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John's lectures > Clinical development 1 > Flashcards

Clinical development 1 Flashcards

1
Q

What do you get after a successful clinical development programme?

A

A licence - that has regulatory approval
A summary product characterisation - (SmpC)
EMA and MHRA approval

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2
Q

What are the requirements to obtain a licence approval of a new drug?

A

Efficacy
Safety
Quality - contents of formulation including excipients/ manufacturing routes and stability

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3
Q

Describe what efficacy is? and list what it determines

A

It shows that the drug works to treat the intended disease / It determines which patients should be given the drug/ the range of the dose/ should everyone be given the drug

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4
Q

How do you demonstrate efficacy?

A
  1. Clinical trials in relevant patient population
  2. The phamacological dose response curve to determine the range of dose
  3. include a placebo comparator
  4. Using relevant end points
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5
Q

What are endpoints?

A

They are what clinical trails are designed to measure
They need to relate to the disease being studied
They need to change in response to the drug being tested
A change should predict a beneficial effect on the disease being studied

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6
Q

What are endpoints used to make decisions on?

A
  1. Prescribing decisions by doctors
  2. Licence approval or rejections
  3. Investment decisions by pharma companies
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7
Q

What do end points need to be?

A
  1. Biologically plausible
  2. Robust
  3. Objective
  4. Validated
  5. Reproducible between individuals
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8
Q

How can end points be measured?

A

Using biomarkers

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9
Q

What are biomarkers?

A

They are a characteristics that are measured and evaluated as an indicator an indicator of normal biological process, pharmacological process.

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10
Q

What are the pros and cons of biomarkers?

A

Pros - easy to measure/rapid change in response to intervention / much smaller and cheaper trials
Cons - Change in biomarkers linking to clinical benefits to patient is not proven

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11
Q

What is a surogate end point?

A

a biomarker that is intended to substitute for a clinical endpoint/ it is expected to predict clinical benefits

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12
Q

What are the pros and cons for surogate endpoint?

A

Pros - Generally accepted or predictive use/ Change over shorter time than clinical endpoints
cons - If it shows safety concerns then may not be sufficient

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13
Q

What are clinical endpoints?

A

A biomarker that is a direct measure of how patients feels

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14
Q

What are the pros and cons of clinical endpoints?

A

Pros - Definitive

Cons - Takes a long time to collect/ trials are big and expensive

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15
Q

What is end point 3/ the final endpoint?

A

Death/ pain/ quality of life/ Bp

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16
Q

What is safety?

A

It is essential to maintain the integrity of volunteers and patient in trials taking medicine/ it can never be proven

17
Q

No agent with a pharmacological effect will ever be safe. TRUE OR FALSE?

A

TRUE

18
Q

Safety is the most important and most controlled part of the clinical trial/ pharamcovigilance. TRUE OR FALSE?

A

TRUE

19
Q

What are adverse event?

A

A change in the subject related to clinical trial that is not part of the efficacy measurements

20
Q

What is an adverse reaction?

A

An adverse event that is considered potentially related to an experimental therapy

21
Q

What is included in a summary product characterization?

A
  1. Maximum dose of drug that can be used
  2. Who should not be given the drug or at reduced dose
  3. Are there any interactions with other drugs
  4. A description of most important and frequent expected adverse effects and their likely frequency
  5. Are there any routine follow ups and monitoring e.g blood tests of liver function that patients should have
22
Q

Everything done in clinical trials is to not compromise the safety of patients. TRUE OR FALSE?

A

TRUE

23
Q

Doctors must factor in that what is included in a SmpC is based on clinical trials within a few patients and not the entire population. TRUE OR FALSE?

A

TRUE

24
Q

What are the possible reasons for the decline in drug development?

A
  1. The bar of approval by regulatory affairs is getting higher
  2. The science is getting harder/ most of simple drugs receptors have already been found
  3. The world is more expensive /site investigation and end point measurements
  4. Market access - Getting regulatory approval and SmpC does not get you on commercial basis for sales
25
Q

What are the competitive advantages that a drug needs to have in order to be highly commercialized?

A
  1. Comparative clinical trials
  2. Cost effectiveness or utility (NCE)
  3. Lower dose
  4. Easier to take
  5. More patients can use the drug
  6. Fewer drug to drug interactions
26
Q

What is target product profile?

A

Before starting you should know where your heading, this can be revisited over the period of the clinical trial development

27
Q

Who produces and owns the target product profile?

A

Produced and owned by product team , regulatory affairs and manufacturing specialists

28
Q

What is included in a target product profile?

A

Mechanism of action - What pharmacological effect does the NCE have and how should that relate to efficacy in the disease?

Indication - Not just which disease, but which patients and subset of patients with the disease – e.g. early disease or late, out-patients or in-patients or in intensive care

Efficacy - What is the key efficacy endpoint for: licensing approval & reimbursement access and how much of a change in that endpoint is needed?

Tolerability - How much risk is acceptable. Are there any safety risks that limit current therapies that should be avoided?

Administration - How is the drug to be given and how often?
Are there key drug:drug interactions to be avoided

Cost -Cost-effective (small molecule, not biologics pricing

John's lectures (2 decks)

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