National Academies Press: OpenBook

Neuroscience Trials of the Future: Proceedings of a Workshop (2016)

Chapter: Appendix B: Workshop Agenda

« Previous: Appendix A: References
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

B

Workshop Agenda

NEUROSCIENCE TRIALS OF THE FUTURE: A WORKSHOP

March 3–4, 2016

Keck Center
500 Fifth Street, NW, Room 100
Washington, DC 20001

Background: Although major strides have been made over the past two decades in basic neurosciences, the pace of translation into more effective treatments has eluded the field. Among the many factors contributing to this reality are the standard clinical trial methods that have barely changed, perhaps with the exception of increased use of electronic data acquisition and analysis.

Clinical trials in neuropsychiatric disorders continue to suffer from high failure rates even with biological targets that are well validated. Even in the hands of experienced investigators, the now commonplace problem of poor assay sensitivity, and attendant trial failure, have adversely affected pharmaceutical and device development. Signal detection in central nervous system trials is regularly beset by high placebo or nonspecific response, intrasubject variability of endpoints, intersubject and intersite variability in multicenter trials, poor treatment adherence, and weak patient engagement and retention. The net effect of these challenges has been to simply increase the trial sample size in an attempt to control type II error. Yet, promising early clinical data often are not

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

replicated in larger registration trials, and Phase III failure rates in neuroscience randomized controlled trials remain among the highest in medicine. The apparent unsustainability of the current clinical development scenario has driven many large pharmaceutical companies entirely out of investment in neurosciences.

Quite apart from the business perspective, the fact that many early-stage clinical trials misleadingly provide a signal (a type I error) raises the question of whether volunteering for these trials is in the best interest of trial subjects, in particular, and for the patients with that particular disorder in general.

Better methods, from clinical study design through execution and evaluation, could help restore the integrity, feasibility, acceptability, efficiency, and economic viability of clinical neuropsychiatric development. However, in order to use innovative approaches to address these challenges, buy-in and acceptance from the regulatory community will be important. For example, adaptive trials could offer a more efficient means of addressing experimental questions involving multiple uncertainties, although they are often infrequently used. In addition, understanding the utility of wearable and patient monitoring devices (and the data generated) in neuroscience clinical trials is needed. Given the current challenges in neuroscience clinical trials, this public workshop will bring together key stakeholders to discuss opportunities to improve the integrity, efficiency, and validity of clinical trials for nervous system disorders (focusing specifically on Phase II and Phase III trials).

Meeting Objectives:

  • Examine assay sensitivity challenges in clinical trials for nervous system disorders, including causes of type I error in early trials and poor signal detection and type II error in later stage trials.
  • Explore opportunities to improve clinical trial methodology for nervous system disorders, including strategies for:
    • Guiding the selection of patient populations, such as using endophenotyping to increase the yield of responders and using genomics, proteomics, and imaging biomarkers to “stage” nervous system disorders.
    • Increasing patient engagement through all phases of the clinical trial (i.e., recruitment, screening, and posttrial) and improving adherence and retention.
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
  • Using patient-centric technologies (e.g., wearables) and integrating such real-world, real-time data with traditional clinical data.
  • Improving monitoring during clinical trials.
  • Leveraging recent advances in diagnostics, biomarkers, and endpoints to develop more efficient clinical trials.
  • Using novel trial designs (e.g., adaptive, enrichment, and platform design studies) for nervous system disorders, including associated regulatory challenges and opportunities.

March 3, 2016

8:30 a.m. Opening Remarks

ATUL PANDE, workshop co-chair

Chief Medical Officer and Executive Vice President

Tal Medical

RICHARD KEEFE, workshop co-chair

Professor of Psychiatry and Behavioral Sciences

Duke University School of Medicine

Neuroscience Clinical Trials: Challenges and Opportunities

8:45 a.m.

STEVEN ROMANO

Senior Vice President and Chief Science Officer

Mallinckrodt Pharmaceuticals

9:05 a.m.

SHITIJ KAPUR

Executive Dean and Head of School

Institute of Psychiatry, Psychology & Neuroscience

King’s College London

9:30 a.m.

Discussion Among Speakers and Workshop Participants

9:45 a.m.

BREAK

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

SESSION I: CLINICAL TRIAL DESIGN

Session Objectives: Discuss current challenges to clinical trial design for nervous system disorders. Explore elements of clinical trial protocols that might be improved and lead to more efficient trials. Discuss how novel trial designs (e.g., adaptive, enrichment, and platform design studies) might be used for nervous system disorders.

10:00 a.m.

Session Overview and Objectives

STEPHEN BRANNAN, session moderator

Vice President of Clinical Research and Medical Affairs

Forum Pharmaceuticals

Biomarkers

10:10 a.m.

ANIL MALHOTRA

Director, Psychiatry Research, Zucker Hillside Hospital

Professor, Molecular Medicine and Psychiatry

Hofstra Northwell School of Medicine at Hofstra University

10:25 a.m.

ALICE CHEN-PLOTKIN

Assistant Professor of Neurology

Perelman School of Medicine, University of Pennsylvania

Diagnosis and Patient Identification

  • Discuss alternatives to the Diagnostic and Statistical Manual of Psychiatric Disorders (DSM).

10:40 a.m.

SARAH MORRIS

Acting Head, NIMH RDoC Unit

Program Officer, Schizophrenia Spectrum Disorders Research Program

National Institute of Mental Health

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

10:55 a.m.

ROBERT BILDER

Professor-in-Residence, Department of Psychiatry and Biobehavioral Sciences,

University of California, Los Angeles

Editor-in-Chief, Diagnostics in Neuropsychiatry

Statistical Approaches and Considerations

11:10 a.m.

MICHAEL PENCINA

Director of Biostatistics, Duke Clinical Research Institute

Professor of Biostatistics and Bioinformatics

Duke University

Novel Clinical Trial Designs

11:25 a.m.

ERIK SNOWBERG

Professor of Economics and Political Science

California Institute of Technology

SYLVAIN CHASSANG

Professor of Economics and Public Affairs

Princeton University

11:45 a.m.

JAIDEEP KAPUR

Study Chair, Established Status Epilepticus Treatment Trial (ESETT)

Eugene Meyer III Professor of Neuroscience, Neurology

University of Virginia School of Medicine

12:00 p.m.

Panel Remarks

Additional Panelists:

TIFFANY FARCHIONE

Deputy Director, Division of Psychiatry Products

Center for Drug Evaluation and Research

Food and Drug Administration

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

CARLOS PEÑA

Director, Division of Neurological and Physical Medicine Devices

Office of Device Evaluation

Center for Devices and Radiological Health

Food and Drug Administration

12:30 p.m.

Discussion Among Speakers and Workshop Participants

1:00 p.m.

LUNCH

SESSION II: CLINICAL TRIAL METHODOLOGY

Session Objectives: Explore opportunities to improve clinical trial methodology for nervous system disorders, including strategies for patient selection, engagement, and retention. Examine the extent to which current diagnostic methods contribute to the inherent variability in study populations. Discuss the utility of patient-centric technologies (e.g., wearables) and how such real-world, real-time data might be integrated with traditional clinical data to improve the integrity and efficiency of trials. Consider lessons learned and best practices from other therapeutic areas that might be applied to neuroscience clinical trials.

1:45 p.m.

Session Overview and Objectives

AMIR KALALI, session moderator

Head, Neuroscience Center of Excellence

Quintiles

2:00 p.m.

Transforming Clinical Trials with Technology (guided panel discussion)

  • Discuss opportunities to improve patient engagement and retention.
  • Discuss how to improve patient adherence to assigned treatment.
  • Discuss opportunities to improve patient assessments.
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
  • Consider the potential applications of emerging technologies (e.g., wearables) for clinical trials.
    • What are common clinical applications of these technologies?
    • What are the known benefits and risks associated with use? What are the scientific controversies behind this evidence?

CARLOS RODARTE

Chief Executive Officer

Health Rhythms

JOHN REITES

Head, Digital Health Acceleration

Quintiles

DREW SCHILLER

Chief Technology Officer and Co-Founder

Validic

GLEN DE VRIES

President and Co-Founder

Medidata Solutions, Inc.

KARL KIEBURTZ

Robert J. Joynt Professor in Neurology

Senior Associate Dean for Clinical Research

Director of the Clinical & Translational Science Institute

University of Rochester Medical Center

3:15 p.m.

BREAK

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Lessons Learned from Other Therapeutic Areas

3:30 p.m.

Oncology

PERRY NISEN

Chief Executive Officer

Sanford Burnham Prebys Medical Discovery Institute

3:45 p.m.

Cardiology

ADRIAN FELIPE HERNANDEZ

Professor of Medicine

Duke Clinical Research Institute

Duke University School of Medicine

4:00 p.m.

Discussion Among Speakers and Workshop Participants

4:45 p.m.

Day One Wrap-Up

Workshop Co-Chairs

5:00 p.m.

ADJOURN DAY ONE

March 4, 2016

8:30 a.m.

Day Two Opening

ATUL PANDE, workshop co-chair

Chief Medical Officer and Executive Vice President

Tal Medical

RICHARD KEEFE, workshop co-chair

Professor of Psychiatry and Behavioral Sciences

Duke University School of Medicine

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

KEYNOTE SESSION III: INTERNATIONAL REGULATORY LANDSCAPE

Session Objectives: Consider the regulatory landscape for neuroscience clinical trials. Explore differences in regulatory pathways among countries and consider the impact.

8:50 a.m.

Session Overview and Objectives

THOMAS LAUGHREN, session moderator

Director

Laughren Psychopharm Consulting, LLC

Key Regulatory Opportunities for Neuroscience Clinical Trials

9:00 a.m.

Regulatory Opportunities and Challenges in the United States

ROBERT CALIFF

Commissioner of Food and Drugs

Food and Drug Administration

9:20 a.m.

Regulatory Opportunities and Challenges in Europe

LUCA PANI

Director General

Italian Medicines Agency (AIFA)

9:40 a.m.

Discussion Among Speakers and Workshop Participants

10:45 a.m.

BREAK

SESSION IV: ETHICAL CONSIDERATIONS

Session Objectives: Examine ethical, legal, and social questions about neuroscience clinical trials. Consider potential data protection and human subjects’ issues that might arise as clinical trials continue to transform.

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

11:00 a.m.

Session Overview and Objectives

PETRA KAUFMANN

Director, Office of Rare Diseases Research and Division of Clinical Innovation

National Center for Advancing Translational Sciences

Data Protection

11:10 a.m.

FRANK ROCKHOLD

Senior Vice President, Global Clinical Safety and Pharmacovigilance

GlaxoSmithKline

Professor of Biostatistics and Bioinformatics (starting March 2016)

Duke University School of Medicine

Human Subjects Protection

11:25 a.m.

GREG KOSKI

President and Co-Founder

Alliance for Clinical Research Excellence and Safety (ACRES)

11:40 a.m.

EMIL CHIAUZZI

Research Director

PatientsLikeMe

12:00 p.m.

Discussion Among Speakers and Workshop Participants

12:30 p.m.

LUNCH

SESSION V: REIMBURSEMENT

Session Objectives: Consider how data collected by payers are used to inform reimbursement decisions and influence the long-term translation of products in the marketplace. Consider economic outcome measures used to determine payer practices. How will and should these measures be worked into future clinical trials?

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

1:15 p.m.

Session Overview and Objectives

DANIEL BURCH, session co-moderator

Vice President and Global Therapeutic Area Head for Neuroscience

Pharmaceutical Product Development (PPD)

MICHAEL POLLOCK, session co-moderator

Vice President, Real World Outcomes

Pharmaceutical Product Development (PPD)

Improving the Evidence Base for Reimbursement

What evidence is needed from research to align with insurance policies and evidence criteria?

1:25 p.m.

RHONDA ROBINSON BEALE via teleconference

Senior Vice President and Medical Officer

Blue Cross of Idaho

Challenges of Generating the Required Evidence: An Industry Perspective

1:40 p.m.

PAUL STANG

Vice President, Global R&D Epidemiology

Janssen Research and Development

Pragmatic Trials: Challenges and Opportunities for Neuroscience Trials

1:55 p.m.

MARK CZIRAKY

Co-Founder and Vice President of Research

HealthCore, Inc.

2:10 p.m.

Discussion Among Speakers and Workshop Participants

2:45 p.m.

BREAK

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×

SESSION VI: MOVING FORWARD

Session Objectives: A panel will synthesize and discuss key highlights from the workshop presentations and discussions, including identifying next steps and promising areas for future action and research.

3:00 p.m.

Panel Discussion: Session Moderators

ATUL PANDE, workshop co-chair

RICHARD KEEFE, workshop co-chair

STEPHEN BRANNAN, session I moderator

AMIR KALALI, session II moderator

THOMAS LAUGHREN, session III moderator

PETRA KAUFMANN, session IV moderator

DANIEL BURCH AND MICHAEL POLLOCK, session V moderators

3:45 p.m.

Discussion Among Session Moderators and Workshop Participants

4:15 p.m.

Closing Remarks from the Workshop Co-Chairs

4:30 p.m.

ADJOURN WORKSHOP

Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 79
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 80
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 81
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 82
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 83
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 84
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 85
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 86
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 87
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 88
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 89
Suggested Citation:"Appendix B: Workshop Agenda." National Academies of Sciences, Engineering, and Medicine. 2016. Neuroscience Trials of the Future: Proceedings of a Workshop. Washington, DC: The National Academies Press. doi: 10.17226/23502.
×
Page 90
Next: Appendix C: Registered Attendees »
Neuroscience Trials of the Future: Proceedings of a Workshop Get This Book
×
Buy Paperback | $46.00 Buy Ebook | $36.99
MyNAP members save 10% online.
Login or Register to save!
Download Free PDF

On March 3-4, 2016, the National Academies of Sciences, Engineering, and Medicine’s Forum on Neuroscience and Nervous System Disorders held a workshop in Washington, DC, bringing together key stakeholders to discuss opportunities for improving the integrity, efficiency, and validity of clinical trials for nervous system disorders. Participants in the workshop represented a range of diverse perspectives, including individuals not normally associated with traditional clinical trials. The purpose of this workshop was to generate discussion about not only what is feasible now, but what may be possible with the implementation of cutting-edge technologies in the future.

  1. ×

    Welcome to OpenBook!

    You're looking at OpenBook, NAP.edu's online reading room since 1999. Based on feedback from you, our users, we've made some improvements that make it easier than ever to read thousands of publications on our website.

    Do you want to take a quick tour of the OpenBook's features?

    No Thanks Take a Tour »
  2. ×

    Show this book's table of contents, where you can jump to any chapter by name.

    « Back Next »
  3. ×

    ...or use these buttons to go back to the previous chapter or skip to the next one.

    « Back Next »
  4. ×

    Jump up to the previous page or down to the next one. Also, you can type in a page number and press Enter to go directly to that page in the book.

    « Back Next »
  5. ×

    Switch between the Original Pages, where you can read the report as it appeared in print, and Text Pages for the web version, where you can highlight and search the text.

    « Back Next »
  6. ×

    To search the entire text of this book, type in your search term here and press Enter.

    « Back Next »
  7. ×

    Share a link to this book page on your preferred social network or via email.

    « Back Next »
  8. ×

    View our suggested citation for this chapter.

    « Back Next »
  9. ×

    Ready to take your reading offline? Click here to buy this book in print or download it as a free PDF, if available.

    « Back Next »
Stay Connected!