Background <p>The Norwegian public–private partnership, CONNECT, identified the reimbursement submission case of selpercatinib—a first-line targeted therapy for RET fusion-positive non-small cell lung cancer (NSCLC)—as a unique opportunity to assess the role of structured expert elicitation (SEE) in conjunction with phase II evidence.</p> Objective <p>This study aims to compare the potential of using SEE and other sources of external evidence with phase III clinical trial results to explore the possibility of facilitating earlier (conditional) reimbursement decisions.</p> Methods <p>We conducted a series of cost-effectiveness analyses, grouping different sources of evidence (i.e., published clinical evidence and SEE) to estimate the long-term health and economic consequences of selpercatinib versus pembrolizumab plus pemetrexed and platinum-based chemotherapy for patients with advanced RET fusion-positive NSCLC in Norway. We developed a probabilistic partitioned survival model to estimate and compare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) associated with evidence potentially available early in the product lifecycle, including SEE, with those using phase III evidence.</p> Results <p>ICERs consistently ranged between $8257 and $19,819 per QALY gained when we used SEE, either alone or in combination with additional external evidence, or when we used the phase III randomized controlled trial (RCT) evidence.</p> Conclusions <p>The consistent cost-effectiveness results between pre-submission and post-submission phases support the potential of SEE to complement health technology assessment (HTA) and potentially inform earlier (conditional) reimbursement decisions for this single case study. However, future research should focus on refining SEE protocols for continued methodological development and validation to improve generalizability and applicability.</p>

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Integrating Structured Expert Elicitation with External Evidence to Inform Earlier Reimbursement Decisions: A Norwegian Case Study of Selpercatinib for Non-Small Cell Lung Cancer

  • Yancy S. Wu,
  • Emily A. Burger,
  • Fredrik Holmboe,
  • Magnus Bangum,
  • Francisco Oteiza,
  • Christoffer Bugge,
  • Erik Magnus Sæther,
  • Eline Aas

摘要

Background

The Norwegian public–private partnership, CONNECT, identified the reimbursement submission case of selpercatinib—a first-line targeted therapy for RET fusion-positive non-small cell lung cancer (NSCLC)—as a unique opportunity to assess the role of structured expert elicitation (SEE) in conjunction with phase II evidence.

Objective

This study aims to compare the potential of using SEE and other sources of external evidence with phase III clinical trial results to explore the possibility of facilitating earlier (conditional) reimbursement decisions.

Methods

We conducted a series of cost-effectiveness analyses, grouping different sources of evidence (i.e., published clinical evidence and SEE) to estimate the long-term health and economic consequences of selpercatinib versus pembrolizumab plus pemetrexed and platinum-based chemotherapy for patients with advanced RET fusion-positive NSCLC in Norway. We developed a probabilistic partitioned survival model to estimate and compare costs, quality-adjusted life-years (QALYs), and incremental cost-effectiveness ratios (ICERs) associated with evidence potentially available early in the product lifecycle, including SEE, with those using phase III evidence.

Results

ICERs consistently ranged between $8257 and $19,819 per QALY gained when we used SEE, either alone or in combination with additional external evidence, or when we used the phase III randomized controlled trial (RCT) evidence.

Conclusions

The consistent cost-effectiveness results between pre-submission and post-submission phases support the potential of SEE to complement health technology assessment (HTA) and potentially inform earlier (conditional) reimbursement decisions for this single case study. However, future research should focus on refining SEE protocols for continued methodological development and validation to improve generalizability and applicability.