Patient Preference Workshop

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

Thursday, Dec 7 (Day 1): 8:30 a.m. – 5:00 p.m.
Friday, Dec 8 (Day 2): 8:30 a.m. – 1:00 p.m.
 

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 PDF iconDetailed PDF Agenda and PDF iconSpeaker Biographies 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)

PDF iconIntroduction to Session 1
Anindita Saha (FDA/CDRH)

PDF iconFDA Perspective on Patient Preference Information in Medical Product Evaluation
Anindita Saha (FDA/CDRH) and Million A. Tegenge (FDA/CBER)

PDF iconIndustry Perspective on PPI to Support Medical Product Development and Evaluation
Bennett Levitan (Janssen R&D)

PDF iconAcademic Perspective on Patient Preference Research
John F. B. Bridges (Johns Hopkins University)

PDF iconPatient 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)

PDF iconIntroduction to Session 2
Martin Ho (FDA/CDRH)

PDF iconScientific Fundamentals of PPI Studies
Juan Marcos Gonzalez (Duke University)

PDF iconPanel 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)

PDF iconIntroduction to Case Study 1
Michelle Campbell (FDA/CDER)

PDF iconFDA Clinical Perspective
Gregory Reaman (FDA/OCE)

PDF iconPatient 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)

PDF iconIntroduction to Case Study 2
Telba Irony (FDA/CBER)

PDF iconFDA Regulatory and Clinical Background
Heather Benz (FDA/CDRH)

PDF iconPatient Partnership Perspective
Catherine Kopil (Michael J. Fox Foundation)

PDF iconCERSI - 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)

PDF iconPatient Group Success in Patient Preferences
Andrea Ferris (LUNGevity Foundation)

4:45 pm

PDF iconDay 1 Closing Remarks
Kathryn M. O’Callaghan (FDA/CDRH)

 

Day 2: Friday, December 8, 2017

Time

Topic

7:30 am

Registration

8:30 am

PDF iconWelcome 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)
PDF iconSession 4 Handout

PDF iconIntroduction to Session 4
Heather Benz (FDA/CDRH)

FDA Perspective
Vishal Bhatnagar (FDA/CDER) and Million A. Tegenge (FDA/CBER)

PDF iconCERSI Perspective
Liana Fraenkel (Yale University)

PDF iconConsortium Perspective
Stephanie Christopher (Medical Device Innovation Consortium)

PDF iconPatient 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)

PDF iconIntroduction to Session 5
Mimi Nguyen (FDA/CDRH) and Michelle Tarver (FDA/CDRH)

PDF iconPatient Perspective
Cynthia Grossman (FasterCures)

PDF iconFDA Perspective
Ebony Dashiell-Aje (FDA/CDER)

PDF iconIndustry Perspective
Matt Reaney (Sanofi)

PDF iconProfessional Society Perspective
Shelby D. Reed (ISPOR)

PDF iconAcademic Perspective
C. Daniel Mullins (University of Maryland)

PDF iconPanel 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

PDF iconThe CERSI Success Story: How the CERSI Collaborations Have Helped Advance the Field of Patient Preference
Frank F. Weichold (FDA/ORSI)

12:45 pm

PDF iconSummary of the Day and Wrap-Up
Telba Irony (FDA/CBER) and Frank F. Weichold (FDA/ORSI)

 

Contact Information

For questions please contact: [email protected].