Glossary of words and terms used in this website

Advocacy groups and patient organizations (also known as patient advocacy groups, or PAGs) support the needs of patients. They may support research for a specific condition, promote awareness of it, and educate the community about it.

Clinical trial sponsors initiate the trial. Sponsors can be organizations or individuals, such as doctors, medical institutions, foundations, voluntary groups, pharmaceutical companies, or federal agencies.

In a comparator-controlled clinical trial, the investigational drug is compared with a drug that’s already approved for use. This helps the trial team to see if the investigational drug could have benefits over drugs that are already approved.

The consent process involves learning and discussing the details of a clinical trial before deciding whether to take part.

The ICF must be read and signed before a person can join a trial. It contains key information about the trial, such as why it’s being conducted, how long it will last, the health assessments involved and the potential risks and benefits. The ICF is not a contract, and if the person decides to join the trial, they can still withdraw at any time. If you do not understand English, a translator or interpreter may be provided.

In a controlled clinical trial, a group (or groups) of participants will not be given the investigational drug, instead they will be given a control (either an approved treatment for the condition or a placebo).

In a double-blind trial, neither the people who take part nor the trial doctor will know the treatment each participant is getting. Some trials are done this way because knowing which treatment each participant is getting can affect the results of the trial, through a concept called bias.

The EMA is an agency of the European Union (EU). The EMA protects public and animal health in EU member states, as well as the countries of the European Economic Area, by ensuring that all medicines available on the EU market are safe, effective and high quality.

An independent team of people who review trial protocols to protect the rights and welfare of the people who take part.

In the United States, the FDA is responsible for making sure medical treatments are safe and effective for people to use. The FDA staff meet with researchers and perform inspections of clinical trial sites to protect the rights of patients and will verify the quality and integrity of the data.

Health assessments in a trial could be questionnaires or medical tests. These allow researchers to collect information about the health of people taking part in a clinical trial, as well as to help researchers answer the trial questions.

An independent team of people who review studies to protect the rights and welfare of people who take part in clinical trials.

A type of clinical trial in which participants are assigned to groups that receive one or more intervention/treatment (or no intervention) so that researchers can evaluate the effects of the interventions on biomedical or health-related outcomes. The assignments are determined by the trial’s protocol. Participants may receive diagnostic, therapeutic, or other types of interventions.

A drug being tested in a trial. An investigational drug has not been approved by a regulatory authority for any use or has not been approved for the specific use or dose being studied.

Advocacy groups and patient organizations (also known as patient advocacy groups, or PAGs) support the needs of patients. They may support research for a specific condition, promote awareness of it, and educate the community about it.

A placebo is an inactive substance that looks like the drug or treatment being tested. Comparing results from the two groups suggests whether changes in the test group result from the treatment or occur by chance.

The written description of a clinical trial. It includes the trial’s objectives, design, and methods. It may also include relevant scientific background and statistical information.

Each country has its own regulatory authority with its own regulations, or laws, for conducting a clinical trial. The regulatory authority reviews and approves the protocol, and ensures that the clinical trial follows national regulations.