Answer to a call for help

I responded to a blog  from a man dealing with  a difficult cancer .   He expressed hope that an mRNA vaccine could help him.  He was frustrated with the FDA.  So am I . Here is my comment

I am a medical oncologist and hematologist in Seattle. The information you supply certainly justifies your desperation. It is not sufficient for me to supply any practical advice.

 The options available to you are both too numerous and too few.  I agree that the American (and international) systems for therapeutic approvals are so slow and cumbersome that people are denied potentially useful interventions and die waiting for an acceptable statistical confidence interval. The systems (like the FDA) justify this based on the dangerous unknowns that devolve from unjustified approvals. (They are very fond of the fact that thalidomide [which can cause horrible birth defects] was not approved in this country until it was shown effective in multiple myeloma; and by then, the birth defect side effect known, its administration severally restricted [ allowing the drug company, Celgene, to gain enormous, outrageous profits]. The parenthetical comment is important.  It demonstrates the web of interests that are victimizing you and others like you.

 Drug companies have adopted a "your money or your life” pricing policy which makes the typical new oncology  drug cost $1,000.00 Per DAY ( $360,000. per year).  The rules of the FDA (the product of lobbying by drug companies, insurance companies and other .. with a few good intentions...) restrict its mission to "safety and efficacy." Theoretically cost does not enter into its considerations.  But FDA approval has become very defined.  New drugs are approved ONLY in certain combinations (often forcing the use of toxic medicines in addition to the newly approved one) for very specific stages of very specific diseases. Only certain brands of therapies are approved for specific stages of specific diseases, although nearly identical medicines exist. This practice means that there is no competition for price. Use of the medicine for a less advanced stage of the disease can be denied insurance coverage because it is not approved.  It is particularly egregious in the field of checkpoint inhibitor therapy, an immune therapy that allows the immune system to work harder than it ordinarily would by removing some of the checkpoints. [ I presume this type of therapy has been explored with you]

 

 Approvals (now) require prior “lines” of treatment. A number of older (often more toxic) treatments must be administered   before the patient qualifies for the newly approved treatment. All these restrictions serve to  severely limit the range of utilization of these drugs and, at the same time, keep the prices exorbitantly high by eliminating competition. It is worth remembering that Medicare still cannot negotiate the price of these drugs.

 

At the same time, there are subtle and overt advertising campaigns aimed at you and aimed at your doctors  and probably aimed at the approval process to advance certain therapeutic options regardless of their probability of success in your case. I think a degree of skepticism must accompany the hope generated by new therapies like mRNA vaccines. It is hard to get a good handle on the probability of success. But great effort is spent to make the therapy appear appealing and promising.

 

You, and people like you, are in the midst of a battle that is primarily motivated by economic considerations. Hugely successful therapies that are effective in a broad range of cases will be approved rather rapidly, at least for a very limited spectrum of disease. Otherwise, the system fails so miserably it would be abandoned. But anything less than that is going to be subject to a long and arduous process that will restrict its usage and increase its cost.