The following links may be useful to those working toward the development of new medicines for Orphan, Neglected and Tropical Diseases.
Orphan Drug Application process
Orphan designation can be requested for a drug or biological product to treat certain diseases or conditions that meet criteria specified by the FDA. Receipt of such designation confers specified benefits on the sponsor, including tax credits for clinical testing, waiver of the prescription drug user fee at the time of submission of a request for marketing authorization and FDA guidance during clinical development.
For more information, see: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/default.htm
FDA Guidance on the process: https://www.fda.gov/Drugs/GuidanceComplianceRegulatoryInformation/Guidances/ucm423881.htm
The EMEA has a similar program, also with benefits for the sponsor. See the Common EMEA/FDA application form: Commom EMEA:FDA ODA
Tips: Tips for Submitting an Application for Orphan Designation
Orphan Products Grants Program
The FDA’s Orphan Products Grants Program is designed to encourage the development of drugs to treat rare or orphan diseases or conditions. Especially sought are treatments for diseases that presently have no therapy or for which the new treatment would be significantly superior to existing therapy. For information, forms and submission deadlines for applications, see: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/WhomtoContactaboutOrphanProductDevelopment/default.htm
Tropical Disease Priority Review Voucher Program
A Tropical Disease Priority Review Voucher is a “reward” that may be obtained by a sponsor filing an NDA for any of several designated Tropical Diseases, presently including: Tuberculosis, Malaria, Blinding trachoma, Buruli Ulcer, Cholera, Dengue/Dengue haemorrhagic fever, Dracunculiasis (guinea-worm disease), Fascioliasis, Human African trypanosomiasis, Leishmaniasis, Leprosy, Lymphatic filariasis, Onchocerciasis, Schistosomiasis, Soil transmitted helminthiasis and Yaws. Once obtained, the voucher can be used by the sponsor to obtain a priority review (defined as a review conducted with a PUDUFA goal date of 6 months) of another application. Importantly, a priority review voucher may be transferred from the sponsor who obtains it to another sponsor. This program is intended to encourage the development of new medicines for neglected tropical diseases. Download: https://www.fda.gov/downloads/Drugs/Guidances/UCM080599.pdf
Therapeutics for Rare and Neglected Diseases (TRND)
A program within NIH that provides drug discovery and development expertise to academics and others seeking to develop new medicines for Neglected Diseases. http://www.ncats.nih.gov/research/rare-diseases/trnd/trnd.html
ClinicalTrials.gov web
Registry and results database of publicly and privately supported clinical studies of human participants conducted around the world. https://register.clinicaltrials.gov/
FDA guidance on Investigational New Drug (IND) Applications
Detailed information on the content and format required for the submission of an IND to the FDA. http://www.fda.gov/Drugs/DevelopmentApprovalProcess/HowDrugsareDevelopedandApproved/ApprovalApplications/InvestigationalNewDrugINDApplication/default.htm
Drugs approved by the FDA
Orange Book: Listing of all drugs currently approved for human use by the FDA http://www.accessdata.fda.gov/scripts/cder/ob/default.cfm
Green Book: Listing of all veterinary drugs approved by the FDA http://www.fda.gov/AnimalVeterinary/Products/ApprovedAnimalDrugProducts/UCM2006464
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