A Comparison of Currently Approved Small Interfering RNA (siRNA) Medications to Alternative Treatments by Costs, Indications, and Medicaid Coverage.

Small interfering RNA (siRNA)-based medications offer the ability to target previously undruggable targets and have now received FDA approval in five instances for orphan or uncommon diseases. The current siRNA "-sirans" are directed towards hepatic molecular targets. Because they are not conventional drug formulae, their ultimate clinical success will require overcoming multiple barriers beyond their pharmacology.