DOES PARTICIPATION IN THE UK CANCER DRUGS FUND RESOLVE UNCERTAINTY AND IMPROVE STRENGTH OF CLINICAL EVIDENCE DURING NICE TECHNOLOGY APPRAISALS
Author(s)
Paul Okediji, MBChB1, Catrin Austin, PhD2, Sarika Paul, PhD2, Stephen Norton, PhD2, Martin W. Njoroge, PhD2, Milena Wobbe, PhD2, Steve Williamson, MSc, BPharm2.
1National Institute for Health and Care Excellence (NICE), London, United Kingdom, 2National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom.
1National Institute for Health and Care Excellence (NICE), London, United Kingdom, 2National Institute for Health and Care Excellence (NICE), Manchester, United Kingdom.
OBJECTIVES: The reformed Cancer Drugs Fund (CDF) was launched in July 2016 to enable conditional patient access to cancer medicines with clinical uncertainty at initial NICE appraisal. No comprehensive evaluation of whether CDF participation strengthens evidence certainty between CDF entry and exit currently exists. This evaluation aims to determine whether uncertainties in clinical evidence identified at CDF entry are resolved at NICE reassessment and whether cancer medicines demonstrate a difference in clinical benefit between CDF entry and exit appraisals.
METHODS: Using a mixed methods approach, quantitative data was collected from NICE appraisal documents including final guidance and data collection arrangements (DCAs) for each CDF topic since July 2016. Changes in overall survival (OS) and progression-free survival between CDF entry and exit, and committee opinion on whether uncertainties were resolved following CDF participation were determined. Qualitative data on the value of evidence generated in the CDF is being collected via semi-structured interviews with key NICE and NHS England stakeholders.
RESULTS: Preliminary analysis of 12 randomly selected CDF topics showed 30 named uncertainties across entry DCAs (range: 1-4 uncertainties per topic). Of the 30 identified uncertainties, 37% were resolved, 17% partially resolved, and 47% remained unresolved at NICE reassessment. Median OS increased in 2 (of 3) topics with comparable data (range: 0.03-26.5 months). Hazard ratios increased in 4 topics (mean change: 0.05), indicating attenuation of relative treatment effect. Full quantitative results across all identified CDF topics and qualitative interview findings will be available by autumn 2026.
CONCLUSIONS: While CDF participation can facilitate full uncertainty resolution in some topics, preliminary data indicates that a substantial proportion of uncertainties remain unresolved. A more in-depth analysis of the relative impact of these unresolved uncertainties on the HTA process will be undertaken. Final results will help inform an ongoing internal review of NICE's managed access program.
METHODS: Using a mixed methods approach, quantitative data was collected from NICE appraisal documents including final guidance and data collection arrangements (DCAs) for each CDF topic since July 2016. Changes in overall survival (OS) and progression-free survival between CDF entry and exit, and committee opinion on whether uncertainties were resolved following CDF participation were determined. Qualitative data on the value of evidence generated in the CDF is being collected via semi-structured interviews with key NICE and NHS England stakeholders.
RESULTS: Preliminary analysis of 12 randomly selected CDF topics showed 30 named uncertainties across entry DCAs (range: 1-4 uncertainties per topic). Of the 30 identified uncertainties, 37% were resolved, 17% partially resolved, and 47% remained unresolved at NICE reassessment. Median OS increased in 2 (of 3) topics with comparable data (range: 0.03-26.5 months). Hazard ratios increased in 4 topics (mean change: 0.05), indicating attenuation of relative treatment effect. Full quantitative results across all identified CDF topics and qualitative interview findings will be available by autumn 2026.
CONCLUSIONS: While CDF participation can facilitate full uncertainty resolution in some topics, preliminary data indicates that a substantial proportion of uncertainties remain unresolved. A more in-depth analysis of the relative impact of these unresolved uncertainties on the HTA process will be undertaken. Final results will help inform an ongoing internal review of NICE's managed access program.
Conference/Value in Health Info
2026-11, ISPOR Europe 2026, Vienna, Austria
Value in Health, Volume 29, Issue 12S
Code
HPR240
Topic
Clinical Outcomes, Health Policy & Regulatory, Health Technology Assessment
Topic Subcategory
Coverage with Evidence Development & Adaptive Pathways, Reimbursement & Access Policy
Disease
No Additional Disease & Conditions/Specialized Treatment Areas, Oncology