Introducing 1st Rare Disease Advocacy World (RDAW) USA 2015 Meeting
Patient advocacy groups are one of the most important powerful stakeholders that contribute to the success of orphan drug development and changes in policies for global regulatory agencies.
Just recently in September 2014, the European Medicines Agency (EMA) announces the launch of a pilot program to involve patients in the benefit-risk evaluation of medicines at the regulatory agency’s Committee for Medicinal Products for Human Use (CHMP). CHMP is the EMA committee responsible for preparing the agency’s opinions on questions concerning human medicines – it conducts the initial benefit-risk analysis of medicines. In August 2014, Rona Ambrose, Canada’s Minister of Health, announces the launch of a pilot project “ … targeting patient input from Canadians with rare diseases to help inform future reviews of orphan drugs …. The Pilot Project will simulate how input from patients will be gathered and incorporated into the drug submission review process once the Orphan Drug Framework is in effect.” The FDA has started the Patient-Focused Drug Development (PFDD) initiative to obtain and better understand a patient’s perspective about their disease and to get patient feedback on treatments.
In recognition of the power, strength, passion, and compassion of patient advocacy organizations, there is a new portion of the World Orphan Drug Congress USA 2015, devoted to this trend. Rare Disease Advocacy World (RDAW) is a conference of its own which leverages the success of the world’s largest orphan drug event – The World Orphan Drug Congress USA – to grow the breadth and scope of the event even further and embrace and empower rare disease advocacy like never before. RDAW brings stakeholders together at this co-located conference. RDAW will be a 2-day event that will provide opportunities for patient advocacy groups to learn more about:
• Achieving a scientific mindset
• Understanding your disease
• Characterizing your patients
• Developing a strategic plan
• Raising awareness and raising funds.
Several experts in the field will be hosting a series of case studies and workshops:
• Pat Furlong, President & CEO, Parent’s Project Muscular Dystrophy (PPMD)
• Nancy Goodman, Founder & Executive Director, Kids V Cancer
• Eric Hoffman, Director, Research Center for Genetic Medicine, The George Washington University.
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