1. Office of Orphan Products Development (OOPD) Gayatri R. Rao, M.D., J.D. Acting Director NORD Corporate Council May 15, 2012

2. OOPD Updates Orphan Drug Designation Program Orphan Products Grants Program Humanitarian Use Device (HUD) Designation Program Pediatric Device Consortia Grants Program Outreach Efforts What’s On the Horizon?

3. Orphan Designation Highlights ~3740 Designation requests ~2600 Products have received Orphan Designation (~70%) 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09 10 11 Year Number of Designation Requests Number of Orphan Designations

4. Orphan Drug Approval Highlights FY 2011 – 26 approvals Total (since 1983) – 400 Number of Approvals 83 84 85 86 87 88 89 90 91 92 93 94 95 96 97 98 99 00 01 02 03 04 05 06 07 08 09 10 11 Year

5. Orphan Drug Designation Program 1/1/2012 – To Date # of Designation Requests Received 82 # of Designations Granted 54 (majority rec’d in 2011) # of Drugs Approved for Rare Diseases 8 7 designated 1 funded by OPG pgm Drugs Approved in 2012 that were also Designated 1.Glucarpidase 2.Ivacaftor 3.Mitomycin C 4.Mifepristone 5.Pazopanib 6.Everolimus 7.Taliglucerase Ivacaftor received 1-year funding from OPG program for $350K for a phase 2, RCT assessing safety, tolerability, PK and PD; results were submitted in support of approval Source: OOPD Database

6. Size of Populations (1983-2011) Number of Designations and Approvals 0-910-1920-2930-3940-4950-5960-6970-7980-8990-99 100- 109 110- 119 120- 129 130- 139 140- 149 150- 159 160- 169 170- 179 180- 189 190- 199 200 & up US prevalence (in thousands) of diseases for which products received an orphan designation Designations Approvals

7. Orphan Drug Regulations Proposed rule issued on Oct. 19, 2011 – (76 Fed. Reg. 64868) –Received 13 comments Clarify existing regulations Goal is to finalize rule in 2012

8. Orphan Products Grants Program Current annual budget is $14M –No increase in annual budget since 2005 Funded studies contributed to > 45 marketing approvals

9. Orphan Products Grants Program Overall: ~2000 applications, over 500 studies funded –FY 2011: Received 94 applications, funded 14 (~15%) –FY 2012: Received 125 applications, anticipate funding 5-10 new awards

10. HUD Designation Program 2012 – To date 11 HUDs received 3 HUDs designated Total2011 # of HUDs Received 28321 # of HUDs Designated 183 (65%) 17 (majority rec’d in 2011) # of HDEs Approved 566 3 labeled for peds 1 funded by OPG pgm Devices labeled for peds Berlin Heart EXCOR® Pediatric Ventricular Assist Device (VAD) NeuRx DPS™, Diaphragm Pacing System Elana Surgical Kit Berlin Heart EXCOR® Pediatric VAD received 3-year funding for ~$1.19M which funded a pivotal study in support of HDE approval Source: OOPD Database

11. HUD Designation Program Draft HUD guidance issued Dec. 2011, responsive in part to Sec. 740 Report and the 2010 IOM Report –Received 5 comments –Goal is to finalize guidance this year Pediatric Medical Devices –GAO Report – Pediatric Medical Devices Pre-2007 – FDA received & designated on average 1HUD/year Post-2007 – FDA received & designated on average 5 HUDs/year –Whether the profit incentive for HDE should be expanded?

12. Pediatric Devices Consortia Grants Program Total $8 million awarded since 2009 –>130 pediatric devices evaluated and/or assisted Currently fund 5 consortia 1.Drs. Geiger and Muelanaer and the University of Michigan – Pediatric Medical Device Institute Pediatric Device Consortium 2.Dr. Harrison and the UCSF Pediatric Device Consortium 3.Dr. Boyan and the Atlanta Pediatric Device Consortium 4.Dr. del Nido, M.D. and the Pediatric Cardiovascular Device Consortium 5.Dr. Garcia and the MISTRAL Pediatric Device Consortium Pediatric Devices Consortia Workshop – September 24, 2012, at FDA “Programs such as the pediatric device consortia can foster an environment for device innovators to share ideas and advance the development of pediatric medical devices.” -- GAO Report, December 2011

13. Outreach Efforts FDA Rare Disease Patient Advocacy Day –Held Mar. 1, 2012 –~400 participants (on-site + webinar) –Engage with patient advocates re FDA regulatory process Future? –Brown bag, focused webinars –Once every leap year

14. What’s on the Horizon Workshop on NH Studies of Rare Diseases –May 16-17, 2012, at NIH –https://www.team- share.net/Natural_History_Studies_Rare_Diseases/ overview.aspxhttps://www.team- share.net/Natural_History_Studies_Rare_Diseases/ overview.aspx The Science of Small Clinical Trials Workshop –November 27-28, at FDA Orphan Drug/HUD Designation and Orphan Grants Workshop (co-sponsored with EMA) –October 12, 2012, at FDA Regulatory Science

15. What’s on the Horizon Potential legislative changes IRDiRC –Joined the Steering Committee FDA Rare Disease Council –Consists of OOPD, CDER, CBER, CDRH, OSHI, and CFSAN –Coordinate and collaborate on matters related to rare diseases

16. 30 th Anniversary of the ODA!

17. Questions? For more information on OOPD’s programs, check out www.fda.gov/orphan Still have questions? Email us at orphan@fda.hhs.gov ORorphan@fda.hhs.gov Call us at 301-796-8660