Patient Preference Workshop

Advancing Use of Patient Preference Information as Scientific Evidence in Medical Product Evaluation

Thursday, Dec 7 (Day 1): 8:30 am – 5:00 pm
Friday, Dec 8 (Day 2): 8:30 am – 1:00 pm

Tommy Douglas Conference Center (TDCC)
10000 New Hampshire Ave, Building 9
Silver Spring, MD 20903

Hosted by Johns Hopkins, University of Maryland, UCSF-Stanford, Yale-Mayo Clinic, and the Georgetown University Center of Excellence in Regulatory Science and Innovation and the U.S. Food and Drug Administration [Center for Devices and Radiological Health (CDRH), Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), and the Office of Chief Scientist, Office of Regulatory Science and Innovation].

About the workshop

Patients have unique perspectives about the value of the potential benefits and the impact of potential harms and burdens of their medical treatments. Scientists, clinicians, medical product developers, and regulators play critical roles in evaluating the benefits and risks of medical products. However, only patients live with their medical conditions and make choices regarding their own care. Reliable and accurate methods are needed in order to effectively incorporate patients’ values into decision-making processes.

Patient preference information is qualitative input or quantitative data elicited from patients about the desirability or acceptability of outcomes or other attributes of medical products, focusing particularly on trade-offs. The continued development of methods to elicit patient preference information and enhance its regulatory applications has the potential to contribute to a better understanding of the benefit-risk profiles of some medical products.

This workshop will:

Present current progress on incorporating patient preference information into medical product benefit-risk assessments
Provide examples of how patient preference can be collected, analyzed, and presented in a way that matters to stakeholders
Explore methods for appropriate measurement, interpretation, and adoption of patient preference information in a regulatory context
Identify future research and capacity needs in order to improve the use of patient preference information in a regulatory context

Workshop agenda, slides and recording

The agenda is shown below, and slides can be downloaded by clicking on the title of each talk below. A Detailed PDF Agenda (PDF) and Speaker Biographies (PDF) are also available for download.

The workshop recordings can be accessed here:

Day 1: Thursday, December 7, 2017

Time	Topic
7:30 am	Registration
8:30 am	WELCOME SESSION: Patient Input and Regulatory Science Introduction Carol Linden (FDA/ORSI) Welcome Remarks RADM Denise Hinton (FDA/OCS) Welcome from the CERSIs G. Caleb Alexander (Johns Hopkins University)
8:45 am	SESSION 1: Fundamental Concepts and Regulatory Context of PPI to Support Medical Product Development and Evaluation Chair: Anindita Saha (FDA/CDRH) Introduction to Session 1 Anindita Saha (FDA/CDRH) FDA Perspective on Patient Preference Information in Medical Product Evaluation Anindita Saha (FDA/CDRH) and Million A. Tegenge (FDA/CBER) Industry Perspective on PPI to Support Medical Product Development and Evaluation Bennett Levitan (Janssen R&D) Academic Perspective on Patient Preference Research John F. B. Bridges (Johns Hopkins University) Patient Perspective on Landscape K. Kimberly McCleary (FasterCures) Discussion and Q&A
10:15 am	Break
10:30 am	SESSION 2: Scientific Fundamentals of PPI Studies Chairs: Martin Ho (FDA/CDRH) and Leslie Wilson (UCSF) Introduction to Session 2 Martin Ho (FDA/CDRH) Scientific Fundamentals of PPI Studies Juan Marcos Gonzalez (Duke University) Panel Discussion and Q&A Martin Ho (FDA/CDRH) Telba Irony (FDA/CBER) Laura Lee Johnson (FDA/CDER) Leslie Wilson (UCSF-Stanford CERSI) Fadia T. Shaya (University of Maryland CERSI) Erica S. Spatz (Yale-Mayo Clinic CERSI) Brett Hauber (University of Washington) Juan Marcos Gonzalez (Duke University) Becky Noel (Eli Lilly and Company)
12:15 pm	Lunch
1:15 pm	SESSION 3: Discussion on In-Depth Case Studies Chair: Telba Irony (FDA/CBER) & Michelle Campbell (FDA/CDER) Introduction to Session 3 Million Tegenge (FDA/CBER)
1:20 pm	Case Study 1: Rare Pediatric Cancer Moderator: Michelle Campbell (FDA/CDER) Introduction to Case Study 1 Michelle Campbell (FDA/CDER) FDA Clinical Perspective Gregory Reaman (FDA/OCE) Patient Study Perspective Deborah A. Marshall (University of Calgary) Patient Perspective Nancy Goodman (Kids V Cancer) Panel Discussion and Q&A Michelle Campbell (FDA/CDER) Gregory Reaman (FDA/OCE) Paul G. Kluetz (FDA/OCE) Leslie Wilson (UCSF-Stanford CERSI) Deborah A. Marshall (University of Calgary) Nancy Goodman (Kids V Cancer) Tamar Krishnamurti (University of Pittsburgh) Elisabeth (Liz) Piault-Louis (Genentech)
2:45 pm	Break
3:00 pm	Case Study 2: Neurological Degenerative Disease Moderator: Telba Irony (FDA/CBER) Introduction to Case Study 2 Telba Irony (FDA/CBER) FDA Regulatory and Clinical Background Heather Benz (FDA/CDRH) Patient Partnership Perspective Catherine Kopil (Michael J. Fox Foundation) CERSI - Preference Study Perspective Ellen M. Janssen (Johns Hopkins University) and Ira Shoulson (Georgetown University) Panel Discussion Telba Irony (FDA/CBER) Heather Benz (FDA/CDRH) Kerry Jo Lee (FDA/CDER) Ellen M. Janssen (Johns Hopkins University) Ira Shoulson (Georgetown University) Catherine Kopil (Michael J. Fox Foundation) Janel Hanmer (University of Pittsburgh) Kara L. Haas (Johnson & Johnson)
4:25 pm	Introduction to Patient Group Kathryn M. O’Callaghan (FDA/CDRH) Patient Group Success in Patient Preferences Andrea Ferris (LUNGevity Foundation)
4:45 pm	Day 1 Closing Remarks Kathryn M. O’Callaghan (FDA/CDRH)

Day 2: Friday, December 8, 2017

Time	Topic
7:30 am	Registration
8:30 am	Welcome and Patient Preference Remarks Theresa M. Mullin (FDA/CDER)
8:45 am	SESSION 4: CDRH Preference Sensitive Areas Discussion: Diseases and Conditions Where Patient Preference Studies Could Be Useful Chair: Heather Benz (FDA/CDRH) Session 4 Handout Introduction to Session 4 Heather Benz (FDA/CDRH) FDA Perspective Vishal Bhatnagar (FDA/CDER) and Million A. Tegenge (FDA/CBER) CERSI Perspective Liana Fraenkel (Yale University) Consortium Perspective Stephanie Christopher (Medical Device Innovation Consortium) Patient Partnership Perspective Frank Hurst (FDA/CDRH) and Melissa West (Kidney Health Initiative) Q&A
10:00 am	Break
10:15 am	SESSION 5: Capacity Building and Sustainability Chair: Mimi Nguyen (FDA/CDRH), Michelle Tarver (FDA/CDRH) and Fadia Shaya (University of Maryland) Introduction to Session 5 Mimi Nguyen (FDA/CDRH) and Michelle Tarver (FDA/CDRH) Patient Perspective Cynthia Grossman (FasterCures) FDA Perspective Ebony Dashiell-Aje (FDA/CDER) Industry Perspective Matt Reaney (Sanofi) Professional Society Perspective Shelby D. Reed (ISPOR) Academic Perspective C. Daniel Mullins (University of Maryland) Panel Discussion and Q&A Mimi Nguyen (FDA/CDRH) Cynthia Grossman (FasterCures) Ebony Dashiell-Aje (FDA/CDER) Matt Reaney (Sanofi) Shelby D. Reed (ISPOR) C. Daniel Mullins (University of Maryland) Joseph S. Ross (Yale University) R. Scott Braithwaite (New York University)
12:30 pm	The CERSI Success Story: How the CERSI Collaborations Have Helped Advance the Field of Patient Preference Frank F. Weichold (FDA/ORSI)
12:45 pm	Summary of the Day and Wrap-Up Telba Irony (FDA/CBER) and Frank F. Weichold (FDA/ORSI)

Contact info

[email protected]