• Supporting rare disease patient advocacy with epidemiological insights

    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. Notably, more than half of the novel drug approvals by the FDA’s Center for Drug Evaluation and Research last year […]

  • The role of precision epidemiology in R&D decision-making: Spotlight on rare genetic diseases

    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 […]