The NY Times has a compelling article on clinical trials. They are costly, slow, bureaucratic. But the most recent twist is that with "personal medicine" they no longer work.
Here is the issues. Classic clinical trials assumed the same therapeutic was give or not to a group of patients. The evaluation was then with or without and did it work and was it better than the previous treatment. Simple, yes?
But as the article noted:
A few converging forces make this bottleneck more consequential than ever. The first force is scientific progress itself. Science is creating treatments that the regulatory system was not designed to handle. The most striking case is that of personalized medicine: Thanks to technological advances in recent decades, we can now sequence a patient’s tumor to find out its specific vulnerabilities, and increasingly we can tailor therapies to the needs of specific individuals.
The problem is that the therapeutic is fundamentally different for each patient since we are examining the genes in the tumor cells. There are two basic approaches. One is the recent one treating melanoma as we have discussed previously using the immune system. The other is a antibody drug conjugate approach as we proposed for prostate cancer. Each therapeutic is different and designed specifically for the patients malignancy, cell by cell. Apparently one has gotten through the quagmire of FDA approval. Will others?
The article is broad and compelling, The US is often the slowest to get through as well as the costliness. Hopefully there will be a focus on improving personalized treatments. But the politics of the FDA and HHS may be the worst problem in the process and many lives may be lost as a consequence.
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