Skip to main content

Causal Estimands: A Common Language for Missing Data

  • Chapter
  • First Online:
Biopharmaceutical Applied Statistics Symposium

Part of the book series: ICSA Book Series in Statistics ((ICSABSS))

  • 660 Accesses

Abstract

What are estimands, and what is there connecting with missing data? The topic of estimands is all about interpreting the results of our statistical analyses in the context of the original scientific questions that we intend to answer in a clinical trial. Missing data, a ubiquitous problem in clinical trials, require additional assumptions and choices on how to collect and analyze data that can have an unexpected impact on the interpretation of these analyses.

This is a preview of subscription content, log in via an institution to check access.

Access this chapter

Chapter
USD 29.95
Price excludes VAT (USA)
  • Available as PDF
  • Read on any device
  • Instant download
  • Own it forever
eBook
USD 84.99
Price excludes VAT (USA)
  • Available as EPUB and PDF
  • Read on any device
  • Instant download
  • Own it forever
Softcover Book
USD 109.99
Price excludes VAT (USA)
  • Compact, lightweight edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info
Hardcover Book
USD 109.99
Price excludes VAT (USA)
  • Durable hardcover edition
  • Dispatched in 3 to 5 business days
  • Free shipping worldwide - see info

Tax calculation will be finalised at checkout

Purchases are for personal use only

Institutional subscriptions

Notes

  1. 1.

    We ignore pathological cases such as multiple maxima to keep the discussion simple.

  2. 2.

    Nuisance parameters such as the variance are ignored for now to simplify the exposition.

  3. 3.

    The model can be extended further by allowing R to take on more than two values indicating multiple response patterns. This will be necessary for applying PMMs.

References

  • Carpenter, J., & Kenward, M. (2012). Multiple imputation and its application. Statistics in practice. Wiley.

    Google Scholar 

  • de Yebenes et al (2011). Pridopidine for the treatment of motor function in patients with Huntington’s disease (MermailHD): A phase 3, randomised, double-blind, placebo-controlled trial. Lancet Neurol, 2011(10), 1049–57.

    Google Scholar 

  • Gelman, A., & Hill, J. (2007). Data analysis using regression and multilevel/hierarchical models. Analytical Methods for Social Research. Cambridge University Press.

    Google Scholar 

  • Hollis, S., & Campbell, F. (1999). What is meant by intention to treat analysis? Survey of published randomised controlled trials. BMJ, 319(7211), 670–674.

    Google Scholar 

  • Lewis, J. A., & Machin, D. (1993). Intention to treat—Who should use ITT? British Journal of Cancer, 68(4), 647–650.

    Google Scholar 

  • Little, R. J., & Rubin, D. B. (1987). Statistical Analysis With Missing Data. Wiley.

    Google Scholar 

  • Little, R. J., & Rubin, D. B. (2000). Causal effects in clinical and epidemiological studies via potential outcomes: Concepts and analytical approaches. Annual Review of Public Health, 21(1), 121–145.

    Google Scholar 

  • MacKay, R. J., & Oldford, R. W. (2000). Scientific method, statistical method and the speed of light. Statistical Science, 15(3), 254–278.

    Google Scholar 

  • Mallinckrodt, C. H., Clark, W. S., & David, S. R. (2011). Type I error rates from mixed effects model repeated measures versus fixed effects anova with missing values imputed via last observation carried forward. Drug Information Journal, 35(4), 1215–1225.

    Google Scholar 

  • Mallinckrodt, C. H., Lin, Q., Lipkovich, I., & Molenberghs, G. (2012). A structured approach to choosing estimands and estimators in longitudinal clinical trials. Pharmaceutical Statistics, 11(6), 456–461.

    Google Scholar 

  • Mayo Foundation for Medical Education and Research. Huntington’s disease. Retrived June, 2017 from http://www.mayoclinic.org/diseases-conditions/huntingtons-disease/basics/definition/con-20030685.

  • National Research Council. (2010). The prevention and treatment of missing data in clinical trials.

    Google Scholar 

  • Pawitan, Y. (2001). In all likelihood: Statistical modelling and inference using likelihood. OUP Oxford: Oxford science publications.

    Google Scholar 

  • Phillips, A., Abellan-Andres, J., Soren, A., Bretz, F., Fletcher, C., France, L., Garrett, A., Harris, R., Kjaer, M., Keene, O., Morgan, D., O’Kelly, M., Roger, J. (2017). Estimands: Discussion points from the PSI estimands and sensitivity expert group. Pharmaceutical Statistics, 16(1), 6–11. PST. 1745.

    Google Scholar 

Download references

Author information

Authors and Affiliations

Authors

Corresponding author

Correspondence to Steven A. Gilbert .

Editor information

Editors and Affiliations

Rights and permissions

Reprints and permissions

Copyright information

© 2018 Springer Nature Singapore Pte Ltd.

About this chapter

Check for updates. Verify currency and authenticity via CrossMark

Cite this chapter

Gilbert, S.A., Tan, Y. (2018). Causal Estimands: A Common Language for Missing Data. In: Peace, K., Chen, DG., Menon, S. (eds) Biopharmaceutical Applied Statistics Symposium . ICSA Book Series in Statistics. Springer, Singapore. https://doi.org/10.1007/978-981-10-7820-0_15

Download citation

Publish with us

Policies and ethics