Healthcare Insights

Sean Vander Linde

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How to be a Rare Disease advocate? Design a patient-focused business strategy

By Sean Vander Linde on May 23, 2019 4:44:04 PM

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.  

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Topics: Insights, Rare diseases, Pharmaceuticals, Gene therapy, Commercial growth, Strategy

An Orphan Journey: Commercializing Rare Disease Drugs

By Sean Vander Linde on Feb 13, 2019 3:37:48 PM

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 [1]. 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 [2]. 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. 

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Topics: Insights, Rare diseases, Pharmaceuticals, Gene therapy, Commercial growth, Strategy

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About Kaiser's Healthcare Insights

Kaiser's Healthcare Insights blog shares company expertise on hot market trends, behaviors, and news that are shaping the global healthcare space. These posts share commercial growth strategies and tactics for leading pharmaceutical, medical device, and health IT companies to drive top-line growth and a unique competitive advantage within their respective spaces.

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