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PHRM 510 > Lecture 14: Drug Development and Biosimilars > Flashcards

Lecture 14: Drug Development and Biosimilars Flashcards

1
Q

What Constitutes a “Drug”?

A

A drug is a substance that produces change in cellular or physiologic functioning

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2
Q

Drug development influenced by:

A

High cost of research; complex regulatory environment & legislations;
insurer reimbursements; and patient perspectives

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3
Q

Some of factors that influence drug development include

A 
 Development cost (~ $1.3 - $2.8 billion or more) and time (can be as much as or >15 years)
 Regulatory approval process
 Changes in laws
 Patient acceptance
 Insurance reimbursement
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4
Q

Investing in new drug development is

A

expensive and risky

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5
Q

FDA Regulates drugs under two legislative acts

A
  • Food, Drug, and Cosmetic Act – for small molecule drugs

- Public Health Service Act – for biologicals

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6
Q

The FDA’s mission is to promote and protect the public health by promoting:

A

safe and effective products reach the market in a timely safe and effective products reach the market in a timely way
- However: once FDA determines a drug is safe and effective, it has little control over
its use in medical practice, such as for non-approved indications (“off-label” use)

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7
Q

Specific FDA Function/Role in Drug Development

Process and Drugs in the Marketplace

A
  • Approves drugs on the basis of purity, safety, efficacy
  • Regulate all labeling of prescription and nonprescription drugs
  • Advertising OTCs the responsibility of the Federal Trade Commission (FTC)
  • Regulate manufacturing processes of drugs and institution of drug recalls
  • Regulate bio-equivalence for generics and bio-similars
  • Monitor safety of US blood supply
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8
Q

Conceptualization

A

performed by independent investigators, small research companies, academic institutions & large pharmaceutical companies

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9
Q

Intramural

A

NIH investigators perform research

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10
Q

Extramural

A

independent investigators win research grants to perform research

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11
Q

Drug development has

A

3 stages prior to approval

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12
Q

STAGE 1

A

Drug discovery
Discovery ( Laboratory based)

5,000 – 10,000 compounds may be synthesized and tested
- 250 “may” make it to pre-clinical testing

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13
Q

STAGE 2

A

Pre-clinical Testing

  • Tests performed to determine if candidate molecule safe to test in humans
  • Candidates for drugs are first tested in animals
  • Very few candidate materials for drugs make it through pre-clinical testing
  • < 0.1% of potential candidate drugs go from discovery to clinical testing stage
  • Varies in number of years it takes
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14
Q

STAGE 3

A

Clinical testing has 3 DISTINCT PHASES with distinct goals
- Investigational New Drug (IND) application must be submitted to FDA
in order to begin clinical testing in humans

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15
Q

STAGE 4

A

Post approval and post clinical testing (also called Phase IV)

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16
Q

To begin clinical testing,

A

Investigational New Drug (IND) Application must be filed

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17
Q

When all clincial testing is complete,

A

The New Drug Application (NDA) must be filed

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18
Q

PHASE IV

A

addition research post-marketing by FDA

- Used to identify events not detected during clinical testing

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19
Q

IND

A

Investigational New Drug

20
Q

NDA

A

New Drug Application

21
Q

3 phases before drug approval : Clinical testing

A
  • Phase 1: Learn how the drug works in (small number) of humans – set dosage
  • Phase II: Demonstrate efficacy (proof of concept) and safety (toxicity) in patients
  • Phase III: Provide strong evidence of efficacy and safety by studying candidate drug
    in a few thousand people with target disease in randomized, controlled trials using
    doses and settings more like “real world” practice
22
Q

Phase I Clinical Testing Goals Include

A
  • Learn how candidate drug works in HEALTHY humans
     Studies of <100 healthy individuals
     Establish dose range, metabolism, excretion, toxicities
     Usually takes one year or less to complete
23
Q

Phase II Clinical Testing Goals Include

A
  • Study candidate drug in a few hundred people with target disease
  • Efficacy (proof of concept) and safety (toxicity) in patients
  • Single arm studies (No comparison group)
  • Usually takes up to two years to complete
    (occurs after Phase I testing is completed)
24
Q

Phase III Clinical Testing Goals Includ

A

Study a few thousand people with target disease in randomized, controlled trials
 Strong evidence for efficacy and safety
 Use doses and settings more like real-world practice
 Success is necessary to move to the next stage of approval
 Usually takes three to four years to complete
(occurs after Phase I and Phase II testing is completed

25
Q

Efficacy

A

Extent to which a specific intervention, procedure, regimen, or service
produces a beneficial result under ideal conditions
 “Ideal conditions” are achieved in clinical trials

26
Q

Effectiveness

A
  • Measure of success of a therapeutic intervention carried out in a typical
    environment on typical patients
  • “real-world” conditions
27
Q

Post-Clinical testing

A

NDA submitted to FDA upon successful completion of clinical testing (Stage 3)
 NDA specifies the following information about the candidate drug:
 Manufacturing
 Action in the body
 Marketing
 Labeling and toxicologic information

28
Q

Phase IV testing and evaluation

A

Post-marketing surveillance in larger samples and for longer time frame
 evaluation of safety and effectiveness not detected in earlier trials
 “real-world” settings, “real-world” patients, and very large numbers of people

29
Q

MedWatch

A
  • FDA’s safety information adverse event reporting program
     Health professionals encouraged to report adverse events in their patients
     Manufacturers are required to report adverse events to FDA
30
Q

Adverse Event Reporting System

A

Supports the FDA’s post-marketing safety surveillance program
 Contains adverse drug event reports FDA received from manufacturers required by regulation

31
Q

ANDA

A

Abbreviated New Drug Application

32
Q

Abbreviated New Drug Application (ANDA). What is ANDA

A
  • submitted to FDA for a drug already “approved” but required when requesting:

 Change from prescription to over-the-counter status
 Change in ingredients, dosage, manufacturing, formulation, or labeling

33
Q

Who submits to ANDA

A

Manufacturers of generics or biosimilars
- FDA examines the bio-equivalence, bioavailability, and
pharmacokinetic/pharmacodynamic properties

34
Q

Intellectual Property (IP) rights

A

protect innovators & provide
incentives for drug development

But: allow Pharma Companies to have a monopoly & charge higher prices

35
Q

Drug Price Competition & Patent Term Restoration Act of 1984

(Hatch Waxman Act) & PPACA

36
Q

Hatch-Waxman Act Characteristics

A
  • Applies to small molecule drugs
    Innovator data exclusivity for 5 years from FDA approval*
     Additional 3 years of data exclusivity for new indications
     Additional 6 months of exclusivity for pediatric applications
     No new clinical trials required for generics (ANDA)
     Regulation pertains to small-molecule drugs
37
Q

Hatch-Waxman Act

A

established the approval pathway for generic drug products, under which applicants can submit an abbreviated new drug application (ANDA)

38
Q

Biologic

A

are a large, new part of the Rx marketplace
- Very important uses in major diseases
 Very, VERY expensive medications

39
Q

Majority of Biologicals Use Living Cells to

Produce a Protein Product

A

Insert gene encoding the protein of interest
 Cells require proper conditions for optimal growth
(temp, pH, oxygen, feeds, etc.)
 Culture and fermentation can take weeks
 Complex Purification Steps
 Safe product with desired potency

40
Q

Small molecule drugs vs. Biological drugs

A

Biological drugs are large and have a high MW. They are complex. Produced in living cell structure. Unstable and immunogenic

Small molecule drugs are non-immunogenic, stable, produced by chemical synthesis

41
Q

BLA

A

Biologic License Application- which includes

- Manufacturing info to demonstrate company can properly and consistently manufacture the product

42
Q

The biological product is licensed and can be marketed in the US only if:

A

FDA determines that the data shows the biological product is safe and effective for it’s proposed use, and
-the biological product can be manufactured in a way that ensures a quality product

43
Q

Biologics Price Competition and Innovation Act (2009)

Abbreviated approval pathway in place under the 2010 Patient Protection and
Affordable Care Act (PPACA)

A

Allows for licensing with less than full complement of pre-clinical and clinical data
 Focus on the manufacturing process
 “highly similar” with no clinically significant differences in safety, purity, potency,
immunogenicity
 Tested against a reference product, NOT against placebo!
 “The goal is not to independently establish safety & effectiveness of proposed product

44
Q

Biosimilarity”

A

A biosimilar product is a biological product that is approved based on a showing that it is
highly similar to an FDA-approved biological product, known as a reference product, and
has no clinically meaningful differences in terms of safety and effectiveness [purity,
potency] from the reference product

Only minor differences in clinically inactive components are allowable in biosimilar
products”

45
Q

Interchangeable means

A

An interchangeable biological product may be substituted for the reference product
by a pharmacist without the intervention of the health care provider who prescribed
the reference product

-They have different brand names,
making their identification more confusing

PHRM 510 (11 decks)

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