Month: November 2014

Where Is The FDA?

MedPage Today recently published a four part series describing the results of a remarkable investigation in conjunction with the Milwaukee Journal Sentinel. It is the story of very expensive cancer drugs that have been released on the market with no evidence of efficacy or proof of safety.  This is explained as being the result of desperate cancer patients and organizations advocating quick FDA approval of any drug that might hold hope. 54 such drugs were examined. The average charge for these medications is $10,000/month but as high as $40,000/month. That $40,000 drug has been shown to extend life by 4 months. 40 of the drugs were approved without showing any evidence that they prolonged life or improve the quality of life. Looking at 26 of those approved between 2004 and 2011 only three were found to increase survival. This was by 10 months and  8 weeks and the third improved the 3 year survival rate from 61% to 70%.  Not very impressive.

What is the approval process? Instead of requiring proof of efficacy as measured by improved quality and quantity of life the FDA approves cancer drugs based on surrogate markers, supposedly showing that the drug is able to cause shrinkage or slowing of progression of a cancer. The FDA then gives the drug company many years to come up with better studies of safety and efficacy. The companies stall on this but they continue to collect exorbitant charges. Side effects, including drug caused deaths, are ignored. As Richard Deyo says “Maybe you live a month longer with a new drug but you are having horrible symptoms or horrible quality of life in the meantime”

What are the forces that create this perversion of research and approval? Yes, societal pressure is one of them. But we must not forget that cancer drugs are a $28 billion industry (in 2013). This is a strong motivation for an industry that has a history of distorting and profiteering from tainted research and marketing.  Add to that the fact that half of the FDA drug evaluation budget is paid for by the very pharmaceutical companies it is supposed to monitor.  Ironically, the FDA is now judging its own performance by using surrogate criteria, i.e., the number and speed of drug approvals. It certainly shouldn’t take $800 million to rubber stamp a bunch of applications. They should be able to do that before lunch. Maybe, just maybe, we need to replace the FDA with a true watchdog agency that has strong scientific integrity and maximum immunity from conflicts of interest, and political micromanagement. Also read

The Value-Based Hospital May Not Be

The report by the Boston Consulting Group (BCG), “The Value-Base Hospital” is interesting but disappointing. “Value” is one of the three current catchwords that is used throughout the health care industry (“Quality” and “Patient-Centered” being the other two) . We know that the BCG is off to a bad start when they state, “By definition, health outcomes are specific to a given disease, medical condition, or procedure.” I don’t know where they get that definition but my 50 years of medical practice has always been informed by the understanding that health care and outcomes are specific to patients (and no 2 patients are the same). Medical conditions don’t walk into the hospital. Patients come to the hospital. They almost always have accumulated a list of diagnoses and conditions, some of which are accurate, some wrong, and a lot of “sorts of”s and “atypical”s and “suspected”s.  And they come with a variety of needs, goals, expectations, strengths and attitudes. If there is any value it has to be for each individual patient, not each condition. And only the patient knows what is of value.

The authors’ lack of awareness of the difference leads them to a narrowed appraisal of the STEMI trial. They summarize their perspective by saying, “The study found that routine thrombus aspiration before PCI did not significantly reduce mortality and, therefore, did not contribute to health care value.” What the study really found was no difference in mortality at 30 days, not 90 days, not 1 year (as in the TAPAS trial), but 30 days. And there was no other end point such as post hospital angina, shortness of breath or return to normal activity. The study wasn’t designed to detect any “health care value”. Whether or not thrombus aspiration has any value needs much more sophisticated investigation. As an aside it should be noted that Value is Quality divided by Cost. As, such, the term Value is often misused in place of Quality. Health outcomes are a measure of Quality, not Value. When stakeholders talk of value they usually mean decreased cost to them.

The International Consortium of Health Outcomes Measurement defines health outcome by saying “Outcomes are the results people care about most when seeking treatment, including functional improvement and the ability to live normal productive lives”.  For instance, for low back pain they consider the following parameters: major surgical complications, need for reoperation, need for pain medications, work status, health-related quality of life. The last three of these are important but very subjective and can be measured or scored only by creating arbitrarily weighted number scales. The BCG admits that the usual quality metrics are rejected by physicians as not relevant to patient care. However, the attempt to replace Quality” with “Outcome” is equally naïve and, by necessity, has to focus on events and procedures, not medical care. And it ignores the larger issues such as whether or not the procedures were indicated or avoidable in the first place.

Attempts to measure outcomes for medical conditions introduce a subjective quicksand of forms, questionnaires, and multiple inputs for every hospitalization or patient care episode. And, with all of that, they represent only one moment in time. As an example see the Movement Disorder Society’s 31 page Unified Parkinson’s Disease Rating Scale .

We have a long way to go before we can measure “Quality” of medical care and, as disappointing as it may be, we may never be able to measure it anymore than we can measure the quality of a poem, a symphony or a sunset or, for that matter, a kiss, the touch of a hand, or the healing power of caring. But that doesn’t mean we can’t strive for personal and compassionate excellence of medical care.