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Nov 5, 2013

Genetic Alliance & PhRMA Partner to Drive Patient-Focused Drug Development

  • The Genetic Alliance and the Pharmaceutical Research and Manufacturers of America (PhRMA) started an initiative to explore the use of a technology-enabled, crowdsourcing approach to patient engagement as a complement to ongoing patient-focused drug development efforts under the Prescription Drug User Fee Act (PDUFA V).

    As part of the reauthorization of PDUFA, the FDA committed to gain the patient perspective on 20 disease areas in public meetings to be held between 2012 and 2017.

    “PhRMA is fully supportive of FDA’s efforts to advance patient-focused drug development and seeks to contribute additional patient-driven data that reflect a range of patient perspectives on issues such as severity of conditions and unmet medical needs over time,” said Sascha Haverfield, Ph.D., vp of scientific and regulatory affairs at PhRMA.

    After issuing a Request for Proposals, Genetic Alliance chose advocacy organizations representing three disease areas that will be the focus of FDA patient-focused drug development public meetings in 2014 and 2015. The patient communities in these three disease areas will utilize a crowdsourcing, technology-enabled method to gathering input from a diverse set of patients on key benefit-risk questions.

    “Using the Platform for Engaging Everyone Responsibly (PEER), there is an opportunity to demonstrate the power of a secure, crowdsourced approach to provide additional insight into patients’ experience with a disease or condition,” explained Sharon Terry, president and CEO of Genetic Alliance. “The organizations we selected are experts at broad and diverse engagement from the very people that have a vested interest in patient-focused drug development. We are excited to engage these communities.”

    The following organizations were selected to engage the sickle cell disease community: North Alabama Sickle Cell Foundation, Sickle Cell Disease Association of America-Southern Connecticut, Sickle Cell Warriors, and the William E. Proudford Sickle Cell Fund.

    The Coalition for Pulmonary Fibrosis was chosen to engage the idiopathic pulmonary fibrosis community.

    The Gastroparesis Patient Association for Cures and Treatments, and Share and Care Cockayne Syndrome Network were selected to engage the irritable bowel syndrome, gastroparesis, and gastroesophageal reflux community.

    Genetic Alliance will provide the participating organizations with a customized version of the PEER system, through which they will engage in a systematic collection of affected individuals’ perspectives on a condition’s severity and current unmet medical needs, according to Terry, who added that Private Access is Genetic Alliance’s technology partner for this project.



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