Precision Medicine: Its Future

A decade ago there was a big push for the area of Precision Medicine. In reality it has turned into more of a personalized medicine and the biggest focus has been on cancer therapeutics. JAMA has noted:

Oncology has been the primary focus of precision medicine. One recent analysis by the IQVIA Institute found that 97 new anticancer medications have been approved since 2011. A 2018 study published in JAMA Oncology found that 31 genome-targeted or genome-informed anticancer drugs were in use as of January 2018. Precision oncology has had some major successes. Imatinib has a 95% response rate in patients with chronic myeloid leukemia and extends quality-adjusted life by about 9 years; venetoclax has an 80% response rate in patients with chronic lymphocytic leukemia who have a 17p deletion. The new chimeric antigen receptor T cell (CAR-T) therapy tisagenlecleucel has a 62% remission rate at 24 months among patients with acute lymphoblastic leukemia. And yet, the overall effect of precision medicine on care for patients with cancer has been modest. The 2018 JAMA Oncology study estimates that only 8% of patients with cancer are eligible for precision medications approved as of January 2018 and only 5% would actually benefit from them. Even among patients who respond, incremental survival provided by many drugs is measured in months. Partly because of cost-effectiveness concerns, of the 54 new anticancer drugs launched between 2013 and 2017, only 80% were available in Germany by the end of 2018 and only 69% were available in France (96% were available in the United States).

These new therapeutics are quite costly to develop and produce and they often work in about 40% of the cases. This may not be great but on the one hand it is better than nothing and on the other hand it allows for examination of why not in the remaining 60%.

Cost is always an issue.  However there are a multiplicity of issues. The treatment with these therapeutics is in reality a massive clinical trial. CAR-T cells, as we have written, are very powerful and on the downside there is the impact of cytokine storms. Just like old time chemotherapy the CAR-T can attack everything the patient has. 

Yet the progress has been beneficial and we anticipate that in the next twenty years we will see it stabilized and solving many things which are now death sentences.