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Advancing Rare Disease Treatments Through Innovation and Policy Change

EveryLife Foundation is dedicated to accelerating biotechnology innovation for rare disease treatments. We advocate for scientifically sound policy changes to enhance the efficiency and predictability of drug development. Our mission is to ensure that no disease is too rare to receive treatment, and all therapies must be safe and effective. Successful initiatives show momentum for more legislation supporting rare diseases. By encouraging industry to repurpose existing drugs and creating specialized FDA review divisions, we aim to dramatically increase treatment availability in the coming years.

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Advancing Rare Disease Treatments Through Innovation and Policy Change

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  1. EveryLife Foundation • Dedicated to accelerating biotechnology innovation for rare disease treatments • Advocating practical and scientifically sound change in policy and law to increase the efficiency & predictability of the development process through scientific analysis and dialogue, grassroots support & expert-led workshops • We believe: • No disease is too rare to deserve treatment • All treatments should be safe & effective • We could be doing more with the science we have

  2. Successes in FDASIA showed there is momentum for more rare diseases legislation • Patients are motivated & ready to take action • Rep. Upton is actively seeking proposals to improve FDA, spur drug development & innovation

  3. CureTheProcess – 2Small policy changes that will dramatically increase the availability rare disease treatments in the next 5-10 years • Specialize:Create more specialized FDA New Drug Review Divisions; give reviewers sufficient time and opportunity to stay connected to the scientific and academic community • Rationalize:Allow for a more scientific rationalized application of the ICH guidelines for safety studies • Incentivize: Create an additional market incentive to encourage industry drug sponsors to repurpose major market drugs for rare diseases

  4. The OPEN ACTOrphanProductExtensionsNowAcceleratingCures&Treatmentswww.CureTheProcess.orgwww.EveryLifeFoundation.org Be Sure to Follow us • http://www.facebook.com/EveryLife4RareDiseases • http://twitter.com/#!/curetheprocess

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