Patients/caregivers

Clinical trials help doctors to understand how to treat a certain disease in the most innovative way possible. The beneficiary can be the patient themselves, or in the future, other people with a similar pathology. Furthermore, if you take part in a clinical trial you could be one of the first people to benefit from an innovative treatment.

Currently, the best way to find out about the possibility of participating in a clinical trial is to talk to your doctor or your patient organisation. You can also do a search on the available platforms.

• RNEC
• EudraCT
• Clinicaltrial.gov

• International Clinical Trials Registry Platform (ICTRP)

• PtCRIN

It is necessary to understand that for each identified study, there is a Protocol where essential safety conditions for the participants are defined, as well as the best way to answer specific scientific questions. In this protocol, there is a set of previously defined participation requirements that must be fulfilled by the patient. The requirements are divided into Inclusion Criteria (allow the patient to participate) and Exclusion Criteria (make it impossible for the patient to participate). Generally, the criteria relate to factors such as gender, age, degree of disease, other pathologies or treatments.

After showing interest in participating in a trial, the patient or volunteer is interviewed by the investigators, where they can clarify questions and understand in detail the objectives and risks associated with the trial. They will be given an Informed Consent Form, where they sign and express their willingness to participate freely.

Once accepted as a participant, depending on the clinical trial being tested and its design, you may be assigned the therapy in question or the control (after a process of randomisation). This control may be a placebo (a sham treatment without any effect) or a standard treatment of recognised efficacy already in use in clinical practice. In the randomisation process, you will be assigned to either one of the two groups (treatment or control).

The treatment will have a previously defined duration, during which the patient will be periodically monitored.

There is an extensive body of European and Portuguese legislation that guarantees citizens that clinical trials are designed and conducted in the safest possible way for their participants. Extensive prior scientific evidence of a high degree of confidence is required to indicate safety before administration in humans (e.g. prior animal studies).

It is known, however, that unanticipated adverse reactions may occur during the study. It is important to stress that it is not a risk-free activity. To this end, promoters and researchers are required to take out civil liability insurance, which will respond for any damages caused to the participant.

Portuguese law does not currently allow any payment for participation in clinical trials, in order not to promote financial incentives or benefits, in order to protect participants. However, participants will be reimbursed for all expenses related to their participation in the study, such as absence from work or travel.

In Portugal, participation in clinical trials should not be seen as a complementary way of obtaining income, but rather as a voluntary collaboration for the evolution of science.

There is an extensive body of European and national legislation that ensures standards and good practices with regard to clinical trials. You can check the legislation here.

In Portugal, before the start of a new clinical trial with medicines, the proposal must be reviewed and approved by the Ethics Committee dor Clinnical Research (CEIC), and authorised by the national competent authority, INFARMED – National Authority of Medicines and Health Products, I. P. From this assessment, it is concluded whether the possible risks outweigh the benefits, so that the study can proceed.

Examples of how clinical trials can help society in terms of health:

• Prevent disease by testing a vaccine

• Detecting or diagnosing diseases by testing a new type of test

• Treating diseases by testing new medicines

• Testing new uses (indications) of already marketed medicines

• Finding out how patients can control their symptoms or improve their quality of life – for example, by testing how a particular diet affects a disease

If the clinical trial shows positive results, it is up to regulatory agencies to decide whether the new therapy can proceed to the market.

Yes, children and adolescents can participate in Clinical Trials. In the particular case of newborn or pre-school children (up to 7 years old), they simply need parental/guardian consent for their participation.

In the case of children (up to 13 years of age) and adolescents (from 14 to 17 years of age), there is also the possibility that they may participate in clinical trials. The consent of their parents (both parents) or legal guardians for their participation is fundamental and imperative, as well as the assent of the minor participants.

In the case of adults without capacity of judgement, they may also take part in clinical trials. However, they are considered to be incapacitated subjects  not able to give informed consent, and all information must be clarified in an appropriate way, taking into account their level of understanding. Their legal representative must also give informed consent.

It is always part of the Informed Consent signed in advance by the patient that he or she may leave the clinical trial at any time. The patient may decide to leave the trial if his clinical condition worsens or if he feels that the treatment has no effect. The patient may even choose to leave at any time, with no obligation to give any satisfaction.