The CADTH Therapeutic Review of treatments for transplant-ineligible multiple myeloma patients represents several firsts for the organization. It is CADTH’s first therapeutic review in oncology, and its first involving international collaboration. It also showcases many of the progressive aspects of CADTH’s therapeutic review process.
Multiple myeloma is a type of cancer that affects plasma cells, which help the immune system fight infections. It mainly affects patients who are 65 or older and while it remains incurable, patients can live with this cancer for many years and over time they may receive several different forms of treatment.
The treatment landscape for multiple myeloma is complex and continuously evolving, especially for the many patients who aren’t eligible for the preferred form of first-line treatment — high-dose chemotherapy followed by an autologous stem cell transplant. There are more than 20 combinations of therapies available for these transplant-ineligible patients that contribute to both survival and quality of life. The choice of treatment often depends on patient characteristics, personal preferences, experience with previous therapies and, importantly, which treatments are funded by regional cancer centres.
And while there is a wealth of scientific literature on the available treatments, head-to-head comparisons of first-line and subsequent-line therapies for transplant-ineligible patients are few. The result is often uncertainty among decision-makers about both their relative effectiveness and the optimal order (or “sequencing”) of treatments.
The CADTH Therapeutic Review will compare the clinical effectiveness and cost-effectiveness of treatments for transplant-ineligible multiple myeloma among patients who are newly diagnosed and those diagnosed with relapsed and/or refractory multiple myeloma. (Refractory means the cancer doesn’t improve with treatment, or it stops responding to treatment).
Notably, this is CADTH’s first Therapeutic Review to involve international collaboration. CADTH is working with the Erasmus School of Health Policy and Management (ESHPM) in the Netherlands on both the clinical and economic research. The ESHPM team is updating two of its existing network meta-analyses on this topic and, together with CADTH staff and Canadian clinical experts, will use real-world data from a Canadian database and the outcomes of the network meta-analyses to develop a living economic model on the cost-effectiveness of treatment sequences.
This decision to create a living economic model offers several important benefits. It means that new therapies can be incorporated into the model in a timely manner, as they become available in Canada. Further, this living economic model will assist payers as they look to manage their formularies and identify the most cost-effective treatment sequences.
In addition to international collaboration, Canada’s national multiple myeloma patient advocacy group is also contributing to the project. Myeloma Canada surveyed patients to complement patient group input that was submitted to CADTH as part of previous reimbursement reviews of multiple myeloma treatments.
A CADTH Therapeutic Review is an assessment of the publicly available evidence about a category or class of drugs. It involves:
In some instances, a therapeutic review may involve revisions to existing CADTH drug reimbursement recommendations, if applicable based on the evidentiary outcomes. Stakeholder feedback will be sought throughout the process via public calls for feedback. Consult our Therapeutic Review Framework and Process for more details.
Symptomatic multiple myeloma is an incurable plasma cell neoplasm with an estimated 3,400 new cases in Canada annually with a higher incidence in males. In Canada, the 5-year net survival rate for multiple myeloma is 44%.
The preferred first-line treatment for newly diagnosed patients with multiple myeloma is high-dose chemotherapy followed by autologous stem cell transplantation. However, the majority of patients are not eligible for this procedure due to underlying health risks. For these patients, a number of multi-drug regimens can be offered as first- or subsequent-line treatment. Many of these treatments have been reviewed by CADTH’s pan-Canadian Oncology Drug Review expert committee.