Clinical trials I II III

Clinical trials and Phases

1. CLINICAL TRIALS Presentation by G. Mohana Priya S. Nandhini G. Nantha Kumar Course Teacher: P. Mala, Assistant Professor, Department of Biotechnology, PMIST. 3 April 2020 Drug Discovery and Development 1

2. Content : Drug discovery process. Clinical trials. Phases of clinical trials. Benefits of clinical trials. Risk of clinical trials. 3 April 2020 Drug Discovery and Development 2

3. Picture courtesy: https://www.slideteam.net/media/catalog/product/cache/960x720/5/_/5_staged_drug_discovery_process_Slide01.jpg 3 April 2020 Drug Discovery and Development 3

4. What is clinical trials? Clinical trials are scientific studies that allows to find ways to prevent, screen for, diagnose, or treat disease. i.e., these studies follow strict, scientific standards which protect patients and help produce reliable clinical trial results. The clinical trials are research studies involving people. Clinical trials produce high – quality data for healthcare decision making. They test whether particular treatment are safe and how well they work. 3 April 2020 Drug Discovery and Development 4

5. We need to know: Does a treatment work? Does it work better than other treatments? Does it have any side effects? 3 April 2020 Drug Discovery and Development 5

6. Clinical trials can study many things, such as: New drugs not yet approved by the U.S. FDA New uses of drugs already approved by the FDA, New ways to give drugs, such as in pill form, Use of alternative medicines, such as herbs and vitamins, New tests to find and track disease, and Drugs or procedures that relieve symptoms. 3 April 2020 Drug Discovery and Development 6

7. Phases of clinical trials: Preclinical Phase I Phase II Phase III FDA review Phase 4 3 April 2020 Drug Discovery and Development 7

8. Picture courtesy: https://www.google.com/url?sa=i&url=https%3A%2F%2Faidsinfo.nih.gov%2Funderstanding-hiv-aids%2Fglossary%2F63%2Fapproved-drug&psig=AOvVaw1-Fxj9RWbHnggL7jYMTSkH&ust=1583599820378000&source=images&cd=vfe&ved=0CA0QjhxqFwoTCKD3spunhugCFQAAAAAdAAAAABAM 3 April 2020 Drug Discovery and Development 8

9. Phase I : First test of a drug in humans, usually includes a small number of healthy volunteers(20 to 100), who are generally paid for participating in the study. These trials are often called clinical pharmacological trials. i.e., they are designed to establish the safety, tolerability, therapeutic action, pharmacokinetic and pharmacodynamic characteristics, and sometimes the initial efficacy rate in humans. This also investigates the side effects that occur as dosage levels are increased. About 70% of experimental drugs pass this phase of testing. 3 April 2020 Drug Discovery and Development 9

10. Phase II : In phase II , a drug is evaluated for its efficacy and safety in patients with specific diseases. This phase of testing can last from several to two years, and involves up to several hundred patients. Often these studies are meant that neither the patients nor the researchers know who has received the experimental drug. This allows investigators to provide the pharmaceutical company and FDA with comparative information about the relative safety and effectiveness of the new drug As a rule, these are Placebo – controlled trials. 3 April 2020 Drug Discovery and Development 10

11. Phase II are divided into phases IIa and phases IIb. Phase IIa – to evaluate short-term safety of a drug. Phase IIb – to confirm clinical efficacy of a drug and determine the therapeutic dose range. About one-third of experimental drugs successfully complete both phase Ii a and phase Ii b studies. 3 April 2020 Drug Discovery and Development 11

12. Phase III: In phase III of clinical trials, the study drug is given to large groups of patients(thousands) of different age groups, with different concomitant diseases. This trials investigate all aspects of treatment including assessment of risk/benefit ratio. This large – scale testing, which can last several years , provides the pharmaceutical company and the FDA with a more thorough understanding of the effectiveness of the drug or device, the benefits and range of possible adverse reactions. 3 April 2020 Drug Discovery and Development 12

13. Phase III trials are often randomized controlled studies. 70% to 90% of drugs that enter phase III studies successfully complete this phase of testing. Phase III clinical trials may be divided into phases IIIa and IIIb as well Once phase III is complete, a pharmaceutical company can request FDA approval for marketing the drug. 3 April 2020 Drug Discovery and Development 13

14. Phase IV: Phase IV studies, often called Post Marketing Surveillance Trials , are conducted after a drug or device has been approved for consumer sale. The purposes of these trials are to identify differences between a new pharmaceutical product and other drugs in this pharmacological group , to compare its efficacy with already marketed analogs, and to demonstrate a new drug benefit in relation to health economics and to identify and to determine previously unknown or ill-considered side effects as well. As a result, the safety and efficacy of a drug may be reviewed periodically according to new clinical data on its use. 3 April 2020 Drug Discovery and Development 14

15. Benefits of clinical trials: You may get a new treatment for a disease before it is available to everyone. You play a more active role in your own health care . Researchers may provide you with medical care and more frequent health check-ups as part of your treatment. You may have the chance to help others get a better treatment for their health problems in the future. You may be able to get information about support groups and resources. 3 April 2020 Drug Discovery and Development 15

16. Risk of clinical trials: New drugs and procedures may have side effects or risks unknown to the doctors. New drugs and procedures may be ineffective, or less effective, than current approaches. Even if a new approach has benefits, it may not work for you. 3 April 2020 Drug Discovery and Development 16

17. Reference: https://www.nia.nih.gov/health/clinical-trials-benefits-risks-and-safety https://www.medtran.ru/eng/trials/trial_phases.htm https://www.centerwatch.com/clinical-trials/overview https://www.roswellpark.org/clinical-trials/risks-and-benefits 3 April 2020 Drug Discovery and Development 17