1. In 1983 the Orpan Drug Act was passed. Under what conditions can a drug qualify for this status?
    Any rare disease or condition or condition which (a) affects less than 200, 000 persons in the US or (b) affects more than 200, 000 persons in the US but for which there is no reasonable expectation that the cost of developing and making available in the US will be recovered from the sales. (Chapter 2)
  2. What does the Orphan Drug Act guarantee the developer of the product?
    The developer of the product gets 7 years of market exclusivity following FDA’s approval of the product. Other incentives are tax credits for clinical research undertaken by the sponsor to generate required data for the product. (Chapter 2)
  3. What act authorized ANDAs and specified that only bioequivalence trials are required?
    Hatch-Waxman Act (drug Price competiton and patent term restoration act) (Chapter 2)
  4. What was the agreement with FDA and industry under PDUFA of 1992 and MPDUFA of 2002?

    A. FDA can collect fees from companies and FDA had to meet timelines
    B. FDA got to yell at companies and companies had to bitch about it
    FDA had set timelines in response to company lobbying efforts
    C. FDA established fees for Premarket apporoval applications, supplements and 510(k) in exchange for timeline commitments
    D. A and D
  5. What is a Notice of Change?
    Clinical Trial sponsor can make modifications to any investigational device or study protocol that doesn’t effect safety or study design by submitting the notice of change within 5 days after instituting the change.
  6. What clinical trials have to be registered registry databank?

  7. Under FDAAA of 2007, what is required for data from clinical trials that will be used as a basis for an efficacy claim or postmarketing studies? (regarding making it public)
    They are required to be posted online. $10000 penalty may be charged, if not corrected within 30 days, another $10, 000 may be charged per day.
  8. What is required of the sponsor regarding submission timelines for DTC advertising?
    Sponsor is to submit to FDA no later than 45 days before dissemination.
  9. True or false. Regulations have the force of law, where guidance documents and guidelines establish principles or practices but do not have the force of law.
  10. What is a Unified Agenda?
    It is a regulatory agenda that is published semi-annually that summarizes each agency’s planned rulemaking activities for the next six months.
  11. Public comments to new regulations/revisions are submitted during the comment period. Upon expiration of the typical 60 day period what actions can the FDA take?
    • 1. Promulgate interim or final rules
    • 2. Extend the comment period
    • 3. Abandon its intention to promulgate a rule
  12. What is the purpose of an FDA petition and what must it contain?
    • Petitions are requests to FDA to issue, change, or cancel a regulation.
    • A petition must contain:
    • 1. Action requested
    • 2. Statement of grounds
    • 3. Environmental impact
    • 4. Official certification statement
    • 5. Petitioner identifying information
  13. What are the 5 drug pathways?
    • 1. New drugs
    • 2. OTC
    • 3. Generic
    • 4. Grandfathered drugs
    • 5. 505(b)(2) New Drug Application
  14. What would constitute a “grandfathered drug”
    • - Generally recognized as safe, can be introduced without an NDA provided claims and formulation remain the same
    • - They include all drugs marketed prior to 1938 and are exempt from safety and efficacy requirementsSome drugs marketed prior to 1962 are exempt from efficacy requirements. FDA did an efficacy “proof” in 1962
  15. What is a 505(b)(2) NDA?
    This permits applicant to submit data not developed by applicant for approval by the FDA. Applicants can rely on published literature or agency’s previous findings of safety and effectiveness for an approved drug.
  16. What are the 9 medical device pathways?
    • - Establishment registration and device listing
    • - Traditional 510(k)
    • - Abbreviated 510(k)
    • - Special 510(k)
    • - Humanitarian device exemptions (HDE)
    • - De novo classification
    • - Traditional premarket approval (PMA) - Modular PMAProduct development protocol (PDP)
  17. What registration pathway would you use for a Class 1 medical device?
    Establishment Registration and device listing.
  18. What are the requirement pathways for traditional 510(k)?
    • - Class II device and some Class III
    • - Requires demonstration of “substantive equivalence” to a “predicate” device previously cleared
    • - Modifications that affect safety and effectiveness are subject to 510(k) submission requirements
  19. An FDA petition must contain which of the following?

  20. Drugs may be eligible for over-the-counter status when:

  21. Biologics are cleared for marketing through which process?

    d. All of the above
  22. A Special 510(k) relies on the following information:

  23. Which act required rulemaking meetings to be open to the public?

  24. What is the difference between the USC and CFR?
    • USC is the US code
    • CFR – official compilation of regulations
  25. how are veterinary drugs managed and by which division?
    • CVM - centre for vet medicine,
    • INAD - investigational new animal product
    • NADA - new animal drug application
    • Biologics are covered by US Department of agriculture
  26. how do animal drug submissions differ from human?
    • the ability to use known data from teh development of a drug for humans or other animal species if applicable
    • the requirement of safety and efficacy for smaller number of animals (vs. large human numbers)
    • ther relevence of species, class, breed of animal as well as geographic differences
    • the differentiation of drugs developed for treatment of companion animals vs. treatment of food animals
  27. To be eligible for an ANDA submission, the generic product must show bioequivalence to the reference listed drug (RLD). Under what conditions would a product get a waiver?
    products that have high solubility-high permiability rating such as steriles, otics (ear), and ophthalmics (eye)
  28. What is the orange book?
    The orange book identifies the RLD and provides information on applicable patents and their expiration dates.
  29. What are the 3 major parts of the ANDA
    • Chemistry and Manufacturing
    • Bioequivalnce Study
    • Administrative information (labels, patents)
  30. What are the typical BE studies to prove equivalance to a RLD?
    typically two-way crossover studies in the fasted state, if there is a food effect indicated in the label, a fed state study is also conducted
  31. For bioequivalence studies done in support of an ANDA what is not required?

  32. An ANDA must contain a statement by the applicant one of the following patent certifications applies to the RLD:
    • paragraph I: patent information has not been filed
    • paragraph II: patent has expired
    • paragraph III: patent will expire at a specific date, and the sponsor of the ANDA does not intend to market the product before that date
    • paragraph IV: the patent is invalid or will not be infringed by the proposed drug product
  33. What is a 30-month stay
    if an ANDA applicant certifies that the patent is invalid, or ANDA will not infringe the patent. They have to contact the patent owner (NDA holder), if the NDA holder brings an action for patent infringement within 45 days, the FDA will not approve the ANDA for 30 months, or per court orders
  34. What are the rules for 180 day exclusivity.
    If one or multiple companies submit ANDA with a paragraph IV challenging the patent on the same first day. This provides a shared incentive for multiple companies willing to challenge a listed patent
  35. what is the average ANDA review and approval time`
    2 years
  36. What is a suitability petition?
    Filed by sponsor, seeks permission to file ANDA for the drug product and contains comparisons and contrasts between the generic drug and the RLD. CRF314.93 for what characteristics a generic drug not identical to the RLD. FDA has 90 days to respond.
  37. What is the difference from a branded generic and an authorized.
    • Branded generic - approved by the ANDA process but distinguishes itself from other generics with a brand name.
    • Authorized generic - licencing agreement between the innovator and generic drug manufacturer. The generic manufacturer markets the generic under a different name. The generic can be added to the generic NDA as a supplement or described in the NDA Annual Report.
  38. What is a "drug"
    A product used in diagnosis, curing, mitigating, treating or preventing a disease or affecting a structure or function of the body.
  39. What is considered a "new drug"
    • new drug use of any substance which composes the drug
    • there is a new drug use of a combination of approved drugs
    • the proportion of the ingredients in combination has changed
    • there is a new intended use for the drug
    • the dosage, method or duration of administration or application is changed
  40. CBER or CDER who regulates biologic therapies?
  41. What is SPA?
    • Special Protocol Assessment. Within 45 days of reciept FDA evaluates certain protocols.
    • covered protocols:
    • - animal carcinogenicity protocols
    • - final product stability protocols
    • - Phase III
  42. why would an orphan drug designation be revoked?
    if the sponsor provided false evidence on the application or if it turned out that there was not enough drug product to support the indication. if post designation prevelance exceeds 200, 000 the status cannot be revoked.
  43. Fast Track designation?
    • designed to expedite development and review for new drugs designed to meet unmet medical needs for serious/life threatening diseases.
    • Request can be made at any time, causes hightened FDA interest, increases chances of priority review (6 mo vs. 10 mo) and rolling FDA eligibility.
  44. what is a P-IND number?
    if there was a pre-IND meeting, this is the number given to the IND, the final IND number will be the p-IND number, minus the p.
  45. How do you get IND approved.
    Trick question! IND is not approved its a request for exemption from federal law. Federal law requires that prior to transporing or distributing a drug in interstate commerce an NDA or BLA must be approved by FDA.
  46. what is form 1572
    • contains study detail, can have more than one form/IND
    • one form per study
  47. Form 1571?
    contains sponsor and drug information and an indication of what the submission contains
  48. what is the physician's labeling rule?
    • for the product insert. contains 3 parts:
    • 1. highligts (1 page)
    • 2. contents
    • 3. full prescibing information
  49. REMS
    • Risk evaluation and mitigation strategy. Must be submitted with each new product, product dependant. Can be as simple as including patient insert and as complicated as a physician training program.
    • At min. it indicates a post approval evaluation at 18 months, 3 years, 7 years
  50. What is a PAI
    GMP pre-approval inspection. if the site hasnt been recently inspected by FDA with a favorable outcome. An adequate PAI is required for approval. Can be done as early as 90 days after submission.
  51. What are the periapproval activities for an NDA?
    • 120 day safety update
    • Label negotiations
    • National Drug Code designation
  52. Establishment Licencing
    Required for all establishments involved in manufacture, preparation. propagation, compounding, processing. Use FDA form 2656 to apply. Use form 2657 to list drug products.
  53. NCEs get 5 years of exclusivity. How can additional exclusivity be granted to a drug product?
    • 1. 6 months pediatric exclusivity
    • 2. up to 3 years exclusivity for a change in approved drug product where approval required new clinical investigations
  54. What are ther 6 most common uses of the 505(b) route?
    • 1. change in drug product form
    • 2. change in active ingredient
    • 3. new combination product
    • 4. OTC switch
    • 5. new indication
    • 6. NCE that is a prodrug or active metabolite of a drug already approved
  55. what is the difference between drug CGMP and dietary supplement CGMP?
    Drug CGMP require process validation
  56. What are dietary supplement claims NOT allowed under DSHEA?

  57. What are some prohibited statements for dietary supplements under the DSHEA?
    Manufactures cannot claim, or express an implied claim that a product will diagnose, mitigate, treat, cure, or prevent a specific disease or class of diseases.
  58. If the standards of relevant monograph are met for the OTC product, what is the approval process?
    no approval is required.
  59. What is the official definition of devices
    • an instrument, apparatus, implement, machine, implant, in vitro agent including a component part or accessory which is:
    • - recognized in the official national formulary or untited states pharmacopoeia
    • - intended for use in the diagnosis of disease or other conditions, or in teh cure, mitigation, treatment, or prevention of disease in man or other animals
    • - intended to affect hte structure or any function of the body
    • - does not achieve any of its primary intended purposes though chemical actions within or on the body
    • - does not depend on being metabolized for the achievement of its primary purpose
  60. CDRH?
    FDA's Centre for Device and Radiological Health regulates firms that manufacture, repackage, relabel, import medical devices in US
  61. What are the general controls for all med devices?
    • 1. establishment licence
    • 2. listing with FDA
    • 3. GMP manufacturing
    • 4. labelling in accordance to labelling regulations
    • 5. submission of a premarket notification before marketing a device (unless exempt)
  62. which level of med devices require pre market approval?
    III - devices for which insufficient information exists to assure safety and effectiveness solely through general or special controls
  63. HUD?
    Humanitarian use device... for less than 4000 patients in the US. If application accepted, device eligible for HDE.. humanitarian device exemption - exempt from effectiveness requirements. Can only be used in area where IRB process is established
  64. 510(K) exemption
    to be submitted when a premarket authorization is not required. Tells the FDA that this device is safe and effective as a predicate device
  65. how can a device be substantially equivalent
    • has the same intended and the same technological characteristics as a predicate
    • OR
    • has the same intended use as the predicate and different technological characteristics and the info submitted to FDA does not raise new questions of safety and effectiveness and demonstrates that the device is at least as safe and effective as the legally marketed device
  66. IDE
    Investigational device exemption... lets a device be used in a clinical study to collect safety and effectiveness data required to support a PMA or 510K (not usually required)
  67. How long does the FDA have to respond to a citizen's petition?
    180 days of reciept
  68. what should be contained in a citizen petition?
    • action requested
    • statement of grounds
    • environmental impact
    • economic impact
    • a certification by the submitter
  69. what are suitability petitions?
    • a petition which seeks to file an abbreviated application for a new drug whose active ingredient, route of admin, dosage form or strength, differ from that approved drug.
    • FDA has 90 days to approve/deny
  70. what is a determination meeting for a med device?
    limited to PMAs or product development protocol applications, the purpose is to determine the type of valid scientific evidence needed to demonstrate that a device is effective for tis intended use
  71. what are type b meetings?
    regular meetings scheduled within 60 days of request. Pre-submission, pre-IND, end of phase I, end of phase II/pre phase III
  72. how long does FDA have to schedule a type C meeting?
    75 days within request.
  73. yThe following studies are not subject to GLP except

  74. In GLP.. distinguish between ther responsibilities of the testing facility management, study director, and Quality Assurance unit (QAU)
    • Testing facilities management - responsible for designating a study director, and replacing if necessary. Has authority to provide resources and implement changes necessary to comply with GLP requirements and take corrective action.
    • Study director - overall responsibility for study design, conduct, and reporting
    • QAU-maintain master schedule, and documentation. Ensure no deviation without proper documentation. Inspect studies at intervals and prepare a statement to include in the final study report that specifies dates of inspection and findings.
  75. True or false. Quality Assurance Unit must review and approve all the standard operating procedures applicable to a nonclinical test laboratory.
  76. For nonclinical studies, corrections to the signed and dated final study report may be made in the form of a report ammendment by the:

  77. What is HIPAA?
    • Health Insurance Portability adn Accountability Act - establishes min requirements for protecting individually identifiable medical records - Privacy Rule.
    • Protects health status and payment for health sevices
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