First Test Flashcards
1
Q
Need Finding Perspective
A
Patient - stress, pain, lost functions, death
Provider - malfunction, risk, uncertainty
Payer - cost, inefficiency
2
Q
Observations methods:
A
- read published data
- ask questions, interviews
- watch people do stuff
- work as part of a team
3
Q
Disease fundamentals
A
- anatomy & physiology
- pathophysiology (physiology of disease)
- clinical presentation: symptoms, signs
- clinical outcomes: most common outcome of disease
- epidemiology: causes, distribution, control
- economic impact: cost
4
Q
Need Screening: Treatment Options
A
- behavioral/ lifestyle modifications
- pharmacologic/ biologic therapy
- radiation
- minimally invasive therapy: laparoscopy, catheter
- open surgery
5
Q
Need screening: stakeholder concerns
A
Stakeholders: payers, patients, providers
Concerns: clinical outcome, safety, convenience, risk, ease of use, cost, reputation, economic impacts, competition
6
Q
need screening: market analysis
A
- market segmentation (disease, treatment, providers, payers)
- market size
- market dynamics
7
Q
Amount spent on healthcare per person per year in the US
A
$8000
8
Q
Typical cost to develop a drug in the US
A
$1 B+
9
Q
FDA has authority over
A
- sale of drugs
- sale of medical devices
- transportation of drugs across state lines
10
Q
Affordable Care Act of 2010
A
- the essential health benefit package, a minimum set of services covered by insurers
- employer mandate (requires employers to insure workers)
- individual mandate
11
Q
A US patent provides you with the right to:
A
Exclude others from practicing your invention
12
Q
The leading cause of death in developing countries
A
perinatal conditions
13
Q
Four stages of technology life cycle
A
- Emergence (losing money)
- Growth
- Maturation (gains money)
- Decline (gains less and less money)
14
Q
Disruptive Innovations:
A
-New technology, renders old technology obsolete
-Previously unmet medical needs, or dramatic reduction in cost,
or improvement in access
15
Q
Sustaining Innovation
A
- Extends life of current tech.
- improved patient experience, responsive to payers’ policies, or providers’ practices
- improved performance, efficacy, or reduced side effects
- responsive to changes in market, marketing strategies
16
Q
Occupational Safety
A
- Occupational Safety and Health Act established OSHA in 1970
- Part of the Department of Labor
- issues standards such as “bloodborne pathogens”, “nonionizing radiation”, “occupational exposure to dangerous chemicals in labs”
- Enforces standards by training, outreach, inspections
17
Q
Examples of OSHA guidance: Hierarchy of Control
A
- engineering controls (fume hoods, biological safety cabinets)
- administrative controls (chemical hygiene plan, standard operating procedures)
- work practices (no mouth pipeting, hand washing, chem. substitution)
- personal protective equipment (gloves, goggles, respirators)
18
Q
Employer Responsibilities
A
- Follow all OSHA relevant health and safety standards
- inform employees about hazards through training, etc.
- Keep accurate records of work-related injuries and illnesses
19
Q
Occupational Safety at University of Arizona
A
- Risk Management Services (RMS) coordinates university risk management efforts
- General Lab Safety Training
- Speciality Training
20
Q
What laws protect pregnant women?
A
- Pregnancy Discrimination Act (PDA) of 1978: issued pregnant women as a protected group
- Americans with Disabilities Act of 1990: amended in 2008 to say some pregnant health concerns qualify as disability
- Equal Employment Opportunity Commission (2014): issued a guidance to PDA (reasonably accommodate pregnant women) (not a law)
- NO OSHA laws
21
Q
What are specific rights of pregnant women
A
- Employers can’t refuse to hire you because of your pregnancy if you are capable of job responsibilities
- Can’t be fired because of pregnancy
- Women can decide when to stop working (the same way as disability leave)
- You may request appropriate accommodations
22
Q
Pregnancy Discrimination Act Limits
A
- Only applies to companies with 15 or more employees.
- EEOC exempts employers if they prove keeping you on in pregnant condition would pose unreasonable hardship on company
23
Q
Controlled Substances
A
- Any drug regulated by government
- Regulate by Drug Enforcement Agency & Department of Justice
- Title 21 USC: Controlled Substance Act (CSA)
- Medical use: II-IV
- No medical use: I
24
Q
Controlled Substances Details
A
- Security and Record management required by DEA
- requirements for storage, tracking, use, purchase, and disposal
- PI required to have DEA registration certificate
- purchase of narcotics must have statement signed by Principal Investigators that the drugs will be used in line with policies
25
Animal Use
- Regulated by Animal Welfare Act of 1966
- Administered by the US Department of Agricultural
- rats, mice, and birds are not protected
- Farm animals are not protected
26
Funding for research from Public Health Services
- research must resubmit the Animal Welfare Assurance form every 5 years and update annually to ensure research follows Public Health Services policies
- By PHS mandate, each institution must have a Institutional Animal Care and Use Committee (IACUC) to oversee animal use
27
Examples of animal use
- research involving live animals
- collection of any living animal byproducts
- collection of tissues post-mortem for research or teaching
- field trapping of animals
- handling animals for UA course or teaching activity
- observational field studies
- production of custom antibodies
- routine care and use of livestock
28
Activities involving animals not overseen by IACUC
- teaching or research with invertebrate species
- purchase of commercially available antibodies
- purchase of commercially available animal products
29
Structure of Human Subject Protection in the US
Ethical Principle: Belmont Report, 1978
Law: 45 CFR, part 46 (Protection of Human Subjects, 1981)
Policy: "The Common Rule", 1991
Compliance: Institutional Review Board
30
Belmont Report
1978
- Respect to persons
- Beneficence
- Justice
31
Research Involving Humans - Laws
- protections for pregnant women, human fetuses, and neonates in research
- additional protection for research involving prisoners
- protection for children in research
- registration for IRB
32
Human Subject
living person who a researcher gathers data from by:
- intervention: physical procedures
- interaction: communication or interpersonal contact between investigator and subject
- Private Information: information about subject which they can reasonably expect not to be shared publicly
- Identifiable information: self explanatory
33
Research involving Humans - Policy
- A Policy for the Protection of Human Research Subjects has been adapted by several agencies aka the "Common Rule", of 1991
34
Research Involving Humans: Assurances and Reviews
-The Federal-Wide Assurance is an institutional assurance accepted and approved by the OHRP (institution must train investigators)
-Each FWA holder must designate at least one IRB registered with
OHRP
35
Definition of Medical Device
- intended to affect the structure or function of body of man or animal
- does not achieve its primary intended purpose by chemical means
- is not dependent on being metabolized
36
Drug definition
Articles (other than food) that affect the structure or function of the body of man and animals
37
Combination Product definition
A product comprised of two or more regulated components (biologic, drug, or medical device)
38
Food and Drug Administration (Devices)
- Regulates broad range of medical devices
| - Has authority to regulate medical devices before and after they reach market
39
Class I Med. Dev.
- minimal risk
| - subject to general controls: registration, labeling, etc
40
Class II Med. Dev.
- does not sustain or support human life
- mild risk (Ex: wheelchairs)
- subject general controls and special controls: Premarket Notification 510(k), mandatory performance standards
41
Class III Med. Device
- sustains or supports life
- potential risk
- general controls and Premarket Approval
42
Premarketing Requirements for Med Dev
- 510(k) exemption and listing
- 510(k) premarket notification
- PMA or premarket approval
43
510(k) Exempt
- Not reviewed by FDA
- mostly class I med devices but some class II that are deemed exempt
- FDA has database of exempt devices
- must still meet general controls requirements
44
510(k) Premarket Notification
- class II devices, some class I
- low to no risk devices
- must show substantial equivalence to FDA approved predicate dev.
- 510(k) forms includes name of dev., description of device, comparison to predicate device(s), and purposed labeling (directions for use, labeling)
45
Premarket Approval
- Class III device (most risk), similar to drug approval process
- No standard form for PMA
- must include results of nonclinical testing and results of clinical tests for safety and efficacy
46
Investigational Device Exemption (IDE)
- request to allow clinical trials on unapproved device as part of PMA or general research
- Significant risk (needs approval by FDA and IRB), Non-Significant risk (approval by IRB only), Exempt studies (No IDE required)
- includes sponsor information (files IDE), investigator information (could be outsourced like Contract Research Organization), and human subject protection information
47
Protocol of Clinical Investigations
1. Statement of objectives
2. method of selection of subjects
3. method of observation and recording data
4. comparison of results with control
5. methods of data analysis
48
FDA response to IDE
- approval or disapproval/withdrawal, or request for more information
- investigation can not be done until 30 days after FDA receives IDE
49
Marketing Status of Med Dev
- FDA Listed
- FDA cleared (premarket notification 510(k) submitted)
- FDA Approved (accepted PMA)
50
Drug approval process
Drug discovery -> preclinical trials -> clinical trial -> FDA review -> LG Scale MFG
51
Pharmacology and Toxology
- Animal Testing (one rodent & one nonrodent species, use few animals)
- Pharmacology: pharmacodynamics (interaction w/ bio matrix, receptors) & pharmacokinetics (absorption, distribution, metabolism, excretion)
- Toxicity (single dose acute toxicity w/ 14 day observation)
52
Investigational New Drug (IND) application
- Requirement for all clinically tested drugs
- Needed for drug transport across state lines
- Commercial & non-Commercial (investigator IND, emergency use IND, treatment IND) IND's
- content includes clinical protocols, chemistry, manufacturing and control (CMC) information
- PharmaTox info
53
Participants of clinical trials
- investigator (files IND)
- sponsor (a physician)
- sponsor-investigator
- human subject
54
Drug Master File (DMF)
-FDA submitted filed with confidential information on facilities, processes, articles
55
FDA responses to IND
- discussion of deficiency
- appeal and resumption
- clinical hold
- inactive status
- termination
56
Clinical Testing Phases
- Phase I: to establish safety and safety dose range(20-80 subjects)
- Phase II: efficacy for specific indication (several hundred subjects)
- Phase III: compare drug to current standard of care (several hundred or thousands of subjects)
57
Clinical Study Designs
-uncontrolled
-controlled
-randomized
-blinded
-double blinded
-open-label trial
GOLD STANDARD: Randomized Control Trial
58
New Drug Application (NDA)
- Sponsor proposes FDA grants permission for sale of drug
- FDA reviewer checks: safety & efficacy, appropriate purposed labeling, methods used in manufacturing
- Contains CMC info, Pharmatoxic info, clinical data, pharmokinetics and bioavailability
59
Intellectual Property (IP)
- any product of human mind
- has value in market place
- can be reduced to tangible form
- has 4 forms (patents, copyrights, trademarks, trade secrets)
60
Patents (first to file wins)
- a right to exclude other from practicing your invention
- Title 35 "patents" of the US code
- US authorizes US patent and Trademark Office
- judicial power is in district courts
- 3 types: utility (useful, novel, non-obvious, specific), design, and plant (asexual))
61
utility patent
- contains: title page, specifications aka disclosure (background and detailed description), and claims
- must be non-obvious, novel, useful, statutory class (article of manufacturing, composition, machine, method, or a new use of the above)
62
US patent filing process
- Provisional Patent application (establish filing date, patent pending status, limit 12 months until standard patent)
- Standard Patent application (20 year protection for utility patent, 14 year for design patent)
- Continuation patent application (new embodiments)
- Continuation in part (new claims and new embodiments)
- Divisional Application
- Reissue
63
International Patent Protection
- Regulated by Patent Cooperation Treaty 1970
- PCT is administered by World Intellectual Property Organization
- First phase is international, second phase is national (file with each state)
64
IP Counsel
- Prosecution: Applicant- Patent Office interaction related to patent application and maintenance (DIY, Patent Agent, Patent Attorney)
- Litigation: legal actions related to infringement of an issued patent
65
Trade Secrets
-Information that has economic value to holder based on secrecy and is subject to effort to maintain secrecy
66
Uniform Trade Secrets Act
- Tenth amendment creates federal preemption (powers not given to federal government are given to states and people)
- Uniform Trade Secrets Act is a proposed law that states may choose to follow (hence trade secrets are protected by states)
67
International Trade Secret Protection
- The Agreement in Trade-Related Aspects of Intellectual Property Rights (WTO) 1994
- Economic Espionage Act of 1996
- North American Free Trade Agreement (NAFTA) , 1994
68
Trade Secret Discovery
- Improper means of discovery: bribery, theft
| - Proper means: reverse engineering, independent invention, published literature, licensing
69
Trademarks
- requires registration with USPTO
- requires efforts to maintain
- if not maintained it is subject to dilution or abandonement
70
Copyright (includes software)
- Exclusive right to use and distribute original work granted to its creator
- in 1989, US signed the 1886 Berne Convention
- copyrights are automatic
- has fair use and first sale doctrines
- Sonny Bono Copyright Term Extension Act of 1998
71
copyleft license
- any modifications to work are freely available, any derivatives of work are public
- example:GNU General Public License
72
Creative Commons License
allows to define which specific rights the author reserves
73
Who gets most patents
company: IBM
Biomed company: johnson and johnson
University: University of California
74
What can you do with intellectual property?
- commercialize (develop a product)
- sell it (trade rights for money)
- license it
- put it on a shelf (patent troll)
75
Do's and don't's of keeping a notebook
- Do: identify the notebook, use bound (numbered) book, date & sign each page
- Don't: back-date, remove page, white off anything
76
Authorship
- First Author: major role in everything
- Senior Author: PI
- Corresponding author: usually senior author that corresponds with editor of journal
77
Drug Development Costs and Time
10 - 15 years
| $1,000 M and more
78
Goals of SBIR program
- stimulation of tech. innovation
- meet federal needs
- aid disadvantaged persons
- increase private-sector commercialization
79
Agencies involved in SBIR/STTR
Department of Energy, Dept. of Defense, NASA, National Science foundation, dept. of health and human resources
Total is $2.5B
80
Phases of SBIR
Phase I: establish technical merit
Phase II: continue R&D efforts
Phase III: pursue commercialization objectives
95% grants and 5% contracts
81
3 "I"s of Biomedical Engineering
1. Identify Need
2. Invent the solution
3. Implement
82
Private Investors
- Angels: emerging markets, $50-$250 k
| - Venture Capital
