Wednesday, May 15, 2019

Medical trial metrics

The following explains basically what kind of analysis is done on the data from a trial to evaluate how successful is a given treatment. If you read the links below, in bold red, you are set. The others are for context, etc. (you can skip as you gotta get moving :) )
  1. Metrics: Definition of the outcome parameters of a clinical trial (endpoints) to judge a given treatment. Things like OS, PFS, ORR... Basically these are measures till a given endpoint (when something happens to the patient). For instance, time till the patient dies or the tumor disappears... (for the math see #2):
    1. A paper giving a bit more of background on this. 
    2. And another document from fda giving even more info. 
    3. The Clinical Viewpoint: Definitions, Limitations of RECIST, Practical Considerations of Measurement
    4. Which Metrics Are Appropriate to Describe the Value of New Cancer Therapies?
  2. Statistics: The above are the parameters to be analyzed, but what are/how to understand the statistics of those parameters?
    1. Hazard Ratio (HR):  the chance of an event occurring in the treatment arm divided by the chance of the event occurring in the control arm. A nice explanation (but no math) with examples is here. "A hazard ratio of 2 means that a treated patient who has not yet healed by a certain time has twice the chance of being healed at the next point in time compared to someone in the control group." "The hazard ratio is equivalent to the odds that an individual in the group with the higher hazard reaches the endpoint first." Note: odds in gambling is the ratio of something happening vs not happening. For instance, a 2 to 1 odds ratio, means that the probability of something happening is double than not happening but that actually means that it will happen 2 out of 3 times.
      1. And check this out for math with examples.
      2. For the hardcore math, maybe this paper can help.
Work in progress but for the time being...

Cheers!

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