Regulatory policies and guidelines – are these enabling timely development of Orphan medicines globally?

Description:The Orphan Medicines Regulation has been in place for more than 20 years in the European Union, and new legislative proposals are expected to change the way developers work collaboratively across stakeholders to develop, approve and provide access to medicines for rare diseases in Europe. The key question is whether these regulations will positively impact the lives of patients with rare disease.

The medicine development landscape has also been evolving at an unprecedented pace in terms of new types of medicines (e.g. ATMPs) as well as evidence generation techniques through the use of RWD/RWE and digital health technologies (DHTs). These new guidelines and ways of working are also becoming the key tools for ensuring timely development of Orphan medicines in Europe.

As other agencies or organisations globally are putting in place legislations or guidelines to incentivise the development of Orphan medicines, it will be important also for them to consider what an optimal framework looks like and whether convergence of requirements, processes and practices is possible.

This session will provide an overview of the main expected changes in the orphan policy environment in Europe and put these in perspective in an international, multistakeholder discussion to debate what legislative procedures and guidelines are helpful to enable timely availability of Orphan medicines globally.