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
Highlighted 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, Article e077391. [DOI] [Repository]
de Haart, K., Asao, K., Ataher, Q., Geier, J., Hillen, J., Huang, K., Mol, P. G. M., Rivera, D., Wang, H., Yang, H., Zhan, S., Hoogendoorn, W. E., & Bloem, L. T. (2025). Long-term follow-up after authorization of gene therapy: leveraging real-world data. Drug Discovery Today, 30(5), Article 104337. [DOI] [Portal]
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. [DOI] [Repository]
Publications
2025
Scholarly publications
Zafiropoulos, N., Pignatti, F., Kouroumalis, A., Guizzaro, L., Karres, D., Demolis, P., Tenhunen, O., Janssens, R., Taams, A., Bloem, L. T., Koenig, F., & Posch, M. (2025). A model structure for describing uncertainties in benefit-risk assessment of oncology drug applications. Frontiers in Medicine, 12, 1589578. [DOI]
Verschueren, M. V., Verschueren, D. V., van de Garde, E. M. W., & Bloem, L. T. (2025). A Bayesian Approach to Compare Accumulating Survival Data From Clinical Practice With RCT Data: A Case Study in Non-Small Cell Lung Cancer Patients. CPT: Pharmacometrics and Systems Pharmacology. Advance online publication. [DOI]
de Haart, K., Asao, K., Ataher, Q., Geier, J., Hillen, J., Huang, K., Mol, P. G. M., Rivera, D., Wang, H., Yang, H., Zhan, S., Hoogendoorn, W. E., & Bloem, L. T. (2025). Long-term follow-up after authorization of gene therapy: leveraging real-world data. Drug Discovery Today, 30(5), Article 104337. [DOI] [Portal]
MOLecular Evaluation of Melanocytic Ambiguous Tumours (MOLEMAT) Investigators, Ebbelaar, C. F., Jansen, A. M. L., Speet, L. C. M., Schutgens, F., Zoetemeyer, S., Cleton-Jansen, A.-M., van Dijk, M. R., Breimer, G. E., Bloem, L. T., de Leng, W. W. J., van Doorn, R., Suijkerbuijk, K. P. M., Schrader, A. M. R., & Blokx, W. A. M. (2025). Clinical outcomes and genomic profiles of MAP2K1-mutated primary cutaneous melanocytic tumours. EBioMedicine, 114, Article 105643. [DOI] [Portal]
Verschueren, M. V., Tassopoulou, V. P., Visscher, R., Schuurkamp, J., Peters, B. J. M., Koopman, M., van de Garde, E. M. W., Egberts, A. C. G., & Bloem, L. T. (2025). Definitions, measurement, and reporting of progression-free survival in randomized clinical trials and observational studies of patients with advanced non-small-cell lung cancer treated with immunotherapy: a scoping review. ESMO Real World Data and Digital Oncology, 7, Article 100118. [DOI] [Portal]
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. (2025). Stimulating development of innovative medicines in the European Union: does a new definition for unmet medical need add value? Drug Discovery Today, 30(1), Article 104251. [DOI] [Portal]
Hogervorst, M. A., Soman, K. V., Gardarsdottir, H., Goettsch, W. G., & Bloem, L. T. (2025). 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, 28(1), 161-174. [DOI] [Portal]
2024
Scholarly publications
van Hattem, C. C., de Jong, A. J., de Groot, J. S., Hoekman, J., Broekman, K. E., Sonke, G. S., van Hennik, P. B., & Bloem, L. T. (2024). Factors affecting the feasibility of post-authorisation RCTs for conditionally authorised anticancer medicines: a multistakeholder perspective from a qualitative focus group study. BMJ Open, 14(11), Article e084483. [DOI] [Portal]
Zinken, J. F., Pasmooij, A. M. G., Ederveen, A. G. H., Hoekman, J., & Bloem, L. T. (2024). Environmental risk assessment in the EU regulation of medicines for human use: an analysis of stakeholder perspectives on its current and future role. Drug Discovery Today, 29(12), Article 104213. [DOI] [Portal]
Verschueren, M. V., Abedian Kalkhoran, H., Deenen, M., van den Borne, B. E. E. M., Zwaveling, J., Visser, L. E., Bloem, L. T., Peters, B. J. M., & van de Garde, E. M. W. (2024). Development and Portability of a Text Mining Algorithm for Capturing Disease Progression in Electronic Health Records of Patients With Stage IV Non-Small Cell Lung Cancer. JCO clinical cancer informatics, 8, Article e2400053. [DOI] [Portal]
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. [DOI] [Portal]
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, 202, Article 106871. [DOI] [Portal]
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. [DOI] [Portal]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
Professional publications
Hoek, J. M., Warmerdam, D. O., Berends, S. E., Vingerhoed-van Aken, B. E., Driessens, M. H. E., Fabriek, B. O., Hernandez, J. F., Meij, P., van Oers, C. C. M., Stolk, P., ten Ham, R., & Bloem, L. T. (2024). Shaping the potential of a Dutch ATMP network: Report of the RSNN SIG Advanced Therapies and FAST workshop on 11 June 2024. Utrecht University. [DOI] [Portal]
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. [DOI] [Portal]
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. [DOI] [Repository]
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. [DOI] [Repository]
Professional publications
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
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. [DOI] [Repository]
2017
Other output
Shakir, S., Bloem, L., Bruin, M. L. D., Hoekman, J., Leufkens, H., & Ryan, P. (2017). Practical Points to Address Uncertainties in Risk Management Planning.
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. [DOI]
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. [DOI] [Repository]