Challenges that characterize weight-loss drug trials necessitate good trial design — as highlighted by the STEP 5 trial of semaglutide.
This is a preview of subscription content, access via your institution
Access options
Access Nature and 54 other Nature Portfolio journals
Get Nature+, our best-value online-access subscription
$29.99 / 30 days
cancel any time
Subscribe to this journal
Receive 12 print issues and online access
$209.00 per year
only $17.42 per issue
Buy this article
- Purchase on Springer Link
- Instant access to full article PDF
Prices may be subject to local taxes which are calculated during checkout
References
Patel, M. R., Donahue, M., Wilson, P. W. & Califf, R. M. Am. Heart J. 151, 633–642 (2006).
US Department of Health and Human Services Food and Drug Administration. https://www.fda.gov/media/71252/download (2007).
Garvey, W. et al. Nat. Med. https://doi.org/10.1038/s41591-022-02026-4 (2022).
US Department of Health and Human Services Food and Drug Administration. https://www.fda.gov/regulatory-information/search-fda-guidance-documents/multiple-endpoints-clinical-trials-guidance-industry (2017).
European Medicines Agency Committee for Medicinal Products for Human Use. https://www.ema.europa.eu/en/documents/scientific-guideline/ich-e9-r1-addendum-estimands-sensitivity-analysis-clinical-trials-guideline-statistical-principles_en.pdf (2020).
European Medicines Agency. https://www.ema.europa.eu/en/ich-e9-statistical-principles-clinical-trials (1998).
Wharton, S. et al. Int. J. Obes. (Lond) 45, 923–933 (2021).
Simons-Morton, D. G., Obarzanek, E. & Cutler, J. A. J. Am. Med. Assoc. 295, 826–8 (2006).
Wilding, J. P. H. et al. N. Engl. J. Med. 384, 989–1002 (2021).
National Institute of Health and Care Excellence. https://www.nice.org.uk/guidance/gid-ta10765/documents/final-appraisal-determination-document (2022).
Rubino, D. et al. J. Am. Med. Assoc. 325, 1414–1425 (2021).
Author information
Authors and Affiliations
Corresponding author
Ethics declarations
Competing interests
A.I.A. is an investigator in the ISAP trial, testing oral semaglutide in people at risk for dementia. A.I.A. is supported by the NIHR Oxford Biomedical Research Centre (BRC).
Rights and permissions
About this article
Cite this article
Adler, A.I. Endpoints and estimands: understanding trials of weight-loss drugs. Nat Med 28, 2005–2006 (2022). https://doi.org/10.1038/s41591-022-02032-6
Published:
Issue Date:
DOI: https://doi.org/10.1038/s41591-022-02032-6