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Rare Diseases and FDASIA Gayatri R. Rao MD, JD Director Office of Orphan Products Development (OOPD), FDA Rare Disease Congressional Caucus Briefing November.

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Presentation on theme: "Rare Diseases and FDASIA Gayatri R. Rao MD, JD Director Office of Orphan Products Development (OOPD), FDA Rare Disease Congressional Caucus Briefing November."— Presentation transcript:

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

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: nizationCharts/UCM pdf OOPD Office of Orphan Products Development CDERCBER CDRH Center for Devices and Radiological Health CTP Center for Tobacco Products OSMP Office of Special Medical Programs RDP Rare Diseases Program

3 For Complete FDA Organizational Chart see: /OrganizationCharts/UCM 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

4 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

5 Orphan Drug Highlights Designations ~ 3700 Designation requests received ~ 2550 Products granted designation (~70%) Designations Approvals Year Number of Orphan Designations and Approvals Approvals >400 Drugs brought to market FY 2011 – 26; FY 2012 (to date) – 17

6 Size of Populations ( ) Number of Designations and Approvals & up US prevalence (in thousands) of diseases for which products received an orphan designation Designations Approvals

7 HUD Designation Program 2012 – To date 20 HUDs received 12 HUDs designated Total2011 # of HUDs Received # of HUDs Designated 183 (65%) 17 (majority rec’d in 2011) # of HDEs Approved 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

8 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

9 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

10 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

11 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

12 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, Kicked off bi-monthly process consultation meetings –Oct. 25, 2012 – Open public meeting to discuss the proposed set of disease areas

13 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”

14 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

15 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

16 Questions? For more information on OOPD’s programs, check out


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