Pharmacy 2500 MidTerm

1951 Durham-Humphrey Amendment

(prescription) - defining the kinds of drugs that cannot be safely used w/o medical supervision and restriction to sale

Developing New Drugs basics

-CDER 'main consumer watchdog' evaulates new drugs before sale, providing info to use drugs wisely and correctly, benefits > risks, brand name vs generic -drug companies test and send data to FDA who evaluates it -tested on lab animals before in humans

Approval of Drugs basics

-drug's effects have been reviewed by CDER and determined health benefits > risks -analysis of the target condition & available treatments -assessment of benefits and risks from clinical data want 2 well-designed clinical trials -strategies for managing risks - approved drug label, clearly describing effects/detection, some high risk drugs need to implement a Risk Management and Mitigation Strategy (REMS)

prescription drugs are:

-prescribed by a doctor, bought at a pharmacy, prescribed for 1 person only, the most strictly regulated medications (include pills, tablets, IV, patches, biologics etc) -pharmaceutical products regulated by FDA can include medical devices and tests (pacemakers, HIV diagnostic test)

FDA is then divided into Divisions & Centers (2)

1. CDER 2. CBER

FDA (Food and Drug Administration) Responsibilities (5)

1. protect the public health by assuring foods (except agriculture products) are safe, wholesome, sanitary and properly labeled; ensures human/vet drugs/vaccines and biological products/devices are safe/effective 2. Protect the public from electronic product radiation 3. Assure cosmetics and dietary supplements are safe & properly labeled 4. regulate tobacco products 5. advancing public health by speeding product innovations

FDAMA regulations (4)

1. reauthorization of PDUFA for 5 more years - higher charges, shortening drug review time 2. improve patient access to already approved experimental drugs - allow chronic patients access 3. allow pharma companies to disseminate economic outcomes of their products to companies who are involved in insurance coverage and drug pricing 4. preserve presumption that 2 adequate and well-controlled studies are needed to prove product's safety

when was the FDA established?

1930

The Federal Food, Drug and Cosmetic Act 1938

1937: Sulfanilamide An antibiotic accidentally contained diethylene glycol, killing 107 people, establishing a need to ensure drug safety before putting meds on the market leading to the approval of The Federal Food, Drug and Cosmetic Act 1938

Drug (FDA Definition)

A substance 1. recognized by an official pharmacopoeia or formulary 2. Intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease 3. intended to affect the structure or any function of the body 4. intended for use as a component of a medicine -this includes biologics

1. An inactive ingredient in a drug does all of the following EXCEPT... a. Cause a physiologic change in the body b. Stabilize the drug c. Impact how the drug travels in the body d. Give the drug flavor

A.

1. How often must a biopharmaceutical company submit a submission to the FDA for updating a flu vaccine? a) Once every year b) Once every other year c) Once every two years d) Once every five years

A.

1. Which of following is not a way that the FDA defines a drug? A. intended for use in hospitals, pharmacies, at home, etc. B. recognized by an official pharmacopoeia or formulary C. intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease D. intended to affect the structure or any function of the body E. intended for use as a component of a medicine

A.

2. What was the majority type of treatment that was approved using a breakthrough therapy application? a) Cancer b) Inherited disorders c) Cardiovascular conditions d) Infectious diseases

A.

3) Which of the following is NOT one of the goals of GCP? a) To ensure all data during trials is sent to the FDA for approval. b) To protect the rights, safety and welfare of humans participating in research. c) To assure the quality, reliability and integrity of data collected. d) To provide standards and guidelines for the conduct of clinical research.

A.

3) Which part of rational drug design directly depends on basic science? a. Target Selection b. in vitro & in vivo Studies c. Lead Discovery d.Medicinal Chemistry

A.

A phase 3 clinical trial has just begun for the new drug, Buckeyecillin. The study design states that this will be a randomized, two-armed study with a control group receiving a placebo and the treatment group receiving Buckeyecillin. Only the investigators will know who is receiving which medication. This study is: A. A single-blind interventional study B. A double-blind observational study C. A double-blind interventional study D. A single-blind observational study

A.

In which of the following situations would it be most likely that a drug with serious side effects be approved? A. A new drug that cures Ebola, for which no cures exist currently B. A new treatment for use during an asthma attack C. A new drug that lowers cholesterol D. A new treatment for the common cold that reduces a cold's duration by 3 days

A.

The FDA writes guidances and regulations for the drug industry, and takes into consideration pharmaceutical company input. Guidances are__________ and regulations are __________; companies who do not follow guidances or regulations are subject to __________. A. not legally binding ; legally binding ; fines B. not legally binding ; not legally binding ; fines C. legally binding; legally binding; fines D. legally binding ; legally binding ; jail time

A.

The purpose of this expedited process is to allow drugs for serious conditions that fill an unmet need to be approved based on a surrogate endpoint. A. Accelerated Approval B. Breakthrough Therapy Designation C. Fast Track Designation D. Priority Review

A.

This is sent to the FDA and includes information on the safety, efficacy and proposed labeling of a new drug, such as an antidepressant. A. New Drug Application (NDA) B. Branded Ligand Application (BLA) C. Biologic License Application (BLA) D. New Doctor Application (NDA

A.

What is an umbrella study? A. A trial that enrolls patients with the same cancer, but different genetic mutations to look for potential treatments B. A trial in which a group of subjects are followed for long periods of time to watch for a specific outcome, such as development of a disease C. A trial that enrolls patients who do not currently have a disease, but are at risk of developing the disease to study early intervention D. A trial in which investigators observe human subjects to assess different health outcomes

A.

What would NOT be a valid reason to exclude someone from a clinical trial? A. The person is 85 years old and, in the investigators' personal opinions, older adults are not reliable patients B. The person is unable to provide informed consent C. The person has multiple diseases and the treatment may interfere with their other medications D. The patient has type II diabetes, high blood pressure and high cholesterol and the results of the study will be too difficult to interpret based on their health

A.

When can the fast track designation be requested by the drug company? A. At any point during the drug development process B. Only after the drug has entered clinical trials C. Only during the pre-clinical trial phase D. At any point after the drug application has been submitted

A.

Which drug law improved patient access to already approved and experimental drugs? A. FDAMA B. PDUFA C. the Kefauver-Harris Drug Amendments D. 21st century cures act

A.

Which of the following is NOT an expedited process designated by the FDA? A. Breakthrough Discovery B. Priority Review C. Fast Track D. Accelerated Approval

A.

Which of the following statements about a drug's formulation is FALSE? A. A drug's formulation has NO impact on which organs the drug can reach in the body. B. A drug's formulation is also known as the drug's dosage form. C. The formulation determines how Long a drug will last. D. A drug's formulation determines how fast the drug will work.

A.

Which of the following would be considered a surrogate endpoint? A. progression-free survival B. overall survival C. time to remission D. overall cure rate

A.

6. How does the FDA continue to monitor drugs once they have been marketed? (select all that apply) a. A computerized database system for all approved drug and biologic products b. Health professionals and patients voluntarily report any reactions or problems with medications c. A FDA representative is stationed at each US hospital to watch out for patients with severe drug reactions d. Patients must contact the FDA and tell them how they are doing on their new medication

A. , B.

Which of the following are incentives of the orphan drug designation? (Note: This answer has multiple answers; please check all that are correct) A. 7 years market exclusivity instead of 5 years B. Tax increases to the drug company C. The possibility of the drug company receiving new grants D. 8 years market exclusivity instead of 5 years

A., C.

What are the four steps in rational drug design? (Check all that apply) A. Medicinal Chemistry B. Pharmacology C. Lead Discovery D. Target Eradication E. Tentative Discovery F. Target Selection G. in vitro & in vivo Studies H. human trials

A., C., F., G.

1.) Which of the following is not a directory that oversees the core functions of the FDA? - A.) Medical Products & Tobacco B.) Medical Research & Cosmetics C.) Food's & Veterinary Medicine D.) Global Regulatory Operations

B.

3. On average, it takes $1.3 billion to bring a new molecule to the market. Where does the majority of this money go? A. Post-Approval Studies B. Clinical Trials 1,2,3 C. Synthesis and Extraction D. Formulation and Stability

B.

4) Explanation of research is the first part and participation being voluntary is the last part in the _________ a) Belmont Report b) Basic Elements of Informed Consent c) Declaration of Helsinki d) Nuremberg Code

B.

4.) Which of the following is not an ethical principal defined in The Belmont Report? A.) Justice B.) Risk C.) Respect for persons D.) Beneficence

B.

The Alzheimer's Association announces that it is paying for clinical trials to begin on a new drug, Remembral, that has shown promise in slowing the progression of Alzheimer's in animal models. The clinical trials will be performed at the University of Montana under Dr. Patty Potter. They are currently looking for volunteers to participate in the trial. Who is the principal investigator of this trial? A. The volunteers B. Dr. Patty Potter C. The Alzheimer's Association D. University of Montana

B.

Which of the following about generic drug names is TRUE? A. The country of origin's government assigns generic drug names B. drug makers usually propose names according to their compound's chemical make-up C. must meet standards set by the NIH

B.

Which of the following is NOT an incentive of the orphan drug designation? A. Tax credits to the drug company B. 9 years market exclusivity instead of 5 years C. The possibility of the drug company receiving new grants

B.

Which of the following is NOT included in a New Drug Application (NDA)? A. Data from human trials B. Information on the profit margin of the drug C. Data from animal trials D. Proposed label for the drug

B.

Which of the following statements is FALSE regarding the experimentation, or testing, of unapproved drug products? A. Sometimes the unapproved drug, in rare cases, can be given to severely ill patients when no other treatment exists. B. It can take up to 5 years to get a new molecule approved by the FDA C. About a quarter of pharmaceutical spending is spent on clinical studies D. Clinical studies utilize animal testing before beginning clinical studies on humans.

B.

You learned about active ingredients and inactive ingredients in the first lecture. Which of the following is FALSE? A. An active ingredient can be formulated and delivered in several ways such as orally or through the skin B. An active ingredient is synthesized in a lab or isolated from nature to be delivered with inactive ingredients only in rare circumstances C. An active ingredient is any component that provides pharmacological activity and may have a direct effect on the structure or function of the body D. Inactive ingredients are part of a drug product's formulation, which can influence the effects of the drug product on the body

B.

2. Which of the following is TRUE about experimental drugs? (select all that apply) a. Have been approved by the FDA b. Can be used if it has the potential to help a dying patient c. Can be prescribed by a healthcare provider d. In pre-clinical and clinical studies

B. , D.

10. What is the process called when an expedited authorization and use of an unapproved product or the off-label use of an already approved product in a declared emergency involving a chemical, biological, radiological, or nuclear agent? A. New Drug Authorization B. Crisis Drug Authorization C. Emergency Use Authorization D. Pandemic Drug Authorization

C.

2) If a pre-approved drug is improved and sold, how long is the window of market exclusivity? a. 5 years b. 4 years c. 3 years d. The drug doesn't get market exclusivity since it took ideas from a pre-existing drug.

C.

5) Which of the following is an example of early prevention clinical research? a. Research on how to slow down progressive cancers b. Research on the side effects of lead poisoning c. Research on the cause of type 2 diabetes d. Research on chemical change in brain cells after brain death

C.

A biologic can be a range of product types. The following is FALSE regarding biologic? A. One example of a biologic active ingredient is a vaccine B. Biologics differ from small molecule drugs such as aspirin in both size and complexity C. A biologic is not a type of drug according to the FDA definition of a drug D. Biologics include proteins, which may be derived from living systems

C.

A group of women with breast cancer are studied and found that a majority of the women had been exposed to second-hand smoke during their lifetime, which may have contributed to their cancer development. This is known as what type of study? A. Prospective study B. Interventional study C. Retrospective study D. Double-blinded study

C.

Nira Shaw and her team have developed a new drug to treat multiple sclerosis. Studies on mice and rats have given promising results and Dr. Shaw would like to begin testing the drug on humans. What must she do first? A. The drug must show efficacy in humans B. The drug's active ingredient must be approved by the FDA as a new chemical entity (NCE) C. Submit the drug to the FDA to get it approved as an Investigational New Drug (IND) D. She must submit a New Drug Application (NDA)

C.

The FDA writes guidances and regulations for the drug industry, and takes into consideration pharmaceutical company input. If a company is found in violation of an FDA guidance, what could be their punishment? A. Guidances are legally binding and the company could be shut down B. Guidances are legally binding and those found in violation may go to jail C. While guidances are not legally binding, the company could be fined D. The company will only be warned because guidances are not legally binding

C.

What are the 4 parameters that predict the pharmacological profile of a drug? A. Absorption, Dispersal, Management and Elimination B.Adherence, Dispersal, Medication and Elimination C. Absorption, Distribution, Metabolism and Excretion D. Application, Distribution, Management and Expulsion

C.

What is "an expedited authorization and use of an unapproved product or the off-label use of an already approved product in a declared emergency"? A. new drug authorization B. prescription drug authorization C. emergency use authorization D. biologic use authorization

C.

Which drug law establishes new expedited product development programs, such as the Breakthrough Devices program? A. PDUFA B. FDAMA C. 21st Century Cures Act D. the Kefauver-Harris Drug Amendments

C.

Which of the following examples may be awarded 3 years of market exclusivity? A. The generic drug, Headache-Be-Gone, has just been introduced to the market. B. Brutus Pharmaceuticals has submitted a new drug to the FDA, and the FDA has just awarded the drug NCE status. C. Braxtocillin was originally approved to treat infections, but Buckeye Pharmaceuticals has just re-submitted the drug to be approved for a second indication, which is treatment of high blood pressure. D. Summers Medical Products, has just submitted an IND to the FDA to perform clinical trials on their experimental drug, medosporin.

C.

Which of the following would be responsible for poor treatment of subjects in a clinical trial? a. the sponsor b. the institutional review board c. the principal investigator d. the biomedical researcher

C.

Why is it important to have good manufacturing practice? A. It provides guidelines for the proper care of a patient by their primary care physician. B. It creates legislation that provides data privacy and security provisions for safeguarding medical information. C. It assures the identity, strength, quality, and purity of drug products D. It's a standard for the design, performance, monitoring, recording, analysis and reporting of clinical trials or studies meant to protect the rights of human subjects.

C.

Q: The FDA oversees which of the following centers?

CDER and CBER

1) Which of the following choices describes a phase 1 clinical trial? a. Includes up to 5000 patients b. Testing efficacy of the drug c. Volunteers are patients that have pre-existing health issues d. Establishes safety of the drug

D.

2) The World Medical Association created the __________. a) National Research Act b) Belmont Report c) Nuremberg Code d) Declaration of Helsinki

D.

2.) Which of the following is not a source of compounds that are tested as potential drugs - A.) Bioengineering B.) Naturally Sourced C.) Synthetic Production D.) Genetically Derived

D.

5. Which historic event led to the creation of the Nuremberg Code? a. Tuskegee syphilis experiments b. Nuclear testing at Bikini Atoll c. The discovery of immortal HeLa cells d. WWII and Nazi experiments on humans

D.

How long does the FDA have to review an NDA to determine if it is sufficiently complete to conduct a full review? A. 90 Days B. 55 Days C. 74 Days D. 60 Days

D.

Prescription drugs need to first undergo experimentation in preclinical and clinical studies. Before a drug can enter clinical studies: A. The pharmaceutical company must submit a New Drug Application (NDA) B. The drug's active ingredient must be approved by the FDA as a new chemical entity (NCE) C. The drug must show efficacy in humans D. The drug must first be approved as an Investigational New Drug (IND)

D.

The FDA must evaluate the NDA and weigh the effectiveness of the drug against the potential safety concerns. This is known as: A. The safety-effectiveness ratio B. The safety-benefit ratio C. The side effect-dose ratio D. The risk-benefit ratio

D.

The FDA oversees which of the following centers? A. Center for Clinical Evaluation and Research B. Center for Drug Evaluation and Research C. Center for Biologic Evaluation and Research D. Center for Drug Evaluation and Research and the Center for Biologic Evaluation and Research

D.

What is a chemical library? A. An approach where biologically active compounds are specifically designed or chosen to work with a particular drug target. B. A type of procedure that occurs in a whole, living animal C. A method used by chemists to model molecule-target binding using a computer simulation. D. A collection of information on drug molecules that include their structure, purity, quantity and physiochemical characteristics

D.

Which of the following is NOT part of Good Manufacturing Practice (GMP)? A. Obtaining appropriate quality raw materials B. Assuring the purity of the drug products C. Investigating product quality deviations D. Assuring the integrity of the data collected

D.

Which of the following is considered the most important document in the history of the ethics of medical research because it serves as a blueprint for today's principles and ensures the rights of subjects of medical research? A. The Belmont Report B. The National Research Act C. Declaration of Helsinki D. The Nuremburg Code

D.

Which of the following was created as a DIRECT response to research atrocities in the U.S. such as the Tuskegee syphilis studies? A. The Nuremberg Code B. The Belmont Report C. The Declaration of Helsinki D. The National Research Act

D.

4) CDER does limited research in which areas of drugs? A) Quality B) Safety C) Efficacy D) Toxicity E) Both A,B

E.

8. Which of the following is not a type of clinical research? A. Treatment B. Prevention C. Early Detection/Screening D. Diagnostic E. All of the above are different types of clinical research

E.

Which of the following is considered a basic element of informed consent? Select all that apply. A. A description of any reasonably foreseeable risks or discomforts to the subject. B. A description of any benefits to the subject or to others which may reasonably be expected from the research C. An explanation of whom to contact for answers to questions about the research and research subjects' rights. D. A statement that participation is voluntary and that refusal to participate will involve no penalty. E. All of the above

E.

US Pharmacopeia 1820

Established the first compendium of standard drugs for the United States, by physicians in D.C

priority review

FDA aims to take action on application review in 6 months instead of 10, directs attention and resources to evaluate drugs that would significantly improve treatment, diagnosis and prevention of serious conditions

drug indication

FDA approves a drug for 1 or more indications. A drug indication is a particular use for the drug, such as treating diabetes. A group of people can also be specified for use found on drug label

FDA Organization

FDA is an agency under the U.S department of health and Human Services, consisting of the Office of the Commissioner and 4 directorates overseeing the core functions of the agency

4 Directorates of FDA

Medical Products & Tobacco, Foods & Veterinary medicine, Global regulatory operations & polycy and operations

prescription drugs are NOT:

OTC meds and supplements

Bureau of Chemistry 1862

Pres. Lincoln appointed chemist Charles Wetherill to serve in the new Department of Agriculture, beginning of the bureau of chemistry - predecessor to FDA

Enforcement of Federal Regulations FDA

Some (like FDA's egg safety regs.) address a specific problem, while others (citizen petition regulations) are administrative or procedural. A regulation is a general statement issued by an agency, board, commission etc that has the force and effect of law. Congress often grants agencies the authority to issue regulations, sometimes requires agencies to issue a regulation or grants discretion to do so. Many laws passed by Congress give Federal agencies some flexibility in how to implement them. Federal regulations specify details and requirements necessary to implement and enforce legislation enacted by Congress

Code of Federal Regulations - CFR

The codified regulations of the Federal government based on the final agency regulations published in the Federal Register. Title 21 reserved for Food and Drug administration, revised yearly in April

1962 Kefauver-Harris Amendment

Which amendment stated that all new drugs marketed had to be SAFE and EFFECTIVE.

Accelerate Approval

applied to promising therapies treating serious/life-threatening conditions; approval of drug demonstrates an effect on a 'surrogate endpoint'. long-treatment drugs are required to conduct post-marketing clinical trials to verify effects and the FDA can withdraw approval

active ingredients

component of the medicine that provides the actual pharmacological activity - also be the component that has a direct effect on diagnosis, cure, prevention etc or the component that affects the structure or function of the body synthetic or natural

breakthrough therapy

designation expedites development and review of drugs intended to treat a serious condition and allow for substantial improvement, also eligible for fast track (can mix designations)

small molecule vs biologic

difference in size of therapeutic agent, natural products are not always biolgics, like asprin. biolgics are much larger

Accelerated Approval Pathway (1992)

due to protests surrounding HIV/AIDS

drug development designations (4)

encourages development of certain drugs, addressing specific needs, drugs are available to patients quickly include: accelerate approval, fast track, breakthrough therapy, priority review

inactive ingredients

included in a drug but has no therapeutic affect; may have an impact on how the drug gets in and around the body and are included to help stabilize the drug and may include flavorings, binding agents, preservatives etc

Q: Which of the following is FALSE about the FDA?

it oversees the Department of Health and Human Services (DHHS)

biologics

made from a variety of natural resources (human, antibodies, animals and microorganisms) and may also be produced by biotech methods. ex) allergenic extracts, blood, gene therapy, devices/test kits, human tissue, vaccines etc

Enforcement of Federal Guidances FDA

not legally binding, but rather strong suggestions from the FDA, pharma companies can still be fined by the FDA if they don't adhere

CDER (Center for Drug Evaluation and Research)

performs essential public health task by ensuring safe & effective drugs are available to improve health of people in the US

fast track

process designed to facilitate the development/advance the review of drugs that treat serious conditions and fill an unmet medical need based on promising data; drug companies must request a fast track process

1997 Food and Drug Administration Modernization Act (FDAMA)

reauthorized the Prescription Drug user fee act of 1992 and mandated the most wide-ranging reforms in agency practices since 1930, focusing on reforming regulation of food, meds and cosmetics

1992 Prescription Drug User Fee Act (PDUFA)

requires drug and biologics manufacturers to pay fees for product applications and supplements, along with other services, required the FDA to use these funds to hire more reviewers to assess applications

Q: The FDAMA of 1997 focused on reforming the regulation of food, mes, and cosmetics. Which of the following was part of the revision?

requires two well controlled studies to prove a drug's safety and efficacy

CBER (Center for Biologics Evaluation and Research)

similar to CDER, applies to biologics like proteins, vaccines, enzymes etc.

changes in formulation of _______ active ingredients can have __________ effects and last different amounts of time

similar; different ex) birth control: pill vs IUD

formulations

the physical form where a drug is produced and dispensed, like a tablet, capsule, IV etc. the formulation of the drug has a large impact on how that drug affects the body, it also helps determine how fast the drug will work, where the drug goes and how long it will last ex) if a drug is taken orally it'll work slower than an IV

Q: The FDA writes guidances/regulations for the drug industry and takes into consideration pharmaceutical company input. If a company is found in violation of FDA guidance, what could be their punishment?

while guidances are NOT legally binding, the company could be fined