Lec 4 – Identification of the Clinical Need (The Patient)
• High cost and time consuming drug development process means we should aim to
avoid failure mid way through this process
A way to do this is to have a plan at the start of the end result
,-Consider what needs you are addressing
-Could use a Target Product Profile (TPP) to do this
• Stakeholders in Healthcare NEEDS
-Provider
-Payers eg Government, Insurance Companies
-Pharmaceutical Industry
-Patients
So must consider more than just the patients needs to be successful
-Providers Needs – Is there benefit to patient whilst considering the ability the provider
has to give this care
-Payers Needs – Is benefit worth the cost?, consider long term of this
-Pharmaceutical Industry Needs – Is there a market?
-Patients Needs – Will it improve/cure?
Regulators also involved – buffer between drug development and patients, ensuring any
new products meet certain requirements to be deemed as ‘safe’
Their Needs – Is the intended benefits delivered to patients?
Many others involved at each stage of development process who each have needs
These include – biologists, chemists, DMPK (drug metabolism PK) department,
marketing, safety
• Target Product Profile (TPP)
-A summary of the goals of the drug development program
-Includes:
ESSENTIALS
Indications/Use (which disease, symptoms and population)
Dosage Forms and Strengths
, Dosage and Administration (consider dosing regimen and duration)
Contraindications, Warnings and Precautions
Adverse Effects
OPTIONAL
Efficacy Profile (what will your product do in terms of efficacy)
Standard of Care (competitors-so what is already out there)
Differentiation Claims (why is it better than existing treatments)
Anticipated Launch Date
Territory
• Understanding the clinical needs requires you to look at:
-The disease -clinical presentation, how its measured (any biomarkers), affected
population (ensure this is specific enough so you can prove efficacy), is there dif
groups of the disease, what is the burden, prevalence
Consider what you will address in a disease and what specifically you will target (cure
vs treatment)
-Treatments - what current therapies are available and how do they work, what are
the positives and negatives about them, from this is there a new market or any
unmet needs
If you are able to assess the need, disease burden and competitor then you can
minimise the risk of failure during drug discovery
Ensure you have right disease, for right target, and right patient
Lecture talks about COPD and ADP Kidney Disease as examples (can refer to slides to
see the process mentioned above in action)
-Population Specificity – between two subtypes of COPD – emphysema and chronic
bronchitis, have differences in symptoms, so treatment will differ in tackling these
symptoms, hence need to specify which subgroup you are targeting
-Understand disease characteristics
--- allows identification of target,
, ---and assessment if compound is suitable for development, find out via experiments
to show its efficacy -such as assay development to assess drug compounds potency,
or biomarkers to show target engagement/efficacy
-Is there a market?
How does it compare with other existing treatments
Is drug cost effective
Lec 5 - Human Factors in Product Development (The Patient, The Medicine, The
Pharmacist)
• Human Factors Def
-How humans interact with the environment surrounding them
Here are some proper definitions (DON’T MEMORISE)
• High cost and time consuming drug development process means we should aim to
avoid failure mid way through this process
A way to do this is to have a plan at the start of the end result
,-Consider what needs you are addressing
-Could use a Target Product Profile (TPP) to do this
• Stakeholders in Healthcare NEEDS
-Provider
-Payers eg Government, Insurance Companies
-Pharmaceutical Industry
-Patients
So must consider more than just the patients needs to be successful
-Providers Needs – Is there benefit to patient whilst considering the ability the provider
has to give this care
-Payers Needs – Is benefit worth the cost?, consider long term of this
-Pharmaceutical Industry Needs – Is there a market?
-Patients Needs – Will it improve/cure?
Regulators also involved – buffer between drug development and patients, ensuring any
new products meet certain requirements to be deemed as ‘safe’
Their Needs – Is the intended benefits delivered to patients?
Many others involved at each stage of development process who each have needs
These include – biologists, chemists, DMPK (drug metabolism PK) department,
marketing, safety
• Target Product Profile (TPP)
-A summary of the goals of the drug development program
-Includes:
ESSENTIALS
Indications/Use (which disease, symptoms and population)
Dosage Forms and Strengths
, Dosage and Administration (consider dosing regimen and duration)
Contraindications, Warnings and Precautions
Adverse Effects
OPTIONAL
Efficacy Profile (what will your product do in terms of efficacy)
Standard of Care (competitors-so what is already out there)
Differentiation Claims (why is it better than existing treatments)
Anticipated Launch Date
Territory
• Understanding the clinical needs requires you to look at:
-The disease -clinical presentation, how its measured (any biomarkers), affected
population (ensure this is specific enough so you can prove efficacy), is there dif
groups of the disease, what is the burden, prevalence
Consider what you will address in a disease and what specifically you will target (cure
vs treatment)
-Treatments - what current therapies are available and how do they work, what are
the positives and negatives about them, from this is there a new market or any
unmet needs
If you are able to assess the need, disease burden and competitor then you can
minimise the risk of failure during drug discovery
Ensure you have right disease, for right target, and right patient
Lecture talks about COPD and ADP Kidney Disease as examples (can refer to slides to
see the process mentioned above in action)
-Population Specificity – between two subtypes of COPD – emphysema and chronic
bronchitis, have differences in symptoms, so treatment will differ in tackling these
symptoms, hence need to specify which subgroup you are targeting
-Understand disease characteristics
--- allows identification of target,
, ---and assessment if compound is suitable for development, find out via experiments
to show its efficacy -such as assay development to assess drug compounds potency,
or biomarkers to show target engagement/efficacy
-Is there a market?
How does it compare with other existing treatments
Is drug cost effective
Lec 5 - Human Factors in Product Development (The Patient, The Medicine, The
Pharmacist)
• Human Factors Def
-How humans interact with the environment surrounding them
Here are some proper definitions (DON’T MEMORISE)
