Due to the small population size and geographic spread of individual rare diseases, clinical trial research in rare diseases requires a tailored approach to overcome these obstacles and still contain costs and shorten timelines. With over 900 common genetic disorders identified, state-of-the art healthcare facilities and regulatory processes in line with international standards, the Middle East, North Africa (MENA) region has the potential to contribute greatly to the advancement of rare disease research. Since 2008, the global top 10 Pharmaceutical industry sponsors in rare disease research have collectively conducted more than 70 rare disease studies in the region. Clearly the capacity is available, only the potential has yet to be further utilized.

This paper is relevant for all clinical trial sponsors looking to expand into new markets as well as academics and patient advocacy groups looking to understand the rare disease prevalence in the region. Inside you will find analysis of the rare disease clinical research landscape as well as clinical trial trending and the main sponsors involved since 2008. The capacity of MENA to contribute to rare disease research is discussed in depth on the country level, with three case studies of missed opportunities, in which, despite high disease prevalence there was little to no regional participation in clinical trials.

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A link to the customized interactive LongTaal Rare Disease Dashboard containing an overview of active rare disease clinical studies in the region, sponsor and country comparisons and an overview of global rare disease epidemiology has been made publically available through the end of May 2019 complementary of Clinart MENA.

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