FDA’s New Breakthrough Therapy Designation: What does it mean for Pricing and Market Access?
By Ram Subramanian, Tory Sheppard, Brad Rubin, and Chris Kramer
The U.S. Food and Drug Administration’s (FDA) new breakthrough therapy designation represents perhaps the most important effort to date to reduce the time required to bring new drugs to market. According to the FDA a breakthrough therapy is a drug that is:
Intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and
That may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development, as indicated by preliminary clinical evidence.
The breakthrough therapy designation allows for early and positive awareness of a drug’s therapeutic value, signaling that the product delivers substantial improvement on at least one clinical endpoint.
Currently, there are 20 known breakthrough designations (Figure 1), and 8 of those have gone to novel oncology therapies that are currently being developed. With faster approval times for breakthrough products, pricing and market access (P&MA) activities for these drugs could fall on the critical path. By filing for approval on the basis of compelling Phase 2 data, a breakthrough-designated product could potentially come to market approximately 3 years sooner than a drug without the breakthrough designation (Figure 2).