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Missing Endpoints

Rare Disease Advocates’ Relevance in Clinical Trial Design and the Consequences of Non-Inclusion
The stakes are extraordinarily high for both orphan drug developers and rare disease communities. Trials often fail to measure functional outcomes that reflect patients’ greatest unmet needs. Failure to identify outcome measures that capture what patients value most contribute to delays, or worse denials, to therapy. Industry-Advocacy partnerships are key to mitigating the risk of missing endpoints. Moving forward it is critical for Industry to adopt internal strategies to fill the empty chair at the table and allow Rare Disease Advocates (RDAs) to influence and support clinical trial design.

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ThinkGen was founded in 2010 and has had a successful history delivering actionable results that help clients thrive in competitive and complex markets. Our team brings together both market research expertise from the vendor side, and long-term marketing and sales experience from the client side.

Contact Us if you are interested in collaborating with RAM members on the development of a whitepaper and/or an initiative that provides real world insights into the various complexities of the rare disease landscape.

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