In order to develop a new drug, it is necessary to synthesize/develop a promising molecule (lead) that is a candidate for becoming a new drug for a given therapeutic indication(s) and perform the first tests and chemical optimization on its effectiveness, non-toxicity and characteristics that allow its formulation to become a drug. The potential drug should then proceed to preclinical in vitro and in vivo testing to determine whether it is safe to be tested in humans.
The clinical trial stage (performed in humans), with the drug already formulated in some pharmaceutical form, comprises phase I, II and III studies. The time it takes for a drug to be released to the market from the discovery of the molecule is approximately 12 to 15 years. On average, USD 1.38 billion is spent for each new drug developed.
A clinical study is a controlled scientific research for the purpose of answering a question about a specific therapeutic intervention, which may be with a drug, a health product, a vaccine, etc., usually to evaluate its safety and efficacy.
Informed consent is the process in which all candidates volunteering to participate in a clinical study receive detailed information and have the opportunity to clarify questions about the trial so that they can make a decision on whether or not to participate. It is an ongoing process throughout the duration of the research to inform and explain any updates or modifications to the methodology or the test product so that participants can continuously reaffirm their desire to remain in that study.
The consent process is conducted by the team responsible for the research in a given institution through a document called the Informed Consent Form, which is approved by an Ethics Committee and includes details on the study, such as its purpose, duration, required procedures, key contacts, risks and potential benefits involved in their participation. Anyone who decides to participate in the study must sign the Informed Consent Form. Regardless of the signature of this document, the participant may decide to leave the study at any time.
A protocol is a detailed plan that determines what will be done in a given clinical study. The plan is carefully designed to protect participants’ health and well-being above all else, as well as to provide data on specific hypotheses about a particular drug. A protocol describes the study participation criteria, the testing schedule, procedures, medications and dosages, and the duration of the study. While participating in a clinical study, participants who follow a protocol are regularly examined by the study team to monitor their health and to determine the safety and effectiveness of their treatment.
A placebo is an inert tablet, liquid or powder that has no active ingredient in its formulation. In clinical studies, experimental treatments may be compared to placebos to assess the experimental treatment’s effect on indications where there is no established treatment standard. In some studies, a group of participants called the control group will receive a placebo instead of an active drug or experimental treatment, or have a placebo added to the base therapy to provide masking of the treatment.