Emerging new therapy classes (e.g. CAR-T and gene therapies) with potential curative benefits across severe and rare diseases are likely candidates for expedited regulatory approval but their high prices will incur strong payer scrutiny. Previous innovative therapy classes can provide valuable insights in this regard. Oligonucleotide therapies (antisense oligonucleotides
(ASOs), morpholinos, aptamers, and siRNAs) can target and modify transcripts of specific disease-causing genes. This research evaluates their regulatory, reimbursement and commercial success.
Authors: Bebee T1, Macaulay R2
1PAREXEL International, Horsham, PA, USA, 2PAREXEL International, London, United Kingdom
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