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.

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The Rare Advocacy Movement (RAM) is a group of patient advocacy professionals dedicated to the overall advancement of the rare disease patient advocacy professional ecosystem and landscape. Membership and participation in RAM is subject to the observance of the established Code of Conduct for Membership ("Code of Conduct") and the acceptance of the established and adopted RAM Process Document. All members and affiliates of RAM vow to abide by the established Code of Conduct. Any RAM member or affiliate who may violate the Code of Conduct in parts and/or in its entirety will be subject to immediate removal from the RAM membership and/or affiliation.