The Rare Diseases Forum Innovation in Trial Design Workshop · 2019-09-23  · 5. The Rare Diseases...

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The Rare Diseases Forum Innovation in Trial Design Workshop Veronica Miller, PhD Forum for Collaborative Research UC Berkeley School of Public Health September 23, 2019 Washington, DC

Transcript of The Rare Diseases Forum Innovation in Trial Design Workshop · 2019-09-23  · 5. The Rare Diseases...

  • The Rare Diseases Forum Innovation in Trial Design Workshop

    Veronica Miller, PhDForum for Collaborative Research

    UC Berkeley School of Public Health

    September 23, 2019

    Washington, DC

  • Thanks & Acknowledgments

  • The Forum for Collaborative ResearchCatalyzing Clinical Research to Improve Global Health

  • 4 The Rare Diseases Forum September 23 2019 WORKSHOP

    Guiding Principle

    Once new drug candidates and therapeutic strategies are identified, their efficient and safe development is in the best interest of all stakeholders, most of all, the patients.

    “The Forum accelerates drug development by increasing efficiency through collaboration, not by lowering standards.”Veronica Miller PhD, Executive Director, The Forum for Collaborative Research

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    The Concept Enhanced clarity

    Innovation

    Collaboration

    Redundancy

    Development time

    Risk

    ONE TABLE

    Regulators

    Industry

    Academia

    Patients

    THE FORUM

    “Starting with HIV, the Forum consistently and effectively applies the multi-stakeholder model to advance the development path for new treatments in multiple disease areas. ” Eric A. Hughes, MD, PhD, Development Unit Head, Novartis Pharma AG

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    Disease Areas

    In order of appearance The HIV Forum 1997- present The HCV Forum 2006-2016 (completed) The Liver Forum 2014 - present The CMV/transplantation Forum 2014- present The HBV Forum 2016 - present The PSC Forum 2017 - present

    The Rare Diseases Forum 2018 →

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    Results Advance development of regulatory strategies Evolving science and evolving consensus

    Generate evidence through collaboration Efficient use of data

    Provide mechanism for patient-centered drug development Provide mechanism for innovation in data use and analytics

    “The Forum addresses cutting edge regulatory science and policy issues with proven results.”George Hanna MD, VP Infectious Diseases Global Clinical Development, Merck & Co., Inc.

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    Rare Diseases Forum Steering Committee Co-Chairs

    Academic: Marshall L. Summar MD NCMC/NORD

    Industry: John F. Crowley Amicus Therapeutics

    Forum: Veronica Miller PHD UC Berkeley

    Members Regulatory –US FDA

    Dragos Roman MD CDER/DGIEP

    Dina Zand MD CDER/DGIEP

    Rachel Witten MD CBER/OTAT

    Christine Mueller MD OOPD

    Regulatory – EMA

    Violeta Stoyanova MD COMP

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    Sponsors

         

  • 10 The Rare Diseases Forum September 23 2019 WORKSHOP

    Sponsors

  • 11 The Rare Diseases Forum September 23 2019 WORKSHOP

    Rare Diseases Forum Workshop 1Innovation in Trial Designs “ Bringing Lessons from the Oncology Experience to Rare Diseases” Planning Committee Members

    Barry Byrne, MD, University of Florida

    Chaohong Fan, MD, FDA/CBER

    Dina Zand, MD, FDA/CDER

    Ke Liu, MD, FDA/CBER

    Keith Gregg, PhD, BioMarin

    Laura Lee Johnson, PhD, FDA/CDER

    Marie-Laure Nevoret, MD, Regenxbio

    Nick Kenny, PhD, Syneos Health

    Olivier Danos, PhD, Regenxbio

    P.J Brooks, PhD, NIH/NCATS

    Rachel Witten, MD, FDA/CBER

    Sandi Lehrman, MD, Patient Advocate

    Stephen Pakola, MD, Regenxbio

    Tim Miller, PhD, Abeona Therapeutics

    Vivian Fernandez, Regenxbio

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    Forum RD Team Luis Javier Hernandez Rare Disease Forum Project

    Manager

    Leighla Dergham U Maryland Intern

    Terry Daniels Office Administrator

    Brenda Rodriguez Development Director

    Prism Event Management Paula Blay

    Mairead O’Reilley

  • The Rare Diseases ForumGoals & Objectives

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    Facilitate Development of New Therapies for Rare Diseases

    Innovation in trial design, including seamless/adaptive designs and master protocols

    Best practices and uses of natural history, registries and other sources of evidence

    Use of the totality of evidence and treatment assessment outcomes Biomarkers and disease intermediates Innovation in biostatistics

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    Rare Diseases Forum – Workstreams –Working Groups

    I. Legal and scientific basis for establishing evidence –treatment assessment outcomes

    1. Legal underpinnings for use of evidence to establish patient benefit2. Assessing clinically meaningful treatment outcomes

    b. Use of digital technology in quantification of treatment outcomes

    3. Encyclopedia of innovation

    II. Innovation in clinical trial design

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    Goals for Today: Innovation in Trial Design

    Draw key lessons from oncology experience Consider application to rare diseases Establish concrete next steps for RD Forum

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    Perspectives

    Many different perspectives “Master protocols are the way to go” “Single arm studies w natural history comparators are the way to go” “Bayesian approaches in RD is not possible bc we do not have enough priors” “Basket approaches will not work if endpoints are not validated”

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    CollaborationCooperationCommunicationCommunity

    One DrugOne TrialOne Disease

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    Rules of the Game

    Open, constructive, dialogue and deliberation Bring your expertise Leave your hat at the door

    What’s said in the room, stays in the room Reports and publications not for attribution

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    Participants speak as individuals and express views that may not represent those of their organizations

    Social Media: Quotes of speakers/panelists/audience

    Detailing meeting proceedings

    Photos during receptions

    Referencing attendance

    Interactions during breaks

  • Lessons from OncologyPanel 1: Platform Trials

  • Lessons from OncologyPanel 2: Basket Trials

    Slide Number 1Slide Number 2Slide Number 3Guiding PrincipleThe ConceptDisease AreasResultsRare Diseases Forum �Steering Committee SponsorsSponsorsRare Diseases Forum Workshop 1�Innovation in Trial Designs “ Bringing Lessons from the Oncology Experience to Rare Diseases” �Planning Committee MembersForum RD TeamSlide Number 13Facilitate Development of New Therapies for Rare DiseasesRare Diseases Forum – Workstreams –Working GroupsGoals for Today: Innovation in Trial DesignPerspectivesSlide Number 18Rules of the GameSlide Number 20Slide Number 21Slide Number 22


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