About FDA

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:

http://www.fda.gov/ForIndustry/UserFees/PrescriptionDrugUserFee/ucm327281.htm#contact_information

Not sure who  to call or not quite ready for clinical trials, but have some questions

Contact OND Rare Diseases Program

Lucas Kempf - Lucas.Kempf@fda.hhs.gov
or
CDERONDRareDiseaseProgram@fda.hhs.gov

General Information

Rare Diseases Program
CDER/OND

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)

http://www.fda.gov/Drugs/default.htm

Center for Biologics Evaluation and Research (CBER)

http://www.fda.gov/BiologicsBloodVaccines/default.htm

Center for Devices and Radiological Health (CDRH)

http://www.fda.gov/MedicalDevices/default.htm

Office of Orphan Product Development (OOPD), FDA

Where can I get information about developing products for
rare diseases & conditions?

OOPD home page

http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/default.htm

Orphan Drug Act

http://www.fda.gov/regulatoryinformation/legislation/significantamendmentstothefdcact/orphandrugact/default.htm

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 doeshttp://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 programhttp://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 conditionshttp://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
    

 

Page Last Updated: 12/11/2018
Note: If you need help accessing information in different file formats, see Instructions for Downloading Viewers and Players.
Language Assistance Available: Español | 繁體中文 | Tiếng Việt | 한국어 | Tagalog | Русский | العربية | Kreyòl Ayisyen | Français | Polski | Português | Italiano | Deutsch | 日本語 | فارسی | English