4 - Non-Inferiority Trials** Flashcards

1
Q

Define superiority trial

A

Trial designed to show that tx A is better than tx B

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2
Q

Define non-inferiority trial

A

Trial to test whether tx A is not worse than tx B, by a prespecified margin

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3
Q

Define equivalence trial

A

Trial to test whether tx A is equal to tx B

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4
Q

When should a non-inferiority trial be used?

A

When new tx has other advantages than primary outcomes (ex: route of administration, side effects, monitoring schedule)

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5
Q

What are the hypotheses for a superiority trial?

A
  • Null = no difference in tx

- Alternative = difference in tx

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6
Q

What are the hypotheses for a non-inferiority trial?

A
  • *Opposite to superiority
  • Null = difference in tx
  • Alternative = no difference in tx
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7
Q

What determines sample size?

A
  • Power
  • Type 1 error rate
  • Effect size
  • Underlying event rate
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8
Q

What is the difference between superiority and non-inferiority trials when determining statistical significance?

A
  • Superiority compare to reference line (0 for comparing difference or 1 for comparing relative risk or hazard risk)
  • Non-inferiority compare to NI margin
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9
Q

Should intent to treat or per protocol be used in non-inferiority trials?

A
  • Both

- Results should agree or conclude inferior

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10
Q

Are failed superiority trials the same as non-inferiority trials?

A

No

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11
Q

Describe the non-inferior checklist

A
  • Was study designed as non-inferiority trial?
  • Has clinical effectiveness of standard tx been well established and consistent?
  • Is non-inferiority margin defined and its derivation understood?
  • Is sample size calculated based on alpha, beta, and non-inferiority margin?
  • Was planned sample size achieved in trial?
  • Was the result achieved in the active control arm as expected?
  • Were ITT and per protocol analysis performed and do they give a consistent answer?
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12
Q

What is an acceptable NI margin?

A

30%

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