Treat the Pathway, not the Gene (from Precision Medicine and the Reinvention of Human Disease)

Treat the key pathway, not the genetic mutation (fromPrecision Medicine and the Reinvention of Human Disease)Some of the earliest and most successful Precision Medication drugs have targeted specific mutations occurring in specific subsets of diseases. One such example is ivacaftor, which targets the G551D mutation present in about 4% of individuals with cystic fibrosis [135]. It is seldom wise to argue with success, but it must be mentioned that the cost of developing a new drug is about $5 billion [136]. To provide some perspective, $5 billion exceeds the total gross national product of many countries, including Sierra Leone, Swaziland, Suriname, Guyana, Liberia, and the Central African Republic. Many factors contribute to the development costs, but the most significant is the incredibly high failure rate of candidate drugs. About 95% of the experimental medicines that are studied in humans fail to be both effective and safe. The costs of drug development are reflected in the rising costs of drugs.When a new drug is marketed to a very small population of affected individuals, the cost of treating an individual may be astronomical. Americans should not pin their hopes on the belief that one day, the FDA or CMS (which administrates Medicare) will step in and put a stop to the price rises. The Food and Drug Administration can approve or reject drugs, but it does not regulate prices. Likewise, Medicare is not permitted to consider cost when it decides whether a treatment can be...
Source: Specified Life - Category: Information Technology Tags: clinical trials convergent pathways cost of precision medicine precision treatment Source Type: blogs