PPT-BioSafe Introduction and rare disease activities

Tim MacLachlan PhD DABT Chair BioSafe Leadership Committee Executive Director Biologics Safety Assessment Preclinical Safety Novartis The opinions expressed in this presentation reflect those of the presenters or speakers only and do not . activities are based. They constitute key instruments for increasing knowledge on Rare Diseases (RD) by pooling data for fundamental and clinical research, epidemiological research, and real-life pos “. 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). Oct. 16th, 2014. The National Association of Insurance Commissioners – Model Insurance regulations – . Joel White, Horizon Government Affairs. 2014 . M. idterm . E. lections Forecasting. . - . Nick Manetto, FaegreBD . 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. Introductions. Introduction to Staff and to RDLA. Monday. : . Rare Disease Day at NIH . . . Cocktail . Reception . & Documentary . Screening. Tuesday. : Legislative Conference. Wednesday. 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 . caregivers, and the dedicated researchers seeking to improve their lives. Through the stories of real people, we present the everyday realities and challen 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 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 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 . to. . care. and . on. European . cooperation. EESC . public. . hearing. :. Ensuring. . strong. European . solidarity. . for. . rare. . disease. . patients. Prof. Dr. . Ildiko. . Horvath. 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 .