patient focused drug development · • narcolepsy – sept. 24, 2013 • neurological...
TRANSCRIPT
Patient Focused Drug Development
Kim McCleary Director of Strategic Initiatives, FasterCures
December 6, 2013
Washington Representatives Retreat
Three Roles – Three Perspectives
Past President & CEO The CFIDS Association of America
Consultant to Unite Narcolepsy initiative
Director of Strategic Initiatives FasterCures
• Alpha-1 antitrypsin deficiency • breast cancer • chronic Chagas disease • female sexual dysfunction • fibromyalgia – NEXT: Dec. 10 • hemophilia A, hemophilia B, von
Willebrand disease, and other heritable bleeding disorders
• HIV – June 14, 2013 • idiopathic pulmonary fibrosis
• IBS/gastroparesis/gastroesophageal reflux disease
• lung cancer – June 28, 2013 • myalgic encephalomyelitis/chronic
fatigue syndrome – April 25, 2013 • narcolepsy – Sept. 24, 2013 • neurological manifestations of inborn
errors of metabolism • Parkinson's disease and Huntington's
disease • pulmonary arterial hypertension • sickle cell disease – Feb. 7
Opportunities to Participate
Comments to docket
(unlimited) Range: 14-228
Live webcast
(up to ~1,000)
In-person meeting
(up to ~150)
Panelists
(10)
Surveys, other means to expand engagement
4
Use of polling questions with audience response tools to quantify answers (stakeholders only)
Biggest participation challenge
Similar Campaigns
• Survey based on FDA’s questions
• Webinar series to educate, engage & empower patients
• Survey based on FDA’s questions • Piloted questions from NHC
Patient Stratification Tool
• Used multiple choice & open text formats
• Webinar series to educate, engage & empower patients • Added coaching opportunities
• Created a matrix of patient archetypes
Outcomes: ME/CFS Successful lead condition in PFDDI series
Set new participation records (rare or prevalent)
2,046 survey respondents – largest patient-based data set about narcolepsy
Interim analysis of 1,350 respondents’ answers issued prior to FDA meeting; final dataset submitted to FDA via docket
Data set has attracted attention from researchers; now available on Sage Bionetwork’s Synapse platform under a Creative Commons 3.0 license
Newly mobilized patient community; elevated presence
Outcomes: Narcolepsy
NHC Patient Stratification Tool
Sample Question from NHC’s PST
Looking Ahead 16 more meetings in series; 2 on calendar, 10 other conditions
selected, 4 more to be added in 2015
FDA is open to participating in similar meetings hosted by orgs – example of Duchenne muscular dystrophy policy forum on Dec. 12
Broader distribution of National Health Council’s Patient Stratification Tool & implementation manual to assist groups to elicit patient perspectives, collect information & report findings
Genetic Alliance + PhRMA collaboration to help selected groups collect information for PFDD using PEER Platform
FasterCures collaborating with other organizations including NHC, NORD, PAN, Alpha-1 & industry partners, to expand opportunities for patient perspective to be included drug development and regulatory decision-making
From John Jenkins, FDA (2010)
Patient Participation in FDA Regulatory Decisions
One patient representative appointed by FDA to advisory committee that reviews NDA
Industry sponsors must refrain from pre-approval marketing; some confusion about how to engage with patients & when it’s permissible pre-approval
“Voice of the Patient” report series (selected conditions)
Decision Factor Evidence & Uncertainties Conclusions & Reasons
Analysis of Condition
Current Treatment Options
Benefit
Risk
Risk Management
Benefit-Risk Summary Assessment
From “Structured Approach to Benefit-Risk Assessment in Drug Regulatory Decision-Making,” issued by FDA, February 2013
Condition
Product
Patient perspective will inform these 2 rows
FDA Benefit-Risk Framework
Is it possible for patients to communicate their benefit expectations & risk tolerances?
• Measurement/methodology? • Reliability? • Durability? • Generalizability? • Only in the face of specific choices, or more
broadly?
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