FDA’s Center for Biologics Evaluation and Research (CBER) and Center for Drug Evaluation and Research (CDER) initiated the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program to help further accelerate the development of novel drug and biological products for rare diseases. Selected participants will be able to obtain frequent advice and enhanced communication from FDA staff to address program-specific development issues, including but not limited to:

• clinical study design,  
• choice of control group, 
• fine-tuning the choice of patient population, 
• leveraging nonclinical information, and 
• product characterization. 

The pilot was announced in the Federal Register, which included the following eligibility criteria for participation: 

• For both CBER and CDER, the program is open to sponsors of products currently in clinical trials under an active investigational new drug application (IND) and IND has been submitted in or converted to Electronic Common Technical Document (eCTD) format, unless the IND is of a type granted a waiver from eCTD format. 
• For both CBER and CDER, the program is open to sponsors who have demonstrated substantial effort to ensure that Chemistry, Manufacturing, and Controls (CMC) development aligns with clinical development. 
• For CBER, eligible products must be a gene or cellular therapy intended to address an unmet medical need as a treatment for a serious rare disease or condition, which is likely to lead to significant disability or death within the first decade of life. 
• For CDER, products must be intended to treat rare neurodegenerative conditions, including those of rare genetic metabolic etiology. 

The agency looks forward to ongoing engagement with the sponsors selected to participate, and hopes the insight gained will provide information on how best to facilitate more efficient development of potentially life-saving and life-changing therapies with rare disease indications. 

For questions about the START Pilot Program, please contact CBER’s Office of Communication, Outreach and Development (ocod@fda.hhs.gov) or CDER’s START Pilot Program mailbox (CDER.STARTProgram@fda.hhs.gov). 

Related information: 

• Federal Register: Support for Clinical Trials Advancing Rare Disease Therapeutics Pilot Program; Program Announcement 
• Scientific Challenges and Opportunities to Advance the Development of Individualized Cellular and Gene Therapies; Request for Information 
• Opportunity for Feedback on Development and Dissemination of Educational Materials on Rare Disease Drug Development 
• CDER’s Accelerating Rare disease Cures (ARC) Program 
• Demonstrating Substantial Evidence of Effectiveness for Human Drug and Biological Products