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CPPS 304 > Drug Evaluation and Approval > Flashcards

Drug Evaluation and Approval Flashcards

1
Q

When was the Penicillin Structure described?

A

1945

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2
Q

What was synthesized based on the Penicillin structure?

A

More Beta-Lactams

Ampicillin
Carbenicillin
Methicillin

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3
Q

When was the mechanism of penicillin described?

A

1965

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4
Q

Describe the Discovery of Aspirin.

A

Willow Bark had been used as an analgesic and antipyreti for centuries - Hippocrates wrote about using willow leaves for fever. (400 BC)

Salicylic Acid - the active ingredient was isolated in the 1700s.

Were able to synthesize in the 1800s.

SA was very hard on the stomach. Topically used to burn off warts.

**Felix Hoffman (Bayer Pharmaceuticals) - Acetylated SA **to make Acetylsalicylic Acid (Aspirin) started selling in 1899.

Aspirin became the “wonder drug” intially used for Pain/Inflammation and Fever and later routinely used as an Antiplatelet (1980s).

Mechanism of Action was not descibed until the 1970s.

Antiplatelet Propertied discovered -1940s

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5
Q

Describe the Discovery of Anti-Retrovirals.

A

AIDS (Acquired Immune Defiency Syndrome) - was first identified in 1981.

100% Mortalilty Rate

Mid-1980s - Discovered how contracting HIV leads to the developement of AIDS. At this time everyone who got HIV developed AIDs.

**Very Few Antiviral drug on the market at this time. (ONLY Herpes and Influenza)
**
The fight against AIDs was considered an internation emergency and significant resources were directed to research.

In a few years, researchers determined:

HIV was a retrovirus.

Key Enzyme in its replication was a reverse Transcriptase.

Started researching drugs to inhibit Reverse Transcriptase.

AZT (Azidothymidine) was found efficacious in treating the virus. Was Previously a failed anti-cancer drug in the 1960s.

AZT Approved by 1987 (Record Time)

Resistance soon Developed but other therapies were found.

1996 - Combination Therapy made HIV very treatable.

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6
Q

What are the steps in HIV replication and what drugs target these steps?

A

Step 1: Attachment/ Entry; CCR5 Inhibitor Drugs

Step 2: Transcription; Reverse Transcriptase Inhibitors

Step 3: Integration into Genome; Interase Inhibitors

Step 4: Assembly/Maturation; Protease Inhibitors

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7
Q

What are the 2 fundamental approaches for drug discovery?

A

Compound-Centered - 20th Century

Target Centered - 21st Century

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8
Q

What is the Compound-Centered Approach for drug discovery?

A

Test promising compund for pharmacologic activity.

Without fully characterized your target.

Receptors were not characterized until the 1970s.

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9
Q

What are the 2 technologies that the Compound-Centered Approach is facilitated by?

A

Combinatorial Chemistry

High throughout screening.

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10
Q

What are all the sources of compounds in the compound centered approach?

A

Natural Products: Penicillin, Opioids

Endogeneous Compounds: Cortisol, Insulin

Re-purposing Existing Known Compounds

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11
Q

Describe the Process of Combinatorial Chemsitry?

A

Use a known compound Precursor (Opium).

Machine that allows for the generation of a large number of compounds from a small number of precursors.

Can then be screened for effectiveness.

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12
Q

Describe the process of High-Troughput Screening?

A

Automated System that allows for the rapid screening of thousands of compounds.

Assay for:

Receptor Binding

Biochemical/Cellular Target

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13
Q

What is the target-centred approach fo drug discovery characterized by?

A

Identify/Characterize the target first, then design a dig that “hits that target”.

Rational Drug Design

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14
Q

What techniques is target-centered drug discovery facilitated by?

A

Understanding the mechanisms of the disease.

Ability to full characterize receptors through protein crystallography.

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15
Q

What drug is used in immunotherapy of cancer?

A

Pembrolizumab

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16
Q

How does the Pembrolizumab and Immunotherapy work?

A

Pembrolizumab targets programmed cell death protein (PD-1)

Tumours bind to PD-1 and Turn of the T-Cell Immune response to the timour.

Pembrolizumab prevents this - T Cells remain activated to attack tumour cells.

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17
Q

How long does a patent last in Canada?

A

20 years from the Date of Issue.

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18
Q

What do you do after you discover a drug molecule?

A

Patent It

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19
Q

What is a patent?

A

A patent grants exclusivity to the holder of the patent for marketing of the product, and protects the intellectual property behind that product.

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20
Q

What is a Generic Version of a drug refer to?

A

A drug that has been copied after the patenting period.

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21
Q

What are the effects of increasing or decreasing patent protection?

A
Comsumers: Want Less Drug Corporations: Want More
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22
Q

What was done to address uncertainty with patenting?

20 Year Patent starts ticking as soon as it is issued, however what is it takes 18 years to bring said dug into the market.

A

New Rule: Allows 8 years exclusivity post-market approval.

If it took 18 years, you would still get 8 years Exclusivity of drug.

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23
Q

How can a patent be challenged in Canada?

A

If a generic manufacturer can prove that a manufacturer is not marketing their product then they can infringe upon the patent.

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24
Q

Why do drug companies want fast evaluation and approval processes?

A

The longer it takes to gain approval to sell the drug, the less time the company will have exclusive sales.

Patent Clock Starts Ticking

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25
Q

What are the 2 stages of studying/testing does health Canada do?

A
  1. Pre-Clincal (Animal Studies, Key Elements)
  2. Clinical (Approval Process Phase 1-4, Limitations)

Lots of drugs screened out in preclinical

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26
Q

What are the Key Elements of Pre-Clincal Testing?

A

Pharmacodynamics: How Selective is the Drug
Pharmacokinetics: Half-Life, Routes of Elimination

Toxicology: Therapeutic Index

Pharmaceutical Development: What dosage forms are preferred. (Tablets or Injections etc)

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27
Q

What are the Regulatory Agencies in Canada, USA and Europe?

A

Canada: Health Canada

USA: Food and Drug Administration (FDA)

Europe: European Medicines Agency (EMA)

FDA approved drugs must still be approved by Health Canada.

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28
Q

What agency is considered the most thorough and best?

A

FDA

Drug Reviews between agencies can be simultaneous.

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29
Q

What branch at Health Canada is responsible for Rx drugs and approval?

A

Therapeutic Products Directorate (TPD)

Split into 15 offices,

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30
Q

Where are clinical trials typically carried out?

A

Multiple sites internationally.

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31
Q

What is the purpose of regulatory bodies conducting site visits (inspections)?

A
  • Ensures tha proper protocols are followed.
  • Sites not following protocol may have their data excluded.
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32
Q

What is Phase 1 of Clinical Trials, what is the focus and what are the goals?

A

First Time using drug on humans.

Size: Small Trials

Trial Design: Open Labelled (No blinding, everyone sees identity of the drug) or Uncontrolled.

Person Type: Use Healthy Volunteers - Unless Patient Needs it if there is no other option.

Focus:
* Pharmacokintics
* Dosing
* Safety

Goals:
How is the drug metabolized?
What is the range of doses?

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33
Q

What is Phase 2 of Clinical Trials, what is the focus and what are the goals?

A

Size: Medium Size Trial (N = Few Hundred)

Person Type: Used in Patients fully.

Trial Design: Open Labeled, single(Physician knows identity of drug), or double blind (No-one knows identity).

Focus:
* Efficacy
* Safety
* Pharmacokinetics

34
Q

What is Phase 3 of Clinical Trials, what is the focus and what are the goals?

A

Size: large N = several hundred to thousands.

Person Type: Patients

Trial Design: Double Blind

**Focus: **
* Efficacy and Safety

Goals:
* Is the drug more efficacious than placebo?
* Is the drug as safe as placebo?

35
Q

What happens after you pass Phase 3 clinical trials?

A

Recieve a Notice of Compliance (NOC) from Health Canada.

36
Q

What is a Notice of Compliance?

A

Notice of Compliance means that the manufacturer has complied with all the requirements set forth by Health Canada in the Food and Drugs Regulations.

They are Now free to market their drug in Canada.

37
Q

What is a Notice of Compliance with Conditions (NOC/c)?

A

A notice of compliance with conditions is reserved for certain drugs that show promise for life threatening conditions. Allowed to fast track trial stage to get drugs on the market.

Manufacturers are allowed to forego larger confirmatory trials in the approval process but are required to complete at a later date.

The trials that need to be completed at a later date are the conditions.

38
Q

What is the official source of information on a drug?

A

The Product Monograph

39
Q

What are the three sections of the Product Monograph?

A
  • Health Professional Information
  • Scientific Information
  • Consumer Information
40
Q

What information does the Health Profession Info contain in the PM?

A
  • Indications
  • Safety: Contraindications warnings, adverse reactions, drug interactions
  • Dosing
  • Pharmacology: Pharmacokinetics, Pharmacodynamics
41
Q

What is the most important piece of information in the PM?

A

Indications

Described the condition in which the drug is approved for use.

Drugs can have multiple indications - Supported by evidence in trials.

42
Q

What is an example of a lipid lowering drug which the PM describes can be used to treat Hypercholesterolemia, clinical atherosclerotic cardiocascular disease, and lowering of low density lipoprotein cholesterol?

A

Evolocumab

43
Q

What is referred to as the “label” for a drug?

A

The PM, Specifically Indications/Health Professional

44
Q

What is it called when a drug is used in a condition in which it is not indicated?

A

Off Label Use

When a drug is used off-label, there is higher risk for the physician if something goes wrong.

45
Q

What is contained in the Scientific Information section of the product Monograph?

A
  • Pharmaceutics: Chemical Name and Properties
  • Clinical Trials
  • Detailed Pharmacology: Includes Pre-Clinical Data
  • Toxicology: Includes Pre-Clincal Data
46
Q

What are some examples of drug withdrawals?

A
Even in well established drug classes.
47
Q

What is phase 4 of trials?

A
  • Post Marketing Surveillance
  • Much larger sample, but not usually a controlled trial.
  • Tests Safety and Requirement
  • This is not a requirement, and usually poor;y done.
48
Q

What are problems with the current drug approval system?

A
  • Clincal Trials + Rx drugs are Very expensive
  • Trials not large enough to uncover rare serious side effects
  • Trials are not long enough to uncover long term toxicity issues.
  • Long approval process of therapies are harmful to patients relying on fast drug access.
49
Q

What is one way to try and combat some of the problems with the system?

A

Repurposing

If done with a drug that is currently approved for another indication, at least Phase 1 can be skipped.

50
Q

Whats is repurposing of a drug?

A

Occurs when a drug already approved previously is re-purposed for a new indication.

51
Q

What are some examples of repurposed drugs?

A
  • AZT
  • Sildenaful (Viagra)
52
Q

How did companies use repurposing in the case of Metformin?

A

Companies “mined” data fom clinical trials and registried of Metformin in Type 2 diabetes.

Found that patients on Metformin had a lower risk of developing cancer.

53
Q

What is is roadblock for repurposing drugs?

A

Drugs usually no longer on Patent.

Required trials for new indication cost alot of money.

Not worthwhile to the company if drug is off patent.

Note: A patent can be taken out for a new formulation of an existing drug.

54
Q

What was Sildenafil (Viagra) originally made for?

A

An antihypertensive

55
Q

What does a formulary mean?

A

List of drugs

Practical Case - List of drugs in relation to a drug plan.

In SK the Drug Formulary = list of drugs that are covered.

56
Q

What is the maximum charge in SK to fill a prescription?

A

25$ for 14&under and 65orolder

57
Q

If a drug is not in the ____ it is generally not ____.

A

If a drug is not in the Formulary it is generally not Covered.

58
Q

What case is there additonal coverage?

A

Low income families / High drug cost relative to income.

59
Q

What it third party coverage?

A

Private Insurers - Covers Drug on top of Provincial

List of Drugs in their formularies that are covered - closesly reflect provincial ones.

Small Detuctable - Coverage = 80 to 100 %

60
Q

What are other examples of drug plans?

A
  • Non Insured Health Benefits
  • Indigenous Population
  • Veteran’s Affairs
  • Workman’s Compensation Board
61
Q

How are decisions made as to which drugs will be covered?

A

The decision lies with each drug plan
- Guided by the CDA Formulary Review Process (Reimbursement Reviews)

62
Q

What is the CDA and what is the goal?

A

Canada Drug Agency

Independent Non Profit Organizaiton founded in 1989.

Goal: To facilitate the appropriate and effective utilization of health technologies within health care systems across Canada.

63
Q

Prior to the CDA process how did the reimbursement reviews take place?

A

Each province had its own public drug plan and seperate review process.

64
Q

What were reasons why First Ministers intiated the need for a single review and listing process?

A
  • A consistent Approach
  • Reduced Duplicaiton
  • Maximized use of limited resources and expertise
  • Equal Access to evidence and Advice
65
Q

What is the reimbursment review process?

A
Note: CDA cares about Economics unlike Health Canada
66
Q

Who are all on a review team?

A
  • Two clincal reviewers
  • Two Economic Reviewers
  • Informatics Specialist

Clinical Expert = Top in the Field, Opinion Leader

67
Q

How is a systematic Review of Literature conducted?

A
  1. Develope Review Protocol
  2. Select Studies
68
Q

What is a Protocol defined by?

A
  • Population - Interested In
  • INtervention - Your Drugs
  • Comparator - Drugs comparing it to
  • Outcome
  • Study - Type of Study

PICOS

69
Q

What types of Studies are selected?

A
  • Publish and Unpublished
  • Required to disclose All studies.
70
Q

What is pharmacoeconomics and what is it used for?

A

**Method for comparing costs, risks, and benefits or different therapies. **

Used to: Determine the most cost-effective therapies.

71
Q

How is cost effectiveness evaluated?

A

Using clinical Outcomes and Data, expressed QALY’s (Quality of Life)

72
Q

What are the different costs that economists look at?

A
73
Q

How long are reviews completed in?

A

45 Days - Both clinical and economic review.

Review sent back to manufacturer for their comment - May be edited based on comments.

74
Q

Where is a Final version of a review sent after its completed?

A

CDEC Meeting

Monthly Meeting

75
Q

What is CDEC?

A

Canadian Drug Expert Committee

Bunch of Expert People

76
Q

What does the CDEC do?

A

Look at Review, Vote on if should be covered or not.

Recommendations sent to the Provincial Drug Plans.

77
Q

How aften to drug plans take CDEC recommendations?

A

93%

78
Q

What is a unique feature of the CDA Formulary Review Process compared to Health Canada and FDA?

A

Transparency

79
Q

What are the components of the CDEC Recommendation?

A
  • Brief Description of Drug
  • Recommendation and Reasons’
  • Consideration
  • Includes a summary of trial data.
80
Q
A

CPPS 304 (10 decks)

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