OBJECTIVES: Innovative therapies for treating multiple myeloma (MM) face various value demonstration and value recognition challenges, which may lead to delays in reimbursement and patient access. We sought to evaluate the current state of play related to these challenges within an HTA context and provide recommendations for overcoming these challenges.

METHODS: We conducted a targeted literature review to identify the main challenges and performed case studies in Canada, France, Germany, and the United Kingdom to evaluate the assessment of therapies in practice and methods to manage uncertainty. Based on this, we propose recommendations for manufacturers and HTA bodies to optimize evidence-based and timely access to new MM therapies.

RESULTS: The key value demonstration and recognition challenges relate to the large number of potential comparator treatments, generalizability of clinical trial results, clinical trial design, equity of patient access, value attribution in combination therapies and subsequent indications, use of surrogate endpoints, and suitability of outcome measures. The national case studies showed considerable heterogeneity in the HTA outcomes of MM therapies over the last decade. Methods used in practice to manage uncertainty included managed entry agreements, coverage with evidence development, use of real-world evidence (RWE) and external comparator data. Solutions identified include evaluating therapies by treatment class exposure rather than line of therapy and employing external control arms using RWE in the absence of phase III trial data.

CONCLUSIONS: Various challenges exist in generating good-quality evidence for MM therapies. Many of these are shared with other therapy areas, but their combination can make value demonstration and recognition of MM therapies particularly challenging. Addressing them requires a mix of approaches dealing with unique aspects of MM therapies and solutions that can also apply to other innovative therapies, ensuring consistency in HTA and decision-making is not unduly compromised.