“Guiding principles for patient participation in the generation of benefit-risk data”: Chapter Outline Leo Russo on behalf of the Group 2 Topic 3 sub-team CIOMS XI F2F Meeting October 23-24th, 2018 Berlin, Germany

Sub-team Update Monthly calls established. Three have been held. Members (13): Mick Foy, Linda Härmark, Stephen Heaton, Kaisa Immonen, Marie Lindquist, Tsunehiro Oi, Sten Olsson, Ravi Patel, Brian Edwards, Leo Russo, Martina Schaeublin, Panos Tsintis, Christian Thomeczek. Goals : Maintain focus on Benefit-Risk data. Process: assess existing relevant work, synthesize it, identify important gaps, and make recommendations. Strive to assess and make recommendations on a global basis.

Guiding principles for patient participation in the generation of benefit-risk data Current Environment (Note: landscape assessments are needed here) Settings and context Post-authorization safety studies (PASS) Post-authorization effectiveness studies (PAES) Adverse Event Management: Spontaneous reports Patient support programs Expanded access programs (cohorts) and compassionate use programs (named patients) Observational studies including outcomes research, patient registries Internet access/Social media Personal sensors and wearable devices (new forms of data) Patient preference studies Risk minimisation measures including registries used for Restricted Access Clinical Trials (pre- and post-authorisation) Traditional RCTs Pragmatic, Large Simple Trials Legal Actions Crisis management

Guiding principles for patient participation in the generation of benefit-risk data Current Environment (cont) Channels of Communication Patients- Regulatory Authorities Patients-Industry Patients-Healthcare Professionals Patients-Patients/Patient Organizations Rules and Criteria for engagement Regulators Patient Organizations Medical Societies (i.e. outside US and EU) Methods of Communicating Data Patient Reported Outcomes (PROs)

Guiding principles for patient participation in the generation of benefit-risk data The Patient’s Perspective (writing led by a patient) Primary data collection vs. Secondary Use of Patient’s Data. How do patients’ views on privacy and consent differ across these scenarios? Do patients understand the connection between their data and public health? Gaps, Needs, and Untapped Opportunities Mobilizing patients, caregivers, and patient families as advocates for open access to patient benefit-risk data. Increasing patient engagement in regards to providing their data. Engaging military and prison populations Challenges Meeting Legal requirements (ex. GDPR) The Evolving Global Environment on Data Privacy Utilizing a very large volume of data (experience with RWE) Social media-establishing its reliability, its value, and guard against manipulation. Societal Attitudes towards industry and regulators/government partnering with patients.  Create higher levels of engagement across patient populations Training needs including capacity and capability Research involving children at different ages including informed assent. Vulnerable populations

Guiding principles for patient participation in the generation of benefit-risk data Future directions Conclusions and recommendations Case Study (i.e. lessons learned). Appendices

Next Steps Clarify our Landscape Assessment needs. Identify patient to lead “Patient Perspective” section. Linda has approached an external person Finalize Chapter Outline Identify Section Leads (2 per section) Use breakout session to develop working plan to take forward.