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.
Awareness of the vast impact of rare diseases, which affect approximately 300 million people globally (1), has significantly increased over the years, leading to increased funding and investment in rare disease research and therapy development. Although more than half of the novel drug approvals by the FDA’s Center for Drug Evaluation and Research last […]
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