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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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/OrganizationCharts/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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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

OMPTCDER

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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What is the Advantage to Designation?• When a drug or biological product is designated an orphan,

certain financial incentives can flow:– Tax Credits – 50% of clinical trials costs

– Waiver of User Fees - $1.9 M

– 7-year Marketing Exclusivity

• When a device is designated as a HUD, device is eligible for an alternate pathway to market – Humanitarian Device Exemption (HDE)– Exempt from an effectiveness showing; in lieu need only show

probable benefit

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Orphan Drug HighlightsDesignations~ 3700 Designation requests received~ 2550 Products granted designation (~70%)

0

50

100

150

200

250Designations

Approvals

Year

83 84 85 86 87 88 89 90 91 92 93 94 95 9 6 97 98 99 00 01 02 03 04 05 06 07 08 0 9 10 11

Nu

mb

er

of

Orp

ha

n D

esi

gn

atio

ns

an

d A

pp

rova

ls

Approvals>400 Drugs brought to marketFY 2011 – 26; FY 2012 (to date) – 17

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Size of Populations (1983-2011)

0

50

100

150

200

250

300

350

400

450

500

Nu

mb

er

of

De

sig

na

tion

s a

nd

Ap

pro

vals

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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HUD Designation Program

2012 – To date20 HUDs received12 HUDs designated

Total 2011

# of HUDs Received

283 21

# of HUDs Designated

183(65%)

17(majority rec’d in

2011)

# of HDEs Approved

56 63 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

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FDASIA and PDUFA Performance Goals: Provisions Related to Rare Diseases

• Expedited Approval for Serious or Life-Threatening Diseases/ Conditions– Accelerated Approval– Breakthrough Therapies

• External Experts• Patient-Focused Drug Development• Rare Pediatric Disease Voucher

Program• Meetings and Guidance

Development• 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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Expedited Approval for Serious or Life-Threatening Diseases/ Conditions: Accelerated Approval

• Based on a determination that the product has an effect on a:– Surrogate endpoint that is reasonably likely to predict

clinical benefit OR– 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

• Takes into account severity, rarity, or prevalence, as well as lack of alternative treatments

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10

• Designate drug as a breakthrough therapy if:– Intended to treat a serious or life-threatening

disease/condition AND – Preliminary clinical evidence indicates that drug may

demonstrate substantial improvement over existing therapies on 1 or more clinically significant endpoints

• Submit request for designation with original IND or later

• If designated:– Eligible for everything Fast Track receives – 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

External Experts

• Ensure that opportunities exist for consultations with stakeholders for topics, including for example:– Severity of rare diseases– Unmet medical need associated with rare diseases– Willingness and ability of individuals with a rare disease

to participate in clinical trials– Assessment of benefits and risks of therapies to treat

rare diseases

• Develop and maintain a list of external experts for consultation

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Patient-Focused Drug Development• FDA to conduct 20 meetings on different disease

areas to obtain patient perspective on disease severity or unmet medical need– Sept. 24, 2012 – Published a preliminary list of

nominated disease areas and the criteria used for nomination

• Included rare diseases (e.g., sickle cell disease, amyloidosis, hereditary angioedema)

– Oct. 10, 2012 - Kicked off bi-monthly process consultation meetings

– Oct. 25, 2012 – Open public meeting to discuss the proposed set of disease areas

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13

Rare Pediatric Disease Priority Review Vouchers• Similar to the Tropical Disease Priority Review

Voucher Program

• Uses priority review vouchers as an incentive to develop drugs and biologics for “rare pediatric diseases”

• Authorized until “the last day of the 1-year period that begins on the date that FDA awards the third rare pediatric disease priority voucher”

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Meetings and Guidance• Public Meetings

– Issues in clinical trials for drugs for rare diseases (e.g., endpoint selection, use of surrogate endpoints, patient reported outcomes)

– Ways to encourage/accelerate development of new therapies for pediatric rare diseases

• Guidance Development– 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.)

– FDASIA provisions related to accelerated approval, breakthrough therapies, etc. likewise call for guidance development

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15

Humanitarian Device Exemption

• Expands profit-making to HDE devices that – Don’t occur in pediatrics OR – Occurs in pediatrics in such number that development of

device is “impossible, highly impracticable, or unsafe“

• Changes the definition of annual distribution number (ADN)– Equals the # of devices needed to treat/diagnose/cure

4,000 individuals

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