The United States Food and Drug Administration is a federal agency of the Department of Health and Human Services. The FDA is responsible for protecting and promoting public health through the control and supervision of food safety, tobacco products, caffeine products, dietary supplements, prescription and over-the-counter pharmaceutical drugs (medications), vaccines, biopharmaceuticals, blood transfusions, medical devices, electromagnetic radiation emitting devices (ERED), cosmetics, animal foods & feed and veterinary products.
An artificial heart is a device that replaces the heart. Artificial hearts are typically used to bridge the time to heart transplantation, or to permanently replace the heart in the case that a heart transplant is impossible. Although other similar inventions preceded it from the late 1940s, the first artificial heart to be successfully implanted in a human was the Jarvik-7 in 1982, designed by a team including Willem Johan Kolff, William DeVries and Robert Jarvik.
An orphan drug is a pharmaceutical agent that, developed to treat certain rare medical conditions, would not be profitable to produce without government assistance, due to the small population of patients affected by the conditions. The conditions are referred to as orphan diseases.The assignment of orphan status to a disease and to drugs developed to treat it is a matter of public policy that depends on the country. Designation of a drug as an orphan drug has yielded medical breakthroughs that might not otherwise have been achieved, due to the economics of drug research and development. Examples of this can be that in the U.S. and the EU, it is easier to gain marketing approval for an orphan drug. There may be other financial incentives, such as an extended period of exclusivity, during which the producer has sole rights to market the drug. All are intended to encourage development of drugs which would otherwise lack sufficient profit motive to attract corporate research budgets and personnel.
The United States Federal Food, Drug, and Cosmetic Act is a set of laws passed by the United States Congress in 1938 giving authority to the U.S. Food and Drug Administration (FDA) to oversee the safety of food, drugs, medical devices, and cosmetics. A principal author of this law was Royal S. Copeland, a three-term U.S. senator from New York. In 1968, the Electronic Product Radiation Control provisions were added to the FD&C. Also in that year the FDA formed the Drug Efficacy Study Implementation (DESI) to incorporate into FD&C regulations the recommendations from a National Academy of Sciences investigation of effectiveness of previously marketed drugs. The act has been amended many times, most recently to add requirements about bioterrorism preparations.
The Food and Drug Administration's (FDA) New Drug Application (NDA) is the vehicle in the United States through which drug sponsors formally propose that the FDA approve a new pharmaceutical for sale and marketing. Some 30% or less of initial drug candidates proceed through the entire multi-year process of drug development, concluding with an approved NDA, if successful.
A medical device is any device intended to be used for medical purposes. Significant potential for hazards are inherent when using a device for medical purposes and thus medical devices must be proved safe and effective with reasonable assurance before regulating governments allow marketing of the device in their country. As a general rule, as the associated risk of the device increases the amount of testing required to establish safety and efficacy also increases. Further, as associated risk increases the potential benefit to the patient must also increase.
Sarepta Therapeutics, Inc. is a medical research and drug development company with corporate offices and research facilities in Cambridge, Massachusetts, United States. Incorporated in 1980 as AntiVirals, shortly before going public the company changed its name from AntiVirals to AVI BioPharma soon with stock symbol AVII and in July 2012 changed name from AVI BioPharma to Sarepta Therapeutics and SRPT respectively. As of the end of 2019, the company has two approved drugs.
Drug development is the process of bringing a new pharmaceutical drug to the market once a lead compound has been identified through the process of drug discovery. It includes preclinical research on microorganisms and animals, filing for regulatory status, such as via the United States Food and Drug Administration for an investigational new drug to initiate clinical trials on humans, and may include the step of obtaining regulatory approval with a new drug application to market the drug. The entire process – from concept through preclinical testing in the laboratory to clinical trial development, including Phase I–III trials – to approved vaccine or drug typically takes more than a decade.
A ventricular assist device (VAD) is an electromechanical device for assisting cardiac circulation, which is used either to partially or to completely replace the function of a failing heart. The function of a VAD differs from that of an artificial cardiac pacemaker in that a VAD pumps blood, whereas a pacemaker delivers electrical impulses to the heart muscle. Some VADs are for short-term use, typically for patients recovering from myocardial infarction (heart attack) and for patients recovering from cardiac surgery; some are for long-term use (months to years to perpetuity), typically for patients with advanced heart failure.
Clinical research is a branch of healthcare science that determines the safety and effectiveness (efficacy) of medications, devices, diagnostic products and treatment regimens intended for human use. These may be used for prevention, treatment, diagnosis or for relieving symptoms of a disease. Clinical research is different from clinical practice. In clinical practice established treatments are used, while in clinical research evidence is collected to establish a treatment.
Rintatolimod, sold under the tradename Ampligen, is a medication intended for treatment of chronic fatigue syndrome (CFS). There is some evidence it may improve some CFS symptoms.
The Center for Drug Evaluation and Research is a division of the U.S. Food and Drug Administration (FDA) that monitors most drugs as defined in the Food, Drug, and Cosmetic Act. Some biological products are also legally considered drugs, but they are covered by the Center for Biologics Evaluation and Research. The center reviews applications for brand name, generic, and over the counter pharmaceuticals, manages US current Good Manufacturing Practice (cGMP) regulations for pharmaceutical manufacturing, determines which medications require a medical prescription, monitors advertising of approved medications, and collects and analyzes safety data about pharmaceuticals that are already on the market.
The Prescription Drug User Fee Act (PDUFA) was a law passed by the United States Congress in 1992 which allowed the Food and Drug Administration (FDA) to collect fees from drug manufacturers to fund the new drug approval process. The Act provided that the FDA was entitled to collect a substantial application fee from drug manufacturers at the time a New Drug Application (NDA) or Biologics License Application (BLA) was submitted, with those funds designated for use only in Center for Drug Evaluation and Research (CDER) or Center for Biologics Evaluation and Research (CBER) drug approval activities. In order to continue collecting such fees, the FDA is required to meet certain performance benchmarks, primarily related to the speed of certain activities within the NDA review process.
Expanded access or compassionate use is the use of an unapproved drug or medical device under special forms of investigational new drug applications (IND) or IDE application for devices, outside of a clinical trial, by people with serious or life-threatening conditions who do not meet the enrollment criteria for the clinical trial in progress.
Fast track is a designation by the United States Food and Drug Administration (FDA) of an investigational drug for expedited review to facilitate development of drugs that treat a serious or life-threatening condition and fill an unmet medical need. Fast track designation must be requested by the drug company. The request can be initiated at any time during the drug development process. FDA will review the request and attempt to make a decision within sixty days.
Thoratec Corporation is a United States-based company that develops, manufactures, and markets proprietary medical devices used for mechanical circulatory support for the treatment of heart-failure patients worldwide. It is a global leader in mechanical circulatory support devices, particularly in ventricular assist devices (VADs).
Avita Medical is a clinical and commercial company developing and marketing a range of respiratory and regenerative products. The first regenerative medicine product brought to the market by Avita Medical was ReCell spray-on skin for the treatment of burns. The two latest products are ReNovaCell, for Aesthetics and Plastic applications including skin trauma, and ReGenerCell for the treatment of chronic wounds. The Avita Medical regenerative product range is currently marketed in Europe, the Middle East, Africa (EMEA) and Australia.
The Food and Drug Administration Safety and Innovation Act of 2012 (FDASIA) is a piece of American regulatory legislation signed into law on July 9, 2012. It gives the United States Food and Drug Administration (FDA) the authority to collect user fees from the medical industry to fund reviews of innovator drugs, medical devices, generic drugs and biosimilar biologics. It also creates the breakthrough therapy designation program and extends the priority review voucher program to make eligible rare pediatric diseases. The measure was passed by 96 senators voting for and one voting against.
Breakthrough therapy is a United States Food and Drug Administration designation that expedites drug development that was created by Congress under Section 902 of the 9 July 2012 Food and Drug Administration Safety and Innovation Act. The FDA's "breakthrough therapy" designation is not intended to imply that a drug is actually a "breakthrough" or that there is high-quality evidence of treatment efficacy for a particular condition; rather, it allows the FDA to grant priority review to drug candidates if preliminary clinical trials indicate that the therapy may offer substantial treatment advantages over existing options for patients with serious or life-threatening diseases. The FDA has other mechanisms for expediting the review and approval process for promising drugs, including fast track designation, accelerated approval, and priority review.
Due to the many regulations in the industry, the design of medical devices presents significant challenges from both engineering and legal perspectives.
