Improvement of Pediatric Drug Development: Regulatory and Practical Frameworks

Tsukamoto, Katusra K.; Carroll, Kelly A.; T, Onishi; Matsumaru, Naoki; Brasseur, Daniel; Nakamura, Hidefumi

doi:10.1016/j.clinthera.2016.01.012

Cited by 33 publications

(28 citation statements)

References 10 publications

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“…[11] In spite of the overall agreement regarding the importance of these initiatives, this work is a test of patience, and progress may seem slow. [8,12,13] It is therefore important to keep on monitoring the use of medicinal products in children both locally and globally. The current study is a part of this international work to focus on the improvement in both clinical practice and the availability of drugs for children and is the first study on OL and UL drug use in hospitalised children in Norway.…”

Section: Introductionmentioning

confidence: 99%

Off-label and unlicensed medicines to hospitalised children in Norway

Teigen

Wang

Truong

et al. 2016

Journal of Pharmacy and Pharmacology

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ObjectivesThe aim of this study was to investigate the use of off‐label (OL) and unlicensed (UL) medicines to hospitalised children in Norway, to add to the current knowledge on use of medicines in this vulnerable patient group.MethodsThe study was performed as a cross‐sectional prospective study. Medication was classified as on‐ or off‐label based on the comparison with the SmPC regarding age, indication, dosage, route of administration and handling of the product. UL products were classified as imported or pharmacy produced.Key findingsMore than 90% of children receiving medicines in our study were given OL or UL medicines. More patients received OL (83%) than UL (59%). Route of administration was the most frequently observed OL category. The vast majority of the OL prescriptions were for ‘off‐patent’ products. One‐third of products prescribed were UL.ConclusionsThe study confirms that medicines to children in hospital to a significant degree are being used outside or without authorisation, in spite of recent paediatric regulatory initiatives. More data are still needed on efficacy and safety of medicines used in children, data to be incorporated in the SmPC. In addition, suitable formulations are needed to ensure optimal dosing and adherence without risky manipulations.

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Section: Introductionmentioning

confidence: 99%

Off-label and unlicensed medicines to hospitalised children in Norway

Teigen

Wang

Truong

et al. 2016

Journal of Pharmacy and Pharmacology

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“…As a special and vulnerable group, recruitment and ethical issues make the conduction of clinical trials in children more challenging. How to further improve or innovate the technical methods of clinical trials, improve the use of efficient databases such as electronic medical record system, and strengthen the cooperation among industries, administrative departments, medical institutions, research doctors and subjects, to further promote the development of clinical trials of pediatric drugs and the application of data, has long been a problem of discussion [10] . On January 3, 2020, the State Drug Administration has issued the Notice of State Drug Administration on Guiding Principles for Issuing Real World Evidence to Support Drug R&D and Evaluation (Trial) (No.1, 2020) [11] .…”

Section: Discussionmentioning

confidence: 99%

Current Situation of Clinical Trials of Drugs for Children's Indications in China

Liu

Song

ran³

et al. 2020

Preprint

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Objective: To analyze the current situation of clinical trials of drugs with indications for children in China in order to provide references for research and development of drugs for children and in formulating relevant policies. Methods: Drug clinical trials with indications for children registered before January 9, 2020 were retrieved based on the drug clinical trial registration and information publicity platform. The data were extracted and statistically analyzed by excel 2010 and SPSS 22.0, respectively. Results: There were 256 clinical trials of drugs with indications for children, which accounted for 2.61% of the total registered trials. The overall average annual growth rate of the number of trials from 2007 to 2019 was 43.30% (P=0.0000). The host company and the lead organization were mainly located in the eastern and northern parts of China. There were 67 trials (26.17%) with children as subjects; 102 trials (39.84%) with research drugs only for children; the drug type was mainly the chemical drug, and indications mainly included infections, nervous and mental diseases and respiratory diseases. Conclusion: The prospects associated with pediatric drug development are positive, but it still needs new incentives or technical guidance to further promote.

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“…Drug development in pediatric patients is often complicated by challenges, such as ethical and legal aspects, appropriate pediatric formulations, small sample sizes, and slow recruitment into trials. 5 Prior to the passage of the Best Pharmaceuticals for Children Act (BPCA) in 2002 and the Pediatric Research Equity Act (PREA) in 2003, few pediatric drug product trials were submitted to the US Food and Drug Administration (FDA). The FDA has the authority to require pediatric studies under certain circumstances (PREA), and also to incentivize drug developers to conduct pediatric studies (BPCA).…”

Section: Articlementioning

confidence: 99%

Combined Pediatric and Adult Trials Submitted to the US Food and Drug Administration 2012–2018

Tanaudommongkon

Miyagi

Green

et al. 2020

Clin Pharma and Therapeutics

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Despite legislation incentivizing and requiring drug companies to conduct pediatric clinical trials, there still is a 9-year delay in drug approval for pediatric labeling after the initial adult drug approval. The aim of this study was to review the experience of the US Food and Drug Administration (FDA) with combined pediatric and adult trials as a means for expediting pediatric approval and labeling. Combined pediatric and adult trials submitted to the FDA from 2012 to 2018 were reviewed. Only the publicly available labels and reviews were utilized for this analysis. Combined trials were identified for 72 products, with a total of 156 combined adult and pediatric trials. The therapeutic areas with the largest number of combined trials were in pulmonology for products reviewed under the Best Pharmaceuticals for Children Act (BPCA) and/or the Pediatric Research Equity Act (PREA), and hematology reviewed under the Orphan Drug Act (ODA). All drugs that utilized combined pediatric and adult clinical trials were approved simultaneously for both the adults and that part of the pediatric population. A separate pediatric subgroup efficacy analysis was reported in 57% and 48% of products under BPCA/PREA and the ODA, respectively, with a separate safety analysis in 48% and 38% of these products. When considering both BPCA/PREA and orphan drug studies, all the combined pediatric and adult trials allowed concurrent approval and labeling for part of the pediatric population at the time of the adult approval. Medications are frequently used off-label in pediatric patients. 1 Approximately 62% to 85% of drugs prescribed for children are used off-label, 2 with higher rates for children hospitalized in neonatal and pediatric intensive care units. 3 Although off-label use does not imply improper, contraindicated, or illegal use, off-label use often lacks substantial evidence for efficacy in contrast to that required for approval. The lack of pediatric use information for approved drugs is also associated with an increased risk for adverse drug reactions. 4 Pediatric healthcare providers are then faced with the practical dilemma as to how to prescribe off-label medications for children due to a lack of reliable information.

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Improvement of Pediatric Drug Development: Regulatory and Practical Frameworks

Cited by 33 publications

References 10 publications

Off-label and unlicensed medicines to hospitalised children in Norway

Off-label and unlicensed medicines to hospitalised children in Norway

Current Situation of Clinical Trials of Drugs for Children's Indications in China

Combined Pediatric and Adult Trials Submitted to the US Food and Drug Administration 2012–2018

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