In this series, Kaiser will explore how rare disease companies can develop commercial growth strategies to succeed against the challenges of a high cost and highly specialized market.
2018 saw 91 orphan drug approvals by the FDA – nearly 3 times greater than in 2013 and the highest number of approvals yet – along with over 400 designations for the candidates in development . 2019 is expected to bring even more approvals as more companies begin to realize R&D investments in rare diseases. Driven by high pricing potential, significant unmet needs, and favorable regulatory policy, orphan drug sales are expected to balloon to reach $262B in the next 5 years . The market continues to grow more competitive and new technologies are emerging, such as Spark Therapeutics’ Luxturna, which marks the first FDA approval in a new class of one-time gene therapy treatments targeting inherited diseases. In this environment, companies will need to shift their focus to how they can play strategically and optimize the value of their orphan therapies.