Analysis of real-world data for health technology assessment submission

Vitaccess is partnering with a pharmaceutical company on an international, patient-centric real-world evidence study. This observational longitudinal study is designed to increase understanding of the impact of living with a rare autoimmune, neuromuscular disease.

The Institute for Clinical and Economic Review undertook an assessment of treatments for this disease and requested evidence on important contextual considerations, including extent of unmet need, caregiver impact, and complexity of current treatment regimens. The client also sought to influence the proposed approach to economic modelling.

• • To compile and execute an analysis plan of the real-world observational study data to meet the requirements of the ICER draft scoping document.

  • To support the client in providing input to ICER’s approach to economic modelling and analyses.

• • Scope: US.

  • We developed a de novo analysis plan, to meet ICER’s data request and align with the client’s objectives on product positioning and demonstration of cost-effectiveness, including:

(i) Analyses of correlations between EQ-5D utility scores and the range of variables that could potentially be used to define model health states, to demonstrate which would be most appropriate for economic modelling

(ii) Comparison of the burden of disease in the target eligible patient population for the client’s product and the wider disease population

(iii) Exploration of the relationships between disease severity, as measured by activities of daily living score, and direct and indirect costs

(iv) Analysis of categories of pharmacological treatments used by disease severity.

• • Analysis plan covering level of aggregation, medication, hospital-based resources, primary care healthcare visits, societal burden, and targeted analyses based on economic model/health state requirements

  • Preliminary summary data

  • Draft analysis report, including data tables

  • Final analysis report

This study provided data on health-related quality of life, healthcare resource use, caregiver burden, and work productivity, which were not available from clinical trials.

The data analysis provided RWE on key contextual considerations and the optimal approach to economic modelling for HTA review.

The Vitaccess Real™ study platform is flexible and adaptive and, with the support of in-house strategic consultancy, provided the capability to:

• • Access detailed data quickly, enabling rapid response to time-critical requests, e.g., providing detailed contextual data for HTA

  • Design and execute a supplementary analysis plan to respond to evolving client needs

  • Provide robust, evidence-based argumentation on alternative approaches to economic modelling.