In the current paradigm, cancer therapies are measured for superiority in comparison to placebo or existing treatment, based on average survival of a tested group of patients. This is true for many deadly and rare diseases. However, the comparison is based on a misconception of a silver bullet “cure” for a given population or group of patients. Approving new therapies on the basis of comparative average population efficacy for one drug against another drug or standard of care leaves the vast majority of patients deprived of treatments that could work for them, but which do not produce average population superiority.
“in most CER [comparative effectiveness research] studies, it is the drug or treatment with the larger average effect on an entire population that “wins.” In the president’s [Obama’s] hypothetical, the blue pills are “just as effective” as the red ones because, on average, they do as much good for patients. But the average patient is not the same as any particular individual patient. Declaring a treatment most effective based on an average is a medical and an economic error…First, individuals differ from one another and from population averages. Therefore, what may be on average a “winning” therapy may simply not work for a large number of patients. Conversely, a drug that is less effective on average may still be the best, or only, choice for a sizable proportion of patients.” Project FDA Report Blue Pill or Red Pill: The Limits of Comparative Effectiveness Research; Tomas J. Philipson, University of Chicago, Eric Sun, Stanford University No. 4 June 2011
This is a profound problem in cancer when therapies usually do not impact outcomes for more than 30% of the population. That means 70% of patients do not have a treatment that will work for them. When approval is based on improving a population average, it deprives all those for whom existing therapies do not work from having any other options. In cancer, that is the vast majority of patients who cannot be surgically remediated. The same can be said for ALS, Parkinson’s, MS, and countless other diseases.
Copyright Fight to Live 2019