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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary Appendix C Resources A number of resources mentioned throughout the workshop are available on the internet. The following list is provided as a starting point for those interested in the development of drugs for rare and neglected diseases. The Forum does not endorse any particular programs, publications, or websites. ORGANIZATIONS AND INITIATIVES National Organization for Rare Disorders (NORD) http://www.rarediseases.org/ FasterCures, The Center for Accelerating Medical Solutions http://www.fastercures.org/ See white paper: Entrepreneurs for Cures: The Critical Need for Innovative Approaches to Disease Research, May 30, 2008. http://www.fastercures.org/objects/pdfs/white_papers/FastercuresWP_Innovation_052808.pdf Drugs for Neglected Diseases initiative (DNDi) http://www.dndi.org/ U.S. FOOD AND DRUG ADMINISTRATION (FDA) FDA http://www.fda.gov/
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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary FDA Office of Orphan Products Development http://www.fda.gov/orphan/ Funding FDA Orphan Products Grants Program http://www.fda.gov/orphan/grants/index.htm FDA Cooperative Research and Development Agreements http://www.fda.gov/oc/ofacs/partnership/techtran/default.htm Guidance for Industry Fast Track Drug Development Programs—Designation, Development, and Application Review http://www.fda.gov/cder/guidance/5645fnl.pdf Formal Meetings with Sponsors and Applicants for PDUFA Products http://www.fda.gov/cder/guidance/2125fnl.pdf E 10 Choice of Control Group and Related Issues in Clinical Trials http://www.fda.gov/cder/guidance/4155fnl.pdf Special Protocol Assessment http://www.fda.gov/cder/guidance/3764fnl.PDF Code of Federal Regulations (CFR) and Federal Food, Drug, and Cosmetic Act (FFDCA) Title 21: Food and Drugs (21 CFR) Chapter I: Food and Drug Administration, Department of Health and Human Services (Parts 1–1299) http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/cfrsearch.cfm 21 CFR § 316 Orphan Drugs http://www.accessdata.fda.gov/scripts/cdrh/cfdocs/cfcfr/CFRSearch.cfm?CFRPart=316 Priority Review and Priority Review Voucher, FFDCA, Section 524 http://www.fda.gov/opacom/laws/fdcact/fdcact5b.htm
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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary NATIONAL INSTITUTES OF HEALTH (NIH) NIH Office of Rare Diseases http://rarediseases.info.nih.gov/ Funding NIH National Center for Research Resources (NCRR), Clinical and Translational Science Awards (CTSA) http://www.ncrr.nih.gov/clinical_research_resources/clinical_and_translational_science_awards/ Small Business Innovation Research (SBIR) and Small Business Technology Transfer (STTR) Programs http://grants1.nih.gov/grants/funding/sbirsttr_programs.htm Guidelines and Standardized Agreements Uniform Biological Material Transfer Agreement (UBMTA) http://www.autm.net/aboutTT/aboutTT_umbta.cfm Principles and Guidelines for Recipients of NIH Research Grants and Contract on Obtaining and Disseminating Biomedical Research Resources http://www.ott.nih.gov/policy/reasearch_toolarchive.html EXAMPLES OF DATA AND MATERIALS SHARING REPOSITORIES NIH AIDS Research and Reference Reagent Repository https://www.aidsreagent.org/Index.cfm MR4: Malaria Research and Reference Reagent Resources Program http://www.mr4.org/ BEI Resources: Biodefense and Emerging Infectious Diseases Research Resources Repository http://www.beiresources.org/ ScienceCommons Biological Materials Transfer Project http://sciencecommons.org/projects/licensing/ LONI Image Data Archive https://ida.loni.ucla.edu/login.jsp?project=ADNI%2f
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Breakthrough Business Models: Drug Development for Rare and Neglected Diseases and Individualized Therapies - Workshop Summary Genetic Alliance BioBank http://biobank.org INTELLECTUAL PROPERTY MANAGEMENT Socially Responsible Licensing at Berkeley—Humanitarian Use Clauses in Contracts http://ipira.berkeley.edu/—then “Socially Responsible IP Management” link CLINICAL TRIALS Small Clinical Trials: Issues and Challenges (IOM report, full text free online) http://www.iom.edu/CMS/3740/5483.aspx ClinicalTrials.gov Registry http://clinicaltrials.gov/ Declaration of Helsinki, the World Medical Association http://www.wma.net/e/policy/b3.htm
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