1. Trial Data Must Be Accessible: Why Collecting the Same Outcomes Isn’t Enough Outcomes in Trials and Systematic Reviews: Why We Should be Paying More Attention
Nicole Fusco
Society for Clinical Trials & International Clinical Trials Methods Conference
Liverpool, UK
May 8, 2017

2. Declaration of interests
May 2014-Oct. 2016: PCORI ME Nov Present: Thomas Greene fund for research scholarship

3. Trial information and data are available from multiple sources
Public Sources
Non-Public Sources
Journal Articles
Clinical Study Reports
Conference Abstracts
Information from Regulators
Individual Patient Data
Trial Registries

4. Five elements of a completely specified outcome
Time-Points
Domain
e.g., 1 month
e.g., Pain intensity
Method of Aggregation
Specific measurement IV II
e.g., 0-10 scale
e.g., Mean
Specific Metric
e.g., Value at a time-point
III
Saldanha IJ, et al. PLoS One 2014

5. Few domains, large number of outcomes
4 domains  64 defined outcomes
Mayo-Wilson E, et al. Submitted 2017

6. Example of multiple outcome definitions
With just 4 pre-specified outcome domains, we observed 214 defined effectiveness outcomes across 21 trials 21
Trials
Mayo-Wilson E, et al. Submitted 2017

7. Trials reported different defined outcomes
No outcomes reported in all trials
116/214 (54%) outcomes reported in only one trial

8. Effectiveness outcomes in public vs. non-public sources
We had non-public sources for 6/21 trials
More than half (110/214) of effectiveness outcomes appeared only in non-public sources

9. Adverse events in clinical trials

10. Adverse events in public vs. non-public sources
We had non-public sources for 6/21 trials
Most adverse events (83%) appeared only in non-public sources

11. Few AEs are reported in public sources, compared with non-public sources
This shows only 6 trials – we assume that the other trials are similar. Public sources for other trials report about the same number of AEs, so we assume that there are AEs unreported for those trials *
*Trial not published

12. Few trials report number of patients experiencing any AE
3 trials: AE data only in public sources
Using only public sources, we have data from only 5 trials
21 trials testing gabapentin for neuropathic pain
10 trials: reported AE data
9 trials: reported meta-analyzable AE data
2 trials: AE data in both public and non-public sources
Make sure to talk about the so what!!!! Before MA box, remind that we want to combine trials to inform policy
11 trials: did not report AE data
1 trial: did not report meta-analyzable AE data
4 trials: AE data only in non-public sources

13. Conclusions Effectiveness outcomes
Hundreds of different outcomes reported
4 domains  214 defined outcomes
Trials don’t report overlapping outcomes
Many outcomes were not reported in public sources
Adverse events
Hundreds of different adverse events reported
Most adverse events were not reported in public sources
This is a problem in general – not just for this case example

14. Multiple Data Sources (MUDS) Team
Steering Committee
Dickersin, Kay - PI (KD)
Fusco, Nicole (NF)
Li, Tianjing (TL)
Mayo-Wilson, Evan (EMW)
Tolbert, Elizabeth (ET)
Conception & design, funding
Doshi, Peter (PD)
Vedula, Swaroop (SV)
KD, TL
Protocol development, study implementation
Cowley, Terrie (TC)
Haythornthwaite, Jennifer (JH)
Hong, Hwanhee
Payne, Jennifer (JP)
Singh, Sonal (SS)
Stuart, Elizabeth (ES)
EMW, KD, TL, NF, ET, JE
Data acquisition
Bertizzolo, Lorenzo (LB)
Ehmsen, Jeffery (JE)
Gresham, Gillian (GG)
Heyward, James (JHe)
Lock, Diana (DL)
Rosman, Lori (LR)
Suarez-Cuervo, Catalina (CS)
Twose, Claire (CT)
KD, NF, EMW, TL, SV
Analysis and interpretation of data
Canner, Joseph (JC)
Guo, Nan (NG)
Hong Hwanhee (HH)
Stuart, Elizabeth (ES)
NF, EMW, KD, TL
Systematic Review Data Repository
Jap, Jens (JJ)
Lau, Joseph (JL)
Smith, Bryant (BS)
Ancillary studies
Golozar, Asieh (AG)
Hutfless, Susie (SH)
EMW, KD, TC

15. Thank you!

16. References
Saldanha, Ian J., et al. "Outcomes in Cochrane systematic reviews addressing four common eye conditions: an evaluation of completeness and comparability." PloS one 9.10 (2014): e