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Office of Orphan Products Development (OOPD)

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Office of Orphan Products Development (OOPD) Gayatri R. Rao, M.D., J.D. Acting Director NORD Corporate Council May 15, 2012 OOPD Updates Orphan Drug Designation ... – PowerPoint PPT presentation

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Title: Office of Orphan Products Development (OOPD)


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
  • Whats On the Horizon?

3
Orphan Designation Highlights
3740 Designation requests 2600 Products have
received Orphan Designation (70)
Number of Designation Requests
Number of Orphan Designations
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
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
Year
4
Orphan Drug Approval Highlights
FY 2011 26 approvals Total (since 1983) 400
5
Orphan Drug Designation Program
  • Drugs Approved in 2012 that were also Designated
  • Glucarpidase
  • Ivacaftor
  • Mitomycin C
  • Mifepristone
  • Pazopanib
  • Everolimus
  • Taliglucerase

1/1/2012 To Date
of Designation Requests Received 82
of Designations Granted 54 (majority recd in 2011)
of Drugs Approved for Rare Diseases 8 7 designated 1 funded by OPG pgm
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)
Designations
Approvals
Number of Designations and Approvals
0-9
10-19
20-29
30-39
40-49
50-59
60-69
70-79
80-89
90-99
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
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 gt 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
Devices labeled for peds Berlin Heart EXCOR
Pediatric Ventricular Assist Device (VAD) NeuRx
DPS, Diaphragm Pacing System Elana Surgical Kit
Total 2011
of HUDs Received 283 21
of HUDs Designated 183 (65) 17 (majority recd in 2011)
of HDEs Approved 56 6 3 labeled for peds 1 funded by OPG pgm
Source OOPD Database
Berlin Heart EXCOR Pediatric VAD received 3-year
funding for 1.19M which funded a pivotal study
in support of HDE approval
  • 2012 To date
  • 11 HUDs received
  • 3 HUDs designated

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
  • gt130 pediatric devices evaluated and/or assisted
  • Currently fund 5 consortia
  • Drs. Geiger and Muelanaer and the University of
    Michigan Pediatric Medical Device Institute
    Pediatric Device Consortium
  • Dr. Harrison and the UCSF Pediatric Device
    Consortium
  • Dr. Boyan and the Atlanta Pediatric Device
    Consortium
  • Dr. del Nido, M.D. and the Pediatric
    Cardiovascular Device Consortium
  • 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
Whats 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.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
Whats 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
30th Anniversary of the ODA!
17
Questions?
  • For more information on OOPDs programs, check
    out
  • www.fda.gov/orphan
  • Still have questions?
  • Email us at orphan_at_fda.hhs.gov OR
  • Call us at 301-796-8660
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