Ophthalmic Research Training Services

Clinical research is a branch of medical science that determines the safety and effectiveness of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatment, diagnosis or for relieving symptoms of a disease.

The term clinical research refers to the entire biography of a drug from its inception in the lab to its introduction to the consumer market and beyond. Once the promising candidate or the molecule is identified in the lab, it is subjected to pre-clinical studies or animal studies where different aspects of the drug (including its efficacy and toxicity) are studied.

In the United States, the data obtained from the pre-clinical studies are submitted as an Investigational New Drug (IND) to the Food and Drug Administration (FDA) for permission to conduct human studies.

In the European Union, the European Medicines Agency (EMA) acts in a similar fashion for studies conducted in their region. These human studies are conducted in four phases in research subjects that give consent to participate in the clinical trials.

Phase 1 Clinical Research Trials usually deal with investigating the studied drug in a minor number of research subjects who are healthy volunteers. This phase is mainly targeted at identifying the safety, tolerability, and the general mechanism of the action of the drug in humans.

These studies are usually conducted in tightly controlled clinics called CPUs (Central Pharmacological Units), where participants receive 24-hour medical attention and oversight. In some diseases in which the therapy under study is known to be too toxic for healthy subjects (some cancer medications, for instance), phase 1 trials are performed in patients with diseases to test these parameters. The goal of phase 2 Clinical Research Trials is to grasp additional understanding of the studied drug's safety and efficacy. It also determines the appropriate dose to be administered to deliver the desired treatment effect while minimizing the safety risk of future research subjects. This usually requires more than 100 patients to demonstrate relevant results, although the actual number of subjects varies widely based on the disease under study. Thus, multiple clinics are utilized to recruit subjects with the disease under study to sufficiently enroll the study(ies) in a reasonable period of time. Multiple phase 2 clinical research studies are often required to define the appropriate patient population to study during phase 3. Once the drug is deemed a potentially safe and effective candidate in Phase 2, it is then studied in Phase 3 clinical research trials. This phase often exposes more than 1000 research subjects with the disease, and is usually performed at many clinics (sometimes well over 100) to enroll the trial (or trials). There is a focus on the effectiveness of the study drug in a variety of demographic and socioeconomic subjects with variants of the disease under study. A comparison is usually made with standard drugs available on the market. It is imperative that the drug is shown to be effective and safe in this phase.

When phase 3 Clinical Research Trials are completed (as well as the data demonstrating safety and efficacy of the study drug), a New Drug Application (NDA) containing all manufacturing, pre-clinical, and clinical data is filed with the FDA for review. If deemed safe and effective, the FDA grants approval of the NDA, which then allows the company to market the product. This approval usually comes with strict requirements for the company to conduct additional studies to keep the NDA active (usually involving pediatric trials and additional safety trials).

In phase 4, the aim is to further characterize the safety of the drug through the identification of unknown adverse effects and to potentially research new therapeutic indications. Companies often use this phase to gain exposure to different physicians and clinics, which aids in the marketing of their product. The entire process of a drug from lab to market may take approximately 12 to 18 years (but not always), costing billions of dollars. Clinical research continues throughout the lifetime of the drug to include post marketing surveillance where a periodic 'progress report' is submitted to the regulatory authorities once every 2 years after the drug is released into the market, and Pharmacovigilance where the safety of marketed drugs, biologics or medical devices are monitored.

The focus of clinical research is wide enough to include important items such as data management, medical writing, regulatory consultation, and biostatistics.