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pharmacology > Drug Development in the UK L8 > Flashcards

Drug Development in the UK L8 Flashcards

1
Q

what is the first stage of drug development

A

drug discovery

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2
Q

what are the two ways targets for drugs are identified

A
  1. target bases
    - look for proteins that are liked to certain diseases
  2. phenotypic
    - testing drug on tissue/ cell/ organ to see it phenotypical effects and to see if they are what you want
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3
Q

what happens after target is identifed

A
  1. lead identification
  2. lead modification
    - does lead molecule need to be modified to be even more effective
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4
Q

what methods are used to measure protein interaction on drug

A
  1. HTS – High Throughput Screening
    Global protein profiling
    Protein-protein interaction profiling
  2. SAR - Structure Activity Relationships Predicting biological activity from molecular structure.
  3. Chemoproteomics
    Selectivity and/or drug affinity profiling
    - important to measure protein interaction as if our drugs interact with other proteins, can stop the drug from binding to target
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5
Q

what is the second stage of drug development

A

preclinical

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6
Q

what are the main things researched during preclinical

A

toxological effects

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7
Q

what is GLP and what is its purpose

A

Good Laboratory Practice
Quality control measures for uniformity, consistency, reliability, reproducibility, and integrityin pre clinical studies
they cover range of drugs not just for humans

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8
Q

give examples of GPL tests

A
  • physical-chemical testing
  • toxicity studies
  • mutagenicity studies
  • environmental toxicity studies on aquatic and terrestrial organisms
  • studies on behaviour in water, soil and air; bioaccumulation
  • studies to determine pesticide residues in food or animal feedstuffs
  • studies on effects on mesocosms and natural ecosystems
  • analytical and clinical chemistry testing
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9
Q

what does The FDA Modernization Act 2.0 say

A

some drugs do not needed to be tested on animals before humans
- up to researchers to decide

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10
Q

what is ICH and what is its purpose

A

An international harmonised approach (ICH) to ensure consistency in the evaluation, approval and use of new therapeutic substances.
they Produce Technical Guidelines for Regulatory Authorities to implement which focus on safety, efficacy and quality

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11
Q

what are the technical guidelines

A

Cover specific types of molecule
Small molecular weight chemicals
Biologics
Gene therapy products etc

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12
Q

what does safety, efficacy and quality mean

A

Safety
- characterise and integrate pharmacology and toxicology activity to justify first in human studies and to estimate the maximum safe starting dose.
- provide ongoing assessment of safety to support longer term clinical trials and ultimately marketing authorisation
Efficacy
- characterise and integrate non clinical and human pharmacodynamics to provide an assessment of overall therapeutic efficacy
Quality
- ensure that drug substance is synthesised in a consistent and controlled way to optimise drug product quality characteristics

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13
Q

what are the two regulation bodies in UK and what do they assess

A
  1. MHRA (medicines and healthcare product regulation agency)
  2. European medicines agency
    - Positive pre-clinical data can be submitted for a clinical trial authorisation (UK).
    Allows FIM studies to commence based on supplied data.
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14
Q

what is MHRA split into and what do they do

A
  1. MHRA
    - Regulation of medicines and medical devices
  2. NIBSC
    - national institute for biological standards and control
    - standardisation and control of biological medicines
  3. CPRD
    - clinical practice research data link
    - anonymise NHS primary care data for observational research purposes
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15
Q

what are the stages to Drug marketing authorisation – drug license by MHRA

A

part 1: identification of product
-Administrative data incl; summary of product characteristics (SPC) and expert reports
part 2: product manafacture
- Composition and method of production
- Control of starting materials, control tests on intermediate/final products
- Stability tests
- Bioavailability/bioequivalence
part 3: pre-clinical data
- GLP / Regulatory toxicology
part 4: clinical results
- suggest trial protocol

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16
Q

what is the 3rd stage to drug development

A

clinical trails

17
Q

what is a therapeutic trial

A

A carefully, and ethically, designed experiment with the aim of answering some precisely framed questions.
In its most rigorous form it demands equivalent groups of patients concurrently treated in different ways or in randomised sequential order in cross over designs.
- doesn’t always need large amounts of patients

18
Q

what are the clinical trials categories
- what question are they trying to answer

A
  1. treatment
  2. prevention
  3. diagnosis
  4. screening
  5. quality of life
19
Q

what are the clinical trials design type

A
  1. randomised control studies (blind or unblind)
  2. non-randomised control studies (unblind)
  3. Historical control studies (unblinded)- everyone gets it and compare to previous control people
  4. Cross over trial design- each patient is own control
  5. Factorial design- measure 2 things at once
  6. Hybrid design
20
Q

what factors affect the reliability of a trial

A
  1. bias
    - placebo
  2. controls
  3. blind
    - single, double, dummy
21
Q

what is the placebo effect

A

A psychological response to a treatment that has no intrinsic drug activity.
- All trials subject to possible psychological impact on therapeutic outcomes.
- Physiological conditions do not change with a placebo.

22
Q

what factors effect the outcome of a trial

A
  1. demographics
  2. ethics
  3. randomisation
23
Q

what do clinical trials measure

A
  1. clinical measures
    - effects on different organs
  2. PK measures
    - C max, T max, t1/2 , AUC (0-t)
    - Bioavailability, Clearance (Cl), Elimination (k), Disposition (Vd).
  3. PD measures
    - Target affinity (receptor occupancy), dose-response.
    - Biomarkers, clinical measures (cognitive function etc).
  4. safety measures
    - Extent of exposure (definition of dosing regime)
    - Common adverse (serious) events
    - Common laboratory tests (Liver and Renal function)
24
Q

how many different trials are there

A

4

25
Q

what are phase 1 trials

A

these are first human trials
Aims to establish:
The PK/PD properties of the drug in humans
whether drug gets to the tissue of interest
modulates a molecular target or cognate biochemical pathway
produces the anticipated downstream biological

The toxicological properties of the drug in humans
establishment of the maximally tolerated dose (MTD)

The appropriate route and frequency of administration of the drug to humans (drug dose and schedule).

Emphasis remains on safety

Average duration 1 year

26
Q

what are phase 2 trials

A

exploratory/confirmatory efficacy (large scale) dose range finding
Aims to establish:
To characterise and integrate non clinical and human pharmacodynamics to provide an assessment of overall therapeutic efficacy (ICH guidelines)
First evidence of clinical effectiveness in humans

Additional safety information
Pharmacodynamic dose-ranging

Emphasis on efficacy and safety.

Design influenced by phase I results

27
Q

during phase 2 trials, therapeutic efficacy is examined
what does this involve

A

Involves participants with target disease – 100’s patients
Can also include refractory patients.

Uses target route of administration.

Measures
Confirm primary pharmacology through POP studies
Validation of clinical endpoints or surrogates
Drug interactions
Effectiveness in intended patient population under defined conditions of usage
Human variability (gender, ethnicity etc)

28
Q

what are the outcomes of phase 2

A
  1. Evidence of efficacy or in-efficacy in target condition
     Extension studies determine possible kinetic/dynamic causes.
  2. Decreased safety profile
     Re-assessment of candidate risk:benefit ratio
  3. Possible dosing regime information/adjustments
  4. Positive safety and efficacy data will allow drug candidate to progress to
29
Q

what are phase 3 trials

A

pivotal efficacy and safety
Aims to establish:
The potential role of the new drug in routine clinical practice.
Efficacy on a substantial scale
Safety
Comparison to other therapeutic alternatives

Emphasis on efficacy

1000’s + participants with target condition.

More complex study design, documentation and personnel requirements.
Strict entry and exclusion criteria.

Will dictate whether a drug gains approval for general medical use.

30
Q

what are phase 4 trials

A

pharmacovigilance and long-term safety
Post-licensing studies
To investigate new indications for drug

In specific patient sub-groups

To investigate safety concerns

31
Q

what is a negative of drug development

A

high attrition
100s of drugs start off but only very few meet all the regulations

32
Q

who are NICE and what do they do

A

national institute for health and care excellence
provides national guidance and advice to improve health and social care
Utilise health economic techniques to support health policy decisions

33
Q

what do NICE produce

A

Produce
Technology appraisals – use of new and existing medicines/treatments
Clinical Guidelines - specific diseases/conditions

pharmacology (13 decks)

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