1 in 2 rare diseases do not have a dedicated foundation or research support group1. In the US, that number translates to close to 15 million patients navigating a complex journey without a dedicated team supporting them and ultimately seeking out a life-changing treatment. As the rare disease market continues to grow and evolve, so will the focus on patient engagement and advocacy, led in part by patient advocate leaders and community groups.
In this second installment of Kaiser's rare disease blog series, we explore the importance of stakeholder education in rare disease drug development. Read more on how companies can effectively work with key stakeholders in the rare disease space to better meet the needs of the patient population and succeed in the market.
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.