Clinical Trials

The new legal basis for clinical research with medicinal products: The Clinical Trials Regulation (CTR)

As of January 31st, 2022, clinical research on medicinal products in the European Union is governed by the new Clinical Trials Regulation (CTR), specifically Regulation (EU) No 536/2014. This regulation supersedes the former legislation on clinical trials, namely the Clinical Trials Directive (CTD), Directive 2001/20/EC. The primary aim of the new regulation is to streamline the clinical trial application process and harmonise the assessment of interventional clinical trials among the EU Member States.
Further, the European Medicines Agency has introduced the Clinical Trials Information System (CTIS). This portal enables sponsors/CROs to centrally submit a clinical trial application and receive a consolidated decision covering the relevant national competent authorities and Ethics Committees.

Starting from January 31, 2023, all new clinical trial applications must be submitted through CTIS. Trials submitted before may fall under the governance of the previous CTD if desired by the sponsor. Nevertheless, by January 31, 2025, all clinical trials will be mandated to transition to the new system.
 

Various types of studies: Observational studies versus clinical trials

In order to decide whether a planned study falls under the CTR or not, it is important to differentiate between observational studies and clinical trials.

• Observational (non-interventional) studies monitor patients under the conditions of current medical practice and do not assign a patient to a particular therapeutic strategy in advance according to a study protocol. This type of studies is excluded from the scope of the clinical trials regulation.
• Clinical trials are research studies that prospectively test a medicinal product in humans. A clinical trial is designed to learn for example if a new treatment is more effective, has fewer side effects than existing treatments or improves the patients’ quality of life.
   

There are commonly three main phases of clinical research prior to a marketing authorisation application:

• Phase I clinical trials are conducted to determine the safety of a new medicinal product, treatment, or treatment combination. Subjects can be healthy or have the condition being studied.
• Phase II clinical trials gather more information about the safety and efficacy of the medicinal product in a larger group of patients. Further, different dose regimens may be studied.
• Phase III clinical trials test a treatment that showed promising results in a phase II clinical trial. Phase III are commonly confirmatory trials in patients comparing the new treatment with the standard treatment to determine if it is more effective, has fewer side effects, or both. Patients are usually assigned to different treatment groups.