Abstract
BACKGROUND: The Ibrance® Patient Program was established to provide access to palbociclib for UK National Health Service (NHS) patients with metastatic breast cancer (MBC), pending a funding decision. METHODS: Non-interventional cohort study involving a retrospective medical record review of patients commenced on palbociclib between April and December 2017 at eight UK centres. Primary outcomes included clinicopathological characteristics, treatment patterns, clinical outcomes and selected adverse events. RESULTS: Overall, 191 patients were identified, median age of 57.0 years (range 24.3-90.9); 30% were diagnosed with de novo MBC; 72% received first-line and 10% as ≥ second-line treatment. Median progression-free survival (95% CI) was 22.8 months (16.5-not reached [NR]) in first-line; NR in patients with de novo MBC; 7.8 months (6.8-NR) in ≥ second-line (median follow-up: 24 months). Median overall survival (OS) was NR in the overall cohort; OS rate (95% CI) at 24 months was 74.2% (67.1-81.9%) in first-line; 82.1% (72.6-92.8%) in patients with de novo MBC; 55.0% (37.0-81.8%) in ≥ second-line. Forty-seven per cent of patients developed grade 3-4 neutropenia; 3% febrile neutropenia. CONCLUSION: This study supports the effectiveness of palbociclib and demonstrates the benefit to patients of early access schemes that bridge the gap between regulatory approval and NHS funding for new medicines. CLINICAL TRIAL REGISTRATION: Clinical trial: ClinicalTrial.gov:NCT03921866.