EFPIA / EFSPI Estimand Implementation Working Group (EIWG)

2023 Achievements

  1. Non-inferiority sub-team
    • PSI 2023 presentation
    • Preparation of paper on estimands in non-inferiority trials
    • Preparation of PSI 2024 proposal including a panel of regulators
  2. Training sub-team
    • Organized the webinar "Proposing Estimands from Different Perspectives", which showed a case study in heart failure presented by the perspective of multiple stakeholders (patient, clinician, regulator, health technology assessor and statistician). The webinar had a record of more than 800 people registered to attend. (March 2023)
  3. Communications sub-team
    • Participated in APBG Estimands Webinar
    • Preparation of PSI 2024 proposal on estimand thinking and statistical leadership
  4. Estimation sub-team
    • Preparation of manuscript on treatment policy estimation
  5. Reporting sub-team
    • Agreed to collaborate with NIH on trial disclosure and estimands
    • Invited presentation at JSM 2023 by Melanie Wright “Realizing the benefits of estimands to increase transparency in the reporting and communication of trial results”
    • Preparation of manuscript on the reporting of estimands and intercurrent events in clinical study reports
  6. Intercurrent Events sub-team
    • Newly formed to tackle uncertainty around the meaning of different intercurrent event strategies, which is a major barrier to effective implementation of the estimands framework
    • Identified several key areas of uncertainty to target in 2024
  7. Estimands in Early Phase [I/II] & Clinical Pharmacology Studies sub-team
    • Submitted and received referee comments on paper “Tutorial on How Estimands can be Applied to Bioequivalence and Related Clinical Pharmacology Trials”. (Hope for open access in 2024).
    • Presented to PSI conference (linked to above bioequivalence topic) “Does it Make Sense to Apply the Estimand Framework to Clinical Pharmacology Trials”. June 2023
    • Reviewed and submitted comments for FDA draft guidance : Statistical Approaches to Establishing Bioequivalence (December 2022)
    • Knowledge sharing and discussion on a number of topics.
  8. HTA and RWE sub-team
    • Antonia Morga and Pepa Polavieja presented on behalf of the group on “The ICH E9(R1) Addendum in the context of Health Technology Assessments: methodological considerations and recommendations” at an Estimand session during the PSI conference in London (June 2023)
    • The group presented a poster at ISPOR Europe (November 2023) titled: “Estimands in Health Technology Assessments: methodological considerations and recommendations”