Without caution, fast track regulatory paths can be the “route to non-reimbursement” for cell and gene therapies, according to Eric Faulkner, vice president of global real-world-evidence at Novartis Gene Therapies. He advised that to succeed with payers, companies selling cell and gene therapies need to prove transformative effect and think “outside the box” when it comes to generating evidence and communicating with payers.
Novartis Gene Therapies
Novartis is the company behind the gene therapy Zolgesnma (onasemnogene abeparvovec) for the rare disease spinal muscular atrophy. It won approval from the US Food and Drug Administration in May 2019 after being awarded fast track, breakthrough therapy and priority review designations.
It was conditionally approved in the EU in May 2020 after receiving support through the EU’s priority medicines (PRIME) Scheme. Prior to its approval, it was initially accepted for regulatory review under the European Medicines Agency’s accelerated access mechanism but later revered to the standard review timetable.
Novartis also holds the ex-US rights to Luxturna, a gene therapy for inherited retinal dystrophy developed by Spark Theraputics. Luxturna won EU approval in November 2018. It was approved in the US in December 2017 after the FDA granted the application priority review and breakthrough therapy designations.
Securing reimbursement for gene and cell therapies is challenging for a number of reasons, including high prices for products, small...
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