Policy approach to optimize multiple myeloma treatment sequencing practices

As a consequence of major therapeutic advances that have transformed multiple myeloma (MM) into a manageable chronic condition, treatment sequencing has become highly complex. According to current practices, novel therapies are predominantly reserved for later disease stages, yet limited evidence suggests that this approach yields optimal long-term outcomes. Understanding the drivers of current practices and identifying strategies to strengthen evidence-informed sequencing are therefore critical.

This paper synthesizes insights from a multi-stakeholder discussion involving patient representatives, clinical experts, health technology assessment (HTA) specialists, and industry representatives. Drivers of current sequencing practices include constraints of clinical development pathways, limitations of guideline development, methodological challenges within HTA, financial pressures, and the high disease burden in advanced disease stages. Several options for optimizing treatment sequencing have been explored, including new evidence-generation approaches, whole-disease modeling, meaningful patient involvement, and expanding evaluation frameworks.

Uncertainty plays a central role in shaping sequencing practices in MM, underscoring the need for better evidence to guide decisions and avoid suboptimal use of treatment options. As the therapeutic landscape continues to expand, sequencing will be even more complex, which necessitates international coordinated efforts. The multi-stakeholder discussion presented here represents an initial step toward enabling such joint actions.