scholarly journals The National Paediatric Medicines Forum: Who we are and what are we doing

2021 ◽  
Author(s):  
Deshina Naidoo ◽  
Mary McCaskill ◽  
Michael Petrovski ◽  
Sean Turner ◽  
Sonya Stacey ◽  
...  
Keyword(s):  
Paediatric Medicines

Ethical, Legal and Regulatory Issues of Paediatric Translational Research. Call for an Adequate Model of Governance

2020 ◽  
Vol 27 (3) ◽  
pp. 213-231
Author(s):  
Annagrazia Altavilla ◽  
Viviana Giannuzzi ◽  
Mariangela Lupo ◽  
Donato Bonifazi ◽  
Adriana Ceci
Keyword(s):  
Translational Research ◽  
Research Infrastructure ◽  
Paediatric Research ◽  
Adequate Model ◽  
Regulatory Issues ◽  
Societal Issues ◽  
Translational Approach ◽  
Legal Frameworks ◽  
Ethical Oversight ◽  
Paediatric Medicines

Abstract The lack of paediatric medicines, including innovative and advanced ones, is a long-lasting and well-known problem at European and international levels. Despite the existing legal frameworks and incentives, children remain deprived of many kinds of therapy because of challenges faced in appropriately study and tailoring medicinal and other products for them. In this context, the necessity to foster paediatric research addressing unsolved and uncovered issues within a ‘translational approach’ has appeared. This article, after having clarified the concept of translational research in the perspective of the establishment of a European paediatric research infrastructure (RI), will identify and point out ethical, legal and regulatory issues particularly relevant in a children’s rights perspective. It concludes asking for the setting up of an adequate model of governance within a future RI, including adequate and independent ethical oversight and a pluridisciplinary common service dealing with ethical, legal and societal issues relevant for children.

scholarly journals Changes in availability of paediatric medicines in Australia between 1998 and 2002

2005 ◽  
Vol 59 (6) ◽  
pp. 736-742 ◽  
Author(s):  
Jocelyn Chui ◽  
June Tordoff ◽  
David Reith
Keyword(s):  
Paediatric Medicines

scholarly journals Cardiovascular Paediatric Medicines Development: Have Paediatric Investigation Plans Lost Heart?

Pharmaceutics ◽  
2020 ◽  
Vol 12 (12) ◽  
pp. 1176
Author(s):  
Bethany Faulkner ◽  
M. Begoña Delgado-Charro
Keyword(s):  
Cardiovascular Diseases ◽  
Health Technology ◽  
European Medicines Agency ◽  
Therapeutic Area ◽  
The European Union ◽  
Off Label Use ◽  
Off Label ◽  
Treat Heart Failure ◽  
Paediatric Medicines

This work aimed to establish whether paediatric needs in cardiovascular diseases have been met by paediatric investigation plans (PIPs) produced since the development of the European Union Paediatric Regulation in 2007. The European Medicines Agency repository was searched for patterns in the development of paediatric medicines in general. Next, positive PIPs related to cardiovascular diseases were scrutinized for outcomes and compared to specific paediatric cardiovascular needs. In total, 1866 PIPs were identified with 12% corresponding to decisions taken for cardiovascular medicines. However, despite this therapeutic area having the greatest number of overall PIPs, only 14% of established needs in paediatric cardiovascular diseases were addressed by PIPs with positive decisions. Further, 71.9% of PIPs with decisions in cardiovascular disease corresponded to full waivers, so the product would not be studied in paediatrics. Despite the progress found in overall numbers of PIPs published, cardiovascular products are still commonly used off-label in paediatrics. Particularly, there is a need to develop products to treat heart failure and hypertension, two areas with clear unmet clinical needs in paediatrics. A case study on valsartan showed that industry, regulators, health technology assessment bodies, and prescribers should work together to reduce off-label use of paediatric cardiovascular diseases (CVD).

scholarly journals P23 Accelerating and de-risking the production of paediatric oral formulations

2020 ◽  
Vol 105 (9) ◽  
pp. e18.1-e18
Author(s):  
Dilawar Khan ◽  
Daniel Kirby ◽  
Simon Bryson ◽  
Maryam Shah ◽  
Mohammed Afzal
Keyword(s):  
Paediatric Population ◽  
European Medicines Agency ◽  
List Type ◽  
Scanning Calorimetry ◽  
Glycopyrronium Bromide ◽  
Paediatric Regulation ◽  
Age Appropriate ◽  
Screening Platform ◽  
The Eu ◽  
Paediatric Medicines

Background & AimAs part of the EU paediatric regulation, the paediatric use marketing authorisation (PUMA) was introduced, with an aim to stimulate research in existing compounds that are off-patent and/or to help transform known off-label use into authorised use.1 However, success has been limited, with only a few products gaining a PUMA, such as Sialanar 320 micrograms/mL glycopyrronium (equivalent to 400 micrograms/mL glycopyrronium bromide). A distinct challenge to overcome in this area is the development of more ‘age appropriate formulations’, particularly with an excipient composition and load that is suitable for paediatric patients. This project aims to establish an excipient screening platform, supplemented with analytical characterisation of materials, which will act as a decision making tool to accelerate and de-risk the production of age appropriate paediatric medicines.MethodTo develop this excipient screening platform, a list of drugs that require an age appropriate formulation was produced using the ‘needs for paediatric medicines’ documents provided by the European medicines agency (EMA),2whilst common problematic excipients in paediatrics were identified using an EMA reflection paper.3 Literature and prescribing data were also reviewed to ensure drugs selected would benefit from an age appropriate formulation. Differential scanning calorimetry (DSC) to determine compatibility of selected drugs with widely used excipients was carried out using a TA DSCQ200 instrument (TA Instruments, New Castle, DE) with TA Instruments Universal Analysis 2000 software. Data was collected under nitrogen atmosphere (50 mL min−1) using pierced flat-bottomed TZero aluminium pans (sample mass about 2 mg) and heating rate of 10 °C min−1 in the range from 50 to 400°C. For samples containing both the drug and an excipient, 1 mg of each was measured out and gently mixed with a spatula for one minute.ResultsThe most common class of drugs identified as requiring age appropriate formulations were related to cardiovascular disorders and neurology, whilst the majority of drugs identified also exhibit poor aqueous solubilities. Some identified problematic excipients include ethanol, sodium benzoate and sorbitol; however, these excipients may still be used in paediatric formulations, as long as they are below certain concentrations (for example, ethanol concentration should not exceed 0.5% w/v for under 6 years old). Two drugs identified through the initial screening, carvedilol and nifedipine, were analysed by DSC, alone and then alongside starch from corn and starch 1500; the resulting DSC curves showed no changes in peak size, position (peak onset temperatures for nifedipine and carvedilol were observed at 173.2°C and 117.3°C, respectively) and shape, as well as no additional peaks, therefore suggesting compatibility between the tested samples.ConclusionThis first phase of the development of an excipient screening platform will continue to scan several different excipients with selected active pharmaceutical ingredients (APIs) in order to create compatibility profiles. The excipient screening platform generated will accelerate and de-risk the production of age appropriate formulations, as it would allow screening for potential incompatibilities and acceptability, alongside informing formulation of appropriate oral paediatric dosage forms.ReferencesEuropean Commission. State of Paediatric Medicines in the EU. 10 years of the EU Paediatric Regulation. COM (2017) 626. Available at: https://ec.europa.eu/health/sites/health/files/files/paediatrics/docs/2017_childrensmedicines_report_en.pdfNeeds for paediatric medicines - European Medicines Agency [Internet]. 2019 [cited 28 June 2019]. Available from: https://www.ema.europa.eu/en/human-regulatory/research-development/paediatric-medicines/needs-paediatric-medicinesReflection paper: formulations of choice for the paediatric population [Internet]. European Medicines Agency. 2019 [cited 28 June 2019]. Available from: https://www.ema.europa.eu/en/documents/scientific-guideline/reflection-paper-formulations-choice-paediatric-population_en.pdf

scholarly journals P59 Assessing medicines for safe use in paediatrics

2020 ◽  
Vol 105 (9) ◽  
pp. e37.1-e37
Author(s):  
Mary Worrall ◽  
Anne Fitzpatrick
Keyword(s):  
European Commission ◽  
Assessment Tool ◽  
New Products ◽  
Daily Intake ◽  
European Medicines Agency ◽  
List Type ◽  
Medicinal Products ◽  
Pharmaceutical Development ◽  
Human Use ◽  
Paediatric Medicines

AimThis service review aimed to reassess and upgrade the ‘New Products Assessment Form’ and to develop an assessment tool in line with European regulations governing paediatric medicines. Many medicinal products routinely used to treat the paediatric population have not been studied or authorised for paediatric use, which means there is widespread unlicensed and ‘off-label’ use of medicines. Medicines deemed safe in adult formulations may not be appropriate for paediatric patients. Medicines must therefore be carefully selected based on agreed criteria including, but not limited to: licensing, excipients, administration, labelling, similarity to other products, safety and handling.MethodA literature review was conducted. Guidance, information, and advice was sought from other healthcare institutions, and European guidelines and directives informing current practise around excipients in paediatric medicines. Pharmacy colleagues were consulted during the development of the tool, and an accessible assessment tool was completed for use in a tertiary paediatric hospital.1–4ResultsThis is the first comprehensive ‘New Products Assessment Form’ in the hospital which complies with the European Medicines Agency (EMA) directives governing excipients in paediatric medicines. The document highlights clearly potential issues and risks associated with product excipients, licensing status, warning label guidance and allows for recording of rationale for the selection of medicines. The ‘New Products Assessment Form’ is intended to highlight potential issues associated with excipients and their associated acceptable daily intake (ADI), but it will also highlight other risks associated with medicines used in paediatrics e.g. inadequate labelling, translation requirements for foreign products, sound-alike/look-alike products, safety and handling, and others.ConclusionThis revised assessment tool has been approved for use in the hospital pharmacy. It will be made available in hospital and community pharmacies on request. Use of the tool should be monitored and audited.ReferencesAnnex to the European Commission guideline on ‘Excipients in the labelling and package leaflet of medicinal products for human use’ (SANTE-2017-11668). https://www.ema.europa.eu/en/documents/scientific-guideline/annex-european-commission-guideline-excipients-labelling-package-leaflet-medicinal-products-human_en.pdf. NPPG Neonatal and Paediatric Pharmacists Group Newsletter No 61 Autumn 2016. Excipients in medicines for children. http://nppg.org.uk/wp-content/uploads/2017/04/NPPG-61.pdfQuestions and Answers on Ethanol in the context of the revision of the guideline on ‘Excipients in the label and package leaflet of medicinal products for human use’ (CPMP/463/00) https://www.ema.europa.eu/en/documents/scientific-guideline/questions-answers-ethanol-context-revision-guideline-excipients-label-package-leaflet-medicinal_en.pdfEMA. Guideline on pharmaceutical development of medicines for paediatric use.https://www.ema.europa.eu/en/documents/scientific-guideline/guideline-pharmaceutical-development-medicines-paediatric-use_en.pdf

Paediatric Medicines: A View from Patient Organisations

2006 ◽  
pp. 13-24
Author(s):  
Alastair Kent ◽  
Cor Oosterwijk ◽  
Tsveta Schyns ◽  
Ysbrand Poortman
Keyword(s):  
Patient Organisations ◽  
Paediatric Medicines

Design of methodological tools to assess acceptability of paediatric medicines

2016 ◽  
Vol 511 (2) ◽  
pp. 1143-1144 ◽  
Author(s):  
Punam Mistry ◽  
Hannah Batchelor ◽  
Carly Tibbins
Keyword(s):  
Paediatric Medicines
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