Harmonise clinical trial regulations to boost medical research - and save lives

Clinical trials are an essential part of the development of medical treatments. They involve  testing new medicines or new therapies, as well as optimising existing medicinal products and procedures, by carrying out carefully controlled investigations on patients. Although the majority of these trials are conducted by the pharmaceutical industry, many are driven by pressing public health needs, and by scientific opportunities, rather than by economic interests of private companies.

Such trials often involve diseases that affect a small number of patients, for example, orphan diseases such as cystic fibrosis, or areas which are not seen to offer suitable commercial potential, for example paediatric therapies and neglected diseases endemic to developing countries. Or it may be that the objective is simply to improve existing procedures and prescriptions, for example, finding the optimal drug combination or timing. These trials often require international collaborations between centres in different countries, to facilitate the recruitment of patients and to increase the diversity of ethnicities, which produces results with more general validity.

Complex and inconsistent

Tight national regulations ensure patient safety and the methodological quality of clinical trials. However, these mechanisms are very diverse.  Increasingly complex and inconsistent regulations are causing delays, raising costs and leading many well-conceived trials aimed at addressing important public health problems to either not be conducted, or to be so delayed that their impact is dramatically reduced. In the European Union alone, the total number of applications for clinical trials fell by 25 per cent between 2007 and 2011.

To promote medical research and to help regulators overcome this problem, the OECD is calling on its member governments to harmonise their clinical trial approval processes. The aim is to encourage international collaboration in clinical research and streamline procedures for conducting clinical trials.

Bureaucracy overwhelms academics

The initiative results from an in-depth analysis of the problems encountered in medical research. In 2010, the OECD Global Science Forum initiated a project to investigate existing difficulties and to propose measures to facilitate international cooperation in clinical trials that are set up for non-commercial purposes. This was carried out by a Working Group composed of experts appointed by member state governments through health ministries and /or research ministries, and representatives of major international organisations. The Group’s findings and policy recommendations were published in late 2011 (see report).

As part of the project, the Working Group conducted a global survey among the various stakeholder communities to identify the most critical areas of concern for the conduct of international clinical trials. From the survey findings, it clearly appeared that the lack of harmonisation and problems with interpreting highly heterogeneous regulatory procedures created burdens that were difficult to overcome by many academic sponsors wishing to collaborate with groups in other countries.

In addition to the challenges presented by the existing national regulatory complexity,  it was found that clinical trial investigators also had to abide by administrative requirements that were poorly adapted to the nature of their study. Existing regulations have mostly been developed to guide the conduct of traditional trials for new medicines, which present an unknown risk for patients.  They are often less suited to address the many academic trials that use drugs that are already marketed, and often present lower risks.

Risk-based approach

The set of principles adopted by the Council of the OECD in December 2012 were devised through a consensus effort by scientists, physicians and regulators. It introduces a risk-based oversight and management methodology for clinical trials. This framework combines a stratified approach that is based on the marketing authorisation status of the medical product and can be applied in a common manner across countries’ regulatory frameworks, with a trial-specific approach that considers other issues such as the type of populations involved in a trial, and the informed consent of the patients.

Although this recommendation is primarily driven by the need to facilitate co-operation among academic groups for clinical trials undertaken for non-profit purposes, countries may wish to extend its implementation to the oversight and management of all clinical trials, thus adopting principles similar to those enumerated below regardless of the objective of the trial.

The policy guidance is optimised to reducing the burden of trial oversight as far as possible. It should facilitate the current discussion on the proposed new European Clinical Trial regulations which are based on similar concerns, as well as help other countries that are engaged in revising their existing regulatory mechanisms.  It is expected that this will enhance international cooperation in the field of clinical research, to the benefit of the many patients who require better treatments.

Frédéric Sgard is Project Administrator, OECD Global Science Forum