Rare Diseases Program: Frequently Asked Questions
“Where can I go for help?”
Center for Drug Evaluation and Research (CDER), Office of New Drugs (OND) | ||
|---|---|---|
Where are you in your Development Program? | ||
| Ready/almost ready for clinical trials, IND opened or ready to request a pre-IND meeting | Contact OND review division directly: | |
| Not sure who to call or not quite ready for clinical trials, but have some questions | Contact OND Rare Diseases Program Jonathan Goldsmith - Jonathan.Goldsmith@fda.hhs.gov or Larry Bauer Larry.Bauer@fda.hhs.gov. | |
General Information | ||
Rare Diseases Program | http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm221248.htm | |
Office of New Drugs (OND) | http://www.fda.gov/AboutFDA/CentersOffices/OfficeofMedicalProductsandTobacco/CDER/ucm184426.htm | |
Center for Drug Evaluation and Research (CDER) | ||
Center for Biologics Evaluation and Research (CBER) | ||
Center for Devices and Radiological Health (CDRH) | ||
Office of Orphan Product Development (OOPD), FDA | ||
|---|---|---|
Where can I get information about developing products for | ||
OOPD home page | http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm | |
Orphan Drug Act | ||
Other FDA Resources | ||
|---|---|---|
Cellular and Gene Therapy Products | CBER regulates human gene therapy products - products that introduce genetic material into the body to replace faulty or missing genetic material, thus treating or curing a disease or abnormal medical condition. | http://www.fda.gov/BiologicsBloodVaccines/CellularGeneTherapyProducts/ default.htm |
Drug reviews and approved labels | Reviews of drug applications for approved products, prescribing information (i.e., labeling), and listing of approvals (by month and year) | http://www.accessdata.fda.gov/scripts/cder/drugsatfda/index.cfm |
Enhanced Communication Team | OND office to help with general/jurisdictional questions about the drug development process. Secondary contact for those encountering communication problems with the review team (assists in evaluating the issues and facilitates resolution). | http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ ucm327281.htm |
FDA Basics | Provides information about FDA and what the agency does | http://www.fda.gov/AboutFDA/Transparency/Basics/default.htm |
Information for Healthcare Professionals | http://www.fda.gov/Drugs/ResourcesForYou/HealthProfessionals/default.htm | |
MedWatch | The FDA safety information and adverse event reporting program | http://www.fda.gov/Safety/MedWatch/default.htm |
Office of Health and Constituent Affairs (OHCA) | Information for patients and patient advocates. | http://www.fda.gov/AboutFDA/CentersOffices/OC/OfficeofExternalAffairs/ ucm343095.htm |
Participating in Clinical Trials | Clinical trials are voluntary research studies, conducted in people, designed to answer specific questions about the safety and/or effectiveness of drugs, vaccines, other therapies, or new ways of using existing treatments. | https://clinicaltrials.gov/ |
Expanded Access | Expanded access, also called “compassionate use,” provides a pathway for patients to gain access to investigational drugs, biologics and medical devices for serious diseases or conditions | http://www.fda.gov/ForPatients/Other/ExpandedAccess/ ucm20041768.htm |
FDA Drug Approval Case Study | CDER/PASE has developed a learning tool to advance knowledge, insight and understanding of drug regulatory processes. | http://www.fda.gov/downloads/Training/ForHealthProfessionals/ UCM467907.pdf |
CDER Small Business and Industry Assistance (SBIA) | (SBIA) promotes productive interaction with regulated domestic and international small pharmaceutical business and industry by providing timely and accurate information relating to development and regulation of human drug products. | http://www.fda.gov/Drugs/DevelopmentApprovalProcess/ SmallBusinessAssistance/default.htm |