USPS Midterm 1 > Pharma Quiz 1 > Flashcards

Pharma Quiz 1 Flashcards

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1
Q

Duchenne muscular dystrophy

A

New medication for rare disease
clinical trial of 12 boys, no significant benefit, FDA didn’t approve. Launched in 2017 - price experienced exponentially

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2
Q

Free Market

A

Unregulated system of voluntary, economic exchange. Governed by invisible hand and law of supply and demand. Absence of coerced transactions. Resource allocation pareto efficient

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3
Q

Market equilibrium

A

Q supplied equals q demanded

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4
Q

Rise in supply can be caused by 3 things

A
  1. Lower prod costs
  2. More firms increasing total supply
  3. Stable economic environment
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5
Q

Fall in demand caused by 4 things

A
  1. High price
  2. Change in income
  3. Cheaper alternatives
  4. Access barriers
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6
Q

Market Failures are when

A

prices do not fully reflect the costs or benefits of goods (policy to avoid detriment)

Competitors collude

Transaction costs

Pareto optimal resource allocation violate principles of distributive justice and fairness

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7
Q

Market failures require

A

government interventions

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8
Q

3 focuses of pharma policy over drug life cycle

A
  1. drug discovery and development
  2. Clinical Development
  3. Post marketing
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9
Q

Drug Discovery and Development

A

Free market driven by scientific novelty and investment attractiveness (AMCs, small biotech firms, VCs)

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10
Q

Clinical Development

A

R&D to prove safety and efficacy through clinical trials (Big Pharma)

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11
Q

Post Marketing

A

Assessing quality, safety, effectiveness in rboader pop. Access and afforability, competition, and supply

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12
Q

What % of worldwide pharma market does the US make up in 2021?

A

40%

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13
Q

List price vs next price equation

A

list price - rebates/discounts -wholesaler fees - additional price concessions = manufacturer realized price/net price

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14
Q

What percent of individuals have private health insurance

A

about 2/3

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15
Q

Private health insurance plans composed of

A

pre-paid fixed premiums
cost sharing when utilization

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16
Q

What part of medicare covers prescription drugs?

A

part D, largely funded by premiums

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17
Q

Pharma bro story

A

daraprim for deadly parasitic infection. acquired by martin shkreli, ceo of turing pharma. price went from 13.50 to 750. Was fine a monopoly abuse

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18
Q

key areas responding to market failures

A
  1. resource allocation
  2. price
  3. transaction costs/competition
  4. efficient and equitable use of tax payer contribution
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19
Q

Innovation is

A

any practice, invention, idea, that is percieved as new in a field/industry

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20
Q

Key criticism around innovation theories

A

pro-innovation bias
issues of equality
demand for innovation vs. supply of innovation

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21
Q

5 things we want to see from biopharma innovation

A
  1. breakthrough, first in class
  2. more effective
  3. safer
  4. cost effective
  5. convenience
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21
Q

What is biopharma innovation

A

meds saving and improving lives of pop. Can either better target diseases, or keep patients out of the hospital

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22
Q

Challenges for policymakers regarding biopharma innovation

A

measuring innovation
changing definitions of pharma products
changing definitions of patients
inelastic demand for innovation

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23
Q

4 types of biopharma products

A

small molecules
biologics
me too drugs
orphan drugs

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24
small molecules
new chemical entity approved by an NDA has a trademarked, properitary name, protected by patents
25
large molecules (biologic)
requires living organisms to be products uses a BLA also refers to the originator drug
26
me too drugs
minimal chemical differences make it different from generic (isomer, dosage, form) essentially creates a new monopoly
27
orphan drug
less than 200,000 patients rare health co ndition can be small molecules or biologics, and may have non-orphan inidcations
28
5 steps of Drug Development Process
1. Drug discovery and development 2. preclinical research 3. clinical research 4. fda review and approval 5. post market safety monitoring
29
Who usually does which steps in the drug development process
Academic medical centers do steps 1 and 2. Big pharma do steps 3-5
30
time frames for each step
drug discovery + preclincial = 6.5 years clinical = 7 years approval = 6-10 months
31
Step 1. Discovery and development
New insights, tests, treatments, technologies. Run scientific experiements
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Step 2. Preclinical Development
Research to find potential toxicity in vitro or in vivo.Still must use good lab practices
33
Step 3. Clinical Research
Studies done in people. Must submit IND to Drug before beginning research.
34
What to include in an IND?
animal study data and toxicity manufacturing clinical protocols data from prior research investigator info
35
Phase 1
20-100 (health or with disease) 1.5 years Purpose is to test safety and dosage 70% progress
36
Phase 2
several 100 with disease 2 years purpose: efficacy and safety 33% progress
37
Phase 3
Several thousand Purpose: safety and efficacy 3.5 years 25-30% progress
38
Step 4. FDA review and approval
If NDA/BLA complete, review team has 6-10 months to make decision
39
What 4 dimensions must a drug pass to come to the market?
Safety and efficacy Do benefits outweigh risks Appropriate labelling Adequate manufacturing methods
40
Step 5. FDA post-market safety monitoring
reporting problems Passive surveillance: voluntary reports from providers and consumers If manufacturer receives a report, must send to FDA manufacturer inspections Drug advertising Generic Drugs
41
Zolgensma
first gene therapy for spinal muscular atrophy
42
Pharmaceutical R&D market cap
300 bill
43
Breakdown of funders in R&D
Big biopharma is the largest funder - 300 bn Non-profit and public sectors - more than a quarter Remaining 10% - VCs (APAC)
44
NIh does a TON of funding
very little related to phased clinical development, most the eearly drug discovery and preclinical phases
45
NIH 3 evaluation criteria
1. Importance of Research 2. Rigor and Feasibility 3. Expertise and Resources
46
What determines if a drug is developed up to launch?
Expected financial return
47
Timeline of funding
public sector/non profits fund early VC helps the translation gap Big biopharma helps with late stage
48
Why is the industry concerned about drug pricing regulations
impact ROI, lower biopharma R&D investment and innovation
49
Pure Food and Drug Act of 1906
Spurred by rapid industrialization and poisonous drugs aimed at preservative,s mostly guarded labeling (enforced by bureau of chemistry and poison squad)
50
Food Drug, and Cosmetic Act 1938
Authorized FDA to oversee safety of food, drugs, cosmetics, medical devices mandated food quality standards required drugs be labeled for safe use required drugs be labeled for safe use and prohobited false therapeutic claims clarified fda's right to conduct factory inspections mandated pre-market approval of all new drugs, so manufacturer would have to prove to FDA that drug was safe before it could be sold
51
1951 Durhan-Humphrey Amendment
Establshed two categories Rx OTC
52
1962 Kefauver-Harris Amendment
Thalidomide Disaster Required drugs to prove efficacy Instituted stricter agency control over drug trials GMPs Transferred Rx regulation from FTC to FDA
53
PDUFA of 1992 (Prescription Drug User Fee Act
get fees from manufacturers for approval
54
FDA proactive powers
approval of new drugs/food additives requiring preventive safety
55
FDA reactive
inspections citations/seizures withdrawals
56
FDA enforcement tools
Review/approval of INDs, NDA, Manufacturing inspections Regulatory guidance Court
57
What is a drug? Defined by FDCA section 201
Part B: treating/mitigating/diagnosing disease Part C: Changing function of the body
58
Defining "new drug" under the FDCA
A drug not genrerally recognized by experts as safe and effective for use under the conditions recommended in the drug's labeling
59
Without an NDA, a drug cannot be marked unless
generally recognized as safe and effective grandfathered drug
60
Requirements and legal standards to recieve an NDA
Safety and efficacy Legal standards: safety and effiectiveness FDA will work for labelling
61
OTC products evaluated by...
therapeutic category. Can be marketed through a monograph or the NDA process
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RX to OTC Switch two versions
Full switch Partial switch
63
4 FDA incentives for innovation
1. patents/exclusivity 2. Accelerate Approval 3. Drug Development Designations 4. Economic incentives
64
Accelerated Approval 2 parts
1. Subpart H (accelerated approval programs) Expedite approval of new drug products to treat life-threatening diseases 2. Emergency Use authroizations temproary authorizations during emergency scenarios Go to FDA review after phase 2
65
3 Drug Development Designations
1. Fast Track - unmet medical need 2. Breakthrough therapy - substantial improvement from SOC on a clinicall significant endpoints 3. Priority Review review within 6 months to 10 months (for drugs that will signfiicantly improve treatment, diagnosis, and prevention of a serious condition)
66
Different Economic incentives
Preapproval and post approval rewards Orphan Drug Program Other funding/grant opps Longer exclusivity
67
Tuskegee Syphillis Experiemnt 1932-1972
on black popualtions
68
3 ethical principles of human subject research
autonomy beneficience justice
69
The common rule
What non-FDA studies must conform to
70
practice vs research
practice tries to enhance wellbeing. research si to develop or contirbute to generalizable knowledge (therapeutic misconception)
71
2019, how much did pharma spend on R&D
83 bill, 10x what industry spent in 80s
72
How many new drugs increased compared to previous deacades
by 60%, average 38 drugs 2019-2019. 59 drugs in 2018
73
How much of revenue does pharma spend on R&D
1/4
74
Trends in types of drugs being approved
more specialty, more biologics (oncology, diabetes, and immunology)
75
3 main factors influencing spending on R&D
1. anticipated revenues 2. Costs of prodution 3. Federal poligies and programs influencing supply and demand
USPS Midterm 1
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