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Rare disease caucus
1. Rare Diseases and FDASIA
Gayatri R. Rao MD, JD
Director
Office of Orphan Products Development (OOPD), FDA
Rare Disease Congressional Caucus Briefing
November 14, 2012
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2. Where in the FDA? Office of the
Commissioner
(OC)
Office of Medical
Products and
Tobacco
OMPTOF
Office of Foods
OO
Office of
Operations
OGROP
Office of Global
Regulatory
Operations &
Policy
Center for
Biologics
Evaluation and
Research
Center for
Drug
Evaluation
and Research
For Complete FDA Organizational Chart see:
http://www.fda.gov/downloads/AboutFDA/CentersOffices/Orga
nizationCharts/UCM288864.pdf
OOPD
Office of Orphan
Products
Development
CDER CBER CDRH
Center for
Devices and
Radiological
Health
CTP
Center for
Tobacco
Products
OSMP
Office of Special
Medical Programs
RDP
Rare Diseases
Program
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3. For Complete FDA Organizational Chart see:
http://www.fda.gov/downloads/AboutFDA/CentersOffices
/OrganizationCharts/UCM288864.pdf
Office of the
Commissioner
(OC)
Office of Medical
Products and
Tobacco
OMPT
CDER
Center for Drug Evaluation
and Research
CBER
Center for Biologics
Evaluation and Research
OOPD
Office of Orphan
Products
Development
OSMP
Office of Special
Medical Programs
Step 1:
Orphan Drug
Designation
Humanitarian Use
Device (HUD)
Designation
Step 2:
Marketing Application -
NDA / BLA / HDE
CDRH
Center for Devices and
Radiological Health
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4. What is the Advantage to Designation?
โข When a drug or biological product is designated an orphan,
certain financial incentives can flow:
โ Tax Credits โ 50% of clinical trials costs
โ Waiver of User Fees - $1.9 M
โ 7-year Marketing Exclusivity
โข When a device is designated as a HUD, device is eligible for
an alternate pathway to market โ Humanitarian Device
Exemption (HDE)
โ Exempt from an effectiveness showing; in lieu need only show
probable benefit
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6. Size of Populations (1983-2011)
0
50
100
150
200
250
300
350
400
450
500
NumberofDesignationsandApprovals
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
Designations
Approvals
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7. HUD Designation Program
2012 โ To date
20 HUDs received
12 HUDs designated
Total 2011
# of HUDs
Received
283 21
# of HUDs
Designated
183
(65%)
17
(majority recโd in 2011)
# of HDEs
Approved
56 6
3 labeled for peds
1 funded by OPG pgm
Devices labeled for peds
Berlin Heart EXCORยฎ
Pediatric Ventricular Assist
Device (VAD)
NeuRx DPSโข, Diaphragm
Pacing System
Elana Surgical Kit
Berlin Heart EXCORยฎ Pediatric
VAD received 3-year funding
for ~$1.19M which funded a
pivotal study in support of
HDE approval
Source: OOPD Database
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8. FDASIA and PDUFA Performance Goals:
Provisions Related to Rare Diseases
โข Expedited Approval for Serious or Life-
Threatening Diseases/ Conditions
โ Accelerated Approval
โ Breakthrough Therapies
โข External Experts
โข Patient-Focused Drug Development
โข Rare Pediatric Disease Voucher Program
โข Meetings and Guidance Development
โข Humanitarian Device Exemptions
Reauthorization of
Grants:
1. Orphan Products
Grant Program -
$30M/year
2. Pediatric Devices
Consortia Grant
Program -
$5.25M/year
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9. Expedited Approval for Serious or Life-
Threatening Diseases/ Conditions:
Accelerated Approval
โข Based on a determination that the product has an effect
on a:
โ Surrogate endpoint that is reasonably likely to predict clinical
benefit OR
โ Clinical endpoint that can be measured earlier than irreversible
morbidity or mortality, that is reasonably likely to predict effect
on irreversible morbidity or mortality or other clinical benefit
โข Takes into account severity, rarity, or prevalence, as well
as lack of alternative treatments
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10. 10
โข Designate drug as a breakthrough therapy if:
โ Intended to treat a serious or life-threatening disease/condition
AND
โ Preliminary clinical evidence indicates that drug may
demonstrate substantial improvement over existing therapies on
1 or more clinically significant endpoints
โข Submit request for designation with original IND or later
โข If designated:
โ Eligible for everything Fast Track receives
โ Also get more interactive involvement with review division to
help guide efficient yet scientifically appropriate trial design
Expedited Approval for Serious or Life-
Threatening Diseases/ Conditions:
Breakthrough Therapy
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11. 11
External Experts
โข Ensure that opportunities exist for consultations with
stakeholders for topics, including for example:
โ Severity of rare diseases
โ Unmet medical need associated with rare diseases
โ Willingness and ability of individuals with a rare disease to
participate in clinical trials
โ Assessment of benefits and risks of therapies to treat rare
diseases
โข Develop and maintain a list of external experts for
consultation
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12. Patient-Focused Drug Development
โข FDA to conduct 20 meetings on different disease areas to
obtain patient perspective on disease severity or unmet
medical need
โ Sept. 24, 2012 โ Published a preliminary list of nominated
disease areas and the criteria used for nomination
โข Included rare diseases (e.g., sickle cell disease, amyloidosis,
hereditary angioedema)
โ Oct. 10, 2012 - Kicked off bi-monthly process consultation
meetings
โ Oct. 25, 2012 โ Open public meeting to discuss the proposed set
of disease areas
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13. 13
Rare Pediatric Disease Priority Review Vouchers
โข Similar to the Tropical Disease Priority Review Voucher
Program
โข Uses priority review vouchers as an incentive to develop
drugs and biologics for โrare pediatric diseasesโ
โข Authorized until โthe last day of the 1-year period that
begins on the date that FDA awards the third rare
pediatric disease priority voucherโ
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14. Meetings and Guidance
โข Public Meetings
โ Issues in clinical trials for drugs for rare diseases (e.g., endpoint
selection, use of surrogate endpoints, patient reported outcomes)
โ Ways to encourage/accelerate development of new therapies for
pediatric rare diseases
โข Guidance Development
โ PDUFA Performance Goal (IX.E.): FDA, on an ongoing basis, will
develop guidance to address issues to facilitate development of
drugs for rare diseases (e.g., study design, endpoints, statistical
analysis, etc.)
โ FDASIA provisions related to accelerated approval, breakthrough
therapies, etc. likewise call for guidance development
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15. 15
Humanitarian Device Exemption
โข Expands profit-making to HDE devices that
โ Donโt occur in pediatrics OR
โ Occurs in pediatrics in such number that development of device
is โimpossible, highly impracticable, or unsafeโ
โข Changes the definition of annual distribution number
(ADN)
โ Equals the # of devices needed to treat/diagnose/cure 4,000
individuals
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