Patients', clinicians' and research's priorities on important outcomes in multiple myeloma: A mixed-methods study

Research in multiple myeloma increasingly relies on surrogate end-points to expedite approvals, yet these may not reflect patient priorities. We conducted a mixed-methods study to identify outcomes valued by patients and clinicians and compare them with end-points used in randomized controlled trials (RCTs). Interviews with 10 patients and 6 clinicians identified treatment priorities, which, together with end-points from a systematic review of myeloma RCTs, informed tailored surveys, completed by 117 patients and 105 clinicians. Both groups ranked quality of life (QoL) as most important (odds ratio [OR] 1.01; 95% confidence interval [CI] 0.55-1.87). Clinicians more often prioritized overall survival (OS) (OR 1.92; 95% CI 1.05-3.50) and progression-free survival (PFS) (OR 5.37; 95% CI 1.95-14.79), whereas patients prioritized pain reduction (OR 0.03; 95% CI 0.00-0.23). Compared with RCT end-points, patients emphasized QoL (OR 0.03; 95% CI 0.01-0.07) and pain elimination (OR 0.05; 95% CI 0.02-0.11), while trials favoured PFS (OR 6.33; 95% CI 2.53-15.83) and response (OR 17.75; 95% CI 5.56-56.61). Clinicians aligned with trials on PFS but valued QoL and OS more highly. QoL emerged as a shared priority, underscoring the need for patient-centred trial designs that better capture outcomes meaningful to those living with myeloma.