These pages bring together concepts from across the various blogs and provide additional references for further information.
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  • Learn more about the definition of a rare disease, how it affects individuals throughout their life, the top needs of the rare disease community worldwide, the key international policy milestones that support these needs, and the particularities of this population in regulatory and HTA discussions.

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  • Learn more about important interventions that support newborn health, including genetic screening and the Kangaroo Mother Care Method.

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  • Learn more about the definition of ‘health technology’ and ‘health technology assessments,’ the importance of involving patients in HTA and how to evaluate the impact of their involvement, and the different ways of involving patients during HTA processes.

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  • Understand the importance of partnering with the patient community across the drug development and access continuum, the involvement of patients in regulatory reviews and health technology assessment dialogues, the various methods for patient involvement, and the guiding principles to support compliant partnering with patient communities.

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