Clinical trials vs real life healthcare

Question 1

Why are clinical trials better than real life healthcare? (7)

Answer 1

1) Better standard of care
- Better survival outcomes
- Better care quality

2) Improvement of patient experiences
- More informed on their condition
- Better engagement

3) Increases treatment options eg, in rare cancers

Question 2

What is research waste and how can it be avoided? (4)

Answer 2

Research aims that have no societal benefit
To reduce:
- Ask relevant questions
- Appropriate design and methods
- Accessible full publication
- Unbiased and unstable report

Question 3

What is the difference between external and internal validity? (8)

Answer 3

External validity:
Making sure the results are generalisable to the ‘real world’
- Randomised trials
- Differences in healthcare systems globally
Main aim is effectiveness: does this intervention have benefit in clinical outcomes?

Internal validity
Quality of the research methods and ensuring there is ‘maximum’ benefit in a fair test
- Lack of confounding factors
- Right outcome measures
Main aim is efficacy: does this intervention act well on the right target in a ‘fair test’?

Question 4

How is external validity compromised? (4)

Answer 4

Strict eligibility criteria to reduce confounding factors

• Multimorbidity
• Concomitant medication
• Adherence

Short exposure time
- Long term efficacy and safety?

Question 5

How can limited exposure in a clinical trial reduce external validity? (9)

Answer 5

Long term efficacy

• In chronic conditions
• Comparing standard of care in different regions
• In specific groups

Long term safety

• Rare adverse events
• Delayed adverse events
• Adverse events in specific groups
• Uncertainty and differences in health care systems globally

Question 6

What are some limitations in real world studies? (6)

Answer 6

• Internal validity trade off
• Selection bias
• Recall bias
• Detection bias
• Unclean/ missing data
• Heterogeneity in clinical and lab measurements

Question 7

Who is important to increase implementation speed? (10)

Answer 7

Patients- adherence, engagement
Clinicians- inertia, lack of time in appointments
Healthcare systems- lack of follow up and public awareness
Stakeholders

Question 8

What are the 4 phases in implementation research? (4)

Answer 8

1) Reimplementation
2) Piloting
3) Implementation and evaluation
4) Postimplementation and dissemination