What Celgene's Pomalyst Tells Us About Medical Progress

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  • 02/11/2013
Here's what the pundits will say about Pomalyst,  an immunomodulating drug developed by Celgene for patients who have a form of multiple myeloma (MM) that has mutated around other treatments (this is from the well-known peer-reviewed medical publication, The Daily Beast)

“So far, most new (cancer) drugs offer only marginal extensions of life and few cures. “

Now a look at the facts and where Pomalyst fits in the progress we are making against MM.  Pomalyst was studied in people with MM who had no other options.  As in,  their MM was no longer responding to other drugs and they were going to die.  

Clinical trials showed that median overall survival for people getting Pomalyst was 13.7 months.   

Another study looked at response of patients who were no longer responding to other drugs and whose MM had already come back:

Clinical trials showed halfway into the study that nearly 50 percent of people receiving Pomalyst responded rapidly (as in the MM stopped progressing) and 78 percent had a  median surival time of between 3-6 months.   Patients receiving Pomalyst were seven times more likely to respond (and live) then those who received an old MM drug. 

Now overall survival is the average time half of all patients in a study will live after treatment.   So a median survival of 13.7 months  means  that after 13.7 months, 50% of people with that condition would be alive, and 50% would have passed away.   Which also means that many people in the trials are living much longer than 13.7.   Or in the other study, 3-6 months.   

Several studies have shown  that the median survival of people diagnosed with MM has gone from 3 years in 1997 to up to  20 years.  The first MM drug was introduced in 1998.  Two others were introduced in 2003 (Velcade) and Revlimid (2005) thereafter.    W hen age is controlled for, that  means an increasing number of people with MM can expect to have the same life expectancy as people who do not have the disease.  And we can't measure the full impact of these drugs let alone Pomalyst in a precise manner.   

But we can say that MM drugs have increased average life expectancy more rapidly among MM patients than it has average LE in the population as a whole. 

And we can say that just as a few HIV drugs introduced over a 5 year period dramatically saved lives, so too have a handful of drugs for MM turned an incurable disease into a controllable condition in less than a decade.  And these drugs work because they target specific disease mechanisms in patients for whom they work the best and offer the most hope.   The averages are indicators of profound and rapid gains among more and more people.  

If we had bought the story about marginal benefits 10 years ago and delayed and dragged out approval and use of other cancer drugs, where would we be today?  If we accepted the arrogant assertion that "most new (cancer) drugs offer only marginal extensions of life and few cures.“ how may more people would be dead?   Let me go out on a limb and say that while not all new products are useful and effective,  the current effort to control health care costs is biased and shaped by this naysaying approach to innovation, an approach delays access to new medicines -- indeed an approach that subsidizes a industry of underachieving social scientists to study average cost effectiveness in an era of personalized medicine --  assures more people will die who did not have to or want to.    Recently, the National Pharmaceuetical Council held a conference on The Myth of the Average Patient..  All well and good.  But how about a conference on what CER will cost people seeking better medicines for cancer and Alzheimer's in terms of lives saved.   My late colleague John Vernon and I did a lot of work in this area but somehow the "stakeholders" always ignore the impact of adding CER to the rate of innovation and the number of lives saved.   Why is industry and patient groups silent about the impact of CER on medical progress?  What gives? 

Finally,  why does it take so long to conduct clinical studies.  In both of the trials mentioned above, response was immediate and highly effective.  (Less than 6 months in most cases).  Can't we find other ways to confirm what works and what doesn't?  Here's Eric Topol on this important topic..

We have this big thing about evidence-based medicine and, of course, the sanctimonious randomized, placebo-controlled clinical trial. Well, that's great if one can do that, but often we're talking about needing thousands, if not tens of thousands, of patients for these types of clinical trials. And things are changing so fast with respect to medicine and, for example, genomically guided interventions that it's going to become increasingly difficult to justify these very large clinical trials.

We are not just standing in the way of medical progress, we are putting up obstacles based on outdated science and sanctimony.  


Center for Medicine in the Public Interest is a nonprofit, non-partisan organization promoting innovative solutions that advance medical progress, reduce health disparities, extend life and make health care more affordable, preventive and patient-centered. CMPI also provides the public, policymakers and the media a reliable source of independent scientific analysis on issues ranging from personalized medicine, food and drug safety, health care reform and comparative effectiveness.

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