Repurposing Medicines: an undervalued toolkit in Paediatrics

<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d7244480e141">Background: Repurposing medications is well known amongst adults, having being utilized during the COVID pandemic. The NHS repurposing medications programme current research work continues to prioritise adult repurposing work for rare diseases rather than in Paediatrics. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e143"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e144">Objective: To understand the potential opportunities and challenges to repurposing medications in children. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e146"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e147">Opportunities: Repurposing is a vital tool in decreasing the time a drug is available to patients by shortening the required trial period. It allows clinicians to potentially impact a patient’s life within the disease timeframe rather than years down the line by utilizing data already available. We have safety profiles of medications such as biologics available but do not utilize this across specialities. Paediatric oncology have repurposed medications successfully and could guide future endeavours. One area where repurposing medications would be beneficial is in IgA vasculitis, currently there is limited treatment options, however there are similarities to IgA nephropathy in adults that has multiple treatment options. Repurposing biologics has been already utilized in other inflammatory diseases, Anakinra was utilized in Paediatric inflammatory multi-system syndrome, showing repurposing can be successful. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e149"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e150">Challenges: There are challenges to repurposing any medication. However, in children there are additional layers. There is complexity around medication prescribing and administration to availability of data regarding drug safety. Within Paediatrics, medicines require adapting depending on weight, age, or body surface area adding to difficulties. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e152"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e153">Conclusion: We need to be proactive and utilise information that we do have around repurposing and drug safety profiles linking this to rare diseases in children. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e155"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e156">Repurposing medications needs to occur with the same determination in children as we do in adult diseases. We must not shy away just because there may be more barriers to cross. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e159"/>

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Published
2025-04-12
DOI
https://doi.org/10.14293/idr.25.016cm
Primary Topic
Pharmaceutical studies and practices
Type
article
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article

Repurposing Medicines: an undervalued toolkit in Paediatrics

Daniel B. Hawcutt, Conor C. Murphy, Louise Oni
Pharmaceutical studies and practices
article

Repurposing Medicines: an undervalued toolkit in Paediatrics

Daniel B. Hawcutt, Conor C. Murphy, Louise Oni
article en

Abstract

<p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" class="first" dir="auto" id="d7244480e141">Background: Repurposing medications is well known amongst adults, having being utilized during the COVID pandemic. The NHS repurposing medications programme current research work continues to prioritise adult repurposing work for rare diseases rather than in Paediatrics. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e143"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e144">Objective: To understand the potential opportunities and challenges to repurposing medications in children. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e146"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e147">Opportunities: Repurposing is a vital tool in decreasing the time a drug is available to patients by shortening the required trial period. It allows clinicians to potentially impact a patient’s life within the disease timeframe rather than years down the line by utilizing data already available. We have safety profiles of medications such as biologics available but do not utilize this across specialities. Paediatric oncology have repurposed medications successfully and could guide future endeavours. One area where repurposing medications would be beneficial is in IgA vasculitis, currently there is limited treatment options, however there are similarities to IgA nephropathy in adults that has multiple treatment options. Repurposing biologics has been already utilized in other inflammatory diseases, Anakinra was utilized in Paediatric inflammatory multi-system syndrome, showing repurposing can be successful. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e149"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e150">Challenges: There are challenges to repurposing any medication. However, in children there are additional layers. There is complexity around medication prescribing and administration to availability of data regarding drug safety. Within Paediatrics, medicines require adapting depending on weight, age, or body surface area adding to difficulties. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e152"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e153">Conclusion: We need to be proactive and utilise information that we do have around repurposing and drug safety profiles linking this to rare diseases in children. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e155"/><p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e156">Repurposing medications needs to occur with the same determination in children as we do in adult diseases. We must not shy away just because there may be more barriers to cross. <p xmlns:xsi="http://www.w3.org/2001/XMLSchema-instance" dir="auto" id="d7244480e159"/>

University of Liverpool (GB), Great Ormond Street Hospital (GB), National Institute for Health and Care Research (GB), Kidney Care UK (GB), Alder Hey Children's Hospital (GB), Roland Hill (United Kingdom) (GB), University College London (GB)
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Pharmaceutical studies and practices
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