Polak, Tobias B. http://orcid.org/0000-0002-2720-3479
Cucchi, David G. J. http://orcid.org/0000-0001-6706-5464
Schelhaas, Jasmin
Ahmed, Syed S.
Khoshnaw, Naima
van Rosmalen, Joost http://orcid.org/0000-0002-9187-244X
Uyl-de Groot, Carin A. http://orcid.org/0000-0001-6492-5203
Funding for this research was provided by:
HealthHolland (EMCLSH20012)
Article History
Accepted: 18 April 2023
First Online: 18 May 2023
Declarations
:
: CAU-dG has received unrestricted grants from Boehringer Ingelheim, Astellas, Celgene, Sanofi, Janssen-Cilag, Bayer, Amgen, Genzyme, Merck, Glycostem Therapeutics, Astra Zeneca, Roche, and Merck. TBP works part-time for expanded access service provider myTomorrows, in which he holds stock and stock options (< 0.01%). He is contractually free to publish, and the service provider is not involved in any of his past or ongoing research, nor in this manuscript. DGJC received payments for lectures for Takeda, and conference visit support from Servier, all outside the submitted work. JS, SSA, NK, and JVR do not report any conflicts of interest.
: CAU-dG, JvR and TBP work on a Dutch government grant from HealthHolland. For this grant, they research legal, ethical, policy, and statistical issues of evidence generation in expanded access programs (EMCLSH20012). HealthHolland is a funding vehicle for the Dutch Ministry of Economic Affairs and Climate Policy that addresses the Dutch Life Sciences & Health sector.
: This research does not meet the criteria to qualify as medical research with human subjects and is hence exempt from ethics approval in the Netherlands.
: This research does not include research with (human) subjects and therefore does not require informed consent.
: All code to replicate our findings can be found on the GitHub of the main authorFootnote removed. Aggregated data are available upon reasonable request.
: TBP conceived the idea for this review. TBP, DGJC, SSA, JS, and NK collected and labeled the data. TBP and JvR performed the statistical analysis. TBP drafted the manuscript. DGJC, JvR, and CAU-dG critically revised the manuscript. All authors approved the final version of the manuscript.
: Not applicable.
: Not applicable.
: Not applicable.