Clinical trial phases explained

Dr. GAURAV KUMAR
PHARMD, C.P.P.M,
D.C.M.S, B.Ph.,
1DR.GAURAV KUMAR PHARMD, CPPM, CGCPh 5/21/2019

1. Clinical trials are studies performed with human subjects to test new drugs or
combinations of drugs to improve the diagnosis of disease and the quality of life of the
patient.
2. A clinical trial is a research study designed to test the safety and effectiveness of new
treatments for disease.
3. A clinical trial is a research study conducted to investigate new treatments such as a
new drug compound in human volunteers or research participants.
4. Each clinical trial is designed to learn about a potential treatment and its effect on humans.
5. Any investigation in human subjects intended to discover or verify the clinical,
pharmacological, and other pharmacodynamic effect of an investigational product and to
identify any adverse reactions to an investigational product, and to study ADME of an
investigational product with the object of ascertaining its safety and efficacy.

1. To improve medical knowledge: related to the treatment, diagnosis and prevention of
illnesses.
2. New medication: To determine the correct dose.
3. To improve treatments: Medicine available to patients.
4. To determine whether the drug will treat the disease effectively in humans.
5. Safety of a new drug: Alone and in combination with other therapies.
6. Risks: of new therapeutic, preventative, or diagnostic products or interventions.
7. To determine whether a new treatment is better than standard treatment.

A. Treatment Trials - test new treatments, new combination of drugs or new approaches to
surgery or radiation.
B. Prevention Trials - look for better ways to prevent diseases.
C. Diagnostic Trials - determine better tests or procedures for diagnosing a particular
disease or condition.
D. Screening Trials - test the best way to detect or treat diseases.
E. Quality of Life Trials - explore and measure ways to improve the comfort and quality of
life of people with a chronic illness.

 Clinical trials can be sponsored by pharmaceutical companies, medical technology
companies, biotechnology companies, hospitals and other funding bodies.
 Trials can occur at sites as varied as hospitals, universities, doctors’ offices, community
clinics, or in the offices of clinical-trial contractors.

 First, a Pre-Clinical Trial must be done before the Clinical Trial starts.
 Preclinical trial – research on a new drug or procedure, usually done on animals, to
learn about mechanisms of action, determine how well the treatment works, and see
if it is safe to test on humans.
 Clinical trials are usually conducted in humans in the following phases:

 Also called Human Micro-dosing studies.
 Gives no data on safety or efficacy. being by definition a dose too low to cause any therapeutic effect.
 Subjects: 10-15 healthy volunteers.
 Dose: it is 1/100th of therapeutic dose.
 Primary goal:- Pharmacodynamic and Pharmacokinetic particularly oral bioavailability and half-
life of the drug.

 The first studies in humans are Phase I trials. Initial studies to determine the
Pharmacokinetic and pharmacologic actions of drugs in humans, the side effects
associated with increasing doses, and to gain early evidence of effectiveness; may
include healthy participants and/or patients.
 Initial safety trials on a new medicine. Phase I trials are sometimes conducted in
severely ill patients.
 Pharmacokinetic trials are usually considered Phase I trials regardless of when
they are conducted during a medicine's development.

1. Aim of this trial: is to determine if the treatment is safe, what the expected side
effects are, and to determine a dose for the treatment.
2. These are usually only conducted with a small number of healthy volunteers (20-
80) with close safety monitoring.
3. The trials usually last a few weeks to months.
4. Objective: is to determine whether the drug is safe for use in humans and how the
drug behaves in the human body.
5. Primary goal: To assess the safety (pharmacovigilance),tolerability,
pharmacokinetics, and pharmacodynamis of a drug.

 The aim of these trials: is to establish how well a treatment works.
 Phase II trials are performed on larger groups (100-300) and are designed to assess
how well the drug works, as well as to continue Phase I safety assessments
 These trials are usually conducted with a small number of supervised patients.
 Specialists in the field of the disease will monitor the patients and review their
progress regularly.
 Clinical trials that are conducted in a small number of patients with the illness being
researched upon. The objective is to explore the therapeutic efficacy of the drug and
to further evaluate its safety. A key objective of this phase is to determine the dose
for phase III trial.

 Most phase II studies are randomized trials where one group of patients receives the
experimental drug, while a second "control" group receives a standard treatment or
placebo. Often these studies are "blinded" which means that neither the patients nor
the researchers know who has received the experimental drug. This allows
investigators to provide the pharmaceutical company and the FDA with
comparative information about the relative safety and effectiveness of the new drug.
About one-third of experimental drugs successfully complete both Phase I and
Phase II studies.

 Phase 2a: How much drug should be given
 Phase 2b: How well the drug response to the dose

 These trials involve a larger number of patients. These clinical trials help to
demonstrate or confirm the therapeutic efficacy of the drug and to collect more
information that will allow the drug to be used safely.
 Their aim is to show whether or not a new treatment is better than the current
standard treatment.
 The trial involves 2 groups: one group of patients will get the standard treatment,
and the other group will get the newer treatment. It is usual for the patient and the
doctor not to know which of these treatments the patient is receiving. Because the
trials involve a larger number of patients, this study also gives a better
understanding of the potential side effects of the new treatment. Once Phase III is
complete, a pharmaceutical company can request FDA approval for marketing the
drug.

 studies, often called Post Marketing Surveillance Trials, are conducted after a drug
or device has been approved for consumer sale. Pharmaceutical companies have
several objectives at this stage: (1) to compare a drug with other drugs already in
the market; (2) to monitor a drug's long-term effectiveness and impact on a patient's
quality of life; and (3) to determine the cost- effectiveness of a drug therapy relative
to other traditional and new therapies. Phase IV studies can result in a drug or
device being taken off the market or restrictions of use could be placed on the
product depending on the findings in the study.

 The aim of these trials is long-term surveillance of the treatment. Many thousands
of patients are usually enrolled in phase IV trials.
 Clinical trials that are conducted to gather additional information about a drug's
safety, efficacy, and optimal use. They are also sometimes known as post-marketing
studies.

 A clinical trial is usually conducted at hospitals or clinics. The location depends on
who is conducting the clinical trial.

 A clinical trial is conducted by an Investigator who must be a locally registered
doctor or dentist.

 Clinical trials are an essential part of the regulatory process. They provide the basis
for the development and marketing of new drugs, biological products, and medical
devices. The data collected from clinical trials to provide the evidence to show that
the product is safe and effective is used to support product registration or marketing
authorization of new medicines and medical devices.

 A clinical trial is conducted in accordance with a research plan called a protocol that
has been designed to address the research question and safeguard the rights, safety
and well-being of participants. The protocol outlines the eligibility criteria thereby
defining the population being researched upon for the clinical trial. The eligibility
criteria usually define the age, gender, type and stage of an illness, previous
treatment history, and other medical conditions. Participants must sign an informed
consent form before they can be screened for eligibility assessment for a clinical
trial.

 The length of a clinical trial depends on what is being researched upon. Participants
are informed about the duration of participation in the clinical trial during the
informed consent process.

 A clinical trial will involve new medications or treatments for which not all the side
effects and risks are known. Sometimes the effectiveness of a new treatment will
not be fully known until there are clinical trials. For this reason, new drugs may not
always work, or they may be less effective than the current standard treatment.
Patients must also consider that this treatment may work for some people, but not
necessarily for them. Research also continues to determine why some treatments
work in some people and not in others.

 Clinical trials allow access to new medications and treatments before they would
otherwise be widely available. If the drug is effective, those enrolled in trials will be
among the first to benefit. Being a participant in a clinical trial also improves the
understanding and knowledge of many conditions, and has the potential to benefit
future suffers of your disease. When you are an enrolled participant in a clinical
trial, your health care is provided by a leading physician in that field (e.g. a
consultant or an experienced, senior registrar). This allows close monitoring of any
side effects of the treatment, limits any potential problems, and allows early
recognition of problems. It also ensures that you receive the best available care and
monitoring while you are involved. There is some evidence that suggests cancer
patients who are involved in clinical trials have a better outcome than those who are
not.

 Participants should undergo all the study procedures outlined in the informed
consent form and follow the advice given by the study team.

 In many studies, the new drug is compared to a placebo. A placebo is a product that
looks like the new drug, but it does not have the active ingredient in it. People do
not know that they are getting the placebo.

 Blind and Double Blind Trials are frequently done.
 A Blind Trial is a trial in which the patients do not know if they are receiving the
treatment or a placebo.
 A Double Blind Trial is a trial in which the patients and the researchers do not
know who is receiving the treatment.
 Why would the above be good ideas?

 Randomization is the process by which patients are assigned a group for the
Clinical Trial.
 Groups are assigned randomly, not purposefully.
 Some people will receive the new treatment, some may receive an already approved
treatment, and some may receive a placebo.
 If one treatment is found superior, the trial is stopped so that the fewest patients
possible receive the less beneficial treatment.

 For approximately every 5,000 to 10,000 compounds that enter preclinical testing,
only one is approved for marketing.
 Cost of the failures has to be borne by the price of the one success.

 Below are some estimates on the amount of time it takes for this process in cancer
treatment research.
 Pre-clinical Trials - 4.5 years
 Phases I-III - 8.5 years
 FDAApproval - 1.5 years
 Phase IV - Ongoing for the duration of the use of the drug
 How long is this whole process?

Clinical trial phases explained

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