Possible uses of RWE in HTA

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Darren Robertson
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1 Inclusion of Real-World Evidence (RWE) in Health Technology Assessment (HTA) Feasibility & Technical Issues Keith Abrams, Pascale Dequen, Sylwia Bujkiewicz, David Jenkins, Reynaldo Martina & Nicola Cooper ISPOR, November 2014 Department Health Sciences University of Leicester, UK Possible uses of RWE in HTA Estimating Relative Effectiveness (RE) Mapping between outcomes Exploring heterogeneity & natural history Recent NICE case on use of RWE 1

2 Use of RCTs & RWE in estimating RE (Relative) Clinical Effectiveness RCTs Real World Evidence RCT 1 (A v X) RCT 2 (A v X) RCT 3 (A v X) Registry/Obs (A v X) Adjusting for casemix/confounding Common comparator + variable follow-up IPD available Propensity score/iv methods Incorporating RCTs & RWE in IC/NMA A B A B RWE Study,?adjusted for bias C C Prediction of future RCT using NMA (incl. RWE) A B C D RWE Bridging Study 2

Odds Ratio (log scale) Use of RCTs & RWE in estimating RE (Relative) Clinical Effectiveness Absolute Effectiveness/Decision Making RCT (A v X 1 ) RCT (B v X 2 ) RCT (C v X 3 ) Registry/Obs (X) Target

3 Odds Ratio (log scale) Use of RCTs & RWE in estimating RE (Relative) Clinical Effectiveness Absolute Effectiveness/Decision Making RCT (A v X 1 ) RCT (B v X 2 ) RCT (C v X 3 ) Registry/Obs (X) Target Pop n (X) Use of RCTs & RWE in estimating RE & NH Slope: (95% CI 1.45 to 0.79) unfavourable No-effect line Target Pop n effect favourable Control group risk (%) (log odds scale) Average effect Target Pop n baseline estimated & characterised using RWE 3

4 NICE Example of Use of RWE : Vertebral augmentation 2 procedures to treat painful Vertebral Compression Fractures (VCF) due to osteoporosis: Percutaneous vertebroplasty (PVP) involves injecting bone cement into the vertebral body (with or without stents) Balloon kyphoplasty (BKP) involves inserting a balloon-like device, slowly inflated and filled with bone cement, into the vertebral body Evaluated by NICE in 2013 Both recommended without stenting compared to noninvasive management (OPM) Mortality benefit? Primary outcome measures of interest in assessment of augmentation procedures were pain and functional status All-cause mortality was assessed as a secondary outcome; however, included RCTs were not powered to detect mortality difference and this effect did not achieve statistical significance, even when pooled The effect of vertebral augmentation on mortality is an important, yet inadequately understood issue. Large-scale registry data from Germany and the USA suggests that people with osteoporotic VCFs who have received augmentation have significantly improved survival rates however a definitive causal link could not be established Health Technol Assess.18(17):

5 Research questions: Can we use the observational data available from the US and Germany to estimate mortality differences between treatments for osteoporotic VCFs? Can we use bias adjustment methods to pool both randomised and observational (even after propensity adjustment) data on mortality? What impact do mortality differences have on the costeffectiveness of PVP and BKP vs. non-invasive management? Research questions: Can we use the observational data available from the US and Germany to estimate mortality differences between treatments for osteoporotic VCFs? Can we use bias adjustment methods to pool both randomised and observational (even after propensity adjustment) data on mortality? What impact do mortality differences have on the costeffectiveness of PVP and BKP vs. non-invasive management? 5

6 Power transform prior approach to RWE Corresponding to 30% relative bias Illustration of impact of weight [α] on the overall HR of PVP vs. OPM when pooling randomised data based with an informative prior based on RWE. Cost-effectiveness acceptability? 6

7 Cost-effectiveness acceptability Illustration of CEAC for both treatments at a 20,000/QALY willingness to pay threshold against ascending weight [α] for pooled HR using power transform prior model for RWE: PVP OPM 7

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