RAC Prep Drugs Questions & Answers Rated 100% Correct!!

drug - An article intended for use in the diagnosis, cure mitigation treatment or prevention of disease in man new drug - -New use of a drug substance or component (active ingredient, excipient, carrier, coating). -New use of a combination of approved drugs -Change in proportion of ingredients in a combination drug -New intended use of a drug -Change in dosage, method or duration of administration or application active moiety - Central, active part of a molecule or ion responsible for the drug's physiological or pharmacological action. -excludes those appended portions of the molecule that cause the drug to be an ester, salt (including a salt with hydrogen or coordination bonds), or other noncovalent derivative (such as a complex, chelate, or clathrate) of the molecule new chemical entity (NCE) - A drug that contains no active moiety that has been approved by the FDA in any other application submitted under section 505(b) New molecular entity (NME) - Product that has not been previously available for therapeutic use in humans and is destined to be made available as a prescription-only medicine Orphan drug - drugs for a disease or condition that affect 200K people in US -FDA will provide formal protocol assistance if requested -Grants are available - no fee Rare pediatric disease voucher - Sponsors who submit a marketing application for a rare pediatric disease can be eligible for a Priority Review Voucher for any subsequent applicationOrphan drug annual report - Shall include: -drug development progress -description of upcoming investigational plan -changes that may affect orphan status Fast track designation - Began with FDAMA 1997 For new drugs that: -Show superior effectiveness, effect on serious outcomes or improved effect on serious outcomes -Avoid serious side effects of an available therapy -Improve the diagnosis of a serious condition where early diagnosis results in an improved outcome -Decrease a clinical significant toxicity of an available therapy that is common and causes discontinuation of treatment -Can address emerging or anticipated public health need -More frequent meetings with FDA to discuss the drug's development plan and ensure collection of appropriate data needed to support drug approval -More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers -May be requested at any time during development (including @ IND submission) -FDA will provide response in 60 days (designation or non-designation letter) -Eligible for accelerated approval -Eligible for priority review -Eligibility for rolling review Breakthrough designation - Began with FDASIA 2012 For new drugs that: -An effect on an established surrogate endpoint -An effect on a surrogate endpoint or intermediate clinical endpoint considered reasonably likely to predict a clinical benefit (i.e., the accelerated approval standard)-An effect on a pharmacodynamic biomarker(s) that does not meet criteria for an acceptable surrogate endpoint, but strongly suggests the potential for a clinically meaningful effect on the underlying disease -A significantly improved safety profile compared to available therapy (e.g., less dose-limiting toxicity for an oncology agent), with evidence of similar efficacy Adds to benefits of Fast Track -Intensive guidance on an efficient drug development program, beginning as early as Phase 1 -Organizational commitment involving senior managers -Submit concurrent to IND or no later than EoP2 meeting -FDA will respond within 60 days Accelerated approval - For drugs that: -allows approval based on surrogate or an intermediate clinical endpoint -reduces clinical trial timeline -disease must be serious or life-threatening -treatment must be better than existing treatments Priority review - For drugs that: -provide major advances in treatment or provide treatment where no therapy exists, -eliminates or reduces treatment-limiting reaction, -evidence of enhanced patient compliance, -evidence of safety and compliance in a new subpopulation such as children -Must be requested upon NDA or BLA submission -Can be added to a Fast Track or Breakthrough Therapy, but it must be requested separately. -FDA will respond within 60 days -Faster review than standard applications. 6 mo review instead of typical 10 mo. Rolling review - -Clock doesn't start until all modules have been submitted, but FDA can start review anytime after 1st module submitted-unique to Fast Track and Breakthrough designations -Initiate discussion at pre-NDA or -BLA meeting Preapproval inspection (PAI) - Can occur as early as 90 after NDA submission -successful PAI at facility is required for NDA approval -Can be GMP and/or BIMO inspections -Usually conducted in the following situations: NMEs, original BLAs, priority reviews, sponsor's first application, for cause, application if it has been more than 2 years since last inspection. Drug registration and listing system (DRLS) - Includes prescription and OTC drugs —system to which drug establishment and drug listings are submitted -electronic only now Establishment listing — must be done electronically - Required for all manufacturers (domestic and foreign) and initial distributors (importers) For establishment registration must be completed within 5 days of beginning manufacturing or upon submission of marketing application and then each 12/31. For listing products in commercial distribution due within 3 days of initiation registration and then each 6/30 and 12/31 Proprietary name submission and review - Name submitted after P2 trials or with NDA -For names submitted during IND, FDA will respond within 180 days -For names submitted with NDA, FDA will respond within 90 days -Names will be re-reviewed 90 days before approval to make sure they are still appropriate. Pediatric Study Plan (PSP) - Mandatory for all products covered under PREA -due w/in 60 days of EoP2 meeting, before beginning P3 studies, or if no P2/3 studies, 210 days before NDA/BLA submission -7 month review timelinePediatric Written Request - Issued by FDA and requests pediatric studies be performed -it is a prerequisite for 6 mo pediatric exclusivity -if pediatric data is submitted w/o having received a request, it will invalidate the Sponsor's right to exclusivity -can be requested by submitting a Proposed Pediatric Study Request (response issued by FDA within 120 days) -must be submitted at least 15 months before exclusivity ends since it takes 6 mo to review and there must be 9 mo left to add Proposed Pediatric Study Request - Request for pediatric written request -Response issued by FDA within 120 days New Drug Application (NDA) - Application in eCTD format with FDA Form 356h. Must include the following: -SUMMARY -Labeling -pharmacologic class -brief marketing history -CMC -Nonclinical -Human PK and BA -Micro for anti-invectives -Clinical data -Benefit/risk -TECHNICAL -CMC -Drug substance -Drug product -Stability