Selection of key publications
- Brinkhuis, F., Goettsch, W. G., Mantel - Teeuwisse, A., & Bloem, L. T. (2024). Added benefit and revenues of oncology drugs approved by the European Medicines Agency between 1995 and 2020: retrospective cohort study. The BMJ, 384, e077391. https://doi.org/10.1136/bmj-2023-077391
- Brinkhuis, F., Goettsch, W. G., Mantel - Teeuwisse, A., & Bloem, L. T. (2024). High cost oncology drugs without proof of added benefit are burdening health systems. The BMJ, 384, q511. https://doi.org/10.1136/bmj.q511
- Gort, J., van Hattem, C. C., Bloem, L. T., & ten Ham, R. M. T. (2023). Geneesmiddelen voor Geavanceerde Therapie (ATMP’s) in Nederland: Veldverkenning, Knelpuntanalyse en Activiteitenkaart. Utrecht University. https://doi.org/10.33540/1874/433501
Note: Report in Dutch; translated title: ‘Advanced Therapy Medicinal Products (ATMPs) in the Netherlands: Field Map, Bottleneck Analysis, and Activity Map’
Book chapters
- Bloem, L.T., Van der Elst, M.E., Klungel, O.H. Principles of Pharmacovigilance and Drug Regulation. In: Jose, J., Cox, A.R., Paudyal, V., editors. Principles and Practice of Pharmacovigilance and Drug Safety. 1st ed. Cham: Springer; 2024. p. 135-161. https://doi.org/10.1007/978-3-031-51089-2_6
Publications
2024
Scholarly publications
Hogervorst, M. A., Soman, K. V., Gardarsdottir, H., Goettsch, W. G., & Bloem, L. T. (2024). Analytical Methods for Comparing Uncontrolled Trials with External Controls from Real-World Data: a Systematic Literature Review and Comparison to European Regulatory and Health Technology Assessment Practice. Value in Health. Advance online publication. https://doi.org/10.1016/j.jval.2024.08.002
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. (2024). Patient-reported outcomes of adverse events after COVID-19 vaccination in Nigeria: A mixed methods study. Vaccine, 42(23), Article 126196. Advance online publication. https://doi.org/10.1016/j.vaccine.2024.126196
Bloem, L. T., Van der Elst, M. E., & Klungel, O. H. (2024). Principles of Pharmacovigilance and Drug Regulation. In J. Jose, A. R. Cox, & V. Paudyal (Eds.), Principles and Practice of Pharmacovigilance and Drug Safety (pp. 135-161). Springer. https://doi.org/10.1007/978-3-031-51089-2_6
Jadoenathmisier, K. D., Stoyanova-Beninska, V., Soons, I. M., Leufkens, H. G., Bloem, L. T., & Pasmooij, A. M. (2024). Feasibility of disease terminology systems for mapping orphan conditions and therapeutic indications of designated orphan medicines in the European Union. European Journal of Pharmaceutical Sciences, Article 106871. Advance online publication. https://doi.org/10.1016/j.ejps.2024.106871
Sadreghaemy, M., Gamba, M. A., Bloem, L. T., & Egberts, T. C. G. (2024). Leftover of Amoxicillin Suspension After Use by Children in the Netherlands. Pharmacoepidemiology and Drug Safety, 33(8), Article e5868. https://doi.org/10.1002/pds.5868
van de Voort, T., Moors, E., Hoekman, J., & Bloem, L. T. (2024). Understanding the transformative nature of initiatives challenging the traditional pharmaceutical model in the gene and cell therapy field. Abstract from EASST-4S 2024 Conference, Amsterdam, Netherlands.
Maksimova, M. V., van Thiel, G. J. M. W., Tromp, Y., Lechner, R., van Delden, J. J. M., & Bloem, L. T. (2024). Balancing ethical norms and duties for the introduction of new medicines through conditional marketing authorization: a research agenda. Frontiers in Medicine, 11, Article 1408553. https://doi.org/10.3389/fmed.2024.1408553
Hogervorst, M. A., van Hattem, C. C., Sonke, G. S., Mantel-Teeuwisse, A. K., Goettsch, W. G., & Bloem, L. T. (2024). Healthcare decision-making for tumour-agnostic therapies in Europe: lessons learned. Drug Discovery Today, 29(7), Article 104031. https://doi.org/10.1016/j.drudis.2024.104031
Madrid Paredes, J., Versteeg, J.-W., Vreman, R. A., & Bloem, L. T. (2024). Agreement about Availability of Alternative Treatments for Innovative Drugs Assessed by the EMA and HTA Organizations. Clinical Pharmacology and Therapeutics, 116(1), 136-146. https://doi.org/10.1002/cpt.3252
Brinkhuis, F., Goettsch, W. G., Mantel - Teeuwisse, A., & Bloem, L. T. (2024). High cost oncology drugs without proof of added benefit are burdening health systems. The BMJ, 384, Article q511. https://doi.org/10.1136/bmj.q511
https://dspace.library.uu.nl/bitstream/handle/1874/437614/bmj.q511.full.pdf?sequence=1
https://dspace.library.uu.nl/bitstream/handle/1874/437614/bmj.q511.full.pdf?sequence=1
Brinkhuis, F., Goettsch, W. G., Mantel - Teeuwisse, A., & Bloem, L. T. (2024). Added benefit and revenues of oncology drugs approved by the European Medicines Agency between 1995 and 2020: retrospective cohort study. The BMJ, 384, Article e077391. https://doi.org/10.1136/bmj-2023-077391
Verweij, S., Haverhoek, V., Bergman, E., Westman, G., & Bloem, L. T. (2024). A Natural Language Processing Approach towards Harmonized Communication of Uncertainties Identified during the European Medicine Authorization Process. Clinical Pharmacology and Therapeutics, 115(4), 871-880. https://doi.org/10.1002/cpt.3195
Verschueren, M. V., Peters, B. J., Bloem, L. T., Kruik, V. R., Uitvlugt, E. B., Bijsmans, A. R., Egberts, A. C., & van de Garde, E. M. (2024). Pembrolizumab Plus Chemotherapy Per PD-L1 Stratum In Patients With Metastatic Non-Small Cell Lung Cancer: Real-World Effectiveness Versus Trial Efficacy. Clinical Lung Cancer, 25(2), 119-127.e1. https://doi.org/10.1016/j.cllc.2023.12.011
2023
Scholarly publications
van den Berg, R. B., Laarman, A. R. C., Bloem, L. T., Dijkstra, J. A., Veldkamp, A. I., Allegaert, K., Swart, E. L., & van Weissenbruch, M. M. (2023). Neonatal pain score after use of paracetamol: Is there a relationship with serum trough concentration at steady state in preterm and term neonates? International Journal of Clinical Pharmacology and Therapeutics, 61(8), 354 - 362. https://doi.org/10.5414/CP204431
Verschueren, M. V., Dijs, T., Gulikers, J. L., Veelen, A. V., Croes, S., Hendriks, L. E., Smit, A. A., Bloem, L. T., Egberts, A. C., van de Garde, E. M., & Peters, B. J. (2023). Durvalumab after chemoradiotherapy in patients with stage III non-small-cell lung cancer: real-world outcomes versus clinical trial results. Immunotherapy, 15(11), 839-851. https://doi.org/10.2217/imt-2023-0002
Bloem, L. T., Schelhaas, J., López-Anglada, L., Herberts, C., van Hennik, P. B., & Tenhunen, O. (2023). European Conditional Marketing Authorization in a Rapidly Evolving Treatment Landscape: A Comprehensive Study of Anticancer Medicinal Products in 2006-2020. Clinical Pharmacology and Therapeutics, 114(1), 148-160. https://doi.org/10.1002/cpt.2906
Professional publications
Gort, J., van Hattem, C. C., Bloem, L. T., & ten Ham, R. M. T. (2023). Geneesmiddelen voor Geavanceerde Therapie (ATMP’s) in Nederland: Veldverkenning, Knelpuntanalyse en Activiteitenkaart. Utrecht University. https://doi.org/10.33540/1874/433501
European Commission, Directorate-General for Health and Food Safety, Mantel - Teeuwisse, A., Bloem, L. T., Boon, W., De Bruin, M. L., Hoekman, J., Klein, K., Stolk, P., & Vreman, R. (2023). Study in support of the evaluation and impact assessment of the EU general pharmaceuticals legislation: Analytical Report. Publications Office of the European Union. https://doi.org/10.2875/780874
European Commission, Directorate-General for Health and Food Safety, Mantel - Teeuwisse, A., Bloem, L. T., Boon, W., De Bruin, M. L., Hoekman, J., Klein, K., Stolk, P., & Vreman, R. (2023). Study in support of the evaluation and impact assessment of the EU general pharmaceuticals legislation: Evaluation Report. Publications Office of the European Union. https://doi.org/10.2875/62709
Other output
Hoogendoorn, M., Fabriek, B., & Bloem, L. T. (2023). Regulatory and health technology assessment of cell and gene therapies in Europe: Convergence or divergence? Pharmacoepidemiology and Drug Safety, 32, 16-16.
Eskola, S. M., Jokinen, S., Bloem, L. T., Brouwer, E., Brown, D. A., Camarero, J., De Bruin, M. L. L., Gardarsdottir, H., & Hakkarainen, K. M. (2023). Systematic review of externally controlled (EC) trials, data used in them, and their role in regulatory decision-making. Pharmacoepidemiology and Drug Safety, 32(supplement 1), 260-261. Article 260.
van Hattem, C. C., de Jong, A., de Groot, J. S., & Bloem, L. T. (2023). Feasibility of post-authorization randomized controlled trials for conditionally authorized anticancer medicines-A multistakeholder perspective. Pharmacoepidemiology and Drug Safety, 32, 623-623.
Verschueren, M., Kruik, V., Peters, B., Bloem, L., Bijsmans, A. R., Egberts, T., & van de Garde, E. (2023). Real-world versus clinical trial outcomes of pembrolizumab plus chemotherapy in patients with stage IV non-squamous non-small cell lung cancer. Journal of Thoracic Oncology, 18(4), S72-S72.
2022
Scholarly publications
Ebbelaar, C. F., Schrader, A. M. R., van Dijk, M., Meijers, R. W. J., de Leng, W. W. J., Bloem, L. T., Jansen, A. M. L., Blokx, W. A. M., & on behalf of the MOLecular Evaluation of Melanocytic Ambiguous Tumors (MOLEMAT) investigators (2022). Towards diagnostic criteria for malignant deep penetrating melanocytic tumors using single nucleotide polymorphism array and next-generation sequencing. Modern Pathology, 35(8), 1110-1120. https://doi.org/10.1038/s41379-022-01026-6
Bloem, L. T., Bot, R. E., Mantel-Teeuwisse, A. K., van der Elst, M. E., Sonke, G. S., Klungel, O. H., Leufkens, H. G. M., & Hoekman, J. (2022). Pre-approval and post-approval availability of evidence and clinical benefit of conditionally approved cancer drugs in Europe: a comparison with standard approved cancer drugs. British Journal of Clinical Pharmacology, 88(5), 2169-2179. https://doi.org/10.1111/bcp.15141
Vreman, R. A., Bloem, L. T., van Oirschot, S., Hoekman, J., van der Elst, M. E., Leufkens, H. G., Klungel, O. H., Goettsch, W. G., & Mantel-Teeuwisse, A. K. (2022). The Role of Regulator-Imposed Post-Approval Studies in Health Technology Assessments for Conditionally Approved Drugs. International Journal of Health Policy and Management, 11(5), 642-650. https://doi.org/10.34172/ijhpm.2020.198
Other output
Hogervorst, M., Goettsch, W., Mantel-Teeuwisse, A. K., Bloem, L., & Vreman, R. A. (2022). Uncertainties Around Tumour Agnostic Therapies in Europe: Barriers and Facilitators of Patient Access. Value in Health, 25(12), S266-S266.
2021
Scholarly publications
Ebbelaar, C. F., Jansen, A. M. L., Bloem, L. T., & Blokx, W. A. M. (2021). Genome-wide copy number variations as molecular diagnostic tool for cutaneous intermediate melanocytic lesions: a systematic review and individual patient data meta-analysis. Virchows Archiv, 479(4), 773-783. https://doi.org/10.1007/s00428-021-03095-5
Bloem, L. T. (2021). Evidence generation on benefits and risks of medicines and its impact on regulatory and downstream decision-making. [Doctoral thesis 1 (Research UU / Graduation UU), Universiteit Utrecht]. Utrecht University. https://doi.org/10.33540/888
Bloem, L. T., Karomi, M., Hoekman, J., van der Elst, M. E., Leufkens, H. G. M., Klungel, O. H., & Mantel-Teeuwisse, A. K. (2021). Comprehensive evaluation of post-approval regulatory actions during the drug lifecycle–a focus on benefits and risks. Expert Opinion on Drug Safety, 20(11), 1433-1442. https://doi.org/10.1080/14740338.2021.1952981
Bloem, L. T., Vreman, R. A., Peeters, N. W. L., Hoekman, J., van der Elst, M. E., Leufkens, H. G. M., Klungel, O. H., Goettsch, W. G., & Mantel-Teeuwisse, A. K. (2021). Associations between uncertainties identified by the European Medicines Agency and national decision making on reimbursement by HTA agencies. Clinical and Translational Science, 14(4), 1566-1577. https://doi.org/10.1111/cts.13027
2019
Scholarly publications
Vreman, R. A., Bouvy, J. C., Bloem, L. T., Hövels, A. M., Mantel-Teeuwisse, A. K., Leufkens, H. G. M., & Goettsch, W. G. (2019). Weighing of Evidence by Health Technology Assessment Bodies: Retrospective Study of Reimbursement Recommendations for Conditionally Approved Drugs. Clinical Pharmacology and Therapeutics, 105(3), 684-691. https://doi.org/10.1002/cpt.1251
Bloem, L. T., Mantel-Teeuwisse, A. K., Leufkens, H. G. M., De Bruin, M. L., Klungel, O. H., & Hoekman, J. (2019). Postauthorization Changes to Specific Obligations of Conditionally Authorized Medicines in the European Union: A Retrospective Cohort Study. Clinical Pharmacology and Therapeutics, 105(2), 426-435. https://doi.org/10.1002/cpt.1169
2017
Other output
Bloem, L. T., Mantel-Teeuwisse, A. K., Leufkens, H. G. M., De Bruin, M. L., Klungel, O. H., & Hoekman, J. (2017). Risk factors associated with post-marketing changes in specific obligations of conditionally authorised products in the EU. Pharmacoepidemiology and Drug Safety, 26, 605. https://doi.org/10.1002/pds.4275
2016
Scholarly publications
Bloem, L. T., De Abreu Lourenco, R., Chin, M., Ly, B., & Haas, M. (2016). Factors impacting treatment choice in the first-line treatment of colorectal cancer. Oncology and Therapy, 4(1), 103–116. https://doi.org/10.1007/s40487-016-0020-4