OBJECTIVES : Given increasing payer sensitivity, there is reduced opportunity to gain reimbursement in the usual markets. This study aims to assess the current commercialisation opportunity (CO) for orphan drugs outside Europe as well as any improvements to market access in the mid-term (5 years).

METHODS We measured CO on two dimensions. The first is market access potential (MAP); composed of population size, healthcare expenditure, reimbursement landscape, orphan drug regulatory impact, early access programs and orphan definition. Information on these was gathered through literature reviews, secondary research and CRA internal expertise. The second dimension is level of commercial risk, determined by the Transparency international ‘corruption perception index’. Time to generate early income, determined by time to commercial sales post-registration, was also taken into consideration.

Expected improvements to MAP were further analysed for the mid and highest rated markets through secondary research of expected changes to healthcare expenditure, rare disease policy, and orphan definition.

RESULTS : Japan, South Korea, Australia, Canada, Brazil and Colombia rated the highest in the two dimensions. Turkey, Israel, Saudi Arabia rated closely behind, followed by Mexico, Chile, Bahrain and India.

Taiwan, China and South Africa also represent a medium MAP, but were excluded from further analysis due to their low score on commercial risk.

Further analysis indicates MAP is expected to increase in Canada, following a $1 billion investment to improve national coverage for orphan drugs; and Australia, following a 2017 relaxation of orphan drug definition. While moderate, Bahrain, Israel and Turkey also show potential for improved MAP, likely to vary by indication.

CONCLUSIONS : The level of infrastructure, specific policies for rare disease and low commercial risk in the highest rated markets indicates considerable CO. Updates to rare disease definition and creation of rare disease specific policies should be monitored, as these will improve MAP in the mid-term.