Solutions for Complex Diseases may be Found Within

Precision medicine tackles diseases that are baked into a patient’s genetics. As scientists learn more and more about the behavior of genes, researchers are tirelessly working to identify therapies that overcome genetic shortcomings. This means that patients with a genetic hiccup that triggers a life-threatening medical condition can get treatment for an underlying cell-based dysfunction, not just the symptoms it causes. There is no better example of this than CFTR modulators. They are a class of precision medication designed to correct CFTR, the malfunctioning protein responsible for Cystic Fibrosis. A dramatic account of this therapy’s life changing nature is captured by guest writer and CF patient Gunner Esiason in The Hill.

Esiason also explains that patient access to these revolutionary CF therapies are jeopardized by the use of economic models that determine that life-extending therapies (vs. curative therapies) are not cost-effective. In these economic models, calculations by fiscal intermediaries carry more weight than scripts written by physicians and plans of care crafted by a patient’s care team. The power of the prescription pad belongs with a patient’s physician, not a government bureaucrat driven by cost containment goals. Patients and Providers United amplifies the voices that seek to keep patients and providers – not bureaucratic middlemen – at the center of medical decision making.