RWE4Decisions Symposium: Developing Real-World Evidence to Deliver Innovation in HTA

Welcome coffee

Opening session

Chaired by Laura Batchelor, RWE4Decisions Secretariat

• Welcome
• Real-World Evidence in the 2024-2025 EU health agenda
  

  MEP Tomislav Sokol

  Member of the European Parliament’s Committee on the Environment, Public Health and Food Safety (ENVI) and former rapporteur of the European Health Data Space Regulation

  Recently re-elected as a Member of the European Parliament, MEP Sokol is a substitute member of the European Parliament’s Committee on the Environment, Public Health and Food Safety (ENVI), and of the subcommittee on Public Health (SANT). He is also EPP coordinator on public health.
  He was rapporteur for the European Health Data Space regulation, and is currently rapporteur for the regulation of the General Pharmaceutical Package (GPL).
  Prior to his election as MEP, he has been a Member of the Croatian Parliament from 2017 to 2019, as well as a Senior Lecturer at the Zagreb School of Economics and Management.
  

  Bio

Plenary Session: The Policy Context for Real-World Evidence to Support Innovation in HTA

• Moderated by
  

  François Meyer

  RWE4Decisions Facilitator

  François joined the management team of HAS in 2005, leading the setup of the HTA Division across key areas like pharmaceuticals, medical devices, interventional and diagnostic procedures, and public health programs. He expanded HAS’s role in economic analysis within France’s HTA field. He then focused on international cooperation, serving on the HTAi Board for two terms, coordinating EUnetHTA activities, and representing HAS in projects including Post Launch Evidence Generation with HTA-EMA cooperation. Before joining HAS, François held management roles at the French Medicines Agency (now ANSM). He was active at the EMA, serving on the Committee for Orphan Medicinal Products.

  Bio
• Council of the EU perspective
  

  Enrique Terol García

  Coordinating Advisor on Health, Permanent Representation of Spain to the European Union

  Enrique Terol is Health Counsellor at the Permanent Representation of Spain to the European Union. 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 (ERNs) under the framework of the Directive on Cross-border Health. Between 2021 and 2022 he worked as team leader in the Medical Service of the European Commission.
  

  Bio
• The European Medicines Agencies Network Strategy 2028 and DARWIN EU
  

  Patrice Verpillat

  Head of Real-World Evidence, European Medicines Agency (EMA)

  Dr. 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.

  Bio
• Payer perspective
  

  Christoph Rupprecht

  Head of Department for Health Policy and Health Economics, AOK Rheinland/Hamburg

  Christoph Rupprecht is Head of Department of Health Policy & Health Economics at the Allgemeine Ortskrankenkasse (AOK), a German public health insurance organisation, where he has worked at for the past 23 years.
  Christoph holds degrees from the University of Cologne, the University Diderot Paris VII and the Freie Universität Berlin. His main interests are in regulatory innovation and value, disease management, as well as cross-border cooperation.

  Bio
• Patient perspective
  

  Anne-Pierre Pickaert

  Member, Acute Leukemia Patients Network (ALAN)

  Anne-Pierre Pickaert has over 20 years of public health and access to medicines experience with a wide range of healthcare stakeholders (charity, patient organisation, governmental agency, consultancy and pharmaceutical industry).
  She initially worked as a cancer prevention specialist through nutrition and lifestyle (World Cancer Research Fund and the French National Cancer Institute) and later worked as market access director (Kantar Health, Lundbeck, and Sanofi). Anne-Pierre holds a MSc in Economic Evaluation of Health Technology Assessment from the University of York.
  Anne-Pierre founded Care4Access, a consultancy dedicated to patient engagement and advocacy. She is an engaged patient advocate with Entraide aux Greffés de Moelle Osseuse (EGMOS) and Association Laurette Fugain, two French patient organisations. She is a steering committee member of the Acute Leukemia Advocates Network (ALAN), as well as a board member of the Francophone Society for Bone Marrow Transplant and Cellular Therapies (SFGM-TC).
  

  Bio
• Industry perspective
  

  Alexander Natz

  Secretary-General, European Confederation of Pharmaceutical Entrepreneurs (EUCOPE)

  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 an 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.
  

  Bio
• Q&A

Coffee break

Plenary Session: Launching the new RWE4Decisions Stakeholder actions to Generate Better Real-World Evidence for HTA/Payer Decisions

• Moderated by
  

  Ashley Jaksa

  VP Scientific Strategy and Partnerships, Aetion

  Ashley Jaksa is Market Access Scientific Strategy Lead at Aetion. She focuses on collaborating with decision-makers to enhance their use of RWE and setting methodological guidance for RWE generation. She also works with Aetion’s biopharma clients on incorporating RWE in their global market access strategy. She is passionate about determining how to get the best therapies to the right patients at the right time through the use of the best evidence. Previously, she led analytic services at Context Matters where she consulted with biopharma clients on designing and executing analytic projects. She has presented her research at numerous international conferences including ISPOR, HTAi, and AMCP, and published her findings in various peer-reviewed publications. She holds a bachelor’s degree from the University of Michigan-Ann Arbor and a MPH from Yale University.

  Bio
• Presentation: RWE4Decisions work in 2024
  

  Dr Karen Facey

  RWE4Decisions Facilitator

  Karen 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.

  Bio
• Presentation: Launching the Stakeholder Actions to Generate Better Real-World Evidence for HTA/Payers
  

  Ashley Jaksa

  VP Scientific Strategy and Partnerships, Aetion
• National HTA/Payer perspective
  

  Piia Rannanheimo

  Chief Specialist, Finnish Medicines Agency (FIMEA)

  Piia Rannanheimo is a pharmacoeconomist at the Finnish Medicines Agency (Fimea). She joined Fimea’s HTA-team in 2010. With a background in Pharmaceutical Sciences, she has contributed to numerous national, Nordic and European HTAs and worked as a visiting researcher at CADTH and at the Center for Health Economics at University of York. She also serves as an expert for the Council for Choices in Health Care in Finland.
  She’s also been a member of several working groups led by the Ministry of Social Affairs and Health in Finland, covering rational use of medicines, HTA, RWD and data management. Currently, she’s working as a project manager in a work that aims for the establishment of national medicinal product data repository.
  

  Bio
• HTA/Payer Collaboratives perspective
  

  Shaun Rowark

  Associate Director for Data Access and Analysis, National Institute for Health and Care Excellence (NICE)

  Shaun Rowark has worked in UK healthcare informatics for over a decade, starting out as a data analyst in hospital trusts to now being an associate director overseeing NICE’s access to healthcare data sources and developing its burgeoning data analysis function.
  He has managed several programmes of work including automated analysis and computable guidance, helping to ensure that NICE’s products are suitable for a digital healthcare system.
  Shaun holds a masters degree in Health Informatics from University College London.
  

  Bio
• Patient perspective
  

  Stefan Joris

  Board Chairman, RaDiOrg – Rare Disease Belgium

  Stefan Joris joined the Belgian Cystic Fibrosis Association in 2016. In 2018 he joined the board of Cystic Fibrosis Europe as Secretary and became chairman of the ‘Policy and Public Interest Group’ and in 2019 he became chairman of the board of RaDiOrg, Rare Diseases Organization Belgium.
  In these positions Stefan plays a key role in the growing involvement of patients representatives and patients associations as stakeholders and partners in the evolving health environment. The BCFA also launched a RWE program, VERA, in 2022 to collect QoL data of their patients for primary and secondary use.
  

  Bio
• Industry perspective
  

  Andre Vidal Pinheiro

  Vice-President and Head of Patient Value & Access – Europe & Canada, Takeda

  Andre 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 patient access to Takeda’s innovative therapies.
  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.

  Bio
• Q&A

Closing Session: Way forward – RWE4Decisions agenda to deliver in future

• Next steps for the implementation of EU HTA Regulation
  

  Carlos Martín Saborido

  Advisory Member, Directorate General for Common Portfolio of SNS and Pharmacy Services, Spanish Ministry of Health

  Dr Martín Saborido is a Health Economist working on HTA economic evaluation and RWE applied to HTA. He has been working as a research associate at the Spanish Health Technology Assessment Agency mainly doing HTA reports and in the Regional HTA Unit in Madrid as health economics research fellow. Internationally, Dr Martin has been working in the Liverpool Review and Implementation Group (University of Liverpool) in the UK assessing economic evaluations on behalf of NICE and doing HTA reports for the NIHR Health Technology Assessment programme. He worked as Public Health Economist in the Joint Research Centre (European Commission) developing economic evaluations in the public health frame.
  
  In the private sector, he worked at several pharma consultancies such as RTI Health Solutions and World Health Management. In the academic ground, he has been lecturer in Statistics, Research Methods and Economic Evaluation of Health Technologies, mentoring and guiding several PhD and MSc thesis. In 2020 he gained a permanent position as Scientific Officer in the Department of Health Economics at the National School of Public Health in Madrid.
  In April 2021 he was appointed as Advisory member at the Ministry of Health in the Area of Pharmacy, working on strategy and innovation of pharma policy and price & reimbursement with special interest in RWE for reimbursement decisions. This position was held until November 2024 when he was appointed Head of HTA Agency (Instituto de Salud Carlos III)
  Dr Martin Saborido is currently member of the Advisory Committee of DARWIN EU initiative (EMA) and member of the steering group of RWE4 Decision.

  Bio
• Q&A
• Conclusions and Payer reflections
  

  Jo De Cock

  Senior Adviser and former CEO, Belgian Institute for Health and Disability Insurance (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.

  Bio