New drugs are being introduced daily for both well-known and rare diseases affecting the people around us every day. This month alone, there have been approvals for the treatment of pain, diabetes, ADHD, pancreatic cancer, Schizophrenia, bipolar disorder, and high cholesterol.
The FDA defines a drug as any product intended for use in the diagnosis, cure, treatment, or prevention of a disease and is intended to affect the structure or any function of the body. This is not just limited to medicine; it also includes fluoride toothpaste, antiperspirant, dandruff shampoo, and sunscreen. In order for any drug to be approved by the FDA, it must go through 4 stages: pre-clinical, clinical, NDA (New Drug Application) review, and post-marketing.
Pre-Clinical stage – The drug is developed by the drug sponsor (typically the manufacturer) and the sponsor seeks to have it approved by the FDA so it can be sold in the U.S. The sponsor must first test the drug on multiple species of animals for toxicity. This is used to gather basic information on the safety and effectiveness of the drug. Following the animal testing, he/she will submit an IND (Investigational New Drug) application to the FDA based on the initial testing results including the composition and manufacturing of the drug, and the plan becomes developed for human trial.
The Clinical stage – There are several clinical studies/trials done to emphasize safety and effectiveness of the drug. Following the IND, the drug sponsor holds the first phase of the trial with 20-80 healthy volunteers to emphasize safety. The first phase determines what the side effects are and how it is metabolized and excreted. The next step is to hold the second phase of the clinical trial for hundreds of patients to emphasize effectiveness and determine whether the drug actually works for people who have a specific disease or condition. For controlled trials, those receiving the trial drug will be compared to those receiving another treatment, generally a placebo or a similar drug. Of course, safety is still evaluated in this phase, and the short-term side effects are also studied. At the end of Phase 2, the FDA and the sponsors will meet to discuss how studies will be done in Phase 3. For Phase 3 trials, thousands of patients are used. Phase 3 gathers more information on safety and effectiveness, studies different populations and dosages of the drug, and uses it in combination with other drugs.
The NDA Review stage – First the FDA will meet with the drug sponsor prior to the NDA. The next step is for the sponsor to formally ask the FDA for approval for marketing by submitting the NDA, which includes all animal and human data and information about how the body handles the drug and how it is manufactured. After the application is received, the FDA has 60 days to decide whether to file so it can be reviewed. If it is filed, the FDA Review team is assigned to review the research done on the safety and effectiveness of the drug. The next step in the process is for the FDA to review the professional labeling and communicate appropriate language to health care professionals and consumers. Then, the FDA inspects the manufacturing facilities. After all of these tasks have been completed, the FDA will either approve the NDA review or issue a response at a later time.
During the Post-Marketing stage, the FDA will critically monitor the drug to ensure its safety and effectiveness. Although there were several phases of trials done in the Clinical stage of the process, it is not possible to predict all of the effects the drug may have. The main goal of the post-marketing stage is to detect serious, unexpected adverse effects and take action. During the monitoring of the drug, the sponsor is required to submit safety updates to the FDA.
The FDA does offer an Accelerated Approval program and a Fast Track program for faster approvals for drugs that qualify. The Accelerated Approval program allows for earlier approval for drugs treating serious diseases and filling unmet medical needs. Since the drug can be evaluated by blood tests or x-rays rather than waiting for trial results, it is a faster process. The Fast Track program allows the drug sponsor to submit portions of their application when the information becomes available instead of waiting to submit it all together.
As expected, the process is a serious and timely manner to ensure the U.S. is only selling drugs that are both high in safety and effectiveness. Since the Prescription Drug User Fee Act (PDUFA) was passed in 1992, there have been over 1,000 drugs introduced into the market. Some of these drugs treat cancer, AIDS, and cardiovascular disease.
Our customers and our patients are our top priority. Quality and safety should be a quality that goes uncompromised in any pharmacy but that’s the HeartLight difference. Contact HeartLight Pharmacy today at one of our 3 locations or fill out the Contact Form to be contacted by a HeartLight Specialist in your area.