Successful private–public funding of paediatric medicines research: lessons from the EU programme to fund research into off-patent medicines

The European Paediatric Regulation mandated the European Commission to fund research on off-patent medicines with demonstrated therapeutic interest for children. Responding to this mandate, five FP7 project calls were launched and 20 projects were granted. This paper aims to detail the funded projects and their preliminary results. Publicly available sources have been consulted and a descriptive analysis has been performed. Twenty Research Consortia including 246 partners in 29 European and non-European countries were created (involving 129 universities or public funded research organisations, 51 private companies with 40 SMEs, 7 patient associations). The funded projects investigate 24 medicines, covering 10 therapeutic areas in all paediatric age groups. In response to the Paediatric Regulation and to apply for a Paediatric Use Marketing Authorisation, 15 Paediatric Investigation Plans have been granted by the EMAPaediatric Committee, including 71 studies of whom 29 paediatric clinical trials, leading to a total of 7,300 children to be recruited in more than 380 investigational centres. Conclusion: Notwithstanding the EU contribution for each study is lower than similar publicly funded projects, and also considering the complexity of paediatric research, these projects are performing high-quality research and are progressing towards the increase of new paediatric medicines on the market. Private–public partnerships have been effectively implemented, providing a good example for future collaborative actions. Since these projects cover a limited number of offpatent drugs and many unmet therapeutic needs in paediatrics remain, it is crucial foreseeing new similar initiatives in forthcoming European funding programmes

Ethical dimensions of paediatric nursing: A rapid evidence assessment

© 2016, © The Author(s) 2016. Background: Paediatric nurses often face complex situations requiring decisions that sometimes clash with their own values and beliefs, or with the needs of the children they care for and their families. Paediatric nurses often use new technology that changes the way they provide care, but also reduces their direct interaction with the child. This may generate ethical issues, which nurses should be able to address in the full respect of the child. Research question and objectives: The purpose of this review is to describe the main ethical dimensions of paediatric nursing. Our research question was, ‘What are the most common ethical dimensions and competences related to paediatric nursing?’ Research design: A rapid evidence assessment. Method: According to the principles of the rapid evidence assessment, we searched the PubMed, SCOPUS and CINAHL databases for papers published between January 2001 and March 2015. These papers were then independently read by two researchers and analysed according to the inclusion criteria. Ethical considerations: Since this was a rapid evidence assessment, no approval from the ethics committee was required. Findings: Ten papers met our inclusion criteria. Ethical issues in paediatric nursing were grouped into three areas: (a) ethical issues in paediatric care, (b) social responsibility and (c) decision-making process. Conclusion: Few studies investigate the ethical dimensions and aspects of paediatric nursing, and they are mainly qualitative studies conducted in critical care settings based on nurses’ perceptions and experiences. Paediatric nurses require specific educational interventions to help them resolve ethical issues, contribute to the decision-making process and fulfil their role as advocates of a vulnerable population (i.e. sick children and their families). Further research is needed to investigate how paediatric nurses can improve the involvement of children and their families in decision-making processes related to their care plan

γ-H2AX foci as in vivo effect biomarker in children emphasize the importance to minimize x-ray doses in paediatric CT imaging

Objectives: Investigation of DNA damage induced by CT x-rays in paediatric patients versus patient dose in a multicentre setting. Methods: From 51 paediatric patients (median age, 3.8 years) who underwent an abdomen or chest CT examination in one of the five participating radiology departments, blood samples were taken before and shortly after the examination. DNA damage was estimated by scoring gamma-H2AX foci in peripheral blood T lymphocytes. Patient-specific organ and tissue doses were calculated with a validated Monte Carlo program. Individual lifetime attributable risks (LAR) for cancer incidence and mortality were estimated according to the BEIR VII risk models. Results: Despite the low CT doses, a median increase of 0.13 gamma-H2AX foci/cell was observed. Plotting the induced gamma-H2AX foci versus blood dose indicated a low-dose hypersensitivity, supported also by an in vitro dose-response study. Differences in dose levels between radiology centres were reflected in differences in DNA damage. LAR of cancer mortality for the paediatric chest CT and abdomen CT cohort was 0.08 and 0.13% respectively. Conclusion: CT x-rays induce DNA damage in paediatric patients even at low doses and the level of DNA damage is reduced by application of more effective CT dose reduction techniques and paediatric protocols

A comparison of clinical paediatric murmur assessment with echocardiography

Objective: To compare the clinical acumen of paediatric cardiovascular examination between various hospital paediatrician grades. Design: Prospective data collection of clinical and echocardiography findings on paediatric echocardiography referrals. Setting and patients: All paediatric patients (birth - 14 years) referred for echocardiography, in a regional hospital catering for the island population of Malta. Echocardiography was carried out by three paediatricians with tertiary training in this technique. Main outcome measures: Pre-echocardiography clinical diagnoses were compared with echocardiography results according to grade of referring hospital doctor (ranging from houseman to consultant). Both normal and abnormal hearts at echocardiography were included. Results: Echocardiographers had the highest clinical accuracy and the highest attempts at reaching a clinical diagnosis. Accuracy and attempts at diagnosis decreased as doctor's hospital grade decreased, from consultant to houseman. Ventricular septal defect was the most easily diagnosed lesion. Atrial septal defect was often misdiagnosed as pulmonary stenosis.peer-reviewe

Trends of intervention for paediatric stone disease over the last two decades (2000–2015): A systematic review of literature

Objective: To ascertain the publication trends of interventions for paediatric kidney stone disease (KSD) we conducted a systematic review of literature over the last 16 years. Patients and methods: With a rise of paediatric KSD and related interventions, a systematic review using PubMed was done over the last 16 years for all published papers on ‘Paediatric stone disease intervention – ureteroscopy (URS), shockwave lithotripsy (SWL), percutaneous nephrolithotomy (PCNL), open stone surgery, and laparoscopic stone surgery’. The search was limited to English language articles with a published abstract, whilst case reports, animal and laboratory studies, were excluded. We also analysed the data in two time periods, period-1 (2000–2007) and period-2 (2008–2015). Results: During the last 16-years, 339 papers were published on paediatric stone disease intervention on PubMed. This included papers on URS (95), PCNL (97), SWL (102), open stone surgery (34) and laparoscopic stone surgery (11). During period-1 and period-2 there were 30 and 65 papers on URS, 16 and 81 papers on PCNL, 33 and 60 papers on SWL, nine and 25 papers on open surgery, respectively. When comparing the two periods, there were 92 published papers for all interventions in period-1 and this had risen almost threefold to 247 papers in period-2. Conclusions: Our systematic review shows that intervention for KSD in the paediatric age group has risen over the last 8 years. Whilst URS, SWL, open surgery and laparoscopic surgery have all doubled, PCNL has risen fivefold reflecting an increase in the new minimally invasive PCNL techniques

E-survey of current international physiotherapy practice for children with ataxia following surgical resection of posterior fossa tumour.

ObjectiveTo determine current international practice regarding physiotherapy input for children with ataxia following surgery for posterior fossa tumour. Design: An e-survey covering the following domains: participant demographics, treatment/ intervention, virtual training, intensity/timing of treatment, and aims and outcomes of physiotherapy management.ParticipantsPhysiotherapists involved in the management of children with ataxia following surgical resection of posterior fossa tumour. Participants were contacted via 6 key groups; Paediatric Oncology Physiotherapy Network (POPs), Association of Paediatric Chartered Physiotherapists (APCP), European Paediatric Neurology Society (EPNS), International Society of Paediatric Oncology (SIOP)-Europe Brain Tumour Group, Posterior Fossa Society (PFS), and Pediatric Oncology Special Interest Group (SIG) (American Physical Therapy Association).ResultsA total of 96 physiotherapists participated: UK (n =53), rest of Europe (n = 23), USA/ Canada (n = 10), and Australia/NZ (n = 10). The most common physiotherapy interventions used were balance exercises, gait re-education and proximal control activities. The most frequently used adjuncts to treatment were mobility aids and orthotics. Challenges reported regarding physiotherapy treatment were: reduced availability of physiotherapy input following discharge from the acute setting, lack of evidence, impact of adjuvant oncology treatment, and psychosocial impact.ConclusionThis e-survey provides an initial scoping review of international physiotherapy practice in this area. It establishes a foundation for future research on improving rehabilitation of ataxia in this population

Pediatric Rotavirus Gastroenteritis: A 2 year Analysis to Understand Current Prevalence in Mumbai

Many studies have established the high prevalence of paediatric Rotavirus gastroenteritis in India. The importance of rapid diagnosis of rotavirus infection has also been stressed upon, to initiate prompt rehydration therapy and prevent unnecessary use of antibiotics .We undertook a retrospective analysis of 327 paediatric stool specimens to understand the current prevalence and seasonal distribution of cases in Mumbai and its surrounding areas. Overall Rotavirus positivity rate was 37.9 %, with peak positivity in winter seasons. Infections were more common upto 2 years of age. Incidence of bacterial and parasitic coinfections was low

A feasibility study of signed consent for the collection of patient identifiable information for a national paediatric clinical audit database

Objectives: To investigate the feasibility of obtaining signed consent for submission of patient identifiable data to a national clinical audit database and to identify factors influencing the consent process and its success. Design: Feasibility study. Setting: Seven paediatric intensive care units in England. Participants: Parents/guardians of patients, or patients aged 12-16 years old, approached consecutively over three months for signed consent for submission of patient identifiable data to the national clinical audit database the Paediatric Intensive Care Audit Network (PICANet). Main outcome measures: The numbers and proportions of admissions for which signed consent was given, refused, or not obtained (form not returned or form partially completed but not signed), by age, sex, level of deprivation, ethnicity (South Asian or not), paediatric index of mortality score, length of hospital stay (days in paediatric intensive care). Results: One unit did not start and one did not fully implement the protocol, so analysis excluded these two units. Consent was obtained for 182 of 422 admissions (43%) (range by unit 9% to 84%). Most (101/182; 55%) consents were taken by staff nurses. One refusal (0.2%) was received. Consent rates were significantly better for children who were more severely ill on admission and for hospital stays of six days or more, and significantly poorer for children aged 10-14 years. Long hospital stays and children aged 10-14 years remained significant in a stepwise regression model of the factors that were significant in the univariate model. Conclusion: Systematically obtaining individual signed consent for sharing patient identifiable information with an externally located clinical audit database is difficult. Obtaining such consent is unlikely to be successful unless additional resources are specifically allocated to training, staff time, and administrative support