Companies have historically been unwilling to invest in new treatments for rare diseases; small patient populations limit revenue potential. In response, countries have enacted legislation to encourage drug development in such orphan indications that provides developmental and regulatory incentives. However, the reimbursement/payer hurdle is increasingly becoming the key challenge to overcome in terms of patient access and commercial success. The objective of this research was to understand how orphan drugs (ODs) undergo pricing and reimbursement (P&R) across 25 major and emerging markets.
Authors: Macaulay R, Roibu C, Ivanova H, Campbell J, PAREXEL International, London, United Kingdom
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