Comparative modeling of multiple myeloma across myeloma continuum: prevention, treatment, and health drivers

Multiple myeloma (MM) is a common and lethal hematologic malignancy. Treatments of MM have been rapidly evolving. While these new treatments improve survival considerably, the median survival still ranges from 43-83 months at diagnosis. Among all cancer sites, the management of MM is the costliest, which in part can be attributable to guideline recommended multidrug regimens.

Despite such significant health and economic burdens and rapid changing landscape for MM treatments, MM is not one of the cancer sites in the Cancer Intervention and Surveillance Modeling Network (CISNET). Therefore, MM lacks comparative modeling to set goals and policy prioritization in MM prevention and control. Moreover, unlike breast cancer or colorectal cancer, there exists no population-based screening for MM or risk managed strategies for those with premalignant conditions (MGUS and smoldering MM). MM requires comparative modeling to evaluate promising intervention strategies, particularly at premalignant stages.

To prevent/control this devastating disease, it is imperative to demonstrate the potential benefits and harms of the novel interventions before implementation. It is also important to demonstrate the potential benefits and harms of de-implementation of the existing interventions, in lieu of the novel ones. This Incubator Program will include two modeling groups to conduct comparative modeling under the coordination of the coordinating center. Our Program will evaluate novel strategies in preventing or treating MM with the goal of reducing the health and economic burden of MM.

We plan to comparatively build, calibrate, and validate evidence-based MM modeling across the MM care continuum (Aim 1). Using the proposed comparatively modeling, we will (1) assess the impacts of novel MM prevention strategies in high-risk patients diagnosed with MGUS (Aim 2); (2) evaluate the cost-effectiveness of novel treatment regimens as well as guideline-recommended treatments in patients diagnosed with MM (Aim 3); and (3) assess whether, under what conditions, and in which ways the goal of minimizing health and/or economic burden can be achieved through the proposed novel intervention strategies (Aim 4). The proposed MM Incubator Program is significant in its capability to 1) build evidence-based comparative modeling for MM, a disease area that lacks of such modeling, relative to the areas of solid tumors already with such modeling, to guide interventions and policies; 2) provide evidence-based evaluation before implementation of any costly clinical trial or de-implementation of any outdated intervention; 3) explore novel interventions/treatments at various stages of MM; and 4) examine the value of guideline-recommended therapies, providing evidence to inform changes in guidelines and thus a shift in current clinical practice of MGUS and MM management. The proposed intervention strategies for MGUS and MM patients are innovative, with the goals to prevent and control MM.

Successful completion of this study will provide evidence in tangible metrics to urge a paradigm shift from current MGUS/MM management. It is therefore a vital step to move the field forward.

Grant Number: 5U01CA265735-05
NIH Institute/Center: NIH
Principal Investigator: Su-Hsin Chang