When is Rationing Ethically Accptable?

If I prefer a medicine that produces fewer side effects or marginally better outcomes, how much should you be expected to pay for my preference?

That's a question we in the US run away from. We'd rather say - "rationing is unethical! Period."

In truth, rationing happens all the time. The trend towards requiring us to pay a larger portion of our health care expenses out of pocket aims at making us responsible for our own rationing choices. If my physician recommends a CT scan "so we can be sure..." and I decide it's not worth the cost to me - that's self-imposed rationing. In our personal lives we make rationing decisions based on informal cost-effectiveness decisions every day.

But at the level of policy, acknowledging the need for ethically-grounded rationing is a third rail.

That's why the publication of Cost-Effectiveness of Long-Acting Injectable Paliperidone Palmitate Versus Haloperidol Decanoate in Maintenance Treatment of Schizophrenia is so important. for my field - psychiatry. In addition to providing valuable clinical information, the authors are admirably honest in presenting the rationale for potential rationing decisions.

Here's what the article is about:

Some patients with schizophrenia who benefit from antipsychotic medications follow their regimen reliably. But some don't, and for them a long-acting injectable antipsychotic medication can literally be a life-saver. I remember well a patient of mine who wouldn't take pills and wasn't keen on seeing a "shrink," but who accepted a monthly injection of haldol from his primary care physician. I made a serious joke with my colleague - "I may accomplish some useful things, but you are 'curing' schizophrenia in five minute appointments!" My patient and his family were enormously grateful that he functioned better and was happier than he'd been for a decade.

Paliperidone palmitate is still on patent and theoretically had some advantages over haldol decanoate, a much less costly generic medication. In a well-controlled randomized comparative effectiveness study, the authors compared the two medications, quantifying the differences between them in terms of quality adjusted life years (QALYs). Paliperidone had a slight advantage in terms of side effects, but at a cost of $500,000 per QALY, well-above what virtually every explicit discussion of QALYs has seen as an acceptable cost. Here's part of the author's conclusions:

The results of this study should encourage consideration of older, less expensive drugs, such as HD. Used at moderate dosages in this study, HD’s overall effectiveness and tolerability were only slightly worse...than those of PP, and it had clear advantages in cost-effectiveness...A rational policy for treatment of chronic schizophrenia might limit use of the more expensive [PP] to patients who do not benefit from or cannot tolerate HD. 

My colleague and friend Norman Daniels and I have written extensively about the overall ethics of rationing. I'm proud of the work we've done and believe it's useful. But progress in coming to grips with the need to ration care in a clinically grounded, ethically admirable manner will have to be done specialty by specialty in medicine, in concert with concerned members of the public.

The comparative effectiveness study of paliperidone and haldol illustrates four crucial steps that need to be taken for us as a nation to learn to set limits fairly:

1. Develop clinically and humanly meaningful evidence about key treatment choices.
2. Acknowledge the findings in an explicit, east to understand manner.
3. Do economic analysis to define the costs involved with the choice.
4. Decide whether the differences between the choices are worth the costs entailed.

The fifth and most difficult step is making use of these findings in the real world. In a health system based on competing health plans, health plan A would be reluctant to apply findings like these in its policies before health plan B does the same. If they did, word on the street would be "health plan A RATIONS CARE! How can we tolerate money grubbers like that in our health system?"

Learning to set limits fairly is more of a challenge to the heart than the head. At an intellectual level it's easy to see that limits are necessary. But thus far in the US, health system leaders, health care organizations, and the public have preferred to act as if rationing is evil and can be avoided.

That may have been true in the Garden of Eden. But, alas, that paradise vanished long ago.

Professional Societies and Stewardship in Health Care

Three imaging specialists from Johns Hopkins published a short but VERY important article - "Medical-Imaging Stewardship in the Accountable Care Era" - in the October 29 issue of the New England Journal of Medicine. (Unfortunately, the article is available free only to subscribers.)

The authors are leaders in imaging at Johns Hopkins. In their eminently practical article, they recommend that hospitals and medical groups designate internal experts to set standards for "appropriate" use of imaging studies. CT, MRI and other technologies are, arguably, the most important diagnostic advances in the last 25 years. When used "appropriately" they are the source of enormous benefit. When used "inappropriately" they are the source of high costs without concomitant benefit, and sometimes cause harm when incidental findings lead to unneeded biopsies or other interventions.

I put the word "appropriate" into quotes because in health management jargon it's used as if it's a statement of fact. In reality, it's a contestable judgment about value. The term, like its cousin "medical necessity," allows the health system and political leaders to pretend that we're just dealing with science, and not making value judgments about interventions and resource allocation.

Many health insurers have turned to radiology benefit management companies to oversee the use of imaging services. When this function is carried out well it applies evidence-based criteria to the ordering process and offers educational services to the clinicians whose orders they are reviewing. However, no matter how well the external review process is conducted, an unavoidable "us versus them" dynamic often emerges. In principle, self-management within a hospital or medical group is a preferable approach. Put simply, if it's good clinical care and "appropriate" resource allocation, we clinicians should be doing it ourselves, and not require external "disciplinarians" to enforce good practice.

What I just said is not a critique of radiology benefit management companies, but, rather, a reflection on a structural dynamic. In growing up, when we start to do the "right thing" on our own rather than depending on our parents to guide us, we're carrying out the same actions but in a more mature manner. That's what we should be doing as clinicians in our medical practices!

In "Medical-Imaging Stewardship in the Accountable Care Era," the authors are illustrating the kind of leadership professional societies and leaders within a profession can provide. Evidence based use of imaging and "appropriate" resource allocation are what we should be doing because it's the right way to provide health care. A true profession doesn't require or want health insurers to take responsibility for these core elements of professionalism.

The medical profession hated managed care when it emerged in the 1980s and 1990s. But if we had been managing ourselves in a clinically and socially responsible manner, external review would not have been needed. We left a vacuum. External entities then filled it.

Two teaching cases about unneeded MRIs

Readers who teach may find these two cases useful. But I think they'll be interesting for most thoughtful adults. 

CASE I.  Many years ago I wrote a hypothetical case for a session on rationing for medical students.

Tension Headache

You are a primary care physician at a not-for-profit health maintenance organization (HMO) that serves 500,000 members, paid for by capitation, a monthly premium paid to the HMO by the members’ employer, Medicaid, or Medicare. The premium revenues create a budget to care for the entire HMO population. 

Susan Jackson, a 28 year old office worker, consults you about headaches. For the past month she has had frequent headaches at the end of the work day. The headaches are much less frequent on the weekends. She describes the headaches as a dull, mild to moderate pain, that feels like tightness or a band around her head. She is otherwise healthy. Her neurological examination is normal. She identifies clear stressors at work.

You explain that this is a classical pattern for tension-type headaches and give practical advice on what to do. You predict that the headaches will improve as Susan learns to master the stressors and to build more relaxation into her day. Susan thanks you, but says that a friend who had headaches received an MRI, which showed a tumor that required surgery. With some urgency she says “unless you can guarantee that there is no possibility whatsoever that I have a tumor, I want an MRI. What harm can an MRI do?”

You know that the likelihood of a tumor is vanishingly low, and that established standards of care do not recommend imaging studies in a situation like Susan’s. There is no medical contraindication to getting an MRI, but you know that it would cost between $1500 - $2000, which would come out of the budget for the HMO population. You reflect on what to do, and how to explain your thinking about the MRI to Susan.

CASE II. Today I read the following case on the excellent "Costs of Care" blog:

Doc, I need an MRI

By Patricia Czapp, MD

“Doc, I need an MRI for my back.”

I recognized the voice immediately and turned to greet one of my favorite patients, Mr. P. There he was, smiling, leaning on his walker.

Mr. P visits me several times a day in my primary care office that is essentially in his living room.  The practice itself, sized fewer than 1,000 square feet, is on the first floor of a high-rise apartment building that houses disabled and low-income adults.

My team and I provide primary care to the residents of the building  (a public housing unit) and the surrounding community, a diverse population that has in common these characteristics:  social isolation, low health literacy and low general literacy, a high prevalence of behavioral health problems, and limited transportation.

We came to practice in the building because our health system, Anne Arundel Medical Center, several years ago noted a high number of ED visits from individuals of one address. We visited the address to meet the residents of the building and their landlord, the local housing authority.

We found a population of individuals who were aged beyond their years, suffering from preventable complications of chronic disease and for whom a visit to the hospital met medical as well as nonmedical needs…individuals like Mr. P.

Mr. P is a man living a marginalized existence. He thrives when people take the time to listen to him, touch him, and show him that they care. For many decades , he found this comfort in the ED. When his landlord agreed to try an experiment with us, we came to practice in his building.  Mr. P was one of our earliest patients.

We provide a low-cost alternative to meet his needs and do so with kindness, tolerance and generosity.

“What happened to your back, Mr. P?” I asked.  “Did you fall or hurt yourself”?
“No Doctor, I Just woke up, got out of bed and it hurt real bad for a while.  I could hardly stand up. ”

Rather than lecture him about the lack of medical necessity for an MRI, I accompany him to his modest apartment where we review the condition of his bed and mattress and suggest alternative ways to use pillows to support his back.   Mr. P beams, “Thank you so much.”  And then shuffles toward the Community Room.

If we had not been there to intercept Mr. P, he would have dialed 911. It shocks many to learn that individuals use the ED for nonmedical needs. But for some, this is the only way they feel human.

Our practice has been open for two years. In that time, we have experienced a significant decrease in medical 911 calls, ED visits, admissions and readmissions of residents of the apartment building. They have an alternative now to the ED, and we meet their social needs in their living room – one visit at a time, sometimes multiple times a day…
“Doc, I need a CT scan for my head.”
___________________________________________________________________
Patricia Czapp, MD was contestant of  “The Best Care, The lowest cost: one idea at a time” – a collaboration between Costs of Care, Healthcare Financial Management Association, Strata Decision Technologies, and Yale-New Haven Health.

"Tension headache" works well with medical students and residents. It ends with a resource allocation dilemma. Ms. Jackson wants a guarantee of absolute certainty, which is virtually never possible. The MRI is not required for good clinical care, but if  it cost $15-$20, not $1,500-$2,000, should it be covered because of her understandable anxiety? And, however one thinks through the dilemma, what should the primary care physician actually say to Ms. Jackson?

The second case is a beautiful example of ministering to one's patient - clinical practice as a calling. It's invites learning about "hotspotting" - meeting the real needs of people who are "overutilizing" expensive medical care. Mr. P was suffering, but ED visits were not the best pathway to relief. Dr. Czapp's remarkable practice has the potential to reduce costs even as it provides vastly better care for the population she serves.

Political candidates continue to bash their opponents by accusing them of health care rationing. Cases like these allow us to understand how cost reduction and frank rationing can be conducted in an ethically admirable, clinically sound manner.

Setting Limits Fairly in Singapore (2)

I wrote a post yesterday  before I led a workshop for ethics committees or hospital personnel simply interested in the ethics of setting limits in health care . Today I want to write about what I learned from the 25 wonderfully engaged folks I met with and from conversations subsequent to the workshop. Here are my initial observations:

1. One of the three core components of the framework for setting limits fairly that Norman Daniels and I developed is publicity or transparency. On the basis of our observations in the US, Canada, and the National Health Service in England, we argued that those responsible for setting limits (health plans, government agencies) should share the rationale for policies and decisions with the public. In liberal democracies in the west, this stipulation is widely accepted. My impression is that Singapore has more of a tradition of accepting governance by experts and judging their policies by results, without expecting the level of transparency, access and participation that is especially valued in the US. The group I spoke with suggested that the value we in the US place on transparency is not as strongly shared by the population and government in Singapore. To be useful, transparency requires a public experienced in understanding, evaluating, and interacting with policy makers, and policy makers experienced in a reciprocal role. My tentative conclusion thus far is that for transparency to contribute to improved policy making in Singapore, a period of learning or skill development will be required. I did not recommend a sudden shift in practice, but did suggest development of a strategy for how to engage the public in constructive interactions over time.
2. Physicians in the group told me that it's common for families to press for continued "treatment" even when the clinical staff has concluded that comfort care is the right approach. I asked them whether families doubted their clinical assessment or believed that a miracle might occur. Those weren't the reasons. They explained that there is a strong expectation of filial piety in Singaporean culture, and not pressing for continued active "treatment" felt disrespectful and even shameful to the family. They hoped that as advance directive conversations became better developed they will be able to talk with children about "what would your father/mother have wanted in this situation?" Their sense was that as is true in the US, most elderly people would want to shift to comfort care and not be "flogged" by fruitless interventions. Moving to comfort care could then be seen as the way to show true filial piety!
3. As part of our discussion of the family role in setting limits, a participant told us that because of family guilt if they feel they aren't doing "everything," talking with families about options that from a realistic perspective offer no benefit even though we can never say with absolute certainty that they won't be effective for the first time now can cause real harm to families. She described a situation in which a family sold its home to pay for an intervention that in practical terms was "futile."
4. Singapore health policy has been developed to minimize the risk of what they call the "buffet syndrome" - that full coverage creates "moral hazard" and will lead to excessive utilization. Their system of required savings to pay towards one's own (and one's family's) care is very cleverly engineered to confront Singaporeans with the cost implications of their treatment and, at the same time, to try to make sure that they have resources in their savings accounts to pay for their choices. But with an aging population, more chronic illness, and new technologies, middle class families are facing costs they can't reasonably meet. The group cited another instance where in order to finance valuable treatment a family had to sell its home and impoverish itself. This was consistent with what a taxi driver told me - "In Singapore you can die, but you can't be sick."

Because of its good population health outcomes and low public spending (between 4-5% of GDP) the Singapore health system has received great praise, especially from strong believers in the market and "skin in the game." But because of the high payment expectations it sets for its citizens it has been criticized by liberal believers in comprehensive coverage. From my meeting with the workshop participants, and from a subsequent meeting with officials in the Ministry of Health, I believe the praisers and the critics are both wrong. Singapore hasn't found a magic bullet that can be readily transferred elsewhere. But Singapore hasn't created a cold, uncaring system. They're struggling very thoughtfully with the problem every developed economy is struggling with - how to find the optimal combination of communitarian caring for the population with the individuals responsibility for his own health and for the cost of care for himself and his family. What I would praise and want the US to emulate is the combination of thoughtfulness, analytical clarity, and non-ideological deliberation about managing the dilemmas of modern health care.

Setting Limits Fairly in Singapore (1)

This afternoon I'm speaking to members of the ethics committee (and interested others) at Tan Tock Seng Hospital in Singapore. The hospital was founded in 1844 by Mr. Tan, a wealthy Chinese businessman, who donated $7,000 to found a hospital for the "diseased of all nations." It's a large hospital, with 460 emergency visits and more than 2,000 specialty clinic visits every day.

I've been asked to speak about "Accountability for Reasonableness," the framework for setting limits that Norman Daniels and I described in our book Setting Limits Fairly. From a combination of conceptual analysis and field work done in the U.S. at not-for-profit organizations like Kaiser Permanente, and in Canada and the UK, we concluded that health care limit-setting required a fair process, which the book explicated. The framework emphasizes the role of relevant reasons, transparency about policies and their rationales, and processes for appeal and revision.

The framework has been found useful in liberal western democracies like Canada, the National Health Service in England, and the Scandinavian countries that have universal coverage under publicly established budgets. Singapore is decidedly "first world" in its remarkable economic achievements since independence (1965) - its per capita GDP in 2011 was approximately the equivalent of US$50,000, and the unemployment rate was 2.1%. From talking with Singaporeans and reading, my impression of the society and its governance is that it is much readier to accept expert authority than the US and liberal western democracies.

The ethics committee has asked me to discuss the case of a middle aged man with severe rheumatoid arthritis, for whom a costly new medication has been recommended. In the US context there would be no question as to whether insurance would cover the medicine if the clinical reasoning behind the recommendation was sound. Any debate about coverage would be expected to be open to public scrutiny. From what I learned from my colleagues in rheumatology, a patient in his condition would expect to have access to the medication in the US, and would certainly expect  to be able to challenge a decision not to be given access.

But as a visitor here I'm acutely aware of what a terrible example the US sets as a health system. Our costs are strangling other important societal investments, and because of our out of control costs we've left 50 million to be uninsured. So while I'm proud of the work Norman Daniels and I have done on fairness, I'm agnostic about its relevance for another society with (a) different moral and political traditions and (b) excellent overall health outcomes. I'll be introduced as a visiting expert, but before meeting with a group that provides clinical care and is concerned with ethics, I feel more like a learner than like someone ready to pronounce the truth about setting limits in a society significantly different from my own.

ACOs, Rationing, and Medical Ethics

Magic bullets come and go in the world of health policy, but the accountable care organization (ACO) is likely to be a durable reform. Except for group practices like Geisinger, Harvard Vanguard, and Kaiser Permanente, systems like the VA, and local communities in which independent practitioners have established cooperative collaborations, health care has been grotesquely uncoordinated in the U.S. ACOs speak to that structural lesion.

Title III of the Patient Protection and Affordable Care Act puts ACOs forward as an innovation to promote the quality and efficiency of health care. "Efficiency" isn't controversial. Doing a second MRI because the results from the first one aren't available is pure waste. But not doing an MRI when a plain film, or a careful physical examination and history, could be "adequate," is more complex. Some would call that "efficiency" or "evidence based practice." Others would call it "rationing." But there's no uncertainty about how to label not offering an effective, and desired, service - that's definitely rationing.

In the October 6 issue of JAMA, Dr. Robert Brook of RAND has an important brief article - "What if Physicians Actually Had to Control Medical Costs?" As a thought experiment, Brook imagines that enough money is available for a physician to treat 100 patients with condition A or condition B. Treating each patient costs $1000, and only $100,000 is available. Epidemiological data predicts that the physician will see 100 patients with A and 100 patients with B. The benefit of treating A is four times the benefit of treating B. What should the physician do?

Brook believes, and I agree, that even when we have wrung all efficiency savings out of health care, there will still be beneficial interventions physicians want to provide, and patients want to receive, that our society will not be prepared to pay for. He argues, and I agree, that the medical profession is unprepared for engaging with this eventuality:

Policy makers discuss controlling medical costs, and academics publish articles analyzing cost-control approaches. But physicians seem oblivious to the possibility that, sooner or later, care will need to be explicitly rationed. Physicians who actually order the health-related diagnostics or treatment for which taxpayers pay must decide how they will cope with explicit rationing. Will there be a physician plan or health professional plan to deal with the eventuality of explicit rationing? Should planning begin now instead of waiting until the decision is imminent?

There's no way that the need for rationing could have been part of the federal health reform process. We're not yet mature enough as a body politic to deal with that piece of reality without going ballistic about "death panels." But wishful thinking and political immaturity don't change the fact that rationing happens now, will have to be acknowledged in the future, and is an ethical requirement, not an abomination. Brook concludes, and I again agree:

...an explicit plan for rationing needs to be developed. But who will do it, and how?

Physicians (and other health professionals), patients and the wider public should be the "who?" And with regard to "how?," the experience of the Didcot practice in the U.K. provides a model of deliberative process. With help from two ethicists, the general practice group created guidelines for the practice, reviewed them with members of the practice, and made them public.

No one wants to go first in discussing rationing. To do so would invite savage, know-nothing political attack. But it's important for public deliberation about how to ration in a clinically guided, ethically justifiable and potentially socially acceptable manner to get underway. Accountable Care Organizations are an ideal setting for physicians and patients to join together in this kind of what if scenario planning.

In Defense of "Bean Counters"

I've been speaking about health reform and managed care in different venues around the country. With regard to cost containment I often hear the phrase "bean counters" - as in "Do you want bean counters controlling your life by reducing your medical care to a set of numbers?"

This post is in praise of the much abused act of "bean counting."

When I first began to practice at the not-for-profit Harvard Community Health Plan HMO in 1975 I had a young patient with severe alcoholism. While drunk he got into a fight and was admitted to a psychiatric hospital. I'd been in charge of a psychiatric hospital unit before coming to the HMO and I knew that my patient would be better served by an alcohol treatment program, which would also, it happens, be cheaper. That's the basic definition of win/win.

But when I called the attending psychiatrist about my patient, to urge discharge to my care, I heard him say to the chief of the unit, who had been a teacher of mine during residency - "I have Sabin on the phone...he used to care about patients, but now he cares about money!"

I was really p - - - - d off by the comment, but it led to many years of thinking about how our health system can do a better job of caring about patients and caring about money. (See here for an article I wrote with that title 15 years ago.) I've come to use a framework that encourages all participants in health care to embrace two core values – compassionate fidelity to the needs of individual patients and responsible stewardship of the resources we place in public and private insurance pools.

Fidelity is about individuals. Stewardship is about populations. These two values are the pillars of an ethical health system.

Fidelity - the commitment to seek benefit for the patient and to avoid doing harm - has been at the heart of medical ethics since the time of Hippocrates. As long as health care was a transaction between a single patient and a single physician paid for by the patient’s own funds (or by barter), there was no need for insurance. Population concerns and stewardship were not important considerations.

But as health care became more complex and vastly more expensive societies created insurance mechanisms through which people pool funds to help them pay for care when they need it. In countries like Canada and England, where coverage is universal and paid for by taxes, the ethical importance of stewardship is easy to understand. As important as health care is the public has many uses for its funds. These societies see responsible stewardship as a moral requirement

In the complex U.S. health system it is much harder for the population and its leaders to understand how insurance funds are created. As a result, our society has not recognized stewardship as an ethically justifiable process. That's how we've come to demean attention to the cost of care as “bean counting.”

In the mind of the public (and some political leaders) “stewardship” is often confused with “rationing.” In health care, rationing refers to withholding beneficial services, such as very expensive cancer therapies, because of their cost. In reality, stewardship is the best way to avoid a need to ration! Responsible stewardship by insurers involves (a) seeking elimination of interventions that are not needed but can cause harm (as by side effects), (b) substituting equally effective but less costly interventions, such as generics for branded drugs, or prudent watchful waiting for doing a costly but unnecessary test today, and (c) careful contracting. The more insurers do (a) - (c) the less need society will have to (d) ration - in the sense of deliberately choosing not to provide interventions likely to be beneficial.

Stewardship isn't just a job for public and private insurers. It's best seen as a responsibility for all who are concerned with health and health care. As individuals we are stewards of our own well-being, even if most of us do a poor job. The provider community - physicians, nurses and other clinicians - is in the best position to differentiate between efficient and inefficient health care and to provide what patients need in the right way, at the right place, at the right time. And the employers who pay for insurance would rather invest more in employee wages and research and development than in ever-increasing health care costs.

Ideally, the health reform process would move our society along a learning curve towards better understanding and appreciation of the moral importance of stewardship. But so far the voices changing "bean counters - up against the wall!" have been the loudest.

Why Comparative Effectiveness Research is so Important

I'm on the road now, and yesterday gave the talk I wrote about (here) last week. (I'm not writing about the venue of the talk since it was an internal meeting, not a public session.)

I came away from the event convinced more than ever that a robust program of comparative effectiveness research (CER) is the crucial next baby step for improving the value of what we in the U.S. health "system" do in health care and making the topic of health care costs less toxic. Sadly, the challenge for us in the U.S. is to begin to deal with runaway costs in a serious fashion rather than waiting for the tooth fairy or the Wizard of Oz to make the problem go away. (See "Cost Control: How Incapacitated are We?" by Paul Menzel on the Hastings Center Health Care Cost Monitor for an analysis of the almost total collapse of meaningful cost containment in the health reform process.)

CER studies compare alternative approaches to treating the same condition. It's most straightforward when the comparison is between drug A and drug B, but in principle we can compare a drug to, say, meditation or exercise, as well. The legislation that is moving through Congress is careful to insist that CER cannot be used to manage care or drive insurance coverage. Drug companies are terrified at the prospect that CER will deflate their claims about "me too" drugs. Medical specialists fear that a sham treatment may equal or outperfom their favorite procedures, as has happened in prior research on surgery. It's a fairly safe prediction that the first thing that will happen from CER is....very little.

CER dramatizes the fact that much of what we do in health care is based on faith, not evidence. It treats our beliefs as hypotheses which may be correct but could be off the mark. For healing to occur we must have faith in our doctors. The more evidence our doctors have about what works best the more that faith is warranted and will be rewarded.

Because it's so obvious that when two approaches are equivalent there has to be a VERY good reason for not choosing the less costly alternative, CER will gradually shake us out of the reflexive U.S. attitude that costs should not be considered in delivering care. It's best to see the health reform process as a first step, not a "solution." If Congress can fashion a bill that the President can sign we'll have demonstrated that our fractious and wildly irrational political process can engage with health care. It seems certain that whatever emerges will be profoundly imperfect. But if the body politic and the legislature comes away from the reform process with more confidence about tackling health care, we'll be in a better position to learn from the flaws in what emerges and to take some wiser next steps.

That's where CER comes in. It enacts a scientific approach to a realm that is increasingly dominated by advertising and economic interests. The first useful impact will be when insured folks say "let's bring premiums down by doing what works best at the lowest cost" and taxpayers say "let's put some teeth into Medicare by using the results of CER." Vested interests have blocked these steps in the legislative process so far. But if the public, and our political leaders, see more facts emerging from CER, it will be harder for PAC contributions and advertising flim flam to drive health care.

Can we Discuss "How Much is Life Worth" without going ballistic?

I've been invited to speak next week at a conference based on the article by Tito Fojo and Christine Grady ("How Much is Life Worth: Cetuximab, Non-Small Cell Lung Cancer, and the $440 Billion Question") that I wrote a post about three weeks ago. The opportunity has led me to a clearer perspective on the currently undiscussable topic of health care rationing.

Fojo and Grady's central argument - that we in the U.S. should rein in our expenditures on interventions that produce small benefits at enormous cost is so obviously correct that the question to ask is not "are they right?" but rather "what's preventing us from doing what we so obviously need to do?"

I've thought about that question in light of the decision process that NICE (National Institute for Health and Clinical Excellence) uses for the guidance it provides to the National Health Service. NICE has created an elegant approach to setting limits. The logic of NICE's process is simple. (1) The National Health Service has a budget. (2) Every expenditure has an an opportunity cost and should be compared to alternative uses of the funds. (3) For selected new interventions, NICE reviews the evidence about its clinical effectiveness. (4) If the intervention offers clinical benefit NICE asks how much those benefits cost, defined as cost/QALY (Quality Adjusted Life Year). (5) NICE applies a template of social value judgments to the facts that emerge from its analysis. (6) The template includes a threshold range (20-30 thousand pounds/QALY) to guide decisions and a framework for deciding when and how to make exceptions to the threshold. (7) The entire process is conducted in a public manner, with opportunities to see the reasoning behind the conclusions and to raise challenges.

NICE fine tunes its approach over time, but its fundamental logic is sound. The impediment to us in the U.S. doing what Fojo and Grady urge us to do is not the absence of a method for deciding about the worth of marginal benefit. Any country ready to tackle rationing could take NICE's procedures off the shelf, study them, and adapt the process to its own culture and institutions.

The key word here is "ready." And, as Hamlet told us, "readiness is all!"

Experienced psychotherapists teach that the hardest job in treatment is dealing with the resistances to change. Once these impediments have been worked with well enough change almost takes care of itself. That model applies at the level of social process as well.

In my talk I'll take Fojo and Grady's argument as the starting point. They're obviously right. The folks at the conference are predisposed to agree - that's why they invited me. I'm going to suggest that making decisions about marginal benefit is, at heart, not all that difficult. NICE shows us how to do it. The task for all those who agree with Fojo and Grady is not to persuade others to see the same truth, but to chip away at the impediments to seeing the obvious. I'll write more about chipping away impediments in future postings.

(See here for NICE's Social Values Judgments report and here for reports from the Citizens Council.)

How Much is Life Worth? - An Oncologist's Call to Arms.

Between 1997 and 2004, Medicare spending on cancer drugs rose by 267% compared to an overall rise in Medicare spending of 47%. With an aging population, the cost of cancer drugs will become a progressively greater problem for the U.S. health "system."

A recent issue of the Journal of the National Cancer Institute has a very important article by Tito Fojo, an oncologist at the NCI Center for Cancer Research, and Christine Grady, an ethicist with the NIH Department of Bioethics. "How Much is Life Worth: Cetuximab, Non-Small Cell Lung Cancer, and the $440 Billion Question" calls for a fundamental change in research, the drug approval process, drug pricing, and clinical practice. The analysis and argument will be familiar to ethicists and policy wonks. What's especially important about the article is that Dr. Fojo speaks as a distinguished cancer researcher and addresses his recommendations to fellow oncologists.

Drs. Fojo and Grady don't flinch from using the "c" word - cost:

In some sense, every life is of infinite value, and we naturally avoid confronting the tension between not wanting to put a value on a life and having limited resources. But the spiraling cost of cancer care in particular makes this dilemma inescapable. We, the oncology community, cannot continue to ignore it.

The main example in the article is Cetuximab (Erbitux). Erbitux adds 36 days of life for patients with non-small cell lung cancer, but also adds a number of significant side effects. Conveniently, the study that demonstrated the 36 day extension of survival did not include quality of life measures. At its present cost, adding an average of 36 days of life is the equivalent of $800,000 per non-quality adjusted year of life. Fojo and Grady argue that this is far too much cost for far too little benefit.

The authors urge oncologists to act as true professionals who consider wider societal concerns like the uninsured or the U.S. economy going down the tubes. They don't use the word "narcissistic," but their analysis suggests that a profession that considers only its own perspective is just that. They make six specific recommendations for practice and policy:

1. "Research studies that are powered to detect a survival advantage of 2 months or less should only test interventions that can be marketed at a cost of less that $20,000 for a course of treatment." Here they are using the standard of one quality adjusted year of life for patients treated with dialysis ($129,090) as their standard.

2. Drugs should be priced accordingly.

3. "Drugs shown to be active in one subset of patients should be advocated, approved, and prescribed for that subset only. The marginal benefit, if any, which may be achieved in other patients should not be an excuse to administer a therapy even if it is decided that there is nothing further to be done."

4. FDA approved indications should be strictly adhered to.

5. "The all too common practice of administering a new, marginally beneficial drug to a patient with advanced cancer should be strongly discouraged. In cases where there are no further treatment options, emphasis should be first on quality of life and then cost."

6. "For therapies with marginal benefits, toxic effects should receive greater scrutiny."

Having exposed the chemotherapeutic emperor's new clothes for what they are, Drs. Fojo and Grady conclude:

We must deal with the escalating price of cancer therapy now. If we allow a survival advantage of 1.2 months to be worth $80 000, and by extrapolation survival of 1 year to be valued at $800 000, we would need $440 billion annually — an amount nearly 100 times the budget of the National Cancer Institute — to extend by 1 year the life of the 550 000 Americans who die of cancer annually. And no one would be cured.

The current situation cannot continue. We cannot ignore the cumulative costs of the tests and treatments we recommend and prescribe. As the agents of change, professional societies, including their academic and practicing oncologist members, must lead the way. The time to start is now.

Drs. Fojo and Grady have issued a courageous and compassionate challenge to their fellow medical professionals. It deserves wide attention!

(The article is not yet available without charge, but a summary and paid access to the full text are available here. For Dr. Fojo's web page see here.)

Immigrant Health and Heart Transplants

I'm still jet lagged from 2 1/2 weeks in Europe, so when I woke up in the dark this morning I sought out the online New York Times for solace. Two articles yielded a larger story about health reform.

Eric De La Cruz was a student and part time disc jockey and graphic designer in Las Vegas in his early 20s when the symptoms of progressive cardiomyopathy - weakness of the heart muscle - began to cause symptoms of heart failure. The condition steadily got worse, and it became clear that the only hope for Eric would be a heart transplant. But neither of his employers provided health insurance. And, surprisingly, when he applied for Social Security disability benefits, which would have made him eligible for Medicare, he was turned down.

Eric did qualify for Medicaid, but in Nevada Medicaid covers heart transplants only up to age 20. At this point his sister began a Twitter-based effort to raise money, and within two weeks $1 million had been offered! Eric went to the University of Southern California for evaluation, but his condition was too far advanced, and he died on July 4.

The second article was from Massachusetts. The recession-driven shortfall in revenues and the requirement that the state stay within its budget necessitated cutbacks in the state's health reform program. (The program has led to the lowest uninsured rate in the U.S. - 2.6% compared to a national average of 15%.) The legislature decided to cut most of the $130 million dedicated to 31,000 legal immigrants. But by scrounging up some additional funding and developing a stripped down set of benefits, a relatively comprehensive insurance package will still be available to the legal immigrant group. It won't cover dental, hospice, or skilled nursing care, and will require higher copayments for non-generic drugs, but it would have covered a heart transplant for someone with Eric Cruz's condition.

After his death, Eric's sister Veronica De La Cruz said:

If my brother had been able to buy health insurance, he would never have been in this situation. No one should ever have to go through what we’ve been through. Eric should still be alive.

Veronica is half right. In a wealthy country like the U.S., a healthy young person like Eric should be eligible for a heart transplant. A heart might not be available, but that would be a tragedy, not an injustice.

Ben Kieckhefer, a spokesman for the Nevada Department of Health and Human Services, described the the state's policy clearly and honestly:

All insurance plans decide what things they are going to cover and what things they are not going to cover. Everyone in the state Medicaid program recognized the difficult situation the family was in, but we can’t just start covering something for one individual case.

Unless there is some form of universal or near-universal coverage, as through a single payer system or a requirement that individuals purchase insurance (as in Massachusetts) Eric would not - and should not - have been able to buy insurance. If we allow ourselves to wait until a need appears before we get insured, free-riding would be the rational choice for the individual to make, and insurance costs would be stratospheric in no time.

Affordable insurance depends on social solidarity, with the healthy contributing more than they receive and the sick receiving more than they contribute. In the U.S., however, talking about "solidarity" invokes fear of the other dreaded "S" word - "socialism."

And as the stories from Massachusetts and Nevada show, viable insurance also entails the equally dreaded "R" word - "rationing." Health systems must set limits. A society (like the U.S.) that refuses to ration in a thoughtful, ethically-guided manner, will (like the U.S.) end up rationing in a thoughtless, unethical manner. Whether a society prefers to limit high cost/high tech interventions as Nevada has done, or more mundane but nevertheless valuable services as Massachusetts chose to do, is the kind of policy decision that requires thoughtful democratic deliberation. Sadly, thoughtful deliberation is currently being drowned out by strident advocacy, pervasive misunderstanding, and lies.

(To read more about Eric De La Cruz see here. For the Massachusetts story, see here.)

American Values and Health Reform (3): Medical Progress

This is the third in a series of posts based on “Connecting American Values with Health Reform,” a publication of eleven short essays from The Hastings Center. The aim of the project was to consider what we want health reform to accomplish and to suggest what values our institutions and practices should be built upon, based on the belief that more clarity about underlying values would allow legislators and the public to handle reform more effectively. (See here and here for the two previous posts)

I hope legislators and media folks read Dan Callhan's essay on "Medical Progress: Unintended Consequences." The content will be familiar to those who have followed Callahan's work, but the short piece is provocative, clear, and easy to follow. It provides an ideal basis for a book club type discussion.

Callahan takes off from the observation that "a powerful faith in science as a basic human value, matched by an equally strong belief in medical progress, has been a central feature of American culture from the start." He acknowledges that we are "healthier and more prosperous because of it," but notes that the intensity of our faith in medical progress has put it off limits for ethical inquiry.

With that background Callahan lays out a series of concerns about the policy implications of our unquestioned faith in the value of medical progress:

1. New technologies or intensified use of older ones account for 50% of our annual health care cost increases. "Our technological benefit is turning into our economic bane." Callahan argues that we should do more assessment of cost-benefit ratios and make choices about what we want to pay for.
2. Much of the improvement we have achieved in health and longevity comes from improvements in socioeconomic factors - perhaps 60%. Callahan argues that "one could make a good case that improvements in education and job creation could be a better use of limited funds than better medical care."
3. Given his view that "throwing technology at illness in the name of progress is an increasingly expensive and economically destructive way to go," Callahan urges us "to aim for a better balance between cure-oriented and care-oriented medicine."
4. With regard to prevention, Callahan takes a very tough stand: "the only way to assure a good outcome for prevention programs is to make clear to the public that high cost technologies will be severely limited when the final illness comes. The carrot is that prevention will give us a longer life with a higher quality. The stick will be the message that you should take care of yourself and not expect medicine to save you when your time runs out."
5. As an extension of his comments about prevention, Callahan engages directly with longevity and mortality: "Americans already live, on average, a long life of seventy-seven years. There is no need to go out of our way to chase life extension, or the denial of death, as the sine qua non of medical progress. We need progress in removing the health disparities that keep millions from reaching seventy-seven..." Coming from a younger person this perspective would elicit a torrent of ageism accusations. Coming, however, from someone who, if online information is to be believed, will be 80 next July, Callahan's view may receive the thoughtful and respectful attention it deserves.

Callahan's tone is moderate, but his conclusions are radical:

Serious progress would mean turning back the clock: learning to take care of ourselves, to tolerate some degree of discomfort, to accept the reality of aging and death, and to see our personal doctor as someone as likely to talk with us as to have us scanned. That cluster of backward-looking ideas is what I think of as common sense, affordable progress.

I think most people who have given serious thought to the health system, including conservatives and people of faith, will agree with much of what Callahan says. But in the frenzy of anti-government sentiment that Republicans are fomenting, Callahan's views may elicit an "Aha - they really are planning for euthanasia" reaction from the Rush Limbaugh crowd. The level of political hysteria and public paranoia is so high that even a great communicator like the President would have a lot of difficulty working with Callahan's wise perspective.

Should President Obama come out of the Closet on Rationing?

An editorial in yesterday's Washington Post challenges President Obama to come out of the closet about rationing and fess up to the American public about the need for limits and sacrifice. Here's the essence of the argument:

PRESIDENT OBAMA sometimes presents health-care reform as a pain-free proposition, as simple as choosing the red pill over the blue -- one that's no more effective but costs twice as much. Asked at his news conference whether "the American people are going to have to give anything up in order for this to happen," Mr. Obama's basic answer was no. "They're going to have to give up paying for things that don't make them healthier," he said...

But Mr. Obama's soothing bedside manner masks the reality that getting health costs under control will require making difficult choices about what procedures and medications to cover. It will require saying no, or having the patient pay more, at times when the extra expense is not justified by the marginal improvement in care. Mr. Obama is right that sticking with the status quo is a bad alternative, but he isn't leveling about the consequences of change...

The current system is untenable and getting worse, with employers dropping insurance and premiums rising for those who still have it. Reform is essential. But Mr. Obama does the public a disservice by acting as if it will not require anything from them in return.

Of course the editorial is correct about the need for rationing. But is its conclusion good advice for political leadership?

On May 13, 1940, Winston Churchill spoke to the British Parliament as the new Prime Minister. He didn't mince words about the need for sacrifice. Perhaps the Washington Post has Churchill in mind in their advice to President Obama:

I have nothing to offer but blood, toil, tears, and sweat. We have before us an ordeal of the most grievous kind. We have before us many, many months of struggle and suffering.

You ask, what is our policy? I say it is to wage war by land, sea, and air. War with all our might and with all the strength God has given us, and to wage war against a monstrous tyranny never surpassed in the dark and lamentable catalogue of human crime. That is our policy.

You ask, what is our aim? I can answer in one word. It is victory. Victory at all costs - Victory in spite of all terrors - Victory, however long and hard the road may be, for without victory there is no survival.

Let that be realized. No survival for the British Empire, no survival for all that the British Empire has stood for, no survival for the urge, the impulse of the ages, that mankind shall move forward toward his goal.

Metaphorically, the U.S. is facing the same situation the Brits were in 1940. Our public dream of a Garden of Eden in which all pain and illness can be banished is a "tyranny" that threatens the survival of the country as we know it. But the threat posed by an economic cancer does not galvanize society the way an external enemy does.

So how can the President align the ethics of honesty with the requirements of political effectiveness? If I were advising him I'd argue for statements like this:

My fellow Americans. Our country is facing a grave and devious threat in runaway health care costs. Health care costs are a form of virulent, but potentially treatable cancer. If we let the status quo continue health costs will strangle our economy and rob us of our strength. Al Qaeda's best strategy for weakening us would be to induce us to continue with the status quo...I know that many are afraid that change means rationing. They're 100% right to be concerned. Every other country in the world has chosen to ration health care. Unless we tackle avoidable waste we'll have to do the same thing. Making large changes can be frightening, but we have not been a nation of cowards...

President Obama needs to invoke an external enemy more effectively than he has done thus far. As wrong as they are, that enemy isn't the Republicans or the Blue Dog Democrats - it's the economic cancer of the status quo. His task is made difficult by the fact that the enemy isn't a foreign power - it's our own cowardice about facing facts in the arena of health care. As Pogo said in the 1950s - "We have met the enemy...and he is us!"

To move health care reform forward President Obama needs to channel Winston Churchill more effectively. If he doesn't create more of an external danger we'll just continue to fight with each other. That dynamic has scuttled every previous effort at meaningful reform.

Peter Singer on Rationing

In yesterday's New York Times magazine, Peter Singer presents a patient, lucid explanation of the ethical and economic necessity for health care rationing. (See here.)

If you've read any of my 37 previous postings on rationing you know that I see rationing as (1) obviously necessary, (2) done every day in our health "system," (3) mandatory for ethical health care in the 21st century, because (4) there are more potentially beneficial interventions available than we can afford. As Peter Singer puts it:

When public funds subsidize health care or provide it directly, it is crazy not to try to get value for money. The debate over health care reform in the United States should start from the premise that some form of health care rationing is both inescapable and desirable. Then we can ask, What is the best way to do it?

Given the undeniable truth of Singer's conclusion, how do we account for the steady drum of histrionic rhetoric warning the public that health care reform will mean "rationing"? What comes to mind is Schopenhauer's famous comment on an arcane philosophical argument:

As a serious conviction it could be found only in a madhouse: as such it would then not need so much a refutation as a cure.

I don't believe that the conservative legislators and commentators who warn about rationing are mad. I assume they know that their pretense that rationing is evil and will only occur if President Obama gets his way is nonsense, and are simply playing politics. Alas, the "rationing" accusation is effective politics, and the disinformation it embodies contributes to the madness of our national discourse on health care.

So how can that madness be cured?

Lucid argument of the kind Singer makes is useful, if only to firm up the resolve of the choir to keep singing. But to move the general public we'll need a combination of political leadership and time. If I were a political advisor, which I'm not, here's the line of thinking I'd recommend:

1. Our health system rations now by not insuring close to 50 million and spending so little on primary care that many well insured folks can't get to a personal physician.

2. We know that at least 30% of what we spend on health care is unnecessary and possibly harmful.

3. We need to reform the system so everyone has insurance and can get good care. That reduces the amount of rationing that is going on.
4. We need to wring out the unnecessary clinical and administrative expenditures. By doing that we reduce the potential need for rationing.

5. By taking no action and allowing runaway costs to continue we ensure that more and more poorly thought out rationing will occur, as occurs at present.

6. In case rationing is ever necessary in a reformed health system, let's tip toe up to thinking about how it could be done in the wisest and most ethical manner.

There's so much to do to capture the low hanging fruit of clinical and administrative waste that trying to focus the public on the need to ration is a distraction. Better to use the spectre of rationing as a prod to ourselves and the health system to remove our enormous expenditures on things that aren't needed before we engage in the truly difficult debates about what beneficial interventions we will forgo!

Letter to the Massachusetts Legislature

Dear Massachusetts Legislators -

This fall you will be considering whether to put the Payment Reform Commission's recommendations for moving health care to a system of global budgets into law. (See my post from July 17 for the story.) This is an opportunity we should seize. If we do, we'll rescue our own health care reform process and we'll help the national reform dialogue get real about controlling runaway costs. Here are some of the key reasons for supporting the Commission's recommendations:

1. Cost control. President Obama has told the U.S. public that containing runaway health costs is a moral imperative. We're starting to understand that health care costs are strangling the economy and way out of line with what other countries spend for equal or better results. Cost containment will not happen without true budgets for health care. There is simply no other way.

2. Mature discipline. Learning to live within our means is a key element of becoming a responsible adult. The recession we're in is teaching us a lesson we should have learned in growing up - no matter how many credit cards we have, there's no free lunch! Budgets for health care reward us (patients, the public and the medical community) for using our brainpower to live within the means that society decides to allocate to health care.

3. Reduced waste. Over the years I've asked colleagues I respect most in every medical discipline how much could be saved without loss of quality if they were in charge of their area of medicine. No one ever said less than 25% and a good number said 50%. Research supports the idea that we can remove at least 30% of what we spend on medical care without impairing outcomes.

4. Improved quality. Our fee-for-service system was exactly right in the horse and buggy era, when health care consisted of solo physicians prescribing nostrums for their patients. But it's ideally unsuited to an era that requires coordination among individuals and institutions. Fee-for-service payment penalizes physicians for spending time communicating with colleagues, families, and social agencies. Global budgets encourage these activities. Wider communication and better coordination = improved quality.

When you take up the Payment Reform Commission's recommendations you will hear from genuinely concerned constituents and self-interested fear mongers. Here are some suggestions on how to respond.

1. "This means rationing!" Of course you understand that rationing happens now and, in the sense of allocating our limited funds wisely to multiple good uses, is an ethical requirement, not an abomination. But it will take presidential leadership and time to move public understanding. Given that for now the "R" word is a debate-stopper, you should say that global budgeting is the best way to avoid the necessity of rationing. Cost containment should start by (a) eliminating interventions that are not needed but can cause harm (as by side effects) and (b) substituting equally effective but less costly interventions, such as generics for branded drugs, or prudent watchful waiting for doing a costly test today. The more we do (a) and (b) the less we'll need to do (c) ration - in the sense of deliberately choosing not to provide an intervention likely to be beneficial.

2. "Doctors will earn more by withholding care!" This fear could be real if physicians are allowed to keep large amounts of unspent funds as income. It's OK to point out that fee-for-service payment rewards us for how much we do, even if it's not the right thing. But arguing "fee-for-service is just as bad as capitation" isn't the way to go. You should build into the legislation that unspent funds should primarily be used for innovation and practice enhancement. The public will trust variation in income that's driven by solid quality and patient satisfaction measures, but not variation that tracks with budget alone.

3. "Global budgets let doctors decide everything - what about the idea that patients should be consumers?" Patients should be central to shaping their health care. The key pathway to achieving this is strong partnerships with their clinicians. Collaboration with patients is mainstream medical ethics in the U.S. now - fee-for-service payment isn't required to bring it about. And, legislation can require the accountable medical organizations that it calls for to include patients at the level of the organizations' boards or in advisory committees.

4. "Global budgets will stifle research and innovation!" Global budgets will encourage health care providers to be more hard nosed about the claims of new products. That won't stifle good research. It will, and should, discourage the rampant development of "me too" products that we see today - such as tweaked "new and improved" molecules that miraculously appear just as the parent drug is going off patent. Encouraging our drug and device industry to to invest in products that will achieve the best results at the lowest cost is all to the good.

Massachusetts can take pride in our superb health care assets - schools, hospitals, clinicians, medical groups, researchers, policy mavens, and entrepreneurs. You and the governor have a once in a lifetime chance to draw on our tremendous strengths to catalyze a transformation of our health system. Seize the opportunity!

Medicare Open to All (3)

President Obama's lobbying efforts with governors may be smart politics, but they show the sad state of ethical discourse with regard to health care reform. Here's what I read in this morning's New York Times:

In a meeting last week with five governors — including Republicans who may be more sympathetic to health legislation than those on Capitol Hill — Mr. Obama privately urged them to serve as his emissaries to Congress. He even coached them on the language they should use with lawmakers, two of the governors said, advising them to avoid terms like “rationing” and “managed care,” which evoke bitter memories of the Clintons’ ill-fated health initiative....Instead, he spoke of “evidence-based care,” the practice of using research to guide medical decisions.

Unfortunately, thoughtful rationing and wisely managed care are exactly what our health system needs!

As I've written about before, the public option proposed by the administration has a crucial advantage with regard to the all important goal of cost containment (see here and here for previous posts). At least 30% of the care we currently pay for is useless at best, and potentially harmful through side effects and errors. We can't have an affordable health system without managing our utilization. This won't happen by publishing studies. We have to manage care!

Good insurance companies like Harvard Pilgrim Health Care, the not-for-profit health plan at which I direct the ethics program, know how to do this and can do it in a clinically guided, ethically justifiable manner. But the public rebelled against the idea of insurance company driven managed care, largely because of distrust of for profit insurers, who were seen as withholding necessary care to maximize profit and provide huge executive bonuses. Insurers have largely backed off from managing care, which contributes to the runaway cost trend.

A public program would have a significant advantage for managing care - it is ultimately governed by the public! If it chose not to cover marginally useful care it could be criticized for its decisions, but could not be accused of serving private profit or executive salaries. That potential legitimacy matters a lot for setting limits.

As I've said before, a public program could act as a "down field blocker," taking the lead over time in helping the public understand what it means to set limits fairly. If a public program made managed care more understandable and acceptable, private insurers could follow its lead.

But if a public program simply acted like today's Medicare it would have a significant disadvantage. Medicare is currently not allowed to use cost-effectiveness calculations of the kind every human being uses every day. As a result, its main weapon for cost control is the fees it pays. This is a blunt weapon, and has had the effect of discouraging the service we need most - time spent with patients planning rational treatment and explaining why some things patients may want are not needed.

It's fine for political leaders to avoid using the terms "managed care" and "rationing" as long as they are not deluded by their own rhetoric. "Evidence-based care" and "using research to guide medical decisions" may be more acceptable wording. But evidence doesn't make decisions - doctors do. We know from the Dartmouth Atlas studies that doctors vary enormously in how they respond to evidence. We will have to "guide medical decisions" actively to make health care more affordable!

David Leonhardt on Rationing

In the business section of today's New York Times, economics columnist David Leonhardt has the best short discussion of rationing I've ever seen.

I won't summarize Leonhardt's succinct article. Anyone who's read my postings under the heading of "rationing" has seen all the same views, but Leonhardt has compressed lucid thinking and well-selected facts into 1200 words.

Leonhardt gives a link to a 2006 study by Baiker and Chandra in the Journal of Labor Economics. The findings will be useful for President Obama and his team in making the case that not rationing is an ethically irresponsible approach to health care. As I said in yesterday's post, runaway healthcare costs are not just an economic problem, they undermine health itself via the connection between income, employment and health status.

Here's the abstract from the Baiker/Chandra article:

We estimate the effect of rising health insurance premiums on wages, employment, and the distribution of part-time and full-time work using variation in medical malpractice payments driven by the recent “medical malpractice crisis.” We estimate that a 10% increase in health insurance premiums reduces the aggregate probability of being employed by 1.2 percentage points, reduces hours worked by 2.4%, and increases the likelihood that a worker is employed only part time by 1.9 percentage points. For workers covered by employer provided health insurance, this increase in premiums results in an offsetting decrease in wages of 2.3%.

Let's hope that enough business and labor leaders read Leonhardt's piece. The anti-rationing demagogues are sharpening their sound bite spears. The reform debate needs strong voices from business and labor to tell it like it is!

More Money for Health Care = More Unhappy Doctors

The national debate about health care is heating up rapidly. We're already hearing a lot about whether we'll have the political will to pay for universal coverage (see, for example, this editorial in yesterday's New York Times). But what we really need to do - for the sake of patients, doctors, and the soul of medicine, is to contain costs, not to figure out how to get more money so we can spend even more. Putting more money into the health care system will simply continue the trend towards unhappy and demoralized doctors.

Here's a back of the envelope thought experiment:

(1) Approximately 17% of the U.S. population is uninsured.

(2) But our per capita spending on health care is 40% higher than the next most costly OECD country, while our health status statistics are mediocre.

(3) Experts estimate (and most practicing physicians agree) that at least 30% of the care we deliver (and the costs we incur) are unnecessary.

(4) If we dropped our OECD disparity from the highest OECD expenditure by 1/4 we would save 10% of our per capita spending.

(5) If we eliminated 1/4 of the unnecessary care we would save 7.5% of our per capita spending.

(6) Saving 10% + 7.5% would cover the uninsured.

In my view, what our health system needs is a budget, somewhere in the zone of our current level of expenditure, with increases capped midway between the Consumer Price Index and the projected health inflation trend. Were this to happen (alas, it almost certainly won't) I would predict that in five years we would see an improvement in physician morale and patient satisfaction, both of which are low in the U.S. compared to other countries.

Without a budget we can confidently predict that the cost trend will climb into the economic stratosphere. And faced with continuing health care inflation we'll try to put on the brakes with new forms of external control. That's what we tried in the 1990s with insurance company driven managed care.

Almost 20 years ago Kevin Grumbach and Tom Bodenheimer wrote a brilliant article - "Reins or Fences: A Physician's View of Cost Containment." We can try to tame costs by jerking providers by the reins. But we clinicians won't like that, and when we're unhappy our patients are unhappy. Or we can build budgetary fences and say to doctors, nurses, hospitals et al - "you're smart folks and you care about patients - here's an overall budget...please make it work...for patients and the public."

Faced with a realistic budget we health professionals, over time, would get our act together. We'll organize ourselves into systems like the Mayo Clinic, Geisinger, Kaiser Permanente, and Harvard Vanguard, the group I practiced with for 33 years. Physicians in these accountable group practices are happier with their careers, their patients do better overall, and the per capita costs are lower.

That's the direction we need to go in. But it won't happen without a budget for our health system. That shouldn't be impossible to achieve - we budget for everything else we pay for!

ALS, the Rule of Rescue, and the FDA

The opening paragraph in "Fighting for a Last Chance at Life" in today's New York Times led me to expect what the Brits call "hanging crepe" - using imminent death to blast insurers as uncaring, profit-seekers and the FDA as a callous bueaucracy:

As Lou Gehrig’s disease sapped Joshua Thompson of his ability to move and speak last fall, he consistently summoned one question from within the prison of his own body. “Iplex,” he asked, in a whisper that pierced his mother’s heart. “When?”

Joshua, a vigorous, athletic, married 34 year old father was diagnosed with Amyotrophic Lateral Sclerosis in 2007. Kathy Thompson, Joshua's mother went onto the web, and learned about Iplex, a drug containing an insulin-like growth factor that in 2006 had been reported to stimulate growth of the nerve cells involved in ALS (in tissue culture). The growth factor had been tested on ALS patients and was found to be ineffective, but Iplex combined the growth factor with a protein that might get it into the nervous system more effectively. The New York Times article describes in rich detail the campaign Kathy orchestrated to make Iplex available, despite the lack of FDA approval or evidence - other than anecdotes from Italy, where the government provides it - for its effectiveness.

There are currently no effective treatments for ALS. It is regarded as a uniformly fatal condition. The desperate effort on the part of ALS patients and their families to try any approach about which they have hope is totally understandable. The public policy challenge is - how should a health system respond to patients in a "last chance" situation who want medications (or other interventions) they regard as promising but which have not been shown to be effective?

It's possible that the medication may prove to do more harm than good, but I don't see that as a reason to forbid access. As Kathy Thompson said when safety concerns were invoked - "what, exactly, is safe about ALS?" Patients who are competent to make decisions and who are fully informed about the risks and benefits of a treatment are entitled to decide whether they want to bear the risks for the chance of the gain.

The core issue involves considerations of the common good. If unproven medications are made available, patients in last chance situations will want to try them. I know that I would. But when this happens it is impossible to find out whether the medication really works. In the 1990s many states mandated insurance coverage for high dose chemotherapy and bone marrow transplant for women with advanced breast cancer. The availability of the treatment made it almost impossible to test its effectiveness. When results finally came in it emerged that the treatment was no better than the existing interventions, and often worse. Hundreds of millions of dollars were spent for what, on a population basis, was a net decrement in health compared to alternatives. (The chemotherapy story is told in detail in False Hope.)

As I read the story I thought - the FDA should make the drug available for what is called "compassionate use," but only in the context of a clinical trial. To my surprise and pleasure, that's exactly what the FDA did in March of this year. Here are the key sections of the FDA statement:

FDA's Decision

The FDA and Insmed [the manufacturer of Iplex] have agreed that access to Iplex for investigational use in patients with ALS will occur in two ways under Investigational New Drug applications (INDs):
1. Single-patient INDs requesting “compassionate use” of Iplex for treatment of named patients with ALS, received and date-stamped by FDA’s document room by close of business on March 6, 2009, will be allowed to proceed, and Insmed has agreed to supply Iplex to those patients; and
2. The remaining supply of Iplex, which is very limited, will be used by Insmed to conduct a clinical trial under an IND in which other patients with ALS who are interested in receiving Iplex treatment will be randomly assigned to receive drug through a lottery system.
All patients who receive Iplex under either a single-patient IND or in the Insmed clinical trial must be adequately informed by their treating physician of the possible benefits and risks of the treatment...

Rationale for FDA's Decision

...FDA has attempted to balance the needs of individual patients who are desperately seeking treatment options for this devastating disease with the need to learn if the drug is in fact beneficial, or harmful, in treating patients with ALS. These considerations were weighed over the last few weeks by FDA scientists and physicians, who held a series of meetings with Insmed and internal meetings to discuss the best path forward.

Conclusion

The FDA understands that ALS is a fatal disease with limited to no treatment options. We are very sympathetic to the desperate situation of patients with this terrible disease, and their families, and we remain committed to facilitating the development of effective drugs to combat ALS. Sometimes therapies that appear promising in the preclinical phase (before studies in humans) do not lead to benefits in patients...

The FDA is mindful of the need to strike a balance between access to unproven therapies for patients with limited treatment options, and the ethics of subjecting those patients to drugs with unacceptable risks or unconfirmed benefits. Today we have chosen a means to provide access to Iplex to as many patients as possible, consistent with all of the considerations discussed earlier.

The FDA has done an impressive piece of ethical analysis with regard to Iplex and ALS. It recognizes the dire circumstances of people like Joshua Thompson in last chance situations, the intensity of society's wish to rescue identifiable victims, and the importance of rigorous evaluation of new treatments on behalf of the common good. Hat's off to the FDA!

Octomom, Ethics, and Health Care Limits

This morning I participated in the Massachusetts Association of Health Plans (MAHP) ethics forum. MAHP has been sponsoring 3-4 ethics forums each year since 2002, open to Massachusetts health plans and interested others. Our topic today was "The Octomom Case: Who Should Decide About Fertility Treatment?"

We used the well publicized case of Nadya Suleman ("Octomom") who gave birth to octuplets as a result of in vitro fertilization as an avenue into large issues of health system ethics. Here's how the invitation defined the topic:

There are many ethical dimensions presented in this case. The first is “limits” itself. Should there be enforceable limits to fertility treatments (as opposed to guidelines)? Are we willing to limit choice? And if so, how far are we willing to go? Few would endorse China’s policy of one child per couple, but we intuitively understand that in vitro fertilization resulting in 14 children is too many. Who should set the limit: the patient, the doctor, government or society – or health plan? And is it the same limit for everyone – or are there different limits for those who can afford to pay all costs themselves?

We had decided to open the forum with a mini-debate. I took the roles of Ms. Suleman and Dr. Kamrava, her fertility specialist. Although I've long argued that society and the health professions have a responsibility for stewardship of societal resources and that limits are an ethic ethical obligation, not a moral abomination, I was impressed with how readily I could enter into a role I do not endorse.

The argument I made as Ms. Suleman was based on (a) love of children, (b) not wanting to discard the six embryos that were implanted, (c) my fully informed consent to the procedure and (d) the high value we place on choice in the U.S. As Dr. Kamrava I argued that we ask physicians in the U.S. to respect the uniqueness of each patient. Ms. Suleman loved children, wished for more, and previously had had single births (and one set of twins) when I transferred six embryos to her. I directly responded to the criticism that I had not followed American Society for Reproductive Medicine guidelines by quoting the relevant passage:

For patients under the age of 35 who have a more favorable prognosis, consideration should be given to transferring only a single embryo. All others in this age group should have no more than 2 embryos (cleavage-stage or blastocyst) transferred in the absence of extraordinary circumstances.

I argued that the intensity of Ms. Suleman's love for children and her commitment to not destroying the six remaining embryos, combined with our experience in her previous cycles, constituted "extraordinary circumstances."

The contrary position in the debate was amplified by the group's discussion.

• Individual preference ("patient autonomy") is very important, but it should not be allowed to trump all other values.
• Being part of a multiple birth increases risks for the unborn child. Even though our society allows abortion, it can legitimately decide to limit conception as a way of protecting child health. We protect children after they are born. Why not do so before they are conceived?
• Even if advances in technology eliminated the risks associated with multiple births, so that harm to the potential child would not be a consideration, our resources are not unlimited. Future costs to society should be part of the equation and can be a legitimate basis for establishing a limit.
• We can't expect physicians to be the limit setters. They are (and should be) sensitive to their patient's values and wishes. Many fertility specialists would welcome external limits set by society.

The forum discussion recognized how strongly we in the U.S. value "life, liberty, and the pursuit of happiness." There was some hope that the recession would help us add heightened concern for the commons to our portfolio of guiding values. The group took note of the fact that several European countries regulate the number of embryos that can be transferred - some to two and some to one. They also limit the number of eggs that can be fertilized, which avoids the issue Nadya Suleman invoked - the presence of multiple untransferred embryos.

At the very least, the Octomom case may have created public readiness to accept limits set by insurers on the basis of the American Society for Reproductive Medicine guidelines.