Experience with outcomes research into the real-world effectiveness of novel therapies in Dutch daily practice from the context of conditional reimbursement

Policymakers more often request outcomes research for expensive therapies to help resolveuncertainty of their health benefits and budget impact at reimbursement. Given the limi-tations of observational data, we assessed its usefulness in evaluating clinical outcomes forbortezomib in advanced multiple myeloma patients. Data were retrospectively collectedfrom patients included in the pivotal Assessment of Proteasome Inhibition for Extend-ing Remissions trial (APEX; n = 333) and two groups of daily practice patients treatedwith bortezomib following progression from upfront therapy (n = 201): real-world patientstreated as of May 2009 (RW-1; n = 72) and June 2012 (RW-2; n = 129). Prognosis, treat-ment, and effectiveness were compared. Outcomes research was useful for policymakers foraddressing to whom and how bortezomib was administered in daily practice. It was limitedhowever in generating robust evidence on real-world safety and effectiveness. The qual-ity of real-world evidence on effectiveness was low due to missing data in patient charts,existing treatment variation and the dynamics in care during the novel drug’s initial mar-ket uptake period. Policymakers requesting real-world evidence on clinical outcomes forreimbursement decisions should be aware of these limitations and advised to carefully con-sider beforehand the type of evidence that best addresses their needs for the re-assessmentphase.