Introduction Of Clinical Trials Flashcards
Clinical trial is?
Human experiments involving medical intervention ( I.e drugs, surgical procedures etc)
Importance of clinical trials
To obtain approval for new drug/procedure from a regulatory body
To obtain a new indication for an established drug
Compare 2 treatments
Determine it the new medicine is safe and effective
Phase 0 referred to as?
Pre - clinical
Phase 1 role
Assess the safety of the drug
Phase 2 role
Efficacy and safety of the drug
Phase 3 role
Drug effectiveness and possible adverse receptions
Phase 4 role
Testing drug after market approval
Investigational new drug (IND) application: must include information on?
Animal pharmacology and toxicology studies
Preclinical data used to assess whether the drug is sate for testing in humans
Chemistry and manufacturing information whether the drug is stable, able to be manufactured with high qualify
Clinical protocols describing study designs, safety assurance
Investigators brochure
Investigators brochure contains?
The information investigators need to know in order to conduct a safe and appropriate clinical trial
What happens in phase 1?
New drug tested on a small number of healthy volunteers
Participants given cow dose of drug then incrementally increase the dose of the drug until which could potentially cause some sort of a side effect
Typically last several months, can be longer up to 2 years
What is required in phase 7 before progressing to phase 2?
Drug and food interactions
What happens in phase 2?
Studies safety and effectiveness of the drug to treat a disease
Randomised /non- randomised and often blinded / double-blinded
Can Last for months to years (2-3 years)
What % of drugs successfully complete phase 2 studies?
25%
Phase 2 can be subdivided into what phases?
Phase 2a ( proof of concept)
Phase 2b ( pivot trails)
What happens in phase 2a:
Pilot trials to evaluate safety and efficacy in selected groups of patients with the condition to be treated
- goal is to refine frequency of dosing or particular aspects of safety and efficacy
What happens in phase 2b:
Typically represents the most rigorous demonstration of a drug’s efficacy
- tells us what type of dose to use in phase z
What happens in phase 3?
Large number of participants, diverse patient population which is representative of the main target population
Double blinded and randomised- give existing therapy in market instead of placebo
Confirmation of efficacy, safety and benefit :risk ratio at the selected dose(s)
What does phase 3 provide investors and regulatory body?
With most complete information about the drug’s effectiveness and possible adverse reaction
What phase is a regulatory requirement for substantive evidence?
Phase 3
Usually at least 2 well designed studies (reproducible)
If phase 3 is successful?
Investigators can seek regulatory approval to market the drug
Phase 4 known as?
Post market surveillance trial
What happens in phase 4?
Drug is approved for marketing by regulatory body an can be used in wider community
Strong test of drug safety and effectiveness
Aim of phase 4?
Compare a drug with other drugs already in the market
Monitor a drug’s long-term effectiveness
Determine the cost-effectiveness
In which phase can result in drug being taken off market/restricted?
Phase 4
What to do to get strongest study design
Ensure that study is blinded
Randomised groups
Controlled trial: standard vs new drug compared to standard vs placebo
Examples of clinical trials eligibility criteria
Certain medical condition
Certain stage of condition/disease
Age
Other co-morbidities and drugs that could alter results
Benefits of participating in a clinical trial
Receive new treatment
Close supervision from clinical staff
Be part of research breakthrough that may help others
Risk of participating in a clinical trial
May not benefit from treatment: no effect, unsafe
Side effects
Trials car be angry and require extra treatments, and time in clinic
May not receive new treatment al all
Underrepresented populations in clinical trials
Older adults - may new co-morbidities
Pregnant women-can harm baby
Children under 16-ethical issues
Ethnic minorities
What can be done to increase participation in underrepresented populations?
Require strong justification for exclusion criteria
Encourage clinical tricks specific to the underrepresent group
Publicise trends in the inclusion of these groups in clinical trials to assess progress
Consider adaptive platform trial designs
What are adaptive platform clinical trials?
Based on predefined criteria, you conduct a clinical trial and may decide to adapt it in some way e.g dose, and then analyse data
Population is?
The entire group that you want to draw conclusions about
Sample is?
A group of people who will participate in the study that is representative of the population
Considerations in designing a clinical trial
Safety
Avoid bias
Measurable endpoints
Allows for statistical analysis
Good clinical practice
International ethical and scientific quality standard for designing, conducting, recording and reporting trials that involve the participation of human subjects
Sponsor
An individual, company, institution organisation or group a organisations that take on responsibility for initiation, management and financing (or arranging of financing) of research
Research questions for phase o
Does the drug bind the target protein?
PK
