14.Randomization06.N of 1 - sporedata/researchdesigneR GitHub Wiki

1. Use cases: in which situations should I use this method?

N-of-1 trials are useful in the context of personalized medicine, where different interventions are randomly assigned to the same individual over time

2. Input: what kind of data does the method require?

N-of-1 are often used when the interventions have a rapid onset and washout (few lasting carryover effects).
Requires motivated patients.

3. Algorithm: how does the method work?

Model mechanics

Describing in words

N-of-1 assumes no carryover and also a lack of system change (i.e., changes in the health system besides the change in the intervention)

Describing in images

Describing with code

Breaking down equations

Reporting guidelines

Guidelines for the Content of Statistical Analysis Plans in Clinical Trials [1].

Data science packages

Suggested companion methods

Meta-analyses of n-of-1 trials.

Learning materials

Books *
Articles
- Treatment effects in multicenter randomized clinical trials [2].
- The n-of-1 clinical trial: the ultimate strategy for individualizing medicine? [3].
- Design and Implementation of N-of-1 Trials: A User’s Guide [4].
- Common references for randomized designs

4. Output: how do I interpret this method's results?

Mock conclusions or most frequent format for conclusions reached at the end of a typical analysis.

Tables, plots, and their interpretation

5. SporeData-specific

Templates

N of 1

Data science functions

References

[1] Gamble C, Krishan A, Stocken D, Lewis S, Juszczak E, Doré C, Williamson PR, Altman DG, Montgomery A, Lim P, Berlin J. Guidelines for the Content of Statistical Analysis Plans in Clinical Trials. Jama. 2017 Dec 19;318(23):2337-43.

[2] Senn SJ, Lewis RJ. Treatment effects in multicenter randomized clinical trials. Jama. 2019 Mar 26;321(12):1211-2.

[3] Lillie EO, Patay B, Diamant J, Issell B, Topol EJ, Schork NJ. The n-of-1 clinical trial: the ultimate strategy for individualizing medicine?. Personalized medicine. 2011 Mar;8(2):161-73.

[4] Kravitz RL, Duan N, Eslick I, Gabler NB, Kaplan HC. Design and Implementation of N-of-1 Trials: A User’s Guide. Agency for healthcare research and quality, US Department of Health and Human Services. 2014 Feb.