WIREDGOV

The European Medicines Agency (EMA), the United States Food and Drug Administration (FDA) and Health Canada are co-organising a workshop to discuss the requirements for the development of medicines for pulmonary arterial hypertension (PAH) that address the high unmet medical needs of children.

The event will take place at EMA’s premises in London on 12 June 2017, and a live broadcast will be available on the EMA website.

Pulmonary arterial hypertension is a disorder of the blood vessels leading to the lungs, in which the blood pressure in the pulmonary artery becomes high. PAH is a serious, debilitating disease that affects both children and adults but the condition is rare in children and the signs and symptoms often differ from those seen in adults. There is a need for treatments targeting the paediatric form of the disease; however the conduct of clinical trials has proven difficult due to the rarity and gaps in knowledge of the disease in younger populations.

The workshop will bring together leading experts and stakeholders in PAH across the globe – including regulators, researchers, clinicians, healthcare professionals, patients and pharmaceutical industry representatives.

The objectives are to:

• analyse the problems related to the conduct of clinical trials in children with PAH,
• refine endpoints and study design to address the challenges identified,
• set priorities for future research in specific medicine development aspects,
• provide medicine developers with more guidance specific to global product development, taking into account current limitations in the development.

The workshop will be followed by a meeting between regulators only on 13 June to investigate potential compatible agreements and remaining points to be addressed in the next steps.

A report on the workshop will be published on the EMA website. This workshop follows a European Union/United States strategic meeting in September 2016 on the future of paediatric medicines, the intensification of collaboration and increased convergence to address challenges with paediatric medicines.