Commercialization Imperatives for Success in Rare Diseases
This article was published in the Pharmaceutical Executive which can be accessed via https://www.pharmexec.com/view/commercialization-imperatives-success-rare-diseases
Overview
Since the Orphan Drug Act was passed in 1983, additional legislation including Humanitarian Use Device (HUD) Designation Program, Pediatric Device Consortia (PDC) Grant Program, as well as Orphan Products Natural History Grants Program have helped further stimulate orphan product development. Many companies have been able to access these grants, as well as qualify for incentives such as 50% tax credit for qualified R&D costs, grants for clinical testing, exemption from marketing application fees, and 7-years market exclusivity.
Because of significant unmet need, attractive economics, and recent success in developing cell and gene therapies, many companies have been actively developing novel therapies for rare diseases. In 2018, more than half of the new medical treatments approved by the FDA were for rare diseases. However, significant challenges persist in commercializing these new orphan drugs to ensure timely market access.
This article highlights key challenges and imperatives in commercializing rare disease drugs and commonly used approaches to address these challenges in five key areas:
improving diagnosing,
developing and optimizing treatments,
gaining market access,
optimizing supply chain systems, and
engaging patients.
Authors: Subbarao Jayanthi, Frank Koos, Melissa Morales
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Featured in the Pharmaceutical Executive in Sep 2019