• Target Product Profiles: How genetic stratification shapes rare disease drug development

    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.