* Your assessment is very important for improving the workof artificial intelligence, which forms the content of this project
Download Stephen Smith Medidata Solutions Worldwide
Survey
Document related concepts
Adherence (medicine) wikipedia , lookup
Compounding wikipedia , lookup
Drug interaction wikipedia , lookup
Drug discovery wikipedia , lookup
Drug design wikipedia , lookup
Orphan drug wikipedia , lookup
Electronic prescribing wikipedia , lookup
Neuropharmacology wikipedia , lookup
Prescription drug prices in the United States wikipedia , lookup
Pharmacokinetics wikipedia , lookup
Pharmaceutical industry wikipedia , lookup
Transcript
21st Century Cures Legislation: Modernizing Drug Development in Pursuit of Cures How Laws are Made. House, Senate, President Stephen Smith Chief Patient Advocate Medidata Solutions Worldwide, Volunteer Committee on Federal Legislation: National MPS Society Email: [email protected] Twitter: @SteveSmithPlans 1 Since the 1970s only 500 treatments have been approved for 7000 rare diseases The federal drug and device approval apparatus is in many ways the relic of another era. How Laws are Made. House, Senate, President The 30th of April 2014 3 TimeLine: 21st Century Cures May 2014: start cross-stakeholder discussion May 2015: Passed 51-0 out of Energy & Commerce Committee: Unanimous! July 2015 Passed U.S. House 344-77: strong bipartisan vote! Fall 2015: in Senate HELP Committee. Contact your Senators! Late 2015/Early 2016: POTUS Signs? 21st Century Cures July 2015: “Never before have we had such a clear, crossstakeholder definition of what is wrong with drug development and what we need to do to fix it. “ Stephen Smith, Patient Advocate since 1990 2012 Patient-Advocacy-Driven Changes 6 • Removing barriers to increased research collaboration. • Incorporating the patient perspective into the drug development and regulatory review process. • Measuring success and identifying diseases earlier through personalized medicine. • Modernizing clinical trials. • Removing regulatory uncertainty for the development of new medical apps. • Providing new incentives for the development of drugs for rare diseases. • Helping the entire biomedical ecosystem coordinate more efficiently to find faster cures. • Investing in 21st Century science and next generation investigators. • HR 6 helps keep and create jobs here at home. Highlights of 21st Century Cures Creating a 21st Century Data Infrastructure Accelerated Approvals Incorporate Patient’s Risk Vs. Benefit Validate biomarkers Conduct long-term, natural history studies Incentives to focus on higher hanging fruit Data Sharing, Re-use, from new sources Use of Apps Use of mHealth devices Use of data: Real time, patient reported, EHR, Medicare Example: Three Incentives in 21st Century Cures The Creating Hope Act – up for renewal Rare Pediatric Disease Voucher reduces FDA review by 4 months The Open Act 6 months patent extension for repurposing a big disease drug into a rare disease drug. The Awakening Dormant Therapies Act 6 months patent extension for creating drug from a project that had been shelved Rare Pediatric Disease Voucher Awarded Voucher by FDA For Developing Disease Bought Voucher from Awardee For This Amount BioMarin Vimizim MPS IVa Regeneron $67.5* Asklepion Cholbam Bile Acid Synthesis Disorder Sanofi $245 United Therapeutics Unituxin Neuroblastoma Abbvie $350 * Regeneron used its voucher successfully to speed approval of Praluent a PCSK9 inhibitor for patient with HeFH, or patients with Clinical Atherosclerotic Cardiovascular Disease. Repurpose for Rare Disease Awaken Dormant Projects 11 Google: Twitter: Steve Smith Patient Advocate @SteveSmithPlans Email Full 21st Century Cures Info [email protected] energycommerce.house.gov Contact your U.S. Senators now! Ask them to support 21st Century Cures Senate.gov Aka: Innovation for Healthier Americans 12