FDA Marks Orphan Drug Act Milestone: 30 Year Recognition January 2013

1. FDA Marks Orphan Drug Act Milestone: 30 Year Recognition January 2013

2. One of Many Rare Disease Heroes… Henry Waxman and Orrin Hatch Author and cosponsors of the Orphan Drug Act (ODA)

3. One of Many Rare Disease Heroes… Abbey Meyers Champion for ODA and founder of National Organization for Rare Disorders

4. One of Many Rare Disease Heroes… Media/Entertainment Industry Raising awareness about rare diseases and the need for medical products

5. One of Many Rare Disease Heroes… Rare Disease Advocacy Groups Organization, education, support, research, advocacy, and action

6. One of Many Rare Disease Heroes… FDA Orphan Incentive Programs Advancing orphan products through orphan designations, grants, and facilitation

7. One of Many Rare Disease Heroes… NIH Office of Rare Diseases Research Advancing rare disease information, collaboration, and research

8. One of Many Rare Disease Heroes… Robert Campbell Jr, MD representing Clinician/product developers and advocates; Developed the artificial rib humanitarian use device

9. One of Many Rare Disease Heroes… Hemin and Desiree Lyon representing 1st approved orphan designated drug and patient advocacy

10. One of Many Rare Disease Heroes… Jana Monaco representing Parent advocacy for newborn screening and medical foods

11. One of Many Rare Disease Heroes… Industry-Investors-Academia Developing products in the spirit of the ODA

12. One of Many Rare Disease Heroes… Sharon Terry representing Advocacy for innovative and collaborative approaches for rare disease products

13. One of Many Rare Disease Heroes… NIH Undiagnosed Diseases Program Cross-cutting efforts to understand and treat rare diseases

14. One of Many Rare Disease Heroes… Nancy Wexler, PhD representing Patient advocacy and gene discovery/research

15. One of Many Rare Disease Heroes… Ashley Appell representing Patients living and thriving with a rare disease

16. One of Many Rare Disease Heroes… FDA Offices/Centers Communication, diagnostic and therapeutic product evaluation, safety, and access

17. One of Many Rare Disease Heroes… Myozyme and Tiffany House representing Contributions of patient representatives to the FDA orphan approval process

18. One of Many Rare Disease Heroes… Ronald J. Bartek representing Family member founder of a rare disease advocacy group

19. One of Many Rare Disease Heroes… Pediatric Ventricular Assist Device-Berlin Heart representing ODA Grants and Humanitarian Use Devices for pediatric rare diseases

20. One of Many Rare Disease Heroes… Denise Ney, PhD representing Clinical dietitian/medical foods researcher for metabolic disorders

21. One of Many Rare Disease Heroes… Hal Dietz, MD representing Clinical research in rare diseases

22. One of Many Rare Disease Heroes… FDA/CDER Rare Diseases Program Focused regulatory science and education efforts for rare disease drug development

23. One of Many Rare Disease Heroes… NIH TRND Program Stimulating drug discovery and collaborative preclinical development for rare disease drugs

24. One of Many Rare Disease Heroes… EMA/EURORDIS/ IRDIRC/ICORD representing Collaborative international rare disease efforts

25. One of Many Rare Disease Heroes… Pearl O’Rourke, MD representing IRB efforts on behalf of rare diseases

26. One of Many Rare Disease Heroes… Jouni Uitto, MD, PhD representing Basic research in rare diseases

27. One of Many Rare Disease Heroes… George Harris Carter representing Patient advocacy for drug development and clinical trials

28. One of Many Rare Disease Heroes… Kalydeco representing Personalized medicines for rare diseases

29. One of Many Rare Disease Heroes… Leslie Boyer, MD representing Academic grantees successfully pursuing biological marketing approvals

30. One of Many Rare Disease Heroes… Rodney Howell, MD representing Advocacy for newborn screening/early detection and treatment of congenital disorders

31. FDA Marks Orphan Drug Act Milestone: 30 Year Recognition January 2013