Strategies for Rare Disease Data Analysis – Doing More with Less

What You Will Learn

The pharmaceutical industry’s burgeoning desire to develop rare disease treatments to address significant unmet needs presents many challenges with identifying and utilizing fit-for-purpose data. In addition, patient identification and retention are major hurdles for rare disease development programs. Real-world data (RWD) such as electronic health records (EHR), claims, and genomics can be paired with data visualization and analysis techniques to discover subjects with diagnostic criteria suggestive of a rare disease disorder. Further, descriptive dashboards are useful in determining the viability and value that may come from particular data points collected in a clinical development program. Dashboards can help answer whether the data is fit-for-use, has sufficient quality, and is informative for decision making. Using dashboards from a purpose-built platform like Datacise™ can assist clinicians and other researchers in making key decisions for the planning and evaluation of research trials. Datacise™ has been developed by MMS, a data-focused CRO, as a solution for RWD curations, analytics, and data visualization.

Attendees will learn about:

• The statistical and data analytics landscape related to rare diseases
• Information related to rare disease study design, longitudinal data volume, comparability and appropriateness of using RWD to supplement trial data
• Considerations for data access, quality and standardization
• The selection and definition of informative endpoints -How proper analysis techniques can help accelerate a clinical development program