Skip to main content
Content Starts Here GSA Federal Advisory Committee Act (FACA) Database Skip to main content

Committee Detail

Note: An Annual Comprehensive Review, as required by §7 of the Federal Advisory Committee Act, is conducted each year on committee data entered for the previous fiscal year (referred to as the reporting year). The data for the reporting year is not considered verified until this review is complete and the data is moved to history for an agency/department. See the Data From Previous Years section at the bottom of this page for the committee’s historical, verified data.

Details on agency responses to committee recommendations can be found under the Performance Measures section for each committee in the fields “Agency Feedback” and “Agency Feedback Comment.”


HHS - 127 - Cellular Tissue and Gene Therapies Advisory Committee - Authorized by Law
Hide Section - GENERAL INFORMATION

GENERAL INFORMATION

Committee NameCellular Tissue and Gene Therapies Advisory CommitteeAgency NameDepartment of Health and Human Services
Fiscal Year2022Committee Number127
Original Establishment Date11/28/1990Committee StatusChartered
Actual Termination Date Committee URLhttp://www.fda.gov/AdvisoryCommittees/Committee...
New Committee This FYNoPresidential Appointments*No
Terminated This FYNoMax Number of Members*14
Current Charter Date10/28/2022Designated Fed Officer Position Title*Designated Federal Officer
Date Of Renewal Charter10/28/2024Designated Federal Officer PrefixMs.
Projected Termination Date Designated Federal Officer First Name*Christina
Exempt From Renewal*NoDesignated Federal Officer Middle Name
Specific Termination AuthorityDesignated Federal Officer Last Name*Vert
Establishment Authority*Authorized by LawDesignated Federal Officer SuffixM.S.
Specific Establishment Authority*21 U.S.C. 394Designated Federal Officer Phone*(240) 402-8054
Effective Date Of Authority*11/28/1990Designated Federal Officer Fax*301-595-1307
Exempt From EO 13875 Discretionary CmteNot ApplicableDesignated Federal Officer Email*christina.vert@fda.hhs.gov
Committee Type*Continuing
Presidential*No
Committee Function*Scientific Technical Program Advisory Board
Hide Section - RECOMMENDATION/JUSTIFICATIONS

RECOMMENDATION/JUSTIFICATIONS

Agency Recommendation*Continue
Legislation to Terminate RequiredNot Applicable
Legislation StatusNot Applicable
How does cmte accomplish its purpose?*The Committee reviews and evaluates available data relating to the safety, effectiveness, and appropriate use of human cells, human tissues, gene transfer therapies and xenotransplantation products which are intended for transplantation, implantation, infusion and transfer in the prevention and treatment of a broad spectrum of human diseases and in the reconstruction, repair or replacement of tissues for various conditions. On Dec 9-10, 2021, a web conference was held for the Gene Transfer and Immunogenicity Branch (GTIB) Site Visit Meeting to evaluate their research program and to make recommendations to the committee. On March 10, 2022, the Committee met by web conference in open session to hear an overview of the research programs of the Gene Transfer and Immunogenicity Branch (GTIB), Division of Cellular and Gene Therapies (DCGT), Office of Tissues and Advanced Therapies (OTAT), Center for Biologics Evaluation and Research (CBER). After the GTIB open session, the meeting was closed to the public to make recommendations to the Agency on specific research programs. The recommendations of the committee were utilized by FDA as part of its independent intramural program review of research priorities. On June 9, 2022, the Committee met by web conference in open session to discuss and make recommendations on biologics license application BLA 125755 from bluebird bio, Inc. for elivaldogene autotemcel to treat patients younger than 18 years of age with early cerebral adrenoleukodystrophy. The FDA considered the recommendations of CTGTAC in its review of BLA 125755, including recommendations on post-licensure studies and granted accelerated approval for Skysona (elivaldogene autotemcel). On June 10, 2022, the Committee met by web conference in open session to discuss biologics license applications BLA 125717 from bluebird bio, Inc. for betibeglogene autotemcel for the treatment of patients with β-thalassemia who require regular red blood cell transfusions. The FDA considered the recommendations of CTGTAC in its review of BLA 125717, including recommendations on post-licensure studies and granted approval for Zynteglo (betibeglogene autotemcel). On June 29-30, 2022, the Committee met by web conference in open session to discuss regulatory expectations for xenotransplantation products. The discussion topics include human cells that have had ex vivo contact with animal cells, and animal organs and cells for transplantation into human subjects, both of which are xenotransplantation products. The FDA will consider the recommendations of CTGTAC in evaluating xenotransplantation products.
How is membership balanced?*Members have clinical or preclinical and product experience in the fields of cellular therapies, tissue transplantation, gene transfer therapies, and xenotransplantation including biostatistics, bioethics, hematology/oncology, human tissues and transplantation, reproductive medicine, general medicine and various medical specialties including surgery and oncology, immunology, virology, molecular biology, cell biology, developmental biology, tumor biology, biochemistry, rDNA technology, nuclear medicine, gene therapy, infectious diseases, and cellular kinetics. One member is technically qualified and identified with consumer interests and one non-voting member represents the point of view of industry.
How frequent & relevant are cmte mtgs?*The committee held 3 advisory committee meetings on March 10, 2022, June 9-10, 2022, and June 29-30, 2022, and 1 site visit on Dec 9-10, 2021, in FY 2022. It’s anticipated the committee may have 3 advisory committee meetings and 1 site visit meeting in FY23.
Why advice can't be obtained elsewhere?*Members of the Committee are drawn from academia, research, and/or clinical practice. Their advice and input lends credibility to regulatory decisions. The alternate means of obtaining this advice would involve the recruitment of large numbers of scientists on a full-time basis at maximum rates of compensation.
Why close or partially close meetings?The committee held one closed sessions in FY2022.
Recommendation RemarksThe Agency is in the process of considering the recommendations of the Committee from each meeting held in 2022 (March through June) to implement the necessary regulatory actions
Hide Section - PERFORMANCE MEASURES

PERFORMANCE MEASURES

Outcome Improvement To Health Or Safety*YesAction Reorganize Priorities*Yes
Outcome Trust In GovernmentYesAction Reallocate ResourcesNo
Outcome Major Policy ChangesYesAction Issued New RegulationsYes
Outcome Advance In Scientific ResearchYesAction Proposed LegislationNo
Outcome Effective Grant MakingNoAction Approved Grants Or Other PaymentsNo
Outcome Improved Service DeliveryNoAction OtherYes
Outcome Increased Customer SatisfactionYesAction CommentFDA approves or chooses not to approve an investigational medical product.
Outcome Implement Laws/Reg RequirementsYesGrants Review*No
Outcome OtherNoNumber Of Grants Reviewed0
Outcome CommentNANumber Of Grants Recommended0
Cost Savings*Unable to DetermineDollar Value Of Grants Recommended$0.00
Cost Savings CommentThe utilization of the Cellular, Tissue and Gene Therapies Advisory Committee enables the Agency to obtain required and frequently scarce professional services from medical and scientific experts not otherwise available to the Agency; and to obtain the services of these experts only on an as needed basis rather than on a full time basis. The services of the Committee resulted in advice for the improvement of the public health, for which it is difficult to assign a financial value.Grants Review CommentNA
Number Of Recommendations*97Access Contact Designated Fed. Officer*Yes
Number Of Recommendations CommentThe Committee made 97 recommendations from FY2003 through FY2022.Access Agency WebsiteYes
% of Recs Fully Implemented*84.00%Access Committee WebsiteYes
% of Recs Fully Implemented CommentThe function of an advisory committee is purely advisory in nature. Although the FDA most often accepts the recommendations from its committees, the advice is purely advisory in nature, and therefore, the Agency has the option of not implementing the advice.Access GSA FACA WebsiteYes
% of Recs Partially Implemented*10.00%Access PublicationsYes
% of Recs Partially Implemented CommentThe function of an advisory committee is purely advisory in nature. Although the FDA most often accepts the recommendations from its committees, the advice is purely advisory in nature, and therefore, the Agency has the option of not implementing the advice.Access OtherNo
Agency Feedback*YesAccess CommentN/A
Agency Feedback Comment*The Agency usually does. Product approval issues are first released to the sponsor. When appropriate, information is made available to the public. Actions related to guidance documents or other general matters issues are available publicly when implemented.Narrative Description*FDA's strategic priorities in responding to the public health challenges of the 21st century are to advance regulatory science and innovation; strengthen the safety and integrity of the global supply chain; strengthen compliance and enforcement activities to support public health; expand efforts to meet the needs of special populations; advance medical countermeasures and emergency preparedness; advance food safety and nutrition; promote public health by advancing the safety and effectiveness of medical products; establish an effective tabacco regulation, prevention, and control program; and manage for organizational excellence and accountability. The Cellular, Tissue, and Gene Therapies Advisory Committee supports FDA's mission and strategic action by reviewing and evaluating available data relating to the safety and effective use of cellular therapies, tissue transplantation, gene transfer therapies and xenotransplantation, which are intended for the use in the prevention and treatment of a broad spectrum of human diseases. The Committee also considers the quality and relevance of FDA's research program, which provides scientific support for the regulation of these products. The Committee supports FDA's mission by using science-based risk management in all of its activities. The Committee recommendations provide the most health promotion and protection at the least cost for the public. This Committee assists the Agency in ensuring timely, high quality, cost-effective processes for review of new technologies/pre-market submissions, effective communication and working relationships with stakeholders to enhance U.S. and global health outcomes, accurately analyzing risks associated with medical products, facilitating the development and availability of medical countermeasures to limit the effects of a terrorist attack on the civilian and military populations, protecting the safety and security of biologics (gene therapy, human tissues, and cellular therapies) all key components of FDA's strategic plan objectives.
Hide Section - COSTS

COSTS

Payments to Non-Federal Members*$22,520.00Est Payments to Non-Fed Members Next FY*$32,984.00
Payments to Federal Members*$2,815.00Est. Payments to Fed Members Next FY*$8,245.00
Payments to Federal Staff*$314,749.00Estimated Payments to Federal Staff*$292,290.00
Payments to Consultants*$28,832.00Est. Payments to Consultants Next FY*$35,340.00
Travel Reimb. For Non-Federal Members*$0.00Est Travel Reimb Non-Fed Members nextFY*$0.00
Travel Reimb. For Federal Members*$0.00Est Travel Reimb For Fed Members*$0.00
Travel Reimb. For Federal Staff*$0.00Est. Travel Reimb to Fed Staff Next FY*$0.00
Travel Reimb. For Consultants*$0.00Est Travel Reimb to Consultants Next FY*$0.00
Other Costs$101,457.00Est. Other Costs Next FY*$96,075.00
Total Costs$470,373.00Est. Total Next FY*$464,934.00
Date Cost Last Modified11/8/2022 3:53 PMEst. Fed Staff Support Next FY*1.75
Federal Staff Support (FTE)*2.00Est Cost Remarks
Cost Remarks  
Hide Section - Interest Areas

Interest Areas

Category
Area
Food and Drugs
Food and Drugs
Health
Treatment
Medicine
Diseases
Hide Section - MEMBERS,MEETINGS AND ADVISORY REPORTS

MEMBERS,MEETINGS AND ADVISORY REPORTS

To View all the members, meetings and advisory reports for this committee please click here
Hide Section - CHARTERS AND RELATED DOCS

CHARTERS AND RELATED DOCS

No Documents Found
Hide Section - DATA FROM PREVIOUS YEARS

DATA FROM PREVIOUS YEARS

Committee

Data from Previous Years

 
ActionCommittee System IDCommittee NameFiscal Year
 COM-040502Cellular Tissue and Gene Therapies Advisory Committee2021
 COM-038777Cellular Tissue and Gene Therapies Advisory Committee2020
 COM-036659Cellular Tissue and Gene Therapies Advisory Committee2019
 COM-034874Cellular Tissue and Gene Therapies Advisory Committee2018
 COM-001894Cellular Tissue and Gene Therapies Advisory Committee2017
 COM-002636Cellular Tissue and Gene Therapies Advisory Committee2016
 COM-003779Cellular Tissue and Gene Therapies Advisory Committee2015
 COM-004376Cellular Tissue and Gene Therapies Advisory Committee2014
 COM-006265Cellular Tissue and Gene Therapies Advisory Committee2013
 COM-006946Cellular Tissue and Gene Therapies Advisory Committee2012
 COM-008393Cellular Tissue and Gene Therapies Advisory Committee2011
 COM-008723Cellular Tissue and Gene Therapies Advisory Committee2010
 COM-010099Cellular Tissue and Gene Therapies Advisory Committee2009
 COM-011046Cellular Tissue and Gene Therapies Advisory Committee2008
 COM-012171Cellular Tissue and Gene Therapies Advisory Committee2007
 COM-012534Cellular Tissue and Gene Therapies Advisory Committee2006
 COM-013899Cellular Tissue and Gene Therapies Advisory Committee2005
 COM-014445Biological Response Modifiers Advisory Committee2004
 COM-015907Biological Response Modifiers Advisory Committee2003
 COM-016489Biological Response Modifiers Advisory Committee2002
 COM-017923Biological Response Modifiers Advisory Committee2001
 COM-018696Biological Response Modifiers Advisory Committee2000
 COM-019897Biological Response Modifiers Advisory Committee1999
 COM-020642Biological Response Modifiers Advisory Committee1998
 COM-021451Biological Response Modifiers Advisory Committee1997