Some Information about Clinical Research and Clinical Studies
Clinical research is the process necessary to bring safe and effective new drugs to market for public use. The U.S. Census Bureau estimates that 55% of all Americans take prescription medications on a regular basis and that virtually everyone takes over the counter (OTC) medication at some point. Every prescription or OTC drug currently in use in the United States started out as a concept and/or as an idea. Innovators, whether they are scientists, physicians, or bio-chemical engineers had to take this idea and work it steadily, following specific methodologies, through the clinical study process, to finally turn it into a beneficial, safe and effective “ready-to use” medicine.
In a recently conducted survey, 83% of those who responded agreed that clinical research is very important and that all new drugs must be tested on humans before they can be approved for general use. However, less than 15% of those surveyed had a basic understanding of how clinical research affects them directly. The general public appears to believe that only 32% of those who volunteer for clinical studies receive good medical care. As a result, nationwide, only an alarmingly low 4% to 6% of eligible patients do, in fact, participate in clinical studies.
This less than positive perception shown by the general public is simply contrary to the reality shown in survey results when those who have participated in clinical research studies are polled. Of these, 90% of all voluntary participants rate the quality of care received during the study as excellent and 85% of those who volunteered indicate that they would repeat their experience and would participate in another clinical study, if given the chance.
Because the difference between perception and reality is so dramatic, it is NEMRA’s intention to insure that information is available to those who may have an interest in study participation so they can make informed decisions regarding their involvement in the clinical research process. We understand that volunteering to participate in a clinical research study is not for everyone. Volunteers are the most valuable and the most respected component of the clinical study process; in reality, they are volunteering for our collective benefit. Without volunteers in clinical research new medications that may ultimately help the study participant as well as others suffering from various illnesses would never be developed and would never become available as a resource in the cure.
SOME BASIC INFORMATION REGARDING THE RESEARCH PROCESS:
Clinical research is a long, diligent process involving many steps, but it can be summarized in three major stages:
- Discovery – this is the conceptual stage when an idea for a new medicine begins. Normally, at this stage, the “inventor” or “creator” of the idea will seek to register it and will file for patent protection.
- Pre – Clinical Development – this is where the idea is taken to the experimental stage and the medicine starts to take shape. A large variety of experiments are performed in order to research their associated chemical and physiological interactions. Various models of the therapy are developed and applied, so researchers can observe the medicine’s effects and arrive at adequate levels of dosage for the new medication.
- Human Clinical Studies (Phases I, II, III and IV) – Before a new drug under investigation can be tested on humans, an Investigational New Drug (IND) application must first be filed with the U.S. Food and Drug Administration (FDA). Once this application is approved, clinical studies can then begin.
Clinical Studies – Phase I Studies
Phase I clinical studies focus on the safety, rather than the effectiveness, of a new medicine. During this stage, doses of the chemical compound are administered to a small group of healthy volunteers under the close supervision of a doctor. Doctors monitor participants very carefully to determine how their bodies react to the new drug, whether the compound is sufficiently absorbed into the bloodstream, duration of absorption and what dosage levels are safe. Examples of Phase I studies are our Healthy Volunteer and Drug Interaction Studies.
Clinical Studies – Phase II Studies
The second stage of clinical testing focuses on the medicine’s effectiveness against the illness that it is designed to treat. During Phase II, researchers also determine the most effective dosages for the new medicine and the most appropriate method of delivering the drug (for example: oral tablets, extended release capsules, inhaled systems, dermatological patches or injections). This stage involves testing patients who are in need of treatment and who volunteer for the tests. The patients for Phase II studies are drawn from a variety of research centers and hospitals that can be local, nationwide or worldwide. As it is true for all studies in all phases of drug development, strict guidelines of informed consent are observed so that the risks and potential benefits are clearly explained to patients.
Clinical Studies – Phase III Studies
In the final stage of human clinical studies, researchers aim to confirm the results of earlier tests in a large patient population. This phase can involve thousands of patients in hundreds of hospitals and research centers. The large number of patients involved also allows researchers to establish a broad database of information about the safety and effectiveness of the drug to satisfy government regulatory mandates in the many countries where it will be used. The large group of diverse populations also enables researchers to identify those problems that may affect only a few patients.
Registration and Approval of New Drugs:
Upon successful completion of Phase III studies, the new drug can be registered. A New Drug Application (NDA) is filed with the U.S. FDA (Food and Drug Administration) as well as with any other worldwide regulatory agencies. Upon review and successful approval of this application, the new medicine can be sold and used by the general population of those geographic areas for which approval was gained.
Clinical Studies – Phase IV Studies
These studies are often called “post marketing” studies because the drugs have already been approved and are available for purchase on the market. These studies are typically developed by the sales and marketing department of the pharmaceutical company that sponsored the drug in order to obtain more data on the product and compare its use in the marketplace to that of similar drugs.