Regulatory approval is traditionally the main hurdle for developers bringing new therapies to patients. However, today, physician and payer acceptance is becoming more critical as expedited regulatory pathways provide marketing authorization with more preliminary data to therapies for severe diseases, whilst payers, facing budgetary pressures, demand more evidence to justify prices. Innovative new therapy classes will further amplify this trend: for example, CAR-T cell and gene therapies with potential curative benefits across multiple severe and rare diseases will be likely candidates for expedited regulatory approval but their high prices will incur strong payer scrutiny. Glybera®, the first EC-approved gene therapy, is no longer marketed for commercial reasons, emphasizing these reimbursement and prescribing challenges. Glybera® is an Advanced Therapy Medicinal Product (ATMP), encompassing gene, cell, and tissue-engineered therapies. This research assesses the commercial success of ATMPs.
Authors: Macaulay R1, Gokool N2, Tang M1, Samuels ER1, Langford C2
1PAREXEL International, London, United Kingdom, 2PAREXEL International, Brighton, United Kingdom
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