Patient Involvement in the Development and Safe Use of CIOMS XI Patient Involvement in the Development and Safe Use of Medicines Judith W. Zander, MD Director of Office of Pharmacovigilance and Epidemiology U.S. Food and Drug Administration April 19-20, 2018 www.fda.gov

Proposed Principles to Define Scope Should be consistent with CIOMS Mission: propose limiting the scope to safety Should address areas of current gaps and not include topics that are duplicative of or better addressed by other initiatives (e.g. ICH, IMI2 PREFER, WEB RADR etc.) Should not include processes that are governed by national or regional legislation www.fda.gov

FDA Current Practices Patient Perspective for Medicinal Safe Use Clinical Development Participation of a patient representative on all FDA Advisory Committee Meeting Panels Open testimony by the public at all FDA Advisory Committee meetings and other public meetings and workshops Voice of the Patient: Patient Focused Drug Development Initiative Prescription Drug User Fee Act (PDUFA) > The Voice of the Patient: A Series of Reports from FDA's Patient-Focused Drug Development Initiative Post Market Pharmacovigilance MedWatch Online link: To report MedWatch online for Consumers (https://www.accessdata.fda.gov/scripts/medwatch/index.cfm?action=reporting.home) MedWatch 3500B form: To report MedWatch on paper for Consumers. Also includes instruction (https://www.fda.gov/downloads/AboutFDA/ReportsManualsForms/Forms/UCM349464.pdf) MedWatch Learn for Consumers: This is an instructional video https://www.accessdata.fda.gov/scripts/MedWatchLearn/consumers.htm Framework for benefit risk counseling to patients about drugs with a REMS https://www.fda.gov/downloads/ForIndustry/UserFees/PrescriptionDrugUserFee/UCM577883.pdf Research on Signal Detection in Social Media has not provided evidence additional benefit over traditional methods

Possible Gaps for CIOMS XI to Address Drug Development and Risk Management: Planning and Conduct of Clinical Trials including safety communication with participants approaches to safety data reporting from CT participants ethical aspects for interactions and human protection Risk Management What risk management burdens are acceptable to patients Extending concepts from CIOMS IX (? App for risk management) Pharmacovigilance: Methods for signal detection and evaluation from patient reported data from spontaneous reports and other data collection vehicles (PSP, registries) Suggest future research on the best way to bring the patient perspective rather than individual patients perspective from a diverse community tor regulatory considerations Additional research is needed to optimize signal detection from social media

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What to Report to FDA MedWatch Use the MedWatch form to report adverse events that you observe or suspect for human medical products, including serious drug side effects, medication errors/product use errors, product quality problems, and therapeutic failures for Prescription or over-the-counter medicines, as well as medicines administered to hospital patients or at outpatient infusion centers Biologics (including blood components, blood and plasma derivatives, allergenic, human cells, tissues, and cellular and tissue-based products (HCT/Ps) Medical devices (including in vitro diagnostic products) Combination products Special nutritional products (infant formulas, and medical foods) Cosmetics Foods/beverages (including reports of serious allergic reactions) www.fda.gov

What Not to Report to FDA MedWatch Tobacco: Tobacco product problems should be reported to the Safety Reporting Portal Safety Vaccines: Report vaccine events to the Vaccine Adverse Event Reporting System (VAERS) https://vaers.hhs.gov/esub/index Investigational (study) Drugs: Report investigational (study) drug adverse events as required in the study protocol and send to the address and contact person listed in the study protocol Mandatory reporting by regulated industry: Drugs and Biologics Applicable Regulations Devices Reporting on Dietary Supplements Reporting on Veterinary Medicine Products Reports FDA Does Not Handle (e.g. CPSC, FTC, State Health Departments) and where to send them)