What is a drug study?
In the United States, new drugs designed to treat disease must be approved by the Food and Drug Administration (FDA). Drug companies must design and pay for studies that demonstrate reasonable safety and efficacy to gain FDA approval, after which they may market their drugs to doctors and patients for a profit. Because pharmaceutical sales are so profitable, drug companies are highly motivated to develop new drugs. This effort helps patients who need better drugs to treat disease.
What are the phases of drug study?
Initially, drugs are developed by basic scientists who have reason to believe a new drug may be useful based on its chemical interaction with the body. Initial studies are performed in the laboratory and often involve animal studies. If basic safety criteria are met in the lab, the drugs may be brought to Phase 1 study in which some human subjects (usually, a dozen or two) are given the drug to look for a beneficial effect and lack of adverse effects. If a drug passes Phase 1, a Phase 2 study is designed to help determine the best dose of drug. More patients are recruited for these studies, but often number fewer than one hundred. If successful, a Phase 3 study is planned to gain approval by the FDA. These studies randomly assign drug treatment to patients in a manner that neither the treating doctor nor the patient knows what drug they are using. It is hoped that this method will avoid bias and offer a true assessment of the drug effect. The schedule of administration is completely designed by the drug company, but must be approved by the FDA. Unfortunately, this oversight may lack rigor. Phase 3 studies often include several hundred patients. Key endpoints of treatment are diligently recorded and adverse drug effects are monitored carefully. Finally, after FDA approval, drugs may be studied in a Phase 4 study looking further for adverse effects and/or new uses for other diseases. It is obvious that more is known about the effectiveness and safety of a drug the further along the study sequence it passes. Nonetheless, some adverse effects are not discovered until years after a drug has been approved for use by the FDA.
What are the benefits of participating in a drug study?
Apart from feeling good about being a part of drug development, patients may benefit from participating in drug studies. For example, the patient may gain access to a helpful drug that would not have been available otherwise. In addition, the drug company usually offers to pay for the medication and treatment visits during the course of the study. They often offer to pay cash to the patient in the form of a stipend for transportation to study visits.
What are the risks of being in a drug study?
Not surprisingly, with potential benefits come potential risks. For example, most drugs that enter the process of study, do not meet with success. That is, they are found to be inferior to existing medications or they are found to have unacceptable adverse effects. This is one risk a patient must accept. Secondly, the decision to treat and method of treatment is guided by strict protocols aimed to favor the study drug; not necessarily in the best interest of the patient. The doctor is given limited independent authority to alter treatment within the study. However, the patient may always exit the study at any time should he or his doctor decide. It is critical to remember that exposure to an unproven drug carries with it the risk of unknown risk of adverse effects. Complicating matters more, the doctor is highly compensated by drug companies to recruit patients for drug studies. During these times of decreasing physician compensation by Medicare and insurance companies, doctors may be under financial duress to cover the high cost of office overhead. There is a risk that the doctor may be motivated more by financial compensation than the best interest of the patient. Hopefully, this risk is not significant. The risk of adverse effects is highest in the early stages of study when less is known about the drug. Therefore, caution must be exercised with phase 1 and phase 2 studies. Phase 3 studies are safer, but still not without risk. Phase 4 studies are the safest because the drug has already been approved for use by the FDA; however, sometimes the full extent of adverse effects is still not known. Therefore, when considering study participation, at a minimum a patient should ask what phase study is being offered them.
So, should I participate in a drug study?
In the end, drug studies are an essential part of discovering new medicines to treat disease. If you feel you may benefit from a study drug and/or currently-approved drugs are not satisfactory to you, a drug study may be worth considering. However, you must accept the risks of being treated by a drug that has not been studied extensively for safety and efficacy in the past and is being used under a protocol designed by a drug company whose primary interest is gaining drug approval by the FDA. If in doubt, it may be best to seek a second opinion from a doctor outside of the drug study.
For a telemedicine consultation with Dr Pautler, please send email request to spautler@rvaf.com. We accept Medicare and most insurances in Florida. Please include contact information (including phone number) in the email. We are unable to provide consultation for those living outside the state of Florida with the exception of limited one-time consultations with residents of the following states: Alabama, Arkansas, Connecticut, Georgia, Minnesota, and Washington.
Copyright © 2022-2023 Designs Unlimited of Florida. All Rights Reserved