Science and imagination are the catalysts for any innovation, but the medical field has its own special process that needs to be followed in order to safely implement new medical therapies. Science begins when someone (a doctor, a nurse, or maybe even a chemist) notices something unexpected and writes it down. That is the spark that starts the scientific method rolling in the direction of a new medication. There are many stages in the process of developing new medications, but the most critical parts are the four stages of clinical trials that are required by the FDA before a new drug can be sold.
The FDA requires that all medications go through the clinical trial process in order to prove two things before going to market. First, and most importantly, a new medication must be safe. This means that a drugs overall benefit must outweigh its overall negative effect for any given dose both in an individual patient and the entire patient population, The cure cant be worse than the disease. Second, a new therapy must be at least as effective as any currently available therapy for a given condition. This means that a pharmaceutical company cannot create a new drug that is (comparatively) less effective then existing medications for the stated indication. The biggest obstacle to proving these things is testing the medication on people in an ethical and safe manner.
In response to instances of abuse in the name of medical research, along with the above requirements, the FDA created regulations specifically designed for the protection of clinical research study participants. First, all aspects of a clinical research trial have to be reviewed and approved by an independent review board (IRB) to make sure that the study run in a safe and ethical manner. Second, the study process cannot take advantage of “at risk” populations such as military personnel, prisoners, the mentally ill, and ethnic minorities among others. And finally, all study participants must undergo the informed consent process before participation in a clinical research trial.
Informed consent is, arguably, the most important part of study participation. Without it the participant has no idea what is being done. The FDA requires that all processes, procedures, and risks involved in study participation be explained in a manner to which the study participant develops a general understanding of what is involved with the study before consenting. In order to make sure that a participant has a complete understanding of the study they are required to read and go over an Informed Consent Form with a knowledgeable member of the study staff. Before signing this form it is highly recommended that the participant ask any and all questions they may have about the study, and take it to their primary care physician if desired. Once this form is signed they have given their consent to be a part of the study.
Patients have the right to take their consent back for any reason and at any time during the study process. The FDA has decided that people who stop participating in a study without notification (lost to follow up) will be counted as dead. If a living participant is counted as dead due to not wanting to participate any more the study statistics can be skewed to a negative result. This can cause an otherwise good medication to be rejected by the FDA. For this reason a lot of study sponsors are requiring study centers to continually try to contact participants who have become lost to follow up. Most studies will require weekly phone calls for the duration of the study, along with certified letters sent to the home, and some will go so far as to hire a company to track down the participant. All of which can be avoided with the simple act of saying “I don’t want to do this any more”. This process covers all phases of clinical trials with a new informed consent form being required at each phase of the trial.
The screening process starts when a potential study participant is first contacted. The prescreening questions are there to determine whether or not a potential participant is likely to meet the study inclusion and exclusion requirements. Once it is determined that a potential patient will likely qualify for the study an appointment is made for the doctor and a study coordinator to go over the consent and do a more in depth screening. Once a consent form is signed a patient will need to have a physical that will include vital signs blood work and possibly some specific testing of the condition being studied. The study doctor will also look at a patient’s medical record and history to make sure that all of the inclusion and exclusion criteria are met, and that it is safe to put the patient on the study. Once all of the results are looked at and the patient meets all of the study requirements they are eligible to be put onto study medication.
Most studies will do a comparison for effectiveness between the study medication and either a placebo (fake), or a comparator (similar medication) in order to determine how effective a medication is. A placebo is mainly used when there is not a standard of care medication for a given condition. It is also possible to have both a placebo and a comparative medication. Patients are given these medications on a “blinded” basis. That means neither the study staff nor the patient know if they are getting the study medication, a comparative medication, or a placebo. The arm of the study a patient goes on is determined through a computerized lottery process known as randomization. Once randomized the patient will stay on the same arm of the study for the duration of the study, and get the same type of medication throughout their study participation.
After a patient is randomized the rest of the study process is basically watching to see what happens. All studies will require periodic office visits to check vital signs, draw blood, and in some cases do an ECG or other test to make sure there aren’t any adverse reactions to the medication and determine how effective the medication is. Some studies require more in depth information about how the medication is working. There are several methods for collecting this information such as; a diary of events, or daily phone calls. If there is some reason that a patient needs to be removed from the study medication, most studies will ask to follow that person for the remainder of the study term in order to make sure that there are no further complications. After the study has ended, all of the data will be reviewed and submitted to the FDA for approval to move on to the next phase of research trials.
Without these regulations pharmaceutical companies would not have to prove that their products are effective, or that they are safe. In todays profit based society it is absolutely imperative to regulate safety into processes that involve human life. The clinical research trial is the medical industries method of ensuring that medical processes and therapies are safe and effective.