The discovery and development phase (also known as basic science research) is conducted to improve knowledge and understanding of a disease, what causes it and how it can potentially be treated. This form of research takes place in a laboratory, with investigators usually focusing on a particular question. Basic science research has provided many important breakthroughs in medical treatments and diagnostic technology.
The pre-clinical research phase is a key step in the development of a potential new treatment and takes place before a clinical trial can begin in humans. The main goal of a pre-clinical research study is to determine safety. A compound will normally only be tested in patients once it has been shown to be effective and safe in clinical trials with an animal model that reliably reflects the human disease.
Clinical research is medical research that is carried out on humans. Individuals volunteer to participate in studies that aim to uncover better ways to treat, prevent, diagnose and understand human disease. Clinical research can include both clinical trials and natural history studies/registries, which provide valuable information about disease progression and characteristics.
Clinical trials are used to determine whether new drugs or treatments are both safe and effective. Carefully conducted clinical trials are the fastest and safest way to find treatments that work. Many new medicines and treatments are found to be helpful and safe in test tubes and in animals. They must also prove safe and effective in humans before doctors can prescribe them.
Ideas for clinical trials usually come from researchers. Once researchers test new therapies or procedures in the laboratory and get promising results, they begin planning Phase I clinical trials. New therapies are tested on people only after pre-clinical studies – laboratory and animal studies – show promising results.
Clinical trials of experimental drugs proceed through four phases:
A registry can be used to gain an overview of the patient population within a particular disease community. It will hold information regarding disease history, characteristics, symptoms, treatments and clinical outcomes. The information collected will provide a valuable resource that can aid research and progress towards therapy development.
Q. What is a protocol?
A. All clinical trials are based on a set of rules called a protocol. A protocol describes what types of people may participate in the trial; the schedule of tests, procedures, medications, and dosages; and the length of the study. While in a clinical trial, participants are seen regularly by the research staff to monitor their health and to determine the safety and effectiveness of their treatment.
Q. Who can participate in a clinical trial?
A. All clinical trials have guidelines about who can get onto the study. Guidelines are based on such factors as age, type of disease, medical history and current medical condition. Before you join a clinical trial, you must qualify for the study. Some research studies seek volunteers with illnesses or conditions to be studied in the clinical trial, while others need healthy volunteers.
The factors that allow someone to participate in a clinical trial are called inclusion criteria and the factors that keep them from participating are called exclusion criteria. It is important to note that inclusion and exclusion criteria are not used to reject people personally. Instead, the criteria are used to identify appropriate participants and keep them safe. The criteria help to ensure that researchers will be able to answer the questions they plan to study.
Q. What is a placebo?
A. A placebo is an inactive pill, liquid, or powder that has no treatment value. In clinical trials experimental treatments are often compared with placebos to assess the treatment’s effectiveness. In some studies, the participants in the control group will receive a placebo instead of an active drug or treatment.
Q. What is a control or control group?
A. A control is the standard by which experimental observations are evaluated. In many clinical trials, one group of patients will be given an experimental drug or treatment, while the control group is given either a standard treatment for the illness or a placebo.
Q. What is a Control Group?
A. A control group in an experiment is the group which does not receive any treatment. This particular group is used as a benchmark against which other test results can then be measured, as this group includes individuals who are very similar in many ways to the individuals who are actually receiving the treatment (in terms of age, gender, race or other factors).
The primary function of a control group is to be used as a point of comparison, so that it can be isolated in testing whether the experiment did (or did not) affect the patients in the ways that were originally intended. Members of the control group are often given placebos and do not know that they are part of the control group. This is so that the researcher can determine the effectiveness of what is being studied.
Q. What is a blinded or masked study?
A. A blinded or masked study is one in which participants do not know whether they are in the experimental or control group in a research study. Those in the experimental group get the medications or treatments being tested, while those in the control group get a standard treatment or no treatment.
Q. What is a double-blind or double-masked study?
A. A double-blind or double-masked study is one in which neither the participants nor the study staff know which participants are receiving the experimental treatment and which ones are getting either a standard treatment or a placebo. These studies are performed so neither the patients’ nor the doctors’ expectations about the experimental drug can influence the outcome.
Q. What protections are there for people who participate in clinical trials?
A. The government has strict guidelines and safeguards to protect people who choose to participate in clinical trials. Every clinical trial must be approved and monitored by an Ethics Committee in the UK (Institutional Review Board in the USA) to make sure the risks are as low as possible and are worth any potential benefits.
An Ethics Committee is an independent committee of physicians, and lay people that ensures that a clinical trial is ethical and the rights of participants are protected.
All institutions that conduct or support biomedical research involving people must have an Ethics Committee that initially approves and periodically reviews the research.
Kim Stratton, Chief Executive Officer at Orphazyme, commented, “We are delighted with FDA’s decision to grant Breakthrough Therapy Designation to arimoclomol for NPC. Arimoclomol has been shown to have a clinically meaningful effect on disease progression in NPC that is further supported by a biomarker effect indicating an effect on the biological underpinnings of the disease and a favorable safety and tolerability profile. We are committed to bringing this product to patients as soon as possible. Breakthrough Therapy Designation is designed to expedite the development and review of products for serious diseases with the direct involvement of senior staff and we look forward to working closely with the FDA to further advance arimoclomol. Our preparations for filing in the US are underway and we are on track to submit a New Drug Application in H1 2020.”Read more