A clinical trial is a research study to answer specific questions about a new medical treatment (medicine/drug, medical device, new therapies, vaccines), or new ways of using known treatments. Clinical trials (also called medical research and research studies) are used to determine whether such new treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that work in people.
Participants in clinical trials can play a more active role in their own healthcare, gain access to new research treatments before they are widely available, and help others by contributing to medical research.
Ideas for clinical trials usually come from researchers. After researchers have tested new therapies or procedures in the laboratory and in animal studies (preclinical studies), the treatments with the most promising laboratory results progress into clinical trials. By conducting clinical trials, more and more information is gained about a new treatment, its risks and how well it may or may not work.
Clinical trials are sponsored or funded by a variety of organizations or individuals such as physicians, medical institutions, foundations, voluntary groups, and pharmaceutical companies. Trials can take place in a variety of locations, such as hospitals, universities, doctors' surgeries, or community clinics.
A protocol is a study plan specific to each clinical trial. The plan is carefully designed to safeguard the health of the participants as well as answer specific research questions. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants following a protocol are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
A placebo us an inactive pill, liquid, or powder that has no treatment value. In clinical trials, experimental treatments are often compared with placebos to assess the treatment's effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.
A control is the standard by which experimental treatments are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
Interventional trials (also called treatment trials) determine whether new treatments, new combinations of drugs, new ways of using known therapies or new approaches to surgery or radiation therapy are safe and effective. These trials might ask a participant to take an experimental new drug or undergo surgery.
Prevention trials look for better ways to prevent disease in people who have never had the disease or to prevent a disease from returning. These approaches may include medicines, vaccines, vitamins, minerals, or lifestyle changes.
Observational trials address health issues in large groups of people. Trial participants may be asked to answer questions about their family histories or give blood samples, but they do not receive treatment for their diseases.
Screening trials test the best way to detect certain diseases or health conditions.
Quality of Life trials (or Supportive Care trials) explore ways to improve comfort and the quality of life for individuals with a chronic illness.
As an innovative pharmaceutical company, Boehringer Ingelheim has a large and experienced global team managing and conducting clinical trials in many indications around the world. Such trials are conducted to establish the potential of drugs under investigation to progress to becoming new medicines. With these trials we evaluate the safety, tolerability and efficacy of drugs. They are fundamental to developing novel treatments of therapeutic benefit.
Patient safety is the prime concern of Boehringer Ingelheim when designing clinical trials. Our trials are conducted according to agreed international standards and in compliance with all respective regulations. All patients who enter into our clinical trials are fully informed about potential risks and the benefits of their involvement and are free to withdraw their consent or discontinue participation at any time with no need for justification.
In clinical trials the effects of drugs under investigation are typically compared to that of placebos, which have no pharmaceutical impact, or with comparable drugs already on the market. Clinical trials are allocated to four main clinical development phases.
Phase I trials study the safety and tolerability of drugs in healthy volunteers. These trials also provide information on the degree and time course of absorption, distribution in the body, metabolic breakdown and excretion of the compound under investigation. These effects are studied at various dosage levels. This phase also seeks to identify whether the new drug interacts with food or other drugs and could reduce or increase the effects of medications taken simultaneously. Phase I trials include normally 20 to 50 volunteers and treatment duration generally ranges from single dose to multiple doses and from a few minutes up to two weeks.
Phase II trials involve patients with the specific illnesses which are targeted by the drug under investigation. These studies, which continue to evaluate drug safety, aim to establish the proof that a new drug is clinically effective in the treatment of the particular disease and what the appropriate drug doses should be. These trials may also result in unanticipated discoveries of treatment for other diseases. Phase II trials can include up to a few hundred patients and treatment duration normally does not exceed three months.
Phase III trials, which only proceed when phase II results have established the proof of concept for short term efficacy and safety for patients. During Phase III trials substantial evidence of the safety and efficacy of drugs under investigation is produced in large patient populations over extended time periods. The regulatory authorities for pharmaceuticals are provided with all information from these trials to enable them to determine whether the drug can be marketed as medicine. Phase III trials include as many as several thousand patients and treatment durations can be up to one year or longer.
Phase IV trials, typically continue to investigate a drug after its initial approval from the regulatory authorities. In this phase the focus is on further evaluation of the use for which approval was secured, for comparison to or combination with other established drugs and to generate more data on safety under broader use. Phase IV trials are an important tool to strengthen the understanding of the drug and to give guidance to prescribers and patients on the safe and appropriate use under various clinical conditions. Regulatory authorities can also request such trials to seek answers to particular issues. Phase IV trials are by definition always performed in the approved indication. They can be both small and also extremely large ( 10-30,000 patients).
* source: based on clinicaltrials.gov, cancer.gov and ich.org FAQs of 20 July 2005 as provided by IFPMA