Drug Development Process_ Fro... (1).pptx

Another brief discussion on drug development and it's administration

1. Author: Rehan Yaqub Kiani Created with Pi Drug Development Process: From Concept to Market

2. CONTENTS 1. Chapter Outline 3. New Drug Application 2. Understanding the Drug Development Process 4. Launch

3. 01 Chapter Outline

4. Market Analysis The typical journey from concept to market for a drug passes through many groups of people, with a variety of responsibilities and skills. Specialty fields in the process include finance, biology, chemistry, law, and medicine. The overall development process is sometimes called integrated drug discovery to highlight the cooperative effort of various teams within a corporation. This chapter presents an overview of the drug discovery process. Awareness of the process as a whole is vital for understanding the specific role of medicinal chemistry in the larger picture. Because this chapter touches on many ideas of drug discovery, some terminology is left loosely defined until it can be properly described in later chapters. The glossary at the end of the text should be helpful.

5. Market Analysis 1 Pharmaceutical companies operate like other for-profit businesses. 3 New drugs are generally developed for diseases affecting a large number of patients. Of greatest interest to the pharmaceutical industry are diseases for which no effective treatments exist. The development of a drug for a new category likely has a large potential market. Desirable new categories include drugs for disorders such as Alzheimer’s disease, obesity, Parkinson’s disease, type 1 and 2 diabetes, and asthma. Creating drugs that are effective in a new category is extremely challenging. The risk of failure is high. From a humanitarian perspective, a market-driven approach to drug development often leaves unaddressed the diseases of less-developed nations. Nations with a low per capita income are less able to contribute to an economically profitable drug. Some scientists have attempted to address this situation. For example, Dennis Liotta of Emory University spearheaded the creation of iThemba Pharmaceuticals in 2009 in Johannesburg, South Africa. iThemba specifically targets novel treatments for HIV/AIDS, malaria, and tuberculosis, three exceptionally common diseases in Africa. The immense cost of developing drugs ranges from US$802 million (2000) to US$1.2 billion (2010). 2 4 Successful drugs often target conditions that require long-term management, like high blood pressure and cholesterol. The following Case Study describes the development of an effective drug for acid reflux, highlights the profitability of such a drug, and demonstrates how quickly competing companies create their own therapies.

6. Learning Goals for Chapter 2 Understand the sequence of events required to bring a drug to market. Know the role of the drug target and how its activity is screened. Recognize the type of information gained in the various animal and human trials. Understand the interaction between regulatory agencies and pharmaceutical companies. Appreciate the overall effort and cost required to develop a new pharmaceutical.

7. 02 Understanding the Drug Development Process

8. Market Analysis From a business standpoint, pharmaceutical companies are no different from any other for-profit business. All sell products to consumers with the intent of generating a profit that will be invested back into the company or distributed to its owners or investors. Just like all businesses, drug companies develop only products that are anticipated to be profitable. The cost of developing a drug and bringing it to market is immense: US$802 million in 2000, according to one estimate. This estimate was revised upward to US$1.2 billion in a follow-up study performed in 2010. Cost estimates vary wildly, but one point is always clear: drug development is very expensive. The profit from each successful drug must be large enough to cover not only the costs of the successful drug but also the expenses of other drugs that were cut in development. For this reason, new drugs are normally developed for diseases and conditions that affect a large number of people. A survey of successful (i.e., profitable) drugs reveals many that treat problems of high blood pressure, high cholesterol, and pain management in arthritis. All affect large segments of the population and treat conditions that require long-term management. A drug for any of these disease conditions will enjoy a large number of patients and subsequent prolonged sales.

9. Target Selection Informed of the specific disease of interest to the pharmaceutical company, the molecular biology team tries to identify a target that may be exploited to change the state of the disease. Many different types of targets are known, but the most frequently encountered are enzymes and receptors. The target is the site of action for the drug and controls a biological process related to the diseased state of a patient. Although a company does not need to know the target of a drug, knowing the target and its structure is extremely beneficial to the discovery team and can make for a more successful drug search.

10. Target Selection Finding a Target The classical approach for discovering a drug target is to study the inner workings and signal pathways in a cell. Traditional Methods Finding a target can be a challenging task for the molecular biology group. The difficulty of this task depends on the target. For diseases that are fairly well known, considerable information likely exists in the literature. Targets that are promising for drug intervention are often described as druggable. In addition to the literature, previous research performed in-house by the pharmaceutical company may provide a wealth of proprietary information to facilitate discovery of a target. Proteins that play key roles in the processes are candidate targets. The use of genomics to find previously unknown proteins with potential value as targets has become much more commonplace.

11. Stage of Drug Development Description Lead Discovery Identifying compounds with modest activity. Lead Optimization Modifying structures to increase activity. Animal Trials Testing on animal subjects for safety. Phase Trials Clinical testing on human subjects for efficacy. Assay Development Quality assay development is crucial for successful drug development. An assay is a method by which the activity of a compound is measured. As compounds are tested or screened in assays, compounds that show activity help steer the medicinal chemistry team toward designing a new compound with even higher activity. 1 Binding Information can tell how a drug interacts with a target. 2 Compound Behavior refers to how a drug is absorbed, distributed, metabolized, and eliminated from an organism.

12. Clinical Trials Before a drug may be tested on humans, it must first be tested on animals. Tests on humans are called clinical trials, and animal trials are often referred to as preclinical trials. The primary goal of clinical trials is to evaluate the drug's effectiveness and safety.

13. Clinical Trials 1 Phase I Trials • The first introduction of the IND into humans. • Focus on safety and tolerance. 2 • Typically involve 20 to 80 subjects. Phase II Trials • The first use of the drug on diseased patients. • Involves several hundred patients. 3 Phase III Trials • Can involve hundreds to thousands of patients. • Focus on effectiveness and long-term safety.

14. 03 New Drug Application

15. New Drug Application The final step in FDA approval is for the pharmaceutical company to file a New Drug Application (NDA). This application contains a full summary of in-house screening, animal trials, and information on the synthetic route. All the previously mentioned stages of the IND’s development are data for presentation to the FDA.

16. 04 Launch

17. Launch Once a drug reaches the market, the long-term activity and safety of the drug continue to be monitored. This ongoing monitoring period is called phase IV, with the FDA overseeing the continued safety and efficacy of the marketed drug. This concludes the structured Markdown presentation from the provided text about drug development. Each section has been formatted according to the specified guidelines while maintaining the integrity of the original content.

18. Thank You