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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