1. Guiding Principles of Engagement Outline
October 2018
Beverly Harrison, Sr. Director and Head, Patient Support, GMO Team, Matthias, Kerry, Nikos, Regina

2. Overview of Guiding Principles of Engagement Chapter1
Principles for patient involvement
Developing patient centric drug development organizations
Best practices and operational considerations
Measures for success
Future Directions (added this section 2 3 4

3. 1
Principles for patient involvement Add case examples(where it worked and where it did not work-get EMA document (scientific advice,leaflet review)
Transparency: Making goals / objectives clear, reducing possibility of misunderstanding, and setting appropriate expectations Patient Bill of Rights-
Communication: Importance of ensuring effective and consistent communication including terminology selection and use
Adding communicating uncertainty
Reciprocity: Mutual benefit, two-way direction flow of value, shared goals – approaching and conducting patient involvement / engagement as a partnership
Initial Source List:
EFPIA Code of Practice on Relationships Between the Pharmaceutical Industry and Patient Orgs
Hoos et. al 2015: Partnering With Patients
DIA Considerations Guide for Patient Engagement
BIO Guiding Principles for Interaction with Patient Advocacy Organizations
PFMD: Patient Engagement Quality Guidance
PFMD: Book of Good Practices
-patient-engagement-manifesto-6- principles-of-partnership/
Discussion Questions:
Other bioethical principles that are relevant and should be included (e.g. autonomy, beneficence, nonmaleficence, justice?) Add Review with bioethicist once available.

4. Developing patient centric drug development organizations2
Developing patient centric drug development organizations
Culture: Embedding patient-centeredness within the organization, attaining buy-in to the value and integrating into considerations for approach to day-to-day work
Capability: Proficiency in ability to apply patient engagement methodologies and other necessary competencies
Supporting infrastructure/ frameworks: Providing the organizational structure and resources necessary for patient engagement to occur efficiently and sustainably. Includes supporting partners (e.g. trial sites, academia)
Initial Source List:
Smith and Benattia 2016: The Patient’s Voice in Pharmacovigilance
Hoos et al. 2015: Partnering With Patients in the Development and Lifecycle of Medicines: A Call for Action.
Lowe et al. 2016: Increasing Patient Involvement in Drug Development
DIA Considerations Guide for Patient Engagement
DIA Capturing the Value of Patient Engagement Presentation
Discussion Questions:
Considerations for companies of different sizes and available resources (e.g. large pharma vs. early / late stage biotech)?

5. Best practices and operational considerations3
Best practices and operational considerations
Selection: Considerations for engaging PAGs vs. Patients, criteria to consider for selection
Training and Education: Enabling stakeholders for increased value
Legal / Regulatory / HCC / Privacy:
Contracts
conflict of interest (patient friendly language)
Compensation (NICE principles)
fair market value (PFMD, WeCan, NHC)
Health literacy (refer to body of workfollow up Kaisa)
Initial Source List:
DIA Considerations Guide for Patient Engagement
PFMD: PE Quality Guidance
PFMD: Book of Good Practices
CTTI: Patient Groups Project
EFPIA: Working with Patient Groups
EUPATI: Guidance for patient involvement
EMA: Patient involvement along medicines lifecycle
NHC: The Patient Information Tool
Smith and Benattia 2016: The Patient’s Voice in Pharmacovigilance
Hoos et. al 2015: Partnering With Patients
MPE/WECAN/PFMD: Reasonable Agreements between Patient Advocacy and the Pharmaceutical Industry
Discussion Questions:
Risk for overlap - do parts belong in other sections? Approach for moving forward?

6. 4
Measures for Success Adding Principles for measuring success (see DIA work)
Tracking the Organization: Establishing current organizational patient engagement baseline to measure and track against
Evaluating Individual Activities: Defining what success looks like before commencing a patient engagement activity including performance indicators
Continuous Improvement: Evaluating every engagement activity for learnings that can be applied to improve future engagement activities
Initial Source List:
DIA Considerations Paper
PFMD Patient Engagement Quality Guidance
DIA Capturing the Value of Patient Engagement Presentation
Pushparajah 2018: Making patient engagement a reality
Discussion Questions:
Ability to create and align key performance indicators for industry and patients?
Involving patients in evaluation of patient engagement activities?

7. Guiding Principles - Notes
Change title of section from “Rules of Engagement” to “Guiding Principles”
In addition to topics outlined in Beverly’s slide deck, the following topics were suggested:
Communications between/amongst stakeholders
Setting expectations
Communicating uncertainty
Ensuring information fed back to participants in patient engagement activites (should be in patient involvement in drug dev section also)
Patient Rights – Elizabeth, Kaisa will look into Patient Bills of Rights and see if applicable here
Contracting (WECAN initiative—patient friendly language)
Health Literacy—refer to body of work
Compensation – Fair Market Value
Add in principles for measuring impact
Data privacy/Data ownership/data sharing, including genetic data, placebo data sharing for future discovery—collaborate with Group 2
Future directions

8. Next Steps Revise outline by November 16
Working Group meeting early Dec to finalize the working outline
January-begin writing sections
Open pre- meeting planned for April 30
Next CIOMs meeting May 2019

9. Writing Guidelines
Be conversational in writing—trying to engage people on ideas and getting them on board, be clear not focused on simple
Use everyday words such as instead of “experience side effects” use “have side effects
Navigation—use subheadings (can’t have too many) no undifferentiated pages
Use bullet points
Headline/Summary—5-9 bullets with key messages; every chapter will require Executive Summary