Centre for Pharmaceutical Policy and Regulation

2025

Biomarkers for neurodegenerative diseases in regulatory decision-making by the European Medicines Agency

Hermans, A. M. M., Bakker, E., Starokozhko, V., den Otter, L., Elferink, A. J. A., Bradshaw, A., Guizzaro, L., Mol, P. G. M. & Pasmooij, A. M. G., Jan 2025, In: Alzheimer's and Dementia: Translational Research and Clinical Interventions. 11, 1, 13 p., e70072.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Differential times of submission and approval of CFTR modulators for the treatment of Cystic Fibrosis in the United States and the European Union

Costa, E., Girotti, S., Mathieu, C., Castellani, C., Ross, J. S., Taylor-Cousar, J. L. & Leufkens, H. G. M., Jan 2025, In: Journal of Cystic Fibrosis. 24, 1, p. 125-132 8 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Duration of Time Intervals for Risk Minimization Measure Effectiveness Studies

Essink, S. C. M., Zomerdijk, I. M., Goedecke, T., Straus, S. M. J. M., Gardarsdottir, H. & De Bruin, M. L., Apr 2025, In: Clinical Pharmacology and Therapeutics. 117, 4, p. 1106-1114 9 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Evolving Recommendations for Patient Populations Among Oncology Medicines: A Quantitative and Qualitative Analysis

Hogervorst, M. A., Vreman, R. A., Oduol, T. A., Mantel-Teeuwisse, A. K., Goettsch, W. G. & Kesselheim, A. S., 10 Mar 2025, (E-pub ahead of print) In: Clinical Pharmacology and Therapeutics.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

Managed Entry Agreements for High-Cost, One-Off Potentially Curative Therapies: A Framework and Calculation Tool to Determine Their Suitability

Callenbach, M. H. E., Vreman, R. A., Leopold, C., Mantel-Teeuwisse, A. K. & Goettsch, W. G., 2025, In: PharmacoEconomics. 43, 1, p. 53-66 14 p., 103433.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Medicines not recommended for inclusion in the who essential medicines list: a retrospective observational study

Costa, E., Del Grosso, V., Cappello, B., Genazzani, A. A., Huttner, B., Leufkens, H. G. M., Magrini, N., Nonino, F., Wirtz, V. J., van den Ham, H. A. & Moja, L., 17 Mar 2025, In: Frontiers in Medicine. 12, 9 p., 1517020.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Patient satisfaction with prescription opioid education in primary care

Davies, L. E. M., van den Ham, H. A., Schellekens, A. F. A., Philbert, D. & Bouvy, M. L., Feb 2025, In: Patient Education and Counseling. 131, 7 p., 108572.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Recommendations for essential medicines for multiple sclerosis in low-resource settings

Saylor, D., Rijke, N., McDonell, J., Laurson-Doube, J., Avasarala, J., Baldin, E., Banerjee, T. K., Bogdanovic, I., Bove, R., Chawla, D. K., Costello, K., Del Giovane, C., Abkari, N. E., Filippini, G., Foschi, M., Gonzalez-Lorenzo, M., Helme, A., Jacobs, D., Kalincik, T. & Mantel-Teeuwisse, A. & 19 others, Minozzi, S., Navas, C., Nonino, F., Ojo, O. O., Ozcan, B., Pasi, E., Peryer, G., Prato Chichiraldi, A., Ridley, B., Sokhi, D. S., Traboulsee, A., Tramacere, I., Tye, J. S. N., Vecchi, S., Viswanathan, S., Xie, F., Zeineddine, M., Schunemann, H. & Piggott, T., Apr 2025, In: Multiple Sclerosis Journal. 31, 4, p. 454-473

Research output: Contribution to journal › Article › Academic › peer-review

Risk Minimisation Measures of Advanced Therapy Medicinal Products Authorised in the EU Between 2009 and 2023: A Cross-Sectional Study

Essink, S. C. M., Zomerdijk, I. M., Straus, S. M. J. M., Gardarsdottir, H. & De Bruin, M. L., 10 Apr 2025, (E-pub ahead of print) In: Drug Safety.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

Stimulating development of innovative medicines in the European Union: does a new definition for unmet medical need add value?

Bloem, L. T., Leufkens, H. G. M., Berends, S. E., Vreman, R. A., Hollak, C. E. M., van Weely, S., de Lannoy, L. M., Bertens, P. J. A. & Pasmooij, A. M. G., Jan 2025, In: Drug Discovery Today. 30, 1, 104251.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Time and cost involved in measuring access to medicines: the case of Albania

Petro, E., Joosse, I. R., Siebers, A. C. M., Martopullo, T., Hart, R. P. M., Mantel-Teeuwisse, A. K., van den Ham, H. A. & Suleman, F., Dec 2025, In: BMC Research Notes. 18, 1, 104.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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2024

A blueprint for health technology assessment capacity building: lessons learned from Malta

Abraham, K., Kvamme, I., Magrin Sammut, S., de Vries, S., Formosa, T., Dupree, R., Corro Ramos, I., Goettsch, W. & Franken, M., 29 Feb 2024, In: International Journal of Technology Assessment in Health Care. 40, 1, 6 p., e11.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Access to childhood cancer medicines in South Africa: a health systems analysis of barriers and enablers

Joosse, I. R., van den Ham, H. A., Mantel-Teeuwisse, A. K., Perumal-Pillay, V. A. & Suleman, F., 12 Jul 2024, In: Journal of Pharmaceutical Policy and Practice. 17, 1, 21 p., 2372033.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Added benefit and revenues of oncology drugs approved by the European Medicines Agency between 1995 and 2020: retrospective cohort study

Brinkhuis, F., Goettsch, W. G., Mantel - Teeuwisse, A. & Bloem, L. T., 28 Feb 2024, In: The BMJ. 384, 12 p., e077391.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Adoption of new medicines in primary care: A comparison between the uptake of new oral anticoagulants and diabetes medicines

Dankers, M., Hek, K., Mantel-Teeuwisse, A. K., van Dijk, L. & Nelissen-Vrancken, M. H. J. M. G., Mar 2024, In: British Journal of Clinical Pharmacology. 90, 3, p. 819-827 9 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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An Inclusive Civil Society Dialogue for Successful Implementation of the EU HTA Regulation: Call to Action to Ensure Appropriate Involvement of Stakeholders and Collaborators

Desmet, T., Julian, E., Van Dyck, W., Huys, I., Simoens, S., Giuliani, R., Toumi, M., Dierks, C., Dierks, J., Cardone, A., Houÿez, F., Pavlovic, M., Berntgen, M., Mol, P., Schiel, A., Goettsch, W., Gianfrate, F., Capri, S., Ryan, J. & Ducournau, P. & 2 others, Solà-Morales, O. & Ruof, J., Mar 2024, In: Journal of market access & health policy. 12, 1, p. 21-34 14 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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A proposed analytical framework for qualitative evaluation of access to medicines from a health systems perspective

Joosse, I. R., van den Ham, H. A., Mantel-Teeuwisse, A. K. & Suleman, F., 7 Jun 2024, In: BMC Research Notes. 17, 1, 7 p., 159.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Availability and stock-outs of paediatric antiretroviral treatment formulations at health facilities in Kenya and Uganda

Jacobs, T. G., Okemo, D., Ssebagereka, A., Mwehonge, K., Njuguna, E. M., Burger, D. M., Colbers, A., Suleman, F., Mantel-Teeuwisse, A. K. & Ooms, G. I., Jul 2024, In: HIV Medicine. 25, 7, p. 805-816 12 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Disease severity-based subgrouping of type 2 diabetes does not parallel differences in quality of life: the Maastricht Study

Werkman, N. C. C., García-Sáez, G., Nielen, J. T. H., Tapia-Galisteo, J., Somolinos-Simón, F. J., Hernando, M. E., Wang, J., Jiu, L., Goettsch, W. G., van der Kallen, C. J. H., Koster, A., Schalkwijk, C. G., de Vries, H., de Vries, N. K., Eussen, S. J. P. M., Driessen, J. H. M. & Stehouwer, C. D. A., Apr 2024, In: Diabetologia. 67, 4, p. 690-702 13 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Evaluating progress towards implementation of the European HTA Regulation: Insights generated from the European Access Academy's multi-stakeholder survey

Brinkhuis, F., Ruof, J., Ham, H. V. D., Gianfrate, F., Strammiello, V., Berntgen, M., Pavlovic, M., Mol, P. G. M., Wasem, J., Dyck, W. V., Cardone, A., Dierks, C., Schiel, A., Solà-Morales, O., Goettsch, W. & Julian, E., Nov 2024, In: Health Policy and Technology. 13, 5, 100930.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Evaluation of alignment of the reimbursement medicines list for children in Albania with the WHO essential medicines list for children

Petro, E., Perumal-Pillay, V., Mantel-Teeuwisse, A. K., van den Ham, H. A. & Suleman, F., 2024, In: Journal of Pharmaceutical Policy and Practice. 17, 1, p. 1-18 18 p., 2290100.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Feasibility of disease terminology systems for mapping orphan conditions and therapeutic indications of designated orphan medicines in the European Union

Jadoenathmisier, K. D., Stoyanova-Beninska, V., Soons, I. M., Leufkens, H. G., Bloem, L. T. & Pasmooij, A. M., Nov 2024, In: European Journal of Pharmaceutical Sciences. 202, 106871.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Identifying Medicine Shortages With the Twitter Social Network: Retrospective Observational Study

Postma, D. J., Heijkoop, M. L. A., De Smet, P. A. G. M., Notenboom, K., Leufkens, H. G. M. & Mantel-Teeuwisse, A. K., 6 Aug 2024, In: Journal of Medical Internet Research. 26, 13 p., e51317.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Illustrating the financial consequences of outcome-based payment models from a payers perspective- the case of autologous gene therapy atidarsagene autotemcel (Libmeldy®)

Callenbach, M. H. E., Schoenmakers, D., Vreman, R. A., Vijgen, S., Timmers, L., Hollak, C. E. M., Mantel-Teeuwisse, A. K. & Goettsch, W. G., Aug 2024, In: Value in Health. 27, 8, p. 1046-1057 12 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Importance of aligning the implementation of new payment models for innovative pharmaceuticals in European countries

Kaló, Z., Niewada, M., Bereczky, T., Goettsch, W., Vreman, R. A., Xoxi, E., Trusheim, M., Callenbach, M. H. E., Nagy, L. & Simoens, S., 2024, In: Expert Review of Pharmacoeconomics and Outcomes Research. 24, 2, p. 181-187 7 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Increasing uptake through collaboration in the development of core outcome sets: Lessons learned at OMERACT 2023

Shea, B., Pardo, J. P., Grosskleg, S., Beaton, D. E., Conaghan, P., Goettsch, W., Hofstetter, C., Maxwell, L., Musaus, J., Ollendorf, D., Schultz, G., Stevens, R., Strand, V., Tugwell, P., Williamson, P., Tunis, S. & Simon, L. S., Jun 2024, In: Seminars in Arthritis and Rheumatism. 66, 6 p., 152438.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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International regulatory and publicly-funded initiatives to advance drug repurposing

Spin, E. L., Mantel-Teeuwisse, A. K. & Pasmooij, A. M. G., 31 May 2024, In: Frontiers in Medicine. 11, 11 p., 1387517.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Monitoring Access to Child Medicines: Introducing a Standardized Set of Age-Appropriate Medicines

Joosse, I. R., Mantel-Teeuwisse, A. K., Suleman, F. & van den Ham, H. A., Mar 2024, In: Children. 11, 3, 266.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Navigating the path towards successful implementation of the EU HTA Regulation: key takeaways from the 2023 Spring Convention of the European Access Academy

Brinkhuis, F., Julian, E., van den Ham, H., Gianfrate, F., Strammiello, V., Berntgen, M., Pavlovic, M., Mol, P., Wasem, J., Van Dyck, W., Cardone, A., Dierks, C., Schiel, A., Bernardini, R., Solà-Morales, O., Ruof, J. & Goettsch, W., 2 Jul 2024, In: Health Research Policy and Systems. 22, 1, 12 p., 74.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Opportunities and challenges for decentralized clinical trial approaches: European health technology assessment perspective

Trials@Home Consortium, Mar 2024, In: Value in Health. 27, 3, p. 294-300 7 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Outer packaging labelling of medicines in Southern African Development Community (SADC) countries: comparative analysis of requirements and transition terms for harmonisation

Narsai, K., Masekela, F. B., Leufkens, H. G. M. & Mantel-Teeuwisse, A. K., 20 Jan 2024, In: BMC Health Services Research. 24, 1, 7 p., 111.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Patient-reported outcomes of adverse events after COVID-19 vaccination in Nigeria: A mixed methods study

Ogar, C. K., Gilbert, H. N., Bloem, L. T., Leopold, C., Bassi, P. U., Katagum, Y. M., Osakwe, A. I., Opadeyi, A. O., Oreagba, I., Mbo, D. N. D., Mantel-Teeuwisse, A. K. & De Bruin, M. L., 3 Oct 2024, In: Vaccine. 42, 23, 11 p., 126196.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Roadmap to Innovation of HTA Methods (IHTAM): insights from three case studies of quantitative methods

Jiu, L., Wang, J., Versteeg, J.-W., Zhang, Y., Liu, L., Somolinos-Simón, F. J., Tapia-Galisteo, J., García-Sáez, G., Hogervorst, M. A., Li, X., Mantel-Teeuwisse, A. K. & Goettsch, W. G., 5 Nov 2024, In: International Journal of Technology Assessment in Health Care. 40, 1, p. 1-10 10 p., e49.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Successes and pitfalls in orphan drug development for sickle cell disease

Costa, E., Isgrò, A., de Montalembert, M., Leufkens, H. G. M., Ware, R. E. & De Franceschi, L., 28 May 2024, In: Blood advances. 8, 10, p. 2455-2465 11 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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The caregiver’s experience of childhood cancer treatment in South Africa

Joosse, I. R., van den Ham, H. A., Mantel-Teeuwisse, A. K. & Suleman, F., 29 Feb 2024, In: Journal of Pharmaceutical Policy and Practice. 17, 1, 19 p., 2312382.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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The impact of scheduling ketamine as an internationally controlled substance on anaesthesia care in Sub-Saharan Africa: a case study and key informant interviews

Ooms, G. I., Usman, M. A., Reed, T., van den Ham, H. A. & Mantel-Teeuwisse, A. K., 7 May 2024, In: BMC Health Services Research. 24, 1, 10 p., 598.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Tools for assessing quality of studies investigating health interventions using real-world data: a literature review and content analysis

Jiu, L., Hartog, M., Wang, J., Vreman, R. A., Klungel, O. H., Mantel-Teeuwisse, A. K. & Goettsch, W. G., 13 Feb 2024, In: BMJ Open. 14, 2, 15 p., e075173.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Trends in orphan medicinal products approvals in the European Union between 2010–2022

Bouwman, L., Sepodes, B., Leufkens, H. & Torre, C., 27 Feb 2024, In: Orphanet Journal of Rare Diseases. 19, 1, 16 p., 91.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Uncertainty In Long-term Relative Effectiveness Of Medicines In Health Technology Assessment: Comparing Assessments Of Medicines For Spinal Muscular Atrophy, Cystic Fibrosis, And Hypercholesterolemia

Versteeg, J.-W., Vreman, R., Mantel-Teeuwisse, A. & Goettsch, W., Oct 2024, In: Value in Health. 27, 10, p. 1358-1366 9 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Uptake of orphan drugs in the WHO essential medicines lists

Costa, E., Moja, L., Wirtz, V. J., van den Ham, H. A., Huttner, B., Magrini, N. & Leufkens, H. G., 1 Jan 2024, In: Bulletin of the World Health Organization. 102, 1, p. 22-31 10 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Use of antibiotics in the early COVID-19 pandemic in Poland, the Netherlands and Spain, from erraticism to (more) logic

Opalska, A., Gardarsdottir, H., Kwa, M., Wojkowska-Mach, J., Sabate, M., Ballarin, M. E., de Groot, M. & Leufkens, H., 2024, In: European Journal of Clinical Pharmacology. 80, 10, p. 1581–1589 9 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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2023

A critical review of methodologies used in pharmaceutical pricing policy analyses

Joosse, I. R., Tordrup, D., Bero, L., Mantel-Teeuwisse, A. K. & van den Ham, H. A., Aug 2023, In: Health Policy. 134, 104576.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Adverse Events to SARS-CoV-2 (COVID-19) Vaccines and Policy Considerations that Inform the Funding of Safety Surveillance in Low- and Middle-Income Countries: A Mixed Methods Study

Ogar, C. K., Quick, J., Gilbert, H. N., Vreman, R. A., Mantel-Teeuwisse, A. K. & Mugunga, J. C., Apr 2023, In: Drug Safety. 46, 4, p. 357-370 14 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Alignment in the registration, selection, procurement and reimbursement of essential medicines for childhood cancers in South Africa

Joosse, I. R., Van Den Ham, H. A., Mantel-Teeuwisse, A. K. & Suleman, F., 20 Sept 2023, In: BMJ Global Health. 8, 9, e012309.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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A systematic review of policies regulating or removing mark-ups in the pharmaceutical supply and distribution chain

Joosse, I. R., Tordrup, D., Glanville, J., Mantel-Teeuwisse, A. K. & van den Ham, H. A., Dec 2023, In: Health Policy. 138, p. 1-9 9 p., 104919.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Duration of Effectiveness Evaluation of Additional Risk Minimisation Measures for Centrally Authorised Medicinal Products in the EU Between 2012 and 2021

Essink, S. C. M., Zomerdijk, I. M., Straus, S. M. J. M., Gardarsdottir, H. & De Bruin, M. L., Oct 2023, In: Drug Safety. 46, 10, p. 1007-1020 14 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Guidance on using real-world evidence from Western Europe in Central and Eastern European health policy decision making

Németh, B., Kamusheva, M., Mitkova, Z., Petykó, Z. I., Zemplényi, A., Dimitrova, M., Tachkov, K., Balkányi, L., Czech, M., Dawoud, D., Goettsch, W., Hren, R., Knies, S., Lorenzovici, L., Maravic, Z., Piniazhko, O., Zerovnik, S. & Kaló, Z., Apr 2023, In: Journal of Comparative Effectiveness Research. 12, 4, 10 p., e220157.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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HARmonized Protocol Template to Enhance Reproducibility of hypothesis evaluating real-world evidence studies on treatment effects: A good practices report of a joint ISPE/ISPOR task force

Wang, S. V., Pottegård, A., Crown, W., Arlett, P., Ashcroft, D. M., Benchimol, E. I., Berger, M. L., Crane, G., Goettsch, W., Hua, W., Kabadi, S., Kern, D. M., Kurz, X., Langan, S., Nonaka, T., Orsini, L., Perez-Gutthann, S., Pinheiro, S., Pratt, N. & Schneeweiss, S. & 2 others, Toussi, M. & Williams, R. J., Jan 2023, In: Pharmacoepidemiology and Drug Safety. 32, 1, p. 44-55 12 p.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Health technology assessment in Türkiye: Current status and perspectives on future implementation

Atikeler, E. K., Fasseeh, A. N., Mantel-Teeuwisse, A. K., Caliskan, Z., Oner, Z. G., Kizilay, H., Kalo, Z. & Goettsch, W., Mar 2023, In: Health Policy and Technology. 12, 1, 7 p., 100701.

Research output: Contribution to journal › Article › Academic › peer-review

Open Access

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Health technology assessment of tests for SARS-CoV-2 and treatments for COVID-19: A proposed approach and best-practice recommendations

COVID-19 HTA Best-Practice Development Group, 24 Apr 2023, In: International Journal of Technology Assessment in Health Care. 39, 1, 6 p., e24.

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