Neuroscience-Estimands European SIG


This SIG will have two connected topics:

  1. General biostatistics Neuroscience community
  2. Working groups on estimands in neuroscience and other topics in neuroscience


Publications and slide decks

The group did work on the impact of Covid-19 pandemic on clinical trials in Neuroscience and provides recommendations. This is thought to be a living document with regular updates.

The impact of Covid-19 on clinical trials in NS V1.0 final


Joint EFSPI/BBS Workshop  on the application of the estimand framework: A Neuroscience perspective

November 3, 2020

This seminar covers the use of estimands in Neuroscience. Neuroscience is a heterogeneous therapeutic area in which development pathways and study analyses can substantially differ between different indications. Efficacy is usually based on longitudinal assessments which makes the application of the estimand framework specifically interesting and rich with many specific aspects from which we all can learn a lot.

A European scientific interest group (SIG) sponsored by EFSPI and PSI is working out in some typical Neuroscience indications how to best apply the estimand framework. This work is in progress and the seminar will provide an overview on the status of the working group including an opportunity to discuss issues and provide feedback. Finally, since the SIG also worked on the impact of Covid-19 pandemic in Neuroscience this topic will also be covered in the seminar.  The seminar is not an introduction into the estimand framework. For more information see .

Below are the presentations of the workshop and a Here is the link  to the recording.

Estimands in NS workshop_V3f

Outline of estimands proposal in progressive MS f

Outline of estimands proposal in MigrainePain_BBS f



Impact of Covid-19 on NS Trials NN f





Biostatistics Neuroscience community:

  • To support/strengthen the role o biostatistics in the edvelopment of new medicine for Neuroscience
  • To identify areas where collaboration between companies makes sense

Working group on estimands:

  • Potential overview on how to formulate estimands in different CNS indications
  • Link the estimand question to statistical analyses in general
  • Define situations in which MMRM can still be used even when theoretically addressing a treatment policy estimand
  • Alternative methods to MMRM for treatment policy estimands
  • Sensitivity analyses
  • Out of scope:
    • Specific estimands for AD (separate working group under ASA)
    • Future use of MMRM in AD (separate working group under ASA)



  • White paper and/or publication
  • If needed potential consultation with HAs to get agreement on new standard method replacing MMRM and/or agreement on continued use of MMRM under certain conditions


The working group on AD under ASA includes a substream on estimands in AD and possible replacements for MMRM. This work will however, unlikely cover all work needed for Neuroscience in general, as there are specific differences in AD versus overall Neuroscience.

Certain areas of high unmet medical need and restricted development options like rare diseases in Neuroscience would not be addressed by the AD working group.



Biostatistics Neuroscience community

  • Meets regularly ~4 times a year via TC
  • Arrange f2f meetings at conferences if possible
  • Only few representatives from each company to participate
  • Transparent to the outside world
  • Open to large and small pharmaceutical industry
  • Exchange methodological information
  • Do not exchange molecule/project specific information
  • Can initiate working groups for working on CNS specific methodological issues together where it makes sense to do it together

Working group on estimands

  • Every company interested can join and add statisticians to the group
  • Monthly TC to organise work would seem meaningful



  1. Membership (see table below)
  2. Information sharing: via the EFSPI and PSI websites, EFSPI and PSI Newsletters, webinars, presentations at conferences, publications in mainstream journals.


Name Company/Institution
Andrew HartleyPPDI
Akiko OkamotoJ&J
Charlie CaoBiogen
Christopher MillerAsztrazeneca
Christopher CoffeyUniversity of Iowa
William S ClarkLilly
David S KellerPfizer
Fabian ModelHoffmann-La Roche
Georg KralidisIpsen
Ih ChangBiogen
John O'GormanBiogen
Khadija RantellMHRA
Klaus Groes LarsenLundbeck
Hong Liu-SeifertLilly
Markus AbtHoffmann-La Roche
Marisa BacchiJ&J
Mette Krog JosiassenLundbeck
Sofia MosesovaDenali
Nikolaos SfikasNovartis
Paul Delmar (Chair)Roche
Peter QuargNovartis
Ulrich FreudensprungBiogen
Veronique RobertServier
Weining (Winnie) RobiesonAbbvie
Hans Ulrich BurgerHoffmann-La Roche

Get in touch

If you would like to get involved with the SIG, please contact Paul Delmar.


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