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Statutory regulations: Investigating safety and or efficacy of a medicine

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Introduction

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.

Resources

  • 1. NIHR Clinical Trials Toolkit: Is the trial within the scope of the Regulations?

    NIHR Clinical Trials Tool Kit guidance on the definition of a clinical trial of an investigational medicinal product (CTIMP): i.e. the type of studies that fall under the provisions of the Medicines for Human Use (Clinical Trials) Regulations.

    Legal Requirement for some em studies (not applicable for all) Date added: 23 October 2008
  • 2. NIHR Clinical Trials Toolkit

    This web-based resource is designed to support clinical trialists and regulators in ensuring clinical trials of investigational medicinal products are conducted in accordance with the Medicines for Human Use (Clinical Trials) Regulations.

    Legal Requirement for some em studies (not applicable for all) Date added: 29 October 2008
  • 3. ATMP: Advanced Therapy Medicinal Product Regulations (EC) No 1394/2007

    European Regulations for the conduct of clinical trials of Advanced Therapy Medicinal Products (ATMPs). These include trials of gene therapies, stem cell therapy, and tissue engineered therapy. The Regulations include a working definition of trials covered.

    Legal Requirement for some em studies (not applicable for all) Date added: 18 November 2008
  • 4. MHRA: Advanced Therapy Medicinal Products

    MHRA guidance on how advanced therapy medicinal product trials (ATMPs) are regulated in the UK (including tissue engineering, gene therapy and stem cell therapy).

    Legal Requirement for some em studies (not applicable for all) Date added: 18 November 2008
  • 5. MHRA: Borderline Products

    MHRA Borderline Products pages provide definitions of what constitutes a medicine / medicinal function.

    Legal Requirement for some em studies (not applicable for all) Date added: 18 November 2008
  • 6. UK Stem Cell Tool Kit

    A regulatory tool for those conducting human stem cell research in the UK.

    Legal Requirement for some em studies (not applicable for all) Date added: 18 January 2010