Control Groups in Randomized Clinical Trials (RCTs) Supporting Approval of Drugs for Systemic Rheumatic Diseases, 2012-2022
Authors' conclusion
These findings suggest that the quality of control groups in RCTs leading to SRD drug approval needs improvement
Study context
- Population
- Pivotal RCTs supporting systemic rheumatic disease (SRD) new drug approvals by the Food and Drug Administration (FDA), January 2012 to October 2022pivotal RCTs
- Participants
- 65 rcts
- Outcome
- Proportion of RCTs using optimal control groups
Methodological detailsmeta-research of pivotal RCTs supporting FDA approvals
- Meta-research of pivotal RCTs supporting FDA approvals
Main finding
As reported in the abstract
RCTs using optimal control groups
RCTs using optimal control groups
16
Observational data: associations, not effects.
Further findings
| Outcome | Values |
|---|---|
| Response rate in placebo groups (all placebo-controlled trials) | Placebo median 26.0% (IQR 19.2%–32.3%) |
Trials with no active groups that were suboptimal
- Suboptimal among 55 trials with no active groups
- 47 trials [85.5%]
Systemic arthritis trials with suboptimal control groups (subgroup)
- Suboptimal control groups among 56 systemic arthritis trials
- 49
- Placebo or dose-response controls
- 47
- Active controls
- 2
Other SRDs: placebo or dose-response controls considered optimal
- Trials considered optimal
- 8
Response rate, investigating vs placebo groups
- RR mostly exceeding 1.50; lowest (anifrolumab)
- 0.90; 95% CI, 0.69-1.17
- RR highest (mepolizumab)
- 11.00; 95% CI, 2.69-44.96
- Significant improvement in response rates of investigating compared with placebo groups
Original publication
Control Groups in RCTs Supporting Approval of Drugs for Systemic Rheumatic Diseases, 2012-2022. JAMA Netw Open. Published 1 November 2023. doi:10.
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