Responsibility

 Two weeks ago, I went to Warsaw, Poland to participate in an award ceremony.  The State of Israel presents an award to people who bravely helped Jews, like my parents, who were hunted by the Nazi regime and their local sympathizers.  My wife, in the course of writing a book and blog  about my parents' experiences in the holocaust, identified the descendants of  the people who help my parents hide.  This led to their certification, by the Israeli holocaust center, Yad V'shem, as "Righteous among the Nations."  The honor was bestowed in the Royal Palace in Warsaw.  I spoke at the ceremony about the extraordinary bravery of these people and how they permitted my birth.  

I recognize that I exist because of the courage of these people.  I feel that I owe the world a measure of courage, as a partial payment for this benevolence.  

I am a physician, a hematologist and oncologist.  I try to help people with very serious, often life threatening, diseases.  I am in a field that has made remarkable  progress since I  entered it, more than 30 years ago.  I try to channel that progress into better outcomes for my patients.

I perceive  several forces in this field.  There is a strong force for progress, bringing new medicines to improve outcomes.  There are economic forces, resulting in exorbitant price tags for  many of these new medicines.  Typically, these new medicines are priced at $300 per pill, $100,000. per year.  Some are even more costly. There are forces to test and validate  the efficacy  of these new approaches through "well designed clinical trials" Below the surface, there are considerations for payers (insurance companies and governments), stockholders in drug companies, researchers and their careers. 

I am obligated, by virtue of the rescues that brought me life, to focus on the benefit of the patient.  I must rescue the patient from all the considerations that ignore or impede her benefit.  That means practicing beyond medicine, practicing honesty. 

I have patients who have been encouraged to enter clinical trials, when information is emerging that an approved therapy (for a different disease) has demonstrated efficacy for their problem.  There is often an issue of the adequacy of evidence, in the absence of a "well designed clinical trial." One does not have the right to claim efficacy prematurely.  But sometimes, it is clear that the probability of a good outcome is far better with the treatment that is "rumored" to be effective.  I have used the "rumored" medicines ( imatinib, venetoclax, Pembrolizumab) as an early adopter and seen them become standard months or years later.  This is a kind of rescue that I can be proud of.

There is certainly a place for clinical trials.  There are situations where a clinical trial is the best alternative for the patient, as well as for the scientific community and the drug company.  But the risk/benefit needs to be assessed in perfect honesty, free from the pressures of economics and institutional politics.  This kind of patient protection can  call for courage.  I want to have that courage.  I want to encourage that bravery in my colleagues .