To encourage manufacturers to invest in the development of orphan drugs (ODs), various incentives have been introduced. However, ODs and gene therapies (GTxs) still need to overcome a series of challenges posed by national health technology assessment (HTA) bodies, which are unique in each country.
In Cell & Gene, Steven Kelly, Ioanna Stefani, Nimisha Raj, and Charlotte Poon review and compare the reimbursement recommendations for ODs issued by four European HTA bodies (France, Germany, England and Scotland) and assess the potential effect of HTA outcomes on the time to reimbursement (T2R). The authors found that, overall, concessions introduced in the HTA process for ODs have a positive impact on minimizing rejections and accelerating the T2R. However, obstacles remain to capture the full value of ODs and GTxs within the HTA process. Achieving more favorable outcome ratings, avoiding restrictions, or addressing uncertainty with a patient access scheme all lead to prolonged appraisal times. Manufacturers are therefore still required to carefully consider their launch strategy. Additionally, further flexibility to address uncertainty in the HTA processes is needed to improve reimbursement timelines and outcomes for ODs and GTxs.
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