Patient engagement at the EMA 27 November 2018 Patient engagement at the EMA IAPO Congress, 25 May 2018 Latin American Patient Academy Presented by Nathalie Bere Public Engagement Department

The European Medicines Agency (EMA) 27 November 2018

Patient engagement at EMA; our experience

Interaction with patients: a progressive journey… 27 November 2018 Interaction with patients: a progressive journey… Public Hearings Involving young people Patient & Consumer Working Party (PCWP) Dialogue with patients Working group with patients 1996 2003 2006 2017 1995 2000 2005 2014 Ongoing… EMA created Patients become Committee members Framework of interaction with patient & consumer organisations Public Engagement Department Systematic inclusion of patient input along medicine lifecycle

Increasing involvement in EMA activities 27 November 2018 Increasing involvement in EMA activities

Involvement along the entire medicine regulatory lifecycle 6

Different ways to engage: Members of EMA Management Board Members of EMA Committees Representing the patient community Patients and Consumers Working Party (PCWP) EMA consultations Workshops Representing their own organisation Scientific Advice procedures Expert meetings Committee consultations Review of documents Patients as individual experts

Learn by experience! Be flexible!

Engagement methodologies; a menu of options One size does not fit all! Surveys Face to Face Preference Elicitation Committee meetings In writing

Support and training is vital! Webpages Annual training day Info-sheets One-to-one personalised support Videos; EMA basics

Who do we engage with? Any organisation representing EU patients or consumers (must meet eligibility criteria : application form on EMA website) Any individual patient or carer can register to work with the EMA (application form on EMA website)

Patients and Consumers Working Party (PCWP) Platform for dialogue and exchange between the EMA and patient organisations Representation from 20 organisations, EMA committees & Management Board Four plenary meetings per year, and consulted as and when needed

Wider Engagement; share practices and promote multi-stakeholder discussions EMA cross-agency departments Healthcare professionals; GPs, pharmacists, nurses.. National Competent Authorities EMA/FDA exchange Patient initiatives

70% of comments led to changes Feedback Meeting minutes/report Thank you acknowledgement Show when input is taken into account Review of documents 70% of comments led to changes Monitoring and measuring Questionnaires sent to patients and assessors who participate Leads to proposals for improvement Continuous monitoring Demonstrate value

Example of value of input ; Scientific Advice procedures 2017: 90 responses

Patient input changed final advice in 26% of cases Patient input impacted final advice in 43% of cases

Examples Safety reporting: selection of Black Triangle based on recommendations by patients and consumers (PCWP) Expert meeting on use of retinoids: Patient representatives provided useful information on current use and awareness of Pregnancy Prevention Plans. They suggested that stronger, simpler and clearer messages and use of reminders and digital technology to improve compliance, especially considering the younger population Scientific Advice : Patient representative highlighted the burden of lung (re-) biopsy procedures and advised to limit them to strictly required cases Committee Expert meeting: Patient representatives highlighted that some non statistically significant differences in endpoints can be life-changing for Duchenne patients in daily activities.

Scientific Advice assessor Feedback Deputy Executive Director: “Over the past 20 years, I have seen many changes at the Agency, in particular the Agency’s interactions with civil society representatives, which have evolved and strengthened over time” “The PCWP in its ten years of existence has been a catalyst for a profound culture change at EMA and to some extent at national agencies”. PCWP Patient co-chair “I have found this experience extremely interesting and enriching and I am very honoured to have been able to take part” Patient involved “This patient provided exceptionally valuable and knowledgeable input and participated in discussions at a very differentiated level” Scientific Advice assessor “I felt that the patients opinions and information are taken seriously for further decisions” Patient involved “Members of the scientific team have been very helpful to phone me and to explain the procedures” Patient involved

Challenges Patients availability and language barriers Ensuring support to enhance participation Providing a clear definition of patients role to manage expectations Handling potential conflicts of interest Representation – how to also gather information from larger groups How to demonstrate the value / impact of patients

Summary messages Engaging with patients; brings everyday aspects of living with a disease into scientific discussions helps bridge the gap between clinical trial data and real world data increases transparency, awareness and understanding Initiate engagement in a stepwise approach; learn together what format works best; Ensure engagement is mutually beneficial Engaging with patients leads to more meaningful outcomes for everyone!

Questions? Nathalie Bere Patient Engagement Public Engagement Department nathalie.bere@ema.europa.eu www.ema.europa.eu European Medicines Agency 30 Churchill Place • Canary Wharf • London E14 5EU • UK Telephone +44 (0)20 3660 8452 Send a question via our website www.ema.europa.eu/contact