Introduction

1. This document gives guidance on the procedures that should be followed in the United Kingdom when proposals are made to conduct gene therapy research on human subjects. It details the information that should be submitted in order to enable the Gene Therapy Advisory Committee (GTAC) to assess the acceptability of gene therapy research proposals.

2. Some guidance is also given on the requirements of other regulatory bodies or committees, including Local Research Ethics Committees, the Medicines Control Agency, the Health and Safety Executive and the Department of Environment, Transport and the Regions.

3. The guidance should be read in addition to general guidance on clinical trials and research governance in the NHS (see Further Reading).

4. Supplementary guidance and reports have been issued by GTAC in relation to monitoring of patients enrolled in adenoviral gene therapy studies and in utero gene therapy. Both can be obtained as stand-alone documents from the GTAC web-pages. (www.doh.gov.uk/genetics/gtac.htm).

GTAC review process

1. 5 GTAC reviews proposals to conduct gene therapy research and provides advice in related areas. GTAC reviews are in addition to those of local research ethics committees (LREC), whose roles and responsibilities are unchanged.

2. 6 GTAC approval should be obtained before NHS personnel conduct any gene therapy research on human subjects, whether conducted on NHS or other premises, in the UK. GTAC approval should also be sought where any part of gene therapy research on human subjects takes place in the UK. This would include enrolment, monitoring, follow-up and other study related procedures. NHS personnel who conduct gene therapy research overseas are encouraged to submit protocols to GTAC for information (this should include any information that will be given to prospective participants).

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