By ANISH KOKA

Frances Oldham Kelsey by all accounts was not mean to have a consequential life.  She was born in Canada in 1914, at a time women were meant to be seen and not heard.  Nonetheless, an affinity for science eventually lead to a masters in pharmacology from the prestigious McGill University.  Her first real break came after she was accepted for PhD level work in the pharmacology lab of a professor at the University of Chicago.  An esteemed professor was starting a pharmacology lab and needed assistants, and the man from Canada seemed to have a perfect resume to fit.  That’s right, I said man.  Frances was thought to be a man’s name, and the acceptance letter accepted Mr. Frances Oldham.  Given the times, her Canadian mentor advised the young Frances she not write to inform the Chicago professor of the mistake but to simply sign the acceptance letter as Miss Oldham.  The rest, as they say, is history.  Ms. Frances Oldham arrived in Chicago in 1936, and just two years later was asked to work on figuring out what caused one of the worst poisonings in American history by the nascent US Food and Drug Administration (FDA).

The FDA at the time was a small organization within the federal government that had come into being a few decades earlier after the passage of one of many progressive laws passed to protect consumers from rapacious pharmaceutical companies of the day.  At the time there was no standard for claims that could be made to an unsuspecting public and no requirement that drug companies specify what ingredients were being consumed by the public.  The companies of the day would take alcohol, water and coloring mixed together – give the formulation a name, get a US patent, and make millions by heavily marketing testimonials of cures to all that ails directly to the consumer.  At one point, it was estimated that there was more alcohol being sold via ‘patent medicines’ than at liquor stores.

Sadly, it was not medical professionals that put a stop to this, but muckraking journalists like Samuel Hopkins Adams who exposed the seamy underside in a series of articles for Collier’s Weekly entitled The Great American Fraud.   Popular outrage followed publication of this series in 1905 and in response, the first draft of the FDA came into being by order of Congress in 1906.

The initial purpose of the FDA was a small, but important one – ensure the correct labeling of drugs being sold to the public.  The 1906 act – fought tooth and nail by industry – mandated that ingredients such as alcohol, cocaine, heroin, morphine and cannabis be accurately labeled with contents and dosage.  To understand the scope of the problem at the turn of the century, understand that Coca-Cola (Coke) was sold initially as a ‘patent medicine’ that was marketed as a cure for headaches, impotence, morphine addiction as well as a number of other ills.  The main ingredients?  Cocaine and Caffeine.  Current drinkers of Coke will be happy to know that cocaine was eliminated from the formula in 1903.

The FDA as we know it now is a result of the passage of the Federal, Food, Drug and Cosmetic Act (FFDCA) of 1938 that came as a reaction to national tragedy in 1937 that once again drove home the point that the public needed protection from private corporations.   The matter was a simple one that involved an ingredient that actually worked – sulfanilamide.  Sulfanilamide was an antibiotic that killed bacteria by preventing the synthesis of Folic Acid.  Humans are relatively primitive and don’t make Folic acid – so it isn’t toxic to humans.  That is, unless you’re the chief chemist of  S. E. Massengill Company, and you create a preparation of sulfinilamide that is dissolved in di-ethylene glycol (DEG).  DEG is toxic to humans – the first case reports of toxicity had been reported six years earlier.  Unfortunately the chemist, Harold Watkins, who worked for the company was unaware as this was not widely known at the time.  Watkins simply added some raspberry flavor to the sulfa dissolved in DEG, and the company founded by a Tennessee medical student who saw more opportunity in selling drugs to doctors than practicing medicine started distributing the drug widely.  No animal studies or any type of premarket testing was required at the time, and so the drug went straight from the lab to the consumer.  More than a hundred people died.

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By DAVID SHAYWITZ, MD

Randomized control trials – RCTs – rose to prominence in the twentieth century as physicians and regulators sought to evaluate rigorously the performance of new medical therapies; by century’s end, RCTs had become, as medical historian Laura Bothwell has noted, “the gold standard of medical knowledge,” occupying the top position of the “methodologic heirarch[y].”

The value of RCTs lies in the random, generally blinded, allocation of patients to treatment or control group, an approach that when properly executed minimizes confounders (based on the presumption that any significant confounder would be randomly allocated as well), and enables researchers to discern the efficacy of the intervention (does it work better – or worse – than controls) and begin to evaluate the safety and side-effects.

The power and value of RCTs can be seen with particular clarity in the case of proposed interventions that made so much intuitive sense (at the time) that it seemed questionable, perhaps even immoral, to conduct a study. Examples include use of a particular antiarrhythmic after heart attacks (seemed sensible, but actually caused harm); and use of bone marrow transplants for metastatic breast cancer (study viewed by many as unethical yet revealed no benefit to a procedure associated with significant morbidity).

In these and many other examples, a well-conducted RCT changed clinical practice by delivering a more robust assessment of an emerging technology than instinct and intuition could provide.

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By PRADHEEP J. SHANKER, MD

Sadly, the case of Alfie Evans came to a close this week, as he passed away in his hospital room surrounded by his parents. The debate over the medical ethics involved goes on.

Ultimately, there are extensive moral, philosophical, and medical issues involved with the policies over these cases. They are complicated, messy, and often times heart wrenching. But let’s put some misconceptions aside to begin with, some propagated by the most extreme and emotional participants in this debate.

Those of us that took issue with the handling of this case for the most part do not believe the doctors involved were evil, murdering individuals. There was no malicious intent from the NHS or physicians involved. I am sure the physicians meant well, from their point of view.

A second point: this was not a case about preservation of resources for the greater good. In this case, the parents had found alternative sources to fund the care they wished for their son.  So those arguing that we need to make such decision to prioritize money for those that can be aided the most is largely off target, and not relevant to the case at hand. I also don’t believe that the single payer system of the NHS in England inherently caused their mistakes; I think any system that is blind to its own deficiencies could lead to such mistakes.

That said, what were the issues that were in dispute here?

First and foremost, what was the ultimate intent of the care providers in this specific case?  Both sides basically admitted, early on, that Alfie’s prognosis was dire.  The reality is this child was likely going to die, and even the experts preferred by the parents readily admitted this in court documents.

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By ADRIAN GROPPER, MD

Seema Verma is right, US health care will be transformed if we empower patients and physicians through access to information. Don Rucker is right to focus attention on APIs to enable the transformation. A year and a half into the new administration and the massively bipartisan 21st Century Cures Act, the Department of Health and Human Services (HHS) is having to navigate between the shoals of highly unpopular Meaningful Use regulations and the apparent need for regulation to undo the damage of market consolidation that they caused. From my perspective, it looks like HHS is doing a good job.

Prediction is a dangerous game but it’s necessary for investments that depend on health information technology. Nowadays, pretty much everything in healthcare depends on information technology, particularly if we need effective quality measures to enable transition to value-based healthcare.

Based on Verma’s most recent remarks, it’s safe to predict that HHS will use the power of the $900 Billion purse as a way of avoiding regulation as it tries to break down the oligopoly of the consolidated “integrated delivery networks” and their even more consolidated EHR vendors. What’s more interesting is to anticipate how Rucker’s recent remarks about Persistent Access will be translated into decision support information for patients and physicians that will actually drive the practice innovation Verma is talking about.

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By NIRAN AL-AGBA, MD

Baltimore County, Maryland is one hour north of Washington DC, where politicians appear impotent to contain runaway healthcare expenditures.  In January 2014, the Centers for Medicare and Medicaid Services (CMS) in partnership with the state of Maryland, piloted an “All Payer Model,” where every insurer, including Medicare and Medicaid, paid a fixed annual amount irrespective of inpatient or outpatient hospital utilization.  Maryland agreed to transition hospitals from fee-for-service arrangements to this global capitation model over five years. 

Capitation, in general, reimburses a fixed amount per patient, unrelated to service volume.  This sets an artificial fiscal ceiling and disincentivizes hospitals, physicians, and other healthcare personnel to provide healthcare. The philosophy is if hospitals or physicians reduce their output and save money, the unused funds can be kept by the organization. The basic premise of capitation pays hospitals, physicians, and others to AVOID providing care, an unfortunate consequence. 

Maryland is experimenting with global capitation, which allots a fixed sum to an institution from each payer, making revenue predictable, while at the same time, encouraging stewardship by the hospital to allocate funds wisely.  When expenses are lower than the prearranged sum, that hospital retains the leftover funds as additional profit.  To ensure care is not withheld to increase revenue, quality measures are assessed and shared publicly.  A 2015 report in the New England Journal of Medicine showed expenditure reductions of 0.64% and inpatient admissions decreased by 5%.   However, with unproven payment arrangements, unintended consequences always occur. 

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By STEVEN FINDLAY

It’s now clear that two public assessments of President Trump’s health since 2015—the only ones we know about—were seriously compromised.   

The import of this has been eclipsed by other (more salacious) recent events—Stormy Daniels, etc.   But what has transpired raises troubling questions and should prompt a reassessment of how candidates for president and presidents are medically evaluated, and the public’s right to that information.     

I’ve written two pieces for THCB on Trump’s physical and mental health.  You can find them here and here. 

The first assessment of Trump’s health, conducted in 2015 by his personal physician of 35 years, Harold Bornstein, is now under a dark cloud.   Bornstein told CNN this month that Trump dictated the contents and language of a one-page letter signed by Bornstein and released publicly by the Trump campaign in the early months of the campaign.    

The letter aimed to assuage concerns about Trump’s age and health status.  Clinton and Trump were two of the oldest candidates ever to make a presidential bid and neither had shared much information about their health status up to that point; both were under pressure to do so.

“He dictated that whole letter. I didn’t write that letter,” Bornstein told CNN.  He had previously admitted (in August 2017) that he had typed the letter in his office in just five minutes while a limo sent by Trump waited outside.   

At the time, the four-paragraph letter seemed suspicious, to say the least.   It didn’t contain any details of test results or the like.   Instead, the letter made unusual and hyperbolic statements about the president’s health such as: “His [Trump’s] physical strength and stamina are extraordinary.”  And:  “If elected, Mr. Trump, I can state unequivocally, will be the healthiest individual ever elected to the presidency.”Continue reading…

BY MICHAEL MILLENSON

Trump appointees cheered by both Republicans and Democrats. Venture capitalists venting about too much investment cash. Data nerds decrying the deification of artificial intelligence.

For two days, Health Datapalooza 2018 offered a glimpse of a Washington where all sides work in harmony “to improve Americans’ health through better data,” in the words of Eric Hargan, deputy secretary of Department of Health and Human Services (HHS).

Not to mention the goal of improving health care economics. Enable digital health entrepreneurs to earn millions of dollars in profits, goes the logic, and their innovations will help the feds and others avoid paying many more millions of dollars in health care bills.

Health Datapalooza began nine years ago as a showcase for public-private data partnership. The shining example back then was the way the release government meteorological data had paved the way for online apps like weather.com. What was significant at this year’s event was not so much the sweeping rhetoric as the signals sent by HHS that it will accelerate the push by previous administrations towards value-based payment.

So, for instance, Seema Verma, the administrator of the Centers for Medicare & Medicaid Services (CMS), said CMS will ask private insurers and state Medicaid programs to require hospitals to provide patients with their own data electronically. The Medicare program wants to make that requirement part of the “conditions of participation” for hospitals in Medicare; i.e., do this or you can’t participate in the program that’s your largest customer.

“The expectations of CMS have changed,” said Verma. “Patients can never again be kept in the dark with regard to their health care information.”

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By GILBERT BENAVIDEZ

The Centers for Medicare and Medicaid Services (CMS) recently announced that Medicare Advantage (MA) plans could, in 2019, expand the health-related benefits they offer. In the announcement CMS wrote that it would

“allow supplemental benefits if they compensate for physical impairments, diminish the impact of injuries or health conditions, and/or reduce avoidable emergency room utilization.”

Such supplemental benefits could include things that are not normally thought of as “health care,” like, for example, groceries, air conditioners for beneficiaries with asthma, and even provider organized Lyft and Uber rides to and from and medical appointments.

While MA covers all Medicare services, MA plans are already permitted to offer extra coverage for supplemental benefits. Previously, MA supplemental benefits had to have a primary purpose of preventing, curing, or diminishing an illness. This ruled out those that might affect health outside the traditional health system, like groceries and non-ambulance transportation. CMS’s new regulation will permit such nontraditional MA benefits so long as they “increase health and improve quality of life.”

You may question why health care plans would include these types of benefits. The answer: If health is a puzzle, medical care is only one piece. The rest of the puzzle is filled in with pieces like environment, diet, and socio-economic status. CMS’s new regulation is intended to more directly address these social determinants of health.

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