About the
Investigational Drug
Clinical Research Studies: What They Are and What They Offer
When considering participation in a clinical research study, it’s important to know this.
Clinical research studies are sometimes referred to as "clinical studies" or "clinical trials," or collectively as "clinical research," "medical research," or "research studies." As well, sometimes the words "study" or "studies," or "trial" or "trials" are used as shorthand terms. The information provided here applies equally, regardless of which term is used.
Clinical research studies are an important part of the development of medical treatments. Clinical research studies are designed to test investigational treatments for overall safety and effectiveness in specific patients. The results of clinical research studies will have to provide enough information for governmental regulatory agencies in your country, and their counterparts in other countries, to decide whether to license these investigational treatments so that doctors may prescribe them for their patients.
To do this, investigational treatments may be compared with the current standard of care, a placebo (a substance designed to have no effect on health), or with some other form of treatment.
Participation in a clinical research study is always voluntary, and choosing not to participate will not affect the current medical care that you are receiving.
| The four phases of a clinical research study |
Clinical research studies can happen in one of four phases, depending on the status of the development of the investigational treatment.
- A Phase I clinical research study is designed to determine the effects of an investigational drug in people. It is usually conducted on a small population of healthy people to observe how the body absorbs, tolerates, distributes, and metabolizes the drug.
- A Phase II clinical research study occurs after Phase I studies have been completed successfully. The investigational drug is tested for safety and effectiveness in a larger population of people who have the disease or condition for which the treatment was developed.
- A Phase III clinical research study is the last round of pre-approval testing for an investigational drug. The drug is tested in comparison to a standard or an approved treatment and/or placebo for the disease or condition for which the drug is being researched. The results of these studies usually provide the information for the drug’s package insert and label.
- A Phase IV clinical research study occurs after a drug has been approved by the competent regulatory agencies in your country. The drug may be compared to competitors, or may explore additional patient populations, or may study any specific, unusual, or unexpected side effects.
| Common terms related to clinical research studies |
Blinding: The design of a clinical research study is "blind" when one or more of the involved research parties do not know which medication the participant is taking. Blinding is done to prevent the unintentional biases that can occur when treatment assignments are known.
Double-blind: The design of a research study in which neither the participant nor the investigator knows which medication (or placebo) the participant is taking.
Informed Consent: The voluntary verification of a patient’s willingness to participate in a clinical research study, along with the documentation thereof.
Open-label: This is a research study in which everyone involved (patient, doctor, and study staff) is aware of the drug and dosing being given. In open-label studies, no one receives a placebo.
Placebo: A placebo is an inactive substance designed to resemble the drug being tested. It is used as a control to rule out any psychological effects testing may present.
Randomization: Research study participants are often assigned to different treatment or control groups by chance (like flipping a coin). No particular criteria are used to assign a participant to a particular group, so all the groups in the research study will be equally comparable.
| Who can participate in a clinical research study? |
All clinical research studies have guidelines about who can participate. The participating research physicians use very specific inclusion and exclusion criteria based on age, gender, the type and stage of a disease, previous treatment history, and other medical conditions. A potential participant must meet these criteria before joining a clinical research study. These criteria help research physicians identify appropriate participants, while ensuring that the researchers will be able to answer the questions they plan to study.
| Why should I participate? |
If you choose to take part in a clinical research study, you may:
- Have access to an investigational medication that could potentially be beneficial to you.
- Receive medical care and monitoring of your disease or condition during the course of the research study.
- Help others who have your disease or condition in the future by contributing to medical research.
| How am I protected? |
Governmental regulatory agencies have strict and specific rules for conducting clinical research studies. For example, all clinical research studies must be approved and monitored by an ethics committee. Ethics committees must:
- Ensure that appropriate steps are taken to protect the rights, welfare, and anonymity of all people who voluntarily participate in clinical research studies.
- Review and approve the research study protocol, all written information provided to participants, and methods of recruitment of research physicians and patients.
- Monitor aspects of a clinical research study and prerequisite reporting of unexpected or unknown side effects.
A clinical research study follows a carefully controlled protocol, which is a plan that details what research physicians will do in that study. Researchers will also report the results of the study at scientific meetings, in medical journals, and to various government agencies. All participants’ names remain confidential and will not be mentioned in these reports.
| What happens if I decide to participate? |
Anyone who is eligible and decides to participate in a clinical research study must first go through the Informed Consent process. In this process, the research study – its purpose, duration, required procedures, key contacts, and any possible benefits and risks – is explained in detail in an informed consent form, and all of your questions regarding study participation are answered by the research physician. This process ensures that you understand what participation involves. As a potential participant, you must then decide whether or not you want to participate. Your signature and date of your informed consent on this form make it an important study document. You may withdraw from the research study at any time. Very often, a reason for withdrawal will be asked.
The staff of a clinical research study may include doctors/physicians and nurses, as well as social workers and other healthcare professionals. The clinical study staff checks your health at the beginning of a research study and monitors you throughout the research study. The clinical study staff provides specific instructions for participation and follows the research study protocol carefully in collaboration with the research staff. The procedures you may receive depend on the research study protocol and the disease or condition being researched.
| Making your decision |
Deciding whether to participate in a clinical research study is up to you. If you decide not to participate in a clinical research study, your decision will not have any effect on the care you’re receiving now or in the future. If you decide to take part, you may withdraw at any time during the research study. If you have any questions, don’t hesitate to ask the research study staff. They are there to help you.
To find out if you may be able to participate:
