Quantifying Bias in Real-World Studies: A New Hope for RWD Acceptance or Are HTAers Gonna Hate?

Author(s)

Moderator: Sreeram Ramagopalan, PhD, Global Access, F. Hoffmann-La Roche Ltd, Basel, Switzerland
Speakers: Miguel Hernán, PhD, MD, MPH, ScM, Harvard University, Boston, MA, USA; Pilar Pinilla, MSc, Facultad de Economía, Empresa y Turismo, Universidad de Las Palmas de Gran Canaria, London, UK; Kristian Thorlund, PhD, Cytel, Inc., Waltham, MA, USA; Seamus Kent, PhD, National Institute for Health and Care Excellence (NICE), London, LON, UK

Presentation Documents

Medicinal development for rare-disease has become increasingly reliant on single-arm trials, which poses challenges for health technology assessment (HTA). While external or synthetic control arms (ECAs or SCAs) using real-world data (RWD) are potential tools to aid decision-making for drug reimbursement by providing comparative data, there are understandable concerns with non-randomised comparisons. Statistical techniques such as quantitative bias analysis (QBA) can give decision-makers a measure of uncertainty around the effect estimates from a non-randomised comparison. However the practical utility of QBA in a HTA setting is currently unclear.

The QBASEL study aimed to investigate the utility of QBA methods for assessing uncertainty in RWD SCA- trial comparisons. This demonstration project investigated 14 trials for advanced non-small cell lung cancer with SCAs being generated from RWD from oncology electronic health records from the United States. The trials which differed by sample size, geographical coverage, biomarker usage and line of therapy provide allowing an understanding of when and how QBA methods may be most useful.

This session will have a varied discussion on the merits of QBA, led by a range of professionals across academic, HTA, and industry settings. First, there will be an overview of the general context and implementation of QBA methodology in an SCA setting. Second, panelists will detail the results from the QBASEL study. The session will conclude with a discussion on the applied and pragmatic implications of the QBASEL results on HTA decision-making.

Conference/Value in Health Info

2022-11, ISPOR Europe 2022, Vienna, Austria

Code

111

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