At the end of August the FDA has approved a therapy, Kymriah, developed by Novartis to fight lymphoblastic leukemia. The therapy is based on the genetic modification of the patient cells transforming them in killer of malignant leukemic cells. It is a way for boosting the patient immunologic system.
Clinical trials on patients that did not respond to the classical therapy (roughly 15%) shown that the gene therapy based Kymriah is effective in 83% of them. The gene therapy has some potential dangerous side effects (affecting in some patients their blood pressure) and has to be conducted in a hospital environment.
This is a great news opening up, potentially, a new effective way to cure cancers.
However, the cost of the therapy, 475,000$, is high. Novartis justifies it with the 1 billion $ they spent in the research and clinical trials, along with the limited number of patients that will be potentially using it (in the US there are about 3,000 cases of lymphoblastic leukemia and of these over 2,500 can be cured with the current therapies). Additionally, the current therapies are also expensive in case of bone marrow transplant and intensive chemio-therapy requiring in hospital care.
The cost of therapies has been increasing significantly to the point that many are cautioning that we are moving forward to greater drugs effectiveness but at the same time the price of those drugs is prohibitive for most of the population.
Affordability is becoming as important as effectiveness and innovation without affordability is moot.