“Right to Try” Does Not Help Patients

Co-Blogged by Christopher Robertson and Kelly McBride Folkers (research associate at the Division of Medical Ethics of the NYU School of Medicine)

In 2014, Arizonans overwhelmingly voted in favor of a ballot referendum that claimed to allow terminally ill patients the “right to try” experimental drugs that have not yet been approved by the Food and Drug Administration (FDA). Despite the policy’s broad support, it has yet to help a single patient in Arizona obtain an experimental drug that they couldn’t have gotten before. Thirty-seven other states have also passed right to try bills, but likewise have seen little real impact for patients.

“Right to try” has moved to the federal stage, as the U.S. Senate unanimously passed such a bill last August without even holding a hearing. The House Energy & Commerce Subcommittee on Health considered the bill in an October hearing, but it failed to garner much enthusiasm among committee members. Vice President Mike Pence has advocated for a federal right to try law, and he recently met with FDA Commissioner Scott Gottlieb and House leadership to encourage pass of the bill this year. Continue reading

REGISTER NOW! Will Value-based Care Save the Health Care System?

Will Value-based Care Save the Health Care System?
March 2, 2018 9:00 AM – 5:00 PM
Wasserstein Hall, Milstein East ABC (2036)
Harvard Law School, 1585 Massachusetts Ave., Cambridge, MA

Value-based health care is one of the most pressing topics in health care finance and policy today. Value-based payment structures are widely touted as critical to controlling runaway health care costs, but are often difficult for health care entities to incorporate into their existing infrastructures. Because value-based health care initiatives have bipartisan support, it is likely that these programs will continue to play a major role in both the public and private health insurance systems. As such, there is a pressing need to evaluate the implementation of these initiatives thus far and to discuss the direction that American health care financing will take in the coming years.

To explore this important issue, the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics is collaborating with Ropes & Gray LLP to host a one-day conference on value-based health care. This event will bring together scholars, health law practitioners, and health care entities to evaluate the impact of value-based health care on the American health care system.

This event is free and open to the public, but seating is limited and registration is required. Register now!

Sponsored by the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School with support from the Oswald DeN. Cammann Fund and Ropes & Gray LLP.

Will the Sun Shine All Over Canada? Making Transparent the Financial Relationships of the Medical Industry (Part 2: Towards Effective Transparency)

By Jean-Christophe Bélisle-Pipon

As detailed in Part 1, Ontario government just enacted the Health Sector Payment Transparency Act, a Canadian first in terms of transparency. The act requires that “transfers of value” (or payments), related to medical products (drugs and medical devices), between a payor and a recipient be reported to the Health Ministry. The Act gives the Ministry unprecedented powers to require, analyze, and publish such data online.

A Transformational Act?

Will this act radically transform the practices and the public knowledge that we have about the financial relations of the medical industry? The effective implementation of the regulations will tell us. However, the fact that Innovative Medicines Canada (formerly known as Rx&D, IMC is the organization representing the interests of the pharmaceutical industry in Canada, like PhRMA in the US) has concerns about the Act is a rather positive sign that this legislation might result into pro-social changes. IMC is invoking both ideological concerns (industry’s interactions with HCPs imply cooperation rather than influence) and logistical concerns (“if the threshold for payments is low, a sales representative could easily lose a receipt and forget to report it”), as well as its  own commitment to limiting undue influences. Continue reading

Will the Sun Shine All Over Canada? Making the Financial Relationships of the Medical Industry Transparent (Part 1: Theoretical Transparency)

By Jean-Christophe Bélisle-Pipon

While Canada is often viewed positively for its public, comprehensive, universal, and accessible health care system, not all is rosy. Canada often lags behind other countries in terms of pharmaceutical policies. Sometimes, this is advantageous (e.g., delaying the approval of a product to wait for more clinical data or real-world efficiency, so to better assess risk-benefit and determining the maximum selling price), but more often simply a problem: until recently, transparency in Canada was more a buzzword than a strong and assumed government stance.

However, a few days ago in Ontario, the omnibus Strengthening Quality and Accountability for Patients Act received royal assent, thus enacting the Health Sector Payment Transparency Act. This clearly marked the beginning of shedding light on the financial relationships and payments to health care providers and organizations made by the medical industry (pharmaceutical and medical device companies), the explicit goal being to strengthen patient trust in the health care system (including research and education activities) by allowing patients to assess whether their health care providers are subject to influence by industry and to foster more informed choice. While the United States enacted the Physician Payments Sunshine Act (PPSA) in 2010, which requires payment disclosure, this is a first in Canada.

The main provisions of the Act Continue reading

Register Now! The Policy, Politics & Law of Cancer Conference, Feb 8-9, 2018 at Yale Law School

The Opioid Crisis Requires Evidence-Based Solutions, Part II: How the President’s Commission on Combating Drug Addiction Ignored Promising Medical Treatments

Last year more than 64,000 Americans died of drug overdose, which is “now the leading cause of death” in people under 50. Opioids kill an estimated 91 Americans each day and are responsible for most drug-related deaths in the US. This public health crisis requires solutions that are supported by science and reason instead of emotion and political ideology. In Part I of this three-part series, I discuss how the President’s Commission on Combating Drug Addiction and the Opioid Crisis misinterpreted scientific studies and used data to support unfounded conclusions. In this second part of the series, I explore how the Opioid Commission ignored medical interventions that are used successfully in the U.S. and abroad. In Part III, I will discuss non-medical interventions such as drug checking and safe injection sites. The Commission’s failure to consider these options is likely driven by emotions such as fear and disgust rather than a careful review of scientific evidence.

Medical marijuana is currently accepted in 29 U.S. states and the District of Columbia. It is also permitted in at least 10 countries. However, the Opioid Commission outright rejected calls to consider the use of medical marijuana as an alternative to opioids for managing pain. Prior to the Commission’s first meeting, it solicited input from industry and members of the public on how to address the opioid crisis. In response, it received over 8,000 public comments. According to VICE News, which obtained the documents by submitting a Freedom of Information Act (FOIA) request, most comments were submitted by individuals urging the Commission to “consider medical marijuana as a solution to the opioid epidemic.” A spokesman for the Office of National Drug Control Policy, a body of the Executive Branch that provides administrative support to the Opioid Commission, reports receiving “more than 7,800 public comments relating to marijuana.” Despite these comments, in its final report, the Commission dismissed the notion that marijuana should play a role in treating chronic pain and opioid addiction. Its report cited a recent study from the American Journal of Psychiatry, which concluded that marijuana use was associated with an increased risk of opioid abuse. However, this study relied on data that was collected over twelve years ago. One of its authors, Columbia Medical School Professor Mark Olfson, told CNN that if the data were collected today, they could yield different results.

Continue reading

The Opioid Crisis Requires Evidence-Based Solutions, Part I: How the President’s Commission on Combating Drug Addiction Misinterpreted Scientific Studies

By Mason Marks

The opioid crisis kills at least 91 Americans each day and has far-reaching social and economic consequences for us all. As lawmakers explore solutions to the problem, they should ensure that new regulations are based on scientific evidence and reason rather than emotion or political ideology. Though emotions should motivate the creation of policies and legislation, solutions to the opioid epidemic should be grounded in empirical observation rather than feelings of anger, fear, or disgust. Legislators must be unafraid to explore bold solutions to the crisis, and some measured risks should be taken. In this three-part series on evidence-backed solutions to the opioid crisis, I discuss proposals under consideration by the Trump Administration including recent recommendations of the President’s Commission on Combating Drug Addiction and the Opioid Crisis. Though the Commission made some justifiable proposals, it misinterpreted the conclusions of scientific studies and failed to consider evidence-based solutions used in other countries. This first part of the series focuses on the misinterpretation of scientific data.

Last year more than 64,000 Americans died of drug overdose, which is “now the leading cause of death” in people under 50. Opioids are responsible for most of these deaths. By comparison, the National Safety Council estimates about 40,000 Americans died in auto crashes last year, and the Centers for Disease Control reports that 38,000 people were killed by firearms. Unlike deaths due to cars and firearms, which have remained relatively stable over the past few years, opioid deaths have spiked abruptly. Between 2002 and 2015, U.S. opioid-related deaths nearly tripled (from about 12,000 deaths in 2002 to over 33,000 in 2015). Last year, synthetic opioids such as fentanyl contributed to over 20,000 deaths and accounted for the sharpest increase in opioid fatalities (See blue line in Fig. 1 below). Continue reading

The CVS/Aetna Deal: The Promise in Data Integration

By Wendy Netter Epstein

Earlier this month, CVS announced plans to buy Aetna— one of the nation’s largest health insurers—in a $69 billion deal.  Aetna and CVS pitched the deal to the public largely on the promise of controlling costs and improving efficiency in their operations, which they say will inhere to the benefit of consumers. The media coverage since the announcement has largely focused on these claims, and in particular, on the question of whether this vertical integration will ultimately lower health care costs for consumers—or increase them.  There are both skeptics  and optimists.  A lot will turn on the effects of integrating Aetna’s insurance with CVS’s pharmacy benefit manager services.

But CVS and Aetna also flag another potential benefit that has garnered less media attention—the promise in combining their data.  CVS CEO Larry Merlo says that “[b]y integrating data across [their] enterprise assets and through the use of predictive analytics,” consumers (and patients) will be better off.  This claim merits more attention.  There are three key ways that Merlo might be right. Continue reading

Limited Seats Still Available, Register Now! 12/12: Sixth Annual Health Law Year in P/Review

The Sixth Annual Health Law Year in P/Review symposium will feature leading experts discussing major developments during 2017 and what to watch out for in 2018. The discussion at this day-long event will cover hot topics in such areas as health policy under the new administration, regulatory issues in clinical research, law at the end-of-life, patient rights and advocacy, pharmaceutical policy, reproductive health, and public health law.

Continue reading

2017’s Word Of The Year In Health Law And Bioethics: Uncertainty

This is the first post by Carmel Shachar and I. Glenn Cohen that appears on the Health Affairs Blog in a series stemming from the Sixth Annual Health Law Year in P/Review event to be held at Harvard Law School on Tuesday, December 12, 2017.

2017 was a year of tremendous uncertainty for many areas of public policy. Health care policy was no exception, most prominently with an almost successful push by Congressional Republicans to radically revise the Affordable Care Act (ACA). Medical research and bioethics also faced uncertainty, with the struggle to ethically engage with new technologies and to better understand the boundaries around self-determination. As we look over the past year and anticipate the coming one, the overarching question remains: Is it possible to run a health law and health care system given this level of flux?

Healthcare Policy in Flux

2017 saw a new presidential administration and Congress. Seeking to capitalize on the Republican control of the White House and both Houses of Congress, Congressional Republicans sought to make good on their campaign promise to “replace and repeal” the ACA. The proposed legislation would have dramatically reshaped our health care landscape, including ending Medicaid’s financial status as an entitlement program, and undercutting the health insurance Marketplaces championed by the Obama administration. Despite the fact that the ACA is not yet a decade old, this would have been a seismic shift in the way many Americans receive their health care. […]

Read the full post here.

We will be discussing these issues and more at the Sixth Annual Health Law Year in P/Review conference, held on December 12, 2017, at Harvard Law School in Cambridge, MA. If you find these issues interesting, we invite you to join us as the event is free and open to the public (registered required). For those unable to join us in Cambridge, some of our conference presenters will participate in a blog series to follow  at the Health Affairs Blog. Stay tuned!

TODAY, 12/4 at 5 PM: Health Law Workshop with Rachel Sachs

December 4, 2017 5-7 PM
Hauser Hall, Room 104
Harvard Law School, 1575 Massachusetts Ave., Cambridge, MA

Presentation: “Delinking Reimbursement”

This paper is not available for download. To request a copy in preparation for the workshop, please contact Jennifer Minnich at jminnich@law.harvard.edu.

Rachel E. Sachs is Associate Professor of Law at Washington University School of Law (St. Louis). She is a scholar of innovation policy whose work explores the interaction of intellectual property law, food and drug regulation, and health law. Her work explores problems of innovation and access, considering how law helps or hinders these problems. Professor Sachs’ scholarship has or will have appeared in journals that include the Harvard Journal of Law & Technology, the University of California-Davis Law Review, the Yale Journal of Law & Technology, and the peer-reviewed Journal of Law and the Biosciences. Prior to joining the faculty, Professor Sachs was an Academic Fellow at the Petrie-Flom Center for Health Law Policy, Biotechnology, and Bioethics and a Lecturer in Law at Harvard Law School. She also clerked for the Hon. Richard A. Posner of the U.S. Court of Appeals for the Seventh Circuit. She received her JD magna cum laude from Harvard Law School and a Master of Public Health from the Harvard School of Public Health. She received her AB in Bioethics from Princeton University.

REGISTER NOW (12/12)! Sixth Annual Health Law Year in P/Review

The Sixth Annual Health Law Year in P/Review symposium will feature leading experts discussing major developments during 2017 and what to watch out for in 2018. The discussion at this day-long event will cover hot topics in such areas as health policy under the new administration, regulatory issues in clinical research, law at the end-of-life, patient rights and advocacy, pharmaceutical policy, reproductive health, and public health law.

Continue reading

Roche and City of Hope Claim Pfizer Biosimilar Version of Trastuzumab Will Infringe “At Least” 40 Patents

By James Love

On November 17, 2017, Genentech, a subsidiary of the giant Swiss drug company Roche, together with City of Hope, a charity, filed a complaint in a U.S. District Court, seeking an injunction to block introduction of a Pfizer biosimilar version of Herceptin (INN: trastuzumab), as well as other remedies to infringement, including compensation for Roche’s lost profits if competition occurs. The complaint (Genentech vPfizer, 17-cv-1672, U.S. District Court, District of of Delaware (Wilmington), filed November 17, 2017) illustrates the complexity of the patent landscape on a drug placed on the market more than 19 years ago and the need for compulsory licensing of patents.

Trastuzumab is a very important drug for the treatment of breast cancer that is Human Epidermal growth factor Receptor 2-positive (HER2+). My wife was treated with trastuzumab for several years, and is currently on a follow-on Roche treatment named Kadcyla, which is a combination of trastuzumab and the small molecule DM1. (DM1 is an NIH funded drug now off patent).

The early development of trastuzumab was dramatic, and documented in such accounts as Robert Bazell’s very readable book, Her-2: The Making of Herceptin, a Revolutionary Treatment for Breast Cancer, published in 1998, and the 2008 movie Living Proof, starting Harry Connick, Jr..  Bazell’s book was referred in the New York Times and the New England Journal of Medicine. The Bazell book and the Living Proof movie provide a dramatic account of the unwillingness of Genentech to invest in the research that led to the approval of trastuzmab, and the role of the Revlon Foundation to support Dr. Dennis Slamon’s critical work at UCLA.

Continue reading

What does the Orphan Drug Tax Credit tell us about the Costs of Clinical Trials?

By James Love

Summary:

  • The number of patients enrolled in the trials used to support the registration of novel orphan product are significantly smaller that non-orphan products.  One measure of this is the difference in the enrollment of trials cited in the FDA drug trials snapshots.
  • Since 2015, the average number of trials cited in the FDA trials snapshots for novel drugs were 439 for orphan products, and 2,736 for non-orphans.
  • Data from the Orphan Drug Tax Credit provides insights into the costs of drug development, or more specially, the costs of the clinical trials used to support an FDA approval.
  • From 2010 to 2016, the average qualifying trial costs claimed for the orphan drug credit was $86 million to $102 million, per FDA approved orphan indication (assuming 2 to 3 year average years of lag between the credit claimed and the approval date).  Companies were able to take a credit of $43 to $51 million, on average, for each FDA approval.
  • The $86 to $102 million in pre-credit outlays is far lower than the average of $965 million on trial costs for a new drug approval, estimated by DiMasi and others in 2016.  Some of the differences are explained by the smaller trials for orphan drugs and other differences in methodologies, although both figures include the costs of failed trials and exclude pre-clinical or cost of capital costs.
  • In 2013, the last year for which we have actual rather than projected data on the credit (from the IRS Statistics of Income), the total amount of the credit from all 132 corporate tax returns that claimed the credit was just over $1 billion, nearly the same amount as the DiMasi estimate of $965 million for a single drug. But in 2013, the FDA granted 265 orphan designations and approved 33 orphan indications, including 8 novel products which were approved for an orphan drug lead indication.
  • The data from the orphan drug tax credit illustrates the large gap between the known facts about the costs for R&D for orphan drug development, and the astronomically larger R&D costs claimed by DiMasi (and frequently quoted by other researchers, policy makers and journalists) as averages that should guide policy making.
  • These data underline the need for greater transparency of R&D costs, and more sophistication and realism by policy makers regarding the costs of research and development for drugs qualifying as orphan products.
  • The data from the orphan drug tax credit also provides additional perspective on the estimates of drug development costs provided by Vinay Prasad and Sham Mailankody in their 2017 JAMA paper.

Continue reading

Save The Date! 2/22/18: The Jaharis Symposium on Health Law and Intellectual Property

On February 22, 2018, join DePaul University, located in downtown Chicago, for The Jaharis Symposium on Health Law and Intellectual Property: Technological and Emergency Responses to Pandemic Diseases.

Hosted by DePaul University’s Mary and Michael Jaharis Health Law Institute and the Center for Intellectual Property Law and Information Technology (CIPLIT®), this one day conference will focus on “best practices” in response to emerging pandemic diseases.

Connect with keynote speakers Lawrence Gostin–University Professor and Faculty Director, O’Neill Institute for National and Global Health Law, Georgetown University– and Richard Wilder–Associate General Counsel, Global Health Program, Bill and Melinda Gates Foundation.  They will be joined by other esteemed panelists during this timely and important discussion.

@DepaulHealthLaw

REGISTER NOW (12/12)! Sixth Annual Health Law Year in P/Review

The Sixth Annual Health Law Year in P/Review symposium will feature leading experts discussing major developments during 2017 and what to watch out for in 2018. The discussion at this day-long event will cover hot topics in such areas as health policy under the new administration, regulatory issues in clinical research, law at the end-of-life, patient rights and advocacy, pharmaceutical policy, reproductive health, and public health law.

Continue reading

TOMORROW, 11/9! Book Launch: Specimen Science – Ethics and Policy Implications

Book Launch: Specimen Science: Ethics and Policy Implications
November 9, 2017 12:00 PM
Countway Library, Lahey Room
Harvard Medical School, Boston, MA

In September 2017, MIT Press will publish Specimen Science: Ethics and Policy Implications, co-edited by Holly Fernandez Lynch (outgoing Petrie-Flom Executive Director), Barbara Bierer, I. Glenn Cohen (Faculty Director), and Suzanne M. Rivera. This edited volume stems from a conference in 2015 that brought together leading experts to address key ethical and policy issues raised by genetics and other research involving human biological materials, covering the entire trajectory from specimen source to new discovery.  The conference was a collaboration between The Center for Child Health and Policy at Case Western Reserve University and University Hospitals Rainbow Babies & Children’s Hospital; the Petrie-Flom Center  for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School; the Multi-Regional Clinical Trials Center of Harvard and Brigham and Women’s Hospital; and Harvard Catalyst | The Harvard Clinical and Translational Science Center. It was supported by funding from the National Human Genome Research Institute and the Oswald DeN. Cammann Fund at Harvard University.

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REGISTER NOW (12/12)! Sixth Annual Health Law Year in P/Review

The Sixth Annual Health Law Year in P/Review symposium will feature leading experts discussing major developments during 2017 and what to watch out for in 2018. The discussion at this day-long event will cover hot topics in such areas as health policy under the new administration, regulatory issues in clinical research, law at the end-of-life, patient rights and advocacy, pharmaceutical policy, reproductive health, and public health law.

Continue reading

Book Launch: Specimen Science: Ethics and Policy Implications

Book Launch: Specimen Science: Ethics and Policy Implications
November 9, 2017 12:00 PM
Countway Library, Lahey Room
Harvard Medical School, Boston, MA

In September 2017, MIT Press will publish Specimen Science: Ethics and Policy Implications, co-edited by Holly Fernandez Lynch (outgoing Petrie-Flom Executive Director), Barbara Bierer, I. Glenn Cohen (Faculty Director), and Suzanne M. Rivera. This edited volume stems from a conference in 2015 that brought together leading experts to address key ethical and policy issues raised by genetics and other research involving human biological materials, covering the entire trajectory from specimen source to new discovery.  The conference was a collaboration between The Center for Child Health and Policy at Case Western Reserve University and University Hospitals Rainbow Babies & Children’s Hospital; the Petrie-Flom Center  for Health Law Policy, Biotechnology, and Bioethics at Harvard Law School; the Multi-Regional Clinical Trials Center of Harvard and Brigham and Women’s Hospital; and Harvard Catalyst | The Harvard Clinical and Translational Science Center.  It was supported by funding from the National Human Genome Research Institute and the Oswald DeN. Cammann Fund at Harvard University.

Continue reading

Errors in Patent Grants: More Common in Medical Patents

By James Love

Recently I have become interested in the frequency of a “certificate of correction” on a granted patent, after two efforts to establish federal rights in patents granted.

The first case involved the University of Pennsylvania.  We had identified five patents on CAR T technologies granted to five inventors from the University of Pennsylvania where there was no disclosure of federal funding on the patents when they were granted by the USPTO, as is required by law.  All five patents had been filed in 2014.    We had reason to believe the five patents should have disclosed NIH funding in the invention, and we were right. But the error had been corrected by Penn, and five “certificate of correction” documents were granted by the USPTO in May 2016, something we had overlooked, in part because the corrections to patents are published as image files, and were not text searchable.

The second case involved the Cold Spring Harbor Laboratory.  KEI had identified two patents listed in the FDA Orange Book for the drug Spinraza,  which were assigned to Cold Spring Harbor, and which had not disclosed federal funding.  KEI was interested in pursuing a march-in case for Spinraza, on the grounds of excessive pricing.  The cost of Spinraza in the first year was $750,000, and the maintenance doses were priced at $375,000 per year.  Researchers listed on the two patents had received funding from the NIH to work on the subject of the two patents. Continue reading