7:30-8:15 am

8:15-8:30 am

8:30-10:00 am

Session 1: Exploring the Concepts of Safety and Tolerability – Incorporating the Patient VoiceChair: Bindu Kanapuru, MD – Medical Officer, Division of Hematology Products, Office of Hematology and Oncology Products (OHOP), FDA

Presentations:

• The Patient Perspective: James (Randy) Hillard, MD – Professor of Psychiatry, Michigan State University
• Safety vs Tolerability – A Clinician’s Perspective: Crystal Denlinger, MD, FACP – Associate Professor, Department of Hematology/Oncology; Chief, Gastrointestinal Medical Oncology; Director, Survivorship Program; Deputy Director, Phase 1 Program, Fox Chase Cancer Center
• Safety vs Tolerability – The Canadian Regulatory Perspective: Katherine Soltys, MD – Acting Director, Bureau of Medical Sciences, Therapeutic Products Directorate, Health Products and Food Branch, Health Canada
• Patient Advocate and Survivor: Karen E. Arscott, DO, MSc – Associate Professor of Medicine-Patient Advocate and Survivor, Geisinger Commonwealth School of Medicine

Panel Discussion: 

Additional Panelists:

• Daniel O’Connor, MB, ChB, PhD, MFPM – Expert Medical Assessor, Medicines and Healthcare products Regulatory Agency (MHRA)
• Eric Rubin, MD – Vice President and Therapeutic Area Head, Merck & Co., Inc.
• Paul G. Kluetz, MD – Acting Associate Director of Patient Outcomes, OCE, FDA
• Selena R. Daniels, PharmD, MS – Team Leader, Clinical Outcome Assessments Staff (COA Staff), OND, CDER, FDA

Q & A

10:00–10:30 am

10:30 am – Noon

Session 2: Assessment of Safety and Tolerability – Emerging Patient-Reported MethodsChair: Steven Lemery, MD, MHS – Lead Medical Officer (Team Leader), OHOP, FDA

Presentations:

• Adverse Event Reporting – CTCAE and PRO-CTCAE: Lori Minasian, MD, FACP – Deputy Director, Division of Cancer Prevention, National Cancer Institute (NCI), National Institutes of Health (NIH)
• Overview of the PRO-CTCAE Industry Working Group – Objectives, Goals, Activities: Sheetal Patel, PhD – Outcomes Research Scientist – Oncology, Genentech, a member of the Roche Group; Co-Chair, PRO-CTCAE Industry WG
• Experience of Implementing PRO-CTCAE in Clinical Trials: Anna Rydén, PhD – Director, Patient Science, AstraZeneca
• Toxicity over Time (ToxT) – Longitudinal Adverse Event Analysis in Cancer Clinical Trials: Gita Thanarajasingam, MD – Senior Associate Consultant, Division of Hematology, Mayo Clinic; Assistant Professor of Medicine, Mayo Clinic College of Medicine

Panel Discussion: 

Additional Panelists:

• Christopher R. Blackburn – Cancer Patient and Senior Corporate Development Manager, GZA GeoEnvironmental (unable to attend)
• Daniel O’Connor, MB, ChB, PhD, MFPM – Expert Medical Assessor, MHRA
• Rajeshwari (Raji) Sridhara, PhD – Division Director, Division of Biometrics V, Office of Biostatistics (OB), Office of Translational Sciences (OTS), CDER, FDA

Q & A

Noon – 1:00 pm

1:00–2:30 pm

Session 3: Analysis and Display of PRO-Based Tolerability Data – Metrics and Paths ForwardChair: Laura Lee Johnson, PhD – Deputy Director, Division of Biometrics III, OB, OTS, CDER, FDA

Presentations:

• Regulatory Considerations in Europe for Trials Collecting PRO Data on Tolerability: Yolanda Barbachano, PhD – Senior Statistical Assessor, Licensing Division, MHRA
• Defining the Population, Missing Data vs Completion Rate and Presentation Methods: Mallorie H. Fiero, PhD – Mathematical Statistician, Division of Biometrics V, OB, OTS, CDER, FDA
• Analysis Strategies for PRO CTCAE: Diane Fairclough, DrPH – Professor, Biostatistics, Colorado School of Public Health

Panel Discussion: 

Additional Panelists:

• Corneel Coens, MSc – Lead Statistician, QoL Department, EORTC
• Joseph Cappelleri, PhD, MPH, MS – Senior Director of Biostatistics, Pfizer, Inc.
• Sandra A. Mitchell, PhD, CRNP – Research Scientist and Program Director, Outcomes Research Branch, NCI, NIH
• Paul G. Kluetz, MD – Acting Associate Director of Patient Outcomes, OCE, FDA

Q & A

2:30–3:00 pm

3:00–4:30 pm

Session 4: From Individual Symptoms to Overall Side Effect BurdenChair: Paul G. Kluetz, MD – Acting Associate Director of Patient Outcomes, OCE, FDA

Presentations:

• Concise Measurement of Cancer Treatment Side Effect Burden and its Relationship to Outcomes: David Cella, PhD – Professor and Chair, Department of Medical Social Sciences, Feinberg School of Medicine, Northwestern University
• Symptoms and Functional Interference During Cancer Treatment: Charles S. Cleeland, PhD – McCullough Professor of Cancer Research, University of Texas MD Anderson Cancer Center
• How EORTC Considers Overall Side Effect Burden and Strengths/Limitations of Summating Individual Symptoms into An Overall Side Effect Score: Galina Velikova, BMBS(MD), PhD, FRCP – Professor, University of Leeds

Panel Discussion:

Additional Panelists:

• Mary Lou Smith, MPA, MBA, JD – Co-Founder, Research Advocacy Network
• Daniel O’Connor, MB, ChB, PhD, MFPM – Expert Medical Assessor, MHRA
• Ethan Basch, MD, MSc – Director, Cancer Outcomes Research Program, University of North Carolina
• Michelle Campbell, PhD – Reviewer and Scientific Coordinator, COA Qualification Program, COA Staff, OND, CDER, FDA

Q & A

4:30 – 5:00 pm

5:00 pm