Comparative Effectiveness

QUALITY: Comparing Perspectives in Comparative Effectiveness Debate

Paul Testa — Mon, 16 Feb 2009 16:13:00 -0500

The New York Times' Robert Pear examines the content and contentiousness of the comparative effectiveness provisions of the $787 billion stimulus package.

HR 1 provides $1.1 billion (pdf starting on page 156) to AHRQ, NIH and the HHS to evaluate the relative effectiveness of different health care services and treatment options. The goal is to create a process of funding and disseminating comparative effectiveness research that is transparent, professional and free from conflicts of interests. As the Dartmouth Atlas' Elliott S. Fisher, MD, tells Pear, the funding would be used to try to answer questions such as:

Is it better to treat severe neck pain with surgery or a combination of physical therapy, exercise and medications? What is the best combination of "talk therapy" and prescription drugs to treat mild depression?

How do drugs and "watchful waiting" compare with surgery as a treatment for leg pain that results from blockage of the arteries in the lower legs? Is it better to treat chronic heart failure by medications alone or by drugs and home monitoring of a patient's blood pressure and weight?

In health care, though, simple questions rarely have simple answers. Nor will answers be static; biomedical science changes at a pretty astonishing clip. We wrote about this topic twice last week, and we expect to hear more about it as the health reform debate intensifies. Last week Kyle Noonan wrote about some of the alarmist rhetoric surrounding the issue, and Joanne Kenen wrote a longer piece about how comparative effectiveness fits into "health" as well as health care reform. We also want to point out Bob Laszewski's posts at the Health Care Policy and Market Place Review, which has provided interesting coverage on this topic. In his most recent post he worries that the final bill's focus on clinical effectiveness will not translate into cost effectiveness. Commenters on the post have suggested that getting the clinical data right is the hard part and that once that information is available it will be easier to work out the questions of cost and value.

Beginning to measure what works best for what patients or populations under what circumstances can provide a baseline. Providers and patients will have access to information that will help them better understand their choices and the likely consequences. Providing that information in a manner that is transparent, credible, and easily understood will go a long way to ensuring that all Americans receive the most effective care whether they live in Florida or Nebraska or California.

HEALTH REFORM: Relax, Folks

Kyle Noonan — Fri, 13 Feb 2009 18:55:00 -0500

It's not just the blogosphere that's agog about some of the pretty astonishing things being said about comparative effectiveness. Steven Pearlstein's Washington Post column debunks some of the wild arguments being made against the comparative effectiveness and health IT provisions in the stimulus bill. Pearlstein provides an insightful look at how the controversy got started and addresses the alarmist rhetoric. He rightly points out:

There's nothing particularly new about comparative effectiveness research—the National Institutes of Health, along with the Agency for Healthcare Research and Quality, have been doing it for years, with a budget last year of about $335 million....nearly all experts agree (comparative effectiveness research) is a necessary first step to reforming a broken health-care system.

Pearlstein punches holes in the argument that the research would lead to denial of appropriate care and makes the case that our health system would be, well, healthier if we had better information on what works and what doesn't:

And there is no reason we cannot set up reasonable procedures, overseen by independent health professionals, to protect patients who can demonstrate a special need for a treatment that is not normally cost-effective.

...(O)urs is an economy that is sinking under the weight of a health-care system that costs twice as much as any in the world while delivering poorer health outcomes. The cost of health care has crippled entire industries, disadvantaged our companies in international competition and brought millions of families into bankruptcy. Worst of all, in denying vital medical services to the 40 million Americans without health insurance, we engage in the most immoral kind of medical rationing imaginable—rationing by the ability to pay.

By the way, in this blog's Thursday post about comparative effectiveness and partisanship, we asked, "Where is Judd Gregg?" An hour or two later, we found out...

QUALITY: Comparative Effectiveness Is About Caring Effectiveness

Joanne Kenen — Thu, 12 Feb 2009 15:19:00 -0500

Imagine plunking down more than $2 trillion a year and not knowing what you are getting for it. Imagine that what you are purchasing gets more complicated and convoluted every year. Imagine that lives, literally, hang in the balance.

Then imagine that someone comes up with a sensible approach to solving or at least shrinking the problem. And they get hit on the head for their efforts.

Welcome to the world of comparative effectiveness.

The partisan warfare over the stimulus bill (which frankly has surprised some of us with its intensity—Judd Gregg where are you?) gives us glimpses of some ugly battles ahead in health care. Comparative effectiveness research is an early battleground. Advocates range from the Institute of Medicine to the AARP; both Sen. John McCain (R-AZ) and President Barack Obama advocated some form of the research during the presidential campaign. The Medicare prescription drug law, which was largely written and backed by the Republicans, explicitly authorized comparative effectiveness research. But critics, who as the Wall Street Journal reported this week include powerful drug companies and medical device makers, say it is a first step toward government rationing or penny-pinching-driven medicine. Conservative commentators have launched a full-scale assault on the concept using some pretty shrill and scary imagery.

But comparative effectiveness is just what it says. Comparing two or more treatments, therapies, drugs and devices, and figuring out which one works better for patients. There is room for legitimate debate—indeed, the debate has already begun and has been part of the stimulus fight—about whether costs should be an intrinsic part of that equation, or whether cost-benefit analysis should be a separate, second step. But the primary goal of comparative effectiveness is just what it sounds like. Compare two or more drugs, devices, or other interventions, and figure out which works best in which circumstances. If a really cheap old medication works way better than a really expensive new one, it should be pretty darn clear which should be the first line of treatment for most individuals. If a really expensive new drug prolongs lives or controls symptoms or eases pain, then we'll have reason to spend the money (why some of these drugs cost so much is a whole different issue, as Gooznews often reminds us). In many many cases, the answers won't be so clear-cut, or they will change over time as we learn more. But we'll have data and science—not well intentioned guesswork or pervasive marketing—on which to base clinical decisions. Yes, the goal is to spend smarter. But it's also to make people healthier. Health is, after all, the point of health care.

The AARP's CEO Bill Novelli put it aptly:

They're at it again. Opponents of health reform are now using scare tactics in a misguided attempt to stop progress in its tracks, blocking attempts to fix the broken health care system that is hurting American families and our economy.

The latest attacks revolve around a smart policy in the economic recovery package that would fund ‘comparative effectiveness research'—a wonky term that just means giving doctors and patients the ability to compare different kinds of treatments to find out which one works best for which patient.

Opponents—like some drug companies and medical device makers—don't want this research. They fear it will cut the profits they make on ineffective drugs and equipment.

But they won't tell you that this research could save your life by giving your doctors better information so they can prescribe the best treatments available to you.

Really, it's not like the AARP is going to be out there agitating for something that will prevent older Americans from getting good health care!

But if you don't like what the AARP and Consumers Union discovered in their recent research and forum on the subject, see what the Institute of Medicine has to say. IoM had a conference with top policy experts on evidence-based medicine in 2007 and outlined the main issues in comparative effectiveness in a 32-page report last summer. The IoM wrote:

Medical care decision-making is now strained, at both the level of the individual patient and the level of the population as a whole, by the growing number of diagnostic and therapeutic options for which evidence is insufficient to make a clear choice. Biomedical insights and medical innovation continue to advance opportunities to increase the health and life-span of the American public, yet to capitalize fully on this potential requires enhanced capacity to ensure that decisions, in the face of increasing complexity, can be supported and guided by the best available scientific information.

The information gap is growing daily:

As the boom in pharmaceuticals, devices and biologics has left us with a need for information on clinical utility and as innovations in the pipeline come to fruition, the information gap will widen. The rate with which new interventions are introduced into the medical marketplace is currently outpacing the rate at which information is generated on their effectiveness and the circumstances of best use. If trends continue, the ability to deliver appropriate care will be strained and may be overwhelmed.

So when a doctor prescribes something for us or our families, we want to know that the doctor has a scientific reason for doing so. When an insurer says we can or cannot have something, we want to know the insurer has a scientific, not monetary, reason for doing so. When a drug or device comes on the market, we want to know it has a scientific reason for being there. As the Institute of Medicine and others have pointed out, there are lots of unresolved questions about who should do the research, how it should be funded, and precisely how to use the data in our payment policies. But there are answers to those questions about the framework. And once we answer them, we can get on to the questions that really matter: What works?

COSTS: What's Not to Like About A Cheaper Diuretic?

Joanne Kenen — Wed, 03 Dec 2008 14:11:00 -0500

The latest installment of the New York Times "The Evidence Gap" series ran over the holiday weekend and looked at why doctors by and large kept prescribing expensive brand name blood pressure drugs years after a huge $130 million government-funded clinical trial known as Allhat showed that older generic diuretics worked better for far less money.

As the Wall Street Journal's Health Blog put it, "Study Found Cheap Blood Pressure Meds Are Best. No One Cared."

On one level, the Times article provided an illuminating look at how the losers in this study—name-brand drug companies—sowed doubt about the accuracy and reliability of the Allhat study as they increased efforts to market their drugs.

But another subtler aspect of the story reminds us that comparative effectiveness research—which we strongly support—is not easy. By the time one data set is analyzed and interpreted, new drugs have come on the market and others become available as generics. The target is always moving. The paper quoted Dr. Sean Tunis, a former chief medical officer for Medicare, as saying that while he supports comparative-effectiveness studies "they are hard to do, expensive to do and provoke a lot of political pushback." Dr. Tunis who now runs the nonprofit Center for Medical Technology Policy added, "There's a lot of magical thinking that it will all be science and won't be politics."

Dr. Carolyn Clancy, director of the federal Agency for Healthcare Research and Quality, noted that traditional clinical trials aren't the only way to gather data about comparative effectiveness on patients in the real world. Her agency has been using insurance records, for instance. Other data could become available to researchers if the U.S. finally got its electronic health records and health IT act together. As Merrill Goozner notes in his blog, patient outcome registries can cause their own debates, but that was how scientists uncovered the Vioxx problems.

COST: Small Successes in a "Mega-Mess"

Joanne Kenen — Mon, 01 Dec 2008 17:45:00 -0500

The Washington Post reviews the growing consensus and awareness about the amount of waste in our health spending. The piece includes "one small vignette" of success in what one health executive called a "mega-mess," and one note of hope that people will soon be in the right places to start fixing it.

"We're not getting what we pay for," says Denis Cortese, president and chief executive of the Mayo Clinic. "It's just that simple."

The "vignette" involves Starbucks, Aetna, and Seattle's Virginia Mason Medical Center, which as we've noted in the past has a reputation as a health-care efficiency innovator. Apparently all that coffee-pouring is hard on one's back, so Starbucks was spending big bucks on MRIs. Even though there is little scientific data that MRIs help in such cases. Ceci Connolly writes:

So they flipped the process, trying physical therapy first. To make up for some of Virginia Mason's lost revenue, Aetna increased its payment for the therapy. Today, the majority of Starbucks employees with back trouble return to work within 48 hours without an MRI or a prescription.

Lower costs. Better outcomes.

Getting there is hard, though, requiring changes in the culture of medicine, in the expectations of patients, in the way we pay doctors, even the congressional politics of health care decision-making. But former Senate Majority Leader Tom Daschle, poised to be the next Health and Human Services Secretary, has spoken and written widely on the need to make health spending more evidence-based, apolitical, and rational. And Peter Orszag, outgoing head of the Congressional Budget Office and incoming director of the Office of Management and Budget, says we'd save bundles even if we capture only a third of the waste. "Given the scale of it," Orszag told the Post, "I am puzzled as to why we are not doing more to improve the efficiency of the health system."

QUALITY: These Statements Have Not Been Evaluated by the FDA

Paul Testa — Mon, 24 Nov 2008 20:48:00 -0500

Can we interest you in some unapproved drugs?

Medicaid is interested. So is the FDA. And Senator Chuck Grassley (R-IA). It seems that from 2004 to 2007, Medicaid spent nearly $198 million paying for prescription drugs that had not gone through FDA safety and effectiveness reviews, according to analysis of federal data by the Associated Press.

Bringing in the bigger picture, the AP notes:

At a time when families, businesses and government are struggling with health care costs and 46 million people are uninsured, payments for questionable medications amount to an unplugged leak in the system.

The FDA estimates that unapproved drugs account for 2 percent of all prescriptions, or about 72 million scripts per year. (Informative FDA video with great sound track here) Many of these drugs provide little or no benefit to the patient. Some are potentially harmful, even deadly, contributing to the some 1.5 million Americans, killed sickened or harmed by preventable medication errors each year.

How did we get here?

In 1962 Congress passed a law requiring the FDA to test the safety and effectiveness of all new drugs entering the market. But today's class of unapproved drugs were already on the market, and were "grandfathered" in, allowing their continued sale today.

The concern over unapproved drugs spiked in the 1980s, when E-Ferol, a high potency vitamin E injection, was linked to the deaths of some 40 premature babies. Since then the FDA has taken steps to remove unapproved drugs from the market and encourage their manufacturers to adhere to FDA guidelines. Critics, as the AP notes, suggest this case-by-case basis is not enough. Senator Grassley has long been outspoken on the issue, last year asking the Office of the Inspector General of HHS to investigate.

Does the FDA have bigger fish to fry? Maybe. But the AP's findings provide further evidence of the lack of knowledge and coordination that exists in our fragmented system. Weeding out old cough remedies from the 1920s is just one of the benefits from the clinical and comparative effectiveness data that a more integrated health care system would provide. (And of course some of these old cheap drugs, once properly reviewed, could turn out to be better options than their expensive new cousins) After all, when you're recovering from the hantavirus, you'd like to know what's snake oil and what's safe.

COST: Boston Hospital Says "Uncle," Buys Robot

Joanne Kenen — Mon, 24 Nov 2008 18:56:00 -0500

Months ago, in one of several posts about the daVinci Robot Surgical System,(also here and here), we wrote about Paul Levy's struggle over whether to purchase one for Beth Israel Deaconess Medical Center in Boston, where he is CEO. Levy revisited the topic in a post he called "Uncle!"

He's still not convinced whether the clinical efficiency measures up to the manufacturers' marketing efficiency, but concluded he had to surrender to the technology arms race... one reason which health costs are pushed ever higher. He wrote:

Without making any representations about the relative clinical value of this robotic system versus manual laparoscopic surgery, I am writing to let you know we have decided to buy one for our hospital. Why? Well, in simple terms, because virtually all the academic medical centers and many community hospitals in the Boston area have bought one. Patients who are otherwise loyal to our hospital and our doctors are transferring their surgical treatments to other places. Prospective residents who are trying to decide where to have their surgical training look upon our lack of the robot as a deficit in our education program. Prospective physician recruits feel likewise. And, these factors are now spreading beyond urology into the field of gynecological surgery. So as a matter of good business planning, concern for the quality of our training program, and to continue to attract and retain the best possible doctors, the decision was made for us.

It's one of many reasons we look forward toward health reform that makes use of more evidence-based analysis of what works, and what's a worthy investment.

PS. Speaking of robots, we recently had to do a little consumer research on replacing vacuum cleaners (didn't they used to last more than five years?) and just out of curiousity, we checked on those little round do-it-themselves vacuum robots. They may be cute, but apparently some of them have an unfortunate propensity for shutting doors behind them and locking themselves in the room. We trust the problem has no surgical equivalent...

HEALTH REFORM: Setting the Stage...

Joanne Kenen — Wed, 19 Nov 2008 16:26:00 -0500

A few health tidbits in Washington’s musical chairs before January.

Peter Orszag, the respected head of the Congressional Budget Office, looks likely to head up the White House Office of Budget and Management. Orszag "gets" health care, and he has been extremely effective in his two years at CBO in helping others in Washington "get" it too. He is one big reason why you hear a different dialogue around town these days, a dialogue that reflects understanding of the huge amount of waste and poor quality built into our anachronistic payment and delivery sytem. Orszag appears to live, breathe, eat and sleep Dartmouth Atlas data, and wants us to invest in comparative effectiveness research so we don't deliver and pay for care of "dubious value."

Orszag understands that health spending—not demographics of aging boomers per se—is the crux of our fiscal challenges. He also built up the health economics expertise at CBO in preparation for Congressional action next year. His likely successor is Doug Elmendorf, according to several news reports. Elmendof who, directs the Hamilton Project at the Brookings Institute, is also no stranger to health care. An analyst at the CBO from 1993-95 he worked primarily on national health care reform.

Over in the Senate, HELP committee chairman Sen. Edward Kennedy said he is divvying up some of the policy tasks among senior committee Democrats as he puts together health reform legislation. Tom Harkin of Iowa will lead a working group on prevention and public health (including tobacco, a long-time interest). Barbara Mikulski of Maryland will focus on quality. Hillary Clinton gets insurance coverage (unless of course she ends up as Secretary of State..) Noteworthy absence: Chris Dodd, Kennedy's close friend and ally and the second-ranking Democrats on the committee. Dodd is currently Banking committee chairman.

COST: Who Decides?

Joanne Kenen — Tue, 04 Nov 2008 19:57:00 -0500

We write often about how inefficiently we provide care in the U.S., and how a top order of business for the next administration should be establishing a comparative effectiveness entity to help sort out what works and what doesn't. A story in today's New York Times about a court ruling on Medicare spending stresses the importance of having independent analysis about effectiveness.

The article reports that a Federal District Court in Washington blocked the Bush administration's effort to save money on Medicare by paying only for the least expensive treatment for a condition—in this legal case, chronic obstructionary pulmonary disease.

The administration wanted the Health and Human Services Secretary to have more discretion over Medicare payment policies. The judge said Congress sets detailed formulas.

We think this begs an important question. Do lawmakers really know whether drug A or drug B is really better for COPD? Do cabinet secretaries? The answers should rather be based on independent, reliable, untainted science and public health data. You can't make cost-benefit analysis without knowing the benefits. And if we really make an effort to get good data, to pay for effective quality care and not waste so much money on unproven or outright unnecessary care (maybe $700 to $800 billion a year), the cost piece of that equation may not be quite so daunting.

HEALTH REFORM: How "Evidence-Based Baseball" Can Help Us Fix Health Care

Joanne Kenen — Fri, 24 Oct 2008 15:51:00 -0400

None of us need to be reminded that we live in a time when every dollar, including every health care dollar, has to count. We highly recommend a terrific New York Times op-ed today by the unlikely trio of Sen. John Kerry, former House Speaker Newt Gingrich and Oakland A's General Manager Billy Beane. They call for government-backed, truly independent comparative effectiveness program to find out what works in medicine and what we should stop wasting our money on. And remember, they penned this piece even before this week's headlines about the widespread practice of doctors prescribing placebos.

Wonder why the Oakland A's are relevant? Read the piece to capture the whole lovely argument but the gist is that baseball has experienced what the authors call a "data-driven information revolution. Number crunchers now routinely use statistics to put better teams on the field for less money." We've learned from the Dartmouth Atlas and numerous other researches that more isn't always better in health care, that lower-cost hospitals get as good or better results than the bigger spenders. Well, the Tampa Bay Rays (and we know how it must have hurt Boston-based Sen. Kerry to write these lines) have the second-lowest payroll in baseball. Yet they are the ones in the series while the bigger bucks teams are watching it on TV. Here are a few highlights from the peice:

Remarkably, a doctor today can get more data on the starting third baseman on his fantasy baseball team than on the effectiveness of life-and-death medical procedures....

Evidence-based health care would not strip doctors of their decision-making authority nor replace their expertise. Instead, data and evidence should complement a lifetime of experience, so that doctors can deliver the best quality care at the lowest possible cost.

Working closely with doctors, the federal government and the private sector should create a new institute for evidence-based medicine. This institute would conduct new studies and systematically review the existing medical literature to help inform our nation's over-stretched medical providers. The government should also increase Medicare reimbursements and some liability protections for doctors who follow the recommended clinical best practices.

They pack a lot in here, including a potential bipartisan fix to the long-running fight in Congress about malpractice reform. We've strongly backed comparative effectiveness before, it's got growing (though admittedly not universal) bipartisan support in Congress. Obama and McCain both endorse the concept. Let's hope the current economic crisis makes this a top priority for Congress next year, even if certain sectors in the health care industry don't like it. As the authors conclude, "The best way to start improving quality and lowering costs is to study the stats." We won't go quite so far as to suggest that we should scrap complex quality reports that people don't understand and replace them with "doc trading cards" with cost, complication and mortality rates instead of RBIs, hits and errors. But you get the idea.

(Gooznews also has feisty post on this topic today.)