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Statutory regulations: Investigating safety and or efficacy of a medicine

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Studies designed to discover or verify the clinical, pharmacological and/or other pharmacodynamic effects of one or more medicinal products, identify any adverse reactions or study the absorption, distribution, metabolism and excretion, with the object of ascertaining the safety and/or efficacy of those products, are defined as clinical trials of investigational medicinal products. This definition includes pharmacokinetic studies.  All clinical trials of Investigational Medicinal Products (CTIMP) must be initiated, conducted and managed in line with the Medicines for Human Use (Clinical Trials) Regulations 2004.

Small scale, proof of concept trials or early human safety studies of potential medicinal interventions will be classified as a clinical trial under the Regulations, and must be conducted in line with the provision of the Regulations.

Trials of advanced therapy medicinal products (which includes some trials of gene therapy, stem cell therapy or tissue engineered medicinal products) also require authorisation by the MHRA, in line with the Advanced Therapy Medicinal Products Regulations (EC) No 1394/2007. More information is available on this Tool Kit at 'Statutory regulations: gene therapy, xenotransplantation and stem cell therapy on this Tool Kit' or at 'Statutory regulations: collecting or using human tissue' on this Tool Kit.

The UK Stem Cell Tool Kit guides those conducting human stem cell research through the regulatory requirements (a link for the Tool Kit is provided in the resources below).  The Tool Kit was developed jointly by UK regulators as a reference tool for those who wish to develop a programme of stem cell research and manufacture, including clinical applications.