Module232025

12/05/2025

Drug development takes place in an ecosystem

Drug development and access to novel or essential medicines involve various stakeholders and decision-makers, each with different objectives, at different stages.

Key Points: • Identifying Barriers: Recognize obstacles within the ecosystem, which may include issues related to content, processes, or capacity. • Developing Solutions: Collaborate to create solutions and provide guidance. • Education and Implementation: Educate stakeholders on the solutions to ensure consistent implementation. Feedback Loop: Establish a feedback mechanism to learn from and adapt to any failures.

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Special Consideration: • Acknowledge the specific needs and challenges in pediatric drug development.

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Paediatric Drug Development Landscape

• Paediatric drug development takes place in the rare disease space , is highly regulated and a global enterprise , with not only the patient but also the parent in mind.

• Growing pipelines of innovative products :

• How can we identify and support completion of development efforts in children for products that address existing unmet medical needs ? • There is a need to foster an innovative R&D environment that allows for the evolution of scientific knowledge and considers changing evidence and unmet needs – rational behind stepwise PIP • Regulatory decision making on mandated paediatric developments cannot occur in isolation and requires acknowledgement of broader implications.

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EMA: Regulatory Decisions On Pediatric Cancer Drugs ‘Cannot Happen In Isolation’

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