Rare Diseases. Innovative Approaches to Therapy and Patient Care and the Establishment of Domestic Orphan Drug Production

5 June, 14:30–16:00 Congress Centre, zone D, conference hall D4
Broadcast
Orphan diseases are a heavy medical and social burden for any state. Roughly 70% of orphan diseases are genetic, progressive, and lead to either disability or early mortality. Moreover, the rarity of such diseases is what causes problems with their diagnosis. Insufficient data about them prevents us from determining their prevalence and, as such, does not provide pharmaceutical companies with the opportunity to forecast the potential market and the efficiency of investments. Some countries struggle to conduct local clinical trials due to their low number of patients with orphan diseases. These factors limit the ability to provide drugs to patients with orphan diseases. To expand such opportunities and jumpstart the market for innovative orphan drugs, it is crucial to strengthen tools for international cooperation, from developing therapies, conducting full-scale clinical studies, collecting and analysing data from real clinical practice, and creating common registries, to consolidating demand and developing mechanisms for joint international procurements. Given the economic sanctions in Russia, the problem of access to innovative pharmaceutical technologies must be solved by improving national technological sovereignty. How can we ensure the creation of domestic developments for molecular genetic research into orphan diseases? How can we promote the creation of patient registries? What regulatory and financial practices could stimulate the development of innovative orphan drugs? How can we ensure the availability of orphan drugs, including for adult patients? What effective tools to support the provision of orphan drugs can be borrowed from international experience?

Broadcast

Image