Clinical Trials - Five frequently asked Questions and their Answers
Published: 08.15.2021
Why are clinical trials set up? And what rules and framework conditions are they subject to? Here are the answers to five frequently asked questions about clinical trials with medicinal products.
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What is a clinical trial?
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Who plans clinical trials?
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What are the framework conditions for clinical trials?
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How do clinical trials work?
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What happens after the end of a clinical trial?
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1) What is a clinical trial?
A clinical trial describes a scientific investigation with pharmaceuticals on human subjects who participate voluntarily. Clinical trials also include research with human biological material and health-related patient data. The aim is to prove the efficacy of new medications and to test their safety in order to improve health, prevention, diagnosis, cure and care of diseases. Clinical trials are essential for the development and approval of new medications and are a prerequisite for regulatory approval.
There are different clinical studies. For example, medications go through several study phases on their way to approval, which focus on different aspects. In phase I trials, healthy study participants receive low doses of the investigational product in order to investigate its safety, tolerability and effect. It is only in phases II and III that the investigational product is testet on real patients. From phase to phase, manufacturers receive more and more information about the effects and risks of their medication.
A distinction is also made in clinical trials between intervention studies and observational studies. Interventional studies carry out a planned and targeted measure on patients - for example, by administering a medication within randomly assigned control and treatment groups. An observational study, on the other hand, analyzes the effects of a therapy over a longer period of time under real everyday conditions without targeted interventions in the treatment.
2) Who plans clinical trials?
Clinical studies are financed by private or state stakeholders - such as pharmaceutical companies, clinics, research institutes, doctors or foundations. They organize the planning and execution of the study themselves or hand over the research project as a sponsor to a so-called Clinical or Contract Research Organization (CRO), which is specialized in clinical studies and takes care of the organization, implementation and evaluation of study projects.
3) What are the framework conditions for clinical trials?
The conduct of each study requires the approval of the responsible authorities. In Germany, these are the Federal Institute for Drugs and Medical Devices (BfArM) and the Paul Ehrlich Institute (PEI). In addition, the responsible ethics committee, which consists of lawyers, scientists, doctors, theologians and non-professionals - representing the public and patients - must give an approving assessment. With regard to the German Medicinal Products Act (AMG), they weigh up the conditions under which the planned study can be carried out from an ethical, medical and legal perspective. In doing so, they ensure compliance with other regulations or laws such as Good Clinical Practice (GCP) or the General Data Protection Regulation (DSGVO). They also check whether medical facilities, doctors and technologies used are suitable for the clinical trial. This procedure serves in particular to protect the study participants.
4) How do clinical trials work?
Before the start of a clinical trial, the trial management draws up a trial plan with the help of experts. This defines and describes all measures of the study, including objectives, current research status, hypotheses, treatment, inclusion criteria for participants (such as demographic data and medical history), evaluation methods, benefit/risk assessment of the medication under investigation or information on the storage and handling of personal data. The protocol also defines the responsibilities of all those involved in the study.
After the trial protocol has been approved by the authorities and the ethics committee has given a positive assessment, patients are recruited. Before enrollment in the study, patients must be fully informed and subsequently sign the informed consent form and patient information. During the study period, all necessary data is collected and evaluated in regular safety reports. Competent authorities, ethics committees and an expert committee, which is set up in some studies for monitoring purposes, regularly check the data and safety. Finally, the study is statistically analyzed and documented in final reports.
Hospitals, university clinics or doctors' practices are among the so-called study sites in which clinical studies are conducted. Multicenter studies are usually carried out as part of drug research and involve a network of several institutions. Clinical trials can often also take place on an international level.
5) What happens after the end of a clinical trial?
Depending on the country, regulatory approval can be centralized at the European Medicines Agency (EMA) or the national authorities. In Germany, the Federal Institute for Drugs and Medical Devices (BfArM) and the Paul Ehrlich Institute (PEI) are responsible - depending on the product in question. Approval documents submitted by the pharmaceutical company include documents on the pharmaceutical quality of the form of therapy, such as the shelf life of the drug, and all results from the study phases. Ideally, the investiated medicinal product should have passed through phases I to III. If there are only few alternative available treatment methods, approval with restrictions can already be granted after phase II. In this case, the phase III study must be completed before a normal approval can be granted.
The responsible regulatory authority reviews the results of all clinical trials on the basis of the documents submitted. This process can take around a year. The authorities are primarily concerned with weighing up the risk-benefit ratio. Approval is only granted if the benefits of the therapy outweigh the risks.
Even after approval, the new medication is still under observation by the medical profession, authorities and manufacturers, for instance in phase IV studies. These are used to constantly update the risks and side effects of a therapy. It is also observed whether the drug can also be used for other conditions. In this case, the approval process starts again in phase II.