National Survey Suggests that Off-Label Status is Material to Informed Consent

By Christopher Robertson

As many readers of this blog know, the FDA requires that, prior to entering the market, companies prove safety and efficacy for each intended use of their products, but physicians are then free to prescribe the products for any other uses.  (Companies are not allowed to promote off-label uses however.)

A recent national survey by Consumer Reports includes two interesting findings:

  1. About two-thirds (63%) of Americans “would not take a doctor prescribed medication that has been approved by the FDA, but not for their specific condition.”
  2. Almost all Americans (94%) “say they have never been told by a physician that a medication they were taking was not approved by the FDA for their condition.”

Patients are right to be skeptical of off-label uses, though they may not appreciate just how common they are.  In fact, most off-label use is unsupported by scientific evidence as to safety and efficacy.  A new report by the FDA illustrates several off-label uses that were subjected to rigorous clinical trials and turned out to be ineffective or dangerous.   For example, Aliskiren is approved for treatment of hypertension and was used off-label for prevention of congestive heart failure (CHF) complications.  A large trial showed that, although it did not significantly improve CHF mortality, it did significantly increase rates of kidney failure for CHF patients.  We do not know how many other off-label uses would fail if similarly tested.   Read More

The ACA’s Real Effect: Moving the Goalposts

By Christopher Robertson

“I believe and I look forward to working with you to make certain that every single American has access to the highest-quality care and coverage that is possible. … [W]e believe it’s appropriate to put in place a system that gives every person the financial feasibility to be able to purchase the coverage that they want for themselves and for their family.”

That quote is not from Barack Obama.  It’s from Trump HHS nominee Tom Price, and it shows just how successfully the ACA has shifted the American political landscape towards universal coverage. As I argued earlier this month in STAT, with Glenn Cohen and Holly Fernandez Lynch, the debate is now about how to get universal health insurance coverage, rather than whether to do so.

Republicans will of course favor market-oriented approaches, and they will find difficulty conceiving a plan that is farther to the right than the ACA itself while actually achieving the goals that Price promises.  But for now, even if the ACA is soon repealed, it has succeeded in moving the goalposts for health policy.

Income-Scaling of Cost-Sharing Gains Traction

By Christopher Robertson

With 148,000 members, the American College of Physicians (ACP) is the largest medical-speciality organization.  This summer, its board released a new report on the growing financial burdens faced by patients who enjoy health insurance but are nonetheless exposed to unbearably large costs for healthcare.  At the end of the day, cost-sharing is just the absence of insurance for those costs.

ACP calls for a range of reforms, including “income-adjusted cost-sharing approaches that reduce or directly subsidize the expected out-of-pocket contribution of lower-income workers to avoid creating a barrier to their obtaining needed care.”  As I have argued, the Affordable Care Act includes income-based subsidies for cost-sharing in the Marketplaces, but these are currently being challenged in court, and do not apply to the employer-based system or Medicare, which together cover the vast majority of patients.

Hillary Clinton has also advanced a plan to create progressive refundable tax credits for people who spend more than 5% of their income out-of-pocket.   The advantage of such a tax-based approach is that it reaches patients regardless of where they get their insurance (except for Medicare, which is excluded).  The disadvantage is that it leaves people in a state of financial insecurity until they get their refunds.  A better approach would scale cost-sharing exposure in the first place, a power that I have suggested is already available under Federal law and which is self-funding.

Fighting the Next Pandemic: Airline Vaccine Screens

By Christopher Robertson

Whether it is Ebola, H1N1, the season flu, or the next nasty bug that we cannot yet even imagine, if we wanted to efficiently spread the disease, one could not do much better than packing several Flight routeshundred people into a cylinder for a few hours, while they eat, drink, defecate, and urinate.  Even more, to make sure that the disease cannot be contained in a particular locality, we could build thousands of those cylinders and move them rapidly from one place to another worldwide, remix the people, and put them back in the cylinders for return trips back to their homes, schools, and jobs.

We are (hopefully) not going to stop airline travel.  But we can make it a lot safer, by ensuring that almost everyone who boards these flights is vaccinated.  That’s the thesis of a new paper out this week.

Airlines carry two million people every day.  And, prior research has shown that airline travel is a vector of disease.  In fact, when the September 11 attacks caused airline travel to fall, seasonal flu diagnoses fell too.

The threat of pandemics is quite real, and more generally, the mortality and morbidity associated with infectious disease is a severe public health burden.  About 42,000 adults and 300 children die every year from vaccine-preventable disease.  New vaccines are on the horizon.

Arguably, airlines have market-based and liability-based reasons to begin screening passengers, whether for vaccinations generally or for particular ones during an outbreak.  Although the states have traditionally exercised the plenary power to mandate vaccinations, and have primarily focused on children in schools, the U.S. federal government also has substantial untapped power to regulate in this domain as well.

Recent Developments in Off-Label Promotion

By Chris Robertson

July has been a busy month for those following the controversy around off-label promotion of drugs and devices.  As many on this blog know, federal law requires that prior to marketing any drug or device, companies must prove to the FDA’s satisfaction that it is safe and effective for all intended uses.  If the company reveals that it intends unapproved uses,  sales of the drug or device are illegal.  Nonetheless, physicians can prescribe “off-label,” and companies are free to sell for those known-but-not-intended purposes.

This carefully-wrought policy may seem convoluted, but it serves important epistemic and economic purposes, as I have argued elsewhere.  This month, I have a new draft paper on SSRN, assessing recent assertions of a First Amendment right to promote for uses not approved by the FDA, and consider whether such a right would be equally applicable to drugs that have no FDA-approved label at all. I worry that the entire pre-market approval regime may be at stake. Feedback on that intentionally-provocative analysis is quite welcome.

On Wednesday, two medical device company executives, were convicted of promoting a product “to deliver steroid medications to patients’ sinuses, though it was only approved by the U.S. Food and Drug Administration for keeping sinuses open.”  The prosecutors thought the case was particularly egregious, because the company had intended the broader use to deliver medicine all along, but sought to mislead the FDA, denying it the chance review the safety and efficacy of the real intended use.  The jury instructions and verdict form  are particularly interesting, to see how the government’s trial strategy avoids the holding of a Second Circuit case of Caronia, which overturned a conviction on First Amendment grounds.  I’ll return with some analysis later. Read More

NOW AVAILABLE FOR PRE-ORDER! Nudging Health: Health Law and Behavioral Economics

This volume, edited by I. Glenn Cohen, Holly Fernandez Lynch, and Christopher T. Robertson, stems from the Petrie-Flom Center’s 2014 Annual Conference “Behavioral Economics, Law, and Health Policy.” Pre-order your copy today!

Nudging HealthBehavioral nudges are everywhere: calorie counts on menus, automated text reminders to encourage medication adherence, a reminder bell when a driver’s seatbelt isn’t fastened. Designed to help people make better health choices, these reminders have become so commonplace that they often go unnoticed. In Nudging Health, forty-five experts in behavioral science and health policy from across academia, government, and private industry come together to explore whether and how these tools are effective in improving health outcomes.

Behavioral science has swept the fields of economics and law through the study of nudges, cognitive biases, and decisional heuristics—but it has only recently begun to impact the conversation on health care. Nudging Health wrestles with some of the thorny philosophical issues, legal limits, and conceptual questions raised by behavioral science as applied to health law and policy. The volume frames the fundamental issues surrounding health nudges by addressing ethical questions. Does cost-sharing for health expenditures cause patients to make poor decisions? Is it right to make it difficult for people to opt out of having their organs harvested for donation when they die? Are behavioral nudges paternalistic? The contributors examine specific applications of behavioral science, including efforts to address health care costs, improve vaccination rates, and encourage better decision-making by physicians. They wrestle with questions regarding the doctor-patient relationship and defaults in healthcare while engaging with larger, timely questions of healthcare reform.

Nudging Health is the first multi-voiced assessment of behavioral economics and health law to span such a wide array of issues—from the Affordable Care Act to prescription drugs.

Read the introduction on SSRN and pre-order your book now!

Blinding as a Solution to Bias

Blinding Bias

We’re pleased to announce that a new book by Petrie-Flom Center affiliates Christopher Robertson and Aaron Kesselheim will be available this week.  The edited volume — Blinding as a Solution to Bias: Strengthening Biomedical Science, Forensic Science, and Law — grew from a 2013 conference at Harvard co-sponsored by the Petrie-Flom Center.  From the fascinating history of blinding (starting in Ben Franklin’s living room to test the healing powers of Mesmerism) to the sham surgeries being used today, and the future novel uses of blinding in the courts, the book moves the debate forward.  What are the advantages and limitations of blinding compared to other solutions for biases? How can we quantify the biases in unblinded research? Can we develop new ways to blind decision-makers?  What are the ethical problems with withholding information?  Fundamentally, questions about who needs to know what open new doors of inquiry for the design of scientific research studies, regulatory institutions, and courts.  With a foreword by Larry Lessig, the book surveys the theory, practice, and future of blinding, drawing upon leading authors with a diverse range of methodologies and areas of expertise, including medicine, law, forensic sciences, philosophy, economics, psychology, sociology, and statistics.  It is available for pre-order from the publisher and Amazon.com.

Some Commentary on How to Think About Secondary Research with Biospecimens

The public comment period on the NPRM to revise the Common Rule has just closed, and now we wait to see what happens (if anything), and when.  One of the most controversial proposals in the NPRM would require at least broad consent for secondary research with biospecimens (i.e., research on specimens originally collected for another purpose, either clinical care or a different study), regardless of whether those specimens retain identifiers.  This is a substantial change from the status quo, which does not require consent for such research with de-identified specimens.  How should we feel about this status quo, and the proposed change?  My own view is that it’s really not so bad: the risks to individual research participants are quite low, and the current approach facilitates critically important scientific advancement.  There is certainly room for improvement, e.g., to impose punishment on those who would act to re-identify de-identified specimens without permission, to inform the public that such research takes place, and to educate them about its value, perhaps allowing those who still feel very strongly that they prefer not to be included an opportunity to opt-out.  But what has been actually proposed has more problems than what it would replace, and in fact, wouldn’t solve some of those it seems to be a response to.

Rebecca Skloot feels otherwise.  She is the author of a book called The Immortal Life of Henrietta Lacks, which chronicles the origin of one particularly important cell line – HeLa  – derived from cells that had been excised from Ms. Lacks in the course of a 1951 surgery to treat her cancer, and later used for research without her knowledge or permission.  Ms. Lacks was poor, uneducated, and black, and her descendants have also faced more than their fair share of adversity.  Ms. Skloot paints a compelling story of exploitation, but in my opinion, it is much more effective as a narrative about the horrible and enduring legacy of racism in this country than as proof that researchers who conduct secondary research with biospecimens without consent (as permitted under the current regulations, remember) or even without profit-sharing have behaved badly. After all, if individual risks are low and social benefits high – both true – then what’s the problem?  And it is far from clear that specimen sources deserve compensation for no other reason than that their discarded material actually proves valuable to scientists.  Nonetheless, the book has been used as a rallying cry by people from all walks of life who believe that they should be allowed to control whether, and potentially how, their specimens are used for research. Indeed, The Immortal Life of Henrietta Lacks is probably the single most important development that pushed the proposed revisions to the Common Rule forward, for the first time since they were released in 1991.

Read More

Bill Sage Webcast on Health Law v. Health Policy

As part of the Regulatory Science series at University of Arizona:
Health Law and Health Policy: A Frictional Account
William M. Sage, MD, JD, University of Texas
Today 12/2 — Noon (AZ Time) / 2pm Eastern / 11am Pacific
The talk will be webcast live, and available as an archive:

https://streaming.biocom.arizona.edu/event/index.cfm?id=26074

Participants in the live webcast will have the opportunity submit questions and comments.  Please do!

Wednesday Webcast: “Gene Patenting, Innovation Incentives, and the Future of Intellectual Property” by Derek Bambauer

By Christopher Robertson

This week, my colleague Derek Bambauer will speak as part of the Regulatory Science series at the University of Arizona.  Free CLE attendance form and readings are available.

Tune in at 12:00pm (Pacific) / 3:00pm (Eastern) on Wed Sept 16.

https://streaming.biocom.arizona.edu/event/?id=26071

The talk will also be archived at the same link.