How Laws are Made. House, Senate, President 1 21 st Century Cures Legislation: Modernizing Drug Development in Pursuit of Cures Stephen Smith Chief Patient Advocate Medidata Solutions Worldwide, Volunteer Committee on Federal Legislation: National MPS Society Stephen Smith

Since the 1970s only 500 treatments have been approved for 7000 rare diseases

How Laws are Made. House, Senate, President The federal drug and device approval apparatus is in many ways the relic of another era. The 30 th of April

TimeLine: 21 st 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 : strong bi- partisan vote! Fall 2015: in Senate HELP Committee. Contact your Senators! Late 2015/Early 2016: POTUS Signs?

21 st Century Cures July 2015: “Never before have we had such a clear, cross- stakeholder definition of what is wrong with drug development and what we need to do to fix it. “ Stephen Smith, Patient Advocate since 1990

6 Patient-Advocacy-Driven Changes 2012

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.

Creating a 21 st 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 Highlights of 21 st Century Cures

Example: Three Incentives in 21 st 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

Awarded Voucher by FDA For Developing DiseaseBought Voucher from Awardee For This Amount BioMarinVimizimMPS IVaRegeneron$67.5* AsklepionCholbamBile Acid Synthesis Disorder Sanofi$245 United Therapeutics UnituxinNeuroblastomaAbbvie$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.

11 Repurpose for Rare Disease Awaken Dormant Projects

12 Contact your U.S. Senators now! Ask them to support 21 st Century Cures Senate.gov Aka: Innovation for Healthier Americans Google: Steve Smith Patient Advocate Full 21 st Century Cures Info energycommerce.house.gov