Personalized trial designs

10 important questions on Personalized trial designs

What is an ethical consideration when giving a new drug to a rare disease?

It is ethical to give a placebo

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.

  1. N-of-1 design
  2. Multiple baseline design
  3. Alternating-treatment design
  4. Changing-criterion design

What is the bi-phasic AB design?

A = baseline phase
B = intervention phase
=> track the change in outcome compared to baseline
=> you can not control for external variables
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What is the alternating treatment design?

You change treatment in the same person.

Pro: quick comparison
Con: interaction effects. The result can not be long term.

What are pros and cons of the n-of-one?

Pro:
  • 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
Cons:
  • 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?

Assess the variability in a patientgroup => in that way you can choose the most suitable patient.

Which studies do not have a control?

1-phase design
- 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?

=> pathways can also be important for less rare patients groups.

The question on the page originate from the summary of the following study material:

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