GUIDANCE 1 December 18, 2017 - U S Food and Drug ... · PDF fileguidance 1 december 18, 2017 page 1 1 2 u.s. food and drug administration 3. center for drug evaluation and research

GUIDANCE 1 December 18, 2017
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2 U.S. FOOD AND DRUG ADMINISTRATION
3 CENTER FOR DRUG EVALUATION AND RESEARCH
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6 PUBLIC WORKSHOP ON
7 PATIENT-FOCUSED DRUG DEVELOPMENT:
8 GUIDANCE 1
9 COLLECTING COMPREHENSIVE AND REPRESENTATIVE INPUT
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12 FDA White Oak Campus
13 10903 New Hampshire Avenue
14 Building 31, Room 1503 (Great Room)
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20 Reported by:
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Silver Spring, MD 20993
Monday, December 18, 2017
Irene Gray,
Capital Reporting Company
www.CapitalReportingCompany.com 202-857-3376
http:www.CapitalReportingCompany.com

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1 A P P E A R A N C E S
2 Conny Berlin,
3 Global Head Quantitative Safety and Epidemiology,
4 Novartis
5 Kunthel By, PhD
6 Division of Biometrics V, Office of Biostatistics,
7 Office of Translational Science, Center for Drug
8 Evaluation and Research, U.S. Food and Drug
9 Administration
10 Michelle Campbell, PhD
11 Clinical Outcome Assessments Staff, Office of New
12 Drugs, Center for Drug Evaluation and Research,
13 U.S. Food and Drug Administration
14 Meghana Chalasani,
15 Decision Support and Analysis Team, Office of
16 Program and Strategic Analysis, Office of
17 Strategic Programs, Center for Drug Evaluation and
18 Research, U.S. Food and Drug Administration
19 Steve Cohen, PhD
20 Vice President, Division of Statistical and Data
21 Sciences, RTI International
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2 Selena Daniels, PharmD
6 Ebony Dashiell-Aje, PhD
10 Sara Eggers, PhD
11 Decision Support and Analysis Team, Office of
12 Program and Strategic Analysis, Office of
13 Strategic Programs, Center for Drug Evaluation and
15 Sonya Eremenco,
16 Associate Director, Patient-Reported Outcome
17 Consortium, Critical Path Institute (C-Path)
18 Sheri Fehnel, PhD
19 Vice President, Patient-Centered Outcomes
20 Assessment, RTI Health Solutions
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2 Richard Gershon, PhD
3 Professor of Medical Social Sciences and
4 Preventative Medicine, Northwestern University
5 Gary Globe, PhD
6 Director, Global Health Economics, Amgen
7 Telba Irony, PhD
8 Deputy Director, Office of Biostatistics and
9 Epidemiology, Center for Biologics Evaluation and
11 Laura Lee Johnson, PhD
12 Acting Director, Division of Biometrics III,
13 Office of Biostatistics, Office of Translational
14 Science, Center for Drug Evaluation and Research,
16 Meena Khare, PhD
17 Senior Advisor for Statistical Programs, Division
18 of Research and Methodology, National Center for
19 Health Statistics, Centers for Disease Control and
20 Prevention
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2 Scott Komo, DrPH
3 Division of Biometrics III, Office of
4 Biostatistics, Office of Translational Science,
5 Center for Drug Evaluation and Research, U.S. Food
6 and Drug Administration
7 Kerry Jo Lee, MD
8 Office of New Drugs, Center for Drug Evaluation
9 and Research, U.S. Food and Drug Administration
10 Isabelle Lousada,
11 President and CEO, Amyloidosis Research Consortium
12 Kimberly McCleary,
13 Acting Executive Director and Managing Director,
14 FasterCures
15 Susan McCune, MD
16 Director, Office of Pediatric Therapeutics, Office
17 of the Commissioner, U.S. Food and Drug
18 Administration
19 Megan Moncur,
20 Office of Biostatistics and Epidemiology, Center
21 for Biologics Evaluation and Research, U.S. Food
22 and Drug Administration

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2 Theresa Mullin, PhD
3 Director, Office of Strategic Programs, Center for
4 Drug Evaluation and Research, U.S. Food and Drug
5 Administration
6 April Naegeli, DrPH
7 Research Scientist, Eli Lilly and Company
8 Sally Okun,
9 Vice President, Advocacy, Policy and Patient
10 Safety, PatientsLikeMe
11 Elektra Papadopoulos, MD
12 Associate Clinical Outcome Assessments Staff,
13 Office of New Drugs, Center for Drug Evaluation
14 and Research, U.S. Food and Drug Administration
15 Elisabeth Piault-Louis, PharmD
16 Associate Director Patient-Centered Outcomes
17 Research Oncology, Genentech, a member of the
18 Roche Group
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A P P E A R A N C E S
Tejashri Purohit-Sheth, MD
Clinical Deputy Director, Division of
Anesthesiology, General Hospital, Respiratory,
Infection Control and Dental Devices, Office of
Device Evaluation, Center for Devices and
Radiological Health, U.S. Food and Drug
Administration
Kai Ruggeri, PhD
Director, Global Research Analytics for Population
Health (GRAPH), Columbia University
Elizabeth Stuart, PhD
Associate Dean for Education; Professor,
Department of Mental Health, Department of
Biostatistics, Department of Health Policy and
Management, Johns Hopkins Bloomberg School of
Public Health

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A P P E A R A N C E S
Pujita Vaidya,
Acting Director, Decision Support and Analysis
Team, Office of Program and Strategic Analysis,
Office of Strategic Programs, Center for Drug
Evaluation and Research, U.S. Food and Drug
Administration
Celia Witten, MD, PhD
Deputy Director, Center for Biologics Evaluation
Research, U.S. Food and Drug Administration

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C O N T E N T S
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Welcome
Pujita Vaidya 12
Opening Remarks
Theresa Mullin, PhD 18
Patient-Focused Drug Development:
Defining Key Terminology
Meghana Chalasani 27
Overview of FDA's Approach to PFDD Guidance 1
Laura Lee Johnson, PhD 32
Session I: Defining Research Objectives and
Methodological Considerations for Designing
Studies to Collect Patient Experience Data
Moderator: Michelle Campbell, PhD 47
FDA Presentation:
Ebony Dashiell-Aje, PhD
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Kunthel By, PhD 55
Moderated Panel Discussion 63
Kunthel By, PhD; Steve Cohen, PhD;
Ebony Dashiell-Aje, PhD;
Richard Gershon, PhD; Meena Khare, PhD;

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C O N T E N T S
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Elisabeth Piault-Louis, PharmD
Audience Question and Answer 112
Session II: Methodological Considerations for
Data Collection, Analysis and Operationalization
Moderator: Scott Komo, DrPH 144
FDA Presentation
Selena Daniels, PharmD 146
Moderated Panel Discussion 156
Steve Cohen, PhD; Selena Daniels, PharmD;
Sheri Fehnel, PhD; Gary Globe, PhD;
Isabelle Lousada; Kai Ruggeri, PhD
Audience Question and Answer 201
Session III: Translating Best Practice into Real
Practice - Developing Guidin

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GUIDANCE 1 December 18, 2017 - U S Food and Drug ... · PDF fileguidance 1 december 18, 2017 page 1 1 2 u.s. food and drug administration 3. center for drug evaluation and research