Skip to main content

Thank you for visiting nature.com. You are using a browser version with limited support for CSS. To obtain the best experience, we recommend you use a more up to date browser (or turn off compatibility mode in Internet Explorer). In the meantime, to ensure continued support, we are displaying the site without styles and JavaScript.

  • News & Views
  • Published:

Clinical trials

Endpoints and estimands: understanding trials of weight-loss drugs

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

Buy this article

Prices may be subject to local taxes which are calculated during checkout

References

  1. Patel, M. R., Donahue, M., Wilson, P. W. & Califf, R. M. Am. Heart J. 151, 633–642 (2006).

    Article  Google Scholar 

  2. US Department of Health and Human Services Food and Drug Administration. https://www.fda.gov/media/71252/download (2007).

  3. Garvey, W. et al. Nat. Med. https://doi.org/10.1038/s41591-022-02026-4 (2022).

    Article  PubMed  Google Scholar 

  4. 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).

  5. 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).

  6. European Medicines Agency. https://www.ema.europa.eu/en/ich-e9-statistical-principles-clinical-trials (1998).

  7. Wharton, S. et al. Int. J. Obes. (Lond) 45, 923–933 (2021).

    Article  Google Scholar 

  8. Simons-Morton, D. G., Obarzanek, E. & Cutler, J. A. J. Am. Med. Assoc. 295, 826–8 (2006).

    Article  CAS  Google Scholar 

  9. Wilding, J. P. H. et al. N. Engl. J. Med. 384, 989–1002 (2021).

    Article  CAS  Google Scholar 

  10. National Institute of Health and Care Excellence. https://www.nice.org.uk/guidance/gid-ta10765/documents/final-appraisal-determination-document (2022).

  11. Rubino, D. et al. J. Am. Med. Assoc. 325, 1414–1425 (2021).

    Article  CAS  Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Amanda I. Adler.

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

Reprints and permissions

About this article

Check for updates. Verify currency and authenticity via CrossMark

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

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/s41591-022-02032-6

Search

Quick links

Nature Briefing

Sign up for the Nature Briefing newsletter — what matters in science, free to your inbox daily.

Get the most important science stories of the day, free in your inbox. Sign up for Nature Briefing