Medical researchers are constantly on the lookout for new or improved methods of treating illness or disease. However, it cannot be widely used until years of careful testing have been completed if they discover something valuable. Medical research studies are what connect medical research to the availability of a drug to physicians and patients. Clinical trials, drug trials, and drug studies are other names for research studies.
- Research studies are designed to test the effect of a medication or treatment on a group of volunteers, assess a drug’s ability to treat a medical condition, monitor the drug’s safety, and look for potential side effects.
- Research studies are carried out by trained doctors, nurses, and researchers. The study coordinator is in charge of the study’s day-to-day operations. The protocol is carried out under the supervision of the principal investigator (usually a physician).
- Pharmaceutical companies or other health organizations may fund research studies and design the protocol, a detailed set of guidelines—multi-center research conducted at several locations.
- In conducting research studies, there are three phases or steps. Before a new drug can be approved for public use, complete all three of these steps.
- Successfully, and all results must be known.
- Phase I studies are conducted on healthy volunteers who agree to take the study drug to assist doctors in determining the drug’s safety and the presence of any side effects. In addition, studies are conducted to determine how the drug is absorbed, metabolized, and excreted. Phase I studies typically involve a small number of subjects (20-100). Approximately 70% of new drugs will make it through this stage.
- Phase II studies assess the new drug’s efficacy in patients suffering from the disease or disorder being treated. The primary goal is to determine the new drug’s safety and effectiveness. Hundreds of patients usually take part. These studies are typically “dual-blind, randomized, and controlled.” The effect of the active drug compared to the effect of a placebo (inactive or “sugar” pill) in controlled studies. In a double-blind study, neither the investigator nor the study subjects know who is receiving the active drug and receiving a placebo. One-third of the medicines studied thoroughly in both Phase I and Phase II.
- Patients with the disorder being treated by the new drug are also used in phase III studies. These studies are carried out to understand better the effectiveness, benefits, and side effects of the study drug. These studies involve many subjects, ranging from hundreds to thousands—seventy to ninety percent of new medicines that enter Phase III studies complete this phase. If the results show a positive effect and safety profile, the company will submit the data and request FDA approval to market the drug.
- Participating in a research study is similar to going to a clinic or doctor’s office but with more personal attention. The subject of the study may have been referred by their doctor or may have learned about it elsewhere.
- Typically, preliminary screening for the study is done over the phone. The study’s actual age, symptoms, and medical history are reviewed, and the study’s details are discussed. If the caller appears to be eligible for the research and is interested in participating, they are invited to come in for the initial, or screening, visit.
- The screening is performed in a clinic, office, or hospital. The subject and the supervising physician sign the informed consent form after reviewing the information gathered over the phone. The subject receives a copy. May perform a physical examination, blood tests, and other tests. Following this, most studies have a period, usually a few weeks, where baseline information, such as the severity and frequency of symptoms, is collected.
- The patient returns to the clinic for the randomization visit at the end of the screening period. If the patient’s baseline data indicate that they are eligible for the study, they then randomized (usually by computer) to receive either a placebo or an active drug.
- During the treatment period, the subjects take the study medication regularly and keep track of their symptoms. Throughout the treatment period, the study coordinator makes regular visits. Medication use and symptoms are reviewed at the end of the treatment period. The study medication’s potential side effects are documented. Many studies include a follow-up period after the treatment period to assess how symptoms and possible side effects have changed. There may be one more visit or phone call to see how the subject is doing since stopping the study drug.
Consultation is a formal process for gathering input from our stakeholders. Stakeholders include all individuals and organizations who influence or are influenced by our work, such as:
- patients, carers, users of pharmacy services and members of the public
- pharmacists and pharmacy technicians
- pharmacy owners
- professional bodies and organizations
- other regulators
We believe people who are impacted by our work must have a say in how we operate. Effective consultation is critical to assist us in improving our work. It informs us and helps us achieve our purpose of protecting, promoting and maintaining the health, safety and wellbeing of patients and public members by upholding standards and public trust in pharmacy.