Footnotes

• Contributors All authors substantially contributed to the conception of the work. JB drafted the manuscript. AS, AB-H, MP, JR, AW-K and AG revised the manuscript critically for important intellectual content. All authors approved the final version of the manuscript.

• Funding The authors have not declared a specific grant for this research from any funding agency in the public, commercial or not-for-profit sectors.

• Disclaimer The views expressed in this article are those of the contributors and should not necessarily be interpreted as the position of the European Medicines Agency or any of its committees.

• Competing interests AB-H is a member of the Compassionate Use Advisory Committee (CompAC), an external, expert panel of internationally recognised medical experts, bioethicists and patient representatives formed by NYU School of Medicine in collaboration with Janssen. CompAC advises Janssen about requests for compassionate use of its investigational medicines, and NYU receives administrative funding from Janssen to facilitate the CompAC Committee. Moreover, AB-H co-chairs the NYU Langone Health Working Group on Compassionate Use and Pre-Approval Access

• Patient consent Not required.

• Provenance and peer review Not commissioned; externally peer reviewed.