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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 Brought to you by
- 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 Brought to you by
- 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 Brought to you by
- 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 Brought to you by
- 5. Orphan Drug Highlights Designations ~ 3700 Designation requests received ~ 2550 Products granted designation (~70%) 0 50 100 150 200 250 Designations Approvals Year 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 0 9 10 11 NumberofOrphanDesignationsandApprovals Approvals >400 Drugs brought to market FY 2011 – 26; FY 2012 (to date) – 17 Brought to you by
- 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 Brought to you by
- 7. HUD Designation Program 2012 – To date 20 HUDs received 12 HUDs designated Total 2011 # of HUDs Received 283 21 # of HUDs Designated 183 (65%) 17 (majority rec’d in 2011) # of HDEs Approved 56 6 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 Brought to you by
- 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 Brought to you by
- 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 Brought to you by
- 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 Brought to you by
- 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 Brought to you by
- 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, 2012 - Kicked off bi-monthly process consultation meetings – Oct. 25, 2012 – Open public meeting to discuss the proposed set of disease areas Brought to you by
- 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” Brought to you by
- 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 Brought to you by
- 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 Brought to you by
- 16. This platform has been started by Parveen Kumar Chadha with the vision that nobody should suffer the way he has suffered because of lack and improper healthcare facilities in India. We need lots of funds manpower etc. to make this vision a reality please contact us. Join us as a member for a noble cause. Brought to you by