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Finding Patients With Rare Diseases: Two Truths and a Lie

Sponsored by 81qd | Mar 1, 2021 8:00am The hope for many companies developing drugs for rare diseases is this: The significant unmet need, given a dearth of treatment options, will drive demand and thus product uptake. Unfortunately, the reality is more challenging. While we have seen a significant increase in the number of drugs for rare diseases, accurate diagnosis rates of rare diseases continue to trail behind those of more prevalent diseases. The average time to diagnosis for a patient with a rare disease is 5 to 7 years. The delay is driven by: Lack of awareness among health care professionals (HCPs) of symptoms and markers

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