question

Role of Regulatory Affairs

answer

Liason between FDA and company; Liason between company and FDA; Liason between functional areas within company

question

Role of the Regulatory Professional

answer

Long-term strategic planning; Risk analysis; Tactical and operational support; Provide updates on regulations and guidance documents; Monitor current FDA environment; Ensure compliance with regulations

question

Regulatory Divisions

answer

Clinical; CMC; Post-marketing; Regulatory Operations; Regulatory Compliance

question

Regulatory Professionals Skills

answer

Ability to understand the technology; knowledge of regulations, guidelines and information sources; Communication and negotiation skills; Flexibility and understanding

question

Pure Food & Drugs Act of 1906 to ensure:

answer

Purity; Quality; Label claims

question

Pure Food & Drugs Act of 1906 brought forth:

answer

Dept of Agriculture, Bureau of Chemisty; Interstate commerce; adulteration and misbranding; criminal penalties - seizure of product; standards - USP, NF

question

Food Drug and Cosmetic Act of 1938

answer

required all new drugs to be safe(BUT NOT EFFECTIVE; BIG DISTINCTION); required submission of safety data before marketing; adulteration and misbranding; Food, Drug, Cosmetic, Device Areas; prohibited acts; enforcement: criminal prosecution, injuctions, seizure, factory inspection

question

Kefauver-Harris Amendments of 1962

answer

required all new drugs to be SAFE & effective (DRUG EFFICACY AMMENDMENT); FDA required to review all new drugs approved between 1938 and 1962 as safe to determine if effective; also known as "Drug Efficacy Amendments"; manufacturers must comply with GMP; NDA must be approved before marketing; prescription drug advertising under FDA supervision

question

Evolution of Drug Regulation brought forth:

answer

Safety, Efficacy, and Expanded Access

question

Evolution of Drug Regulation regulations:

answer

Safety, Efficacy, and Expanded Acess - Orphan Drug Act 1983; Generic Drug Enforcement Act 1992; Prescription Drug User Fee Act (PDUFA) 1992; (Speed & Structure) FDA Modernization Act (FDAMA) 1997

question

PDUFA (1992)

answer

Prescription Drug User Fee Act; Fees for marketing application and supplements; intended to fund more reviewers; applies to drugs and biologics

question

motivation of PDUFA

answer

to accelerate FDA review

question

FDAMA (1997)

answer

Food and Drug Modernization Act

question

what FDAMA did

answer

codified imporved review procedures and communications; Clinical trials registry established; FDA has authority to monitor post-market studies; Extended PDUFA for 5 more years

question

Critical Path Initiative

answer

plan to modernize the drug development process; modernization of scientific tools in drug development (computer modeling, in vitro tests, innovative trial design, qualified biomarkers)

question

Regulatory Pathways: Notice and Comment Process

answer

Advance notice of proposed rulemaking or notice of intent to publish in Federal Register; publish final rule and codify in the Code of Federal Regulations (CFR)

question

proposed rule:

answer

comment period 60 days

question

Code of Federal Regulations (CFR)

answer

the codification of the general and permanent rules and regulations published in the Federal Registr by the executive departments and agencies of the Federal Govt

question

regulations are given in part numbers

answer

"Title #" CFR "Part and subpart number" (i.e. 21 CFR 312 addresses the IND regulations)

question

Code of Federal Regulations (CFR) Title 21 Food & Drugs; Drugs section :

answer

200-299

question

Code of Federal Regulations (CFR) Title 21 Food & Drugs: Drugs for Human Use Section:

answer

300-460

question

Code of Federal Regulations (CFR) Title 21 Food & Drugs: Biologics Section:

answer

600-680

question

IND stands for:

answer

Investigational New Drug Application

question

NDA stands for:

answer

New Drug Application

question

Unlike the CFR, Guidance Documents are not:

answer

law

question

Guidance Documents represent:

answer

current FDA thinking and should (must) be followed; FDA references these documents

question

NOAEL stands for

answer

No observed adverse effect level

question

NOAEL is used when

answer

one cannot conduct nonclinical trials

question

Dept of Agriculture was created

answer

1862

question

the Dept of Agriculture brought forth:

answer

the Division of Chemistry & the Food and Drug Administration

question

the Food & Drug Administration was created

answer

1930

question

the Dept of Health & Education was created

answer

1953

question

the Dept of Health and Human Services was created

answer

1979

question

Congress created FDA by statute in

answer

1988

question

(Mission Statement) The FDA is responsible for

answer

protecting the publich health by assuring the safety, efficacy and security of human and veterinary drugs, biological products, medical devices, our nations food supply, cosmetics, and products that emit radiation.

question

(Mission Statement Cont'd) The FDA is also responsible for

answer

advancing the public health by helping speed innovations that make medicines and foods more effective, safer, and more affordable; and helping the public get the accurate, science-based info they need to use medicines and foods to improve their health.

question

The FDA Commissioner ensures:

answer

that the Agency carries out its mission of protecting and advancing the public health

question

The Commission was appointed by _______ the Pesident and confirmed by _____teh Senate in ______

answer

the President; the Senate; 1988

question

FDA Structure:

answer

Commisioner; CDER; CBER; CDRH; CFSAN; CVM

question

CDER

answer

drugs

question

CBER

answer

biologics

question

CDRH

answer

devices

question

CFSAN

answer

food & safety

question

CVM

answer

veterinary

question

New Drug Development Timeline: (1) Pre-Clinical Testing, R

answer

range: 1-3yrs (Avg 18mo); Initial Synthesis; animal testing **short term**

question

New Drug Development Timeline: (2) Clinical R

answer

range: 2-18yrs (Avg 5yrs); Phase 1,2&3; **long-term**

question

Between Pre-clinical testing R and Clinical R Phases on New Drug Development Timeline there is a:

answer

30-Day Safety Review

question

New Drug Development Timeline: (3) NDA Review time

answer

FDA time to review NDA; Range: 2mo - 7yrs (Avg 24 mo) from submission of NDA to approval of NDA

question

New Drug Development Timeline: (4) Post-Market Surveillance

answer

Adverse reaction reporting; surveys/sampling/testing; inspections

question

NME

answer

New Drug Application

question

FD Act

answer

"articles intended for use in the diagnosis, cure, mitigation, treatment, or prevention of disease and man….and articles (other than food) intended to affect the structure or function of the body of man…."

question

NCE

answer

New Chemical Entities

question

definition of a Biologic

answer

Biologics Act of 1902 - any virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, or analogous product, or arsphenamine or its derivatives (or any other trivalent organic arsenic compound), applicable to the prevention, treatment, or cure of diseases or injuries of man.

question

Section 201 (p)(1) of the FD Act - New Drugs

answer

not meant for animals; not generally recognized as safe and effective; must be the subject of an FDA approved new drug application prior to marketing

question

New Drugs as defined in 21 CFR 310.3

answer

there is a new use of any substance (e.g. active ingredient, excipient, carrier, coating, etc. that composes the drug); there is a new drug use of a combination of approved drugs (drug A + drug B = drug C); the proportion of ingredients in combination is changed; there is a new intended use; the dosage, method or duration of administration or application is changed.

question

the following are subject to FDA approval:

answer

a drug containinng a new chemical or molecular entitiy; a drug containing an existing active ingredient that has never been approved in the US; previously approved, but proposed for a new indication; previously approved but in a different dosage form or route of administration.

question

New Drugs: Exceptions are

answer

"Grandfather Drugs"; Homeopathic Drugs; Dietary Supplements

question

CDRH

answer

Center for Drug Evaluation and Research; responsibility is to ensure drugs are safe and effective; responsibility for both prescription and over-the-counter drugs

question

sponsor responsibility w/New Drugs

answer

it is the responsibility of the company seeking to market a drug to test it and submit evidence that it is safe and effective. Physicians, statisticians, chemists, pharmacologists, and other scientists review the sponsor's new drug application contating the data and proposed labeling.

question

Overview of primary steps to approval w/new drugs

answer

(1) scientific test designed to provide data on a product's safety and effectiveness; (2) presentation and submission of these data in regulatory submissions; (3) FDA's review of these submissions.

question

Nonclinical Testing is testing for

answer

Safety

question

the purpose of nonclinical testing is:

answer

(1) to seek some evidence of the drug's biological activity (2) to show that it is reasonably safe for initial administration into humans

question

Nonclinical testing should follow:

answer

Good Laboratory Practices (GLP)

question

General animal welfare provisions contained in federal state animal protection statutes

answer

GLP

question

nonclinical testing means a new drug is labeled:

answer

"Caution-contains a new drug for investigational use only in laboratory research animals, or for tests in vitro. Not for use in humans."

question

Safety in nonclinical testing is usually demonstrated by:

answer

(1) compiling existing non-clinical data from past in vitro lab or animal studies (2) compiling data from previous clinical testing (3) undertaking new pre-clinical studies

question

toxic and pharmacological effects are studied in:

answer

nonclinical testing

question

genotoxicity screening is performed in:

answer

nonclinical testing

question

drug absorption, metabolism, distribution, and excretion (how is the drug cleared from the body?) is tested in:

answer

nonclinical testing

question

FDA expectations of nonclinical testing are:

answer

(1) pharmacological profile (2) acute toxicity of the drug in at least 2 species of animal (3) conduct short-term toxicity studies which mimic the duration of the proposed clinical studies

question

IND can be thought of as:

answer

the product of a successful preclinical development program

question

A sponsor assembles and submits an IND if:

answer

based on preclinical studies, the pharmacology of a compound justifies further development and it is adequately safe for initial small-scale clinical studies

question

A drug that is used in clinical investigations in humans

answer

Investigational New Drug

question

Such use of an investiagtional new drug (a drug that is used in clinical investigations in humans) is covered by the:

answer

Investigational New Drug Application (IND)

question

An Investigational New Drug Application (IND) is also called:

answer

Notice of Claimed Investigational Exemption for a New Drug Form FDA 1571

question

21 CFR 312

answer

IND regulations

question

What an IND is NOT

answer

a marketing application (it only allows a sponsor to test the drug)

question

an IND is a request

answer

for exemption from the federal statue which prohibits transport of unapproved drugs in interstate commerce

question

A sponsor requests this type of mtg depending on what the purpose is

answer

Type A,B or C

question

A formal request for an FDA meeting should be made in writing to the agency (clock starts ticking) and should list:

answer

objectives and discussion points

question

An IND Type A, B, or C meeting w/FDA will be schedule

answer

within 60 days of agency's written receipt of request

question

4 weeks prior to Type A,B, C mtg this must be sent

answer

a pre-meeting package

question

Product name; chemical name and structures; proposed indications; dosage form, route and duration administration; purpose of meeting/list of objectives; agenda/specific questions; Clinical data; preclinical datat & phase 1 protocol; chemistry, manufacturing, and control data

answer

Background package for FDA IND Type A, B, C mtg

question

if a sponsor has no clinical data to submit w/background package for IND Type A, B,C mtg, the sponsor can submit:

answer

a rational for what the chemical will do

question

A ____ is filed by companies whose goal is to obtain marketing approval of a new product

answer

Commercial IND

question

Three types of Non-Commerical IND's are:

answer

(1) Investigator IND (2) Emergency Use IND (3) Treatment IND

question

____ IND is submitted by an investigator who initiates and conducts the investigation

answer

Investigator IND

question

The motivation of an Investigator IND is NOT

answer

commercial

question

A physician must submit a research IND to propose:

answer

studying an unapproved drug or an approved product for a new indication or in a new patient population

question

An Emergency Use IND is also called:

answer

"Compassionate Use" or "Single Patient" INDs

question

An Emergency Use IND allows the FDA to authorize use of an:

answer

experimental drug in an emergency situation that does not allow time for submission of an IND.

question

___ IND is also used for patients who do not meet the criteria of an existing study protocol or if an approved study protocol does not exist

answer

Emergency Use IND

question

A _____ IND is submitted for experimental drugs showing promise in clinical testing for serious or immediately life-threatening conditions while the final clinical work is conducted and the FDA review takes place

answer

Treatment IND

question

IND maintenace includes the following three:

answer

(1) Amendments (protocol and safety) (2) safety reports (3) annual reports

question

21 CFR 312.21 specifies:

answer

IND Phases

question

Phase 1: drug never in humans before; Phase 2: clinical safety data (testing for efficacy) & prep for phase 3 study; Phase 3: "label enabling study" & package insert info w/enough S data

answer

3 Phases of an IND

question

____ may be submitted for one or more phases of an investigation. The clinical investigation of a previously untested drug is generally divided into three phases. Although in general the phases are conducted sequentially, the may overlap.

answer

An IND

question

Risk vs Benefit Phase (or) At what dose can we get the most benefits w/the least risk

answer

Phase 1 of IND process

question

Phase __ typically closely monitored and may be conducted in patients or normal volunteer subjects

answer

Phase 1 of IND process

question

Phase 1 is designed to determine:

answer

the metabolism and pharmacological actions of the drug in humans, the side effects associatd with increasing doses.

question

while Phase 1 studies are intended to gain early evidence of effectiveness….

answer

you almost never get early evidence of effectiveness bc the dose is too small or the patient population is too small to be accurate

question

Phase 1 studies aer designed to gather sufficient info about:

answer

the drug's pharmacokineticsadn pharmacologicl effects to permit the design of well-controlled, scientifically valid, Phase 2 studies.

question

Number of subjects and patients included in a ___ study are generall in the range of __ to __.

answer

Phase 1; 20-80

question

Phase 1 studies also include studies of drug metabolism, structure-activity relationships, and mechanism of action in humans, as well as:

answer

studies in which investigational drugs are used as research tools to explore biological phenomena or disease processes.

question

___ studies are typically well controlled, closely monitored, and conducted in a relatively small number of patients, usually involving no more than several hundred subjects.

answer

Phase 2

question

Phase 2 studies include:

answer

the controlled clinical studies conducted to evaluate the effectiveness of the drug for a particular indication of indications in patients with the disease or condition under study and to determine the common short-term side effects and risks associated with the drug.

question

Phase 3 studies are after:

answer

a sponsor gets efficacy data from Phase 2 study

question

Study to develop package insert

answer

Phase 3

question

the goal of expanded controlled and uncontrolled trials during phase 3 studies are to

answer

try to remove bias

question

____ study are performed after preliminary evidence suggesting effectiveness of the drug has been obtained

answer

Phase 3

question

Phase 3 studies are intended to gather the additional information about the effectiveness and safety that is needed to evaluate:

answer

the overall benefit-risk relationship of the drug and to provide an adequate basis for physician labeling

question

Phase 3 studies include from several

answer

hundred to thousand subjects.

question

Form FDA 1571

answer

cover sheet for IND application

question

the name, address, and telephone number of the sponsor, the date of the application, and the name of the investigational new drug.

answer

form FDA 1571

question

identification of the phase or phases of the clinical investigation to be conducted

answer

form FDA 1571

question

a commitment not to begin clinical investigations until an IND coverign the investigations is in effect

answer

form FDA 1571

question

a commitment that an Institutional Review Board (IRB) that complies with the requirements set forth in part 56 will be responsible for the initial and continuing review and approval of each of the studies in the proposed clinical investigation and that the investigator will report to the IRB proposed changes in teh research activity in accordance with the requirements of part 56

answer

form FDA 1571

question

a commitment to conduct the investigation in accordance with all other applicable regulatory requirements.

answer

form FDA 1571

question

the name and title of the person responsible for monitoring the conduct and progress of the clinical investigations

answer

form FDA 1571

question

the name(s) and title(s) of the person(s) responsible under 312.32 for review and evaluation of info relevant to the safety of the drug.

answer

form FDA 1571

question

if a sponsor has transferred any obligations for the conduct of any clinical study to a contract research organization, a statement containing the name and address of the contract research organization, identification of the clinical study, and a listing of the obligations transferred.

answer

form FDA 1571

question

the signature of the sponsor or the sponsor's authorized rep.

answer

form FDA 1571

question

A table of contents and introductory statement and general investigational plan (what you plan to do statement)

answer

CFR IND requirements

question

A brief summary of previous human experience with the drug. (you can compare experience to that of other companies)

answer

CFR IND requirements

question

if the drug has been withdrawn from investigation or marketing in any country for any reason related to safety or effetiveness, identification of the country(ies) where the drug was withdrawn and the reasons for the withdrawal.

answer

CFR IND requirements

question

tox study ADME

answer

Absorbtion Distribution Metabolism Excretion

question

A brief description of the overall plan for investigating the drug product for the following year. (based on pk data)

answer

CFR IND requirements

question

Investigator's Brochure

answer

CFR IND requirements

question

A brief description of the drug substance and the formulation, including the structural formula, if known.

answer

CFR IND requirements

question

A summary of the pharmacological and toxicological effects of the drug in animals, and, to the extent known, in humans.

answer

CFR IND requirements

question

A summary of the pharmacokinetics and biological disposition of the drug in animals and, if known, in humans.

answer

CFR IND requirements

question

A summary of information relating to safety and effectiveness in humans obtained from prior clinical studies.

answer

CFR IND requirements

question

A description of possible risks and side effects to be anticipated on the basis of prior experience with the drug udner investigation or with related drugs, and of precautions or special monitoring to be done as part of the investigational use of the drug.

answer

CFR IND requirements

question

Protocols

answer

CFR IND requirements

question

Everyone has done "this" so you can do can/will do the same

answer

Class effect

question

Chemistry, manufacturing, and control information (CMC)

answer

CFR IND requirements

question

Labeling

answer

CFR IND requirements

question

Environmental analysis requirements

answer

CFR IND requirements

question

Pharmacology and toxicology information

answer

CFR IND requirements

question

Previous human experience with the investigational drug

answer

CFR IND requirements

question

3 main components of the IND

answer

(1) nonclinical (preclinical animal testing); (2) Chemistry, Manufacturing, and Controls - CMC Data (3) Clinical Data

question

IND components module

answer

(1) Administrative (2) Summary (3) CMC (4) Nonclinical Data (5) Clinical Data

question

GXP

answer

good clinical practices

question

GLP

answer

Good Laboratory Practices (GLP)

question

GMP

answer

good manufacturing practices

question

The first step for ___ ___ ___ in Nonclinical studies is to develop some in vivo and in vitro data

answer

New Molecular Entities (NME) - per nonclinical studies

question

FDA is willing to discuss non-clinical testing requirements with

answer

Sponsors (nonclinical studies)

question

Nonclinical studies must be conducted according to

answer

GLP

question

____ provide basic pharm/tox information

answer

animal studies (nonclinical studies)

question

long-term animal studies are performed even after

answer

human clinical trials are underway (per nonclinical studies)

question

Allow safe introduction of molecule into humans

answer

goal of nonclinical testing

question

determine minimum recommended starting dose (based on the NOAEL - no observed adverse effect level

answer

goal of nonclinical testing

question

understanding of pharmacokinetics (PK) and pharmacodynamics (PD)

answer

goal of nonclinical testing

question

PK is the study of

answer

what the body does to the drug (extent and duration of exposure; ADME)

question

PD is the study of

answer

what the drug does to the body (dose responses; Adverse effects on physiologic systems)

question

Choice of ____ ____ in nonclinical testing is based upon regulatory requirements, relevance of species, and/or existing disease models

answer

animal species

question

organization and personnel

answer

GLP

question

testing facilities

answer

GLP

question

facilities operation

answer

GLP

question

test and control article characterization

answer

GLP

question

study protocol

answer

GLP

question

records/reporting

answer

GLP

question

equipment design

answer

GLP

question

FDA inspections

answer

GLP

question

drug structure, proposed indication, target population, route of administration, duration or use, and special characteristics are ___ ___ ___

answer

Nonclinical testing considerations

question

EQSM

answer

Efficacy Quality Safety Multidisciplinary

question

_____ studies let you determine if you have enough info to allow to dose safely

answer

toxicity

question

Acute, Chronic, Carcinogenicity, Genotoxicity, and reproductive ____ are all types of ___ ____

answer

toxicity; toxicity studies

question

Information pertaining to the composition, manufacturer, stability, and controls used for manufacturing the drug substance and drug product. This information is assessed to ensure that the sponsor can adequately produce and supply consistent batches of the drug

answer

Chemistry, Manufacturing, and Controls (CMC )

question

drug substance studies, drug product studies, placebo & drug and biologics specific information all are types of

answer

CMC Information

question

drug ____ is the active form, not the final form

answer

substance (ie: acetomenaphin)

question

drug _____ is the final dosage form (what you take)

answer

product (ie: tylenol)

question

clinical protocol, informed consent form, and investigator's brochure are all

answer

clinical items

question

____ protocol is a document given to patients that explains everything (risks vs benefits, etc)

answer

clinical

question

Central focus of FDA IND review, rationale for study, design of study, endpoints, and assessments are all part of the

answer

clinical protocol

question

Determining appropriate clinical success points and if conducting appropriate testing can be considered

answer

endpoints

question

____ _____ Form is required per 21 CFR 50 (Protection of Human Subjects)

answer

Informed Consent

question

the Informed Consent Form provides subjects with

answer

a description of study procedures and risk/benefit

question

_____ _____ is a 30 day FDA multidisciplinary review of clinical, nonclinical, and CMC datat

answer

IND submission

question

IND's are NOT

answer

approved; if 30 days lapses w/out hold from FDA you move forward

question

30 day notification of protocol status as part of the IND process is ___ ____ and ____ ___ ___

answer

clincal hold; no clinical hold

question

Most INDs today are submitted in

answer

CTD format

question

Module 1 Administratie; Module 2 Summary; Module 3 Quality (CMC); Module 4 Safety (Nonclinical); Module 5 Efficacy (Clinical)

answer

CTD Structure

question

21 CFR 314

answer

NDA regulations

question

New Drug Application and Biologics License Application are essentially

answer

the same document

question

BLA

answer

Biologics License Application

question

NDA regulations 21 CFR 314 is a __ not a ___

answer

law; guidance

question

these documents are required for FDA review and approval prior to the marketing of a drug or biologic

answer

NDA Regulations 21 CFR 314

question

SAP

answer

statistical analysis program

question

meeting with the reviewing division to discuss the presentation of the info in the dossier

answer

Pre-NDA/BLA Mtg

question

this meeting should be conducted prior to the filing of a license application to give sponsor's an early indication regarding the division's opinion of their data

answer

Pre-NDA/BLA Mtg

question

Majority of NDA license applications are submitted in the

answer

CTD format

question

NDA process overview is comprised of

answer

topline data

question

Form ____h - Application to market a new drug, biologic, or an antibiotic for human use

answer

356h

question

User Fee cover sheet, paient information financial disclosure, overall table of contents (Index), labeling, and technical sections are all ____ _____

answer

NDA Components

question

The _____ ______ includes info from the entire NDA; summarizes drug safety and effectiveness (this defines yoru application); summarizes drug risk/benefit; received by all reviewers; 50-200 pgs

answer

NDA Content

question

____/____ defines your NDA application

answer

drug risk/benefit

question

CMC, Pharmacology/Toxicology, human pharmacokinetics, microbiology, clinical, risk/benefit are all _____ sections of the NDA

answer

Technical

question

API

answer

active pharmaceutical ingredient

question

(1) composition, manufacture, specifications of Drug Substance & drug product (2) manufacturing development (3) methods validation (4) process controls (5) identity, purity, quality, strength - all are components of the

answer

CMC Section

question

Physiochemical characteristics, stability, method of synthesis or isolation, purification, process controls, identity, strength, quality, purity; specifications & analytical methods; impurity testing - all part of CMC Drug ____

answer

Substance

question

Manufacturing components; statement of drug prodcut composition; specifications and analytical methods; in-process controls; master batch record; container closure system; stability - proposed expiration dating; environmental impact assessment are all part of CMC Drug ____

answer

Product

question

Data from animal studies in the IND and any other un-submitted data; studies of the pharmacological action of the drug as it relates to its proposed therapeutic indication; reproductive toxicology are all part of

answer

nonclinical NDA section

question

in pharmacology, ______ is used to describe the fraction of an administered dose of medication that reaches the systemic circulation

answer

bioavailability

question

Pilot ADME studies are; bioavailability/bioequivalence, dosage form; pharmacokinetics studies; in vivo studies; in vitro studies to examine release rate of the drug substance from the dosage form are all part of____ ____

answer

human PK and Bioavailability studies

question

_____ Section is only required for certain categories of drugs (such as anti-infectives)

answer

Microbiology

question

Microbiology Section reports on how the target organism is affected - the ______ _____ of activity

answer

biochemical basis

question

Mechanisms of resistance, spectrum of activity, and methods to evaluate activity are part of the

answer

Microbiology Section

question

List of investigatros, INDs, and prior NDA's; Overview of clinical investigations; Integrated summary showing effectiveness (ISE); details of any CRO involvement; Description/analysis of each controlled study, protocols and statistics; description of uncontrolled studies are all part of the Clinical ___ _____

answer

Data Section

question

Common Technical Document (CTD) structure:

answer

(1) Module 1 Administrative (2) Module 2 Summary (3) Module 3 Quality - CMC (4) Module 4 Safety (nonclinical); (5) Module 5 Efficacy (Clinical)

question

FDA NDA review time goals are established under

answer

PDUFA

question

approval time

answer

time from the first NDA submission to NDA approval

question

priority NDA review:

answer

6 mo. Review performance goal. Product is determined to provide significant therapeutic or public health advance "Fast Track"

question

standard NDA review:

answer

10-12mo review performance goal

question

Once an NDA has been filed, new and/or revised data or info can be submitted as

answer

amendments

question

medical reviewer; pharm/tox reviewer; CMC reviewer; statistics; microbiology; bioresearch monitor (verifies data) are all part of the

answer

NDA review team

question

A sponsor does NOT file an NDA, it _____ and NDA

answer

submits

question

core team has a 45 day meeting to determine if NDA can be filed or labeled

answer

"refused to file" (RTF)

question

RTF letters are usually within ____ days of NDA receipt

answer

60

question

if omissions from NDA are not on the critical path review then may NOT lead to

answer

RTF

question

FDA will usually inform sponsor of "_____ ______" w/in 14 day of the 60-day application filing date

answer

substantive deficiencies

question

Experts on the reivew team evaluate respective sections; consultation with other experts may be requested; usually results in questions for the sponsor - "Discipline Review Letters" (possible deficiencies) and "Information Request Letters" (clarification); pre-approval inspections

answer

NDA review process

question

NDA Decision Process

answer

follows technical reviews: usually coordinated by the medical reviewer; unresolved issues may be brought before an Advisory Committee

question

approved products are often subject to the following post-approval changes:

answer

CMC, Clinical, Labeling

question

Advertising/Labeling is regulated by:

answer

the Division of Drug marketing, Advertising,and Communications

question

Division of Drug Marketing, Advertising, and Communications reviews package labeling, advertising, and attends drug conferences to ensure

answer

compliance

question

advertising/labeling that is not in compliance can be labeled

answer

false/misleading claims

question

"Fair balance"

answer

if you state a positive abou the data, you should also state a negative (adv/labeling)

question

21 CFR 312, Subpart D of IND regulations is

answer

good clinical practices GCP

question

"21" of 21 CFR means

answer

food & drugs

question

Good Clinical Practice: Consolidated Guidance is under

answer

ICH E6

question

Responsibility (Investigator & Sponsor - ultimate responsibility); record keeping; and essential documents are all found in

answer

ICH E6

question

GCP Documentation Includes

answer

Clinical Protocol; Investigator's Brochure (overview for rationale of study); Informed Consent; Declaration of Helsinki

question

Declaration of Helsinki

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**Do no harm** fundamental principle is respect for the individual, their right to self dtermination and the right to make informed decisions regarding participation in research, both initially and during the research. The investigator's duty is solely to the patient or volunteer......teh sugject's welfare takes precendence over interests of science and society

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proper informed consent procedures; protocol compliance (site compliance & sponsor compliance); updating investigators - sponsors req'd to do this for any new safety ino; & appropriate documentation are all

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GCP considerations

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_____ commitees provide FDA with independent advice from outside experts on issues related to human and veterinary drugs, biological products, medical devices, and food.

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Advisory

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Members of an _____ ______ include a Chair, several members, plus a consumer, industry and sometimes a representative

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Advisory Committee

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Although the committees provide advice to the Agency, final decisions are made by the

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FDA

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Anesthetic and Life Support Drugs; Anti-Infective Drugs; Antiviral Arthritis Drugs; Cardiovascular and Renal Drugs; Reproductive Health Drugs; Drug Safety and Risk Management; Endocrinologic and Metabolic Drugs; & Gastrointestinal Drugs are all types of

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Advisory Committee

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Significant public interest, controversy, and additional expertise are all reasons why the FDA may call an

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advisory committee meeting

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sponsor has to provide a briefing document for an

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advisory committee meeting

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advisory committees can have

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open and closed sessions; open sessions often have public comment

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the term "____ _____" can refer to either a durg or biologic intended for use in a rare disease or condition

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orphan drug

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A drug or biologic becomes an orphan drug when:

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it receives orphan-drug designation from the Office of Orphan Products Development at the FDA.

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Orphan-drug designation qualifies the sponsor to receive _____ _____ from the Gov't in exchange for _____ _____ ____ for a rare disease/condition

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certain benefits; developing the drug

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Ophan drugs must go through the ____ _____ _____ process like any other drug or biologic which evaluates for safety and efficacy

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FDA marketing approval

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21 CFR 316.20

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Content and format of a request for orphan drug designation

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A statement that the sponsor requests orphan drug designation for a rare disease or condition, which shall be identified with specificity

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