We find ourselves today at a critical juncture in the market introduction of groundbreaking therapies. After years of promise, 2017 finally marked the first approval of a gene therapy in the US, Novartis’ Kymriah, with the approval of Kite’s Yescarta and Spark Therapeutics’ Luxturna soon thereafter. To date, there are more than 500 clinical trials for gene therapies in the US alone, 34 of which are in pivotal stages, and there is significant activity in other key geographies. The arrival of these therapies portends disruption in the industry—being able to treat heretofore incurable conditions, but at prices that concentrate significant treatment costs for these patients over a short period of time and in the hands of only one industry stakeholder, the pharmaceutical manufacturer.
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Evidence requirements for orphan drugs
CRA’s Life Sciences Practice focuses on global policy, commercialization, and market access issues dedicated to orphan drug issues, including orphan evidence...