@article{AballeaThokagevistkVelikanovaetal.2020, author = {Aball{\´e}a, Samuel and Thokagevistk, Katia and Velikanova, Rimma and Simoens, Steven and Annemans, Lieven and Antonanzas, Fernando and Auquier, Pascal and Fran{\c{c}}ois, Cl{\´e}ment and Fricke, Frank-Ulrich and Malone, Daniel and Millier, Aur{\´e}lie and Persson, Ulf and Petrou, Stavros and Dabbous, Omar and Postma, Maarten and Toumi, Mondher}, title = {Health economic evaluation of gene replacement therapies: methodological issues and recommendations}, series = {Journal of Market Access \& Health Policy}, volume = {8}, journal = {Journal of Market Access \& Health Policy}, number = {1}, publisher = {MDPI AG}, issn = {2001-6689}, doi = {10.1080/20016689.2020.1822666}, pages = {2 -- 16}, year = {2020}, language = {en} } @article{TunisHannaNeumannetal.2021, author = {Tunis, Sean and Hanna, Eve and Neumann, Peter J. and Toumi, Mondher and Dabbous, Omar and Drummond, Michael and Fricke, Frank-Ulrich and Sullivan, Sean D. and Malone, Daniel C. and Persson, Ulf and Chambers, James D.}, title = {Variation in market access decisions for cell and gene therapies across the United States, Canada, and Europe}, series = {Health Policy}, volume = {125}, journal = {Health Policy}, number = {12}, publisher = {Elsevier BV}, issn = {0168-8510}, doi = {10.1016/j.healthpol.2021.10.003}, pages = {1550 -- 1556}, year = {2021}, abstract = {Transformative cell and gene therapies have now launched worldwide, and many potentially curative cell and gene therapies are in development, offering the prospect of significant health gains for patients. Access to these therapies depend on decisions made by health technology assessment (HTA) and payer organizations. We sought to describe the emerging cell and gene therapies market access landscape by analyzing 17 US commercial payer medical policies, and HTA reports from five European countries and Canada. We found that some US health plans applied coverage restrictions more often than others (four plans applied restrictions in all decisions, while four plans applied restrictions in <30\% of decisions). The European and Canadian HTA bodies recommend access to fewer therapies than US health plans, reflecting a more stringent approach in the context of limited evidence and high scientific uncertainty that is commonly associated with these treatments. Our findings suggest that patient access to approved cell and gene therapies is restricted in all regions studied, though the nature of these restrictions differs between US health plans and the European/Canada HTA recommendations. Payers, HTA groups, pharmaceutical companies, and other stakeholders should collaborate to more clearly define the "uncertainties" and develop market access policies that balance benefits of early access with ongoing data collection to close evidence gaps over time.}, language = {en} } @article{KohliMaschioCartieretal.2022, author = {Kohli, Michele A. and Maschio, Michael and Cartier, Shannon and Mould-Quevedo, Joaquin and Fricke, Frank-Ulrich}, title = {The Cost-Effectiveness of Vaccination of Older Adults with an MF59-Adjuvanted Quadrivalent Influenza Vaccine Compared to Other Available Quadrivalent Vaccines in Germany}, series = {Vaccines}, volume = {10}, journal = {Vaccines}, number = {9}, publisher = {MDPI AG}, issn = {2076-393X}, doi = {10.3390/vaccines10091386}, year = {2022}, abstract = {Enhanced quadrivalent influenza vaccines that include an adjuvant (aQIV) or a high dose of antigen (QIV-HD), which stimulate a stronger immune response in older adults than the standard vaccine (QIVe), are now approved. The objective of this research is to compare available vaccines and determine the cost-effectiveness of immunizing persons aged 65 years and above with aQIV compared to QIVe and QIV-HD in Germany. A compartmental transmission model calibrated to outpatient visits for influenza in Germany was used to predict the number of medically attended infections using the three vaccines. The rates of hospitalizations, deaths, and other economic consequences were estimated with a decision tree using German data where available. Based on meta-analysis, the rVE of -2.5\% to 8.9\% for aQIV versus QIV-HD, the vaccines are similar clinically, but aQIV is cost saving compared to QIV-HD (unit cost of EUR 40.55). All results were most sensitive to changes in vaccine effectiveness. aQIV may be cost-effective compared to QIVe depending on the willingness to pay for additional benefits in Germany. As aQIV and QIV-HD are similar in terms of effectiveness, aQIV is cost saving compared to QIV-HD at current unit prices.}, language = {en} } @article{PetykoKaloEspinetal.2021, author = {Petyk{\´o}, Zsuzsanna Ida and Kal{\´o}, Zolt{\´a}n and Espin, Jaime and Podrazilov{\´a}, Kateřina and Tesař, Tom{\´a}š and Maniadakis, Nikos and Fricke, Frank-Ulrich and Inotai, Andr{\´a}s}, title = {Development of a core evaluation framework of value-added medicines: report 1 on methodology and findings}, series = {Cost Effectiveness and Resource Allocation}, volume = {19}, journal = {Cost Effectiveness and Resource Allocation}, number = {1}, publisher = {Springer Science and Business Media LLC}, issn = {1478-7547}, doi = {10.1186/s12962-021-00311-6}, year = {2021}, abstract = {Background Medicines that are based on known molecules and are further developed to address healthcare needs and deliver relevant improvement for patients, healthcare professionals and/or payers are called value-added medicines (VAMs). The evaluation process of VAMs is heterogeneous across countries, and it has been primarily designed for originator pharmaceuticals with confirmatory evidence collected alongside pivotal clinical trials. There is a mismatch between evidence requirements by public decision-makers and evidence generated by manufacturers of VAMs. Our objective was to develop a core evaluation framework for VAMs. Methods Potential benefits offered by VAMs were collected through a systematic literature review and allocated to separate domains in an iterative process. The draft list of domains and their applicability were validated during two consecutive virtual workshops by health policy experts representing countries with different economic statuses, geographical and decision-making}, language = {en} } @article{dePouvourvilleCunninghamFrickeetal.2023, author = {de Pouvourville, G{\´e}rard and Cunningham, David and Fricke, Frank-Ulrich and Lindgren, Peter and Mantovani, Lorenzo and Murphy, Linda A. and Sol{\`a}-Morales, Oriol and Mestre-Ferrandiz, Jorge and Akehurst, Ron}, title = {Across-Country Variations of Real-World Data and Evidence for Drugs}, series = {Value in Health}, volume = {26}, journal = {Value in Health}, number = {4}, publisher = {Elsevier BV}, issn = {1098-3015}, doi = {10.1016/j.jval.2023.01.009}, pages = {3 -- 10}, year = {2023}, abstract = {Objectives: This study aimed to describe the role of real-world data (RWD) and real-world evidence (RWE) in health technology assessment (HTA) in 5 European countries and to identify the hurdles to the acceptance of RWE and suggest directions toward its more effective use. Methods: Authors from France, Germany, Italy, and Sweden used a common template to extract evidence. For England, the Cancer Drugs Fund was described and analyzed as a particular model for the use of RWD to provide evidence for coverage decisions and managed entry agreements. Results: In all countries except Germany, HTA bodies acknowledged the relevance of RWD/RWE to address postlaunch uncertainties. In Germany, evidence from randomized controlled trials remains the gold standard, and evidence based on RWD is generally rejected. Multiple sources of RWD exist, but the quality, the immediate relevance of existing sources, and their interoperability limit their adaptation to the specifics of a given drug. This leads to skepticism about the validity of the evidence. Timing is also a key issue: the production of evidence may not be synchronized with the HTA and pricing bodies' agendas. The Cancer Drugs Fund case emphasizes that a strong partnership among all stakeholders and a pragmatic use of existing data, alongside clinical evidence provided by companies, are key success factors. Conclusions: A continuous investment in national health information systems is a key issue for providing valid RWE. Processes and aids to guide the acceptability and usage of RWE derived from pairing between sources and questions are essential.}, language = {en} } @article{GozzoPatersonWongetal.2022, author = {Gozzo, Lucia and Paterson, Kenneth and Wong, Olivier and Megerlin, Francis and Geldmacher, Jan and Popoli, Patrizia and Jommi, Claudio and Fricke, Frank-Ulrich and De Sol{\`a}-Morales, Oriol and Kamae, Isao and Rasi, Guido and Drago, Filippo}, title = {Towards a European harmonization of health technology assessment recommendations executive paper of European regulatory conference focused on the EU commission proposal to harmonize HTA}, series = {Frontiers in Drug Safety and Regulation}, volume = {2}, journal = {Frontiers in Drug Safety and Regulation}, publisher = {Frontiers Media SA}, issn = {2674-0869}, doi = {10.3389/fdsfr.2022.970661}, pages = {7}, year = {2022}, abstract = {Health Technology Assessment (HTA) is a multidisciplinary process which supports policy "decision-making", including those related to drug reimbursement and price negotiation based on comprehensive clinical and non-clinical evaluations. The discrepancies in HTA recommendations among European countries are related to the differences in health care systems and willingness to invest in health care, differences in assessment methodologies and appraisal practices, and variations in economic constraints, and may produce disparities in terms of patient access to medicines. The political discussion about the adoption of the European Regulation on HTA lasted for years due to concerns about its feasibility and its implications for national health care systems In this paper we highlighted the importance to harmonize HTA process in Europe and some critical issues emerged during the discussion among experts in the field carried out at the European Regulatory Conference.}, language = {en} }