REPORT

Using real world data to advance precision medicine and help rare disease patients

Recent years have seen rapid advances in medical technology, enabling a surge in the development of drugs and biologics addressing rare diseases and niche patient populations. This movement toward precision medicines faces steep challenges, however – chief among them, that of defining and understanding very small patient populations.

To better understand this critical topic, we convened a panel discussion answering key questions such as

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What are some of the toughest challenges companies face in trying to launch therapeutics for rare diseases?

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Faced with a scarcity of data or gaps in the available data, how can developers proceed?

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Where do these real-world data come from and what insights can they give us into a patient population?

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At what point in the development timeline should companies begin collecting and analyzing this data?

Understanding small patient populations and generating current and precise trends and forecasts is critical to bring life-saving treatments to patients.

We’re blending real world data with epidemiology so that our customers have the greatest accuracy to enable the right investment and drive the strategic business decisions.