An Agency Perspective on Plain Language Summaries of Publications The Future of Combination Products in the EU What Can we Learn from Medicine Development & Delivery – DIA 2019, Panel Presentation – Nicholas Brooke Executive Director PFMD An Agency Perspective on Plain Language Summaries of Publications

Patient Engagement Landscape Fragmentation 1 fragmented needs and objectives, 2 fragmented perspectives and ideas 3 fragmented during the lifecycle of medicine development 4 fragmented context from a geographic point of view 5 fragmented leadership (or even no leadership) hence the need of a collaborative platform

PFMD Membership Highlight collaboration with Eupati & co-creation

The Seven PE Quality Criteria The PE Quality Guidance provides practical guidance in planning, developing and assessing the quality of patient engagement activities and projects throughout the development and life cycle of medicines for all stakeholders. The PE Quality Guidance proposes 7 Quality Criteria which have been co- created based on pre-existing patient engagement frameworks published and in the PFMD co-creation efforts that brought together various stakeholders

Quick facts How does it work? How was it developed? The guidance introduces seven quality criteria to assess patient engagement practices. The quality criteria describe the core values that a good initiative should include in its processes. For each criteria guidance questions were developed and there is access to resources including real examples. The tool was co-developed with a large community of stakeholders (76 people from 51 organisations), representing patient associations, industry, academics, researchers and external experts. Who can use the tool? When can it be used? All stakeholders (Patients, Patients Associations, Industry, Researchers, HTAs, HCPs …) working on patient engagement projects. As a planning tool, when starting a new project As an assessment tool for ongoing or finalised projects As a gap analysis tool to help plan future projects

Patient Engagement Meta-Framework Roadmap

Projects to make PE systematically heppen Framework building workstream SYNaPsE Networking & Mapping Tool Fair Market Value Development of a practical PE meta-framework with tools that help all stakeholders to do more qualitative PE. PE Quality Guidance Book of Good Practices PE activities and Do’s and Don’ts How-to modules An interconnected platform of PE actors, initiatives, organisations and resources. SYNaPsE is evolving from landscape mapping to offering a full range of integrated PE management tools There is no agreed system in place for financial compensation that reflects the level of individual contribution and expertise of patient partners. Reasonable legal agreements PE Industry Training Plain Language Summaries for Publications Pledge to Patients This initiative aims to improve legal agreements between patient advocates and pharmaceutical companies while providing adequate protection and rules for both sides. Co-designed with 13+ organisations from around the world. Twofold objective: an e-learning program about PE, as well as an awareness tool for culture change for reaching the early and late majority within the pharma industry. Committing to more meaningful patient engagement in medicines development by listening, co-creating or communicating with patients and sharing it with the world. Co-creation of a practical guidance for writing plain language summaries in a coherent way that brings value to the patient community. Strategic communication activities with PFMD members and collaborators

Overview of the ecosystem 3 8 7 Complex landscape 2 6 5 * for clinical trials publications, various reports etc. 4 1 Guidances PFMD Quality Guidance CIOMS Capacity building /training Envision EFPIA | EPF | EFGCP | Industry Plain language summaries* Clinical trials info access Measurement PFDD NHC (US version) Legal agreements PARADIGM on CAB (on preferred agreements) WECAN survey | guiding principles | reference contracts Patient views PFMD Industry training FDA Fair market value WECAN survey extended survey EFPIA principles NHC calculator Regulatory

Value of Patient Engagement in Clinical Trials The analysis of ‘the patient-centric trials’ by the Economist indicated: drugs developed using patient-centric designs had 20% increased chance to be launched compared to drugs developed without. This remained consistent across the different therapeutic areas covered (neurology, oncology and rare diseases). patient-centric trials took less time to recruit 100 participants (4 months), compared to the other innovation types (6-11 months) and all trials (7 months). 20% Chance to be launched compared to drugs developed without. 4 months Compared to the other innovation types (6-11 months) and all trials (7 months) Source: the Economist Intelligence Unit patient-centric trials based on PubMed, 2018. Data: 2008-2016.

..the benefit is huge Figure 11: Phase II and III likelihood of launch for patient-centric trials vs all trials (%) All trials Patient-centric trials 100 80 60 40 20 All Therapy areas Neurology Oncology Rare diseases (Therapy area) Source: Pharmaprojects® | Pharmaintelligence, 2018. Data: 2012-2017.

…and the trials are much faster Figure 12: Average time to enroll 100 participants for patient-centric vs all trials (months) Rare diseases All patient-centric (Therapy area) Oncology All trials (control) Neurology All trials Patient-centric trials 5 10 15 20 25 30 35 Source: Trialtrove® | Pharmaintelligence, 2018. Data: 2012-2017.

Research on the financial value of Patient Engagement Net present value (NPV) = metric integrating key business drivers of cost, time, revenue, and risk Study: assessing the impact of patient engagement on NPV for a typical oncology development program entering phase 2 or phase 3 Results: Pre-phase 2 project NPV increase of $62MM Pre-phase 3 project NPV increase of $65MM An investment of $100,000 in patient engagement can generate NPV increase that exceeds 500-fold the investment. Source: Therapeutic Innovation & Regulatory Science 2018, Vol. 52(2) 220-229

When it comes to Combined Products ? When it comes to Combined Products