A day to recognize the role of clinical research: International Clinical Trials Day

May 20 marks International Clinical Trials Day, a date designed to highlight the importance of clinical research and the participation of patients and volunteers that contributes to saving lives, to economic and social development and to providing benefits to healthcare systems. From OLIGOFASTX we do not want to miss the opportunity to address the subject and thank all the organizations and people involved in this complex process, because it is thanks to all of them that progress in medicine is possible.

A clinical trial is an experimental evaluation of a product (promising drug or diagnostic/therapeutic technique) that in its application to humans is intended to assess its efficacy and safety. Of the global investment in R&D in the pharmaceutical industry, more than half goes to the design, development and evaluation of clinical trials. This is a necessary step from a regulatory point of view for the proper understanding of drugs. The main objectives of a clinical trial are:

Development of new therapies: Clinical trials make it possible to test molecules that have given very good results in the preclinical phase (cells and animals) as possible new treatments for a wide variety of diseases. These treatments may be effective for patients who have not responded to existing therapies or who do not yet have effective treatments available.
Identification of side effects: It is important that new treatments are safe and have no negative side effects. Clinical trials allow early identification of any potential side effects and can help minimize the risks associated with future treatments.
Improved understanding of diseases: Clinical trials can also help to understand under real-world conditions how a disease affects patients. These data can lead to better diagnosis, as well as to new therapies and more effective treatments.

It is important, therefore, to continue to support and participate in these types of studies in order to continue to improve medical care and find new treatments for diseases and medical conditions.

Their success depends to a large extent on the willingness of the population to participate in them, but also on the quality, ethics and rigor in their design and execution.

In this blog post, we will explore some of the most relevant aspects of clinical trials, their challenges, benefits and how to participate in an informed and conscious way.

Participate in clinical trials

It is important to keep in mind that, although clinical trials are necessary and offer important opportunities, participation must be informed and conscientious. Before making the decision to participate in a clinical trial, it is essential to know the risks and benefits, as well as the guarantees and protections offered by the study. In addition to consent, participation in these studies is conditional on meeting a series of inclusion and non-exclusion criteria. Among the benefits are:

- Access to new treatments: Some people with serious illnesses may not have access to treatments that could improve their quality of life. Clinical trials offer these patients the opportunity to test new treatments that are not yet available to the general public.
- Free medical care: Many clinical trials offer free medical care and medications for participants. This can help people who do not have health insurance or who cannot afford standard treatments.
- Ongoing monitoring: By participating in a clinical trial, you will have access to medical care and expert review. You will be in constant communication with the researchers and physicians who will guide the participants throughout the study.
- Contribution to the advancement of science: Clinical trials are fundamental in medical research and in the search for new treatments for diseases. By participating in a clinical trial, you are helping researchers obtain important data that can lead to significant advances in the field of health.

Patients’ rights

In general, patients in clinical trials have the same rights as any other person receiving medical care. It is important to be aware of these rights and ensure that they are complied with throughout the study process. Clinical trials can be of great benefit to those in need of innovative treatments, but it is vital that these studies are conducted in an ethical manner and protect the safety of the patients.

1. Informed consent: Before participating in a clinical trial, patients should receive detailed information on the study’s goals, procedures, risks and benefits. They must also consent in writing to participate in the study on a voluntary basis, without pressure or coercion from the investigators.
2. Access to information: Throughout the clinical trial, patients have the right to receive relevant information about their health, the progress of the study, and the results obtained. They also have the right to ask questions and receive clear and honest answers.
3. Privacy protection: Medical and personal data of patients in clinical trials must be treated with confidentiality and protected from any malicious use. Patients have the right to know who has access to their information and how it is used.
4. Safety and monitoring: Patients in clinical trials should receive adequate medical care and constant monitoring by investigators. Study safety protocols must be rigorous and designed to minimize any potential risks.
5. Withdrawal from the study: At any time during the clinical trial, the patient has the right to withdraw from the study without having to give explanations or suffer negative consequences in his/her medical care.

Clinical trial innovation: new phases and designs

Clinical trials are an essential tool for research and development of new medical treatments. Three phases are usually distinguished:

Phase I. Healthy volunteers to demonstrate safety and with very low doses until the maximum dose is established.
Phase II. The safety, but also the efficacy of the drug is tested against a control group or placebo.
Phase III. It is tested in a large number of patients prior to full approval to detect any adverse effects.

In addition, in recent years, new designs and phases of clinical trials have been developed with the aim of improving the efficiency, safety and effectiveness of the studies. Some of the most recent advances in clinical trial design are described below:

Phase 0 trials: Phase 0 trials are conducted before the start of conventional clinical trials. Very low doses of drugs are used in a small number of participants to establish their safety and effectiveness. In this way, the feasibility of the large-scale clinical trial can be assessed before investing large amounts of time and money.
Historical trials: Historical trials use data from previously treated patients to evaluate the effectiveness of new therapies or drugs. These types of trials can be especially useful in rare diseases or diseases where it is difficult to recruit enough patients for a traditional clinical trial.
Phase IIb/III: Phase IIb/III clinical trials are combined studies that allow Phase II and Phase III studies to be conducted simultaneously. In this way, data on the safety and efficacy of a drug can be obtained early in the clinical trial process, which can save time and resources.
Adaptive trials: This type of clinical trial uses information obtained in real time to adapt the study design. The data collected are used to make adjustments in the number of patients participating in the study, in the inclusion and exclusion criteria, and even in the study objectives. In this way, faster and more efficient results can be obtained.

From OLIGOFASTX we thank all the people who participate in this type of studies, because thanks to them medicine can advance and make new drugs available to other people whose quality of life can be improved.

Sources of information:

European Clinical Trials Database (EudraCT): is a European Union database containing information on ongoing or completed clinical trials in Europe. Details about the trial, its current status, objectives and inclusion criteria can be found here.
European Medicines Agency (EMA): is the agency responsible for the evaluation, supervision and regulation of medicinal products in the European Union. Its website provides information on clinical trials, including their regulation and requirements for drug authorization.
European Clinical Research Infrastructures Network (ECRIN): It is a network of infrastructures for clinical research in Europe. Its website provides information on clinical trials, their design, regulation and requirements for their execution.
Clinical Trials Methodology Conference (CTMC): is an annual event organized by the EMA to discuss clinical trial methodology and promote its continuous improvement. In addition to live events, they also offer online resources for researchers and patients.
The Lancet: is a leading European medical journal that publishes clinical studies, systematic reviews, articles and commentaries on health-related topics. Its website offers access to relevant articles and clinical studies.

It is important to always consult with your primary care physician before participating in a clinical trial. In addition, it is important to know the risks and benefits of participating in a clinical trial before making a decision.