Pharmaceutical Research and Manufacturers of America (PhRMA) has teamed up with Genetic Alliance, an advocacy group dedicated to transforming health through genetics, to explore a crowd-sourcing approach to supporting patient-focused drug development.

Genetic Alliance has selected advocacy organisations representing three disease areas around which the US Food and Drug Administration (FDA) will hold patient-focused drug development public meetings during 2014 and 2015.

Patient communities in these disease areas will pilot a crowd-sourcing approach, using Genetic Alliance’s Platform for Engaging Everyone Responsibly (PEER), to gathering input from a diverse set of patients on key benefit-risk issues.

PDUFA efforts

The PhRMA-Genetic Alliance initiative is designed to complement ongoing efforts to drive patient-focused drug development under the Prescription Drug User Fee Act (PDUFA).

Title I of the Food and Drug Administration Safety and Innovation Act of 2012, which authorised PDUFA for the fifth time, sets out various performance goals for the FDA during fiscal years 2013 to 2017.

These include a more systematic approach to obtaining patient perspectives on disease severity and currently available treatments in certain areas.

The idea is to help improve benefit-risk assessments in the FDA’s regulatory decision-making by establishing the context in which particular decisions are made.

Community engagement

Genetic Alliance has chosen the following organisations to engage their respective patient communities:

  • Sickle cell disease: North Alabama Sickle Cell Foundation; Sickle Cell Disease Association of America, Southern Connecticut; Sickle Cell Warriors; The William E. Proudford Sickle Cell Fund.
  • Idiopathic pulmonary fibrosis: The Coalition for Pulmonary Fibrosis.
  • Irritable bowel syndrome, gastroparesis and gastroesophageal reflux: The Gastroparesis Patient Association for Cures and Treatments; Share and Care Cockayne Syndrome Network.

In partnership with Private Access, which develops participant-centric access controls and privacy management systems, Genetic Alliance will provide the participating organisations with a customised version of the PEER system for systematic collection of patient perspectives on the severity and unmet medical needs of their condition.