Friday, December 9, 2011

Jeremy Shane: The Best of Times, and the Worst of Times, for Health and Healthcare

Editor's Note: Jeremy Shane is the President and COO of the Health Central Network, based in the Washington DC area.    Jeremy is a leader in both the business side of healthcare and also on the activist side.  So this guest piece reflects not only Jeremy's deep understanding of the industry, and the challenges and opportunities that it faces, but also his passion for Serious Medicine.   As he observes, only a Serious Medicine approach to Alzheimer's, to cite one urgent example, will save the US from economic ruination as future entitlement bills come due.  

Yet ultimately Jeremy sees more good news than bad news on the horizon; so count him as a the rarest of contrarians--a well-informed optimist.

The Best of Times, and the Worst of Times, for Health and Healthcare

A single page--page B4 of Thursday’s Wall Street Journal provides a wonderful synopsis of the state of the US health industry.  The page includes four articles: “Pfizer Cancer Drug Advances,”    “Justices Weigh Patents on Medical Tests,”  “Novartis Retools Brain Work,” and “Astra Cuts Sales Force in US 24%.”

Collectively, these articles paint a hopeful picture for health, but a very difficult outlook for health companies.  Great science will continue to excite and frustrate, scientific breakthroughs will test long-held regulatory approaches and law, and traditional models of drug development are in big trouble.

First, on the critical issue of curing serious disease, is the Pfizer article.  Wednesday an FDA advisory committee gave a unanimous nod to a new Pfizer drug, axitnib, for kidney cancer.   The FDA likely will give its final approval for the drug soon.  Clinical trials showed the drug prolonged survival for kidney cancer patients whose disease stopped responding to initial treatments.  There are two other treatments on the market for advanced kidney cancers.  Pfizer’s drug seems to work as well as the others, and better in some cases.  That’s the good news.  The challenge now is to figure out which types of kidney cancer where each advanced drug is certain to work better.  The only thing worse than a drug that doesn’t work, is a drug that that sometimes works and sometimes doesn’t, but in either case for reasons doctors don’t understand.  It means some patients will have false hope and lose time that could be spent trying something else that might-–or ideally would-–work.  So at the same conference where Pfizer announced results for axitnib, a new study from the PREDICT Consortium, a group of European researchers, was announced to see if different combinations of kidney cancer drugs could be associated definitively with specific tumor types.  That’s progress.

Yesterday’s articles also highlight how science and technology are eroding long-held regulatory approaches.  The FDA will approve Pfizer’s drug as a “second-line” treatment, meaning most patients will get it only after widely-accepted chemotherapy, surgery, or radiation therapies have been tried and have failed.  FDA rules do not prohibit doctors from using second-line drugs as part of an initial treatment plan, but because the drug wasn’t tested with “treatment na├»ve” patients before FDA approval, the choice to use it earlier is riskier for doctors and patients.  Ironically, the same research approaches making highly targeted drugs possible also suggest that tumors evolve once they are bombarded with chemotherapy, making them harder to kill.  Drugs used as second-line therapies might work better in combination with other drugs if used at the outset.  Or, they might not.  But right now, because of FDA rules governing clinical trial, drug manufacturers rarely test drugs on just diagnosed patients if a reasonably effective chemotherapy regime exists (reasonably effective meaning the chemo will shrink the tumor or slow its growth but is unlikely to kill it for good.)  Patients lose as a result.  Oncologists play the odds, more comfortable with tried-and-true though imperfect approaches, unsure in the absence of trial data whether using a second-line drug at the outset will improve survival.  And the specter of a malpractice suit if they try (and fail) cannot be far from the oncologist’s mind.  In time, research will win out, and it is possible that for many kinds of fast-growing or complex tumors, going nuclear as early as possible on multiple biochemical fronts is a surer path to total victory.

Yesterday’s story about the Supreme Court deliberations highlights how scientific and technological advances also are undermining long-held legal standards.  Patent law is intended to encourage new discovery, protecting breakthrough ideas while allowing others to benefit from publication of new approaches, enabling others to build on new discoveries (for a fee) in their own products.  Patent law has become murky in addressing new techniques to improve diagnosis, adjust drug doses, or determine individuals’ risk of disease based on their genetics.  Yesterday’s Supreme Court case, Mayo v. Prometheus, concerns a test that helps doctors pick the best dose for a Crohn’s Disease drug.  Tests like the one in Prometheus are at the heart of targeted medicine.  By definition, for a targeted therapy to be picked, doctors and patients must know what they’re aiming at, requiring sophisticated tests.  The question facing the Justices in this case and one expected next year, is: What makes a test patentable?  If a test merely detects substances that humans naturally produce, is it really creating something new?  What if the substance identified by a test is a rare mutation--it is a natural substance, but absent innovative techniques it might not be identified?  The answers to these questions could have a large impact on the pace at which new cures are discovered.  If patent law is too loose,  and too many patents are issued, patent-holders could extract monopoly prices and stifle innovation.  But allowing too few patents eliminates the incentive to risk money and time to discover new things, since there is no certainty that investment can be recouped.  Whatever the Supreme Court decides this year and next, it seems likely that health innovators will spend more time in court (like software and wireless phone companies) fighting over patents, diverting precious time and money from breakthrough science into law firms’ coffers.

Patent law may be about to become messier but the skirmish obscures the larger import of cases like Prometheus.  Whatever the law, science and technology are changing the way doctors and patients make health decisions.  Bit by bit, test by test, products like those offered by Prometheus are demystifying diagnostic and treatment choices.  As technology takes the margin of error out of previously subjective medical judgments, it will take the premium out of the cost doctors charge to make the diagnostic or treatment choice.  This is not to say all medical science can or should be reduced to a set of algorithms, though companies such as IBM, with its Jeopardy-winning Watson software, may try.  Doctors will continue to add a lot of value in their surgical skill and ability to manage difficult cases.  The body will remain a complex place to do business for a long time yet.  But the way medical professionals work is changing forever and for better.  The more that can be determined about a person’s medical condition objectively and precisely, the more consistent will be choices about treatment, and the more equitably can treatments become accessible to patients whether they are in a vaunted Memorial Sloan Kettering or a health clinic in rural Kansas.  

So, patent law disputes will over time become beside the point, even as they remain costly distractions.  We can hardly expect nine Justices, however learned, to solve a problem that-- regardless of their words--will be overrun by the discovery of scientific fact.  The high priests of the legal profession represent an industry marching ever-deeper into nuance and obfuscation as science marches in the opposite direction, towards clarity and consistency in making treatment decisions.

This brings us to the articles about Novartis and AstraZeneca and the breakdown of the Big Pharma business model.  Over the last twenty years, big pharma funded R&D on many disease areas with fat profit margins from a few multi-billion dollar blockbusters.  Once the FDA approved a drug, pharma companies sent legions of sales reps to knock on doctors’ doors, persuading them to treat patients with the newest compound.  This business model worked well for a while.  New medications came to market could be used by millions or tens of millions of people.  New treatments in many conditions like high cholesterol worked much better than older drugs, or even postponed costly surgeries.  Drug companies learned to create demand in conditions like erectile dysfunction where no real treatments existed, appealing to aging Baby Boomers’ anxieties about youthfulness and self-image.  Over time “lifestyle” drugs attracted elite scorn, but it is worth remembering that for a company like Pfizer, male angst over performance in bed funded research in niche categories like kidney cancer.  (Lilly, maker of the other leading ED drug, Cialis, also plowed profits into drugs for hard-to-treat conditions as well as more broader-scale areas like depression and chronic pain.)

Regardless, the days of blockbuster sugar daddies is over.  Big drugs, such as Pfizer’s Lipitor, are going off patent, crushing profit margins.  And “push” marketing, driven by salespeople pounding the pavement and knocking on doors makes less sense.  Today’s targeted drugs are used by fewer doctors, specializing in less-common conditions.  Even in oncology--a fairly broad specialty--there are only about 30,000 medical professionals in the largest cancer society, compared to the total universe of about 600,000 US doctors who prescribe drugs.  So companies like Astra Zeneca, who have not found many new targeted drugs, are in a bind.  Unable to support speculative research in multiple areas or justify large sales forces, big pharma is laying off marketers and researchers en masse.  Before Astra Zeneca, rounds of layoffs have happened at Merck, Novartis, Pfizer, GSK and others.  

There is another looming threat to American health and welfare that could produce a few blockbusters, Alzheimer’s Disease.  But AD has proven to be a vexing disease to understand.  Researchers are facing hurdles to developing “druggable” compounds, medications that work in predictable ways and can be delivered in consistent doses to deep recesses of the brain.  AD is likely to follow what we are learning about cancer and autoimmune disease.  That is AD will probably come to be described as Alzheimer Diseases, plural, with different varieties triggered by a range of genetic and environmental interactions, requiring a spectrum of combination therapies.  

Companies like Novartis which have tried and failed to develop Alzheimer’s killers now see that brain conditions defy resolution until we take a holistic approach to understanding brain disorders--degenerative ones like Alzheimer’s, developmental ones like autism, and behavioral ones like schizophrenia.  They are taking a step back to study brain anatomy, chemistry, genetics, and immunology, hoping that collaboration on core brain processes will mean many steps forward on multiple fronts.  Novartis, and a other pharma companies who remain interested in brain disorders, realize that even the largest “Alzheimer’s product team” focused solely on Alzheimer’s cannot hope to crack the “case.”  Brain research will be bigger than any company, any patent law, any FDA rule, bigger too than Obamacare or Ryancare since no insurance scheme from left or right can hope to cover the daunting cost of caring for people with multi-decade degenerative, developmental, and traumatic brain conditions.  Only great science and willing research participants--cures, in other words--can.

That, in a nutshell, is the state of the health industry today.  It is tempting to say it’s Dickensian, the best of times and the worst of times, but that gives too much credit to the down side.  Vibrant societies rely on creative destruction, objective truths, enabling new ways for innovators to profit, and citizenry to prosper.  Great science, or cures as most people think of it, requires perseverance and openness to new ways of thinking.  Rent-seekers and beneficiaries of now outdated regulatory schemes will be compelled by public pressure and scientific truth to step aside or join the fight.  If they get seriously ill, you can bet they will. 

Editor's post-script: A very important point is tucked away at the end of the paragraph about the Prometheus case: Now matter how the Supreme Court rules on that case, to the extent that cutting-edge science can be replicated, in the form of both treatments and tests, the problem of doctor-skill differential--which tends to disadvantage rural locales, and other under-served areas--is diminished.  That is, if the necessary intellectual capital is all captured in in the test or the treatment, then the skill of the doctor or healthcare provider is de-emphasized.   Doctors and other healthcare providers will always be vital, but if the test or treatment can be standardized and replicated on a mass scale, then everyone, everywhere, can get the benefit of the best that medicine has to offer.    And that's a good thing. 

Tuesday, December 6, 2011

All Visions Must Pass: Donald Berwick Departs the Centers for Medicare & Medicaid Services

Dr. Donald Berwick, now departed from his 17-month recess appointment as Administrator of the Centers for Medicare and Medicaid (CMS)--he was unable to win confirmation by the US Senate--took some parting shots at "waste" in the system in a New York Times interview. Berwick, who oversaw the spending of more than $800 billion in outlays, declared that as much as 30 percent of health spending is wasted. OK, we're all against waste, but who defines it? The federal government? Has Uncle Sam earned the credibility to make judgements about waste?

His valedictory interview notwithstanding, Berwick is probably best known, to admirers and detractors alike, as America's leading fan of Britain's National Health Service (NHS). As he said to an NHS audience in July 2008, "I am romantic about the NHS; I love it." And he went on to flatter the NHS in rapturous terms: "You are unified, movingly and most nobly, by your nation's promise to make good on an idea: the idea that health care is a human right.  The NHS is a bridge--a towering bridge--between the rhetoric of justice and the fact of justice."

The NHS does, indeed, represent a kind of pinnacle. It is the culmination of a belief system that originated in 19th century Germany. In the early part of that century, G.W.F. Hegel lyricized about the wondrous justice-giving powers of the "universal" bureaucratic state, not so different from the government of his Prussian homeland. Later in that century, another Prussian, Bismarck, reified and solidified Hegel's idealism into the practical reality of a bureaucratic welfare state; neo-Hegelians finally had achieved their utopian vision. For them, the welfare state became a secularized godhead, boasting the power to transubstantiate mere tax money into glorious and ennobling political structures. In the US, neo-Hegelians called themselves Progressives; the English word "Progressive" was inspired by the German Deutsche Fortschrittpartei, the German Progress Party, founded in 1861.

Of course, American progressives, yearning to enact their vision of modernization and uplift, were not inspired only by Bismarck, they were inspired also by the humming factories that improved productivity and generated prosperity. So Henry Ford, having mastered the assembly line, became a hero, as did Frederick Winslow Taylor, the pioneering "efficiency expert." Across the political spectrum, right to left, from the US to the USSR, "Fordism" and "Taylorism" were admired for their industrializing powers. For their part, American progressives reasoned that if factories were efficient thanks to Ford and Taylor, they could be made even more efficient without the "waste" of capitalist competition. And they further reasoned that people like themselves could make the whole nation more efficient. As John Dewey wrote in his 1935 book, Liberalism and Social Action, "Organized social planning . . . is now the sole method of social action."

So it made sense that healthcare, too, should be planned, modernized, and socialized. The Beveridge Report, produced by the British government in 1942 as the blueprint for the NHS, asserted that national healthcare should be seen as part of a "comprehensive policy of social progress."

Later in the 20th century, Dr. Berwick was swept up by the same progressive idea: planners would improve social welfare and, at the same time, eliminate waste. Berwick founded the Cambridge, Massachusetts-based Institute for Healthcare Improvement (IHI), which self-described itself as follows:

IHI was founded in the late 1980s by Don Berwick and a group of visionary individuals committed to redesigning health care into a system no longer plagued by errors, waste, delay, and unsustainable social and economic costs.

Berwick has been open in his admiration of such contemporary efficiency experts as W. Edwards Deming and of companies that have streamlined "just in time" techniques, such as Toyota. And the progressive healthcare dream has stayed steady now for a century; Berwick's declaration that 30 percent of healthcare spending is "wasted," for example, is perfectly congruent with Barack Obama's 2008 promise to eliminate waste and so cut a family's healthcare spending by $2500, or one-third. The promise still stands, of course, even if we are still waiting for the facts to catch up. Indeed, the lag time might lead some to conclude that perhaps the government is not the efficiency machine that Berwick and Obama might wish it to be. The progressive dream of enlightened management, it seems, will never die.

Some things have changed, to be sure, in the 100 years since Teddy Roosevelt ran for president on the Progressive ticket, promising, among other platform planks, national health insurance. For one thing, progressives have figured out how to profit from their progressivism; Berwick's IHI paid him more than $2.3 million in 2008. Indeed, such a fat paycheck is perfectly in keeping with the spirit of an age in which policy experts become rich as well as powerful. White House healthcare czarina Nancy-Ann DeParle, to name another, received $5.8 million as a consultant to health insurance companies in the three years prior to her entry into the Obama administration.

Yet at the same time, there can be no doubt that Berwick has full faith in the transcendence of what he has been doing. As he told the Times, "We are a nation headed for justice, for fairness and justice in access to care." Indeed, he continued, putting the cause of providing universal health insurance in grandiose terms: "There is a moon shot here." By "moon shot," Berwick meant a project that can grip the popular imagination the way it has gripped the imagination of so many Democratic Party intellectuals. Yet if most Americans don't see health insurance in such lustrous terms, well, that is indeed a problem for the latest generation of Hegelians.

In fact, Berwick lamented that the public hadn't yet grasped the greatness of the vision: "Somehow we have not put together that story in a way that's compelling."

One problem, perhaps, lies in Berwick's zeal for health rationing, seen as a necessary component of health justice. Yet zeal is not shared by most Americans. So when Berwick declared in 2009, "The decision is not whether or not we will ration care--the decision is whether we will ration with our eyes open," those words were widely used against him. But Berwick has, in fact gone even further, past the political point of no return:

Any healthcare funding plan that is just, equitable, civilized and humane must, must redistribute wealth from the richer among us to the poorer and the less fortunate. Excellent healthcare is by definition redistributional.

Alas, poor Berwick--we knew him too well. Whereas he saw government-run healthcare as taking us toward the light, others saw darkness. The NHS, for example, looks bad to most Americans with access to Google. Even the briefest search yields up headlines such as this, from the December 1 edition of The Telegraph, the UK newspaper: "Loved ones not always told their relative is on controversial 'death pathway'/ NHS doctors are failing to inform up to half of families that their loved ones have been put on a scheme to help end their lives, the Royal College of Physicians has found." It seems that tens of thousands of NHS patients are being put on what the Telegraph called the "death pathway," as a play on what the NHS calls--using a euphemism for euthanasia--the "care pathway." According to NHS rules caregivers [sic] are allowed to withhold care from terminal patients after receiving consent from the family. Yet a government audit found that some 2500 families, in the city of Liverpool alone, were not so consulted.

It's from reports such as this that some in the US get the idea that eliminating "waste" is code-talk for eliminating patients. So maybe "death panels" in the US aren't such a stretch, after all. That's why most American advocates of national health insurance tend to shy away from any comparison to the NHS. But not, as we have seen, Dr. Berwick, who has always been forthright in his NHS-philia. Most likely that's why he wasn't confirmed by the Senate; when his recess appointment to CMS expired, he had to go back to Cambridge.

No doubt Berwick will soon find a way to pass his vision on to a new generation, but after a century of progressive activism on healthcare, perhaps he--and all of us--might think about a new course of action. It seems that US healthcare advocates have hit the point of diminishing returns; after all, we have had mostly universal healthcare coverage for decades now. Medicaid and Medicare were created in the 60s, and EMTALA gave everyone the right to emergency-room treatment, without regard for ability to pay. Yet such piecemeal approaches did not meet Berwickian efficiency standards, it would seem, and so the Obama administration charged ahead with the Affordable Care Act, signed into law in 2010. And later that same year, the Democrats, of course, were clobbered in the midterm, and now "Obamacare" is still deeply under water politically.

So maybe the Hegelian-Bismarckian-Berwickian vision has played itself out. Maybe it's in the nature of the health-and-welfare state these days that it a) can't keep up with fast-fluxing market forces, b) can't keep up with the even faster changes in social networking and computerized transparency and publicity, c) can't withstand the onslaught of special interests, who honeycomb any kind of legislation with baroque and unpopular mandates and set-asides, d) can't come to grips with the reality that lawyers and judges end up running any public system these days, and e) can't comprehend the fact that the real problem with a disease such as Alzheimer's is not the financing of the disease but, rather, the disease itself. And therefore the whole idea of securing additional healthcare finance for the population is less important than the idea of securing the medicine to keep people healthy in the first place. The best way to eliminate "waste" in the healthcare system is to eliminate the need for so many people to be processed through the system.

We shouldn't be surprised if Berwick writes a book about his experiences in Washington. Here's a suggested title, although he probably won't use it: All Things Must Pass.