Exploring the Potential for EHR-Derived Real-World Data to Reduce Uncertainty in HTA Decision-Making: A Case Study of Long-Term Survival Outcomes


Pittell H1, Kent S2, Groves B2, Mpofu P3, Baxi S3, Copeland A3, Bargo D4, Adamson B3, Jonsson P5
1Flatiron Health, Great Neck, NY, USA, 2National Institute for Health and Care Excellence (NICE), London, LON, UK, 3Flatiron Health, New York, NY, USA, 4Flatiron Health, Brooklyn, NY, USA, 5National Institute for Health and Care Excellence (NICE), Manchester, UK

Presentation Documents

OBJECTIVES: Clinical trials are an important source of evidence for health technology appraisals (HTA). However, a key concern is uncertainty in survival due to immature data. This study investigates whether electronic health record (EHR)-derived data from the US may have the potential to reduce uncertainty in long-term outcomes, using NICE technology appraisal (TA) 531 as a case study.

METHODS: We selected patients with previously untreated, Stage IV NSCLC, with positive or unknown PDL1 status, who initiated first-line pembrolizumab monotherapy between October 2016 and December 2020 from the nationwide de-identified EHR-derived Flatiron Health database. We applied additional lab and ECOG eligibility criteria. Outcomes were overall survival from treatment start and treatment duration. Sensitivity analyses assessed a sub-group with known PDL1 status and a time horizon ending at NICE TA publication.

RESULTS: The study included 1109 patients (median age 72, 50% female). Real-world duration of therapy was median 5.0 months (95% Confidence Interval [CI]: 4.2-5.7). Median overall survival was 13.8 months (95% CI: 11.8-16.2) over the full study period. The sensitivity analysis excluding patients with missing PDL1 status found median overall survival of 14.9 months (95% CI: 12.5-17.6). The shorter time horizon (October 2016 to June 2018) estimated median overall survival of 13.1 months (95% CI: 10.8-NR).

CONCLUSIONS: In this case, EHR-derived data offered longer follow-up time (max 49 months) than the trial follow-up (max 22 months) used for extrapolation. This cohort had a median overall survival of 13.8 months while the trial (n=154) estimated 30.0 months and a similar Medicare claims analysis (n=3079) estimated 11.4 months. Real-world median age was 7-9 years older than the trial. Our study demonstrates that EHRs can be a source of mature data on specific cohorts of interest with potential to contextualize trial evidence and inform HTA-decision making.

Conference/Value in Health Info

2021-11, ISPOR Europe 2021, Copenhagen, Denmark

Value in Health, Volume 24, Issue 12, S2 (December 2021)

Acceptance Code



Clinical Outcomes

Topic Subcategory

Comparative Effectiveness or Efficacy



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