Feedback

Evaluating treatments in rare indications warrants a Bayesian approach

Affiliation
Cytel ,Toronto ,ON ,Canada
Mackay, Emma K.;
Affiliation
Cytel ,Toronto ,ON ,Canada
Springford, Aaron

Evaluating efficacy and real-world effectiveness for novel therapies targeting rare mutations or patient subpopulations with unmet needs is a growing challenge in health economics and outcomes research (HEOR). In these settings it may be difficult to recruit enough patients to run adequately powered randomized clinical trials, resulting in greater reliance on single-arm trials or basket trial designs. Additionally, evidence networks for performing network meta-analysis may be sparse or disconnected when comparing available treatments in narrower patient populations. These challenges create an increased need for use of appropriate methods for handling small sample sizes, structural modelling assumptions and more nuanced decision rules to arrive at “best-available evidence” on comparative and non-comparative efficacy/effectiveness. We advocate for greater use of Bayesian methods to address these challenges as they can facilitate efficient and transparent borrowing of information across varied data sources under flexible modelling assumptions, probabilistic sensitivity analysis to assess model assumptions, and more nuanced decision-making where limited power reduces the utility of classical frequentist hypothesis testing. We illustrate how Bayesian methods have been recently used to overcome several challenges of rare indications in HEOR, including approaches to borrowing information from external data sources, evaluation of efficacy in basket trials, and incorporating non-randomized studies into network meta-analysis. Lastly, we provide several recommendations for HEOR practitioners on appropriate use of Bayesian methods to address challenges in the rare disease setting.

Cite

Citation style:
Could not load citation form.

Access Statistic

Total:
Downloads:
Abtractviews:
Last 12 Month:
Downloads:
Abtractviews:

Rights

License Holder: Copyright © 2023 Mackay and Springford.

Use and reproduction: