Term
| Role of Regulatory Affairs |
|
Definition
| Liason between FDA and company; Liason between company and FDA; Liason between functional areas within company |
|
|
Term
| Role of the Regulatory Professional |
|
Definition
| 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 |
|
|
Term
|
Definition
| Clinical; CMC; Post-marketing; Regulatory Operations; Regulatory Compliance |
|
|
Term
| Regulatory Professionals Skills |
|
Definition
| Ability to understand the technology; knowledge of regulations, guidelines and information sources; Communication and negotiation skills; Flexibility and understanding |
|
|
Term
| Pure Food & Drugs Act of 1906 to ensure: |
|
Definition
| Purity; Quality; Label claims |
|
|
Term
| Pure Food & Drugs Act of 1906 brought forth: |
|
Definition
| Dept of Agriculture, Bureau of Chemisty; Interstate commerce; adulteration and misbranding; criminal penalties - seizure of product; standards - USP, NF |
|
|
Term
| Food Drug and Cosmetic Act of 1938 |
|
Definition
| 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 |
|
|
Term
| Kefauver-Harris Amendments of 1962 |
|
Definition
| 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 |
|
|
Term
| Evolution of Drug Regulation brought forth: |
|
Definition
| Safety, Efficacy, and Expanded Access |
|
|
Term
| Evolution of Drug Regulation regulations: |
|
Definition
| 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 |
|
|
Term
|
Definition
| Prescription Drug User Fee Act; Fees for marketing application and supplements; intended to fund more reviewers; applies to drugs and biologics |
|
|
Term
|
Definition
|
|
Term
|
Definition
| Food and Drug Modernization Act |
|
|
Term
|
Definition
| codified imporved review procedures and communications; Clinical trials registry established; FDA has authority to monitor post-market studies; Extended PDUFA for 5 more years |
|
|
Term
|
Definition
| plan to modernize the drug development process; modernization of scientific tools in drug development (computer modeling, in vitro tests, innovative trial design, qualified biomarkers) |
|
|
Term
| Regulatory Pathways: Notice and Comment Process |
|
Definition
| 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) |
|
|
Term
|
Definition
|
|
Term
| Code of Federal Regulations (CFR) |
|
Definition
| 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 |
|
|
Term
| regulations are given in part numbers |
|
Definition
| "Title #" CFR "Part and subpart number" (i.e. 21 CFR 312 addresses the IND regulations) |
|
|
Term
| Code of Federal Regulations (CFR) Title 21 Food & Drugs; Drugs section : |
|
Definition
|
|
Term
| Code of Federal Regulations (CFR) Title 21 Food & Drugs: Drugs for Human Use Section: |
|
Definition
|
|
Term
| Code of Federal Regulations (CFR) Title 21 Food & Drugs: Biologics Section: |
|
Definition
|
|
Term
|
Definition
| Investigational New Drug Application |
|
|
Term
|
Definition
|
|
Term
| Unlike the CFR, Guidance Documents are not: |
|
Definition
|
|
Term
| Guidance Documents represent: |
|
Definition
| current FDA thinking and should (must) be followed; FDA references these documents |
|
|
Term
|
Definition
| No observed adverse effect level |
|
|
Term
|
Definition
| one cannot conduct nonclinical trials |
|
|
Term
| Dept of Agriculture was created |
|
Definition
|
|
Term
| the Dept of Agriculture brought forth: |
|
Definition
| the Division of Chemistry & the Food and Drug Administration |
|
|
Term
| the Food & Drug Administration was created |
|
Definition
|
|
Term
| the Dept of Health & Education was created |
|
Definition
|
|
Term
| the Dept of Health and Human Services was created |
|
Definition
|
|
Term
| Congress created FDA by statute in |
|
Definition
|
|
Term
| (Mission Statement) The FDA is responsible for |
|
Definition
| 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. |
|
|
Term
| (Mission Statement Cont'd) The FDA is also responsible for |
|
Definition
| 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. |
|
|
Term
| The FDA Commissioner ensures: |
|
Definition
| that the Agency carries out its mission of protecting and advancing the public health |
|
|
Term
| The Commission was appointed by _______ the Pesident and confirmed by _____teh Senate in ______ |
|
Definition
| the President; the Senate; 1988 |
|
|
Term
|
Definition
| Commisioner; CDER; CBER; CDRH; CFSAN; CVM |
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
| New Drug Development Timeline: (1) Pre-Clinical Testing, R&D |
|
Definition
| range: 1-3yrs (Avg 18mo); Initial Synthesis; animal testing **short term** |
|
|
Term
| New Drug Development Timeline: (2) Clinical R&D |
|
Definition
| range: 2-18yrs (Avg 5yrs); Phase 1,2&3; **long-term** |
|
|
Term
| Between Pre-clinical testing R&D and Clinical R&D Phases on New Drug Development Timeline there is a: |
|
Definition
|
|
Term
| New Drug Development Timeline: (3) NDA Review time |
|
Definition
| FDA time to review NDA; Range: 2mo - 7yrs (Avg 24 mo) from submission of NDA to approval of NDA |
|
|
Term
| New Drug Development Timeline: (4) Post-Market Surveillance |
|
Definition
| Adverse reaction reporting; surveys/sampling/testing; inspections |
|
|
Term
|
Definition
|
|
Term
|
Definition
| "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…." |
|
|
Term
|
Definition
|
|
Term
|
Definition
| 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. |
|
|
Term
| Section 201 (p)(1) of the FD&C Act - New Drugs |
|
Definition
| 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 |
|
|
Term
| New Drugs as defined in 21 CFR 310.3 |
|
Definition
| 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. |
|
|
Term
| the following are subject to FDA approval: |
|
Definition
| 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. |
|
|
Term
| New Drugs: Exceptions are |
|
Definition
| "Grandfather Drugs"; Homeopathic Drugs; Dietary Supplements |
|
|
Term
|
Definition
| Center for Drug Evaluation and Research; responsibility is to ensure drugs are safe and effective; responsibility for both prescription and over-the-counter drugs |
|
|
Term
| sponsor responsibility w/New Drugs |
|
Definition
| 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. |
|
|
Term
| Overview of primary steps to approval w/new drugs |
|
Definition
| (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. |
|
|
Term
| Nonclinical Testing is testing for |
|
Definition
|
|
Term
| the purpose of nonclinical testing is: |
|
Definition
| (1) to seek some evidence of the drug's biological activity (2) to show that it is reasonably safe for initial administration into humans |
|
|
Term
| Nonclinical testing should follow: |
|
Definition
| Good Laboratory Practices (GLP) |
|
|
Term
| General animal welfare provisions contained in federal state animal protection statutes |
|
Definition
|
|
Term
| nonclinical testing means a new drug is labeled: |
|
Definition
| "Caution-contains a new drug for investigational use only in laboratory research animals, or for tests in vitro. Not for use in humans." |
|
|
Term
| Safety in nonclinical testing is usually demonstrated by: |
|
Definition
| (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 |
|
|
Term
| toxic and pharmacological effects are studied in: |
|
Definition
|
|
Term
| genotoxicity screening is performed in: |
|
Definition
|
|
Term
| drug absorption, metabolism, distribution, and excretion (how is the drug cleared from the body?) is tested in: |
|
Definition
|
|
Term
| FDA expectations of nonclinical testing are: |
|
Definition
| (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 |
|
|
Term
| IND can be thought of as: |
|
Definition
| the product of a successful preclinical development program |
|
|
Term
| A sponsor assembles and submits an IND if: |
|
Definition
| based on preclinical studies, the pharmacology of a compound justifies further development and it is adequately safe for initial small-scale clinical studies |
|
|
Term
| A drug that is used in clinical investigations in humans |
|
Definition
|
|
Term
| Such use of an investiagtional new drug (a drug that is used in clinical investigations in humans) is covered by the: |
|
Definition
| Investigational New Drug Application (IND) |
|
|
Term
| An Investigational New Drug Application (IND) is also called: |
|
Definition
| Notice of Claimed Investigational Exemption for a New Drug Form FDA 1571 |
|
|
Term
|
Definition
|
|
Term
|
Definition
| a marketing application (it only allows a sponsor to test the drug) |
|
|
Term
|
Definition
| for exemption from the federal statue which prohibits transport of unapproved drugs in interstate commerce |
|
|
Term
| A sponsor requests this type of mtg depending on what the purpose is |
|
Definition
|
|
Term
| A formal request for an FDA meeting should be made in writing to the agency (clock starts ticking) and should list: |
|
Definition
| objectives and discussion points |
|
|
Term
| An IND Type A, B, or C meeting w/FDA will be schedule |
|
Definition
| within 60 days of agency's written receipt of request |
|
|
Term
| 4 weeks prior to Type A,B, C mtg this must be sent |
|
Definition
|
|
Term
| 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 |
|
Definition
| Background package for FDA IND Type A, B, C mtg |
|
|
Term
| if a sponsor has no clinical data to submit w/background package for IND Type A, B,C mtg, the sponsor can submit: |
|
Definition
| a rational for what the chemical will do |
|
|
Term
| A ____ is filed by companies whose goal is to obtain marketing approval of a new product |
|
Definition
|
|
Term
| Three types of Non-Commerical IND's are: |
|
Definition
| (1) Investigator IND (2) Emergency Use IND (3) Treatment IND |
|
|
Term
| ____ IND is submitted by an investigator who initiates and conducts the investigation |
|
Definition
|
|
Term
| The motivation of an Investigator IND is NOT |
|
Definition
|
|
Term
| A physician must submit a research IND to propose: |
|
Definition
| studying an unapproved drug or an approved product for a new indication or in a new patient population |
|
|
Term
| An Emergency Use IND is also called: |
|
Definition
| "Compassionate Use" or "Single Patient" INDs |
|
|
Term
| An Emergency Use IND allows the FDA to authorize use of an: |
|
Definition
| experimental drug in an emergency situation that does not allow time for submission of an IND. |
|
|
Term
| ___ 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 |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
| IND maintenace includes the following three: |
|
Definition
| (1) Amendments (protocol and safety) (2) safety reports (3) annual reports |
|
|
Term
|
Definition
|
|
Term
| 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&E data |
|
Definition
|
|
Term
| ____ 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. |
|
Definition
|
|
Term
| Risk vs Benefit Phase (or) At what dose can we get the most benefits w/the least risk |
|
Definition
|
|
Term
| Phase __ typically closely monitored and may be conducted in patients or normal volunteer subjects |
|
Definition
|
|
Term
| Phase 1 is designed to determine: |
|
Definition
| the metabolism and pharmacological actions of the drug in humans, the side effects associatd with increasing doses. |
|
|
Term
| while Phase 1 studies are intended to gain early evidence of effectiveness…. |
|
Definition
| you almost never get early evidence of effectiveness bc the dose is too small or the patient population is too small to be accurate |
|
|
Term
| Phase 1 studies aer designed to gather sufficient info about: |
|
Definition
| the drug's pharmacokineticsadn pharmacologicl effects to permit the design of well-controlled, scientifically valid, Phase 2 studies. |
|
|
Term
| Number of subjects and patients included in a ___ study are generall in the range of __ to __. |
|
Definition
|
|
Term
| Phase 1 studies also include studies of drug metabolism, structure-activity relationships, and mechanism of action in humans, as well as: |
|
Definition
| studies in which investigational drugs are used as research tools to explore biological phenomena or disease processes. |
|
|
Term
| ___ studies are typically well controlled, closely monitored, and conducted in a relatively small number of patients, usually involving no more than several hundred subjects. |
|
Definition
|
|
Term
|
Definition
| 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. |
|
|
Term
| Phase 3 studies are after: |
|
Definition
| a sponsor gets efficacy data from Phase 2 study |
|
|
Term
| Study to develop package insert |
|
Definition
|
|
Term
| the goal of expanded controlled and uncontrolled trials during phase 3 studies are to |
|
Definition
|
|
Term
| ____ study are performed after preliminary evidence suggesting effectiveness of the drug has been obtained |
|
Definition
|
|
Term
| Phase 3 studies are intended to gather the additional information about the effectiveness and safety that is needed to evaluate: |
|
Definition
| the overall benefit-risk relationship of the drug and to provide an adequate basis for physician labeling |
|
|
Term
| Phase 3 studies include from several |
|
Definition
| hundred to thousand subjects. |
|
|
Term
|
Definition
| cover sheet for IND application |
|
|
Term
| the name, address, and telephone number of the sponsor, the date of the application, and the name of the investigational new drug. |
|
Definition
|
|
Term
| identification of the phase or phases of the clinical investigation to be conducted |
|
Definition
|
|
Term
| a commitment not to begin clinical investigations until an IND coverign the investigations is in effect |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
| a commitment to conduct the investigation in accordance with all other applicable regulatory requirements. |
|
Definition
|
|
Term
| the name and title of the person responsible for monitoring the conduct and progress of the clinical investigations |
|
Definition
|
|
Term
| 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. |
|
Definition
|
|
Term
| 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. |
|
Definition
|
|
Term
| the signature of the sponsor or the sponsor's authorized rep. |
|
Definition
|
|
Term
| A table of contents and introductory statement and general investigational plan (what you plan to do statement) |
|
Definition
|
|
Term
| A brief summary of previous human experience with the drug. (you can compare experience to that of other companies) |
|
Definition
|
|
Term
| 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. |
|
Definition
|
|
Term
|
Definition
| Absorbtion Distribution Metabolism Excretion |
|
|
Term
| A brief description of the overall plan for investigating the drug product for the following year. (based on pk data) |
|
Definition
|
|
Term
|
Definition
|
|
Term
| A brief description of the drug substance and the formulation, including the structural formula, if known. |
|
Definition
|
|
Term
| A summary of the pharmacological and toxicological effects of the drug in animals, and, to the extent known, in humans. |
|
Definition
|
|
Term
| A summary of the pharmacokinetics and biological disposition of the drug in animals and, if known, in humans. |
|
Definition
|
|
Term
| A summary of information relating to safety and effectiveness in humans obtained from prior clinical studies. |
|
Definition
|
|
Term
| 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. |
|
Definition
|
|
Term
|
Definition
|
|
Term
| Everyone has done "this" so you can do can/will do the same |
|
Definition
|
|
Term
| Chemistry, manufacturing, and control information (CMC) |
|
Definition
|
|
Term
|
Definition
|
|
Term
| Environmental analysis requirements |
|
Definition
|
|
Term
| Pharmacology and toxicology information |
|
Definition
|
|
Term
| Previous human experience with the investigational drug |
|
Definition
|
|
Term
| 3 main components of the IND |
|
Definition
| (1) nonclinical (preclinical animal testing); (2) Chemistry, Manufacturing, and Controls - CMC Data (3) Clinical Data |
|
|
Term
|
Definition
| (1) Administrative (2) Summary (3) CMC (4) Nonclinical Data (5) Clinical Data |
|
|
Term
|
Definition
|
|
Term
|
Definition
| Good Laboratory Practices (GLP) |
|
|
Term
|
Definition
| good manufacturing practices |
|
|
Term
| The first step for ___ ___ ___ in Nonclinical studies is to develop some in vivo and in vitro data |
|
Definition
| New Molecular Entities (NME) - per nonclinical studies |
|
|
Term
| FDA is willing to discuss non-clinical testing requirements with |
|
Definition
| Sponsors (nonclinical studies) |
|
|
Term
| Nonclinical studies must be conducted according to |
|
Definition
|
|
Term
| ____ provide basic pharm/tox information |
|
Definition
| animal studies (nonclinical studies) |
|
|
Term
| long-term animal studies are performed even after |
|
Definition
| human clinical trials are underway (per nonclinical studies) |
|
|
Term
| Allow safe introduction of molecule into humans |
|
Definition
| goal of nonclinical testing |
|
|
Term
| determine minimum recommended starting dose (based on the NOAEL - no observed adverse effect level |
|
Definition
| goal of nonclinical testing |
|
|
Term
| understanding of pharmacokinetics (PK) and pharmacodynamics (PD) |
|
Definition
| goal of nonclinical testing |
|
|
Term
|
Definition
| what the body does to the drug (extent and duration of exposure; ADME) |
|
|
Term
|
Definition
| what the drug does to the body (dose responses; Adverse effects on physiologic systems) |
|
|
Term
| Choice of ____ ____ in nonclinical testing is based upon regulatory requirements, relevance of species, and/or existing disease models |
|
Definition
|
|
Term
| organization and personnel |
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
| test and control article characterization |
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
|
Definition
|
|
Term
| drug structure, proposed indication, target population, route of administration, duration or use, and special characteristics are ___ ___ ___ |
|
Definition
| Nonclinical testing considerations |
|
|
Term
|
Definition
| Efficacy Quality Safety Multidisciplinary |
|
|
Term
| _____ studies let you determine if you have enough info to allow to dose safely |
|
Definition
|
|
Term
| Acute, Chronic, Carcinogenicity, Genotoxicity, and reproductive ____ are all types of ___ ____ |
|
Definition
| toxicity; toxicity studies |
|
|
Term
| 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 |
|
Definition
| Chemistry, Manufacturing, and Controls (CMC ) |
|
|
Term
| drug substance studies, drug product studies, placebo & drug and biologics specific information all are types of |
|
Definition
|
|
Term
| drug ____ is the active form, not the final form |
|
Definition
| substance (ie: acetomenaphin) |
|
|
Term
| drug _____ is the final dosage form (what you take) |
|
Definition
|
|
Term
| clinical protocol, informed consent form, and investigator's brochure are all |
|
Definition
|
|
Term
| ____ protocol is a document given to patients that explains everything (risks vs benefits, etc) |
|
Definition
|
|
Term
| Central focus of FDA IND review, rationale for study, design of study, endpoints, and assessments are all part of the |
|
Definition
|
|
Term
| Determining appropriate clinical success points and if conducting appropriate testing can be considered |
|
Definition
|
|
Term
| ____ _____ Form is required per 21 CFR 50 (Protection of Human Subjects) |
|
Definition
|
|
Term
| the Informed Consent Form provides subjects with |
|
Definition
| a description of study procedures and risk/benefit |
|
|
Term
| _____ _____ is a 30 day FDA multidisciplinary review of clinical, nonclinical, and CMC datat |
|
Definition
|
|
Term
|
Definition
| approved; if 30 days lapses w/out hold from FDA you move forward |
|
|
Term
| 30 day notification of protocol status as part of the IND process is ___ ____ and ____ ___ ___ |
|
Definition
| clincal hold; no clinical hold |
|
|
Term
| Most INDs today are submitted in |
|
Definition
|
|
Term
| Module 1 Administratie; Module 2 Summary; Module 3 Quality (CMC); Module 4 Safety (Nonclinical); Module 5 Efficacy (Clinical) |
|
Definition
|
|
Term
|
Definition
|
|
Term
| New Drug Application and Biologics License Application are essentially |
|
Definition
|
|
Term
|
Definition
| Biologics License Application |
|
|
Term
| NDA regulations 21 CFR 314 is a __ not a ___ |
|
Definition
|
|
Term
| these documents are required for FDA review and approval prior to the marketing of a drug or biologic |
|
Definition
| NDA Regulations 21 CFR 314 |
|
|
Term
|
Definition
| statistical analysis program |
|
|
Term
| meeting with the reviewing division to discuss the presentation of the info in the dossier |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
| Majority of NDA license applications are submitted in the |
|
Definition
|
|
Term
| NDA process overview is comprised of |
|
Definition
|
|
Term
| Form ____h - Application to market a new drug, biologic, or an antibiotic for human use |
|
Definition
|
|
Term
| User Fee cover sheet, paient information financial disclosure, overall table of contents (Index), labeling, and technical sections are all ____ _____ |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
| ____/____ defines your NDA application |
|
Definition
|
|
Term
| CMC, Pharmacology/Toxicology, human pharmacokinetics, microbiology, clinical, risk/benefit are all _____ sections of the NDA |
|
Definition
|
|
Term
|
Definition
| active pharmaceutical ingredient |
|
|
Term
| (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 |
|
Definition
|
|
Term
| 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 ____ |
|
Definition
|
|
Term
| 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 ____ |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
| in pharmacology, ______ is used to describe the fraction of an administered dose of medication that reaches the systemic circulation |
|
Definition
|
|
Term
| 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____ ____ |
|
Definition
| human PK and Bioavailability studies |
|
|
Term
| _____ Section is only required for certain categories of drugs (such as anti-infectives) |
|
Definition
|
|
Term
| Microbiology Section reports on how the target organism is affected - the ______ _____ of activity |
|
Definition
|
|
Term
| Mechanisms of resistance, spectrum of activity, and methods to evaluate activity are part of the |
|
Definition
|
|
Term
| 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 ___ _____ |
|
Definition
|
|
Term
| Common Technical Document (CTD) structure: |
|
Definition
| (1) Module 1 Administrative (2) Module 2 Summary (3) Module 3 Quality - CMC (4) Module 4 Safety (nonclinical); (5) Module 5 Efficacy (Clinical) |
|
|
Term
| FDA NDA review time goals are established under |
|
Definition
|
|
Term
|
Definition
| time from the first NDA submission to NDA approval |
|
|
Term
|
Definition
| 6 mo. Review performance goal. Product is determined to provide significant therapeutic or public health advance "Fast Track" |
|
|
Term
|
Definition
| 10-12mo review performance goal |
|
|
Term
| Once an NDA has been filed, new and/or revised data or info can be submitted as |
|
Definition
|
|
Term
| medical reviewer; pharm/tox reviewer; CMC reviewer; statistics; microbiology; bioresearch monitor (verifies data) are all part of the |
|
Definition
|
|
Term
| A sponsor does NOT file an NDA, it _____ and NDA |
|
Definition
|
|
Term
| core team has a 45 day meeting to determine if NDA can be filed or labeled |
|
Definition
|
|
Term
| RTF letters are usually within ____ days of NDA receipt |
|
Definition
|
|
Term
| if omissions from NDA are not on the critical path review then may NOT lead to |
|
Definition
|
|
Term
| FDA will usually inform sponsor of "_____ ______" w/in 14 day of the 60-day application filing date |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
|
Definition
| follows technical reviews: usually coordinated by the medical reviewer; unresolved issues may be brought before an Advisory Committee |
|
|
Term
| approved products are often subject to the following post-approval changes: |
|
Definition
|
|
Term
| Advertising/Labeling is regulated by: |
|
Definition
| the Division of Drug marketing, Advertising,and Communications |
|
|
Term
| Division of Drug Marketing, Advertising, and Communications reviews package labeling, advertising, and attends drug conferences to ensure |
|
Definition
|
|
Term
| advertising/labeling that is not in compliance can be labeled |
|
Definition
|
|
Term
|
Definition
| if you state a positive abou the data, you should also state a negative (adv/labeling) |
|
|
Term
| 21 CFR 312, Subpart D of IND regulations is |
|
Definition
| good clinical practices GCP |
|
|
Term
|
Definition
|
|
Term
| Good Clinical Practice: Consolidated Guidance is under |
|
Definition
|
|
Term
| Responsibility (Investigator & Sponsor - ultimate responsibility); record keeping; and essential documents are all found in |
|
Definition
|
|
Term
| GCP Documentation Includes |
|
Definition
| Clinical Protocol; Investigator's Brochure (overview for rationale of study); Informed Consent; Declaration of Helsinki |
|
|
Term
|
Definition
| **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 |
|
|
Term
| 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 |
|
Definition
|
|
Term
| _____ commitees provide FDA with independent advice from outside experts on issues related to human and veterinary drugs, biological products, medical devices, and food. |
|
Definition
|
|
Term
| Members of an _____ ______ include a Chair, several members, plus a consumer, industry and sometimes a representative |
|
Definition
|
|
Term
| Although the committees provide advice to the Agency, final decisions are made by the |
|
Definition
|
|
Term
| 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 |
|
Definition
|
|
Term
| Significant public interest, controversy, and additional expertise are all reasons why the FDA may call an |
|
Definition
| advisory committee meeting |
|
|
Term
| sponsor has to provide a briefing document for an |
|
Definition
| advisory committee meeting |
|
|
Term
| advisory committees can have |
|
Definition
| open and closed sessions; open sessions often have public comment |
|
|
Term
| the term "____ _____" can refer to either a durg or biologic intended for use in a rare disease or condition |
|
Definition
|
|
Term
| A drug or biologic becomes an orphan drug when: |
|
Definition
| it receives orphan-drug designation from the Office of Orphan Products Development at the FDA. |
|
|
Term
| Orphan-drug designation qualifies the sponsor to receive _____ _____ from the Gov't in exchange for _____ _____ ____ for a rare disease/condition |
|
Definition
| certain benefits; developing the drug |
|
|
Term
| Ophan drugs must go through the ____ _____ _____ process like any other drug or biologic which evaluates for safety and efficacy |
|
Definition
|
|
Term
|
Definition
| Content and format of a request for orphan drug designation |
|
|
Term
| A statement that the sponsor requests orphan drug designation for a rare disease or condition, which shall be identified with specificity |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| The name and address of the sponsor |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| A description of the rare disease or condition for which the drug is being or will be investigated, the proposed indication or indication(s) for use of the drug, and the reasons why such therapy is needed. |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| A description of the drug and a discussion of the scientific rationale for the use of the drug for the rare disease or condition, including all data from nonclinical lab studies, clinical investigations, and other relevant data that are available to the sponsor, whether positive, negative, or inconclusive. copies of pertinent unpublished and published papers are also required. |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| where the sponsor of a drug that is otherwise the same drug as an already approved orphan drug seeks orphan drug designation for the subsequent drug for the same rare disease or condition , an explanation of why the proposed variation may be clinically superior to the first drug. |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| where a drug is under development for only a subset of persons with a particular disease/condition, a demonstration that the subset is medically plausible |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| A summary of the regulatory status and marketing history of the drug in the US and in foreign countries |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| Documentation the disease or condition for which the drug is intended affects fewer that 200,000 people in the US or, if the drug is a vaccine, diagnostic drug, or preventative drug, the persons to whom the drug will be administered in the US are fewer than 200,000 per year as specified in |
|
Definition
| orphan drug application 21 CFR 316.20 |
|
|
Term
| Three reasons to pursue orphan drug status |
|
Definition
| (1) potentially faster development pathway (2) marketing exclusitivity (3) strategic development options |
|
|
Term
|
Definition
|
|
Term
| ____ ____ is a program intended to make promising products for life threatening diseases available on the market on the basis of preliminary evidence prior to formal demonstration of paitient benefit. |
|
Definition
|
|
Term
| Accelerated Approval studies are designed to _____ and the FDA evaluation is performed on the basis of a _____ _____ marker that is considered likely to predict patient benefit. |
|
Definition
| measure; surrogate marker (ie: response rate) |
|
|
Term
| Accelerated Approval granted may be considered a ____ _____ with a written commitment to complete clinical studies that formally demonstrate _____ ____. |
|
Definition
| provisional approval; patient benefit. |
|
|
Term
| Endpoint choice is critical with |
|
Definition
|
|
Term
| Fast Track is part of the |
|
Definition
| Food and Drug Modernization Act of 1997 |
|
|
Term
|
Definition
| Food and Drug Modernization Act of 1997 |
|
|
Term
| ____ _____ is a process designed to facilitate the development, and expidite the review of drugs to treat serious diseases and fill an unmet medical need |
|
Definition
|
|
Term
| Any drug being developed to treat or prevent a disease w/no current therapy obviously is directed at an ____ _____ |
|
Definition
|
|
Term
| A fast track drug must show some advantage over available treatment such as: showing superior effectiveness; avoiding serious side effects of an available treatment; improving diagnosis of a serious disease where early diagnosis results in an improved outcome and/or: |
|
Definition
| decreasing a clinically significant toxicity of an accepted treatment |
|
|
Term
| A drug that received Fast Track designation is eligible for some or all of the following: (1) more frequent meetings w/FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval (2) more frequent written correspondence from FDA about such things as the design of the proposed clinical trials (3) |
|
Definition
| Eligibility for Accelerated Approval, i.e. approval on an effect on a surrogate, or a substitute endpoint reasonably likely to predict clinical benefit. |
|
|
Term
| _____ Review, which means that a drug company can submit completed sections of its New Drug Application (NDA) for review by the FDA, rather than waiting until every section of the application is completed before the entire application can be reviewed. NDA review usually does not begin until the drug company has submitted the entire application to the FDA |
|
Definition
| Rolling Review - part of Fast Track |
|
|
Term
| Rolling NDA sections submitted separately are: |
|
Definition
| CMC, nonclinical, clinical |
|
|
Term
| Resource allocation, strategic advantages, and separately submitted sections are all part of |
|
Definition
|
|
Term
|
Definition
|
|
Term
| A product comprised of two or more regulated components- ie: drug/device, biologic/device, drug/biologic, or drug/device/biologic, that are physically, chemically or otherwise combined or mixed and produced as a single entitity is a |
|
Definition
|
|
Term
| A combination product is also two or more separate _____ packaged together in a single package or as a _____ and comprised of drug and device products, device & biological products, or biological and drug products |
|
Definition
|
|
Term
| A combination product is also any ______ ______, ______, or ______ product packaged separately that according to its proposed labeling is for use only with another individually specified investigational drug, device, or biological product where both are required to achieve the intended use, indication, or effect. |
|
Definition
|
|
Term
| ____ products require the consideration of different regulations (e.g. drug and device); the impact of either product must be considered, and understanding the interactions between products is critical |
|
Definition
|
|
Term
| Label expansion - Post marketing studies can be both |
|
Definition
| sponsor initiated and investigator initiated |
|
|
Term
| post marketing issues include _____ surveillance - _______ |
|
Definition
| safety; pharmacovigilance |
|
|
Term
|
Definition
| Division of Drug Marketing, Advertising, and Communications |
|
|
Term
|
Definition
| advertising; print, tv, radio and info at conferences/tradeshows |
|
|
Term
| _____ reviews package labeling |
|
Definition
|
|
Term
|
Definition
| Prescription Drug Advertising |
|
|
Term
| Regulatory and corporate compliance must ensure compliance with regulation: |
|
Definition
| sales, marketing, research |
|
|
Term
| "Real World" use of drug; expansion of patient population; and off-lable use are all subject to |
|
Definition
| post market safety surveillance; monitoring drug safety following approval. |
|
|
Term
| drug that is identical or bioequivalent to a "Listed Drug" is known as a |
|
Definition
|
|
Term
| _____ Drug is a: new drug that is approved under ND; drug listed with an effective approval in "Approved Drug Products with Therapeutic Equivalence Evaluations" |
|
Definition
|
|
Term
| "The Orange Book" is formally called |
|
Definition
| Approved Drug Products with Therapeutic Equivalence Evaluations |
|
|
Term
|
Definition
| Abbreviated New Drug Application |
|
|
Term
| generic drugs demonstrate ______ with listed drugs |
|
Definition
|
|
Term
| Package Insert (PI); summary of relevant and important drug development info; provides basis of claims; and what you can say about your product are all components of |
|
Definition
|
|
