PPT-The Rare Disease Treatment Approval Process:

Balancing Gold Standard Evidence with PatientCentered Flexibility Sarah L Wicks JD MPH University of Minnesota Rare Disease Day 2020 February 28 2020 1 Overview FDAs Gold Standard for Approval. “. EMERGING THERAPIES FOR RARE DISEASES. ”. Emil D. Kakkis, M.D., Ph.D. .. President and . Founder. CENTER FOR ORPHAN DISEASE RESEARCH . AND . THERAPY SYMPOSIUM. FRIDAY . MAY 2, 2014. Batten Disease. June 29-30, 2010 . FDA Public Meeting. “Considerations regarding the review and . regulation of articles for treatment of rare diseases”. Tracy VanHoutan, . Board Member . of the Batten Disease Support and Research Association (BDSRA). DISEASES. “ARE WE READY FOR THE CHANGE THAT WE NEED TO FIND EFFECTIVE TREATMENTS FOR ORPHAN DISEASE?”. 2. YES!. Rare disease is very ‘popular’!. Rare disease <200,000 Americans have disease. 30-Year History . Since 1983, NORD has ensured that the rare disease patient voice has been at the table . when . important . federal policy . and regulatory . decisions are made. NORD . recognized the need to build a network of rare disease advocates across the U.S. to unite and mobilize patients . Technical Assistance. If . you encounter problems during this training session, call WebEx Technical Support at 1-866-229-3239 and provide the session ID.. If you are unable to join the audio conference, call Global Crossing Ready-Access audio conferencing at 1-800-788-6092.. Marlene E. Haffner, MD, MPH. CEO, Haffner Associates, LLC. Orphan Drugs Summit 2012. Thursday, 27. th. September 2012. Current . Pharma. Trends. Slow . pharma. industry growth. Patent expiration. Issues in Clinical Trial Design for Rare Diseases. Jonathan C. Goldsmith, MD, FACP. Associate Director Rare Diseases Program/Office of New Drugs. Center for Drug Evaluation and Research. FDA. November 7, 2016. 1 / 3 ‘ New scientific paper confi rms 300 m illion people living with a rare disease worldwide Paris, 17 September 2019 - A new scientific paper confirms the number of people living with a rar biomedical R&D industry. CHI represents more than 275 leading medical device, biotechnology, diagnostics and pharmaceutical companies and public and private academic biomedical research organizations ARTICLEAdvancements in science and technology have helped researchers develop new treatments for some of the most common diseases known to man. Diseases that were once considered death sentences are n Rare Disease DayFrequently Asked QuestionsWhat is a rare disease The most complete listing rarediseasesinfonihgov/diseasesWho is affected by rare diseasesManybut not allrare diseases are genetic Some Overview. Milen Minkov M.D., . Ph.D. .. Full. Professor . of. . P. ediatrics. at Sigmund Freud Private University . Director. . of. Pediatric Services at . Rudolfstiftung Hospital. Head, International LCH Study Reference Center at. begs the question: how long will it take other countries to similarly oer access to genomic medicine? What key barriers exist to the implementation of genomic medicine?Given the current disparities i Goals of the diagnosis survey. Measuring the . time necessary to obtain a diagnosis. as a rare disease patient. Understand the . different steps. of the diagnosis journey . Define the . obstacles limiting the access .