B
Workshop Agenda
Multimodal Therapies for Brain Disorders: A Workshop
June 14-15, 2016
Keck Center
500 Fifth Street, NW | Room 100
Washington, DC 20001
Background: Multimodal therapy approaches (i.e., combinations of interventions aimed at different aspects of a disease) are emerging as potential ways to enhance clinical outcomes for patients with psychiatric and neurological disorders. This could include, for example, concomitant prescription of a drug along with a device, biologic, or behavioral/psychosocial intervention (including, e.g., cognitive remediation, lifestyle adjustment, dietary intervention, or animate therapy). Another example would be the simultaneous use of a neuromodulation device during performance of a specific neurocognitive task. While such a multimodal approach is consistent with the common clinical practice of combining interventions, there are important questions regarding how these interventions interact (in terms of additive, subtractive, or synergistic therapeutic as well as adverse effects), how they should be used, in what subsets of patients, and in what amounts or for what period of time. Further discussion is needed about methodologies for determining efficacy and safety in multimodal therapies compared with monotherapies and for comparing across multiple types of multimodal therapy. Multimodal therapy approaches also raise a number of important regulatory issues, both regarding combinations of regulated products (e.g., drug plus de-
vice) and approaches in which only one product is regulated (e.g., drug/device plus psychosocial intervention). Related questions arise for reimbursement, for example, would payers only pay for care if it includes both drugs and psychotherapy if that is what the data show is effective?
This workshop, hosted by the Forum on Neuroscience and Nervous System Disorders, will bring together key stakeholders to examine scientific, clinical, regulatory, and reimbursement issues related to multimodal approaches and identify potential opportunities to enhance clinical outcomes for individuals with psychiatric and neurological disorders. Rather than delving deeply into specific interventions, the workshop will aim to examine general principles, barriers, and potential solutions and opportunities that may apply across multimodal therapy development for brain disorders.
Meeting Objectives:
- Explore recent advances in the development of multimodal therapeutic approaches for psychiatric and neurological disorders and approaches to using these therapies (e.g., earlier versus later in disease progression), and discuss future research needs to further advance understanding of these approaches.
- Highlight disease areas in which a multimodal approach could be particularly useful (e.g., areas in which the pathophysiology is well understood, or areas in which mono-modal approaches have been insufficiently effective).
- Discuss methodologies for establishing efficacy and safety for multimodal therapies compared to monotherapies, including clinical trials and statistical considerations.
- Consider regulatory issues for multimodal therapies, including for approaches in which only one component is regulated (e.g., drug plus psychosocial intervention), and discuss potential opportunities for addressing challenges.
- Consider reimbursement issues for multimodal therapies for nervous system disorders, and discuss potential opportunities for addressing challenges.
- Incorporate lessons learned from other therapeutic areas in which multimodal approaches are more frequently used (e.g., cardiology, diabetes, cancer).
DAY ONE: June 14, 2016
1:30 p.m. |
Opening Remarks and Discussion of Definitions
KARL KIEBURTZ, Workshop Co-Chair Robert J. Joynt Professor in Neurology Senior Associate Dean for Clinical Research Director of the Clinical & Translational Science Institute University of Rochester Medical Center |
SARAH H. LISANBY, Workshop Co-Chair Director, Division of Translational Research National Institute of Mental Health National Institutes of Health |
SESSION I: STATE-OF-THE-SCIENCE IN MULTIMODAL THERAPIES FOR BRAIN DISORDERS
Session Objectives:
- Explore examples of recent advances in the development of multimodal therapeutic approaches for brain disorders and approaches to using these therapies.
- Discuss future research needs to further advance understanding of these approaches.
- Highlight disease areas in which a multimodal approach could be particularly useful.
1:50 p.m. |
Overview and Session Objectives
TIMOTHY STRAUMAN, Session Co-Moderator Professor of Psychology and Neuroscience Duke University |
KEITH HILDEBRAND, Session Co-Moderator Senior Principal Scientist, Technical Fellow Neuromodulation Medtronic, Inc. |
2:00 p.m. |
Part A: Two Pharmacological Interventions Approved as a Co-Delivery Drug/Drug and Drug/Biologic Combinations for Alzheimer’s Disease JAMES HENDRIX Director, Global Science Initiatives, Medical and Scientific Relations Alzheimer’s Association |
2:10 p.m. |
Part B: Concomitant Prescription of Two Interventions with Different Modalities Drug−Device Combinations for Epilepsy MARTHA MORRELL Chief Medical Officer, NeuroPace Clinical Professor of Neurology and, by courtesy, Neurosurgery Stanford University Combining Drugs and Psychosocial Interventions in Adolescents with Bipolar Disorder KIKI CHANG (via WebEx) Professor of Psychiatry and Behavioral Sciences Stanford University Medical Center |
2:30 p.m. |
Part C: Simultaneous Use of Two Modalities in a Single Procedure Combining Devices with Cognitive Enhancement BRUCE LUBER Staff Scientist Experimental Therapeutics & Pathophysiology Branch National Institute of Mental Health Individually Targeted Combination of Transcranial Magnetic Stimulation and a Psychosocial Intervention TIMOTHY STRAUMAN Professor of Psychology and Neuroscience Duke University |
2:50 p.m. | Discussion Among Speakers and Workshop Participants |
3:30 p.m. | BREAK |
SESSION II: REGULATORY AND REIMBURSEMENT CONSIDERATIONS
Session Objectives:
- Consider regulatory and reimbursement issues for multimodal therapies, including for approaches in which two components are regulated (e.g., drug plus device) and approaches in which only one component is regulated (e.g., drug plus psychosocial intervention).
- Explore evidentiary standards needed for regulation and reimbursement, and consider how different approval pathways and evidentiary standards across FDA centers impact multimodal approaches.
- Discuss potential opportunities for addressing challenges.
3:45 p.m. |
Session Overview and Objectives
SARAH H. LISANBY, Session Moderator Director, Division of Translational Research National Institute of Mental Health |
3:55 p.m. |
Part A: Regulatory Considerations
Combination Products at the Food and Drug Administration PATRICIA LOVE Deputy Director, Office of Combination Products Food and Drug Administration Evaluating Multimodal Products at the Center for Drug Evaluation and Research BILLY DUNN Director, Division of Neurology Products Office of Drug Evaluation I Center for Drug Evaluation and Research Food and Drug Administration Evaluating Multimodal Products at the Center for Devices and Radiological Health CARLOS PEÑA Director, Division of Neurological and Physical Medicine Devices Office of Device Evaluation Center for Devices and Radiological Health Food and Drug Administration |
Evaluating Multimodal Products at the Center for Biologics Evaluation and Research WILSON BRYAN Director, Division of Clinical Evaluation & Pharmacology/Toxicology Office of Cellular, Tissue, and Gene Therapies Center for Biologics Evaluation and Research Food and Drug Administration |
|
4:35 p.m. |
Part B: Payer Considerations
Private Payer Considerations RHONDA ROBINSON-BEALE Senior Vice President and Chief Medical Officer Blue Cross of Idaho Perspectives from a Government Payer STEVEN PIZER Associate Professor of Health Economics Department of Pharmacy and Health Systems Sciences Northeastern University and Chief Economist, Health Care Financing & Economics U.S. Department of Veterans Affairs |
4:55 p.m. | Discussion Among Speakers and Workshop Participants |
5:30 p.m. | Adjourn Day One |
DAY TWO: June 15, 2016
8:30 a.m. |
Day Two Opening Remarks
KARL KIEBURTZ, Workshop Co-Chair SARAH H. LISANBY, Workshop Co-Chair |
KEYNOTE TALK: FUTURE DIRECTIONS IN CLINICAL TRIALS AND REGULATORY APPROACHES FOR MULTIMODAL THERAPIES
8:40 a.m. |
ROBERT CALIFF Commissioner of Food and Drugs Food and Drug Administration |
9:00 a.m. | Questions and Answers with Workshop Participants |
9:30 a.m. | BREAK |
SESSION III: ESTABLISHING EFFICACY AND SAFETY IN MULTIMODAL THERAPIES FOR BRAIN DISORDERS
Session Objectives: Discuss methodologies for establishing efficacy and safety for multimodal therapies compared to monotherapies, including clinical and statistical considerations. Potential topics may include
|
|
|
9:45 a.m. |
Session Overview and Objectives
KARL KIEBURTZ, Session Moderator Robert J. Joynt Professor in Neurology Senior Associate Dean for Clinical Research Director of the Clinical & Translational Science Institute University of Rochester Medical Center |
9:55 a.m. |
Platform Trials for Multimodal Therapies
ROGER LEWIS Senior Medical Scientist, Berry Consultants Professor and Chair, Department of Emergency Medicine, Harbor-UCLA Medical Center Quantification of Dose with Devices MAROM BIKSON Professor of Biomedical Engineering The City College of the City University of New York Quantification of Dose in Psychosocial Interventions WOLFGANG LUTZ (via WebEx) Professor and Head of Clinical Psychology and Psychotherapy University of Trier, Germany |
10:45 a.m. |
Discussants:
BILLY DUNN Director, Division of Neurology Products Office of Drug Evaluation I Center for Drug Evaluation and Research Food and Drug Administration RHONDA ROBINSON-BEALE Senior Vice President and Chief Medical Officer Blue Cross of Idaho |
STEVEN PIZER Associate Professor of Health Economics Department of Pharmacy and Health Systems Sciences Northeastern University and Chief Economist, Health Care Financing & Economics U.S. Department of Veterans Affairs |
|
11:15 a.m. | Discussion Among Speakers, Discussants, and Workshop Participants |
11:45 a.m. | LUNCH |
SESSION IV: INDUSTRY PERSPECTIVES
Session Objectives:
- Discuss industry perspectives on particular technical, scientific, and commercial challenges to bringing these systems to market, and examine lessons learned from successful examples of collaborations on multimodal approaches.
- Explore potential mechanisms for addressing challenges and enabling multimodal therapy development programs.
12:30 p.m. |
Session Overview and Objectives
STEVIN ZORN, Session Moderator Executive Scientist in Residence Lundbeck |
12:40 p.m. |
Challenges and Opportunities for Integration of Therapeutic Devices into Psychiatr JEFFREY NYE Vice President, Neuroscience Innovation and Scientific Partnership Strategy Janssen Research and Development, LLC Johnson & Johnson Innovation |
Intrathecal Infusion Therapy for Chronic Pain: Challenges, Lessons, and Opportunities KEITH HILDEBRAND Senior Principal Scientist, Technical Fellow Neuromodulation Medtronic, Inc. Pfizer–MedGenesis Therapeutix Collaboration on Glial Cell Line-Derived Neurotrophic Factor (GDNF) Protein and Convection Enhanced Delivery (CED) Technology for Parkinson’s Disease ERICH MOHR Chair & Chief Executive Officer MedGenesis Therapeutix Inc. CHRISTOPHER SHAFFER Associate Research Fellow Pfizer Inc. |
|
1:20 p.m. | Discussion Among Speakers and Workshop Participants |
2:00 p.m. | BREAK |
SESSION V: MOVING FORWARD
Session Objectives:
- Discuss the roles of NIH, other research agencies, and disease-specific organizations in supporting the development of multimodal therapies.
- Synthesize and discuss key highlights from the workshop presentations and discussions and, most importantly, identify next steps and promising areas for future action and research.
2:15 p.m. |
Session Overview and Objectives
KARL KIEBURTZ, Workshop Co-Chair and Session Moderator SARAH H. LISANBY, Workshop Co-Chair and Session Moderator |
2:20 p.m. |
Role of Research Agencies in De-Risking Multimodal Therapy Development AMIR TAMIZ Program Director, NIH Blueprint NeuroTherapeutics Network National Institutes of Health STUART HOFFMAN Senior Scientific Advisor for Brain Injury Office of Research and Development Department of Veterans Affairs |
2:40 p.m. |
Role of Disease-Specific Research Funding in Multimodal Therapy Development JAMES HENDRIX Director, Global Science Initiatives, Medical and Scientific Relations Alzheimer’s Association BRIAN FISKE Senior Vice President, Research Programs The Michael J. Fox Foundation for Parkinson’s Research |
3:00 p.m. | Discussion with Workshop Participants |
3:15 p.m. |
Panel Discussion: Identifying Gaps, Opportunities, and Next Steps Highlighted in Workshop Presentations and Discussions KARL KIEBURTZ, Workshop Co-Chair and Session III Moderator SARAH H. LISANBY, Workshop Co-Chair and Session II Moderator KEITH HILDEBRAND, Session I Co-Moderator TIMOTHY STRAUMAN, Session I Co-Moderator STEVIN ZORN, Session IV Moderator EMMELINE EDWARDS Director, Division of Extramural Research National Center for Complementary and Integrative Health |
4:00 p.m. | Discussion with Workshop Participants |
4:20 p.m. |
Closing Remarks
KARL KIEBURTZ, Workshop Co-Chair SARAH H. LISANBY, Workshop Co-Chair |
4:30 p.m. | Adjourn Workshop |