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INFOGRAPHIC: Seizing a Rare Opportunity - A Current Snapshot of the Rare Disease Community
Logan Fleck (Sept 25, 2014)
People living with rare diseases face unique obstacles that stand in the way of their treatment. Because a rare disease affects less than 200,000 Americans, these diseases can go largely unrecognized ‐ even amongst health care providers. Technological developments and the expansion of online communities are helping to raise awareness by providing new opportunities for networking and valuable resources for patients and professionals alike.

Tracking down participants for clinical trials can be difficult and trials often occur far from patients’ homes, but drug developers are beginning to use online sharing and social media to spread the word to target communities. Individuals within these communities are also spreading the word and helping to increase involvement in trials where new treatments are in high demand. Because only about 400 approved treatments exist for rare diseases, there is pressure for new medicines to be produced quickly and most development timelines are condensed. The drugs that meet FDA criteria are released and provide great relief to a small market. However, the shortened timeline can increase the opportunity for error to occur outside of the trial and limits the amount of time for observations to ensure a drug’s maximum efficacy.
Related Conference: NORD's Rare Diseases and Orphan Products Breakthrough Summit

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