Clinical development 1 Flashcards
(28 cards)
What do you get after a successful clinical development programme?
A licence - that has regulatory approval
A summary product characterisation - (SmpC)
EMA and MHRA approval
What are the requirements to obtain a licence approval of a new drug?
Efficacy
Safety
Quality - contents of formulation including excipients/ manufacturing routes and stability
Describe what efficacy is? and list what it determines
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
How do you demonstrate efficacy?
- Clinical trials in relevant patient population
- The phamacological dose response curve to determine the range of dose
- include a placebo comparator
- Using relevant end points
What are endpoints?
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
What are endpoints used to make decisions on?
- Prescribing decisions by doctors
- Licence approval or rejections
- Investment decisions by pharma companies
What do end points need to be?
- Biologically plausible
- Robust
- Objective
- Validated
- Reproducible between individuals
How can end points be measured?
Using biomarkers
What are biomarkers?
They are a characteristics that are measured and evaluated as an indicator an indicator of normal biological process, pharmacological process.
What are the pros and cons of biomarkers?
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
What is a surogate end point?
a biomarker that is intended to substitute for a clinical endpoint/ it is expected to predict clinical benefits
What are the pros and cons for surogate endpoint?
Pros - Generally accepted or predictive use/ Change over shorter time than clinical endpoints
cons - If it shows safety concerns then may not be sufficient
What are clinical endpoints?
A biomarker that is a direct measure of how patients feels
What are the pros and cons of clinical endpoints?
Pros - Definitive
Cons - Takes a long time to collect/ trials are big and expensive
What is end point 3/ the final endpoint?
Death/ pain/ quality of life/ Bp
What is safety?
It is essential to maintain the integrity of volunteers and patient in trials taking medicine/ it can never be proven
No agent with a pharmacological effect will ever be safe. TRUE OR FALSE?
TRUE
Safety is the most important and most controlled part of the clinical trial/ pharamcovigilance. TRUE OR FALSE?
TRUE
What are adverse event?
A change in the subject related to clinical trial that is not part of the efficacy measurements
What is an adverse reaction?
An adverse event that is considered potentially related to an experimental therapy
What is included in a summary product characterization?
- Maximum dose of drug that can be used
- Who should not be given the drug or at reduced dose
- Are there any interactions with other drugs
- A description of most important and frequent expected adverse effects and their likely frequency
- Are there any routine follow ups and monitoring e.g blood tests of liver function that patients should have
Everything done in clinical trials is to not compromise the safety of patients. TRUE OR FALSE?
TRUE
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?
TRUE
What are the possible reasons for the decline in drug development?
- The bar of approval by regulatory affairs is getting higher
- The science is getting harder/ most of simple drugs receptors have already been found
- The world is more expensive /site investigation and end point measurements
- Market access - Getting regulatory approval and SmpC does not get you on commercial basis for sales