The era of “pills in a bottle”—dominated by small molecule drugs for chronic conditions—has given way to innovative biologics, specialty drugs, and most recently, cell and gene therapies targeting rare, orphan and complex diseases. Some of these new therapies offer the potential for one-time, curative treatments but come with unprecedented price tags. This action brief provides information on the evolving marketplace, current finance and coverage models, along with an emphasis on solutions that are effective and innovative.