ABIM's New Research on Physicians

"A systematic, intensive study intended to increase knowledge or understanding of the subject studied, a systematic study specifically directed toward applying new knowledge to meet a recognized need, or a systematic application of knowledge to the production of useful materials, devices, and systems or methods, including design, development, and improvement of prototypes and new processes to meet specific requirements"

 - Definition of "Research" - National Institute of Health

It's one thing to ask a doctor to stay current on his knowledge, it's quite another to insist he survey his patients for a private enterprise, especially if that survey represents unvetted independent research.

Recently, a colleague of mine was attempting to maintain his "board certification" credential with the American Board of Internal Medicine (ABIM) and signed up for the ABIM's requirement for a "practice improvement module" worth a required "20 points" of 100 total required before he could sit for his specialty board re-certification examination.  For his module, he optimistically chose to offer a survey created by the ABIM to his patients, receive feedback on how he did on the survey, then repeat the survey to a later set of patients to show "improvement" of care.  In return for his considerable efforts, he would be granted his required "points" from the ABIM so he could qualify to sit for his specialty re-certification examination.

Here is an exact copy of the survey (pdf - 3.52 MBytes) my colleague was sent in its entirety.   He received a packet of 70 of these surveys from the ABIM, neatly shrink-wrapped, to distribute to his patients.

What could go wrong?

First, imagine the time and work involved to distribute these surveys. Whether he provided the survey to his patients himself or he tasked others to do so, what lab result was not reviewed or phone call not answered as a result?  We can only speculate.

Second, informed consent about the true nature of this survey was not obtained from patients nor my colleague.  Rather, my colleague was coerced into purchasing the survey because he might not be able to continue practicing medicine unless he complies with this requirement.  Informed consent would suggest that the doctor and his patients are informed of potential harms or risks involved with the collection of such survey data.    For the patient: what might their responses mean for their doctor's ability to practice medicine?  How might the working relationship with their doctor be degraded or the trust he has in them be compromised?  For the doctor: how are the data collected on the non-secure website protected, how will they be used against him? Will the data be used for future health care policy development or sold to third parties?

I have no doubt that many will see this survey collection as a benign attempt to truly improve a physician's practice or as an opportunity to empower patients with an means of changing physician behavior.  But I suspect these same people never consider the potential negative consequences of such a survey.  The very idea that this survey is a destructive intrusion into the doctor-patient relationship is a foreign concept to its designers.  We can only imagine the moral outrage and disavowal that will arise in the halls of ABIM with such an assessment.  Yet like a bull in a china shop, the collection of anonymous survey data completely disrupts one of the most tenuous and vulnerable relationships in medicine.  It ignores the vulnerable, highly-charged and often emotional circumstances that accompany any visit to a doctor's office while rendering valid concerns a patient might have about their experience into the muddied waters of anonymous data aggregation.  

Also, this unscientific research survey contains a host of dependent variables like age, race and self-assessments of general health status and mental illness.  Self-assessments make a mockery of non-biased data collection, yet the destructive assumptions made throughout the survey are clear: doctors should have unlimited time, provide unlimited access, and perfect manners toward patients without regard to forces (such as this ABIM survey) that increasingly pull them from what they yearn to do: care for their fellow man, woman or child.   This lack of concern with scientific validity and objectivity  leaves the end game of any particular individual or group "findings" only left to the imagination.  If we are going to investigate whether an individual doctor's behavior reflects an age/education/gender/race bias toward their patients (see questions 42 through 46), this is a serious question, deserving of the doctor's consent, and requiring scientific validity far past that of correlational survey data on an n of 70 patients. The possible "end result" or accusation is far too damning.  Or haven't the ABIM committee members thought of that? But we shouldn't worry - patient bias/irrationality/emotionalism is controlled for by question 41 - where the patient provides us with an assessment of his overall mental health.

If doctor's are subjecting themselves to this kind of scrutiny, shouldn't they (and their patients) know how it will be used? Whether aggregated or individual data, this kind of helter-skelter approach is surely designed to lead to progressive "quality" initiatives to adjust doctor's behavior whether findings are valid or not. We are participating in the first step of yet another new initiative in micro-managing and control of the already besieged doctor.

The intrusion of this survey into the sanctity of the doctor-patient relationship by an independent and non-accountable non-profit organization that ignores sound research and ethical principles should be stopped.  It's negative consequences far outweigh any benefit to patients.   In a recent survey of their membership of over 4000 cardiologists nationwide, the American College of Cardiology found that nearly a third of their respondents indicated that the changes imposed by the ABIM's subversive "re-certification" process (that includes these patient surveys as one tool) will affect their future career plans and will likely accelerate their decisions, such as early retirement, part-time work, or transition to non-clinical work.  Approximately one-quarter of physicians in practice for 15 years or more specified that early retirement is a likely outcome.  Exactly how will such a survey help patients already struggling to access care?  Is ABIM responsible for the repercussions of their physician bullying?

I know this is a time of multiple instances of moral outrage and demoralization for physicians.  But I would ask that you take that outrage and forward this survey to colleagues.  I would also ask that you contact your local professional subspecialty organizations, state licensure boards, and appropriate members of Congress to insist on an immediate moratorium to the American Board of Medical Specialties/American Board of Internal Medicine Maintenance of Certification process as it currently exists.

Believe me, this discussion is ongoing and far from over.

-Wes

The Iron Triangle and Evidence-based Medicine

From the Journal of Evaluation in Clinical Practice:

It is naïve to think that we can prevent vested interests from introducing bias. Politicians cannot tally their votes and in sport we rely on umpires, not player, to call the penalties. What are we thinking relying on industry provide evidence about health interventions that they have developed, believe in and stand to profit from? We need to recognize this inherent bias and take action against it.

It is beyond the scope of this paper to discuss practical solutions in great detail, however, we make the following suggestions:

1. The sensible campaign to formalize and enforce measure sensuring the registration and reporting of all clinical trials (see http:// www.alltrials.net/) should be supported – otherwise trials that do not give the answer industry wants will remain unpublished.
2. More investment in independent research is required. As we have described, it is a false economy to indirectly finance industry-funded research through the high costs of patented pharmaceuticals.
3. Independent bodies, informed democratically, need to set research priorities.
4. Individuals and institutions conducting independent studies should be rewarded by the methodological quality of their studies and not by whether they manage to get a positive result (a ‘negative’ study is as valuable as a ‘positive’ one from a scientific point of view).
5. Risk of bias assessment instruments susch as the Cochrane risk of bias tool should be amended to include funding source as an independent item.
6. Evidence-ranking schemes need to be modified to take the evidence about industry bias into account. There are already mechanisms within EBM evidence-ranking schemes to up- or downgrade evidence based on risk of bias. For example, the Grading of Recommendation Assessment, Development and Evaluation (GRADE) system allows for upgrading observational evidence demonstrating large effects, and downgrading randomized trials for failing to adequately conceal allocation (and various other factors). However, currently such schemes are agnostic to the origins of evidence and do not expressly recognize the high risk of bias when the producers of evidence have an invested interest in the results. It would be easy to introduce an evidence quality item based on whether a trial was conducted or funded by a body with a conflict of interest. If so, the evidence could be downgraded. Given the failure of current evidence-ranking schemes to detect and rule out industry-funding bias, this is a necessary step if EBM critical appraisal is to remain credible.

Read the whole thing.

-Wes

h/t: Ivan Oransky on Twitter

A Simple Idea for Collaborative Academic Research

With the rapid dissemination of medical information, global reach of the internet, and realization that more print journals doesn't mean better print journals, today's researchers use a variety of techniques to connect but most, sadly, have not kept pace with the times.

Yesterday, watching the nightly news, I saw something that caught my eye.  It came during a story about yet another shooting in Chicago.  The usual folks were interviewed by the media: distraught family members, friends, and eye witnesses to the crime.  One of the witnesses seemed intelligent, articulate, and surprisingly insightful and there, below his name on screen, was his Twitter handle.

Imagine how researchers could connect if Twitter accounts were added to their author lines in journal articles or next to their e-mail address in the article's contact information section.  Like-minded researchers could instantly connect, follow colleagues with similar interests, or message them questions.  Private conversations could continue via direct messaging or e-mail in follow-up.  Such a system would easily connect like-minded scholars as well as promote an individual's work.  Just as TV stations, news organizations, professional scientific organizations and the entertainment world have embraced the trend, so too should our stodgy academic world.

And why not?

After all, everyone's looking for research funding these days.

Who knows?  Good scientific work might just have a chance of receiving funds from the most unlikely of sources thanks to social media.

-Wes

Judgment versus Big Data

Knowledge in Numbers?

Decisions in medicine are supposed to rest on concrete obervations and hard evidence. 

Often, hard evidence does not exist or when it does, it isn't used.  Why is this? 

Concrete observations, too, are increasingly missed as we stare at computer screens longer and patients less.  Yet we persist. Why?

This is our reality now; our evolving medical world.

But if we stop and think about it, medicine, by definition, is a world of technological faults, systemic frailties, and human inadequacies.  We are convinced we know how a patient dies, for instance, thanks to the wonders of unprecedented imaging capabilities but stand slack jawed when an all-too-underperformed autopsy discloses a surprise cause of death that was completely missed by all.

And our answer to these inadequacies?  Stop doing autopsies.  Even though autopsies have consistently shown that one in four deaths occurs from an unexpected outcome or complication of care.

Why did we stop doing them?  Let me count the reasons: we are human, you see.

History repeats. 

Increasingly we are foregoing clinical judgment and intuition in favor of "Big Data" to make decisions.  We construct 70-page Appropriate Use Criteria for ICD documents that cover (really) just a few special clinical circumstances for patients, as if the authors ever really know a patient's clinical circumstance.  Ask yourself how good we are at predicting the day a person will take their last breath?  Like the weather, life is impossible to predict even when you have a billion data points or more.

Big Data and its certainty are our hottest trend in medicine and academics right now.  We know why this is: we love technology.  It is rational.  It is understandable.  It is linear.  We want, desperately, to understand and compartmentalize our human condition, to minimize its variability, so we can ration our resources logically.  But rather than acknowledging the limitation of such an approach, we forge ahead and create logic from dissociated databases with incomplete or empty data fields based on highly-selected patient populations to make our points.  Outliers are considered nothing more than acceptable loss rations.  We manipulate and massage the incomplete or erroneous data using statistics to make our points seem more valid.  Then, like the azithromycin folly, we extrapolate that data and transmit our firmly held beliefs through government agencies to the masses.  We feel good about our myopic analyses and are happy our academic salary was secured for another day.  In return, the importance of medical judgment, experience, and intuition to medicine are cast aside by our fervent belief that trials, databases, and data manipulation are always free from bias and the influence of greed.

More inadequacies.

But in the face of medical uncertainty, what other than judgment and intuition does a physician have - or a patient have, for that matter?  The real patient that sits before us demands an answer where, more likely than not, no real answer exists. Real concrete clinical challenges are rarely represented in a clinical trial or computer database. So we listen. We observe. We review data. Perhaps we get a second opinion. Patient judgment, life experiences, and intuitions are factored, too. Then we decide, together. Medical judgment and intuition are like that: not all luck, not all logic.


But now with Big Data, the new requirement for wellness and fitness is going to be for patients to keep proper symptoms that stay within the lines.  Symptoms and findings must fit new rubrics.  If they don't, your "caregiver" won't know how to treat you, the computer won't know how to treat you, and the rubric won't know how to treat you.  Who are you to say your symptoms are unique? Who are you to deserve a special look?  In the great cattle call of commoditized medicine created by Big Data, who do you think you are?   A liability risk?  Please, stay normative; align your symptoms with Big Data.  And be happy about it, dear patient, because the ends justifies the means.

Ironically, the folly of man has always been that we think we can have all the answers.  Perhaps we should stop for a moment and really think about what we're creating, courtesy of Big Data.

-Wes


 

Rebuttal: The NEJM's Justification of Medical "Mystery Shoppers"

The proposed examination of access to primary care according to insurance status in nine representative states was largely derailed by physicians and other critics concerned about the potential for government invasion of physicians' privacy. They argued that less controversial survey methods would suffice or that additional studies of the well-known primary care shortage are a waste of public resources. I think these arguments are misguided.

- Karin Rhodes, MD NEJM, July 27, 2011 (10.1056/NEJMp1107779)

Fair enough, Dr. Rhodes. You certainly are entitled to your opinion.

But before I take on my rebuttal to your piece, let's both be clear on a separate issue: what is most misguided about your perspective piece in the New England Journal of Medicine was that comments were not allowed. If they were, the "physicians and other critics" could explain their aversion to these tactics.

So, let me be the first to state my position.

Covert, subversive tactics in research in an attempt to avoid bias carries the risk of introducing additional forms of bias. For instance, when a phone call is made to a doctor's office for a new patient appointment and the problem sufficienctly urgent that other real live patients are rescheduled to accommodate a mystery patient's needs, how, exactly are the affects and costs to the established patients compensated? How will those data be "counted" in your statistics when your one new patient's access if offset by the loss of two follow-up patient's access. Who will explain to those affected by these tactics why they were rescheduled? Will you?

You claim that "the study was intended to generate valid national estimates of primary care capacity before the anticipated expansion of private and public insurance to as many as 38 million currently uninsured Americans."

First, recall the problems with that "38 million" number. Wasn't that number "47 million uninsured not too long ago?" Right off the bat, we see how numbers can be spun in policy circles, Dr. Rhodes. Which leads to the most important question that remains unanswered regarding a study that uses these covert tactics: how will the data be used? Will the data (which most certainly are going to "discover" problems with access) be used to justify mandates to shorten office visits from 7.5 minutes per patient to 7 minutes per patient to improve access? Or might doctors be directed to see more patients that are not insured? Seriously. What policy directives can we expect from these data?

You justify the use of this deceptive practice saying "the use of masking and concealed allocation, widely endorsed for randomized, double-blind clinical trials, lends confidence to the interpretation of results." And yet in the circumstance of randomized, controlled trials, patients must sign informed consent to take part in such a study before they are randomized. Hardly a "mystery" process. Should patients and doctors of prospective clinics not be afforded the same respect who might be asked to take part in your study?

You also seem to feel that a sampling 18% of states (9 of 50) is adequate to formulate conclusions. I find this concerning. National policy development should have representation from all states affected, not a minority. To suggest that the concerns of states with relatively high congestion mirror those with more rural populations is certain to bias policy decisions going forward and, more likely than not, exclude the perspective of less populous states.

So these are just a few of my concerns. There are others. Please note that none of them even begin to address the privacy issues raised by "others." But given the flaws I've outlined, paired with the obvious shortage of physicians that we will encounter in 2014 when the full brunt of the Patient Protection and Affordable Care Act kicks in (not to mention our limited research funding these days), this study certainly does appear to squander our limited public resources. Must we spend our resources to become Masters of the Obvious?

No doubt others would like to share their views, so unlike the New England Journal of Medicine, I'll leave my comments open.

-Wes

Should US Government Research Grants Support Meta-Analyses?

It was another headline busting study this week: Pfizer Drug Tied to Heart Risks - a provocative title no doubt fed to the media from the publisher: the Canadian Medical Association Journal. The study was yet another meta-analysis that culled the world's literature in an attempt to determine if a trend could be found that might implicate Chantix as a causative agent for and increased incidence of heart disease in smokers.

On its surface, the study sounds authoratative, analyzing "14-double-blind randomized controlled trials involving 8216 participants" ranging in duration from "7 to 52 weeks."

Never mind that 57% (25) of adverse events were weighted from one study and that none of the 14 studies had odds ratios that did not cross the unity line.

Despite this, the lead author concluded:

Despite the limitations of our analysis, our findings have potential regulatory and clinical implications.

Sorry, this is not correct. There are no clinical implications of this trial. Like all metanalyses, meta-anaylses simply cannot determine cause and effect. (Note to main stream media: are you folks listening?!?)

That being said, there's another concern I have with studies like this: our deficit and how we're spending our precious research dollars.

This Chantix study's lead author, Sonal Singh, MD MPH, was supported by a grant from the National Center for Research Resources (NCRR), a component of the US National Institutes of Health (NIH) and the NIH Roadmap for Medical Research (grant number 1KL2RR025006-03). This study came from that grant.

But so did meta-analyses on thiazolidinediones and inhaled corticosteroids and their possible risks for leg fractures or heart attacks.

With this grant, here's all you need: pick a drug, any drug. Then go to a computer, do literature searches of other people's work on a particular drug and side effect, then try to find a relationship to something.

That's it. No original ideas. No original hypotheses to test. And all funded by the American taxpayer.

Is this the "roadmap" for medical research that we want for our public scientific dollars?

Certainly we want our researchers to have the freedom to chose an area of research that they feel is important, but when should we insist on some form of accountability for the quality and cost of that research?

-Wes

Addendum: It should be noted that at the time of the above publication, the authors of the above study noted the FDA's announcement of the addition of a warning to the product label for Chantix (varenicline) from the same single 700-patient randomized controlled trial that the authors weighted 57% of their opinion upon, yet the FDA admitted that "the trial was not designed to have statistical power to detect differences between the arms on the safety endpoints."

Disclaimer: I have no financial conflicts of interest to report regarding Chantix or its manufacturer.

Heart Failure Therapy Inappropriately Bashed

People who stretch the limits of science need to be called out.

Especially when their intent appears to be to change therapy recommendations based on conclusions from a retrospective meta-analyses. Meta-analyses are nothing more than selectively-pooled studies aggregated by statistical pseudoscientific hand-waving and data manipulation fraught with so many examples of bias that we have to wonder if the intent of the lead author's comments about such a study to the media were not focused on whether their hypothesis was an appropriate topic for future study, but rather if some just had another axe to grind.

Repeatedly this week we have seen the lead author of the meta-analysis that attempts to determine if QRS width can predict future responders to cardiac resynchronization therapy (CRT) promulgate opinion in the name of science. In fact, the study's lead author, Ilke Sipahi, MD leaps to such amazing conclusions about the study's findings that the mind just boggles. From the Wall Street Journal, to Consumer Reports and now, that bastion of scientific reporting, the New York Times, his comments resulted in a main stream media feeding frenzy on the technology. His conclusion?

"In this in-depth analysis, we found that pacemaker patients with less severe electrical disturbance in their hearts did not receive any benefit whatsoever from these expensive and potentially risky implants. Given the abundance of data showing lack of efficacy in this patient population, current treatment guidelines should be changed."

Wow. I'm speachless. Cause and effect to change medical practice from a meta-analysis! Since when?

Since never.

And how did the mainstream media interpret the study?

Here's a sample comical passage from a Consumer Reports article entitled "Many Heart Failure Patients Don't Benefit from Pacemakers:"

Current American Heart Association guidelines say that patients with a QRS (the measurement of the activity of the heart's left and right ventricles) of greater than 120 milliseconds should be treated with CRT (Cardiac Resynchrtonization Therapy). But the study found that patients with a QRS between 120 ms and 150 ms—38 percent of the participants—received little to no benefit from CRT. In contrast, the 60 percent of people who had a QRS of 150 or higher did benefit.

Said another way, 38% (about 40%) of patients with QRS widths between 120-150 had no benefit to CRT pacing, and 40% of patients with QRS widths greater than 150 also had no benefit to CRT pacing. (Yet a remarkably large 60% of these very sick patients with or without wide QRS complexes - most NYHA Functional Class III - benefitted from the therapy.)

Yep. That's the real data: pre-procedure QRS width does not predict who will respond to biventricular pacing therapy clinically. Some will. Some will not. Truth is, we can't predict a priori which patients will respond to this form of pacing therapy for heart failure. To suggest (suddenly and miraculously) that QRS width can predict response from these poor data aggregations when numerous other studies have failed to show such an effect is an irresponsible leap.

Now, please guys, can we stop bashing the best form of heart failure therapy for patients with wide QRS complexes above and beyond our maximal medical therapy and move on to more important issues like bathing salt bans extolled by our AMA leadership today?

Now there's science!

-Wes

P.S.: (For a much calmer evaluation of the trial and the media's response, see Dr. John Mandrola's blog.)

Reference: Sipahi I, Carrigan TP, Rowland DY, Stambler B, Fang JC.
Impact of QRS Duration on Clinical Event Reduction With Cardiac Resynchronization Therapy - Meta-analysis of Randomized Controlled Trials
Archives of Internal Medicine Published online June 13, 2011.

Disclaimer: Dr. Fisher is on the speaker's bureau for the medical device companies Medtronic and Boston Scientific and earned $2000 from each company in the past year for that teaching. Also, he often cares for real patients with heart failure and, yes, earns a portion of his living implanting CRT devices as a board-certified cardiac electrophysiologist.

Social Media as Research Tool

With overregulation of the clinical research arena, the whole area is ripe for disruption, especially for research performed on rare disorders. Today, it appears that a new mode of assessing efficacy of novel therapies using social media might just be the perfect disruptor:

The new study, published online in the journal Nature Biotechnology, represents an early example of how social networking could play a role in clinical trials, an area of medical science with strict procedures that many would consider especially difficult to apply in the online world.

"The approach has tremendous potential,'' said Lee Hartwell, a Nobel Prize-winning scientist now at Arizona State University, and formerly president of the Fred Hutchinson Cancer Research Center. Standard clinical trials play a central role in the research enterprise of both of those institutions.

Dr. Hartwell, who wasn't involved in the study, said social-network trials aren't likely to replace conventional randomized, double-blinded, placebo-controlled trials, the gold-standard for generating medical evidence. But such trials have become so complicated and time-consuming that new models are needed, he said.

It's about to be a whole new world...

-Wes

Reference:
Wicks P, Vaughan TE, Massagli MP, Heywood J.
Accelerated clinical discovery using self-reported patient data collected online and a patient-matching algorithm Nature Biotechnology 24 April 2011 | doi:10.1038/nbt.1837

Should Authors Write Editorials About Their Own Paper?

In January, 2011, an article authored by Sana M Al-Khatib, MD and others appeared in the Journal of the American Medical Association (JAMA) entitled "Non-Evidence-Based ICD Implantations in the United States." The resultant headlines were widespread, appearing in theheart.org, the Wall Street Journal, ABC News and many, many more news outlets and blogs. So widespread was the story that even the Heart Rhythm Society had to make a statement about this study.

Now, to be fair, the study brought to light the fact that many ICD's were not implanted according to "guidelines." Most of us in the business acknowledge that fact. But there are many many shortcomings to the original article, the least of which were that new guidelines were published in 2008 in response to new data about ICD's, but because CMS still uses outdated 2005 "guidelines," the authors stuck to their original story line.

* sigh *

No matter: the specter that perhaps only 77.5% of America's doctors were implanting ICD's appropriately was just too juicy for mainstream media to ignore.

Fast forward to March, 2011.

A follow-up article by the same group was published more quietly in Circulation: Cardiovascular Quality and Outcomes entitled "Extent of and Reasons for Nonuse of Implantable Cardioverter Defibrillator Devices in Clinical Practice Among Eligible Patients With Left Ventricular Systolic Dysfunction" with Sana M Al-Khatib as second author. In that paper, they looked at just one institution's data for only seven months to find:

Of the 542 potentially eligible patients identified, 224 (41%) did not have an ICD. In the initial adjusted analysis, female sex (odds ratio 1.90; 95% CI, 1.28 to 2.81) and increasing age (odds ratio 1.07; 95% CI, 1.04 to 1.11) were associated with a higher likelihood of not having an ICD. After detailed chart review, of the 224 patients without an ICD, 117 (52%) were ineligible for the device and 38 (17%) patients refused the device, resulting in only 69 (13%) patients eligible for an ICD who failed to receive one.

Realize that this was a retrospective chart review study: not a single patient or doctor was contacted. Further, a tiny sliver of data was evaluated: 7 months of data from one center (we must assume it was the authors' institution since the study was approved by their institutional review board). But even with such a small sampling of data, the authors acknowledge a myriad of reasons patients did NOT get ICD's and only "13 percent" of patients didn't get ICDs who should have.

Interesting... if you can believe the results from such a poor data sample.

But this was not the best part of this story, not by a long shot.

In the same journal, Al-Khatib writes an editorial about their group's work and extols the virtues of their study by stating:

"The authors (ed's note: me included) should be commended for taking on this onerous task. Detailed review of hundreds of charts requires substantial resources. Their research is important because administrative and other national registries have limited clinical information and may not capture vital information, such as contraindications to a particular therapy, patient refusals of recommended interventions, and long-term follow-up."

And if that is not enough, continues, "this study was conducted at one of the premier institutions in this country."

And finally... "this study by Allen LaPointe (ed note: and me, Al-Khatib) is a good addition to the literature because it highlights the importance of verifying findings derived from registries through chart reviews."

Oh, goodness gracious. Can't we at least be the tiniest bit critical of our own work and demonstrate a modicum of humility?

And how much "quality" can we attribute to the editorial efforts of Circulation: Cardiovascular Quality and Outcomes when authors write their own editorials?

Then again, maybe the media spotlight has gone to all of their heads.

-Wes

REFERENCES:

Al-Khatib SM, Hellkamp A, Curtis J, et al., Non-Evidence-Based ICD Implantations in the United States. JAMA 2011 305(1): 43-49.

Allen LaPointe NM, Al-Khatib SM, Piccini JM, et al. 'Extent of and Reasons for Nonuse of Implantable Cardioverter Defibrillator Devices in Clinical Practice Among Eligible Patients With Left Ventricular Systolic Dysfunction' Circulation: Cardiovascular Quality and Outcomes
2011, 4: 146-151.

Al-Khatib SM. 'Toward More Optimal Use of Primary Prevention Implantable Cardioverter-Defibrillators How Do We Get There?' Circulation: Cardiovascular Quality and Outcomes 2011, 4: 140-142.

When Companies Sue Their Clinical Investigators

Imagine: a large stent development company places their new stent in the hands of a clinical investigator. The clinical investigator does the early cases with a new stent, but the results of the study run counter to the company’s expectations. Those problems are reported at a national meeting. The stent company isn't happy that things didn't turn out the way they had expected because they believe the problems identified could be explained away on other grounds.

So what do they do?

Why, not only do they bring suit against the doctor and hospital that did the study, but they also decide to announce their suit in a press release published to every news organization around the world.

Wow.

While I do not know the circumstances surrounding the case, I find it ironic that the company is suing for defamation, when they, themselves, have just electronically done the same thing: defamed the doctor and the hospital in question before trial.

Maybe the doctor didn't want to adjust the results because he believes in his assessments of the complications in this study. Maybe the heavy-handed efforts to discredit his findings are a result of the company's strategic development goals. Then again, maybe the investigator did screw up - does that mean they should resort to a press release and suit? What have others found with their device?

As previously reported in theheart.org:

Assigned to formally comment on the study after Cervinka's presentation, Dr Jeffrey J Popma (St Elizabeth Medical Center and Harvard Medical School, Boston, MA) observed that the study was small but strengthened by "meticulous attention to details, standard clinical, angiographic, and [intravascular ultrasound] IVUS definitions, and concordance of anatomic and clinical end points."

So, he said, "the results are quite believable. And although the conclusion was that this study was just too small to demonstrate clinical benefit, I think it's too large to dismiss the stent thrombosis that occurred following placement of the Genous stent."

Clinical research is difficult enough, but the practice of holding the specter of legal proceedings over the head of clinical investigators when results are counter to corporate expectations should send a warning to any other doctors approached to serve as clinical investigators for this company...

... run, don't walk, away from these guys.

-Wes

Reference:
The earlier review of the disputed report made at the 2009 ACC Meeting in question from theheart.org

So You Want to Be a Clinical Investigator

Young doctors raised in the sheltered environment of hospital systems are finding the foray into a private practice setting increasingly anxiety-provoking thanks to the economic uncertainty of such a practice due to the recent health care reform legislation. Consequently, many are turning to clinical research as an alternative career path. But what does it take to become a productive clinical researcher?

Here are some points to consider:

1. First, think like a medical device company. By and large, companies are usually the ones funding the research. They want to save costs since many companies have limitations to their research budgets. Every step in clinical research, from the design of the trial to the support documentation and prototype patient consent form(s) should be carefully assembled to reduce delays in approval from an hospital's investigastional review board (IRB). Months of delays with IRB's can occur without this documentation and remember, time is money. An acceptable number of patients with the studied ailment must also be seen at your institution. For instance, if the trial requires congenital heart disease patients and your clinic only sees ten of these patients a quarter, you will probably find the trial will be completed before your center has an opportunity to enroll even a single patient. In return, you get nothing more than the enjoyment of completing tons of paperwork.
2. Make sure your center can afford the trial. All research centers require budgets. Most require an itemization of direct costs and then an overhead fee for "indirect costs" (think lights, heating, facilities charges, etc). These costs cut both ways: centers that have "indirect costs" that are excessive will never be approached by companies for research work (unless they are suicidal) and likewise, companies that don't offer some amount for these real-life expenses to conduct the work, shouldn't be performed at your center. Working out the budget needs BEFORE the research is submitted to the IRB will save countless hours of work in the end.
3. Learn to write. Most doctors are great at math and science, but far fewer are skilled at writing. The most productive researchers I have met are not only good doctors, but great writers as well. They can construct an organized research paper complete with a thorough literature review on their first pass. Learn this skill and you will be a highly sought-after researcher.
4. Be a cheerleader. Patients have to believe in the work and so do your referring doctors. If you can't articulate the importance of the trial to these folks, you'll never be successful. Also, since trials take time, keeping your trial at the forefront of your collegue's minds will be one of the toughest challenges that any young researcher will encounter. Other doctors will not have the passion or commitment for your work, so frequent reminders about your trial (along with follow-up on patients that are enrolled) will go a long way to improve trial enrollment. Again, time is money.
5. Be prepared to work after hours. The days of sheltered research time have long since passed. In today's clinical environment, you'll still need to see your usual cauldron of clinic and procedural patients, lest your clincal productivity (and maybe your salary) suffer. While some clinical trials can increase your clinic volume as patients are referred for evaluation in trials, this is usually not the norm.
6. Be timely at returning e-mails and phone calls.
Believe it or not, corporate time is just as important as yours.
7. Be transparent. In today's world, it is not okay to keep your relationships with a device company private. Tell everyone what you're doing and how much you make doing it (they'll find out anyway). If a speaking or consulting junket sounds too good to be true, avoid it. Sticking to this advice will help keep your clinical research career not only viable, but fruitful, for years to come.
8. Get help.
In today's regulatory environment, the number and scope of rules for research are greater than ever. Many centers have people that can help young investigators stay out of trouble. Find out who these people are and use them. Often.

-Wes

How We've Killed Clinical Research

"... I have always depended on the kindness of strangers."

- Blanche DuBois in Tennessee William's play,
A Streetcar Named Desire

Years ago when I began my medical training, I recall enrolling patients for clinical research. In cardiology, there were a myriad of questions that needed to be answered, especially in the area of defining which medications were best to limit the damage caused by a heart attack. Patients routinely participated in large, multi-center prospective randomized trials to answer these questions. It was routine for them not to charged for participating in the trial: the drug(s) and additional testing would be funded by the company whose drug was being studied. Patients enrolled willingly, eager to help advance science and perhaps, in some small way, their fellow man.

It never dawned on me in those early days why hospitals and research centers were so eager to promote research.

Like many things in our profession, research centers realized that these studies could make money. There are always additional expenses required to perform these studies and research centers wanted to make sure they were not left holding the "cost bag" for these trials, so they would pad their budgets for both the "direct costs" of performing the trial and a bit more for "indirect costs" that funded their offices and utilities and perhaps to offset the losses carried by other slow-to-enroll trials.

This strategy worked. Research centers, working earnestly, helped recruit patients by marketing their cutting edge researchers to the populous. Patients came in droves since there was no additional cost on their part to participate. Research centers got additional "indirect" funds from the drug companies to grow. More research was conducted, more patients recruited, and everyone benefited.

Until the costs of health care grew.

About 2001, research was getting expensive for companies. Back then, I noticed a subtle shift occur in the funding of clinical research by the medical device industry. I was involved in Medtronic's Insync trial that tested the first biventricular pacemaker for the treatment of heart failure. As a young electrophysiology researcher, I was stoked: this was exciting new and unproven territory for pacing therapy to venture.

But I remember how the Insync trial budget was formulated because it was quite different than I had remembered with other clinical trials. In this trial, part of the study was completely funded not by the company, but rather a portion of the trial was paid for by the patient's insurer. This was perfectly legal, we were told, since the large companies had to gain approval from our government via the FDA to allow such a payment strategy. Also, insurers were required to pay for clinically-indicated pacemakers. So, since dual chamber pacemakers were already approved for reimbursement by insurers and the "only" new portion of the implant procedure was the placement of a new left ventricular lead, the patient would not be charged for any of the new equipment, and their insurer would "only" be charged for a "regular" dual-chamber pacemaker (mind you, for a pacing therapy that was unproven at the time). Technically, patients still did not have to bear any additional cost of their involvement in the trial and everybody was happy. With this new trial budget strategy now firmly in place, we had, in effect, "relied on the kindness of strangers" (the patient's insurer) to help fund the company's research. No one seemed to notice this shift, and certainly no one minded, since everybody won: the patient got the device, the research center got paid, and the company, too, could even make a little money to offset their expenses in the process.

Naturally, there was a desire for research centers to recruit more and more trials to grow their prestige in the community. Small companies would use research protocols to gain marketshare in competetive hospital accounts using, in part, this patient-subsidized budget strategy. Worse, they would create new add-on trials only marginally relevant for the same growth strategy even after devices were FDA-approved.

Fast forward ten years to our current era.

With the growing cost of health care being offloaded by insurers on the backs of patients, patients have seen their insurance deductibles rise. Employers are finding they can no longer provide "Cadillac plans" to their employees due to costs, and instead move to insurance programs that are less encompassing in an effort to control costs to their employees. As a result, some patients even have "lifetime caps" limits on their insurance. Consequently, patients have become keenly aware of the costs of providing their health care.

I have noticed that patients are increasingly reluctant to participate in clinical research. Perhaps this is because they understand the relationship between ANY health care and THEIR expenses. They know that their insurers might not cover the experimental parts of research trials and are concerned that they'll be left holding the bag. After all, defining where the lines are drawn between what is paid for and what is not in clinical trials is nearly impossible with our current cloudy hospital billing schemes.

Peter Orsag, the White House budget director, has promised comparative effectiveness research will lead us to the promised land of health care cost containment. But given what I see, because patients are footing an increased portion of their health care tab already, do we really think enrolling patients in these trials will be easy going forward?

Reality always trumps the spin in Washington. Unless we see better transparency of costs from all of parties making money in clinical research, we'll never have the patients enrolled to bring any of these trials, comparative or not, to fruition.

-Wes