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

  • Austin Hu

    FDA

  • Charu Gandotra

    FDA

  • Cristina Quarta

    Alexion, AstraZeneca Rare Disease

  • Eli Muchtar

    Mayo Clinic

  • 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

  • Michael Polydefkis

    Johns Hopkins

  • Michelle Kittleson

    Cedars-Sinai Medical Center

  • Ray Comenzo

    Tufts Medical Center

  • Rebecca Hung

    Vanderbilt University Medical Center

  • Sami Khella

    University of Pennsylvania

  • Vaishali Sanchorawala

    Boston Medical Center

  • Yvonne Efebera

    Ohio Health

Subgroups

  • Charu Gandotra, FDA
  • Christie Nie, Prothena
  • John Vest, Alnylam
  • 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
  • Rebecca Hung, Vanderbilt University Medical Center
  • Sarah Cuddy, Brigham and Women’s Hospital
  • Spencer Carter, University of Utah Health

  • Angela Dispenzieri, Mayo Clinic
  • Eli Muchtar, Mayo Clinic
  • 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
  • Christie Nie, Prothena
  • Justin Grodin, UT Southwestern Medical Center
  • Laura Obici, University of Pavia
  • Michelle Carty, Quality Metric
  • Vaishali Sanchorawala, Boston Medical Center

  • Ashish Verma, Brigham and Women’s Hospital
  • Christie Nie, Prothena
  • Eli Muchtar, Mayo Clinic
  • Frank Bridoux, Université De Poitiers, France
  • Julian Gillmore, National Amyloidosis Centre, University College of London
  • Louis Williams, Cleveland Clinic
  • Nelson Leung, Mayo Clinic
  • Ray Comenzo, Tufts Medical Center