Policy in Action

1. Policy in Action Philadelphia Regional Membership Meeting Phone 202.588.5700Fax 202.588.5701 Address1779 Mass. Ave. NW, Suite 500, Washington, DC 20036 rarediseases.org

2. Pub. Policy in Lame Duck (Anyone smell something fowl ?)

3. NORD’s Policy Agenda • Looking forward to the 113th Session of Congress • Expansion of Rare Disease focus in Fed. Gov. • Continuing implementation of critical existing laws • PPACA – Patient Protection Affordable Care Act • FDASIA – FDA Safety & Innovation Act • End of year fiscal policy (a big mess) • Budget authority for FY 2013 (CR) • Sequester, Doc Fix, Tax policy

4. Overview of FDASIA • The user fee reauthorization has two parts– • FDA & Industry: technical goals and commitments agreed upon by the Agency to support the new fee schedule • Congress: statutory language that legally authorizes collection of the fees that Industry pays, cites the commitment by FDA, and mandates changes to policy governing the marketing of new medical products

5. FDA’s Goals & Commitments • Enhancing Regulatory Science and Expediting Drug Development • Enhances communication during drug development • Advances the science of meta-analysis methods • Advances the use of biomarkersand pharmacogenomics • Advances development of endpoint assessment tools • Advances the development of drugs for rare diseases • Enhancing Benefit-Risk Assessment in Regulatory Decision-making

6. Rare Disease Expansions at FDA • The Rare Disease Program at the Center for Drugs will be permanently increased by 5 FTEs • Expansion to the Office of Biologics • Rare Disease Council – an Agency wide effort • Other critical regulatory science initiatives of high value to the rare disease community- • Biomarkers, surrogate endpoint qualification • Risk evaluation, increased focus on patient input

7. Benefit/Risk Assessment • Key themes: • Communication to the FDA by patients about the impact of their disease on quality of life • Transparency of review decisions through qualitative and semi-quantitative metrics • Development of the Risk v. Benefit matrix that FDA will use to profile decision on specific therapy indications • FDA to hold 20 topic specific meetings on risk

8. PPACA: Look to the 50 States • HHS has ceded purview on essential health benefits to the 50 states • Get to know your state’s Health office • Regardless of the election’s outcome, consolidation by payors/providers will continue • Unintended consequences will balloon if PPACA repealed; lack of authority or steering • Know more in 2014 when first marketplaces go live (keep your fingers crossed)

9. Essential Health Benefits • Designed to be the floor for coverage along categories of “essential” benefits • Unclear if access to specialists will be req. • Prescription Drug Benefit minimum unknown • Likely will be a state by state decision • Specialty tiering and cost-shares • Self-funded plan governance will continue under the broad protections of ERISA • Downstream influence on larger health plans, more as expansions in 2015, 2016, etc.

10. End of year Fiscal Debacle • ‘Normal’ schedule for FY 2013 Appropriations • Adjustment to the Sustainable Growth Rate • This is the ‘Doc Fix’ • Origins of the Sequester • Impact of the Sequester, should it come to pass • Effect of election politics on brinksmanship • Take Action with NORD! • www.rarediseases.org/advocacy/take-action