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

Overview of Guiding Principles of Engagement Chapter 1 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

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 https://powerfulpatients.org/2018/09/19/a -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.

Developing patient centric drug development organizations 2 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)?

Best practices and operational considerations 3 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?

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?

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

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

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