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Off-label drug use in paediatric haemato-oncology patients: financial implications and proposed solutions for Belgian patients

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Abstract

Treatment of children with cancer requires access to and reimbursement of effective drugs. Children with haemato-oncological diseases are often treated according to established treatment recommendations or in the framework of late-phase clinical trials. These often involve the use of drugs authorised for adults but which, however, have been used for many years in paediatrics with no perspective of authorisation in children. In Belgium, medicines are predominantly reimbursed based on their authorised indication. As a consequence, many drugs used in paediatric haemato-oncology are used off-label, despite their status of ‘standard of care’. As reimbursement is often not available, alternative ways for funding need to be explored, which causes a significant administrative burden for healthcare providers and emotional distress for the parents. Solutions to organise a systematic reimbursement of standard of care off-label used drugs are described.

Conclusion: A number of structural solutions are proposed, and we hope that they might guide health authorities to provide a solution to the problem caused by the lack of reimbursement of some standard of care medicines for children with cancer.

What is Known:

• Off-label drug use is frequently observed in paediatric haemato-oncology and compromises—in some countries—reimbursement.

What is New:

• An estimation of the impact of non-reimbursed drugs in Belgium is provided.

• Some solutions are presented to overcome this problem in Belgium.

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Abbreviations

EU:

European Union

PHO:

Paediatric haemato-oncology

SmPC:

Summary of product characteristics

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Acknowledgements

The authors thank the board members of the Belgian Society of Paediatric Haematology Oncology for constructive discussions on this theme: Alina Ferster (ULB, Brussels), Nadine Francotte (CHC Espérance, Liège), Bénédicte Brichard (Cliniques St-Luc, Brussels), Caroline Piette (Hôpital de la Citadelle, Liège) and Jutte Van der Werff (UZ Brussel, Brussels.)

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Authors and Affiliations

  1. Tiene Bauters, Pediatric Haematology, Oncology and Stem Cell Transplantation, Ghent University Hospital, C. Heymanslaan 10, 9000, Ghent, Belgium

    T. Bauters & G. Laureys

  2. KickCancer, Public Interest Foundation, Brussels, Belgium

    D. Heenen

  3. Pediatric Haematology/Oncology Unit, Queen Mathilde Mother and Child Center, Antwerp University, Antwerp, Belgium

    K. Norga

  4. Hospital and Faculty of Medicine and Health Sciences, Antwerp University, Antwerp, Belgium

    K. Norga

  5. Pediatric Haematology and Oncology, Cliniques Universitaires Saint-Luc, Brussels, Belgium

    A. Van Damme

  6. Pediatric Haematology and Oncology, University Hospitals Leuven, Leuven, Belgium

    A. Uyttebroeck

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  1. T. Bauters
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  2. D. Heenen
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  3. K. Norga
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  4. A. Van Damme
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  5. A. Uyttebroeck
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  6. G. Laureys
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Contributions

Tiene Bauters: concept, writing, review and final edits; Delphine Heenen: concept, writing, review and final edits; Koen Norga: concept and review; An Van Damme: concept and review; Anne Uyttebroeck: concept and review; Geneviève Laureys: concept, writing, review and final approval.

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Correspondence to T. Bauters.

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The authors declare no competing interests.

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Bauters, T., Heenen, D., Norga, K. et al. Off-label drug use in paediatric haemato-oncology patients: financial implications and proposed solutions for Belgian patients. Eur J Pediatr 180, 3067–3071 (2021). https://doi.org/10.1007/s00431-021-04150-8

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  • DOI: https://doi.org/10.1007/s00431-021-04150-8

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