that it demands of clinicians is poetically captured by a letter penned by novelist John Steinbeck to his personal physician, in which he incisively relates “What do I want in a doctor? Perhaps more than anything else—a friend with special knowledge.”

Effectively transitioning to this horizontal model of therapeutic relationships will require careful planning and implementation.  Indeed, boosting patient involvement in clinical decision making may prove increasingly elusive as medical knowledge continues to grow exponentially complex in substance and scope.  As such, to realize the promises of patient-centered care, clinicians should work to develop concrete plans and quantifiable measures to operationalize the ideal of patient engagement within the context of day-to-day practice.  As a first step in this direction, healthcare providers might consider ways to deliver complex biomedical knowledge to patients and surrogates in ways that are more accessible, timely and actionable.  By coupling enhancements in patient education with efforts to promote patient involvement in clinical decision making, clinicians can play a pivotal role in maximizing the efficacy of healthcare innovations and delivery systems of the future.

Safe Surgery Checklists May Raise Unintended Ethical and Legal Questions, Especially in Low-Resource Settings

By Matt Baum

The WHO Surgical Safety Checklist is unusual as a patient-safety intervention in that it has been widely promoted as universally effective, i.e. effective both in high-income and resource-limited settings; checklists are now used in approximately 1800 hospitals worldwide. In a paper recently published in the journal, BMJ Open, Aveling and colleagues report the results of a qualitative study on the implementation of the WHO checklist in two UK hospitals and two hospitals in resource-constrained settings in Africa.  Their results suggest that the checklist is “no magic bullet” – that if adopted without proper investment and adaptation to the context of the target hospital, the checklist not only may fail to replicate benefits, but can actually levy its own unintended costs – especially in resource-limited settings. Though the study raises a number of interesting questions, given the nature of this blog, I am hoping that we might start a discussion about  those in the domain of ethics and law.

For example, consider the following real case, which was reported in the BMJ paper:

“A patient admitted for cholecystectomy [surgical removal of the gallbladder] suffered hypoxic [oxygen depriviation-related] brain injury and died following surgery. Subsequently, two staff members (not the surgeon) were threatened with guns by the patient’s family, who said that the surgical team had killed the patient. The two staff members were later arrested and criminal charges brought against one of them. One of the questions asked during the police investigation was whether a pulse oximeter [i.e. a tool for measuring blood-oxygen levels] had been used. It had not: according to staff, no pulse oximeter was available for use, even though the checklist requiring use of this equipment was, officially, in use at the hospital.”

The staff members also did not get any legal representation for weeks because there were no clear policies established surrounding who was responsible for providing that counsel.

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OHRP Announces Details of August 28 Public Meeting About SUPPORT and Similar Trials

Update: In other SUPPORT news today, a second group of bioethicists has written to the NEJM in, ahem, support of OHRP’s original criticisms of the SUPPORT trial. Readers may recall that another group of prominent bioethicists had previously published a letter in the NEJM in support of SUPPORT.

OHRP today announced details of the public meeting it previously said it would convene to address the SUPPORT trial and similar trials comparing two or more standard-of-care interventions in which subjects are randomized.

From an OHRP email:

On June 26, 2013, the Department of Health and Human Services (HHS) announced in the Federal Register an August 28, 2013 public meeting to seek public input and comment on how certain provisions of the Federal policy for the protection of human subjects should be applied to research studying one or more interventions which are used as standard of care treatment in the non-research context.

HHS specifically requests input regarding how an institutional review board (IRB) should assess the risks of research involving randomization to one or more treatments within the standard of care for particular interventions, and what reasonably foreseeable risks of the research should be disclosed to research subjects in the informed consent process.

HHS is seeking participation in the meeting and written comments from all interested parties, including, but not limited to, IRB members, IRB staff, institutional officials, research institutions, investigators, research subject advocacy groups, ethicists, and the regulated community at large.  The meeting and the written comments are intended to assist HHS, through the Office for Human Research Protections (OHRP), Office of the Assistant Secretary for Health (OASH), in developing guidance regarding what constitutes reasonably foreseeable risk in research involving standard of care interventions such that the risk is required to be disclosed to research subjects.  HHS is seeking input on a number of specific questions but is interested in any other pertinent information participants in the public meeting would like to share.

More details and deadlines after the jump.

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New JAMA paper “The Looming Threat of Liability for Accountable Care Organizations and What to Do About It”

The Journal of the American Medical Association (JAMA) has just posted online (ahead of print) a new article by Ben Harvey and I entitled “The Looming Threat of Liability for Accountable Care Organizations and What to Do About It.” We discuss the Accountable Care Organizations (ACOs) that that the Affordable Care Act seeks to incentivize, the malpractice and other liability claims they may, and how the lack of an Employee Retirement Income Security Act (ERISA) shield of the kind enjoyed by Managed Care Organizations will impact the liability environment ACOs will face. We also discuss self-help remedies available to ACOs as well as potential congressional intervention to deal with these issues.

A Decade’s Quest for Safer Drugs: Congressional Committee Green Lights Regulation of Drug Supply Chains and Compounding Manufacturers

By Mary Ann Chirba and Alice A. Noble

On May 22. 2013, the Senate Health, Education, Labor and Pensions (HELP) Committee unanimously approved S.959, “The Pharmaceutical Compounding Quality and Accountability Act,” and S.957, “The Drug Supply and Security Act,” (now incorporated into S. 959 as an amendment).  Congressional efforts to enact comprehensive legislation to improve drug safety and secure the nation’s drug supply chain have lingered for over a decade. The lack of federal uniformity has allowed a patchwork of state legislation to emerge, attracting the less scrupulous to those states with the lowest security. The issue finally gained traction among HELP Committee members when 55 people died and 741 more became ill after contracting fungal meningitis from contaminated steroid injections made by the New England Compounding Center (NECC). Committee member Sen. Pat Roberts (R-KS) stated that given prior reports of problems with NECC, this tragedy could have been averted but for a “shocking failure to act” by NECC, state and federal regulators, and Congress.

As NECC’s role in the meningitis outbreak came to light,gaps in regulatory oversight did, too. The federal Food Drug and Cosmetic Act (FDCA)[1] currently recognizes only two categories of pharmaceutical manufacturers: commercial pharmaceutical companies and compounding pharmacies. To qualify as the latter under federal law, the entity must make individual or small batch, patient-specific drugs and do so only with a physician’s prescription for that patient.  Compounded drugs must be either be unavailable in the commercial market or needed in commercially unavailable doses or combinations. The FDCA exempts such compounders from its pre-marketing requirements applicable to commercially manufactured drugs. Thus, federal law clearly covers commercial pharmaceutical manufacturers, state law just as clearly oversees and licenses pharmacies but as the NECC case demonstrates, there is nothing clear about the responsibility for inspecting, licensing or otherwise overseeing compounders that do not fill prescriptions on a per patient basis.

Instead of compounding in response to an individual prescription, the New England Compounding Center made large batches of drugs for institutional buyers such as hospitals. Many of its drugs were commercially unavailable but some were knock-offs of marketed FDA-approved drugs – a practice which is clearly unauthorized. NECC’s business model was certainly not unique; neither was the limited and erratic response of state and federal regulators to complaints about the facility’s unsafe manufacturing practices. Congress knew that large-scale compounders existed along with concerns about their safety. Several members of the Senate HELP Committee had worked on curative legislation for over ten years, but made few inroads until the NECC crisis prompted the  HELP Committee to shift from park into drive.

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More Commentary on Why Patients May Be Discriminated Against

Given my recent piece in the New England Journal on discrimination against patients, particularly obese patients in the context of the Americans with Disabilities Act, I found this NY Times story particularly interesting: Disability and Discrimination at the Doctor’s Office. The Times story focuses on patients who are disabled in more traditional ways, but indicates that the main culprit behind the medical discrimination they may experience is not animus, but rather lack of proper equipment, which can be quite expensive.  Luckily, there may be hope on the horizon, thanks to the ACA.