Personalized trial designs
10 important questions on Personalized trial designs
What is an ethical consideration when giving a new drug to a rare disease?
What are different study designs for rare diseases? Ideal because?
- Single case methodology; including (bi-phasic AB design)
- Single case experimental designs = with randomization
- Ideal because they can provide short term evidence based treatment decisions.
- N-of-1 design
- Multiple baseline design
- Alternating-treatment design
- Changing-criterion design
What is the bi-phasic AB design?
B = intervention phase
=> track the change in outcome compared to baseline
=> you can not control for external variables
- Higher grades + faster learning
- Never study anything twice
- 100% sure, 100% understanding
What is the alternating treatment design?
Pro: quick comparison
Con: interaction effects. The result can not be long term.
What are pros and cons of the n-of-one?
- Statistical power
- Optimal treatment for individuals => Identify components of variation => patient is its own control
- You can say something about the effectiveness on group level
- It is possible in patients with comorbidities => more inclusive patient population
- How variable is the disease => can you see if the drug is effective or not
- Long term effects are challenging
- Generalization => clarify disorder, mutation (loss of function oid), setting and location
How should you statistically analyze the n-of-one?![]()
Which studies do not have a control?
- Multiple measurements possible
Pre-post intervention
Case description
Why is deep phenotyping important?
- Beste care => best patient.The emerging field of personalized medicine aims to provide the best available care for each patient based on stratification into disease subclasses with a common biological basis of disease
- To set good baseline characteristics and outcomes.
What are general challenges for rare diseases?
- Generalizability
- Recruitment is difficult because you haw a low number of patients
- Do interim analysis because patients may stop using the drug
- What if it is effective- is it also cost effective?
What can make research on rare diseases make it cost effective?
The question on the page originate from the summary of the following study material:
- A unique study and practice tool
- Never study anything twice again
- Get the grades you hope for
- 100% sure, 100% understanding
