International Regulatory Frameworks to Accelerate Orphan Drug Registrations

Description: A rare disease is a disease that affects a small percentage of the population.>7000 rare diseases are know to exist today but more than 90% don't have an approved treatment option. 400 million people around the world are affected by a rare disease, half of whom are children. The impact on quality of life and functioning can be devastating for the affected individual and for those around them. Therefore, the importance of enabling rapid access and geographical expansion of life-saving medicines targeting rare diseases. Collaboration between different stakeholders (patients, industry, health authorities) will be key.

Customizing ways of working, by targeting parallel submissions, using core dossiers, ensuring appropriate resources, as well as leveraging rare disease uniqueness by applying most appropriate international regulatory frameworks, can help accelerate access to medicinal products for the benefit of the patients. Expedited Regulatory pathways such as orphan drug designations, fast-track, priority reviews, and reliance are being used to best streamline submissions and expected approval timelines. This effort does not come without challenges, requiring innovative ways of doing, improving collaboration, both with internal and external stakeholders.

A regulatory strategy planned well in advance during early clinical development will allow us identifying early enough any potential hurdle, understand all country-specific requirements, find solutions, and identify how to already accelerate the product development up to the product approval by implementing best international regulatory frameworks, be it at country and/or regional level.