Clinical trials are studies which determine the safety and efficacy of new medications, medical devices or treatments for human use. They are conducted according to a strictly defined set of regulations which are commonly known as the protocol.
The exact course of each individual study varies from one clinical trial to another and depends on the specific goals of the trial. However, they all usually require a visit to the clinical trial location for tests or evaluations to be performed.
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There are various goals of clinical trial research, depending on the study:
- Treatment: evaluating the latest therapies, surgeries and drugs
- Prevention: methods of avoiding contracting a disease or preventing a disease from returning (e.g. medicines, vitamins, vaccines or lifestyle changes)
- Diagnostics: improving medical tests or procedures for diagnosing diseases or medical conditions
- Screening: enhancing disease detection techniques
- Quality of life: advancing methods of improving the quality of life for individuals with chronic or terminal conditions
Who conducts clinical trials?
A: Clinical trials are run by a team of specialized individuals. These include health care providers, both doctors and nurses, administrative staff members and other functionaries such as social workers. Doctors involved in clinical trials are called investigators.
The role of investigators in clinical trials is manifold. At first, they make a preliminary assessment of each participant’s general health. Afterwards, they instruct all participants about the specifics of the trial and relay further instructions. Throughout the trial investigators monitor participants’ progress and monitor their safety. Once the trial is completed, investigators are charged with establishing follow-up meetings.
What clinical trials are available for participants with current medical conditions?
Individuals with current medical conditions interested in participating in clinical trials have two types of options:
- Observational: Participants are observed by staff on site. Changes in their health condition are recorded. No specific treatments are administered in this type of trials.
- Interventional: Specific treatments are administered in this type of trial according to the trial protocol. The participant’s reactions to the treatment are then recorded and measured by investigators.
What are “phases” in clinical trials?
In most circumstances clinical trials are conducted in four phases (I, II, III, IV).
During several months of Phase I, the safety of therapeutic agents is assessed. A small number of healthy volunteers (20 to 100) are enrolled to study the effects of the therapeutic agent on humans to determine a safe dosage range and to identify and investigate possible side effects.
70% of experimental therapeutic agents complete this phase of testing.
Phase II lasts from several months up to two years, when the efficacy and continued safety of the therapeutic agent is tested. Testing in Phase II usually involve several hundred patients and is randomized: one group of patients receives the new therapeutic agent whereas the second â€œcontrolâ€ group receives a conventional treatment or placebo. As a common practice, during these trials, neither patients nor investigators know who receives the experimental drug. This method allows researchers to collect comparative information about the relative safety and effectiveness of the new therapeutic agent.
33.33% of experimental therapeutic agents complete both Phase I and Phase II
Phase III involves randomized, blind and large-scale testing conducted over several years, comprising several hundred to several thousand patients. It allows a pharmaceutical company and the HPB to monitor adverse effects and effectiveness of the researched agents. Once Phase III is complete, a pharmaceutical company can request HPB’s approval to market the therapeutic agent.
70% to 90% of drugs that make it to Phase III successfully complete testing.
Phase IV is conducted after the therapeutic agent has been marketed. The studies are designed to:
- compare the drug with competitors on the market,
- monitor long-term effectiveness and
- determine the cost-effectiveness of a drug therapy relative to competitors.
The results of the studies conducted in Phase IV may result in the drug being taken off the market or restrictions of use being placed on the drug.
How are clinical trials funded?
Clinical trials can be subsidized by a range of organizations and individuals, including but not limited to: medical institutions, foundations, pharmaceutical companies and physicians.
Where are clinical trials held?
Clinical trials can be conducted in any properly equipped facility such as hospitals, doctor’s offices, clinics or universities.
What is the source of the objectives for clinical trials?
The objectives of clinical trials are usually the result of research conducted at pharmaceutical companies, universities and other medical institutions. The research is primarily focused on an improved treatment option or a cure for a disease or medical ailment. After extensive laboratory testing, the research results that hold the most promise are then moved into a clinical trial phase.
What are the benefits and risks of participation in clinical trials?
Clinical trials hold many benefits for their participants, including:
- Access to the newest treatments before they become accessible to the general public
- Receiving the highest quality medical care for the duration of trial
- Being able to play a more active role in your own health care
- Contributing to medical research
- Helping other people with similar conditions
Although there are many benefits of participation in clinical trials, there are also some risks as well:
- Investigational treatments sometimes carry unpleasant, serious or potentially life-threatening side effects
- Treatment may not be effective for you
The process of understanding all the risks and benefits is called the Informed Consent. Rest assured, before participating in any clinical trial all the benefits and risks will be covered with you thoroughly by an investigator.
How long do clinical trials usually last?
Depending on the type of study, a clinical trial can take up only a few hours of your time or require a several week stay. However, most clinical trials do not require a participant to stay overnight in the study location.
Can you participate in more than one clinical trial?
Although you may qualify for multiple trials, normally you can only participate in one given trial at a time. This is due to a minimum waiting period or the â€œwash-outâ€ period between trials. This period usually lasts between one to four months.
What are common clinical trial terms you should know?
The Investigational Treatment is one which is not yet approved for public distribution by a governing agency.
The Protocol is an outline of a clinical trial which details the length of the trial, the required participant characteristics, all the tests, procedures, medications and dosages administered during the trial and other important information. The Protocol is enforced in order to ensure your health as well as optimal answers for the medical research questions.
The Placebo is a substance which has no medically active components. Placebos (sugar pills) are used in the control groups of clinical trials in order to determine the effect of investigational treatments or active drugs.
Control groups are the mean against which the results of the clinical trials investigational treatment are measured. Control groups generally receive either a placebo, standard treatment or no treatment at all.
Side Effects are the undesired outcomes or symptoms of an investigational drug or treatments. One of the goals of clinical trials is to appraise the short and long term Side Effects of an investigational treatment. Before participating in a clinical trial all the possible Side Effects will be explained to you.
What should I know about participating in a clinical trial?
The investigator will provide you with the specifics and detailed information about the trial before you choose whether to participate or not. Before beginning the trial, the investigator has to complete the informed consent process.
As a part of the informed consent process, the investigator provides documents which contain a detailed description of the study, including: the purpose of the clinical trial, duration, details of the investigational treatment, all procedures performed during the trial, contacts and risks and benefits of the clinical trial.
Once you feel comfortable with the trial, you can choose to sign the informed consent document. However, it is important to note that the informed consent document is by no means a contract and that you can withdraw from the trial at any time.
How do I qualify to participate in a clinical trial?
Each trial has specific conditions which have to be met in order to participate. These criteria include age, gender, type and stage of disease, current treatments and other medical treatments specifications.
The terms are not only implemented in order to provide the best possible outcome for the trial but also to safeguard the participant’s safety.
Do you have to be in pain to participate in a clinical trial?
No. There are various clinical trials, some of which may concentrate, for example, on the effects of medicine on healthy volunteers and the testing of vaccines.
How will I be protected in clinical trials?
Volunteers in clinical trials are protected by several different regulations.
The first of these is the exclusion criteria which ensure that several safety factors- e.g. age, gender, medical history-affect a person’s eligibility to enrol in a study.
Furthermore, all clinical trials are supervised by qualified healthcare professionals. Therefore, in the case of an adverse reaction there is always a qualified individual who will ensure the appropriate measure will be taken for the safety of the clinical trial participants.
Lastly, all clinical trial participants have a legal right to withdraw form a clinical trial at any time and all volunteer’s information is confidential and cannot be disclosed.
How should I prepare for a preliminary meeting with the site staff?
To prepare for a first meeting with the investigators it is always recommended to write down questions you may have. Also, bringing a friend or a relative as well as a tape recorder is also suggested.
Can I continue to be looked after by my primary health care provider while in a clinical trial?
Yes. Although clinical trials provide short-term investigational treatments, it is highly recommended that you consult your primary health care provider to ensure that your participation in clinical trials is successfully incorporated into your long-term health care programme