Bioethicist Art Caplan: Right-To-Try Laws For The Terminally Ill Are Bad Policy

A new piece by Bill of Health contributor Art Caplan in Forbes:

Nathan Nascimento thinks that right-to-try laws aimed at the terminally ill are sound public policy. He is wrong.

Mr. Nascimento’s commentary misrepresents the complexities of the drug development process and the issues surrounding granting access to experimental medicines before they have been fully tested.

The overarching issue, despite his rhetoric to the contrary, is that the safety and efficacy profile of a new medicine is not sufficiently understood until after the drug has completed at least a pivotal Phase 3 clinical trial.

The underlying principle of every clinical development program is to understand, via testing first for safety usually in a small number of patients afflicted with the target condition, and, subsequently, in increasing numbers of patients, the benefits as well as the risks of new medicines. Like it or not, this is a time-consuming, expensive, but appropriate and necessary process. []

Read the full post here.

Melinda Buntin on ‘The Week in Health Law’ Podcast

By Nicolas Terry and Frank Pasquale

twihl 5x5This week we welcome Melinda J. Beeuwkes Buntin, Chair of the Department of Health Policy at Vanderbilt University’s School of Medicine. She previously served as Deputy Assistant Director for Health at the Congressional Budget Office (CBO), and worked at the Office of the National Coordinator for Health IT. At RAND, Melinda served as deputy director of RAND Health’s Economics, Financing, and Organization Program and co-director of the Bing Center for Health Economics. Her research at RAND focused on insurance benefit design, provider payment, and the care use and needs of the elderly. For the lightning round, Nic discussed technological improvement of decisionmaking, both for consumers and doctors. Nic also covered CMS’s rejection of Ohio’s request for a new section 1115 demonstration (which would have charged “premiums, regardless of income, to the 600,000 individuals in Ohio’s new adult group, as well as hundreds of thousands of low income parents, foster care youth, and beneficiaries with breast and cervical cancer”). Frank offered a counterintuitive look at the EpiPen and the present technocrat rage to privatize the VA. During the conversation, we covered some topics in CBO modeling, including Melinda’s recent paper on changes in spending by age of beneficiary. Frank mentioned some general concerns about CBO’s modeling raised by Federal Reserve economists, the GAO, Tim Westmoreland (in 2008 and 2007), Maggie Mahar, Timothy Jost, and Bruce Vladeck. We look forward to more conversations on the nature of health cost projections!

The Week in Health Law Podcast from Frank Pasquale and Nicolas Terry is a commuting-length discussion about some of the more thorny issues in Health Law & Policy. Subscribe at iTunes, listen at Stitcher Radio, Tunein and Podbean, or search for The Week in Health Law in your favorite podcast app. Show notes and more are at TWIHL.com. If you have comments, an idea for a show or a topic to discuss you can find us on twitter @nicolasterry @FrankPasquale @WeekInHealthLaw

Subscribe to TWIHL here!

Petrie-Flom seeks Harvard student RA for project on human subjects research

The PFC Logo-New-Horizontal_slidePetrie-Flom Center for Health Law and Policy, Biotechnology, and Bioethics seeks a part-time research assistant for a project with Harvard Catalyst (Harvard Clinical and Translational Science Center) on challenges and innovation in human subjects research, addressing such issues as the regulatory and ethical aspects of research using electronic and online mediums, payment of research participants, models of community engagement and patient-centered research, and participant comprehension in informed consent. The work will initially involve 6-8 hours per week for 3 months. Applicants who have completed at least one year of a graduate program in law, health policy, or a related field are particularly encouraged to apply. Please contact Luke Gelinas, PhD, lgelinas@law.harvard.edu.

The Federal Government Should Consider Medical Marijuana a Potential Ally in the Fight Against Opioid Addiction

By Shailin Thomas

The United States is in the midst of what many are calling an opioid epidemic. According to the American Society of Addiction Medicine, more than 1.9 million people in the U.S. have a substance use disorders involving prescription pain medications, and another 580,000 have substance abuse issues with heroin. The human costs of these rates of addiction are staggering.   Of the approximately 50,000 lethal drug overdoses that happen each year, almost 20,000 are the result of prescription opioids, and another 10,000 are the result of heroin. While prescription painkillers traditionally aren’t as dangerous as heroin, the connection between the two is well established. According to a 2013 survey, about 80% of heroin users started out abusing opioid painkillers.

Despite continued efforts at nearly every level of government, the rates of opioid addiction and overdose have continued to climb. However, researchers have identified an unlikely ally that may have quietly been slowing the rise of opioid use in certain states: medical marijuana.

A study recently released by Columbia University’s Mailman School of Public Health suggests that medical marijuana availability is linked to decreases in opioid usage. The study looked at opioid prevalence in autopsy reports from fatal car accidents over 14 years, and found that states that passed medical marijuana laws in that period saw a relative decrease in opioid prevalence compared to states that didn’t. While this study is making a splash, it’s just the most recent piece in a long line of research into the connection between medical marijuana availability and opioid use. One study published in Health Affairs in July showed that states which implemented medical marijuana laws between 2010-2013 saw a significant decrease in Medicare Part D prescriptions filled for medications for which marijuana is a possible alternative therapy — including opioids. Another study from 2014 showed a 25% decrease in deaths from prescription pain medication overdoses in states that implemented medical marijuana laws. Continue reading

Regulating the Statutory Duty of Candour

By John Tingle

Patients are very much the weaker party in the care equation. Doctors and nurses are always in a much more powerful position as they have the command of a discrete, specialised body of professional knowledge which the patient has not got and often urgently needs. To make the National Health Service (NHS) much more patient centered and to improve the quality of care we need to take account of this imbalance in the care equation which favours healthcarers. We can do this by explaining more carefully to patients about their treatment options and respecting their autonomy as individuals.

All organisations and those working in them need to be honest, open and truthful in all their dealings with patients and the public. A statutory duty of candour now exists in law in the NHS through regulations and is enforced by the Care Quality Commission (CQC), Health and Social Care Act 2008 (Regulated Activities) Regulations 2014: Regulation 20. The CQC can prosecute for a breach of parts 20(2) (a) and 20(3) of this regulation and can move directly to prosecution without first serving a Warning Notice. Additionally, CQC may also take other regulatory action.

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Social Media Use in Research Recruitment: A New Guidance Document from Petrie-Flom and Harvard Catalyst

stethoscope_computerImagine this scenario: you are a researcher conducting a clinical trial on a promising treatment for a rare but serious heart condition. Unfortunately, you are struggling to locate and enroll enough eligible participants and your study is at risk of not completing. Then you discover a Facebook support group for precisely the condition you are studying. The group is open: you do not need to be invited or to suffer from the condition to become a member—anyone can join. Here are the eligible participants you have been looking for!

But what are your obligations in approaching members of this group for recruitment? Would such recruitment be ethically advisable? Under what conditions? And what ethical norms apply when approaching sick and potentially vulnerable people for recruitment over social media? How should you (and the IRB) evaluate this type of activity from an ethical perspective?

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Sarepta: Where Do We Go From Here?

By Rachel Sachs

This morning, the FDA finally reached a decision in the closely watched case of Sarepta Therapeutics and its drug for the treatment of Duchenne Muscular Dystrophy (DMD).  The FDA granted accelerated approval to Sarepta’s drug, Exondys, on the condition that Sarepta complete a new trial demonstrating the drug’s clinical effectiveness.  As regular FDA observers already know, Exondys’ path to approval has been highly contentious.  The key clinical trial used to support approval contained just twelve patients and no placebo, and the FDA Advisory Committee voted against both full approval of the drug and accelerated approval.  The FDA’s 126-page Summary Review, released today with their approval letter, reveals the intense disagreements within the agency over Exondys’ approval.  FDA staff scientists charged with reviewing the drug recommended against approval but were overruled by Dr. Janet Woodcock, Director of the Center for Drug Evaluation and Research.  FDA Commissioner Califf deferred to her decision.

Sarepta presented the FDA with a series of bad options.  DMD is a rare, heartbreaking, and ultimately fatal genetic disease with no other real treatments, and it is clear why patients and their families would fight for access to anything that might work.  At the same time, the FDA has rarely if ever approved a drug on less evidence than that provided by Sarepta, and approving the drug might send a dangerous signal to both the public and other companies investigating rare disease drugs.  The FDA has now made its choice.  Where do we go from here?  In my view, there are at least three key issues to watch going forward. Continue reading

Confidentiality or Public Disclosure: Trump’s Gastroenterologist and an Ethical Dilemma

By Brad Segal

“If elected, Mr. Trump, I can state unequivocally, will be the healthiest individual ever elected to the presidency,” proclaimed Dr. Harold Bornstein. The gastroenterologist’s letter, released on the candidate’s website nine months ago, stumbles from the outset with a typo (“To Whom My Concern,”), then steamrolls over the most basic descriptions of health (medical school teaches us that vital signs are, well, vital), omits information pertinent to the public discourse (why does it fail to mention the medical reason exempting Trump from the Vietnam draft?), and strangely emphases non-medicalized traits (“His physical strength and stamina are extraordinary”). Most experts agree that this medical record, if we can even call it that, is at best hyperbole. It draws grandiose conclusions without medical justification. Even Dr. Bornstein conceded, “In the rush, I think some of those words didn’t come out exactly the way they were meant.”

Just this morning the Trump campaign released a second letter from Dr. Bornstein. But this time the doctor rather humbly concludes, “In summary, Mr. Trump is in excellent physical health.”  These letters from Dr. Bornstein’s letter demonstrate a modern-day moral dilemma in providing care for a party nominee. At conflict is the physician’s professional duty to respect patient confidentiality, and his or her obligations to care for society more broadly.

First, patient-doctor confidentiality is not merely a byproduct of the law—it is a moral obligation grounded in the core tenants of the medical profession. To put it simply, if a patient comes to expect that his doctor will tell the entire community about the patient’s most embarrassing bodily defects, the patient may understandably deny his worsening symptoms of poor health at the next office visit. In the long run, erosion of trust in the medical system could endanger the public’s health–everyone is thus better off when doctors uniformly respect patient privacy. It is important to point out, however, that an informed and competent person can voluntarily waive one’s right to patient-doctor confidentiality, such as when a patient gives a physician the permission to provide updates to family members. Or when then-candidate John McCain instructed his physicians all 1,100 pages in his medical records.

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Knowledge is Power, or Ignorance is Bliss?

By Kyle B. Brothers

You have a rare illness that seems to have a genetic cause. For years you have moved from geneticist to geneticist looking for the cause of your illness, hoping that by finding the precise genetic cause you will discover ways to alleviate your symptoms. You have had five or six genetic tests, but each one has turned up normal. Finally you visit a young geneticist fresh out of training, hoping that she will know of another test to try. She recommends the most comprehensive genetic test of all: whole genome sequencing (WGS). You are ready to immediately get this test when she poses a difficult question: WGS might reveal a cause for your illness, but it might also reveal that you are at risk for developing breast cancer, or schizophrenia, or Alzheimer’s disease. Which of these “incidental” findings do you also want to receive?

Until recently, this genomic “would you want to know” question has lived exclusively in the world of science fiction. Would you want to know what secrets your genome holds about your future? For example, would you want to know how you will die? If you knew what the future is likely to hold, would you feel fatalistic or empowered to take control of it? These questions have been the topic of compelling movies like GATTACA and classic novels like Aldous Huxley’s Brave New World. Continue reading

Mental Health First Aid Training in Prisons, Police Departments, and the Presidential Election

By Wendy S. Salkin

It has been widely reported and acknowledged that many incarcerated Americans live with mental illness. In 2014, the Treatment Advocacy Center and the National Sheriffs’ Association published The Treatment of Persons with Mental Illness in Prisons and Jails: A State Survey, a joint report that included the following findings:

  • In 2012, there were estimated to be 356,268 inmates with severe mental illness in prisons and jails. There were also approximately 35,000 patients with severe mental illness in state psychiatric hospitals. Thus, the number of mentally ill persons in prisons and jails was 10 times the number remaining in state hospitals.
  • In 44 of the 50 states and the District of Columbia, a prison or jail in that state holds more individuals with serious mental illness than the largest remaining state psychiatric hospital. For example, in Ohio, 10 state prisons and two county jails each hold more mentally ill inmates than does the largest remaining state hospital.

Similarly widely reported and acknowledged is that prisons often either cannot or simply do not serve the mental health treatment needs of those housed within their walls. As Ana Swanson of The Washington Post observed:

Unsurprisingly, many prisons are poorly equipped to properly deal with mental illness. Inmates with mental illnesses are more likely than other to be held in solitary confinement, and many are raped, commit suicide, or hurt themselves.

Solitary confinement is often used as a means of separating inmates living with mental illness from the rest of a prison population. As Jeffrey L. Metzner and Jamie Fellner reported in their March 2010 article, “Solitary Confinement and Mental Illness in U.S. Prisons: A Challenge for Medical Ethics”: Continue reading

J. B. Silvers on ‘The Week in Health Law’ Podcast

By Nicolas Terry and Frank Pasquale

Subscribe to TWIHL here!
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We welcomed J. B. Silvers to the podcast this week. J. B. is the John R. Mannix Medical Mutual of Ohio Professor of Health Care Finance, and Professor of Banking and Finance, at the Weatherhead School of Management, with a joint appointment in the Case Western Reserve University School of Medicine.

We asked J. B. many questions about the state of the ACA, hospitals’ adaptation to the rapidly changing policy environment, and ongoing worries about a death spiral on the exchanges. He offered refreshing and insightful perspectives on a range of live controversies in health care finance.

J. B. has served on committees at the National Academies and several national and state commissions. Until recently, he was a board member (12 years) and treasurer of the Joint Committee on Accreditation of Healthcare Organizations (TJC/JCAHO) and a board member of SummaCare Insurance Company (14 years). For seven years Silvers was a commissioner on the Prospective Payment Assessment Commission (now MedPAC) advising Congress on Medicare payment. From 1997 to 2000, while on leave, he served as President and CEO of QualChoice Health Plan and Insurance Company. He currently is vice chair of the board at MetroHealth Medical Center.

The Week in Health Law Podcast from Frank Pasquale and Nicolas Terry is a commuting-length discussion about some of the more thorny issues in Health Law & Policy. Subscribe at iTunes, listen at Stitcher Radio, Tunein and Podbean, or search for The Week in Health Law in your favorite podcast app. Show notes and more are at TWIHL.com. If you have comments, an idea for a show or a topic to discuss you can find us on twitter @nicolasterry @FrankPasquale @WeekInHealthLaw

Medical Malpractice Under a National Health System and the ACA

By Matthew H. H. Young

What will happen to the current medical malpractice system under a single-payer system?

To answer this question, I started by looking at the information provided by Physicians for a National Health Program, whose mission is to replace the ACA (Affordable Care Act) with single-payer. On their website under Single-Payer FAQs, it says:

What will happen to malpractice costs under national health insurance?

They will fall dramatically, for several reasons. First, about one-fourth of all malpractice awards go to pay present and future medical costs (e.g. for infants born with serious disabilities). Single payer national health insurance will eliminate the need for these awards. Second, many claims arise from a lack of communication between doctor and patient (e.g. in the Emergency Department). Miscommunication/mistakes are heightened under the present system because physicians don’t have continuity with their patients (to know their prior medical history, establish therapeutic trust, etc) and patients aren’t allowed to choose and keep the doctors and other caregivers they know and trust (due to insurance arrangements). Single payer improves quality in many ways, but in particular by facilitating long-term, continuous relationships with caregivers. For details on how single payer can improve the quality of health care, see “A Better Quality Alternative: Single Payer National Health Insurance.” For these and other reasons, malpractice costs in three nations with single payer are much lower than in the United States, and we would expect them to fall dramatically here. For details, see “Medical Liability in Three Single-Payer Countries” paper by Clara Felice and Litsa Lambkros.

Let me address the most salient part of the above argument, which states that the significant burden of malpractice recoveries composed of future medical costs will be alleviated because all individuals will be insured. Continue reading

Kidneys and Livers, Made to Order?

By Seán Finan

Last week, Organovo might just have revolutionised the pharmaceutical industry. The San Diego-based company specialises in producing structures that mimic the behaviours and functions of human tissue, using 3D bioprinting. They announced last week that they were beginning the commercial manufacture and sale of their ExVive Kidney. The product models the proximal tubule of the human kidney and holds significant promise for clinical trials of new drugs. The commercialization of the ExVive Kidney follows the release of ExVive Human Liver Tissue in 2014.

In essence, Organovo is using 3D printing technology to produce samples of “human” tissue that can be used to test the toxicity of new drugs. The printing process, known as 3D bioprinting, involves extracting human cells, culturing them and suspending them in a solution. The resulting “bioink” is fed through a modified 3D printer. Layer by layer, the printer deposits cells, producing a mass with a similar structure and density to a sample of, for example, human liver. Just like “organ on a chip” technology, these synthetic liver and kidney samples present significant advantages over traditional clinical testing.

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Vaccinate Your Children (Says a Federal Judge)

Flickr Creative Commons-Dan Hatton

Flickr Creative Commons-Dan Hatton

By Gregory M. Lipper

After nearly four years fighting about whether and when employers may exclude contraceptive coverage from employee health plans (and even block others from providing that coverage), it’s perhaps refreshing to see less controversial cases. And few healthcare-exemption cases are less controversial than those brought by parents who object to vaccinating their children. Although the challenged laws are objectively more intrusive than the contraceptive regulations—vaccination laws require parents to get the offending treatment injected into their children—courts thus far have correctly dismissed these challenges with little fanfare.

This dynamic surfaced again in a recent federal trial-court decision in California, in which the court dismissed a federal and state constitutional challenge to California legislation repealing the “personal belief exemption” to requirements that those entering schools and child-care facilities get vaccinated against diseases—including diphtheria, measles, mumps, rubella, and other dreadful ailments. The court acknowledged that eliminating the personal-belief exemption “raises principled and spirited religious and conscientious objections by genuinely caring parents and concerned citizens,” but stated that “the wisdom of the Legislature’s decision is not for this Court to decide.” Because the legislature decided to scrap the personal-belief exemption, California now exempts only those children (1) with actual medical reasons for avoiding the vaccination, (2) who are home schooled, or (3) who qualify for an Individualized Education Program under federal disabilty law. That’s a much more limited and manageable group of exemptees.

Although quite a relief for those seeking to minimize gratuitous suffering from preventable diseases, the court’s decision implicates several knotty legal issues and is worth exploring further.

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Hospitals Should Think Before Performing Searches for Law Enforcement

In 2012, a Jane Doe suspected of transporting drugs was detained by U.S. Customs and Border Protection (CBP) agents without a warrant, and brought to University Medical Center in El Paso, Texas. Medical Center personnel — under the direction of the law enforcement agents — performed an X-ray, CT scan, and cavity search before determining that the woman was not in fact carrying any controlled substances. A few months after suffering this traumatic — and possibly illegal — event, the woman received a $5400 bill from the Medical Center for the services rendered as part of the search.

While the woman was compensated to some extent — she settled lawsuits with University Medical Center and the CBP to the tune of $1.1 million and $475,000, respectively — her story, and stories like hers, raise important questions about the ways in which hospitals should (or shouldn’t) work with law enforcement to perform invasive searches.

It’s understandable why hospitals and medical professionals are inclined to cooperate with law enforcement requests for invasive procedures and cavity searches — law-abiding citizens often don’t want to obstruct law enforcement agents from doing their jobs. But in the course of bringing suit against University Medical Center, Edgar Saldivar of the ACLU of Texas noted that the hospital and many of its personnel didn’t know where the obligation to assist the CBP stopped. Many medical professional don’t know that — according to the CBP’s own Personal Search Handbook — they are under absolutely no obligation to comply with requests by law enforcement to perform cavity searches with or without a warrant.

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Corralling the Herd: California Medical Board Acts Against Anti-Vax Doc

This summer, California’s strict new childhood immunization law, barring all exemptions except those needed for medical purposes, went into effect for public and private schools, preschools, and day cares. This law was passed as a response to the highly-publicized 2014-2015 multi-state measles outbreak that originated at Disneyland, and also in response to the growing number of California communities with large clusters of families exempting their children from vaccine requirements, putting at risk community protection from vaccine-preventable illnesses.

As I’ve written about before, both here and in articles with Tony Yang, there are many different ways to structure childhood vaccination laws. While much of the attention goes to whether or not states offer parents the right to exempt their children based on religious and/or philosophical grounds — see, for example, the recent American Academy of Pediatrics report supporting mandated vaccination for school and daycare attendance —  there are many other implementation-related details in the laws that can increase or decrease the law’s efficacy at maintaining high vaccination coverage rates. For example, some states may require that exemption requests be filed annually (increases efficacy), some states require only that a form be correctly completed (decreases efficacy), some states allow for historically anti-vax practitioners, such as naturopaths, to complete medical exemption forms (decreases efficacy, and creates a new, permanent loophole for gaining exemptions), some states require that medical exemptions be reviewed and approved by a state public health officer (increases efficacy). Continue reading

Introducing the 2016-2017 Petrie-Flom Student Fellows

The Petrie-Flom Center is pleased to welcome our new 2016-2017 Student Fellows. In the coming year, each fellow will pursue independent scholarly projects related to health law policy, biotechnology, and bioethics under the mentorship of Center faculty and fellows. They will also be regular contributors here at Bill of Health on issues related to their research.

Sean Finan PhotoSeán Finan is an LLM candidate from Ireland at the Harvard Law School. He recently graduated from the LLB programme at Trinity College, Dublin, where he served as a Senior Editor of the Trinity College Law Review. His research interests include the ethical implications of emerging biotechnologies. For his Fellowship project, he intends to investigate the use of morality tests on patent applications as a means of indirect regulation of research.

Wendy Salkin Square HeadshotWendy Salkin is a Ph.D. candidate in the Department of Philosophy at Harvard University. Her primary research is in political philosophy, moral philosophy, social philosophy, and philosophy of law. She also works on questions in feminist philosophy and bioethics. She is writing a dissertation on informal political representation under the supervision of Tommie Shelby, T.M. Scanlon, Richard Moran, and Eric Beerbohm. She holds a J.D. from Stanford Law School and a B.A. in Philosophy and Africana Studies from New York University. For her Fellowship project, she will examine new directions in the debate over lifespan extension.

Segal photoBrad Segal is currently a medical student at Harvard Medical School where he is enrolled in a dual MD/Master of Bioethics degree program. Brad received his BA and BS from UC San Diego where he double majored in Philosophy and Physiology/Neuroscience. In his first year at HMS Brad’s paper on the ethics of organ transplantation was awarded the Henry K. Beecher Prize in Medical Ethics. He has also studied the ethical implications of our evolving understanding of the brain, and has published on whether and when individual genetics and neurobiology should mitigate a criminal defendant’s moral culpability. During his Fellowship he will be studying what ‘harm’ means in the medical context.

Thomas Shailin PhotoShailin Thomas is a second year law student in a joint MD/JD program between Harvard Law School and the New York University School of Medicine. He received a B.S. from Yale University, where he studied cognitive neuroscience — exploring the anatomy and physiology behind social phenomena. His interests lie at the intersection of clinical medicine and the legal forces that shape it. Prior to law school, Shailin worked on both the administrative and clinical sides of health care, and as a research associate at the Berkman Center for Internet & Society. He is currently an affiliate of the Berkman Center and Outreach Editor for the Harvard Journal of Law & Technology. A fervent proponent of privacy and freedom of expression, Shailin has also served on the Board of Directors of the American Civil Liberties Union of Connecticut. For his Fellowship project, he will focus on a tort solution for faulty eyewitness testimony procedures.

Matthew Young HeadshotMatthew H. H. Young is a fourth-year MD candidate at Harvard Medical School and a second-year JD candidate at Harvard Law School. He graduated with honors from Harvard College with a major in Government and a minor in Global Health & Health Policy. He is interested in healthcare system safety, quality, and outcomes, and serves as the Director of Medical-Legal Affairs for the Institute for Patient Safety and Outcomes (IPSO). For his Fellowship project, he plans to develop a patient-centered checklist to improve perioperative safety, quality, and outcomes, and discuss the checklist’s implications.

Patient Safety and Clinical Risk in Neonatal Care

By John Tingle                                     

The CQC (The Care Quality Commission)  is the independent regulator of health and adult social care in England. They make sure that health and social care services provided to people are safe, effective, compassionate, high-quality care and they encourage care services to improve. The  CQC inspects health facilities and they have important statutory regulatory powers and sanctions.They have recently produced a report on neonatal care and  on  providing care for infants in the community who need respiratory support. As well as some positive findings, the report does reveal a number of major patient safety risks and failings.

In England, one in every nine babies is born needing care from neonatal services and  this is on the increase. The care process here can be challenging with sick babies with complex health needs receiving hospital care and then care at home and in the community. The care of the baby traverses’ distinct pathways or care areas and sometimes problems can occur:

A lack of consistency in care and communication across a pathway can result in poor outcomes for both babies and parents.” (p.3)

The report looks at current practice in three different aspects of care: Continue reading

Considering stakeholders in policy around secondary findings in genomics

By Michael Mackley

It took nearly thirteen years and an army of scientists to generate the first sequence of the human genome. Now, patients around the world are having their genomes sequenced every day. Since the first sequence was unveiled in 2003, the cost of whole-genome sequencing (WGS) has dropped from almost $1 billion to less than $1,000—allowing WGS to enter routine clinical care, potentially transforming the way we diagnose and treat disease. Large national initiatives to read individuals’ genomes are helping to drive this transition; the UK’s NHS England is currently sequencing 100,000 genomes, and the USA has plans to sequence 1 million genomes in the near future. A 2015 study predicts that up to 2 billion people worldwide could have their genomes sequenced within the next decade—comparable to the current reach of the Internet. With so many genomes to be sequenced, it is imperative that laws and policy ensure that individuals, and society, are protected from harm. While larger pieces of legislation—such as those protecting against discrimination—are needed internationally, guidance and policies around routine management are also required.

One area of particular concern is that of ‘secondary’ (or ‘incidental’) findings. While WGS provides a valuable opportunity to learn about genetic contributions to disease (‘primary’ findings), it can also reveal genetic information that may not be relevant to the health condition affecting the patient or their family. This includes genetic changes associated with other health conditions—ranging from medically actionable findings, such as genetic predispositions to breast cancer where treatment is available, to non-actionable findings, such as genetic changes associated with an increased risk of Alzheimer’s. The American College of Medical Genetics and Genomics published recommendations suggesting a moral obligation to seek and return actionable secondary findings, fueling significant debate (1,2). Medical Genetics organizations from other countries (including Canada and Europe) have published more conservative guidelines restricting generation of secondary findings, at least until more evidence is available to support (or refute) clinical utility and assess wider impacts. Continue reading

Mylan Announces Generic EpiPen; Baffles Health Policy Wonks Everywhere

For weeks now, the list price of Mylan’s EpiPen ($600 for a two-pack) has been exhaustively covered by journalists, debated by academics, and skewered by policymakers as an example of the pricing excesses of even generic pharmaceutical companies.  Mylan’s latest response to the outrage?  Announce that soon, it will be launching a generic EpiPen at a list price of $300 for a two-pack.  I and others who study these issues full time cannot understand why Mylan thought this would work to quell the widespread indignation over its pricing practices.

The first red flag came when Mylan stated it would launch the product “in several weeks.”  I often find myself defending the FDA against charges that it is too slow to approve new technologies, but let’s face it: it would be shocking news if they were able to approve a new version of anything in just a few weeks.  Mylan has not had this in the works for months, so it seems that the new generic product is literally identical to the branded EpiPen – just with a different label.  So, essentially, Mylan is preparing to cut the price of its product in half.  (Even though that’s still higher than the price was just three years ago, before Mylan began its regular price hikes, and even though this should make us question their justifications for the $600 price.) Great, right?  Not so fast.

What reasons (other than public relations) might Mylan have for introducing an authorized generic of this type and how might they attempt to use the two products to maintain their current level of revenues?  By bringing the first generic EpiPen to market, Mylan has now planted its flag in the generics space.  Although epinephrine (the drug inside the EpiPen) is now generic and cheap to produce and sell, companies do seem to find it difficult to replicate the device portion of the EpiPen, with Sanofi’s product recently removed from the market due to dosing issues and Teva’s application for a generic denied by the FDA with no public explanation just a few months ago.  Mylan has now benchmarked a new price for those products if they return – they must price below $300 for a two-pack to compete effectively with Mylan. Continue reading