1. drug
   1. any substance other than food or device by affecting structure or functions
      1. diagnosis
      2. curing
      3. treatment
2. type of drug based on active ingrediant
   1. pharmacuetical drug
      1. asily identified and ccharacterized
   2. biologics
      1. not easily identified and characterized
         1. antibodies,growth factors, vaccines, blood products, stem cells
      2. include proteins
         1. more than 40 aa
         2. chemicallly synthezsized prot less 100 aa not regulatd as biologic product
   3. drugs can be classified
      1. precripstion
      2. OTC
      3. Generic drugs
         1. storebrands, copies of brand name drugs, same dosage form, strenth, route of adniminastation
      4. orphan drugs
         1. target diseases affecting less than 200,000 patients
      5. dietary supplements
         1. do not require FDA approval prior to marketing
3. FDA regulates
   1. medical devices and diagnostics
      1. pacemakers, in vitro
   2. combinations products
      1. two or more drugs
         1. flu
         2. cough surpressant/pain reliever
4. drug product
   1. a finished dosage form (CFR 21) containing activive drug ingredient generally in association w inactive ingredients
      1. in cinical trials, placebo does not contain active ingredient
5. component
   1. any ingredient for use in manufacturer of drug product (may be purified out)
6. active ingredient
   1. responsible for drug properties of drug product
   2. afect srtucutre an dfunction of man or animal
7. inactive inredient
   1. any component other than inactive (binders, starch, sweeteners,artificial color
8. strengt
   1. conecentration of drug substance
      1. different measurements
         1. vitamins (IU) - international units - is a standard of therapeutic activity
   2. therapuetic activit y as indicated by appropriate lab test or by adequately controlled clinical data
9. Hatch-Waxman Act of 1984
   1. encouraged manufacture of genetics
10. BIologics Price Competition and innovation act 2009 (BPCI)
    1. biosimilars
    2. for analytical technqures analyzing biologics sidegroups
    3. biosimilar approval
       1. no clinalyy meaning difference bw biosimalr and branded
       2. same strength and units of strength
       3. if no concerns on licensed, studies not required
       4. PREA requirements waived if interchangeable
11. Prscription Drug USers Free Act
    1. mandated funding to FDA to hire new drug reviewers to accerlareate approval proces
12. Fodo and Drug Administration SAfety adn Innov Act of 2012 (FDAASIA)
    1. similar requirements
    2. collect user fees for innovators
13. Biosimilar User Free Act of 2012
    1. under, inital fee is only 10% of fee that branded developer pared
       1. for an application requireing clincal data
14. Orphan Drug Act of 1983
    1. drugs meant to treat low amt patients
    2. FDA incentives to encourage development
       1. tax incentives
       2. clincla trial statistical burdens lessened
          1. not many ppl required as for diabetes
       3. patent protection
       4. ammended: Waive PDUFA fees
15. Rare Disease act of 2002
    1. increase funding
16. FDA Organization
    1. Office of Commisioner
       1. office of foods
          1. center for food safety and applied nutr
          2. " for Vet Mediccine
    2. Office of Regulatory Afairs
    3. ?????
17. Lab to Medicine Cabinent
    1. drug dev basis
       1. volumes of papers to FDA in drug development process
          1. discovery
             1. Subtopic 1
          2. optimize chemical
          3. test animals 9preclinal
             1. IND
             2. if approved fda
             3. test phase 1,2,3
             4. if safe and effective in phase
             5. manufacturer files nda
             6. it gets approved
    2. early development
       1. drug disc and lead optmization
          1. how does drug work
             1. what enzyme targeted by drug that stops cancer
          2. optimixzation - change to improve product 9i.e. solubility)
       2. non clincal studies
       3. submission (Investigation of new drug applications
    3. clinical studies
       1. phase 1
          1. drug safety and toxicity
       2. phase 2
          1. efficacy od drug in target dis
          2. Subtopic 2
       3. phase 3
          1. determine increasing doses in larger group of people, maximum dose to be effective
    4. sumbsiion
       1. drug manuf fda
18. product dev objective
    1. define clinical indication
    2. det population for therapy
    3. determine financial value of product
    4. strategy for treatment of disease/condition
    5. demonstrate safety and efficacy of candidate product in patients with disease/condition
19. manufacturing objectives
    1. develop process to produce the product at reasonable cost
       1. comercials by designated date
    2. assemble people w manuf extpertise
       1. we come in to support cliical progr
    3. in what way will product fail?
    4. establish timelines
       1. goals? date
    5. ???
20. Product development team
    1. tens of thousands
    2. less than 100 employees
    3. analytical dev
       1. chmist biologists
       2. Subtopic 2
    4. cliical affairs
       1. statisti
    5. project management
       1. course???
21. department rsponsibilities
    1. research
       1. discover ptimize and char activity of drug
    2. assay development
       1. clincal assays, nalaytical methods 9urine)
    3. process development
       1. optimize cell lines, manufacturing process
          1. small scale to large scale
    4. manufacturing
       1. supply GLP/GMP material as appropriate
    5. clinical devlopment
       1. develop study protocols, manage clincal plan
    6. clincal operations
       1. execute clinical studies, manage the budgest
    7. QA/QC
       1. perform in vitro comparability/consistency asays on product
    8. Regulatory
       1. navigate reg landscapes
          1. always changes, new laws/ammendments, old laws ammends. taken on
             1. can effect profitability of product
    9. businessdevelopment process
    10. ????
22. market analysis
    1. disease incidence
       1. rare, lot of money,
       2. disease resistance
       3. target people who dont respond to first line of drug
    2. treatment
       1. current options
       2. impact on duration
       3. longer life expectancy?
    3. market expectation
       1. sales projections
       2. physician practice
       3. pricing issues
          1. hillary
             1. insurance pays for (we affected)
    4. drug developments spend money for huge obese market instead of third world
    5. vaccines: one time use
23. clincal target
    1. product rationale, there wil always be market for new trag
       1. non responders to currently avaible drugs
       2. development of drug resistance
       3. better side-effect profile, less toxicities
       4. simpler dosing regimens
       5. better dosage form
          1. oral preferred over intravenous
       6. combination therapies
          1. chemotherapies, more effective than single alone
24. process development
    1. " group
       1. optimize cell line, pan proc
       2. Subtopic 2
    2. considerations for biologics
       1. Genetic Stability in such cells (dont want recominant gene to be lost0)
       2. Ease to grow cells
       3. Yiel should be high of recomb protein
       4. Yield of formulated prufied product
25. manufacturing plan
    1. asses drug needs
       1. comparability studies
       2. GLP (non GMP production)
       3. small clinical studies, outsources trials small scale productions
    2. ??
26. Quality plan and strategy
    1. methods (SOPS) writeen implent, and validated
    2. stick with plan! asses that line
    3. ????
27. Regulatory considerations
    1. ?????