Strategies for managing paediatric patients in immunoglobulin clinical trials

Abstract

The continued demand for immunoglobulin treatment for multiple indications has placed considerable strain on the supply of immunoglobulin product. Reliance on a few manufacturers can significantly impact the availability of product. In addition, patient tolerability may vary from one product to another necessitating a choice of products to find the best treatment for an individual patient. Therefore, it is important to conduct clinical trials with new immunoglobulin products to ensure that there is adequate supply and choice of products available on the market. This is particularly important for immunodeficient patients who require treatment with immunoglobulins for life. A requirement for licensing by the Federal Food and Drug Administration and Health Canada is that every immunoglobulin licensing study includes some paediatric patients. Enrolling paediatric subjects in immunoglobulin clinical trials can be challenging due to the need for both consent and assent for enrolment, as well as the increased demands that the study protocol places on the child and family over their usual clinical care. Therefore, it is necessary to utilize strategies that make the demands of the protocol more tolerable for children, and to ensure that the study documentation reflects the unique needs of paediatric patients (Denhoff et al. 2015). Statement of novelty: This paper discusses strategies to facilitate enrolment and adherence to immunoglobulin study protocols that are unique to paediatric patients.