Association of progression-free survival with patient-reported outcomes and survival: results from a randomised phase 3 trial of panitumumab.

In a randomised phase 3 trial, panitumumab significantly improved progression-free survival (PFS) in patients with refractory metastatic colorectal cancer (mCRC). This analysis characterises the association of PFS with CRC symptoms, health-related quality of life (HRQoL), and overall survival (OS). CRC symptoms (NCCN/FACT CRC symptom index, FCSI) and HRQoL (EQ-5D) were assessed for 207 panitumumab patients and 184 best supportive care (BSC) patients who had at least one post-baseline patient-reported outcome (PRO) assessment. Patients alive at week 8 were included in the PRO and OS analyses and categorised by their week 8 progression status as follows: no progressive disease (no PD; best response of at least stable disease) vs progressive disease (PD). Standard imputation methods were used to assign missing values. Significantly more patients were progression free at weeks 8-24 with panitumumab vs BSC. After excluding responders, a significant difference in PFS remained favouring panitumumab (HR=0.63, 95% CI=0.52-0.77; P<0.0001). At week 8, lack of disease progression was associated with significantly and clinically meaningful lower CRC symptomatology for both treatment groups and higher HRQoL for panitumumab patients only. Overall survival favoured no PD patients vs PD patients alive at week 8. Lack of disease progression was associated with better symptom control, HRQoL, and OS.

Impact of palivizumab on expected costs of respiratory syncytial virus infection in preterm infants: potential for savings.

In its clinical assessment of the respiratory syncytial virus (RSV)-specific monoclonal antibody palivizumab, the IMpact-RSV Study Group demonstrated a reduction in hospitalizations for RSV-related lower respiratory tract infection in infants who received prophylaxis compared with infants who did not receive prophylaxis. An assessment of the RSV-related expenses for managing both groups of infants is needed to provide insight into the value of prophylaxis. The present study was conducted to identify and compare RSV-related health care expenditures incurred by infants who did not receive prophylaxis throughout one RSV season and after. Using a decision-analytic model populated with data from the contemporary medical literature, a pharmacoeconomic study was conducted from the perspective of the payer. Probabilities for RSV-related hospitalizations of infants who did and did not receive prophylaxis were abstracted from several published studies. Components of inpatient and outpatient care were identified through examination of hospital records, reviews of the published literature, and consultation with expert clinicians. Charges related to prophylaxis and medical management of infection were abstracted from hospital billing records and published data. Appropriate charges were applied to decision-tree branches and multiplied by in-line probabilities for outcomes. Products at terminal nodes were summed to establish total expected charges for both groups of infants. Widespread clinical use of prophylactic palivizumab would result in incremental expenses < or =$3459 per infant or cost savings < or =$39,107 per infant. The variability in value of prophylaxis derives from the rate of RSV-related hospitalizations in the community and the total health care expense of managing infected infants.