A European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA)

2011 ◽  
Vol 97 (3) ◽  
pp. 185-188 ◽  
Author(s):  
Nicolino Ruperto ◽  
Irmgard Eichler ◽  
Ralf Herold ◽  
Gilles Vassal ◽  
Carlo Giaquinto ◽  
...  
Keyword(s):  
European Medicines Agency ◽  
Paediatric Research ◽  
European Network

scholarly journals Recommendations by the European Network of Paediatric Research at the European Medicines Agency (Enpr-EMA) Working Group on preparedness of clinical trials about paediatric medicines process

2021 ◽  
pp. archdischild-2020-321433
Author(s):  
Angeliki Siapkara ◽  
Claudio Fracasso ◽  
Gunter F Egger ◽  
Carmelo Rizzari ◽  
Cristina Serén Trasorras ◽  
...  
Keyword(s):  
Clinical Trials ◽  
Working Group ◽  
Time Frame ◽  
European Medicines Agency ◽  
Children And Families ◽  
Paediatric Research ◽  
European Network ◽  
Children And Young People ◽  
Present Information ◽  
Patient Advocacy Groups

Conduct of clinical trials in babies, children and young people is often hindered by issues that could have been foreseen before the trial opened; that is, some clinical trials are often underprepared. In order to identify a good approach to trial preparedness, the European Network of Paediatric Research at the European Medicines Agency formed a working group. The Working Group included representation from regulators, industry, academics, paediatric clinical research networks and parents.The Working Group consulted widely about how to prepare for paediatric clinical trials. The Group’s detailed recommendations have been published (https://www.ema.europa.eu/en/documents/other/preparedness-medicines-clinical-trials-paediatrics-recommendations-enpr-ema-working-group-trial_en.pdf).This paper is a summary of the key recommendations including the following: start early, preferably in parallel to designing the medicine’s development plan and individual protocols; identify the rationale and clinical need; listen to the perspectives of children and families, and of patient advocacy groups; identify how many people will be eligible for the trial; identify the resources needed, such as clinical facilities (including play therapy) and out-of-pocket expenditure by participants and their families; use all available data to estimate what is possible; present information about preparedness in a structured way; deploy proportionate resources to support the preparation of trials.A well-prepared, well-designed trial is likely to require fewer changes during its course, be run in a shorter time frame and achieve expected objectives.

scholarly journals Standardising neonatal and paediatric antibiotic clinical trial design and conduct: the PENTA-ID network view

BMJ Open ◽  
2019 ◽  
Vol 9 (12) ◽  
pp. e032592
Author(s):  
Laura Folgori ◽  
Irja Lutsar ◽  
Joseph F Standing ◽  
A Sarah Walker ◽  
Emmanuel Roilides ◽  
...  
Keyword(s):  
Clinical Trial Design ◽  
Evidence Base ◽  
European Medicines Agency ◽  
Efficacy And Safety ◽  
Paediatric Research ◽  
European Network ◽  
Exclusion Criteria ◽  
Clinical Investigator ◽  
Key Stakeholders ◽  
Safety Studies

Antimicrobial development for children remains challenging due to multiple barriers to conducting randomised clinical trials (CTs). There is currently considerable heterogeneity in the design and conduct of paediatric antibiotic studies, hampering comparison and meta-analytic approaches. The board of the European networks for paediatric research at the European Medicines Agency (EMA), in collaboration with the Paediatric European Network for Treatments of AIDS—Infectious Diseases network (www.penta-id.org), recently developed a Working Group on paediatric antibiotic CT design, involving academic, regulatory and industry representatives. The evidence base for any specific criteria for the design and conduct of efficacy and safety antibiotic trials for children is very limited and will evolve over time as further studies are conducted. The suggestions being put forward here are based on the adult EMA guidance, adapted for neonates and children. In particular, this document provides suggested guidance on the general principles of harmonisation between regulatory and strategic trials, including (1) standardised key inclusion/exclusion criteria and widely applicable outcome measures for specific clinical infectious syndromes (CIS) to be used in CTs on efficacy of antibiotic in children; (2) key components of safety that should be reported in paediatric antibiotic CTs; (3) standardised sample sizes for safety studies. Summarising views from a range of key stakeholders, specific criteria for the design and conduct of efficacy and safety antibiotic trials in specific CIS for children have been suggested. The recommended criteria are intended to be applicable to both regulatory and clinical investigator-led strategic trials and could be the basis for harmonisation in the design and conduct of CTs on antibiotics in children. The next step is further discussion internationally with investigators, paediatric CTs networks and regulators.

scholarly journals Guidance on conducting systematic reviews/meta-analyses of pharmacoepidemiological studies of safety outcomes: the gap is now filled

2016 ◽  
Vol 25 (5) ◽  
pp. 425-427 ◽  
Author(s):  
S. Cortese
Keyword(s):  
Systematic Reviews ◽  
Data Extraction ◽  
European Medicines Agency ◽  
Study Quality ◽  
European Network ◽  
Quality Appraisal ◽  
Comprehensive Guidance ◽  
Extraction Study ◽  
Safety Outcomes ◽  
Meta Analyses

Until recently, no comprehensive guidance specifically on the conduction of systematic reviews and meta-analyses of pharmacoepidemiological studies of safety outcomes was available. In December 2015, the European Network of Centres for Pharmacoepidemiology and Pharamacovigilance (ENCePP), a network coordinated by the European Medicines Agency, published their ‘Guidance on conducting systematic reviews and meta-analyses of completed comparative pharmacoepidemiological studies of safety outcomes’, filling an important gap in the field. This paper highlights the ENCePP recommendations in terms of study identification, data extraction, study quality appraisal and analytical plan. Although the ENCePP document should not be considered as definitive, since it will likely be refined following researchers’ feedback, it is expected that it will be highly influential and useful for the field, with the ultimate goal to improve and standardise the conduction and reporting of systematic reviews/meta-analyses of pharmacoepidemiological studies of safety outcomes.

The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance: application to diabetes and vascular disease

2011 ◽  
Vol 11 (6) ◽  
pp. 304-307 ◽  
Author(s):  
Kevin V Blake ◽  
Camilla Smeraldi ◽  
Xavier Kurz ◽  
Peter Arlett ◽  
Stella Blackburn ◽  
...  
Keyword(s):  
Drug Utilisation ◽  
Real Life ◽  
European Medicines Agency ◽  
The European Union ◽  
Medicinal Products ◽  
European Network ◽  
Study Protocols ◽  
Real Life Setting ◽  
The Impact ◽  
Support Decision Making

The European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) is an initiative led by the European Medicines Agency (EMA) aimed at further strengthening the post-authorisation monitoring of medicinal products in Europe by facilitating the undertaking of multi-centre, independent, studies focusing on safety and on benefit/risk. A key objective of ENCePP is to provide a unique point of access for all involved stakeholders, including industry or regulatory authorities, who are seeking collaboration for the commissioning or the performance of post-authorisation studies. The 2010 EMA regulatory action relating to rosiglitazone included a pharmacoepidemiological drug utilisation study to evaluate the benefit–risk profile in a real-life setting and has also led to the commissioning of an ENCePP study to evaluate the impact of risk-minimisation activities. ENCePP seeks to improve the European Union capacity to conduct such studies and thus support decision making. Application of the ENCePP study concept will result in an increase in trust in medicines and their use. In addition, the ENCePP register of studies will serve as a resource to allow for ready access to study protocols and results, thereby ensuring transparency.

scholarly journals The Role of Regulator-Imposed Post-Approval Studies in Health Technology Assessments for Conditionally Approved Drugs

2020 ◽  
Author(s):  
Rick A. Vreman ◽  
Lourens T. Bloem ◽  
Stijn van Oirschot ◽  
Jarno Hoekman ◽  
Menno E. van der Elst ◽  
...  
Keyword(s):  
Health Technology Assessment ◽  
Technology Assessment ◽  
Relative Effectiveness ◽  
Health Technology ◽  
Initial Assessment ◽  
European Medicines Agency ◽  
European Network ◽  
Study Results ◽  
Approved Drugs

Background: The European Medicines Agency (EMA) aims to resolve uncertainties associated with conditionally approved drugs by imposing post-approval studies. Results from these studies may be relevant for health technology assessment (HTA) organizations. This study investigated the role of regulator-imposed post-approval studies within HTA. Methods: For all conditionally approved drugs up to December 2018, regulator-imposed post-approval studies were identified from EMA’s public assessment reports. The availability for and inclusion of study results in relative effectiveness (re)assessments were analyzed for 4 European HTA organizations: NICE (National Institute for Health and Care Excellence, England/Wales), HAS (Haute Autorité de Santé, France), ZIN (Zorginstituut Nederland, the Netherlands) and the European Network for Health Technology Assessment (EUnetHTA, Europe). When study results became available between an HTA organization’s initial assessment and reassessment, it was evaluated whether and how they affected the assessment and its outcome. Results: For 36 conditionally approved drugs, 98 post-approval studies were imposed. In total, 81 initial relative effectiveness assessments (REAs) and 13 reassessments were available, with numbers of drugs (re)assessed varying greatly between jurisdictions. Study results were available for 16 initial REAs (20%) and included in 14 (88%), and available for 10 reassessments (77%) and included in all (100%). Five reassessments had an outcome different from the initial REA, with 4 (2 positive and 2 negative changes) relating directly to the new study results. Reassessments often cited the inability of post-approval studies to resolve the concerns reported in the initial REA. Conclusion: Results from regulator-imposed post-approval studies for conditionally approved drugs were not often used in REAs by HTA organizations, because they were often not yet available at the time of initial assessment and because reassessments were scarce. When available, results from post-approval studies were almost always used within HTA, and they have led to changes in conclusions about drugs’ relative effectiveness. Post-approval studies can be relevant within HTA but the current lack of alignment between regulators and HTA organizations limits their potential.

European Medicines Agency review of post-authorisation studies with implications for the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance

2011 ◽  
Vol 20 (10) ◽  
pp. 1021-1029 ◽  
Author(s):  
Kevin V. Blake ◽  
Stefanie Prilla ◽  
Sophie Accadebled ◽  
Marie Guimier ◽  
Monica Biscaro ◽  
...  
Keyword(s):  
European Medicines Agency ◽  
European Network

RTS,S/AS01, a vaccine targeting pre-erythrocytic stages of Plasmodium falciparum

2017 ◽  
Vol 1 (6) ◽  
pp. 533-537
Author(s):  
Lorenz von Seidlein ◽  
Borimas Hanboonkunupakarn ◽  
Podjanee Jittmala ◽  
Sasithon Pukrittayakamee
Keyword(s):  
Protective Efficacy ◽  
Age Groups ◽  
Sub Saharan Africa ◽  
Phase Iii ◽  
European Medicines Agency ◽  
Older Children ◽  
Pivotal Phase ◽  
Malaria Epidemiology ◽  
Maximum Benefit ◽  
Sub Saharan

RTS,S/AS01 is the most advanced vaccine to prevent malaria. It is safe and moderately effective. A large pivotal phase III trial in over 15 000 young children in sub-Saharan Africa completed in 2014 showed that the vaccine could protect around one-third of children (aged 5–17 months) and one-fourth of infants (aged 6–12 weeks) from uncomplicated falciparum malaria. The European Medicines Agency approved licensing and programmatic roll-out of the RTSS vaccine in malaria endemic countries in sub-Saharan Africa. WHO is planning further studies in a large Malaria Vaccine Implementation Programme, in more than 400 000 young African children. With the changing malaria epidemiology in Africa resulting in older children at risk, alternative modes of employment are under evaluation, for example the use of RTS,S/AS01 in older children as part of seasonal malaria prophylaxis. Another strategy is combining mass drug administrations with mass vaccine campaigns for all age groups in regional malaria elimination campaigns. A phase II trial is ongoing to evaluate the safety and immunogenicity of the RTSS in combination with antimalarial drugs in Thailand. Such novel approaches aim to extract the maximum benefit from the well-documented, short-lasting protective efficacy of RTS,S/AS01.

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