With the advancement of precision medicine, genetic stratification – dividing patients into subgroups based on their specific mutations – is a key starting point in the drug development lifecycle for rare genetic diseases.
Incorporating a clear stratification plan is a key part of developing an effective Target Product Profile (TPP), which serves as a blueprint for product development by defining what “success” looks like early on, and aligning clinical, regulatory, and commercial strategies to meet patient needs, and regulatory and market requirements.
Rare Disease Day presents a time to reflect on the progress made in understanding and addressing the unique challenges that rare diseases bring – and the hurdles that remain. Over the past year, several significant developments have shaped the rare disease landscape, from new regulatory policies and technological advancements to major advocacy efforts.
Rare genetic diseases are challenging to treat due to their complexity, the unique nature of each condition, and the – by definition – small numbers of patients affected by each one. Encouragingly for the rare disease community, the field is moving rapidly with a wide variety of innovative therapies being developed, including gene therapy involving […]
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