New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development
Childhood-onset systemic lupus erythematosus (cSLE, also known as juvenile or paediatric SLE) is a severe autoimmune disease affecting multiple organs and systems, with higher morbidity and severity compared to adult SLE (aSLE). The European Union’s Paediatric Regulation No. 1901/2006 mandates the a...
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Frontiers Media S.A.
2025-07-01
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| Series: | Frontiers in Medicine |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fmed.2025.1583140/full |
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| author | Roberto De Lisa Fernando de Andres-Trelles Fernando de Andres-Trelles Ralph Bax Sara Galluzzo Sara Galluzzo Brian Alward Brian Alward Andrew Thomson Dominik Karres Hermine I. Brunner Nicolino Ruperto Nicolino Ruperto Gunter F. Egger |
| author_facet | Roberto De Lisa Fernando de Andres-Trelles Fernando de Andres-Trelles Ralph Bax Sara Galluzzo Sara Galluzzo Brian Alward Brian Alward Andrew Thomson Dominik Karres Hermine I. Brunner Nicolino Ruperto Nicolino Ruperto Gunter F. Egger |
| author_sort | Roberto De Lisa |
| collection | DOAJ |
| description | Childhood-onset systemic lupus erythematosus (cSLE, also known as juvenile or paediatric SLE) is a severe autoimmune disease affecting multiple organs and systems, with higher morbidity and severity compared to adult SLE (aSLE). The European Union’s Paediatric Regulation No. 1901/2006 mandates the agreement of a paediatric investigation plan (PIP) for new investigational medicinal products (IMPs) to ensure reliable efficacy and safety data for paediatric indications. This study examined the experience of the Paediatric Committee (PDCO) at the European Medicines Agency (EMA) in assessing PIPs for cSLE, highlighting the challenges and potential solutions in the planning of development of novel agents in this disease and providing the academia point of view on key points of cSLE medicines development. Regulatory requirements so far have been rather consistent when a PIP is agreed, and recommend randomised controlled trials to enable a full benefit-risk assessment. However, PIPs are agreed when adult efficacy data are not yet available and as soon as the product lifecycle progresses, new methods and approaches can offer some advantages over randomised controlled trials in this setting and might provide a comparable level of evidence of efficacy in an alternative way. Extrapolation of adult efficacy data to paediatrics is one possible approach, provided that adult trials produce data that can be used for this purpose and that the degree of residual uncertainty is appropriately quantified for the medicinal product in question. The study also highlights the value of enhanced international cooperation among regulatory authorities, developers, and academic institutions in this field to support collaborative efforts among various stakeholders. |
| format | Article |
| id | doaj-art-34907a04cdcb4d0383cf0d3e12911da3 |
| institution | Kabale University |
| issn | 2296-858X |
| language | English |
| publishDate | 2025-07-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Medicine |
| spelling | doaj-art-34907a04cdcb4d0383cf0d3e12911da32025-08-20T03:28:48ZengFrontiers Media S.A.Frontiers in Medicine2296-858X2025-07-011210.3389/fmed.2025.15831401583140New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug developmentRoberto De Lisa0Fernando de Andres-Trelles1Fernando de Andres-Trelles2Ralph Bax3Sara Galluzzo4Sara Galluzzo5Brian Alward6Brian Alward7Andrew Thomson8Dominik Karres9Hermine I. Brunner10Nicolino Ruperto11Nicolino Ruperto12Gunter F. Egger13Paediatric Medicines Office, European Medicines Agency (EMA), Amsterdam, NetherlandsPaediatric Committee (PDCO), European Medicines Agency (EMA), Amsterdam, NetherlandsUniversidad Complutense de Madrid, Departamento de Farmacología y Toxicología, Madrid, SpainPaediatric Medicines Office, European Medicines Agency (EMA), Amsterdam, NetherlandsPaediatric Committee (PDCO), European Medicines Agency (EMA), Amsterdam, NetherlandsBambino Gesù Children’s Hospital, Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS), Rome, ItalyPaediatric Committee (PDCO), European Medicines Agency (EMA), Amsterdam, NetherlandsHealth Products Regulatory Authority, Dublin, IrelandMethodology Workstream, European Medicines Agency (EMA), Amsterdam, NetherlandsPaediatric Medicines Office, European Medicines Agency (EMA), Amsterdam, NetherlandsPediatric Rheumatology Collaborative Study Group Cincinnati Children’s Hospital Medical Center, University of Cincinnati, Cincinnati, OH, United StatesFondazione Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) San Gerardo dei Tintori, Pediatria/Paediatric Rheumatology International Trials Organisation (PRINTO), Monza, ItalyUniversità degli Studi di Milano-Bicocca, Dipartimento di Medicina e Chirurgia, Milan, ItalyPaediatric Medicines Office, European Medicines Agency (EMA), Amsterdam, NetherlandsChildhood-onset systemic lupus erythematosus (cSLE, also known as juvenile or paediatric SLE) is a severe autoimmune disease affecting multiple organs and systems, with higher morbidity and severity compared to adult SLE (aSLE). The European Union’s Paediatric Regulation No. 1901/2006 mandates the agreement of a paediatric investigation plan (PIP) for new investigational medicinal products (IMPs) to ensure reliable efficacy and safety data for paediatric indications. This study examined the experience of the Paediatric Committee (PDCO) at the European Medicines Agency (EMA) in assessing PIPs for cSLE, highlighting the challenges and potential solutions in the planning of development of novel agents in this disease and providing the academia point of view on key points of cSLE medicines development. Regulatory requirements so far have been rather consistent when a PIP is agreed, and recommend randomised controlled trials to enable a full benefit-risk assessment. However, PIPs are agreed when adult efficacy data are not yet available and as soon as the product lifecycle progresses, new methods and approaches can offer some advantages over randomised controlled trials in this setting and might provide a comparable level of evidence of efficacy in an alternative way. Extrapolation of adult efficacy data to paediatrics is one possible approach, provided that adult trials produce data that can be used for this purpose and that the degree of residual uncertainty is appropriately quantified for the medicinal product in question. The study also highlights the value of enhanced international cooperation among regulatory authorities, developers, and academic institutions in this field to support collaborative efforts among various stakeholders.https://www.frontiersin.org/articles/10.3389/fmed.2025.1583140/fullpaediatric systemic lupus erythematosuschildhood systemic lupus erythematosuspaediatric extrapolationpaediatric drug developmentpaediatric medicine developmentEuropean Medicines Agency |
| spellingShingle | Roberto De Lisa Fernando de Andres-Trelles Fernando de Andres-Trelles Ralph Bax Sara Galluzzo Sara Galluzzo Brian Alward Brian Alward Andrew Thomson Dominik Karres Hermine I. Brunner Nicolino Ruperto Nicolino Ruperto Gunter F. Egger New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development Frontiers in Medicine paediatric systemic lupus erythematosus childhood systemic lupus erythematosus paediatric extrapolation paediatric drug development paediatric medicine development European Medicines Agency |
| title | New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development |
| title_full | New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development |
| title_fullStr | New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development |
| title_full_unstemmed | New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development |
| title_short | New medicines for childhood-onset systemic lupus erythematosus: an EU perspective on paediatric drug development |
| title_sort | new medicines for childhood onset systemic lupus erythematosus an eu perspective on paediatric drug development |
| topic | paediatric systemic lupus erythematosus childhood systemic lupus erythematosus paediatric extrapolation paediatric drug development paediatric medicine development European Medicines Agency |
| url | https://www.frontiersin.org/articles/10.3389/fmed.2025.1583140/full |
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