Moretti, F;
Ruiz, F;
Bonifazi, F;
Pizzo, E;
Kindblom, JM;
(2022)
Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project.
British Journal of Clinical Pharmacology
, 88
(12)
pp. 5052-5059.
10.1111/bcp.15190.
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Abstract
The medicine development process is complex and requires time and effort to ensure safety, efficacy and quality. In paediatrics, this process is even more challenging, as it involves a subgroup of the population that already faces a considerable gap in the clinical evaluation of medicines and devices compared to the adult population. Moreover, access to therapies is heavily influenced by national health technology assessment (HTA) recommendations, which often form the basis for pricing and reimbursement decisions that affect the effective availability of treatments within the national health systems. Yet performing an HTA to assess the relative effectiveness and cost-effectiveness of a new children’s treatment has several non-trivial implications, creating a critical issue for the paediatric population. In addition, the advent of innovative health technologies for children emphasises the need to empower the role of HTAs in paediatrics. This article aims at describing the most relevant elements of the drug development process in the paediatric field by focusing on the HTA assessment. Particular attention will be paid to the factors that influence market access for new paediatric medicines and patients' access to treatment. The paper will also highlight some central methodological challenges in conducting HTA in the paediatric field. Finally, the article will provide insight into how initiatives, such as conect4children, may subsequently reinforce HTA awareness in the paediatric community and strengthen collaborations through network mechanisms.
| Type: | Article |
|---|---|
| Title: | Health technology assessment of paediatric medicines: European landscape, challenges and opportunities inside the conect4children project |
| Open access status: | An open access version is available from UCL Discovery |
| DOI: | 10.1111/bcp.15190 |
| Publisher version: | https://doi.org/10.1111/bcp.15190 |
| Language: | English |
| Additional information: | © 2021 The Authors. British Journal of Clinical Pharmacology published by John Wiley & Sons Ltd on behalf of British Pharmacological Society. This is an open access article under the terms of the Creative Commons Attribution-NonCommercial-NoDerivs License, which permits use and distribution in any medium, provided the original work is properly cited, the use is non-commercial and no modifications or adaptations are made. |
| Keywords: | health technology assessment, paediatric, drug development, access to treatment |
| UCL classification: | UCL UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health UCL > Provost and Vice Provost Offices > School of Life and Medical Sciences > Faculty of Population Health Sciences > Institute of Epidemiology and Health > Applied Health Research |
| URI: | https://discovery.ucl.ac.uk/id/eprint/10142000 |
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