title: Investigational new drugs submitted to the Food and Drug Administration that are placed on clinical hold: the experience of the Office of Cellular, Tissue and Gene Therapy.
abstract: BACKGROUND
  Cell and gene therapies are medical products regulated by the U.S. Food and Drug Administration (FDA) within its Center of Biologics Evaluation and Research (CBER) in the Office of Cellular, Tissue, and Gene Therapy (OCTGT). Clinical research using cell and gene therapies in the United States must be conducted under an Investigational New Drug (IND) application. After an initial, 30-day review FDA either places an IND on clinical hold or allows the IND to proceed.
  
  
  METHODS
  We reviewed letters sent by OCTGT to IND sponsors that were placed on clinical hold. We categorized each deficiency and determined its frequency.
  
  
  RESULTS
  We found that similar deficiencies existed across IND applications and we tabulated the most common deficiencies.
  
  
  CONCLUSIONS
  We discussed the deficiencies and the resources that can help individuals avoid those deficiencies. We believe that awareness of the common deficiencies along with the applicable resources can reduce the frequency of clinical holds and allow clinical studies to proceed without delay. We also believe that this information will guide the FDA as to how to facilitate development of safe and effective cell and gene therapies.
authors.affiliation: Food and Drug Administration, Center for Biologics Evaluation and Research, Rockville, Maryland 20852, USA. keith.wonnacott@fda.hhs.gov
authors.forename: K
authors.initials: K
authors.lastname: Wonnacott
authors.forename: D
authors.initials: D
authors.lastname: Lavoie
authors.forename: R
authors.initials: R
authors.lastname: Fiorentino
authors.forename: M
authors.initials: M
authors.lastname: McIntyre
authors.forename: Y
authors.initials: Y
authors.lastname: Huang
authors.forename: S
authors.initials: S
authors.lastname: Hirschfeld
mesh_terms: D064987:Cell- and Tissue-Based Therapy; D002986:Clinical Trials as Topic; D004353:Drug Evaluation, Preclinical; D015507:Drugs, Investigational; D015316:Genetic Therapy; D006801:Humans; D017387:Investigational New Drug Application; D014481:United States; D014486:United States Food and Drug Administration
chemical_list: D015507:Drugs, Investigational