Why are there clinical trials?

Carefully conducted clinical trials are the safest and surest way to find treatments that work in people. A clinical trial is one of the final stages of a long and careful research process which starts with drug discovery, formulation development, animal studies, and toxicology studies in animals and culminates in studies on humans. Studies are done with patients to find out whether promising approaches to disease prevention, diagnosis, and treatment are safe and effective.

What are the phases of clinical trials?

Most clinical research that involves the testing of a new drug progresses in an orderly series of steps, called phases. This allows researchers to ask and answer questions in a way that results in reliable information about the drug and protects the patients.

Clinical trials are usually classified into one of three phases:

Phase I trials: These first studies in people evaluate how a new drug should be given, how often, and what dose is safe. A phase I trial usually enrolls only a small number of patients, sometimes as few as a dozen.

Phase II trials: A phase II trial continues to test the safety of the drug, and begins to evaluate how well the new drug works.

Phase III trials: These studies test a new drug, a new combination of drugs, or a new surgical procedure in comparison to the current standard. A participant will usually be assigned to the standard group or the new group at random. Phase III trials often enroll large numbers of people.

Phase IV trials: These trials typically continue to investigate a drug after its initial approval from the regulatory authorities. In this phase the focus is on further evaluation of the use for which approval was secured, for comparison to or combination with other established drugs and to generate more data on safety under broader use. Phase IV trials are an important tool to strengthen the understanding of the drug and to give guidance to prescribers and patients on the safe and appropriate use under various clinical conditions.

There is a perception that human beings are used as Guinea pigs in Clinical Trial – Is this true?
As mentioned above clinical trials in human beings are conducted quiet late in the drug development process after sufficient safety, toxicology & efficacy data is available in animals. Additional safeguard is in the form of Phase 1 trials which are conducted on small number of healthy human volunteers with very close assessment on their safety. Only after data is available from such studies, patients who suffer from the disease are exposed to the Investigational New Drug. Moreover, at each stage the data is shared with Regulatory agencies [Drug Controller General of India (DCGI), US FDA, EMEA etc] and with Ethics Committee of the Hospital/ Institute where the trial is conducted. Only after approval from both the regulatory agencies and ethics committees, can the trial be started in the next phase. Moreover, all the patients/ human volunteers are given the informed consent form that contains the information on the trial and the study drug which they are encouraged to discuss with their relatives and with their treating physicians and then take informed decision to participate in the clinical study. Additionally, in India, this entire consent process is audio video recorded. Hence it would be unfair to state that patients in trials are used as Guinea pigs.