White Paper: Update on testing for a new drug You are working on a drug called C
ID: 192997 • Letter: W
Question
White Paper: Update on testing for a new drug You are working on a drug called Cerebrolysin (CBL) which is a mixture of peptides that have been shown to treat Alzheimer's disease in mice. You have just finished the preclinical trials and are about to submit your regulatory filings to the FDA when your biggest investor asks for a progress report. They request 2 things. First, explain the steps you have taken in regards to coming up with your product and preclinical trials in animal models and what filing you are about to submit. Then explain the 3 parts of the clinical trials and why you may (or may not) be able to combine the steps of the trials and what filings you have to do when you are done with the trials. rationExplanation / Answer
Drug Development
It is the process of developing a drug right from its starting stage and completes once it is launched in the market. During the process of drug development, after the lead compound of the drug is discovered through drug discovery, through various steps of preclinical trial and clinical trial, it is manufactured and is available in market after being approved drug regulatory regulating body.
The process of drug development requires intensive research and investment, and the average time for any drug to come to market shelves is nearly 10 to 15 years.
a)Basic Research before drug Discovery
b)Drug discovery and development
It includes discovering the lead molecule for the potential drug after learning more about the disease pathway. The scientists make a research on the genetic and cellular factors that causes the particular disease, find out chemicals and biological substances that have drug like effect on these biological markers.
After studying the genetic and cellular factors associated with the disease, scientist tends to find the target on which a potential drug may acts to bring desired effect. After finding out the drug target, many compounds are identified and tried for interaction to bring therapeutic effect.
After finding the potential drug targets there is a comparison made among all so that drug targets for their ability to regulate a compound. After several test, they confirm and validate the drug target for which the potential drug is manufactured.
Compare known substances to each to understand and analyse their likelihood to treat a disease by interacting with a drug target. Sometimes lead compounds can also be developed as collection of individual molecules for imparting properties needed by drugs and all molecules in question are tested. Through the process of lead optimization all the candidates for potential drug formulation are compared where the one with greatest potential for safe usage with maximum benefit in eliminating the diseases is selected.
C) Pre – Clinical Trials – It is testing of the drug in lab and on animals. Both in – vivo(on living animals) and in-vitro(labs) tests are very important in determining the effectiveness of the potential drug component. It is important so that further scrutiny is done on the lead compounds in order to send only 1 or 2 compound to clinical trial.
D) Clinical Trials
It is one of the most crucial steps in drug discovery where drugs are tested on human volunteers to judge its effectiveness and medicinal benefit. It starts with submission of application to regulatory body for permission to start the test on humans. The test includes both administering the drug on healthy volunteers and patients.
Clinical trial is very time consuming and expensive proves which may take on an average 6 to 7 years for completion. Before the trial can begin companies need to file investigational new drug application with FDA which gives the findings of the potential drug candidate and an outline of how clinical trials will be conducted.
A protocol for testing needs to be developed in order to gain approval from the reviewing committee and authority. All the clinical trials protocol must be approved by Institutional review board (committee of physicians, community advocates and others who judge if a clinical trial is ethical or not) or ethics committee.
Phase I clinical Trials – It helps in understanding if the drug is safe for human use and also helps in judging the drug tolerability in humans. It is usually conducted on small number of healthy volunteers – 100 or less. It is usually to study pharmacokinetics as well as pharmacodynamics of the drug.
Phase II clinical trials – In this test is done on patient volunteers who are suffering from the disease the drug is made for. It consists of almost 100 to 500 volunteers. In this comparative study is made between patients who are treated with this particular drug and any other drug that already exist for that particular disease. It determines the safety and effectiveness of the drug. A comparative study is already made between patients who receive the test drug and the ones who are given placebo.
Clinical Phase III – It uses large number of volunteers as last stage of drug trial which may range from 1000 to 5000 patients. It is the last step in determining the safety and effectiveness of the drug on human body. The drug is tested among thousands of patient volunteers which determine its effectiveness.
E) Approval
After all the clinical trials if the drug is safe and effective, the drug manufacturing company needs to submit new drug application(NDA) or Biologics license application to FDA for approval so that it can be manufactured for marketing purpose. The application contains details about all the data, results , effects and analyses of pre – clinical testing and clinical trials. The process of approval may take anywhere between 6 months to two years.
FDA follows expedite approaches in review of medicines if.
After FDA goes through everything in the report submitted and after estimating the potential benefit and safety, decide to grant approval or not. At times additional research may be required and more trials may be required before granting approval. After the drug is approved it can be manufactured and marketed.
F) POST MARKETING MONITOR
After the drug is approved for marketing, it is still under scrutiny with help of post approval research to monitor safety of the drug and its effects on patients. The purpose is to maintain the safety and efficiency of the marketed drug. FDA requires the companies to perform Phase IV trails on broader range of patients for its ongoing effect.
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