Research In Pediatric Population Research Articles

Conducting Patient-Oriented Research in Pediatric Populations: A Narrative Review.

It has become increasingly common for researchers to partner with patients as members of the research team and collaborate to use their lived experiences to shape research priorities, interventions, dissemination, and more. The patient-oriented research (POR) model has been adopted by both adult and pediatric health researchers. This cultural change to conducting pediatric health research brings with it new methodologies, tools, challenges, and benefits. In this review, we aim to provide guidance on how to conduct POR for pediatric populations using examples from the literature. We describe considerations for engagement before the project begins, for engagement across the research cycle, and for measurement and evaluation. We aim to show that conducting POR is feasible, beneficial, and that many common challenges and barriers can be overcome with preparation and usage of specific tools.

Psychiatric Comorbidities in Pediatric Trichotillomania: AMulticenter Cohort Study.

Trichotillomania (TTM) significantly increases the risk of psychiatric comorbidities. Sparse research in pediatric populations necessitates larger studies to assess these risks. This study investigates the risk of developing psychiatric comorbidities in pediatric TTM patients. This case-control study assessed pediatric patients (< 18 years old) with TTM diagnosed between May 18, 2013, and January 1, 2024, using US-based data from the TriNetX global research network. TTM patients (ICD-10 diagnostic category F63.3) aged 18 years or younger at diagnosis and control patients (ICD-10 code Z00.129) matched for age, sex, race, and ethnicity were assessed. Propensity score matching yielded 16,590 patients in each cohort. The analysis assessed subsequent diagnoses of ADHD, conduct disorders, tic disorders, obsessive-compulsive disorder, anxiety disorders, dissociative, stress-related, and somatoform disorders, mood disorders, and suicide attempts compared to controls. TTM patients under 18 years exhibited significantly greater risks of subsequent diagnoses for ADHD (OR: 2.002; CI 1.841-2.178; p < 0.001), conduct disorders (OR: 3.668; 3.2-3.668; p < 0.0001), tic disorders (OR: 2.247; 1.826-2.765; p < 0.0001), obsessive-compulsive disorder (OR: 11.047; 8.822-13.832; p < 0.0001), anxiety disorders (OR: 3.583; 3.387-3.7; p < 0.0001), dissociative, stress-related, and somatoform disorders (OR: 6.179; 3.935-9.701; p < 0.0001), mood disorders (OR: 2.476; 2.288-2.68; p < 0.0001), and suicide attempts (OR: 1.81; 1.121-2.924; p = 0.0139) compared to controls. TTM patients had the greatest risk of psychiatric diagnosis 1 year postindex event. Pediatric TTM patients have higher psychiatric comorbidity risks, necessitating timely intervention and comprehensive management. Dermatologists can facilitate access to behavioral and pharmacological care, enhancing patient outcomes.

Cariprazine in Psychiatry: A Comprehensive Review of Efficacy, Safety, and Therapeutic Potential.

This article provides a comprehensive review of recent developments regarding a new atypical antipsychotic drug - cariprazine - considering the mechanism of action, efficacy, safety, and promising therapeutic option for various psychiatric disorders, including schizophrenia and bipolar disorder, therapy of addictions, and treatment in the pediatric population. Its distinct pharmacological profile, characterized by partial agonism at dopamine D2 and D3 receptors, as well as serotonin receptors - 5HT1A with a preference for the D3 receptor - sets it apart from other antipsychotics. The unique mechanism of action contributes to cariprazine's positive impact on negative symptoms in schizophrenia and an antidepressant effect. Its relatively low risk of adverse effects, such as sedation, metabolic issues, and hypotension, enhances its tolerability. In bipolar affective disorder, cariprazine exhibits effectiveness in managing both depressive and manic episodes. Ongoing research in pediatric populations suggests potential benefits in schizophrenia, bipolar I disorder, and autism spectrum disorder, but further research is necessary to establish safety and efficacy. Moreover, cariprazine shows promise in addiction therapy, particularly with coexisting psychiatric disorders. Continued research and clinical exploration may discover additional insights, broadening its use in diverse patient populations. This article aims to review the role of cariprazine, a dopamine D2/D3 and serotonin 5-HT1A receptor partial agonist, in the management of psychotic illnesses, including schizophrenia, bipolar disorder, addiction therapy, and pediatric treatment.

Current status and challenges of real-world research in pediatric population

近年备受关注的真实世界研究以反映研究对象实际诊疗与健康状况的多源数据为基础，极大拓展了传统临床研究的适用场景。随着真实世界研究在多个国家儿科领域陆续开展，其在指导儿科临床实践、支持儿童药物研发与监管决策等方面发挥着日益重要的作用。本文系统介绍了国内外儿科真实世界研究现状，并就我国儿科真实世界研究面临的挑战进行探讨。.

Somatic Gene Therapy Research in Pediatric Populations: Ethical Issues and Guidance for Operationalizing Early Phase Trials.

Currently, pediatric research involving investigational gene therapies (GT, used without intending to imply a therapeutic effect) targets a broad range of indications (including rare and ultra-rare diseases) that vary in severity and availability of approved disease-modifying therapies. Because of this diversity of circumstances, there is no one-size-fits-all list of ethical concerns relevant to all uses of investigational GTs in children. Here, we review the main ethical issues, specifically those surrounding the current state of knowledge about GT product-related immunogenicity, toxicity, duration, irreversibility, informed consent/assent, trial design (including the question of who 'goes first'), participant and caregiver burdens, and equity in diagnosis and access to research opportunities. Ethical issues that can be anticipated to arise in pediatric GT clinical trials, e.g., the uncertainty and risk of this research, the resultant preclusion of GT trial participants from other research, the length of follow-up monitoring, and the urgency often felt by caregivers dealing with dire, rapidly progressive conditions, should be proactively identified, addressed in accordance with existing best practices, and transparently discussed among all stakeholders.

Acute effects of maximal versus submaximal hurdle jump exercises on measures of balance, reactive strength, vertical jump performance and leg stiffness in youth volleyball players.

Background: Although previous research in pediatric populations has reported performance enhancements following long-term plyometric training, the acute effects of plyometric exercises on measures of balance, vertical jump, reactive strength, and leg stiffness remain unclear. Knowledge on the acute effects of plyometric exercises (i.e., maximal versus submaximal hurdle jumps) help to better plan and program warm-up sessions before training or competition. Objectives: To determine the acute effects of maximal vs. submaximal hurdle jump exercise protocols executed during one training session on balance, vertical jump, reactive strength, and leg stiffness in young volleyball players. Materials and methods: Thirty male youth volleyball players, aged 12-13years, performed two plyometric exercise protocols in randomized order. In a within-subject design, the protocols were conducted under maximal (MHJ; 3 sets of 6 repetitions of 30-cm hurdle jumps) and submaximal (SHJ; 3 sets of 6 repetitions of 20-cm hurdle jumps) hurdle jump conditions. Pre- and post-exercise, balance was tested in bipedal stance on stable (firm) and unstable surfaces (foam), using two variables [center of pressure surface area (CoP SA) and velocity (CoP V)]. In addition, the reactive strength index (RSI) was assessed during countermovement maximal jumping and leg stiffness during side-to-side submaximal jumping. Testing comprised maximal countermovement jumps (CMJ). Results: Significant time-by-condition interactions were found for CoP SA firm (p < .0001; d = 0.80), CoP SA foam (p < .0001; d = 0.82), CoP V firm (p < .0001; d = 0.85), and CoP V foam (p < .0001; d = 0.83). Post-hoc analyses showed significant improvements for all balance variables from pretest to posttest for MHJ but not SHJ. All power tests displayed significant time-by-group interactions for countermovement jumps (p < .05; d = 0.42), RSI (p < .0001; d = 1.58), and leg stiffness (p < .001; d = 0.78). Post-hoc analyses showed significant pre-post CMJ (p < .001, d = 1.95) and RSI (p < .001, d = 5.12) improvements for MHJ but not SHJ. SHJ showed larger pre-post improvements compared with MHJ for leg stiffness (p < .001; d = 3.09). Conclusion: While the MHJ protocol is more effective to induce acute performance improvements in balance, reactive strength index, and vertical jump performance, SHJ has a greater effect on leg stiffness. Due to the importance of postural control and muscle strength/power for overall competitive performance in volleyball, these results suggest that young volleyball players should implement dynamic plyometric protocols involving maximal and submaximal hurdle jump exercises during warm-up to improve subsequent balance performance and muscle strength/power.

A whole blood microsampling furosemide assay: development, validation and use in a pediatric pharmacokinetic study.

Background: Furosemide is a commonly used diuretic for the treatment ofedema. The pharmacokinetics of furosemide in neonates as they mature remains poorly understood. Microsampling assays facilitate research in pediatric populations. Results: We developed and validated a liquid chromatography-tandem mass spectrometry method for the quantitation of furosemide in human whole blood with volumetric absorptive microsampling (VAMS) devices (10μl). Furosemide was stable in human whole blood VAMS under the study's assay conditions. This work established stability for furosemide for 161 days when stored as dried microsamples at -78°C. Conclusion: This method is being appliedfor the quantitation of furosemide in whole blood VAMS in an ongoing prospective pediatric clinical study. Representative clinical data are reported.

The Challenges of Crizotinib Treatment in a Child With Anaplastic Large Cell Lymphoma.

Survival in cases involving childhood malignancy is reaching nearly 80% in high-income countries, yet cancer remains one of the leading disease-related causes of death in children. In adult oncology the role of targeted therapies is established, but information regarding the use of these therapies in children is limited, largely because targeted therapies were developed in the context of adult pathologies. The few pediatric reports regarding crizotinib, an anaplastic lymphoma kinase (ALK) inhibitor, seem promising. This case of an 8-year-old male with an ALK-positive anaplastic large cell lymphoma highlights the challenges of treating children with crizotinib. Our experience with crizotinib was more challenging than described in the limited pediatric reports. Not only was the tumor response poorer than described in the reports, but a substantial amount of side-effects and practical difficulties, such as the method of administration and dosing, made management challenging. Many challenges for the use of targeted therapy in pediatric care currently persist. The limited research in pediatric populations leaves uncertainty regarding efficacy and short- and long-term side effects as well as practical difficulties. Despite a clear underlying biological rationale for certain targeted therapies, their contribution toward improving the outcome of childhood cancer remains largely unclear.

Heart failure is not a determinant of central sleep apnea in the pediatric population.

Adults with heart failure (HF) have high prevalence of central sleep apnea (CSA). While this has been repeatedly investigated in adults, there is a deficiency of similar research in pediatric populations. The goal of this study was to compare prevalence of CSA in children with and without HF and correlate central apneic events with heart function. Retrospective analysis of data from children with and without HF was conducted. Eligible children were less than 18 years old with echocardiogram and polysomnogram within 6 months of each other. Children were separated into groups with and without HF based on left ventricular ejection fraction (LVEF). Defining CSA as central apnea-hypopnea index (CAHI) more than 1/hour, the cohort was also classified into children with and without CSA for comparative study. A total of 120 children (+HF: 19, -HF: 101) were included. The +HF group was younger, with higher prevalence of trisomy 21, muscular dystrophy, oromotor incoordination, and structural heart disease. The +HF group had lower apnea-hypopnea index (median: 3/hour vs. 8.6/hour) and lower central apnea index (CAI) (median: 0.2/hour vs. 0.55/hour). Prevalence of CSA was similar in both groups (p = .195). LogCAHI was inversely correlated to age (Pearson correlation coefficient: -0.245, p = .022). Children with CSA were younger and had higher prevalence of prematurity (40% vs. 5.3%). There was no significant difference in LVEF between children with and without CSA. After excluding children with prematurity, relationship between CAHI and age was no longer sustained. In contrast to adults, there is no difference in prevalence of CSA in children with and without HF. Unlike in adults, LVEF does not correlate with CAI in children. Overall, it appears that central apneic events may be more a function of age and prematurity rather than of heart function.

Fear of cancer recurrence in childhood cancer survivors: A developmental perspective from infancy to young adulthood.

Fear of cancer recurrence (FCR) is among the most prevalent and distressing concerns reported by cancer survivors. While younger age is the most consistent predictor of elevated FCR, research to date has focused almost exclusively on adult cancer survivors. This is despite the fact that children with cancer are more likely to survive compared to adults, and will become regular, lifelong users of the medical system to mitigate the effects of toxic treatment. The early experience of cancer yields unique challenges and circumstances that may impact children's capacity for and experience of FCR across the developmental trajectory. The aim of this paper is to present a developmental perspective of FCR from infancy to young adulthood. Drawing on the developmental science literature and recent theoretical advancements in FCR, we outline cognitive and social factors that may influence children's capacity for and experience of FCR across the developmental trajectory. First, distinct developmental groups that fall under the classification of "childhood cancer survivor," and that all require attention, are summarized. Using the structure of these groupings, we centrally provide a developmental perspective of FCR that offers a useful starting point for research in pediatric populations. Key research gaps, including the need for validated assessment tools to measure FCR in child and adolescent survivors, are highlighted. Consideration of the unique context of childhood is needed to guide the identification of childhood cancer survivors at risk for FCR as well as the development of effective FCR interventions for this population.

A Systematic Review of Structural and Functional Imaging Correlates of Headache or Pain after Mild Traumatic Brain Injury.

Headaches and pain-related symptoms are the most disabling somatic complaints following mild traumatic brain injury (mTBI). In this study, we reviewed the existing literature examining structural differences in brain morphology and axonal connections, as well as functional differences in brain activity and connectivity associated with pain or post-traumatic headache (PTH) following mTBI. We searched MEDLINE®, Embase, PubMed, Cumulative Index of Nursing and Allied Health Literature (CINAHL), Cochrane Central Register of Controlled Trials, and Web of Science databases for: (1) TBI OR concussion OR post-concussion syndrome; (2) pain OR headache; and (3) magnetic resonance imaging OR functional MRI OR diffusion tensor imaging. Inclusion criteria were that articles be original studies written in English about participants with mTBI or concussion diagnosis with results reported about pain and/or headache. Excluded were review articles, case studies, documentaries, and studies related to moderate to severe TBI. Quality was assessed using the Newcastle-Ottawa Scale (NOS) quality assessment tool. Nineteen out of 3439 studies satisfied the inclusion and exclusion criteria. Participants with pain-related symptoms had lower cortical thickness in frontal and parietal cortical areas and spinothalamic tract volume. Differences in axonal connectivity were displayed in the corpus callosum, spinothalamic tract, fornix-septohippocampal circuit, and periaqueductal gray. Less activation in pain-related regions during a heat-pain task-based fMRI was reported in participants with PTH. In conclusion, individuals with pain following mTBI display differences in brain structure and brain function, suggesting irregularities in the descending pain modulatory system. These findings primarily provide information on neuroimaging differences in adults; there is limited research in pediatric populations.

Conducting reproductive research during a new childhood cancer diagnosis: ethical considerations and impact on participants.

Research among adults shows benefits and low perceived burden of engaging in behavioral research. However, questions remain regarding the ethics of conducting behavioral research in pediatric populations during sensitive situations, including during a new life-threatening diagnosis or at end-of-life. We examined reactions to participating in a behavioral reproductive research study among male adolescents newly diagnosed with cancer and their parents, as a step towards optimizing fertility preservation utilization in a population where future infertility is common. Pediatric literature regarding the ethics of behavioral research was reviewed. In our pilot, forty-four participants (19 mothers, 11 fathers, 14 male adolescents newly diagnosed with cancer) from 20 families completed demographic questionnaires and a fertility preservation decision tool developed by the study team. Qualitative interviews exploring the impact of study participation were subsequently conducted. Verbatim transcripts were coded for thematic content using the constant comparison method. Literature review showed positive reactions to research participation among youth/caregivers. In our pilot study, 89% (n = 17) of mothers, 64% (n = 7) of fathers, and 71% (n = 10) of adolescents reported at least one benefit of participating. Eleven percent (n = 2) of mothers, 36% (n = 4) of fathers, and 29% (n = 4) of adolescents said they were not affected; none of the participants reported a negative effect. Consistent with prior literature, our study suggests behavioral reproductive research prior to cancer treatment can offer direct benefits to participants and society, without increasing burden. These findings will inform future interventions to improve long-term psychosocial and reproductive outcomes for youth with cancer.

Patterns of Early Mental Health Diagnosis and Medication Treatment in a Medicaid-Insured Birth Cohort

ImportanceThe increased use of psychiatric services in the US pediatric population raises concerns about the appropriate use of psychotropic medications for very young children.ObjectiveTo assess the longitudinal patterns of psychotropic medication use in association with diagnosis and duration of use in a Medicaid-insured birth cohort.Design, Setting, and ParticipantsA cohort design was applied to computerized Medicaid administrative claims data for 35 244 children born in a mid-Atlantic state in 2007 and followed up for up to 96 months through December 31, 2014. Children were included in the birth cohort if they had an enrollment record at birth or within 3 months of birth and at least 6 months of continuous enrollment from birth. The cohort represents 92.2% of 38 225 Medicaid-insured newborns in 2007.ExposuresMental health treatments from birth through age 7 years.Main Outcomes and MeasuresCumulative incidence of first psychiatric diagnosis and psychotropic medication use (monotherapy or concomitant use of psychotropic medications) from birth through age 7 years, total and by sex, and the cumulative incidence of the use of psychosocial services (age, 0-7 years) as well as the annual duration of medication use (ie, number of days of psychotropic medication use among children 3-7 years of age).ResultsOf the 35 244 children in the cohort, 17 267 were girls and 17 977 were boys. By age 8 years, 4550 children in the birth cohort (19.7% [percentage adjusted for right censoring]) had received a psychiatric diagnosis (International Classification of Diseases, Ninth Revision, Clinical Modification codes 290-319); 2624 of these diagnoses (57.7%) were behavioral (codes 312, 313, or 314). Girls were more likely than boys to receive an incident psychiatric diagnosis of adjustment disorder (355 of 1598 [22.2%] vs 427 of 2952 [14.5%]; P < .001) or anxiety disorder (114 of 1598 [7.1%] vs 120 of 2952 [4.1%]; P < .001). By age 8 years, 2196 children in the cohort (10.2% [percentage adjusted for right censoring]) had received a psychotropic medication. Among medication users, 1763 of 2196 (80.5% [percentage adjusted for right censoring]) received monotherapy, 343 of 2196 (16.4% [percentage adjusted for right censoring]) received 2 medication classes concomitantly, and 90 of 2196 (4.3% [percentage adjusted for right censoring]) received 3 or more medication classes concomitantly for 60 days or more (range, 78-180 days). The annual median number of days of psychotropic medication use among medicated children increased with age, reaching 210 of 365 days for children 7 years of age. Among children 7 years of age, the median number of days of use of an antipsychotic (193 days [interquartile range, 60-266 days]), stimulant (183 days [interquartile range, 86-295 days]), or α-agonist (199 days [interquartile range, 85-305 days]) exceeded half of the year.Conclusions and RelevanceMedicaid-insured children received substantial mental health services and had prolonged exposure to psychotropic medications in the early years of life. These findings highlight the need for outcomes research in pediatric populations.

Methodological and Ethical Issues in Pediatric Medication Safety Research.

In May 2016, the Eshelman School of Pharmacy at The University of North Carolina at Chapel Hill convened the PharmSci conference to address the topic of "methodological and ethical issues in pediatric medication safety research." A multidisciplinary group of experts representing a diverse array of perspectives, including those of the US Food and Drug Administration, children's hospitals, and academia, identified important considerations for pediatric medication safety research and opportunities to advance the field. This executive summary describes current challenges that clinicians and researchers encounter related to pediatric medication safety research and identifies innovative and ethically sound methodologies to address these challenges to improve children's health. This article addresses 5 areas: (1) pediatric drug development and drug trials; (2) conducting comparative effectiveness research in pediatric populations; (3) child and parent engagement on study teams; (4) improving communication with children and parents; and (5) assessing child-reported outcomes and adverse drug events.

Pediatric Leukemia, Psychosocial Dimensions of Cures, and Implications for HIV.

* Abbreviation: ALL — : acute lymphoblastic leukemia Although many aspects of HIV cure research in pediatric populations are entirely without precedent, historical examples demonstrate how curative interventions may transform clinical practice and perceptions of disease over time. The history of pediatric acute lymphoblastic leukemia (ALL) illustrates how the psychological and social dimensions of care became part and parcel of curative efforts. In particular, the tripartite concept of cure devised by physician and bioethicist Jan van Eys provides a framework for approaching curative research and clinical care in a pediatric population. As we slowly move toward an HIV cure, lessons learned from ALL and van Eys’s work may help us implement such a cure more effectively in a pediatric context. Pediatric ALL underwent a transformation from incurable to curable in the second half of the 20th century. Before the 1950s, it was a uniformly fatal disease. Treatments such as blood transfusions could briefly prolong life, but clinical management focused on controlling symptoms and helping patients and their families prepare for death.1 Beginning in the late 1940s, however, new chemotherapies dramatically increased survival rates and, by 1980, ∼50% of patients could expect to survive ≥5 years, creating a real possibility … Address correspondence to Raul Necochea, PhD, Dept of Social Medicine, University of North Carolina School of Medicine, Chapel Hill, NC 27599-7240. E-mail: necochea{at}med.unc.edu

Topical Review: Provider-Patient Interactions: An Important Consideration for Racial/Ethnic Health Disparities in Youth.

To summarize literature on the role of provider-patient interactions (PPI) in racial/ethnic health disparities and consider the relevance of PPI for understanding health disparities in pediatric psychology. Topical review of literature focusing on health disparities and PPI in adult and pediatric populations. For adults, evidence is clear that racial/ethnic minorities experience poorer quality PPI, which may be associated with poorer health outcomes. In pediatric populations, the emerging literature indicates similar associations, and potential promising targets for intervention including information exchange, shared decision-making and patient-centered communication. PPI research in pediatric populations poses a number of methodological challenges including culturally and developmentally sensitive measurement of triadic (caregiver, patient, provider) interactions. Health disparities in PPI exist for racial/ethnic minority youth and may contribute to poorer health outcomes. Pediatric psychologists can make valuable contributions, given their expertise and role within medical settings. Research, clinical, and policy recommendations are discussed.

Very early combination antiretroviral therapy in infants: prospects for cure.

A single case of sustained HIV control in the absence of antiretroviral therapy or HIV-specific immune responses ensued following 18 months of combination antiretroviral therapy initiated at 30 h of age in a perinatally HIV-infected child (the Mississippi child). This case provides proof-of-concept that delay in HIV viremic rebound may ensue following very early treatment (VET) in perinatal infection, likely through marked reduction of latent replication-competent HIV reservoirs. The latent HIV reservoir remains the critical barrier to remission. Several studies indicate that the earlier effective combination antiretroviral therapy is initiated, the smaller the size of the HIV reservoir. The unique ability of perinatally infected neonates to initiate VET at the time of birth maximizes the potential benefits of limiting latent reservoir size and permitting reservoir decay, likely lengthening the duration of remission and limiting the capacity for re-establishment of viremia. This article covers the rationale and feasibility of VET to achieve sustained virologic remission in perinatal infection. Recent studies highlighting the effects of VET on biomarkers of HIV persistence in perinatal HIV infection are reviewed as well as implications and challenges for cure research in pediatric populations.

The European Union Pediatric legislation: impact on pharmaceutical research in pediatric populations

The first European Paediatric Regulation came into force in the European Union on the 26 January 2007. A few years later, its impact on pharmaceutical research starts to be assessed from different perspectives. Such reflection process is quite valuable as it is anticipated that this regulation will be revised in a few years. Major achievements have occurred and have been acknowledged but there are also divergent opinions, and questions or concerns have been expressed, sometimes leading to interesting controversies. This article will review some background and key features of the European Paediatric Regulation and present, through the examples of pediatric psychopharmacology and oncology, its impact on pharmaceutical research with suggestions for future of pediatric research.

Adherence to the immunosuppressive regimen in pediatric kidney transplant recipients: A systematic review

As most prior reviews on NA focus on adult transplant patients, there is a need for a comprehensive overview on adherence to the immunosuppressive regimen in pediatric kidney transplant patients. This systematic review searched for English-language papers (1990-2008) addressing the prevalence of NA to the immunosuppressive regimen, its consequences, determinants, and interventions in pediatric kidney transplant patients (< age 21 yr). We found 36 papers, showing a prevalence of NA (weighted mean) of 31.8% with adolescents being more at risk compared to younger patients. About 44% of all graft losses and 23% of late acute rejection episodes are associated with NA. Most studies investigated socio-economic, condition-related or treatment-related determinants. Only one educational intervention has been tested but yielded inconclusive results. NA to the immunosuppressive regimen is prevalent with serious clinical consequences in pediatric kidney transplant patients, but the economic consequences have not yet been explored. More studies on determinants of NA are needed. The literature currently lacks fully powered RCTs testing adherence-enhancing interventions. The results of this systematic review identify the gaps in the present evidence-based information regarding NA and can be used as a tool to pursue future adherence research in pediatric populations.

Pharmaceutical research in paediatric populations and the new EU Paediatric Legislation: an industry perspective

A large proportion of medicines used in children are prescribed off-label, and children have often been denied access to new or innovative medications. Because such situation is unethical, the need to obtain paediatric information for medicines used in children seems nowadays a matter of consensus on a global basis. Based on this, it was clear in EU, like what has happened in the US, that there was a need for a legal obligation for Pharmaceutical Companies to perform studies. This new European Paediatric Regulation that entered into force in 2007 opens a new era of European drug regulatory history and will offer a major opportunity to improve children's health through advancements in research by providing a new framework for evaluating the efficacy and safety of medicines for children. But, paediatric development remains challenging and the hurdles of conducting research in paediatric population are numerous. The article presents the new European Paediatric Regulation, illustrates its rationale through paediatric psychopharmacology, and discusses some of its consequences on paediatric research from an industry perspective. Recommendations for further international collaboration are also suggested to make global paediatric development plans.