Rare Action network ®

1. Rare Action network ® Presentation by NORD June 16, 2014

2. Confidential & Proprietary Property of NORD NORD • Leading rare disease patient advocacy organization for > 30 years • Principal resource for federal agencies and corporations when addressing questions or issues concerning the rare disease community • Institutional member of the rare disease community having built great working relationships with patients and patient groups, the FDA, NIH, Biotechnology and Pharmaceutical companies, payers, Medical and Scientific Communities

3. Confidential & Proprietary Property of NORD NORD • Pioneered techniques to champion the rare disease agenda • At the head of the rare disease community’s decision making table • Independent and unbiased advocate for patients with rare diseases and the organizations that support them

4. Confidential & Proprietary Property of NORD What is the Rare Action Network? The Rare Action Networkis NORD’s initiative to mobilize patients around the country to advocate for policies that support the needs of patients with rare diseases. It will predominantly work at the state level, where more and more decisions are being made concerning health care, especially therapies and services for patients with rare diseases.

5. Confidential & Proprietary Property of NORD Goals: • Build a unified network of rare disease advocates to take action on issues that impact patients with rare diseases. • Increase awareness of the challenges faced by the rare disease community to those that can take action to make essential changes. • Address issues of access and coverage to essential treatments and therapies for patients with rare diseases at the state level.

6. Confidential & Proprietary Property of NORD Importance of Being “In the States” • States play a large role in determining which healthcare services and treatments patients with rare diseases have access to • The Affordable Care Act added a layer of complexity • Need to maintain a pulse on patient’s experiences • Federal policy advocacy can be guided by on the ground affordability and access to treatments issues • Better prepared to translate state-based issues to federal action when appropriate

7. Confidential & Proprietary Property of NORD Members of the Rare Action Network ®

8. Confidential & Proprietary Property of NORD NORD’s Network: Over 1,500 members NORD has a presence across the entire U.S. through Patient Organization Members (blue pins) Corporate Council Members (green pins) Individual Members and Activists that support NORD’s mission (red pins).

9. Confidential & Proprietary Property of NORD Rare Disease Day Advocacy • NORD’s State House Project has tripled in impact since its launch in 2012. • State House Events took place in 12 states in 2014. • RDD Advocacy directed at officials took place in 25+ states.

10. Confidential & Proprietary Property of NORD NORD Support for the Network • Connecting, educating and training advocates • Regional Meetings • Webinars • Toolkits • Data collection & management • Issue Management Reporting System • Coverage Denial Reporting System • Develop state policy agenda & mobilize network • Written correspondence with elected state officials • Lobby days in State Capitols • Coalition building

11. Confidential & Proprietary Property of NORD Role of members Rare Action Network ®

12. Confidential & Proprietary Property of NORD Help Grow and Build the Network Advocacy is most successful when rare disease interest groups are unitedand bandtogether to circulate common messages and calls for action. • Grow the network by reaching out to your communities • Collaborate and partner with NORD and other members of the network • Raise awareness and recognition

13. Confidential & Proprietary Property of NORD Data Collection & Management Data = Substantiation • NORD will ask members to report issues to determine: • What are some of the immediate needs/problems we’re aware of? • Where are they taking place? • Who is being impacted by them? • Is anyone actively seeking to address this issue? If yes, who? • NORD will survey issues, track trends, provide reports and help mobilize action

14. Confidential & Proprietary Property of NORD State Policy Report Card • Establish the structure and key indicators to be used • Assess both positive and negative state policies • Grade states on their performance • Identify best practices and work with the network to advocate their adoption, or similar, in other states

15. Confidential & Proprietary Property of NORD Education for State Officials • Work with network to develop and distribute educational materials to introduce state officials to rare diseases and salient policies that affect populations • Departments of Insurance, Medicaid Agencies, State Health Insurance Marketplaces, and more. • Support key officials in drafting pro-rare disease policies • Continue education through webinars and briefings

16. Confidential & Proprietary Property of NORD Direct Advocacy • Advocatefor pro-rare disease policies on both the state and federal levels using the following avenues: • In-person lobbying and advocacy • Written Correspondence • Coalition Building • Multi-state campaigns

17. Confidential & Proprietary Property of NORD infrastructure Rare Action Network ®

18. Confidential & Proprietary Property of NORD

19. Confidential & Proprietary Property of NORD State Ambassadors • State Ambassadors • Lead RAN representative for their state • Help new advocates join the network in your state • Work with NORD’s regional managers to identify best practices and issues faced by patients in your state • Represent your state in discussions on content for educational materials and in the development of programs for regional meetings • Participate in legislative campaigns and attend lobby days • Help mobilize your state for successful advocacy • Deputy State Ambassadors • Support the coordination of efforts with your State Ambassador

20. Confidential & Proprietary Property of NORD 2014 Timeline

21. Confidential & Proprietary Property of NORD Questions?

22. Confidential & Proprietary Property of NORD Lisa Phelps, MPH Director of Membership lphelps@rarediseases.org (203) 744-0100 Paul Melmeyer Assistant Director of Public Policy pmelmeyer@rarediseases.org (202) 588-5700 rarediseases.org