Is this double dipping? A new bipartisan House bill on oncology reimbursements

Here is another gem from the House of Representatives: a bipartisan bill to increase Medicare reimbursements to community oncology practices. While at first glance this seems like a reasonable idea, this detail is puzzling:

The so-called "prompt pay" legislation excludes certain discounts extended to wholesalers when calculating Medicare reimbursements and is strongly supported by oncologists.

Confused? Met too. Here is how I understand it. Many community oncology practices have set up infusion clinics, where they administer intravenous chemotherapy on site to their patients. To stock these infusion centers they deal with drug manufacturers and distributors to purchase the drugs at wholesale prices. The bigger the buy, the bigger the manufacturer discount. To the best of my knowledge these discounts are proprietary information, guarded like state secrets. Yet despite these discounts, the clinics charge Medicare a premium for the drugs themselves as well as for the service of administering. The way this legislation looks to me is that it will completely eliminate any reduction in reimbursement related to these discounts. Double dipping, anyone?

Now, I have many friends who are oncologists, and this is really not a slur against them. But these infusion clinics have always represented a cash cow for these practices. And who would not want to have a steady source of income to maintain a robust practice and have some money left over for a life? Again, this is not an indictment of community oncology practices. If, however, one takes an external perspective, this bill becomes something of an anathema to improving efficiency of healthcare delivery. If the reimbursement rates for administering these already exorbitantly expensive drugs improve further, will it not become even more difficult for an oncologist to tread the fine line of the conflict of interest between treatment only when it is in the patient's best interest and treatment for income optimization? Again, I want to point out that I am not singling out oncologists, as it is a part of the human condition to rationalize our selfish decisions by putting them in an altruistic light. And given the amount of uncertainty about who might respond to these drugs, it is easy to convince oneself that a trial of a therapy may be a reasonable idea, with the reimbursements providing a nudge in that direction.

A couple of quotes from the sponsors of the legislation are also worth reprinting:

"On any legislation today, you have to find a way to pay for it. And like any legislation, that's an issue with this one," Whitefield said. 

"But to be truthful, because of the oncologist groups and patient groups and others, we think that there may be some provisions in the healthcare bill that passed last year that we may be able to utilize some of those funds for this. All of it's about healthcare, and if we can convince people that this is more important than the others then we can do it."     

On any legislation today? You mean it has not always been like this? I guess we have all gotten used to credit as a life style, and now it is time to pay the piper.

Now, what about this: "Because of the oncologist groups and patients groups and others..."? Are they saying what I think they are saying? That because groups are likely to benefit are saying that this is vital, it is in fact vital? I also have to wonder who those "others" are. Hmmm, I wonder...

And this: "All of it's about healthcare, and if we can convince people that this is more important than others then we can do this." OK, so the statement is so grammatically abominable and non-sensical that I can interpret it any way I like. And it seems to me that they are implying that increasing these already hefty reimbursements is more important than stuff like paying for prenatal care and immunizations to the poor? And other essential services to the Medicare population? Well, if this is not the a poster child for why we need to be articulating the value of healthcare, I do not know what is.  

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The value of a test

Reading this vintage paper on C diff from the Archives of Pediatric and Adolescent Medicine, I came upon this irresistible conclusion:

Priceless!

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Quality or value? A measure for the 21st century

Fascinating, how in the same week two giants of evidence-based medicine have given such divergent views on the future of quality improvement. Here (free subscription required), Donald Berwick, the CMS administrator and founder and former head of the Institute for Healthcare Improvement, emphasizes the need for quality as the strategy for success in our healthcare system. But here, one of the fathers of EBM, Muir Gray, states that quality is so 20th century, and we need instead to shine the light on value. So, who is right?

Well, let's define the terms. The Merriam-Webster dictionary defines quality as "the degree of excellence." The same source tells us that value is "a fair return or equivalent in goods, services or money for something exchanged." To me "value" is a holistic measure of cost for quality, painting a fuller picture of the investment vis-a-vis the returns on this investment. What do I mean by that?

Simply put, the idea behind value is to establish what is a reasonable amount to pay for a unit of quality. Let's take my used 1999 VW Passat as an example. If my mechanic tells me that it needs to have some hoses replaced, and it will cost me under $100, and the car will run perfectly, I will consider that to be a good value. However, if my transmission has fallen out in the middle of Brookline Ave. in Boston (really happened to me once, many years ago and with a different car), and it will cost me $5,000 to fix, I may say that the value proposition is just not there, particularly given that the car itself is worth much less than $5,000. Given that my budget is not unlimited, I have to make trade-off decisions about where to put my money, so I may instead spend the money on another used Passat that has good prospects.    

But in medicine, we routinely avoid thinking about value. There seems to be an overall impression that if it out there on the market, and especially if it is new, it is good and I am worth all of it. This impression is further enabled by the fact that CMS has no statutory power to make decisions based on value of interventions -- they are legislatively mandated to turn a blind eye to the costs. Does this make sense? How toothless is our comparative effectiveness effort likely to be if it has to ignore half of the story?

Let us now look at my favorite sticky wicket, ventilator-associated pneumonia, or VAP. Now, the IHI bundle aimed at eliminating VAP consists of 5 points of intervention: 1). semi-recumbent positioning, 2). daily screen for readiness to get off mechanical ventilation, 3). daily sedation vacation, 4). prophylaxis against GI bleeding, and 5). prevention of clots. As I have mentioned before elsewhere, adherence of 95% to all these measures is deemed compliance and may be ultimately used as a quality measure by payers to determine levels of reimbursement. And while each of these interventions is basically "motherhood and apple pie", applying them blindly and in toto to 95% of intubated patients may be a strategy for disaster. But what is even clearer is that, in order to implement this and all of the other quality improvement strategies, systems need to be put in place that will safeguard against failing to implement these quality measures. The time and resource expenditures needed to institute and maintain these systems, which have not been described in great enough detail as far as I am concerned, have never been quantified. So, what we are left with is a bunch of interventions that, while looking OK individually in clinical trials (until you really start looking at them critically), are likely providing small, if any, gains in quality at the margins, whose investment-return equation has not even been disclosed, let alone balanced. And because budgets are necessarily limited, as are clinicians' time and cognitive capacities, we need to select a sensible menu of interventions from this practically unlimited feast.

This is the quality conundrum, a clear case of chasing our tails to achieve perfection at the expense of good enough. And while no one in their right mind will argue with the language of improved quality in healthcare, I do think that Muir Gray and his camp are on to something that has been a long time coming. At this time of shrinking budgets, competing priorities and tightening resources, does it not make sense to look at value as a package deal, rather than merely at quality in isolation from its context? Instead of being bombarded by ever-increasing volume of quality measures coming from many directions, would it not be more sensible to prioritize these interventions based on the value that they bring rather than merely on their projected outcomes benefits, so frequently estimated based on data that have very little applicability to the real world? Let's start asking the question: how much quality and at what price? Without paying attention to this critical balance, we will not only bankrupt the system, but also worsen outcomes paradoxically, as we continue to overwhelm clinicians with infinite minutia that may or may not be generating helpful outcomes.

So, in my book, Muir Gray: score; Berwick: keep trying.            

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New treatments: What benefits at what costs

Yesterday brought quite a bit of press coverage to a small biotechnology company in Cambridge called Vertex. All this attention was spurned by their gene therapy trial results in cystic fibrosis. The treatment, aimed at a genetic mutation present in about 4% of all CF sufferers, was able to improve the volume that a patient can force out of his lungs in 1 second by over 10%, from about 65% to 75%. Matthew Herper of Forbes on his blog, while being duly impressed by the results, also cautioned that the annual price tag for this medicine is likely to reach $250,000 per patient. So, what does all of this mean in the context of our ongoing national discussion about the value of therapies? Well, let's break things down a bit.

First, let's talk about CF. This is a genetic disorder that essentially makes mucus very sticky. Among its many effects, in its most familiar manifestation this mucus plugs up the airways making it difficult to breathe and predisposing the person to frequent and serious lung infections. When I was a resident back in the early '90s, I remember a devastating case of a young man in his late teens with CF whom we all knew so well from his frequent admissions for exacerbations. Though he was pretty high on the lung transplant list, he ended up succumbing to a devastating pneumonia in our ICU, leaving behind a devoted sister who had been fortunate enough to benefit from a transplant several years earlier. This was a typical course in those days: a brief life punctuated by frequent exacerbations, hospitalizations, antibiotics, gastrointestinal complications, and early death in the second or at best third decade of life with very little hope of procreation. Over the last 20 years things have changed dramatically in the treatment of CF: fewer exacerbations, much lengthened life expectancy and a good chance of having children. Yet we cannot attribute most of these changes to dramatic new breakthrough therapies. To be sure, while there have been tweaks to how we give antibiotics and how pancreatic enzymes are administered to replace the digestive enzymes that the pancreas in CF is unable to produce, most of the progress can be attributed to the increased attention to detail and the advent of almost ruthless care coordination at specialty centers. As a Fellow in the '90s I participated in a clinic where CF patients were transitioning from care by pediatric Pulmonologists to that by adult doctors. The CF specialist running this clinic did not only know all of his patients and their family members by names, but was available 24/7 to them and to his staff for consultation. This is the kind of dedication and vigilance necessary to improve the outcomes in CF.

Now, let's talk about the lesion addressed in the Vertex trial. The type of chronic lung disease caused by CF is called "obstructive." Simply put, it makes exhaling the air in the lungs difficult to do. On lung testing one manifestation of obstruction is the amount of air one is able to force out of his lungs in the first second of the effort, and this is called the FEV1, or forced expiratory volume in 1 second. Another important measure of the degree of obstruction is the amount of air that this volume expired in 1 second represents as a proportion of all of the air in the lungs that can be expired, known as the FVC or forced vital capacity. We say that if the FEV1/FVC ratio is under 75%, then obstruction is present. The size of FEV1 helps us understand how bad the obstruction is.

With this as a background, the primary outcome in many obstructive lung disease trials is the improvement in the FEV1. In the specific trial discussed, the average starting FEV1 in the intervention group was about 65%, which falls in the mild-to-moderate category of obstruction. What this means in terms of symptoms can vary widely. The 10% absolute improvement seen in the intervention group resulted in the average FEV1 of about 75% after treatment, definitely representing fairly mild obstruction (generally FEV1 over 80% is considered to be in the normal range). And this truly is impressive. However, equally interesting is the information that is not in the press coverage, largely based on press releases and sound bites from company executives, since the peer reviewed study is not available at this point. We are not told, but led to assume that, the control group started out on average with a similar deficit in lung function. We are informed that the treatment patients were 55% less likely than placebo patients to have an exacerbation of their disease, yet we do not know what the absolute numbers are; that is we are not told what proportion in each group had an exacerbation, how frequently or how severely. So, this 55%, in the absence of context, while an attention grabber, is not a substantive number. Herper does tell us that there was a remarkable difference in the weight gain (a desirable outcome in the CF population), on average 6.8 lb in the treatment vs. 0.9 lb in the placebo group. This is truly impressive, though it would be even more so if I knew that the trial was double blind, a piece of information I did not notice in any of the reports. Some of the reports have also alluded to symptomatic improvement in shortness of breath, though nowhere did I see this quantified.

The most important piece of data, however, is conspicuously absent from all the stories. What is the proportion of patients who responded to therapy? Why is this important? Well, we know that far from everyone responds to every treatment that they ostensibly qualify for; this is referred to as the heterogeneous treatment effect, or HTE. It is very likely that the 10% improvement in the FEV1 represents at once an inflated estimate referent to those non- or under-responders and a muted one for those patients with a terrific response. The question of a minimal clinically significant change in the FEV1 has haunted the lung trials community for a long time now. Yet, without setting some threshold for a minimum FEV1 improvement that correlates with a meaningful improvement in symptoms, one cannot quantify how well the drug works and hence articulate its value. This is crucial when trying to justify the ostensibly exorbitant price tag anticipated for this drug. How many patients will we need to treat in order to have one of them respond meaningfully with an improvement not just in a laboratory number, but also in their lives? If this targeted drug produces a desirable response even in 50% of all patients with the specific mutation it targets, then it means that we need to spend $500,000 annually to obtain a meaningful improvement in symptoms in one CF patient. But what if it only works this way in 20%? Then we will need to treat 5 patients with this drug to obtain 1 meaningful response at the price of $1.25 million annually. This becomes a bit more daunting, particularly given that the costs will have to be covered through some kind of public or pooled funds and given that this is one of many therapies in the pipeline likely to come with a similar conundrum.

I am not implying that improving a single life is not worth $1.25 million annually. In fact, it may well be a bargain. My point is that these are the serious discussions we need to have as a society, so that when the time comes to make these choices, the discussion will not be subverted by a few loud voices sensationalizing "death panel" slogans. Manufacturers need to know that they should disclose full data, not just selective tidbits that highlight benefits only, but also those difficult pieces of information that shed light on their costs. On our part, we need to understand the gargantuan effort and resources these companies expend to tame these elusive wild therapies that hold so much more promise in the abstract than they end up embodying.

We tread a fine line here. Information and how we assimilate it are the next frontier for cogent decision making. We need to get educated about this now because this train is leaving the station regardless of how we feel about it.                            

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