23
Nov
2023
PROGRAMME
09:00
Registration opens
09:30 – 09:35
Opening Session
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Introductory remarks from the co-moderators
Hans-Georg EichlerConsulting physician, Association of Austrian Social Insurance InstitutionsHans-Georg Eichler is the Consulting Physician of the Association of Austrian Social Insurance Institutions. He was the Senior Medical Officer of the European Medicines Agency for over 14 years, and Professor and Chair of clinical pharmacology at the Medical University of Vienna. In 2011, he was a Visiting Scholar at MIT, where he participated in the NEW Drug Development ParadIGmS Initiative (NEWDIGS), focused on enhancing the capacity of the global biomedical innovation system to sustainably deliver new, better, affordable therapeutics to the right patients faster. Dr. Eichler is also a lead co-author of a publication focused on the role of RWE in access and reimbursement decisions for personalized medicines, recently published in the Clinical Pharmacology and Therapeutics (CP&T) Journal.
Karen FaceySenior Adviser HTA, FIPRA RWE4Decisions FacilitatorKaren Facey worked as a statistician in the pharmaceutical sector and UK medicines regulation, before becoming founding Chief Executive of the first national HTA agency in Scotland. Since 2003, Karen has been an independent consultant on health policy, HTA and patient involvement. She is passionate about holistic HTA to determine value and use of health service data to improve patient care. Karen is lead co-author of the paper on ‘RWE to Support Payer/HTA Decisions about Highly Innovative Technologies in the EU – Actions for Stakeholders’. She has just completed EC-funded research in the IMPACT HTA project developing tools to support OBMEA.
09:35 – 09:45
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Data policies and patient access – EU Spanish Presidency priorities
Enrique Terol GarcíaCoordinating Advisor on Health, Permanent Representation of Spain to the European UnionEnrique Terol is Health Counsellor in the Permanent Representation of Spain to the European Union and represents the interests of Spain in the area of health. His professional experience includes the clinical practice, managerial positions as Medical Director and CEO of Primary and Specialised Healthcare in private and public institutions and healthcare planning. He was Deputy General Director of Quality and Health Planning of the Ministry of Health of Spain in charge of the development inter alia of the Spanish Strategy of ischemic heart disease, Diabetes, Mental health, rare diseases and Patient safety. He worked as Health Attaché in the Spanish Permanent Representation to the EU and coordinator of the area of Health in the Spanish Presidency of the EU between 2008 and 2011. He also worked as Seconded National Expert and as Policy officer in DG SANTE developing the legal and organisational bases for and the set-up and implementation of the European Reference Networks (ERN) under the framework of the Directive of Cross-border Health. Between 2021 and 2022 worked as team leader in the Medical Service of the European Commission.
09:45 – 10:50
The Policy Context for Real-World Evidence
Presentations and panel discussion
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The role of Real-World Evidence for Sustainable Healthcare Systems
Marco MarsellaDirector of Digital, EU4Health and Health Systems Modernisation, DG SANTE, European CommissionMarco Marsella is Director responsible for Digital, EU4Health and Health Systems Modernisation at DG SANTE, European Commission.
Prior to that, he was Director of ‘Learning, Multilingualism and Accessibility’ DG CONNECT, focusing on policy, research, innovation and deployment of learning technologies and key enabling digital language technologies and services to allow all European consumers and businesses to fully benefit from the Digital Single Market. He has worked on policy development, innovation and research implementation in the areas of digital content, technologies for learning, e-inclusion, assistive technologies and better internet for kids. Prior to joining the European Commission, Marco worked as researcher and AI (artificial intelligence) specialist in Italy. -
Scaling-up Real-World Evidence generation in Europe – DARWIN EU
Patrice VerpillatHead of Real-World Evidence Workstream, European Medicines AgencyDr. Patrice Verpillat is the Head of the Real-World Evidence (RWE) Workstream of the Data Analytics Taskforce at the European Medicines Agency (EMA). He is a medical doctor, specialist in epidemiology. He has worked during 20 years in the pharmaceutical industry where he had positions in several international companies, always dealing with real world data (RWD) and non-interventional studies (NIS) to bring RWE into research, access and life-cycle product management. Dr. Verpillat has published over 70 articles in Medline referenced journals. He has been involved in many organisations such as ENCePP, ICH M14 working group, European pharma association (EFPIA) and ISPE. -
Patients’ perspectives
Dimitrios AthanasiouBoard Member, World Duchenne OrganisationDimitrios is a Board member of the World Duchenne Organization and a member of the Paediatric Committee PDCO of the European Medicines Agency. He is also a member of the EPAG and TAG in EURORDIS. Nationally, he established EUPATI NLT and the Parent Project MDA Hellas. He is a father of a boy with Duchenne Muscular Dystrophy. -
Health technology developers’ perspectives
Alexander NatzSecretary General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)Dr. Alexander Natz is the Secretary General of the European Confederation of Pharmaceutical Entrepreneurs (EUCOPE), a position he holds since 2010. He also works as a lawyer for the law firm Novacos and advises pharmaceutical and biotech companies in regulatory and pricing & reimbursement decisions from a EU and German law perspective. From 2008 to 2013, he was the head of the Brussels office of the German Pharmaceutical Industry Association (BPI). Dr. Natz also worked in the field of competition law with the European Commission and in the pharmaceutical industry. He was a research assistant at Duke University (USA) where he dealt with international pharmaceutical law. -
Q&A
10:50 – 11:00
Learnings about Real-World Evidence Generation and Evaluation
Presentation: RWE4Decisions 2023
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Karen FaceySenior Adviser HTA, FIPRA International RWE4Decisions FacilitatorKaren Facey worked as a statistician in the pharmaceutical sector and UK medicines regulation, before becoming founding Chief Executive of the first national HTA agency in Scotland. Since 2003, Karen has been an independent consultant on health policy, HTA and patient involvement. She is passionate about holistic HTA to determine value and use of health service data to improve patient care. Karen is lead co-author of the paper on ‘RWE to Support Payer/HTA Decisions about Highly Innovative Technologies in the EU – Actions for Stakeholders’. She has just completed EC-funded research in the IMPACT HTA project developing tools to support OBMEA.
11:00 – 11:25
Break
11:25 – 12:20
The Potential for Collecting Real-World Data in Early Access to Inform HTA/Payer Decisions
Presentations and panel discussion
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Review of Early Access Schemes in relation to HTA
Entela XoxiSenior Researcher, Università Cattolica del Sacro Cuore RomaTen years at Italian Medicines Agency (AIFA): Health technical officer pharmacologist (2007 – 2012) and AIFA’s Registries Co-Ordinator (2013 – 2016). IT member at EU Commission Expert Group on Safe and Timely Access to Medicines for Patients (STAMP) & IT member for “Council conclusions on strengthening the balance in the pharmaceutical systems in the EU and its Member States”. IMI ROADMAP Regulatory‐HTA‐Payer EXAG Member (2017 – 2018) and IMPACT HTA WP10 partner (2018 – 2021). Expertise in regulatory science, post-marketing patient registry, managed entry agreements’ implementation. Since 2017 Lecturer with Collaboration Agreement for Research Projects at Catholic University Sacro Cuore Rome (Italy), and consultancy activities. -
Early Access Programme in France (HTA Perspective)
Camille ThomassinHead of the Real-World Evidence Coordination Unit, Haute Autorité de Santé (HAS)Camille Thomassin, PharmD, has been head of the real-life data coordination unit at the HAUTE AUTORITE DE SANTE (HAS) since October 2022. The unit’s mission includes: coordinating the implementation and monitoring of post-reimbursement studies, as well as data collection for early access to medicines. The unit is also committed to meeting and supporting the holders of French and European registers and databases in the generation of data relevant to the HAS’s assessment of healthcare products and technologies. Previously, until 2017, Camille Thomassin was a benefit/risk assessor at the AGENCE NATIONALE DE SECURITE DU MEDICAMENT ET DES PRODUITS DE SANTE, in charge of evaluating clinical trial protocols and clinical trial data for drug marketing authorizations. Then, from 2017 to 2022, she prepared dossiers for the Transparency Commission in the context of drugs’ appraisal, mainly in oncology and hematology. At HAS, she also coordinated the production of scientific advices requested by pharmaceutical companies for drugs and activities with an international dimension, in anticipation of the implementation of the European HTA Regulation. -
Payer perspective
Jo de CockFormer CEO, National Institute of Health and Disability Insurance, Belgium (INAMI-RIZIV)Jo De Cock is the former CEO of INAMI-RIZIV. Prior to this, he was Deputy Director/Councillor for social affairs in the office of the Belgian Prime Minister (1985-1993) and then became adjunct general administrator of the National Social Security Office (1993-1995). Since its creation in 2019, Jo De Cock has been closely involved in the International Horizon Scanning Initiative, an independent entity legally registered at RIZIV/INAMI and a spin-off of the BeNeLuxAI initiative. Jo has driven forward a series of multi-stakeholder roundtables to consider use of RWE to ensure short term affordability, long-term sustainability and optimal patient care. -
Health technology developers’ perspectives
Andre Vidal PinheiroVice-President, Head of Patient Value & Access EUCAN, Global Pricing & Access, TakedaAndre Vidal Pinheiro is Takeda’s Head of Patient Value & Access for Europe and Canada. From informing Takeda’s product development plans that meet HTA-grade evidence requirements, to partnering with stakeholders for value-based healthcare solutions development, Andre and his team focus on accelerating and broaden access of Takeda’s innovative therapies to patients. Andre has been with Takeda since 2017 in multiple global and regional market access roles, and before that with Pricing & Market Access consultancy firms, giving him breadth of experience across several disease areas and therapy lifecycles. A scientist by training (and at heart), Andre holds a PhD in Biochemistry and a degree in Applied Chemistry. He is passionate about combining evidence-based decision-making, clinical development, public health & policy, the power of data, and pragmatism for the benefit of patients, caregivers and society as a whole. -
Q&A
12:20 – 12:55
Closing Session: Building Better RWE for Decisions – What’s Next?
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Looking ahead to the Belgian EU Presidency
Pedro FaconDeputy CEO, National Institute of Health and Disability Insurance, Belgium (INAMI-RIZIV)Pedro Facon is a public management professional in public health, social security and health crisis governance and management. He worked successively in research, teaching, advisory and management positions at the Public Management Institute (KULeuven), the National Institute for Health and Disability Insurance, the Cabinet Office of the Belgian federal minister of Social Security and Public Health and the Belgian Ministry of Health. From October 2020 until April 2022 he was designated Government Commissioner COVID-19 by the Belgian federal government. As from April 2022 he returned to public administration as Deputy General Administrator of the National Institute for Health and Disability Insurance. He’s Fellow at the Medical Campus of the Free University of Brussels and Guest Professor at the Department of Public Health & Primary Care of the University of Ghent.
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RWE4Decisions Priorities – Steering Group Members’ Reflections
Niklas HedbergChief Pharmacist, Swedish Dental and Pharmaceuticals Benefits Agency (TLV)Niklas Hedberg is the Chief Pharmacist at the Swedish national governmental authority, the Dental and Pharmaceuticals Benefits Agency (TLV). Niklas is the Chair of the Consortium Executive Board for EUnetHTA21. He was the Chair of the EUnetHTA Executive Board between 2018 and 2021. Niklas has been working with pricing and reimbursement since 2001. He has held positions as medical assessor and project leader; Head of the Department for New Submissions (between 2009 and 2014) and he is now the chief pharmacist. Niklas has a broad experience of different aspects of value-based evaluation and over time has seen the increasing importance for health technology assessment (HTA) both across health care systems and on local level to prepare accurate decision making. Among Niklas’ special interests have been the early development of joint scientific advice in 2009 an onward (pilots both nationally with MPA and on European level with EMA) and strategic discussions about RWD. -
Matti Aapro MDOncologist, Genolier Cancer CenterMatti Aapro is member of the Board of Directors of the Genolier Cancer Center, Genolier, Switzerland and Immediate Past-President of the European Cancer Organisation. Dr. Aapro is Executive Board member of All.Can and President of Sharing Progress in Cancer Care (SPCC), both not-for-profit associations. He serves the International Society for Geriatric Oncology (SIOG) as Executive Board Member. He is past-President of the Multinational Association for Supportive Care in Cancer (MASCC). He continues to collaborate with the European School of Oncology (ESO) where he an Executive member. He is member of the Board of the French-speaking Society for Supportive Care (AFSOS), as well as Advisor to the Japanese Association for Supportive Care in Cancer (JASCC). He has been member of the Board of the European Organization for Research and Treatment of Cancer (EORTC) and of the Board of the European Society of Medical Oncology (ESMO). He received his medical degree from the Faculty of Medicine, University of Geneva, Switzerland and was a fellow at the Arizona Cancer Center in Tucson, Arizona, USA. He was the founding chair of the Medical and Radiation Therapy Department at the European Institute of Oncology in Milan. His main interests are Breast Cancer, Cancer in the Elderly, Supportive care and access to treatment. -
Simone BoselliPublic Affairs Director, EURORDIS-Rare Diseases EuropeSimone Boselli joined EURORDIS in April 2017 as a Public Affairs Director. As member of the European and International Advocacy team, Simone contributes to European policy development and specifically represents EURORDIS in policy discussions on access to therapies, with a focus on reducing delays and inequalities, on the underlying challenges in the field of the value assessment, pricing and reimbursement of orphan medicines, and on current initiatives towards improved access. With a view to advance rare diseases as a public health priority at a global level, Simone also supports advocacy activities at Rare Diseases International and the further development of NGO Committee for Rare Diseases. He has in- depth expertise in healthcare having devised and implemented advocacy campaigns at EU and national level on a range of global health issues. -
Eline Van OverbeekeDirector, Global Access Strategy, DMD Gene Therapy, PfizerEline is dedicated to advance the implementation of innovative healthcare solutions through patient centricity and multistakeholder collaboration. She is currently Director of Global Access Strategy for DMD Gene Therapy at Pfizer and was previously a Health Economics and Outcomes Research Manager at Pfizer. In her role, Eline has co-led WP2 of the IMI EHDEN project with NICE and has contributed to public private partnership with payers regarding RWD generation and Market Access of highly innovative therapies. Prior to this role, she was affiliated with the University of Leuven in Belgium where she obtained her PhD in Patient and Public related Health Research on the topic of patient preferences and market access of gene therapies. She was a researcher, and later Steering Committee member, on the IMI PREFER project and contributed to ISPOR and HTAi patient preference and patient involvement working groups. During a secondment at the Council of Canadian Academies, she worked on the ‘From Research to Reality’ project regarding the approval and use of somatic gene therapies in Canada, commissioned by the National Research Council Canada.
12:55 – 13:00
Looking ahead to 2024 – the RWE4Decisions agenda
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Hans-Georg EichlerConsulting Physician, Association of Austrian Social Insurance InstitutionsHans-Georg Eichler is the Consulting Physician of the Association of Austrian Social Insurance Institutions. He was the Senior Medical Officer of the European Medicines Agency for over 14 years, and Professor and Chair of clinical pharmacology at the Medical University of Vienna. In 2011, he was a Visiting Scholar at MIT, where he participated in the NEW Drug Development ParadIGmS Initiative (NEWDIGS), focused on enhancing the capacity of the global biomedical innovation system to sustainably deliver new, better, affordable therapeutics to the right patients faster. Dr. Eichler is also a lead co-author of a publication focused on the role of RWE in access and reimbursement decisions for personalized medicines, recently published in the Clinical Pharmacology and Therapeutics (CP&T) Journal.
13:00 – 14:00