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BPH > C1: Where do Drugs and Medicines come from ? > Flashcards

C1: Where do Drugs and Medicines come from ? Flashcards

1
Q

What is the components of QA + what do they control

A
  • GLP - Good Laboratory Practice (Labs and
    Animal tests)
  • GCP - Good Clinical Practice (Human Clinical
    trials)
  • GMP- Good Manufacturing Practice
    (Production)
    — Quality control
  • GPvP- Good Pharmcovigilance Practice (After
    launch)
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2
Q

What is quality by design

A

A systematic approach to development that
begins with predefined objectives and
emphasizes product and process
understanding and process control, based on
sound science and quality risk management

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3
Q

Good clinical practise

A

international ethical and scientific
quality standard for designing, conducting,
recording & reporting trials that involve
participation of human subjects

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4
Q

Good laboratory practise

A

Good laboratory practice embodies a set of
principles that provide a framework within which
laboratory studies are planned, performed ,
monitored, recorded, reported and archived

Assures that data submitted to regulatory authorities
is a true reflection of results obtained during a study
& can be relied upon when making risk/safety
assessments

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5
Q

Good manufacturing practise

A

The part of quality assurance which is aimed at ensuring
that products are consistently manufactured to a quality
appropriate to their intended use

Quality is fitness for purpose

  • Right product
  • Right strength
  • Free from contamination
  • Correct container
  • Correct label
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6
Q

Why Do We Need GMP

A
  • Pharmaceuticals are the only product specifically
    designed to be given to people who are already ill!
  • Pharmaceuticals are potent substances which can
    improve or maintain life
  • Impossible to judge the quality of pharmaceuticals
    visually
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7
Q

What aspects does GMP control

A
  • Personnel
  • Premises and Equipment
  • Documentation
  • Quality Control
  • Complaints and Product Recall
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8
Q

Personnel

A

Key personnel

  • Head of Production
  • Head of Quality Control
  • Qualified Person

Head of Production and Head of Quality Control must be
independent of each other

Qualified person (EU only)-legal and personal liability to
ensure that the principles and guidelines of GMP have been followed
- Training required for all personnel
- Personal hygiene standards must be maintained
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9
Q

Premises

A
  • Minimise the risk of cross contamination
  • Maintenance procedures must be in place
  • The environment in the facility should be controlled to ensure that it does not affect product quality
  • Adequate pest control procedures should be in place
  • Access to the production environment should be controlled
  • The design of production areas should facilitate cleaning
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10
Q

Equipment

A
  • Manufacturing equipment should be designed, located and maintained to suit its intended purpose
  • Equipment should be designed so that it is easily cleaned
  • Effectiveness of cleaning procedures must be demonstrated
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11
Q

General Documentation Principles

A
  • Documentation provides enhanced process control and enables batch history to be traced
  • Records should be made at the time the action is taken
  • Records should be maintained for a year after the end of the product shelf-life
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12
Q

Documentation Used in GMP

A
  • Standard operating procedures (SOPs )- how to carry out procedures
  • Batch records -Provide a detailed history of a product
    batch
  • Labelling (materials, products, equipment, rooms)
  • Log books for equipment and rooms
  • Test methods: there should be written procedures for
    testing materials and products
  • Specifications- physical, chemical or microbiological
    criteria that the material/product must meet
  • Problem investigations-Initiated following an unusual or
    unwanted occurrence.
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13
Q

Complaints + product recall

A
  • Written procedures should be available detailing the action to be taken in the event of complaint
  • Complaint records should be reviewed regularly to identify trends and recurring problems
  • Complaints relating to serious quality problems should be notified to the relevant regulatory authorities
  • It should be possible to initiate recall procedures swiftly
  • Relevant regulatory authorities (MHRA in the UK) must be informed of any product recall.
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14
Q

Complaints + product recall

A
  • Written procedures should be available detailing the action to be taken in the event of complaint
  • Complaint records should be reviewed regularly to identify trends and recurring problems
  • Complaints relating to serious quality problems should be notified to the relevant regulatory authorities
  • It should be possible to initiate recall procedures swiftly
  • Relevant regulatory authorities (MHRA in the UK) must be informed of any product recall.
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15
Q

What are the origins of the pharmaceutical industry?

A

Pharmacy, Chemistry + Microbiology

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16
Q

When was the ‘golden age’ of drug discovery?

A

1940s-1960s is regarded as the “Golden Age”
- wide range of diseases came under control
“miracle cures” – with spectacular benefits to patients

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17
Q

Why may we be entering a new ‘golden age’ of drug discovery?

A

In this century our new ability to develop ‘biologics’

promises another Golden age

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18
Q

What are Biologics

A 
Analogues, extracts, or modified versions of active biological
compounds
- Antibodies
- Proteins
- Nucleic acids
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19
Q

How come the golden age success did not work

A

The same formula for success was used in the 1970s-80s but huge commitment to R&D intensive marketing and promotion

20
Q

What problems are faced by the pharmaceutical industry?

A

1) Tighter Government regulatory controls on development and marketing
2) Governments want to reduce costs of healthcare = - Cheaper ‘ Generics’ now favoured: reduced profits for industry
3) Success of companies depends on their innovation (But other companies may develop similar drugs, which dilutes
any new market)
4) R&D commitment is out of balance with cash flow (It costs $ 100s of millions to bring a new drug
to market; Companies must therefore develop ‘blockbuster’ drugs)
5) Prolonged development time for a new product means a reduced time (for sales and profits) when drug is under patent
—- Patent life is only 20 years, and you may spend 10 years testing, developing and getting approval for the product.
6) Earlier “Random synthesis” approach to finding drugs is now too hit and miss. (To find a new drug you have to be far more sophisticated; Companies must adopt a rational drug design)

21
Q

Major sources of new drugs

A

(A) Chemical synthesis.
(B) Old compounds with a new use.
(C) ‘Natural Products’ Natural molecules with drug activity
from microorganisms, plants, animals and human sources.
(D) New drugs (chemicals or biological molecules)
synthesized using a knowledge of the proposed site of
action. This is called rational or targeted drug design
and is a more effective strategy than random synthesis.
Computer-based molecular models of
drugs/receptors/proteins used.

22
Q

Natural product source

A

Drugs from plants, marine organisms,
bacteria, fungi, animals, human origin.
- 60% current drugs originate plant/microbial sources
- Only a fraction of Earth’s living species have been tested for activity
- Current effort : secondary metabolites
as sources of new drugs.

23
Q

Targeted drug-design & synthesis source

A

Based on current knowledge of cellular/biochemical
mechanisms & receptor sites :
- Most targets are proteins especially G-protein coupled
receptors (GPCRs) & protein kinases.
- Computer/theoretical models are used to predict activity of new molecules
—-QSAR - Quantitative Structure-Activity Relationships
—-CADD - Computer-Aided Drug-Design
- Chemical modifications are used to improve potency,
bioavailability, duration of action and reduce side-effects

24
Q

How is a lead compound identified ?

A
  • Chemical Libraries & computer design
  • New biological targets / medical conditions
  • Advances in biological knowledge
  • Human Genome Project ( completed 2003)
  • Biological agents as drugs (‘Biologics’)
  • Automated Screening Methods
25
Q

What are the key problem issues in drug discovery ?

A

Efficiency and speed because of :
• High failure rate
over 80% of new drug candidates fail to make it to the
commercial market.
• Other companies
may be working on competitor new drugs.
• Patent protection
only lasts 20 years from the discovery of the drug.
After this time, other companies can make and market
‘generic’ medicines containing the same drug.

26
Q

How do we take a lead compound into the development phase

A
  • Multiple screens must show drug activity in the therapeutic areas we are looking for.
  • Once potential drug activity is identified,
    further screens are then performed
  • Optimization of molecular structure to produce a new drug candidate to take into the development phase
    as a ‘lead compound’
27
Q

What is Medicine Development

A

Includes all the activities needed to turn a drug candidate into a medicine

28
Q

What are the major steps of drug development

A
  1. 1 Chemical process development
  2. 2 Absorption, Distribution, Metabolism, Excretion (ADME)
  3. 3 Toxicology
  4. 4 Pharmaceutics & stability testing
  5. 5 Clinical evaluation
  6. 6 Approval and marketing authorisation
29
Q

What is chemical process development

A

Design of a chemical process that allows manufacture of bulk quantities of high quality drug
• Cost effective
• Environmentally friendly
• Scalable to large quantities

A high spec process is required by regulatory authorities
because we need :
– Low levels of impurities (>99%)
– Analytical methods which are sensitive and validated
– Good Manufacturing Practice (GMP)

30
Q

What is ADME

A

To understand how the new drug is handled
by the body. This is undertaken in animal and human (Phase
1) studies.
- Absorption + bioavailability
-Distribution
- Metabolism
- Excretion

31
Q

What is Toxicology

A

Cell culture, bacterial and animal testing = To determine if new drug has toxicity problems; To estimate a dose which achieves maximum effect with minimum toxicity.

In vitro ‘ tests = Bacterial mutagenesis tests (Ames test) are performed at an
early stage. New drug is discarded if any results are equivocal or
fail; Animal and human cell cultures.

Animal testing

  • Acute toxicity Single doses, short-time (weeks)
  • Chronic toxicity 6-12 months of continuous dosing
  • Carcinogenicity 18-24 months
  • Reproductive toxicity (teratogenicity) 18-24 months
32
Q

What is Pharmaceutics & stability testing

A

Pharmaceutics = design of dosage forms (medicines)

Need to optimise the candidate drug compound into a medicine for clinical trials + product/pack market

It undergoes Preformulation, Formulation + Stability testing

33
Q

Describe phase II clinical trials

A

Clinical investigation of therapeutic effect
- About 1 to 2 years
To establish:
- a suitable dose and dose frequency in patients
(may be different to healthy people used in phase I)
- efficacy of the drug in the human disease
- duration of effect
- common side effects/risks
• 100 - 400 patients with the disease
• Dose ranging studies: different doses and dose
frequencies are given
• The study design may be ‘blind’, ‘cross-over’, ‘doubleblind’ to avoid bias
~ 33% new drugs pass Phase II trials

34
Q

Describe phase III clinical trials

A

– Many studies over 3 or more years, thousands of patients in many hospitals.

To demonstrate :
- Better efficacy or benefit-to-risk than
placebo and a competitor’s product
- tough!
- Short and long term safety
- Randomised, double blind, placebo-controlled trials to avoid patient and physician bias.
- Drug related adverse effects, drug interactions

  • If results show a statistically significant improvement on current treatment, then the company will proceed to submitting a drug/medicine dossier to MHRA or FDA for marketing approval.
    ~~25% make it through Phase III
35
Q

Describe phase IV clinical trials

What is the alternate name

A 
Post-marketing surveillance
or ‘Pharmacovigilance’
- Monitoring the use of new medicine
• Thousands / millions of patients.
• Accumulation of data on long-term efficacy, toxicity, side effects etc. Medicine/drug can be withdrawn of significant adverse effects emerge.
• UK ‘Yellow card' reports. 
• How does new drug compare with others?
• Has evidence emerged that drug is useful in other medical conditions?
36
Q

What is regulatory approval and why is it necessary

A

A ‘dossier’ of all evidence is submitted to the ‘Regulatory body’ of all countries you wanna market the pharmaceutical product in.

These are government agencies that regulate and approve medicines.
Successful ‘regulatory approval’ allows registration/ licensing in that country allowing use, sale or prescribing of the drug/medicine.

In the UK, regulatory approval by the MHRA leads to the granting of a Marketing authorisation MA

37
Q

Patenting - key features

A

Protects your exclusive right to manufacture and sale.

  • take 20 years from discovery of the drug
  • 12 years can be taken up in development

The drug, chemical process + method of use can be patented

38
Q

Marketing - Key features

A

Promotion of the new product is essential to receive a return on the development costs and generate profit.

  • To maximise your market share by establishing awareness of new drug in medical + scientific community

Strategy = planned in advance of regulatory acceptance
– size of the existing & potential market
– clinical profile of the drug
– competitor’s drugs

39
Q

What is the role of the MHRA?

A

The UK ‘regulatory body’: the government agency responsible for licensing new medicines for use in the UK.

  • Ensures the safety, quality and efficacy of medicines and medical devices to the public.
  • All new medicines must be approved by MHRA and issued with a ‘marketing authorisation’(MA) before they can be prescribed or sold
  • MHRA approval always states the medical condition the medicine has been approved for use.
  • Enforces medicines legislation and the terms of the MA by inspecting manufacturing facilities.
  • Monitors GLP, GMP, GCP and GPvP
  • Ensures patient information leaflets, advertising etc meet the required standards.
  • Monitors medicines throughout their lifetime from pharmacovigilance data such as the ‘yellow card’ scheme.
  • It works with the police and home office to detect, close down and prosecute illegal factories making counterfeits and unlicensed drugs.
40
Q

What is the role of NICE?

A

National Institute for Health and Clinical
Excellence (NICE).
NICE is a department of the UK National Health Service (NHS).
Approves medicines for use in the NHS, and issues national guidance to promote good health and treatment and prevention of illness.
Decisions and advice are evidence based.
- New medicines must be approved by NICE before they can be
used/prescribed on the NHS
- Health Technologies = NICE issues evidence-based guidance on
use of new and existing treatments.
- Clinical Practice – NICE issues evidence-based guidance on appropriate treatment and care of people with conditions within NHS. Includes surgical procedures.
- Public Health = NICE issues evidence-based guidance to NHS, local authorities and public sector workers on public health.

41
Q

Preformulation (Pharmaceutics + stability testing)

A

– Tests to understand physicochemical & material properties of the new drug
– e.g. solubility/salts/crystal forms
– Stability of drug : chemical and physical
– Compatibility with excipients

42
Q

Formulation (Pharmaceutics + stability testing)

A

– The design and optimisation of new medicines
– Oral administration (tablets/liquid) is preferred but other routes used if oral unsuitable.
– Formulations for early clinical trials are kept simple, just solutions and capsules.
– Later trials will use the proposed commercial product

43
Q

Stability testing (Pharmaceutics + stability testing)

A

– Storage of medicine and pack at different temperatures and humidities
– How rapidly does it degrade?

44
Q

Quality assurance

A

Concept which covers all matters which influence the quality of the medicinal product and ensures that it is suitable for its intended use
- Ensures that the patients are not at risk due to inadequate safety, efficacy or quality
- Applied throughout development process and to
commercial manufacturing process

45
Q

Describe Phase I clinical trials

A

Clinical Pharmacology and Toxicology trials
– To determine ADME, pharmacokinetics, bioavailability
– Dose related side effects
– 50-200 healthy volunteers – don’t have disease
– exclude children, women of reproductive age, elderly.
– single doses/ rising doses of drug are given
– to determine the highest tolerated dose,
– the earliest ‘first in man’ studies often termed Phase 0 ~~70% potential new drugs pass phase I

46
Q

Quality control

A

Analysis of raw materials and finished product. Test data

must fall within defined specification limits

BPH (7 decks)

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