European Paediatric Formulation Initiative Formulating better medicines for children Dr Catherine Tuleu Mrs Smita Salunke Chair of EuPFI EuPFI Senior Scientific Manager Reader, Department of Pharmaceutics UCL School of Pharmacy, UK & Director, Centre for Paediatric Pharmacy Research UCL School of Pharmacy, UK 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 1
Quick facts www.eupfi.org => Non profit organisation => Established in London (UK) in 2007 led by The School of Pharmacy ⇒ ~ 20 Members from industry, academia, hospital and regulatory agencies 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 2
Mission & Objectives To promote and facilitate 1. Identify the issues and challenges preparation of better & safer - development of paediatric medicines for children through formulation & consider ways towards better medications and – Sharing expertise and interactive discussion clinically relevant dosage forms between industry, for children academia, clinical and 2. Promote early pharmaceutical regulatory professionals. consideration for development of EMA observer • paediatric medicines. – Information dissemination EuPFI OBJECTIVES 3. Identify potential information, – Raising awareness EuPFI MISSION knowledge, know-how gaps in (publications, conferences the paediatric formulation etc.) development. – Linking and networking 4. Improve the availability of IQ • information of paediatric SPeaDD-UK (iUK) : tools for • formulations. taste, acceptability and vitro/vivo correlation 24/06/2016 3 EuPFI-IQ, Mcersi-FDA workshop, June 2016
Annual Conferences 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 4
Framework 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 5
TASTE (MASKING & TESTING) Etat des lieux ( Cram et al – 2009) Acceptability/Preference US PFI AGE-APPROPRIATE FORMULATIONS (NIH NICHD) Review on EXCIPIENTS Taste-Masking Taste formulation factors Technologies Assessment affecting E-tongue interlab Approaches to acceptability 3 Papers published work (Pien et al, taste masking (Liu et al, 2015) 40 Excipients 2014) (Walsh et.al, 2015) > 700 users (Salunke et al, 2013, 2014, 2015) Modification vs Key outputs Manipulation of DF Standardization COLLABORATIVE of types of paediatric -Current devices pharmaceutical preps OUTPUTS` challenges + recent BCS Classification (Ernest et.al, 2012) - IQ survey (EuPFI developments paed ? (Batchelor et al, Athens 2014) (Walsh et al, 2012) 2014, 2016) - Devices views/ Risk/benefit - Non-human tools Review – paed experiences by EU framework biopharm method for the evaluation of proposed HCPs GAPS (Batchelor et al 2013) bitter APIs (SPeaDD-UK) (Walsh et al, 2015) for the selection BIOPHARMACEUTICS drug discovery today ADMINISTRATION of AAF (Sam et al, 2012) May 2016 DEVICES 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 6
Curre rrent nt key y priori rities AGE APPROPRIATE BIOPHARM EXCIPIENTS ADMIN. FORMULATIONS/ TASTE • Prioritise research - in • EXPAND STEP DEVICES • Palatability assessment of paediatric DATABASE • Survey – paediatric dosage forms /APIs biopharmaceutics • Identify how patients and ( including in vitro methods) • Review strategies - excipients can carers on use • Dosage form acceptability, safety, bridging studies to affect the of efficacy and dose flexibility support the absorption of administratio development of drugs and also • Modification of dosage forms n devices for paediatric medicines affect GI • Manufacturing challenges/Poorly children physiology in • Review the impact of soluble APIs children food on the predicted in vivo performance of paediatric medicines 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 7
Shared vision Initial discussion Oct. 2012…idea of a ‘ paediatric symposium with FDA’ Oct. 2013…evolved into a transatlantic workshop since Dec. 2014 Bring together subject matter experts/key players/stakeholders in the field to find solutions to - Acceptability assessment of paediatric dosage forms - Derisking food/drink effects when administered with medicines and biopharm considerations - Safety qualification of excipients for paediatric medicines Harmonized Strategies 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 8
For more information read our article "European Paediatric Formulation Initiative - Formulating ideas for better medicines for children in the upcoming AAPS PharmSciTech Special Theme Issue on pediatrics dosage forms www.eupfi.org Thanks to M- Cersi 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 9
BA BACK CK –UP SL SLID IDES 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 10
Key priorities & outputs AGE APPROPRIATE FORMULATIONS Dosage form acceptability, safety, efficacy and dose flexibility • Food/drink effects on administration of medicines • Safety and efficacy of co-administration • Manufacturing challenges in developing paediatric formulations • how formulation factors affect compounding and manipulation - Balancing of a quality target the acceptability of different approval by medicines regulators product profile against technical oral medicines in children and non-approved preparation to challenges and development fulfil the needs feasibility 11 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016
TASTE ASSESSMENT & TASTE MASKING Bring together subject matter experts/key players/stakeholders in the fields of taste evaluation to propose a synopsis (guidance) for acceptable, sound, scientific approaches to address acceptability/palatability assessment of paediatric dosage forms Understand the current state of the art of taste masking technologies and platforms. Focus on the limitations and technology gaps (in formulation platforms suitable for different age ranges) and identify opportunities more effective age-appropriate taste masking technologies. Understanding the current state of the art, and limitations, of taste assessment in vitro and in vivo methodologies. 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 12
WS 1 WS 2 WS 3 WS4 WS5 ADMINISTRATION DEVICES PURPOSE: Highlight all relevant aspects from specific development issues to appropriate standard laboratory test methods and methodologies for handling studies of such devices. In-depth literature research is performed to create an overview on the topic and to come up with guidance on requirements and feasibility. CURRENT PRIORITIES: Evaluate current status of child appropriate administration devices. • Get insight in devices test methods. • Investigate requirements for handling studies. • 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 13 Develop a guideline •
ADMINISTRATION DEVICES WS 1 WS 2 WS 3 WS4 WS5 This reflection paper exploratory survey overview of currently available provided some valuable insights into the views paediatric administration devices and and experiences of European hospital doctors, highlights some of the challenges nurses and pharmacists, regarding oral and associated with, recommendations and respiratory administration devices for recent developments in delivery devices paediatric patients. 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 14
WS 1 WS 2 WS 3 WS4 WS5 PHARMACEUTICAL EXCIPIENTS PURPOSE: To address safety issues and problems associated with the excipients likely to be used in paediatric formulations. CURRENT PRIORITIES: 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 15 15
PHARMACEUTICAL EXCIPIENTS WS 1 WS 2 WS 3 WS4 WS5 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 16
WS 1 WS 2 WS 3 WS4 WS5 BIOPHARMACEUTICS PURPOSE: Understand the current tools available to measure biopharmaceutical performance of paediatric medicines Highlight the importance of additional research into paediatric biopharmaceutics Undertake collaborative research to drive the development of age-appropriate in vitro tools that can be used to predict the in vivo performance of paediatric medicines 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 17
BIOPHARMACEUTICS WS 1 WS 2 WS 3 WS4 WS5 CURRENT PRIORITIES Prioritise research • activity to be undertaken in paediatric biopharmaceutics Review strategies • Note brings together some key Review highlight currently used in issues in direct extrapolation several knowledge gaps in bridging studies to from adults into paediatric current methodologies in support the populations. paediatric biopharmaceutics development of paediatric medicines provide recommendations explore how a paediatric BCS Review the impact of • based on existing knowledge may differ to that in adults food on the to adapt tests to better predicted in vivo represent paediatric patient performance of populations paediatric medicines 24/06/2016 EuPFI-IQ, Mcersi-FDA workshop, June 2016 18
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