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Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines.

Publication ,  Journal Article
Ratitch, B; Goel, N; Mallinckrodt, C; Bell, J; Bartlett, JW; Molenberghs, G; Singh, P; Lipkovich, I; O'Kelly, M
Published in: Ther Innov Regul Sci
March 2020

This paper provides examples of defining estimands in real-world scenarios following ICH E9(R1) guidelines. Detailed discussions on choosing the estimands and estimators can be found in our companion papers. Three scenarios of increasing complexity are illustrated. The first example is a proof-of-concept trial in major depressive disorder where the estimand is chosen to support the sponsor decision on whether to continue development. The second and third examples are confirmatory trials in severe asthma and rheumatoid arthritis respectively. We discuss the intercurrent events expected during each trial and how they can be handled so as to be consistent with the study objectives. The estimands discussed in these examples are not the only acceptable choices for their respective scenarios. The intent is to illustrate the key concepts rather than focus on specific choices. Emphasis is placed on following a study development process where estimands link the study objectives with data collection and analysis in a coherent manner, thereby avoiding disconnect between objectives, estimands, and analyses.

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Published In

Ther Innov Regul Sci

DOI

EISSN

2168-4804

Publication Date

March 2020

Volume

54

Issue

2

Start / End Page

370 / 384

Location

Switzerland

Related Subject Headings

  • Statistics & Probability
  • Research Design
  • Humans
  • Depressive Disorder, Major
  • Data Interpretation, Statistical
  • Asthma
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
  • 1117 Public Health and Health Services
  • 0104 Statistics
 

Citation

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Ratitch, B., Goel, N., Mallinckrodt, C., Bell, J., Bartlett, J. W., Molenberghs, G., … O’Kelly, M. (2020). Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines. Ther Innov Regul Sci, 54(2), 370–384. https://doi.org/10.1007/s43441-019-00065-7
Ratitch, Bohdana, Niti Goel, Craig Mallinckrodt, James Bell, Jonathan W. Bartlett, Geert Molenberghs, Pritibha Singh, Ilya Lipkovich, and Michael O’Kelly. “Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines.Ther Innov Regul Sci 54, no. 2 (March 2020): 370–84. https://doi.org/10.1007/s43441-019-00065-7.
Ratitch B, Goel N, Mallinckrodt C, Bell J, Bartlett JW, Molenberghs G, et al. Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines. Ther Innov Regul Sci. 2020 Mar;54(2):370–84.
Ratitch, Bohdana, et al. “Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines.Ther Innov Regul Sci, vol. 54, no. 2, Mar. 2020, pp. 370–84. Pubmed, doi:10.1007/s43441-019-00065-7.
Ratitch B, Goel N, Mallinckrodt C, Bell J, Bartlett JW, Molenberghs G, Singh P, Lipkovich I, O’Kelly M. Defining Efficacy Estimands in Clinical Trials: Examples Illustrating ICH E9(R1) Guidelines. Ther Innov Regul Sci. 2020 Mar;54(2):370–384.
Journal cover image

Published In

Ther Innov Regul Sci

DOI

EISSN

2168-4804

Publication Date

March 2020

Volume

54

Issue

2

Start / End Page

370 / 384

Location

Switzerland

Related Subject Headings

  • Statistics & Probability
  • Research Design
  • Humans
  • Depressive Disorder, Major
  • Data Interpretation, Statistical
  • Asthma
  • 4905 Statistics
  • 3214 Pharmacology and pharmaceutical sciences
  • 1117 Public Health and Health Services
  • 0104 Statistics