There have recently been a group of "precision drugs" targeted to a specific mutation (e.g., vemurafenib for BRAF V600E mutations in melanoma and ivacaftor for CFTR G551D mutations in cystic fibrosis). While these drugs are highly effective, they are also very expensive, reflecting the R&D costs of developing a drug for only a small group of patients who carry the specific genetic defect. Will the development of many of such drugs, each targeted to a single mutation or small group of mutations, be the modus operandi of the future? Given that there are generally many different mutations in each gene that can cause disease and each mutation generally only explains a fraction of the cases, it seems that we'll need many such drugs. Could this lead to a rise in healthcare costs in the US? How will the economics of drug development need to change to promote the development of these drugs and bring them to the market, while keeping healthcare costs down?
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