How is Clinical Testing for a newly discovered medicines done?

Answer 1

Drug discovery research is usually done at Pharmaceutical companies using cultured cells or small animals to screen for beneficial effects.

If a candidate drug shows promise in curing a test disease, it undergoes numerous safety tests according to Federal Rules and Regulations.

If it passes those tests, and still appears to remedy a disease modelled in animals, the company will accumulate all its manufacturing and testing information, including a plan for testing in humans that has been reviewed by and ethics committee, and send this huge document to the FDA to apply for an IND (Investigational New Drug exemption).

The IND allows use of this experimental drug in human volunteers only under specific conditions defined by FDA Regulations in chapter 21 of the Code of Federal Regulations [21 CFR parts 312, 50, 56 etc.] Otherwise, use of any drug that is not approved by the FDA is a violation of the Food Drug and Cosmetic Law and the persons using it could be arrested. Applying for an IND allows the testers to be exempt from being seen as violating this law.

The first time the drug is tested in human volunteers is called a Phase I test. It is primarily a safety test to determine if the IND drug causes harm.

Before any drug is tested in humans, the protocol for use of the drug, the informed consent document that tells the patient about the risks and benefits (if any) of participating in the study or receiving the drug, and other related documents are presented to an institutional review board (IRB) that determines if the rights and welfare of the human volunteers will be adequately protected and if the informed consent statement honestly describes what will be done, what risks the subjects may experience and who will be responsible for any costs or compensation for injuries related to participation. The IRB members must not have any conflicts of interest with regard to the study team or the sponsor of the study.

The Phase I tests may start with very low doses and increase the dose until some unpleasant or adverse result occurs. This is done to see if the levels of drug that benefitted the animals in the model experiments can be reached in humans without unpleasant symptoms or toxicity. If the drug passes these initial safety tests, then it can undergoe additional safety tests [Phase II and III] in human patients who have the target disease. As it continues to show positive effects without harming anyone, more patients may be enrolled until enough are being given the drug to result in a definitive test of whether the drug actually can cure the disease, prolong life or reduce symptoms. The FDA then looks at all this data and if no serious problems persist they approve the drug for marketing.
While all of what I have said pertains to drug development in the US, the ICH – International Conference on Harmonization of Technical Requirements for Registration of Pharmaceuticals for Human Use guidelines allows pharmaceutical companies in other countries to have their products licensed by the FDA if they meet these harmonized requirements

Answer 2

Target persons identified. Use of drug tentatively explained. Permission from volunteers obtained. Target grouped agewise genderwise body weightwise and otherwise. Trial group and control group are marked unannounced. Minimum dose exhibited. Effects of drug and side effects studied. Recorded .Drug elimination studied. Groups requested to report periodically for follow up. Results published. More the number of target ,more authentic the result. ..