A real-world comparison of effectiveness and safety outcomes between clinical trial eligible and ineligible patients in the British Association of Dermatologists Biologic Interventions Register (BADBIR).

Background: There is preliminary evidence that psoriasis patients eligible for clinical trials of biologics are not representative of real-world patients.
Objective: To determine whether patients enrolled in BADBIR identified as Eligible or Ineligible for clinical trials differed in effectiveness and safety profiles.
Methods: BADBIR is a pharmacovigilance register of psoriasis patients exploring the long-term safety of biologics compared to conventional systemic therapies. Patients with at least 6 months of follow-up who registered on Enbrel (n=1509), Humira (n=4000) or Stelara (n=1627) were included in the analysis. Eligibility criteria were extracted from phase III trials submitted for licensing. The analyses focused on patients classified as Eligible and Ineligible. Effectiveness was calculated by linear regression of absolute change in Psoriasis Area and Severity Index (PASI) from baseline to 12 months. Incidence rate ratios (IRR) were calculated between the eligibility categories for the total number of serious adverse events (SAEs) reported within 12 months of initiating therapy.
Results: 838 (56%) Enbrel, 2219 (56%) Humira and 754 (46%) Stelara registrations were classified as Eligible with 367 (24%), 282 (7%) and 394 (24%) registrations classified as Ineligible, respectively. Absolute changes in PASI at 12 months for Ineligible Humira and Stelara patients were significantly smaller than for Eligible patients (β [95% confidence interval]; 2.7, [0.5, 4.9] and 1.8 [0.1, 3.5], respectively). There was no significant difference in absolute change in PASI for Enbrel patients at 12 months. SAE rates were highest in the Ineligible patients, with significantly higher IRR compared with Eligible patients (IRR [95% confidence interval]; Enbrel 1.9, [1.4, 2.6]; Humira 2.0 [1.5, 2.6]; Stelara 2.8 [2.1, 3.8]).
Conclusion: Patients classified as Ineligible for clinical trials for Enbrel, Humira and Stelara have lower effectiveness and higher SAE rates than those who would be Eligible. Thus, clinical trial findings are not representative of real-world patients.