A clinical trial in the European Union is defined by Directive 2001/20/EC, as a clinical study that aims to investigate the therapeutic effect(s) of medicinal products that have not yet been authorized by regulatory bodies such as the European Medicines Agency (EMA), or the value of authorized drugs in cases other than those for which it has been authorized. In addition, clinical trials are considered those studies that seek to assign specific products to a therapeutic strategy or propose to investigate effective diagnostic or monitoring procedures.
In contrast to popular belief, not all clinical trials are sponsored by pharmaceutical companies – indeed nearly 40% are funded by other stakeholders such as academic institutions and non-governmental organizations.
Directive 2001/20/EC on the implementation of good clinical practice in the conduct of clinical trials on medicinal products for human use, aimed to harmonize the regulatory framework for clinical trials across the 27 Member States of the EU. The Directive went a long way to ensure a high level of patient safety, but its’ complicated rules and procedures, led to an unfavourable regulatory framework for clinical research. Therefore, new therapeutic avenues through simplified, modernized and expedited procedures were considered necessary by stakeholder groups.
In this context, and after an extensive consultation period, the European Commission adopted on 17 July 2012 a Proposal of the European Parliament and of the Council for the revision of the Directive 2001/20/EC on clinical trials. The new Regulation is expected to come into force in 2016.
For more information on the EU legislation, click here
Clinical Trials Register
The launching of an online register by the European Medicines Agency (EMA) in 2011 has given, for the first time, public access to information on clinical trials for medicinal products authorised in the European Union. The database also allows the public to search for information on clinical trials authorised to be carried out outside the EU if these trials are part of a paediatric investigation plan.
For more information, click here
Patients with thalassaemia need to be involved in relevant clinical trials in the following areas:
- Blood Safety
- New Chelation drugs
- Drugs related to HbF induction
- Drugs related to therapy advances
- Gene Therapy
- Bone Marrow Transplantation
- Chronic Viral Hepatitis
Regular updates on news in these areas is available in the Research News section.