PSC Forum Veronica Miller, PhD Forum for Collaborative Research

Slides:

Advertisements

Similar presentations

6th European Patients’ Rights Day The EMA Geriatric Medicines Strategy and the empowered aging patient Francesca Cerreta EMA (European Medicines Agency)

Advertisements

PERSONALIZED MEDICINE. What is Personalized Medicine? Information-based healthcare –Person-by-person: high content, resolution & fidelity –Ushered in.

Susan Boynton, VP, Global Regulatory Affairs, Shire

Retreat Topics iPSC Opportunities in NIAMS Diseases Science Management Forum: Leveraging and Strategic Funding Collaborations Atopic Dermatitis Advancing.

Subject Selection and Recruitment David Wendler Department of Clinical Bioethics NIH, USA.

The ICH E5 Question and Answer Document Status and Content Robert T. O’Neill, Ph.D. Director, Office of Biostatistics, CDER, FDA Presented at the 4th Kitasato-Harvard.

A Collaborative Community-Engaged Approach to Evaluation for the Alliance for Research in Chicagoland Communities M. Mason, PhD, 1,2 B. Rucker, MPH 3,4.

NCI Review of the Clinical Trials Process 6 th Annual National Forum on Biomedical Imaging in Oncology James H. Doroshow M.D. April 7, 2005 Bethesda, Maryland.

The Interagency Food Safety Analytics Collaboration: Moving Forward Together Christopher Braden, MD Director, Division of Foodborne, Waterborne, and Environmental.

Frank Shipley, Chair, S-TEK Subcommittee Mary Mahaffy, Science Coordinator Karen Jenni, Insight Decisions - Facilitator S-TEK Implementation Plan.

Ajaz S. Hussain, Ph.D. Deputy Director Office of Pharmaceutical Science, CDER, FDA ACPS Subcommittee on Manufacturing Science: Identification and Prioritization.

Safe and Effective Medicine for Children Daring to be Rare, BIOTECanada Panel 2015 CADTH Symposium Saskatoon, April

The Innovative Medicines Initiative (IMI) High level the IMI Concept, Strategic Research Agenda and Call topics Eva Lindgren.

The Practical Art of Endpoint Selection: Industry Perspectives A View from the Pharma Industry of the FDA Guidance on PROs Glenn A. Phillips, Ph.D. Director.

The European Innovation Partnership for asthma: an opportunity for change Samantha Walker PhD, Asthma UK Project Coordinator, EARIP (European Asthma Research.

Regulatory Affairs and Adaptive Designs Greg Enas, PhD, RAC Director, Endocrinology/Metabolism US Regulatory Affairs Eli Lilly and Company.

PUTTING THE PATIENT AT THE CENTER OF HEALTHCARE RESEARCH Towards a more inclusive model.

Adaptive Licensing UK. Content What is adaptive licensing? Draft criteria for pilot candidate selection EMA Road Map 2015 and work programme 2012 UK perspective.

Current Plan for Critical Path Initiative Janet Woodcock, M.D. Acting Deputy Commissioner For Operations November 5, 2004.

Creating the Path for Innovative New Therapies Raymond L. Woosley, MD, PhD President, The Critical Path Institute.

Overcoming challenges in pediatric oncology product development: Regulatory oversight of multi-national clinical studies Ursula Kern, Advisory Committees.

The Third Annual Medical Device Regulatory, Reimbursement and Compliance Congress Value-Based Pricing: The Good, The Bad, and The Ugly What Does Value.

Gwendolyn Ryals, Look at Me Artwork from The Creative Center Janey Shin, Director, Real World Evidence Government Affairs and Market Access CADTH Symposium.

Patient Engagement throughout the Biopharmaceutical Lifecycle: Tips for Effective Patient Advocate/Industry Collaboration to Improve Patient Access and.

The opportunities and challenges of sharing genomics data with the pharmaceutical industry Shahid Hanif, Head of Health Data & Outcomes, ABPI DNA digest.

PRAGMATIC Study Designs: Elderly Cancer Trials

How Laws are Made. House, Senate, President 1 21 st Century Cures Legislation: Modernizing Drug Development in Pursuit of Cures Stephen Smith Chief Patient.

Is a Clinical Trial Right for Me?

CLI and Device Intervention Across the Pacific – An FDA View

Placebo / Standard of Care (PSoC)

Health Technology Assessment

Regulation of Medical Devices: Importance of a Globally Harmonized Approach Nicole Taylor Smith, JD September 2017.

Better Evidence. Better Decisions

Patient Focused Drug Development An FDA Perspective

Opioid Management in Primary Care Michael Parchman, MD, MPH

Seeking Treatments for PSC Out of the Desert and into the Woods

FP6 Priority 1, Life Sciences Genomics and Biotechnology for Health

Industry Perspective: Expanded Access Programs

Balancing Regulation and Innovation: An FDA Division of Cardiovascular Devices Perspective Bram Zuckerman, MD, FACC Director, FDA Division of Cardiovascular.

MacColl Center for Health Care Innovation

Better Lives. Together. The Parkinson’s Foundation makes life better for people with Parkinson’s disease by improving care and advancing research toward.

Introduction of New Technology: An FDA Division of Cardiovascular Devices Perspective Bram Zuckerman, MD, FACC Director, FDA Division of Cardiovascular.

Lessons Learned Through HBD: The Regulator’s View - US FDA

Gestora brasileiro focada exclusivamente na área da saúde.

CPT and Disclosure: Connecting Critical Processes

Forum for Collaborative Research:

Regulatory perspective

Non For Profit Model for Rare Disease Therapy Development

Changing the IBD Paradigm

Is a Clinical Trial Right for Me?

PhUSE Computational Science

Harmonization for Patient Centered Universal Health Coverage

Innovative Medicines Initiative:

Issues in Hypothesis Testing in the Context of Extrapolation

Therapeutic Drug Management Quality Project

Physician / Patient Future Collaboration Opportunities

CPT and Disclosure: Connecting Critical Processes

Introduction to TransCelerate

Adapted from a presentation at the Rwanda First National Workshop on

FDA Perspective Marco Cannella, PhD Senior Lead Reviewer

Experimental Medicine Challenge Grants Round 3

Trial Funding and Engagement: The NIH Sponsored CTSA Program

FP6 Priority 1, Life Sciences Genomics and Biotechnology for Health

Patient Involvement in the Development and Use of Safe Medicines Introductory Section – Concepts to Consider CIOMS XI WG Second Meeting October 23-24,

MIDD: Perspectives and Possibilities

An introduction to EMA’s support for medicines development

Building PHN Scientists

Members Meeting Leadership Consortium for a Value & Science-Driven Health System March 21, 2019 Vision  Research  Evidence  Effectiveness  Trials.

How Should We Select and Define Trial Estimands

Approach: The Cuban Regulatory Strategy

Presentation transcript:

PSC Forum Veronica Miller, PhD Forum for Collaborative Research UC Berkeley School of Public Health

The Concept One table Increase Efficiency Decrease Uncertainty Regulators Industry Academia Patients Increase Efficiency Clarity Innovation Collaboration Decrease Uncertainty Redundancy Development time Risk

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 Accelerate drug development not by lowering standards, but by increasing efficiency through collaboration

Characteristics Non-competitive, safe environment Independence Transparency Information democracy Synergy vs. duplication Single Sponsor-Single Agency All Sponsors-Multiple Agencies Cross-Atlantic perspectives reduce interagency discordance

Results Advance development of regulatory strategies Evolving science & 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

Forum for Collaborative Research HIV Forum Liver Forum Rare Diseases-IEM Forum HBV Forum AH-AALD Forum? PSC Forum CMV/Transpl. Forum HCV Forum forumresearch.org

PSC Steering Committee Co-Chairs Keith Lindor AASLD Rob Myers Gilead Regulatory Lara Dimick FDA/CDER/DGIEP Elmer Schabel EMA/BfArM Patient Advocates Ricky Safer PSC Partners Seeking a Cure Martine Walmsley PSC Support Society Marco Marzioni EASL Academia Christopher Bowlus UC Davis Medical Center Gideon Hirschfield University of Birmingham Kris Kowdley Swedish Medical Center Tamir Miloh Baylor College of Medicine Arun Sanyal Virginia Commonwealth University Industry Rob Myers Gilead Stephen Rossi NGM Biopharmaceuticals David Shapiro Intercept

PSC Forum Sponsors

PSC Forum Strategy Start with most urgent questions/issues Assign multi-stakeholder working groups Build trust Allow consensus to evolve Always science based Identify knowledge gaps and mechanisms to address these Build more trust Tackle more challenging issues

Regulatory Perspectives Regulatory decisions (approve or not approve a drug) is based on benefit and risk considerations What do we know about the benefit? How do we measure it? How long will the benefit last? What do we know about risk (potential harms) When could the harm appear? How serious is it?

What is the tolerance for risk?

PSC Forum Work Streams Clinical Trials Inclusion/Exclusion Criteria Endpoints (e.g. ALP fluctuations) Patient Centered Clinical Research PRO tools Data & Data Use

Clinical Trials: Inclusion-Exclusion Working Groups Underlying IBD Concomittant therapies URSO? Drug Targets Patient Populations

Gideon Hirschfield PSC Forum 2

Patient Centered Research Short-term Working Group Landscape Bring everyone up-to-date

Data & Data Use Landscape of registries and databases

Data Gaps? What data do we need to meet regulatory purposes? Identification of surrogate endpoints Validation of endpoints Strategy: Build synergies amongst existing efforts

Potential Working Groups Non-invasive diagnostics/Imaging Pediatrics Symptom management: ITCH?

Acknowledgement Forum Staff PSC Forum SC and Members Jessica Weber MPH, RN PSC Forum SC and Members