Endpoints Working Group

Foundational Evidence for Use in Drug Development and Evaluation

Goals

To accelerate the development of evidence and knowledge needed to optimize the selection, use and evaluation of endpoints in transthyretin (ATTR) and light chain (AL ) amyloidosis clinical trials from the drug developer and drug evaluator perspectives.

Rationale

  • Understanding endpoints are central to optimizing clinical trial design and patient care.
  • Endpoints are affected by design specifications including inclusion/exclusion criteria, type of amyloid, organ involvement, baseline stratification, and trial duration, for example.
  • Quantitative understanding of outcome rates (event rates, expected changes), levels of variability, prognostic factors, associations across outcomes, and clinical meaningfulness, are among other considerations required to effectively select endpoints and incorporate them into trials as well as to interpret findings.
  • As new endpoints are developed or drawn from other therapeutic areas, there will be deficiencies in the knowledge available for making best use of these endpoints in amyloidosis.
  • The Forum provides the best venue for anticipating and addressing these evidence needs, drawing on the perspectives of clinical experts, outcomes research experts, clinical regulators, and drug developers.
  • While there has been regulatory success using some endpoints mostly upon a relatively sparse playing field standardization will be helpful moving forward.

Chairs

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Angela Dispenzieri
Working Group Chair
Mayo Clinic
BIO+
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Michelle Carty
Working Group Co-Chair
Quality Metric
BIO+

Members

  • Amanda Peltier

    Vanderbilt University Medical Center

  • Ashish Verma

    Brigham and Women’s Hospital

  • Austin Hu

    FDA

  • Cara Rosenbaum

    AbbVie

  • Cecilie Herly

    Novo Nordisk

  • Charu Gandotra

    FDA

  • Donny Dietz

    BridgeBio

  • Eli Muchtar

    Mayo Clinic

  • Eugene Brailovski
  • Heather Landau

    Memorial Sloan Kettering Cancer Center

  • Julian Gillmore

    National Amyloidosis Centre, University College of London

  • Julie Rosenthal

    Mayo Clinic Arizona

  • Justin Grodin

    UT Southwestern Medical Center

  • Kristen Hsu

    Amyloidosis Research Consortium

  • Laura Obici

    University of Pavia

  • Louis Williams

    Cleveland Clinic

  • Michael Polydefkis

    Johns Hopkins

  • Michelle Kittleson

    Cedars-Sinai Medical Center

  • Nadine Abdallah

    Mayo Clinic

  • Namphuong Tran

    Janssen

  • Nelson Leung

    Mayo Clinic

  • Nowell Fine

    University of Calgary

  • Patrick Jay

    Alnylam

  • Ray Comenzo

    Tufts Medical Center

  • Sami Khella

    University of Pennsylvania

  • Sarah Cuddy

    Harvard/Brigham and Women’s Hospital

  • Spencer Carter

    University of Utah Health

  • Stathis Kastritis

    National and Kapodistrian University of Athens

  • Tzu-Yun McDowell

    FDA

  • Vaishali Sanchorawala

    Boston Medical Center

  • Yvonne Efebera

    Ohio Health

Subgroups

  • Charu Gandotra, FDA
  • Julian Gillmore, National Amyloidosis Centre, University College of London
  • Julie Rosenthal, Mayo Clinic Arizona
  • Justin Grodin, UT Southwestern Medical Center
  • Michelle Kittleson, Cedars-Sinai Medical Center
  • Nowell Fine, University of Calgary
  • Sarah Cuddy, Brigham and Women’s Hospital
  • Spencer Carter, University of Utah Health

  • Angela Dispenzieri, Mayo Clinic
  • Eli Muchtar, Mayo Clinic
  • Eugene Brailovski, McGill University
  • Heather Landau, Memorial Sloan Kettering Cancer Center
  • Nowell Fine, University of Calgary
  • Ray Comenzo, Tufts Medical Center
  • Vaishali Sanchorawala, Boston Medical Center
  • Yvonne Efebera, Ohio Health

  • Amanda Peltier, Vanderbilt University Medical Center
  • Michael Polydefkis, Johns Hopkins
  • Sami Khella, University of Pennsylvania

  • Anita D’Souza, Froedtert and Medical College of Wisconsin
  • Justin Grodin, UT Southwestern Medical Center
  • Laura Obici, University of Pavia
  • Michelle Carty, Quality Metric
  • Nadine Abdallah, Mayo Clinic
  • Vaishali Sanchorawala, Boston Medical Center

  • Ashish Verma, Brigham and Women’s Hospital
  • Eli Muchtar, Mayo Clinic
  • Julian Gillmore, National Amyloidosis Centre, University College of London
  • Louis Williams, Cleveland Clinic
  • Nelson Leung, Mayo Clinic
  • Ray Comenzo, Tufts Medical Center
  • Stathis Kastritis, National and Kapodistrian University of Athens