With gene therapies for sickle cell disease looming and two new expensive therapies of to a slow start, access challenges could prevent a large portion of patients from receiving treatment, a report from the US National Academies of Sciences, Engineering and Medicine concludes. The report urges development of new payment models, offering five ideas.

The G-BA has explained in more detail the procedures for mandating post-launch evidence generation under new legislation introduced in 2019.

Novartis has completed its absorption of AveXis and rebranded the acquired gene therapy specialist that developed the soon-to-be SMA blockbuster Zolgensma despite struggling to meet the data integrity standards of regulators and big pharma.

Funding will accelerate an FDA registrational study of the company's first liquid biopsy product, as competition increases in early cancer detection.