The development and assessment of biological treatments for children



Smith, Eve MD, Foster, Helen E and Beresford, Michael W ORCID: 0000-0002-5400-9911
(2015) The development and assessment of biological treatments for children. BRITISH JOURNAL OF CLINICAL PHARMACOLOGY, 79 (3). pp. 379-394.

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Abstract

The development of biological agents with specific immunological targets has revolutionized the treatment of a wide variety of paediatric diseases where traditional immunosuppressive agents have been partly ineffective or intolerable. The increasing requirement for pharmaceutical companies to undertake paediatric studies has provided impetus for studies of biologics in children. The assessment of biological agents in children to date has largely relied upon randomized controlled trials using a withdrawal design, rather than a parallel study design. This approach has been largely used due to ethical concerns, including use of placebo treatments in children with active chronic disease, and justified on the basis that treatments have usually already undergone robust assessment in related adult conditions. However, this study design limits the reliability of the data and can confuse the interpretation of safety results. Careful ongoing monitoring of safety and efficacy in real-world practice through national and international biologics registries and robust reporting systems is crucial. The most commonly used biological agents in children target tumour necrosis factor-α, interleukin-1, interleukin-6 and cytotoxic lymphocyte-associated antigen-4. These agents are most frequently used in paediatric rheumatic diseases. This review discusses the development and assessment of biologics within paediatric rheumatology with reference to the lessons learned from use in other subspecialties.

Item Type: Article
Uncontrolled Keywords: adverse drug reactions, biologics, clinical trials, paediatric rheumatology
Depositing User: Symplectic Admin
Date Deposited: 28 Oct 2016 16:15
Last Modified: 19 Jan 2023 07:27
DOI: 10.1111/bcp.12406
Related URLs:
URI: https://livrepository.liverpool.ac.uk/id/eprint/3004182