New rules on health technology assessments in Spain make room for real-world evidence and early dialogue.

MSD welcomes the decision but says the drug should have gone through NICE’s Highly Specialized Technologies route that allows more flexibility regarding uncertainty around rare disease drugs.

Brazil looks to EU, UK and others for inspiration on introducing a regulatory sandbox.

The Dutch health technology assessment institute, ZIN, has called for a debate on how society should tackle obesity and whether expensive treatments should be part of the national prevention strategy.

Overreliance on “problematic” trials while overlooking the value of real-world evidence could affect the development of other rare disease treatments, according to Advanz’s CEO.

High patient expectations also drove Telethon’s decision to bring forward its marketing authorization application plans for its ultra rare disease gene therapy, etuvetidigene autotemcel.