https://nextgen-health.net/ Paediatric medicines news and analysis for pharma and biotech: new treatments, clinical evidence, drug safety, paediatric conditions and policy. en-gb Fri, 23 Oct 2026 11:08:20 GMT https://nextgen-health.net/articles/why-children-wait-for-medicineshttps://nextgen-health.net/articles/why-children-wait-for-medicinesThu, 22 Oct 2026 08:00:00 GMTPolicyHow the US BPCA and PREA and the EU Paediatric Regulation shape children’s medicines, and why paediatric data still arrive years after adult approval. https://nextgen-health.net/articles/the-formulation-problemhttps://nextgen-health.net/articles/the-formulation-problemTue, 20 Oct 2026 08:00:00 GMTPaediatric MedicinesWhy adult medicines often fail children: dose flexibility, taste and excipient safety, and why paediatric formulation belongs early in drug development. https://nextgen-health.net/articles/extrapolation-and-trial-designhttps://nextgen-health.net/articles/extrapolation-and-trial-designSun, 18 Oct 2026 08:00:00 GMTClinical EvidenceHow paediatric extrapolation and the ICH E11A guideline let adult data support children’s trials, shrinking efficacy studies to dosing and safety work. https://nextgen-health.net/articles/gene-therapy-paediatric-pipelinehttps://nextgen-health.net/articles/gene-therapy-paediatric-pipelineFri, 16 Oct 2026 08:00:00 GMTNew TreatmentsWhy gene therapy targets children first: early irreversible disease, newborn screening, AAV durability in growing bodies and one-time treatment costs. https://nextgen-health.net/articles/rare-disease-and-childrenhttps://nextgen-health.net/articles/rare-disease-and-childrenWed, 14 Oct 2026 08:00:00 GMTPaediatric ConditionsMany rare diseases begin in childhood. How orphan and paediatric incentives combine, and why natural history data drive rare paediatric trials. https://nextgen-health.net/articles/pharmacovigilance-small-populationshttps://nextgen-health.net/articles/pharmacovigilance-small-populationsMon, 12 Oct 2026 08:00:00 GMTDrug SafetyRare side effects seldom appear in small paediatric trials. How spontaneous reporting, registries and FDA reviews monitor children’s drug safety. https://nextgen-health.net/articles/eu-pharma-reform-childrens-medicineshttps://nextgen-health.net/articles/eu-pharma-reform-childrens-medicinesThu, 08 Oct 2026 08:00:00 GMTPolicyThe EU pharma reform folds the Paediatric Regulation into one framework. What it means for paediatric investigation plans and mechanism-of-action rules. https://nextgen-health.net/articles/why-paediatric-trials-struggle-to-recruithttps://nextgen-health.net/articles/why-paediatric-trials-struggle-to-recruitSun, 04 Oct 2026 08:00:00 GMTClinical EvidenceSmall populations, competing trials and consent make paediatric trial recruitment hard. How research networks and family-centred design are helping. https://nextgen-health.net/articles/new-paediatric-dosage-formshttps://nextgen-health.net/articles/new-paediatric-dosage-formsFri, 02 Oct 2026 08:00:00 GMTPaediatric MedicinesMini-tablets, orodispersible films and dispersible tablets are replacing liquids in children’s medicines. How they work and what regulators expect. https://nextgen-health.net/articles/car-t-began-with-childrenhttps://nextgen-health.net/articles/car-t-began-with-childrenWed, 30 Sep 2026 08:00:00 GMTNew TreatmentsThe first approved CAR-T therapy, tisagenlecleucel, treated children and young adults with leukaemia. How paediatric cases shaped cell therapy. https://nextgen-health.net/articles/codeine-lesson-paediatric-prescribinghttps://nextgen-health.net/articles/codeine-lesson-paediatric-prescribingMon, 28 Sep 2026 08:00:00 GMTDrug SafetyHow CYP2D6 ultra-rapid metabolism made codeine dangerous for some children, and why the FDA and EMA restricted its use in children from 2013. https://nextgen-health.net/articles/childhood-cancer-drug-developmenthttps://nextgen-health.net/articles/childhood-cancer-drug-developmentSat, 26 Sep 2026 08:00:00 GMTPaediatric ConditionsWhy childhood cancers get few new drugs, and how the RACE for Children Act requires paediatric studies based on a drug’s molecular target. https://nextgen-health.net/articles/uk-paediatric-investigation-plans-after-brexithttps://nextgen-health.net/articles/uk-paediatric-investigation-plans-after-brexitThu, 24 Sep 2026 08:00:00 GMTPolicySince 2021 the MHRA has run its own paediatric regime for Great Britain. How UK paediatric investigation plans work and where they can differ from the EU. https://nextgen-health.net/articles/real-world-evidence-paediatricshttps://nextgen-health.net/articles/real-world-evidence-paediatricsSun, 20 Sep 2026 08:00:00 GMTClinical EvidenceCan health records and registries fill the paediatric evidence gap? How real-world evidence supports dosing, safety and extrapolation, and its limits. https://nextgen-health.net/articles/how-paediatric-doses-are-calculatedhttps://nextgen-health.net/articles/how-paediatric-doses-are-calculatedFri, 18 Sep 2026 08:00:00 GMTPaediatric MedicinesWeight, body surface area or age? How paediatric doses are calculated, and why organ maturation in the first two years makes simple scaling unreliable. https://nextgen-health.net/articles/rsv-prevention-for-infantshttps://nextgen-health.net/articles/rsv-prevention-for-infantsWed, 16 Sep 2026 08:00:00 GMTPaediatric ConditionsNirsevimab, clesrovimab and maternal RSV vaccines are transforming infant RSV prevention. How they work and how health systems choose between them. https://nextgen-health.net/articles/three-ways-to-treat-spinal-muscular-atrophyhttps://nextgen-health.net/articles/three-ways-to-treat-spinal-muscular-atrophyMon, 14 Sep 2026 08:00:00 GMTNew TreatmentsNusinersen, onasemnogene abeparvovec and risdiplam compared: three SMA treatments, three technologies, and why starting before symptoms matters most. https://nextgen-health.net/articles/long-term-follow-up-in-childrenhttps://nextgen-health.net/articles/long-term-follow-up-in-childrenSat, 12 Sep 2026 08:00:00 GMTDrug SafetyMedicines can affect a child’s growth, development and fertility years later. Why long-term follow-up matters, including after gene therapy. https://nextgen-health.net/articles/rare-pediatric-disease-priority-review-vouchershttps://nextgen-health.net/articles/rare-pediatric-disease-priority-review-vouchersThu, 10 Sep 2026 08:00:00 GMTPolicyHow the FDA rare pediatric disease priority review voucher works, why vouchers sell for around $100m, and whether the scheme creates new medicines. https://nextgen-health.net/articles/pbpk-modelling-in-childrenhttps://nextgen-health.net/articles/pbpk-modelling-in-childrenSun, 06 Sep 2026 08:00:00 GMTClinical EvidenceHow physiologically based pharmacokinetic (PBPK) models use organ maturation data to set first doses for children, and what the FDA and EMA expect. https://nextgen-health.net/articles/excipients-in-childrens-medicineshttps://nextgen-health.net/articles/excipients-in-childrens-medicinesFri, 04 Sep 2026 08:00:00 GMTPaediatric MedicinesBenzyl alcohol, propylene glycol and ethanol can harm newborns. Why excipient safety matters in children’s medicines and how formulators design risk out. https://nextgen-health.net/articles/type-1-diabetes-in-childrenhttps://nextgen-health.net/articles/type-1-diabetes-in-childrenWed, 02 Sep 2026 08:00:00 GMTPaediatric ConditionsClosed-loop insulin systems and teplizumab, the first drug to delay type 1 diabetes, are changing childhood T1D care and reviving the case for screening. https://nextgen-health.net/articles/cftr-modulators-younger-childrenhttps://nextgen-health.net/articles/cftr-modulators-younger-childrenMon, 31 Aug 2026 08:00:00 GMTNew TreatmentsHow CFTR modulators such as elexacaftor/tezacaftor/ivacaftor changed cystic fibrosis, and the push to start treatment in children as young as two. https://nextgen-health.net/articles/medication-errors-in-paediatric-carehttps://nextgen-health.net/articles/medication-errors-in-paediatric-careSat, 29 Aug 2026 08:00:00 GMTDrug SafetyWhy children face more medication errors, from tenfold decimal mistakes to mg/mL mix-ups, and the safeguards that work in hospitals and at home. https://nextgen-health.net/articles/paying-for-paediatric-medicineshttps://nextgen-health.net/articles/paying-for-paediatric-medicinesThu, 27 Aug 2026 08:00:00 GMTPolicyWhy health technology assessment struggles with children’s medicines: small trials, hard-to-measure quality of life and lifetime benefits. https://nextgen-health.net/articles/placebo-and-ethics-in-paediatric-trialshttps://nextgen-health.net/articles/placebo-and-ethics-in-paediatric-trialsSun, 23 Aug 2026 08:00:00 GMTClinical EvidenceWhen is a placebo arm ethical in a children’s trial? ICH E11, risk limits, consent and assent, and the trial designs that reduce placebo exposure. https://nextgen-health.net/articles/childrens-medicine-shortageshttps://nextgen-health.net/articles/childrens-medicine-shortagesFri, 21 Aug 2026 08:00:00 GMTPaediatric MedicinesWhy children’s medicines such as liquid amoxicillin run short: few makers, thin margins and volatile demand, and how regulators are responding. https://nextgen-health.net/articles/glp-1-drugs-for-adolescentshttps://nextgen-health.net/articles/glp-1-drugs-for-adolescentsWed, 19 Aug 2026 08:00:00 GMTPaediatric ConditionsSemaglutide and liraglutide are approved for adolescents with obesity. The STEP TEENS results, AAP guidance and the long-term questions still open. https://nextgen-health.net/articles/gene-editing-sickle-cell-younger-patientshttps://nextgen-health.net/articles/gene-editing-sickle-cell-younger-patientsMon, 17 Aug 2026 08:00:00 GMTNew TreatmentsExa-cel, the first CRISPR medicine, treats sickle cell disease from age 12. What gene editing in younger children means for conditioning and fertility. https://nextgen-health.net/articles/how-paediatric-safety-reaches-the-labelhttps://nextgen-health.net/articles/how-paediatric-safety-reaches-the-labelSat, 15 Aug 2026 08:00:00 GMTDrug SafetyHow paediatric study results reach drug labels under PREA, BPCA and the EU Paediatric Regulation, including negative results and key SmPC sections. https://nextgen-health.net/articles/off-label-prescribing-in-childrenhttps://nextgen-health.net/articles/off-label-prescribing-in-childrenThu, 13 Aug 2026 08:00:00 GMTPolicyOff-label prescribing is routine in paediatrics, especially neonatal care. Why it happens, the risks for children and what policy can do to close the gap. https://nextgen-health.net/articles/biosimilars-and-childrenhttps://nextgen-health.net/articles/biosimilars-and-childrenTue, 11 Aug 2026 08:00:00 GMTPaediatric MedicinesHow biosimilars gain paediatric indications through extrapolation, and the practical issues for children: low-dose presentations, devices and switching. https://nextgen-health.net/articles/neonates-least-studied-patientshttps://nextgen-health.net/articles/neonates-least-studied-patientsSun, 09 Aug 2026 08:00:00 GMTClinical EvidenceMost drugs in neonatal intensive care were never tested in newborns. Why neonates are so hard to study, and what is starting to change. https://nextgen-health.net/articles/individualised-genetic-medicineshttps://nextgen-health.net/articles/individualised-genetic-medicinesFri, 07 Aug 2026 08:00:00 GMTNew TreatmentsFrom milasen to bespoke base editing, genetic medicines are being designed for single children. The science, the regulatory hurdles and who pays. https://nextgen-health.net/articles/antipsychotics-young-people-metabolic-riskhttps://nextgen-health.net/articles/antipsychotics-young-people-metabolic-riskWed, 05 Aug 2026 08:00:00 GMTDrug SafetySecond-generation antipsychotics cause more weight gain in children and adolescents than in adults. The metabolic risks, monitoring and management. https://nextgen-health.net/articles/duchenne-and-accelerated-approvalhttps://nextgen-health.net/articles/duchenne-and-accelerated-approvalMon, 03 Aug 2026 08:00:00 GMTPaediatric ConditionsDuchenne muscular dystrophy has tested FDA accelerated approval like no other disease, from eteplirsen to micro-dystrophin gene therapy.