Applicability of Academic Real-World Data Research in the Case Studies of the HTx Project to Practical Health Technology Assessment Work

Carl Björvang¹Johan Pontén¹Anders Viberg¹Andrea Manca²Georgia Salanti³Pekka Siirtola⁴Gema García Sáez⁵Saskia Knies^6,7Diana Delnoij^6,7Dalia M Dawoud^8,9Jamie Elvidge⁸Lifang Liu¹⁰Bertalan Nemeth¹¹Zoltán Kaló^11,12*

• ¹Tandvards- och lakemedelsformansverket, Stockholm, Sweden
• ²Centre for Health Economics, University of York, York, United Kingdom
• ³Institute of Social and Preventive Medicine, Multidisciplinary Center for Infectious Diseases, Universitat Bern, Bern, Switzerland
• ⁴Biomimetics and Intelligent Systems Group, Oulun yliopisto, Oulu, Finland
• ⁵Bioengineering and Telemedicine Group, Centrode Tecnología Biomédica, ETSI de Telecomunicación, Universidad Politecnica de Madrid, Madrid, Spain
• ⁶Zorginstituut Nederland, Diemen, Netherlands
• ⁷Erasmus School of Health Policy & Management, Erasmus Universiteit Rotterdam, Rotterdam, Netherlands
• ⁸Science, Evidence and Analytics Directorate, National Institute for Health and Care Excellence, London, United Kingdom
• ⁹Cairo University Faculty of Pharmacy, Cairo, Egypt
• ¹⁰European Organisation for Research and Treatment of Cancer, Brussels, Belgium
• ¹¹Syreon Research Institute, Budapest, Hungary
• ¹²Center for Health Technology Assessment, Semmelweis University, Budapest, Hungary

Next Generation HTA (HTx) is a recently finalised EU project which aimed to connect academic HTA researchers with HTA practitioners. This paper studies the applicability of the academic RWD research conducted through four case studies within the HTx project to practical HTA work. As a first step HTA agency representatives of the HTx consortium sent a survey out to each case study leader, enquiring about the usefulness of their RWD research methods for HTA agencies in various situations, and barriers to their utilisation. The second step was to conduct follow-up interviews with the case study leaders, to further understand the new RWD research methods and their applicability to HTA practices. The results show a great variety in when and how the methods could be used for practical HTA evaluations. Generally, there was a high potential for the RWD research methods to be used to estimate treatment effects and cost-effectiveness, while they were less adapted for estimating natural disease progression and identifying relevant comparators. However, there were significant barriers to the use of these methods for practical HTA evaluations, even in the aspects of evaluations the methods were designed to handle. These barriers ranged from the availability of RWD and need for partial reliance on RCT data, to required expertise in areas such as data evaluation, statistics and medical knowledge. The case studies show that RWD can be used for a range of HTA aspects and in various situations. However, no RWD research method is a silver bullet that is applicable for all aspects in all situations. As such, they can make significant contributions to the work of HTA agencies, but as part of a wider tool set.