Introduction and Drug Development-Questions and Answers Graded A+

Introduction and Drug Development-Questions and Answers Graded A+ Drug Development Process - ANSWER-The investment of time, money, and scientific effort required to bring a new drug from a new class of medication to the market in the United States is estimated by the Pharmaceutical Researchers and Manufacturers of America (PhRMA) to take about 10-15 years and somewhere around $1-2 billion dollars. Of course exceptions exist. Resources to develop biotechnology drugs are often greater than this, while the time and cost to develop subsequent drugs in a pre-existing family of medications is usually less. The effort to develop and produce generic versions of previously approved brand name drugs is also less. About 30-40 new drugs approved per year by FDA. Main emphasis is on practicality of drug administration which is driven by pharmacokinetic properties (how is it absorbed, excreted, how many times does it need to be taken, etc.) and on drug toxicity (is it safe?). First is animal trials and then next is human trials. Need to see if main emphasis described above works in both animals and then humans. Drug development for most drugs includes the following steps: • Drug discovery - a process whereby many (often thousands, ~5000) of compounds are screened and tested. • Pre-clinical testing - to evaluate the potential risks/benefits of advancing drug candidates into clinical testing. Usually involves ~250 candidates, with about 5 making it to clinical trials. Primary objectives are establishing pharmacokinetics and safety (toxicity profile) • Investigational New Drug application (IND) - required to be filed with FDA prior to initiation of clinical trials. Provides an opportunity for FDA to make suggestions on trial designs before they begin. Takes place after about 3-6 years of drug discovery and pre-clinical testing. • Clinical trials - which are most often further divided into three distinct phases. Phase 1 (20-10 History of FDA Regulation - ANSWER-Oversight of drug safety and efficacy by the FDA has evolved over the years since the original passage of the Food and Drugs Act. This act was passed by Congress and signed into law by President Theodore Roosevelt in 1906. It was designed mainly to prevent the interstate commerce of misbranded and adulterated foods, drinks and drugs. It wasn't until 1938 that the FDA included provisions to help ensure drug safety when the Act was amended and renamed The Federal Food, Drug and Cosmetic Act due to the use of diethylene glycol (antifreeze) as a solvent for the antibiotic sulfanilamide, which lead to the death of hundreds of kids. In 1951 the Durham-Humphrey Amendment was implemented to define the kinds of drugs that cannot be safely used without medical supervision. While The Food, Drug, and Cosmetic Act of 1938 had first distinguished between prescription and nonprescription medicines, the Durham-Humphrey Amendment more clearly defined the two categories. In 1962 the Kefauver-Harris Amendments were passed to further strengthen drug safety oversight and to, for the first time, ensure drug efficacy after the effects of thalidomide as a teratogen were observed leading to birth defects in children. The provisions regarding drug efficacy have since evolved into the three phases of clinical testing most new drugs are now subjected to in the drug development process. By the early 1990s the amount of data being generated by the drug development process was so large the time required for FDA to review and act upon a new drug application request was averaging 3 years. In 1992 the Prescription Drug User Fee Act (PDUFA) was passed to require drug manufacturers to pay fees to the FDA for product applications and other services. These funds are to be used by FDA to support the staff required to handle applications and shorten review t