Real-world data from expanded access programmes in health technology assessments: A review of NICE technology appraisals

Objectives To quantify and characterise the usage of expanded access (EA) data in National Institute for Health and Care Excellence (NICE) technology appraisals (TAs). EA offers patients who are ineligible for clinical trials or registered treatment options, access to investigational therapies. Although EA programmes are increasingly used to collect real-world data, it is unknown if and how these date are used in NICE health technology assessments. Design Cross-sectional study of NICE appraisals (2010-2020). We automatically downloaded and screened all available appraisal documentation on NICE website (over 8500 documents), searching for EA-related terms. Two reviewers independently labelled the EA usage by disease area, and whether it was used to inform safety, efficacy and/or resource use. We qualitatively describe the five appraisals with the most occurrences of EA-related terms. Primary outcome measure Number of TAs that used EA data to inform safety, efficacy and/or resource use analyses. Results In 54.2% (206/380 appraisals), at least one reference to EA was made. 21.1% (80/380) of the TAs used EA data to inform safety (n=43), efficacy (n=47) and/or resource use (n=52). The number of TAs that use EA data remained stable over time, and the extent of EA data utilisation varied by disease area (p=0.001). Conclusion NICE uses EA data in over one in five appraisals. In synthesis with evidence from well-controlled trials, data collected from EA programmes may meaningfully inform cost-effectiveness modelling

Measuring quality of life using patient-reported outcomes in real-world metastatic breast cancer patients

Metastatic breast cancer (MBC) patients are almost always treated to minimize the symptom burden, and to prolong life without a curative intent. Although the prognosis of MBC patients has improved in recent years, the median survival afte

High-Intensity Care in the End-of-Life Phase of Castration-Resistant Prostate Cancer Patients: Results from the Dutch CAPRI-Registry

Background: Intensive end-of-life care (i.e., the overuse of treatments and hospital resources in the last months of life), is undesirable since it has a minimal clinical benefit with a substantial financial burden. The aim was to investigate the care in the last three months of life (end-of-life [EOL]) in castration-resistant prostate cancer (CRPC). Methods: Castration-resistant prostate cancer registry (CAPRI) is an investigator-initiated, observational multicenter cohort study in 20 hospitals retrospectively including patients diagnosed with CRPC between 2010 and 2016. High-intensity care was defined as the initiation of life-prolonging drugs (LPDs) in the last month, continuation of LPD in last 14 days, >1 admission, admission duration ≥14 days, and/or intensive care admission in last three months of life. Descriptive and binary logistic regression analyses were performed. Results: High-intensity care was experienced by 41% of 2429 patients in the EOL period. Multivariable analysis showed that age (odds ratio [OR] 0.98, 95% confidence interval [CI] 0.97–0.99), performance status (OR 0.57, 95% CI 0.33–0.97), time from CRPC to EOL (OR 0.98, 95% CI 0.97–0.98), referral to a medical oncologist (OR 1.99, 95% CI 1.55–2.55), prior LPD treatment (>1 line OR 1.72, 95% CI 1.31–2.28), and opioid use (OR 1.45, 95% CI 1.08–1.95) were significantly associated with high-intensity care. Conclusions: High-intensity care in EOL is not easily justifiable due to high economic cost and little effect on life span, but further research is awaited to give insight in the effect on patients' and their caregivers' quality of life