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                FDA Meetings, Conferences and Workshops

FDA Meetings, Conferences and Workshops

                        FDA Meetings, Conferences, and Workshops: Past Events
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          Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products - 04/27/2023FDA Meetings, Conferences and Workshops Virtual
    Event Title
              Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products


                  April 27, 2023

  

    
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      Meeting Information
                Event MaterialsDate:
          
  


     
           

April 27, 2023   

 
 
        
                                      Time: 
                                      
12:00 PM
    - 4:30 PM
ET

 

  The FDA’s Center for Biologics Evaluation and Research (CBER) Office of Therapeutic Products (OTP) hosted a virtual public listening meeting to solicit input on methods and approaches (e.g., use of real-world evidence (RWE), registries) for capturing post approval safety and efficacy data for cell and gene therapy products. This event was held to meet an FDA commitment that is part of the seventh authorization of the Prescription Drug User Fee Act (PDUFA VII).

The rapid pace of innovation for cell and gene therapy products holds promise for transforming medicine and creating treatment options for patients living with difficult, rare, and often incurable diseases. With an increasing number of cell and gene therapy products in development and the potential for more of these treatments to become available to patients in the future, it is important to understand the full spectrum of long-term effects and collect accurate, timely, and comprehensive data to ensure these products remain safe, effective, and of high quality. This virtual listening meeting was an opportunity for stakeholders to provide input on methods, approaches, logistics, privacy concerns, and other aspects related to efficacy and safety data collection in the post-approval setting for cell and gene therapies.

View the recording for the public listening meeting on Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products:

Recording 1 of 2
	Recording 2 of 2


In addition to holding the virtual public listening meeting, FDA opened a docket, which closed on Friday, May 26, 2023. For access to the docket, to read background documents or the electronic and written/paper comments, go to Regulations.gov and insert the docket number, FDA-2023-N-0398, into the “Search” box and follow the prompts, and/or go to the Division of Dockets Management at 5630 Fishers Lane, Room 1061, Rockville, MD 20852.

About the OTP Public Listening Meeting

Under the Prescription Drug User Fee Act (PDUFA) VII, by the end of fiscal year 2024, FDA is required to convene a meeting to solicit input on methods and approaches (e.g., use of RWE, Deve) for capturing post-approval safety and efficacy data for cell and gene therapy products. There will be a docket for the public comment period, which will be open for 30 days following the meeting. Within six months after the public meeting, FDA will issue a summary report or a transcript of the meeting.

About OTP

OTP, previously known as OTAT, oversees development for a wide variety of biological products, including gene and cell therapies, tissues and tissue-engineered products, xenotransplantation products, and more. OTP strives to lead all regulatory decisions with data, impartiality, and compassion and welcomes the participation of patients and their advocates in formal meetings related to the development of investigational products. Learn more about OTP and view our available resources.

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Event Materials

  Title
            File Type/SizeFDA CBER OTP Listening Meeting: Methods and Approaches for Capturing Post-Approval Safety and Efficacy Data on Cell and Gene Therapy Products
              pdf               (681.99 KB)

                                                                                  Content current as of:
      10/18/2023
         
                   
Regulated Product(s)
		 Biologics
			  			     			  						              
			
			 
			  Gene Therapy
			  			     			  			     TissueMore Meetings,
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        FDA Meetings, Conferences and Workshops

          
                        FDA Meetings, Conferences, and Workshops: Past Events
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