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Off-label drug use in paediatric haemato-oncology patients: financial implications and proposed solutions for Belgian
T Bauters1, D Heenen2, K Norga3,4
1Tiene Bauters, Pediatric Haematology, Oncology and Stem Cell Transplantation, Ghent University Hospital, C. Heymanslaan 10, 9000, Ghent, Belgium. tiene.bauters@uzgent.be.
Insights
Off-label drug use in pediatric hematology-oncology often lacks reimbursement, creating financial and administrative burdens. This study estimates the impact in Belgium and proposes solutions for systematic reimbursement of essential off-label medicines.
Area of Science:
- Pediatric Oncology
- Pharmacoeconomics
- Healthcare Policy
Background:
- Pediatric cancer treatment relies on effective drugs, often used off-label in children.
- Current reimbursement systems in Belgium primarily cover authorized indications, excluding many standard-of-care pediatric oncology drugs.
- Off-label drug use, despite being standard of care, leads to funding challenges and administrative burdens.
Purpose of the Study:
- To estimate the impact of non-reimbursed drugs in Belgian pediatric hematology-oncology.
- To propose structural solutions for systematic reimbursement of off-label standard-of-care medicines for children with cancer.
- To alleviate financial and emotional distress for families and reduce administrative burdens for healthcare providers.
Main Methods:
- Analysis of drug utilization in pediatric hematology-oncology in Belgium.
- Estimation of the financial impact of non-reimbursed off-label drugs.
- Development of policy recommendations for reimbursement structures.
Main Results:
- Significant number of drugs used in pediatric hematology-oncology are off-label and non-reimbursed in Belgium.
- The study quantifies the economic impact of this reimbursement gap.
- Proposed solutions aim to streamline access to essential medicines.
Conclusions:
- Structural solutions are needed to ensure reimbursement for standard-of-care off-label drugs in pediatric oncology.
- Health authorities should consider policy changes to address this critical issue.
- Systematic reimbursement would improve access to treatment and reduce patient/provider burden.
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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