EMA/FDA/Health Canada joint workshop addressing unmet needs of children with pulmonary arterial hypertension

• Date:
  Monday, 12 June 2017, All day
• Location:
  European Medicines Agency, Amsterdam, the Netherlands

This joint workshop organised the European Medicines Agency (EMA), the United States Food and Drug Administration (FDA) and Health Canada brings together experts and stakeholders to discuss requirements for the development of medicines in paediatric pulmonary arterial hypertension (PAH) and to address the need of the current paediatric clinical practice in a timely manner. The objectives are to improve the understanding of problems related to the conduct of clinical trials in the paediatric population, refine endpoints and study design to address the clinical trials challenges in this rare paediatric population, set priorities in future research in the field of pharmacokinetics and pharmacodynamics measurements and post-marketing tools in paediatric PAH medicines and provide medicine developers with more guidance specific to global product development which takes into account current limitations.

Documents

Programme of the EMA-FDA-Health Canada workshop on paediatric pulmonary arterial hypertension (PAH)

Reference Number: EMA/848554/2016 Rev.1

List of participants - EMA/FDA/Health Canada workshop on paediatric pulmonary arterial hypertension (PAH)

Reference Number: EMA/356444/2017 Rev.1

Presentation - Paediatric Pulmonary Arterial Hypertension: Regulators perspective on a global challenge (Cà©cile Ollivier, EMA)

Presentation - Multiregional Regulatory Considerations in Pediatric Drug Development (L.Yao, U.S. FDA)

Presentation - PHA Europe: PH kids

Presentation - In focus – The paediatric PAH population clinicians perspectives (M.Beghetti)

Presentation - EMA/FDA/Health Canada workshop on paediatric pulmonary arterial hypertension

Presentation - Comparison between adult and pediatric populations with I/HPAH and PAH-CHD in the Bologna ARCA registry (N. Galià¨)

Presentation - Paediatric pulmonary arterial hypertension current treatment, needs and challenges (Rolf M.F. Berger, University Medical Center Groningen)

Presentation - Basis for regulatory decision-making: Objectives of paediatric development (A.El-Gazayerly, Dutch Medicines Evaluation Board)

Presentation - EMA/FDA/Health Canada workshop on paediatric pulmonary arterial hypertension: Endpoints (Health Canada)

Presentation - EMA/FDA/Health Canada workshop on paediatric pulmonary arterial hypertension: Hemodynamic Endpoints (C.Garnett, FDA)

Presentation - EMA/FDA/Health Canada workshop on paediatric pulmonary arterial hypertension: Endpoints – Patients perspective (P. Hassel)

Presentation - Use of quantitative tools for study planning purposes and study design optimisation (V. Gigante, AIFA)

Presentation - Methodological considerations (A. Thomson, EMA)

Presentation - Industry perspective on partnering with academia and agencies in drug development for children with PAH (J. Strait, Merck Sharp & Dohme)

Presentation - Patient’s/parent’s view on clinical trial challenges (Deutsche Herzstiftung)

Presentation - EnprEMA network perspectives (M. Turner, EMA)

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