Our new paper explores regulatory developments in early access treatments in non US and EU markets

Developing treatments for rare diseases and ensuring these reach patients quickly has long been a regulatory conundrum. Balancing the need for rapid access to promising drugs with those for adequate real world data and safeguards is challenging. In the US and EU, initiatives by the FDA (i.e. BTD) and EMA (i.e. CMA and PRIME) have accelerated developments and arguably served as a broad guide to other regions. Countries such as Brazil, China, India and Russia, for instance, have developed early access programmes and bilateral agreements between regulators, while governments across the globe are increasingly promoting Early Access pathways (EAPs) and Compassionate Use Schemes (CUSs). Such initiatives are enabling more patients with the rarest ailments to access experimental or unapproved treatments. It’s still early days, of course, and regulatory and market evolution has some way to go before access pathways are streamlined. Yet the potential benefits for patients, regulators and pharma companies in many markets is there to be realised.

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