Lessons Learned: Challenges and Wins...Lessons Learned: Challenges and Wins Risa Hayes, Ph.D. PRO...
Transcript of Lessons Learned: Challenges and Wins...Lessons Learned: Challenges and Wins Risa Hayes, Ph.D. PRO...
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Lessons Learned: Challenges and Wins
Risa Hayes, Ph.D. PRO Consortium Co-Director
THIRD ANNUAL PATIENT-REPORTED OUTCOME (PRO) CONSORTIUM WORKSHOP
April 4, 2012 Silver Spring, MD
Co-sponsored by
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Agenda
• Lessons Learned: Challenges and Wins – Introduction – Risa Hayes, PhD – Eli Lilly and Company
– Asthma Working Group - Linda Nelsen, MHS – Merck Sharpe & Dohme
– Depression Working Group - Steven I. Blum, MBA – Forest Research Institute
– Functional Dyspepsia Working Group - Robyn T. Carson, MPH – Forest Research Institute
– Irritable Bowel Syndrome Working Group - Mollie J. Baird, MPH – Ironwood Pharmaceuticals
– Non-Small Cell Lung Cancer Working Group - Rajiv Mallick, PhD – Daiichi Sankyo
• FDA Response – Laurie Beth Burke, RPh, MPH; Marc K. Walton, MD, PhD
• Open floor discussion 2
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A Consortium of Pharma
2011 • Teleconferencing across 9 different time zones is
only the beginning…
2012 • Challenges: Time, member turnover,
uncertainty, agendas • Wins: Face-to-face meetings, non-
competitive environment 3
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Interactions with the FDA
2011 The good news and the not so good news…
2012 Challenges: Meeting of the minds Wins: Liz, FDA telecons/FTF meetings
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The Process
2011 Making it up as we go along…
2012 Challenges: Physician payment, CIAs,
sharing data Wins: SharePoint, Scientific Data
Disclosure Policy 5
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PRO Consortium Objectives
2011 Broadening our horizons…
2012 Challenges: Keeping in scope Wins: Communication subcommittee,
ePRO subcommittee
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Content Validity Stage (New)
2011 Finding a path forward…
2012 Challenges: Project agreements, PRO
ownership, mixed methods Wins: Vendor selection process, expert
panels, member participation 7
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Lessons Learned: Asthma
Working Group Linda Nelsen
THIRD ANNUAL PATIENT-REPORTED OUTCOME (PRO) CONSORTIUM
WORKSHOP
April 4, 2012 Silver Spring, MD
Co-sponsored by
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Lessons Learned: Depression Working Group
Steven I. Blum Forest Research Institute
THIRD ANNUAL PATIENT-REPORTED OUTCOME (PRO) CONSORTIUM WORKSHOP
April 4, 2012 Silver Spring, MD
Co-sponsored by
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Project Initiation/Management
• Wins: Vendor Selection, Completed Literature/Instrument Reviews, Developed Study Protocol, IRB Approval, Initiated Concept Elicitation Interviews
• Challenges: Execution of Project Agreements, Agreement on Population/Inclusion/Exclusion Criteria, Project Scope
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Expert Panel
• Wins: Established Expert Panel (L. Carpenter, J. Fawcett, M. Thase, M. Trivedi), Held 1st Expert Panel Meeting (WebEx) to Review Study Documents, Scheduled Face-to-Face Item Generation Meeting
• Challenges: Selection/Recruitment Process, Understanding of PRO/DDT Guidance documents, Scheduling, Engagement
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Working Group
• Wins: Added New Member Firm, Project Management, Completed Scientific Data Disclosure Plan, Submitted Two Research Abstracts
• Challenges: Representative Turnover, Revisiting Past Decisions, Revision of Diagnostic Criteria (DSM-5), Engagement/Participation of Members
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Lessons Learned: Functional Dyspepsia
Working Group Robyn T. Carson
Forest Research Institute
THIRD ANNUAL PATIENT-REPORTED OUTCOME (PRO) CONSORTIUM
WORKSHOP
April 4, 2012 Silver Spring, MD
Co-sponsored by
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Scoping Stage Summary Document (SSSD)
• Wins: – Superior responsiveness/engagement between the FDA
GI Division/SEALD and FD WG to reach consensus on the target patient population
• Submitted SSSD and received timely feedback from the FDA (< 60 days)
• Expeditiously granted F2F Type C Meeting • Timely resolution
– SEALD fellow actively involved in FD WG calls to facilitate decision-making on the SSSD revisions
• Challenge: Defining the FD patient population for qualitative research
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Working Group
• Wins: – Very engaged representatives from
member firms – Representatives with different skill sets
(eg, PRO, Clinical, Regulatory as needed) • Challenge: Scheduling conflicts
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Next Steps
• Wins: RFP developed and issued to coordinating committee for approval in a timely manner
• Challenges: – Execution of sponsor contracts and
impact on qualitative research timelines – Carrying the momentum forward from
SSSD stage into qualitative work stage
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Lessons Learned: Irritable Bowel Syndrome
Working Group Mollie J. Baird
Ironwood Pharmaceuticals
THIRD ANNUAL PATIENT-REPORTED OUTCOME (PRO) CONSORTIUM WORKSHOP
April 4, 2012 Silver Spring, MD
Co-sponsored by
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Expert Panel Meeting
• Wins: – Successfully developed items as a team
• KOLs, IBS WG member firms, non-member participants, RTI, and C-Path
– Superior collaboration, communication, and engagement among all team members
– Meeting preparation and document reviews before the meeting enabled decision making
• Challenges: – SEALD presence and feedback in the meeting may have
been advantageous
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Working Group
• Wins:
– Very engaged and active representation
– Non-member participants add value to the discussions
– FDA and SEALD were actively involved early in the process
• Challenges:
– Reaching consensus through biweekly teleconferences, which could ultimately compromise the qualitative research timelines
– Covering all necessary agenda items in biweekly teleconferences
– Difficulty in coordinating schedules for ad hoc teleconferences
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Qualitative Research Stage
• Wins: – Member firms were able to watch and listen to patient
interviews in real time – RTI (vendor) is flexible, collaborative, and knowledgeable
in the PRO GI arena • Challenges:
– Reaching consensus and making decisions in adherence to the agreed upon timelines
– Allow more time in between patient interviews to update and obtain feedback from the IBS WG
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Lessons Learned: Non-Small Cell Lung Cancer
Working Group Rajiv Mallick, PhD (co-chair)
Daiichi Sankyo THIRD ANNUAL
PATIENT-REPORTED OUTCOME (PRO) CONSORTIUM WORKSHOP
April 4, 2012 Silver Spring, MD
Co-sponsored by
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Overview
• Update on progress and issues • CORE Messages adapted to NSCLC
– Classification of Endpoint types – Biomarker, Human-Modulated
– Continuum of Direct vs. More Indirect Patient Benefits
– Direct Benefits: Concept of measurement (proximal vs. distal to core pathophysiology)
– Context of Use
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Update on Progress • Scoping Stage, DDT meeting (July 2011)
– Finalized conceptual framework (living document) • Pulmonary vs. non-pulmonary symptoms • Symptoms vs. impacts (eg. sleep disturbance, energy)
– Context of use • common target population of registration trials – stage III/IV
(exploratory analysis of stage I/II); ECOG PS 0-2 • Known epidemiology: co-morbid COPD
– Endpoints • Improvement or delayed deterioration in pulmonary symptoms
• Interviewed, finalized vendor (HRA) • Brief core messages slide deck adapted to NSCLC
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Types of Endpoint Assessments to Document Tx Benefit - NSCLC
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(ECOG PS) PRO Biomarker (RECIST)
Physiologic or lab findings that can be measured
without human assessment
Unobservable concepts (e.g., feelings, sensations)
Clinical judgment needed
Self-report feasible and appropriate?
No clinical judgment needed
Observable concepts (e.g., signs, events, behaviors)
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Relationship to Treatment Benefit in NSCLC
Direct Measures
Indirect Measures Continuum
PFS in 2nd/3rd line treatment
ECOG PS (maintained/improved)
Symptoms, Functioning, Overall Survival
Tumor Response (CR, PR, SD, PD); PFS in 1st line tx
• Direct assessment (of tx benefit) • Indirect assessment (of tx benefit)
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Direct Evidence of Tx Benefit: Concepts of Measurement
Disease –defining concepts
Proximal disease impact concepts
Distal disease impact concepts
Distal impact on general life concepts
Cough
Shoulder Pain
Dyspnea
Hoarseness
Wheezing
Swelling of the face/neck
Lack of energy
Depression
Social functioning
Overall impact on HRQL
Anxiety Weight loss
Decreased appetite
Memory
Ambulation
Difficulty with activities of daily living
Helplessness/ hopelessness
Independence
Sleep disturbance
Concentration/clarity of thinking Shortness of
breath
Tightness in chest
Phlegm
Swallowing
Loss of stamina
Life interference
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An Endpoint Model displays the role and hierarchy of relevant outcome concepts in clinical trials (i.e., all primary and secondary endpoints)
Overall Survival
Endpoint Heirarchy
Secondary with Hierarchy
http://www.fda.gov/downloads/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/UCM205269.pdf
Primary
Exploratory
Concept Endpoints COA/Biomarker/Survival
Survival
Biomarker (based on RECIST) Biomarker (based on RECIST) PRO PRO
Progression-Free Survival Response Pulmonary symptoms Non-pulmonary symptoms
Context of Use: Endpoint Model
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Panel Discussion 5 Lessons Learned: FDA Perspective
Laurie Burke, RPh, MPH Marc Walton, MD, PhD
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Stages of DDT Qualification
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Stage Start End Initiation •DDT tracking #
assigned •FDA receives Letter of Intent
•FDA request for initial briefing package
Consultation & Advice
•FDA requests initial briefing package •FDA receives initial briefing package
•FDA request for qualification package
Review •FDA receives qualification package
•Qualification letter sent & decision posted on FDA website
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Initiation Stage • Request for DDT# • Letter of Intent
– Concept of measurement – Context of use
• Disease definition • Targeted patient population • Study design considerations • Targeted claim
• If FDA agrees that a COA is needed, and if FDA determines resources are adequate… – FDA agrees to begin the qualification process – FDA requests an initial briefing package
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Briefing Package
• Introduction – Concept of measurement – Context of Use – Overview of current COA development – Plan to involve external expertise
• Summaries – Documentation of content validity – Documentation of other measurement properties – Interpretation of scores – Language translation and cultural adaptation – Administration mode – Data collection – Appendices
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Consultation & Advice Stage
• COA developer submits protocols and study summaries (i.e., briefing packages) for FDA input when needed
• Briefing package reviews with discussion and response from SEALD and other relevant disciplines
• When FDA perceives instrument development is complete, FDA will request a Qualification Package
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Review Stage
• Qualification Package reviewed by SEALD and relevant disciplines
• FDA communicates review conclusions to submitter
• If qualified, a qualification statement is posted on the FDA website
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Lessons Learned—Needs Idenitified Goal: Quicker response and better advice
– FDA staff is becoming more familiar with DDT program Initiation Stage
– Need better disease definition and subpopulation identification in advance
– Need more specificity in naming the proposed concept of measurement and context of use
C and A stage – Need more concise submissions (e.g., study summaries
only) – Earlier submission and advice (generally, sooner is better)
• Review stage – FDA needs to provide submission templates – FDA needs a review MAPP to clarify the review process
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COA Review Status
• Active COA DDTs (26) – Initiation Stage: 8 – Consultation & Advice Stage: 16 (7 from C-Path PRO Consortium)
– Review Stage: 2 • Other COA DDTs (10)
– Declined: 5 – On Hold: 3 – Withdrawn: 2
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Why then a PRO Consortium?
Two roads diverged in a wood, and I— I took the one less traveled by, And that has made all the difference.
Road Not Taken by Robert Frost 36