Introduction and Overview: the FDA and important terms; Exam 1

what are the different drug development designations?

fast track / breakthrough therapy / priority review

what is a prescription drug?

prescribed by a doctor / bought at a pharmacy / prescribed for and intended to be used by one person / most strictly regulated medications can be pills, tablets, injections, patches, inhalants, etc

what are federal regulations?

required or authorized by state / some address a specific problem or known health hazard( FDA egg safety regulation). others are administrative or procedural(citizen petition regulations) / regulation: general statement issued by an agency, board or commission that has the force and effect of law. if broken, can result in punishment / congress grants agencies authority to issue regulations and sometimes requires them to issue a regulation. many laws passed by congress give federal agencies flexibility in deciding how to best implement those laws / specify details and requirements to implement and enforce legislation enacted by congress

what is the Center for Biological Evaluation and Research (CBER)?

similar to the CDER but applies to biologics like proteins, vaccines, enzymes and others

what is the priority review drug development designation?

FDA takes action in 6 months vs 10 months under standard review / direct attention and resources to evaluate drugs that would improve treatment, diagnosis pr prevention of a serious condition

how does the FDA regulate the pharmaceutical industry?

US congress enacts Acts (PDUFA, FDAMA) / FDA then interprets the Acts / the industry participates and uses these documents to better understand how to operate in the healthcare system

what is breakthrough therapy?

a drug development designation / expedites development and review of drugs intended to treat a serious condition / preliminary clinical evidence indicates drug may demonstrate substantial improvement over available therapy / eligible for fast track process / drug company must request

Food and Drug Administration(FDA)

a government body within the US government that is responsible for protecting the healthy by ensuring teh safety, efficacy, and security of human and veterinary drugs, biological products, medical devices and the nation's food supply, cosmetics and products that emit radiation / regulates biologics. extends to 50 states and US territories and possessions

what is a drug(FDA)?

a substance that's.... / recognized by an official pharmacopeia or formulary / intended for use in the diagnosis, cure, mitigation, treatment, or prevent of disease / intended to affect the structure or any function of the body / intended for use as a component of a medicine

what is FDA accelerated approval?

allows approval of a drug that demonstrates an effect on a "surrogate endpoint" that's likely to predict clinical benefit or clinical endpoint that occurs earlier but not as robust as standard endpoint / useful when drug is meant to treat a disease with a long course and extended period of time needed to measure its effect / drug maker needs to do post-marketing clinical trials to verify and describe drug's benefits / FDA can withdraw approval if future trials fail

What is the FDA approval process?

analysis of target condition and available treatment--> ex: a drug to treat a life threatening disease with no other existing therapy may be considered to have benefits that outweigh the risks even if risks would be unacceptable for non life-threatening condition / assessment of benefits and risks from clinical data--> drug maker submits 2 well-designed clinical trials. in rare cases(rare disease), one clinical trial may be enough even if multiple trials not feasible. benefit should outweigh risk / strategies for managing risks--> may need to use Risk Management and Mitigation Strategy (REMs). risk management strategies= FDA approved drug label that explain benefit and risk / drug is approved for one or more indication

What is the FDA responsible for?

assuring that food (except meat from livestock, poultry, and some egg products- US Department of Health) are safe, wholesome, sanitary and properly labeled / human and vet. drugs, vaccines and other biological products and medical devices (human use) are safe and efficient / protect public from electronic product radiation / assuring cosmetic and dietary supplements are safe and properly labeled / regulating tobacco products / advancing public health by helping to speed product innovation

What is the Code of Federal Regulations (CFR)?

codification of general and permanent rules published in federal Register by the Executive departments and agencies of the federal government / title 21 reserved for rules of the FDA / revised early around April

what is an active ingredient?

component of the medication that provides actual pharmacological activity (causes physiological change) / could also be component that has direct effect on diagnosis, cure, mitigation, treatment or prevention of the disease or affects the structure or affect the structure or function of the body / either chemically synthesized or isolated from natural product

what is the fast track drug development designation?

drug company must request to facilitate development and advance the review of drugs that treat serious conditions and fulfill unmet medical needs based on promising animal or human data / can get important drugs to patients earlier

what are inactive ingredients?

no therapeutic effect / may have impact on how drug gets in and around the body / helps stabilize the drug and may include flavorings, binding agents, preservations or others

what are guidances?

not legally binding by t strong suggestions from the FDA / pharmaceutical companies can still be fined by the FDA if they don't follow them

what's the structure of the FDA?

office of the commissioner and four directorates overseeing the core function of the agency (medical products, tobacco, foods, global regulatory operations, policy and operations) / divided into divisions and centers--> CDER and CBER

what is a drug indication?

particular use for the drug (ex: treating diabetes) / group (ex: adults) can be specified / can find by reading the drug label

what is the Center for Drug Evaluation and Search (CDER)?

performs an essential public health task by making sure that safe and efficient drugs are available to improve the health of people in the US / drug companies have to test before selling / if drug's health benefits overweigh known risks, drug is approved for sale (lab and animal tests first and then human and then test to determine if drug is safe when treating a disease and if has a real heal benefit) / doesn't test drug itself but does limited research in areas of drug quality, safety, and effectiveness standards

what are formulations?

physical form in which drug is produced and dispensed (ex: tablet, capsule, etc) / has large impact on how the drug affects the body / helps determine how fast a drug works, where it goes and how long it lasts / orally as tablet= works slowly and effects the whole body / via injection= works faster than tablet and only has a local effect depending on injection site / some formulated to work for a few hours vs others work for a day or longer / changes in formulation of similar active ingredients can have different effects and last different times

what are biologics?

typically injections and include proteins or material derived from living systems / made from a variety of natural resources- human, animal, microorganism- and may also be produced by biotechnology methods / big difference between them and small molecule drugs are size of therapeutic agent / natural products doesn't necessarily= biologics (ex: aspirin isn't)