Daratumumab for Newly Diagnosed Multiple Myeloma Patients: Addressing Ambivalence in Unmet Needs

OBJECTIVES: National Institute for Value and Technologies in Healthcare (NIHO) assessed the drug Darzalex (daratumumab) for treating patients newly diagnosed with multiple myeloma (MM) in combination with lenalidomide and dexamethasone (DaraRd). According to EHA-ESMO Clinical Practice Guidelines, the first option therapies for patients not eligible for autologous stem cell transplantation (ASCT) include DaraRd, daratumumab in combination with bortezomib, melphalan, and prednison (DaraVMP), and bortezomib with lenalidomide and dexamethasone (VRd).

METHODS: NIHO considered DaraVMP, VRd, bortezomib, melphalan, and prednison (VMP), bortezomib with cyclophosphamide and dexamethasone (VCd), and bortezomib with dexamethasone (Vd) as comparators, as these treatment regimens were categorized and used in clinical practice. Professional input was collected via questionnaires from three clinical experts in hemato-oncology. All three experts endorsed categorizing Darzalex in the DaraRd combination, citing superior clinical trial results and an unmet need for innovative treatments.

RESULTS: Based on network meta-analysis (NMA) results, NIHO considered the efficacy of DaraRd to be comparable to the efficacy of DaraVMP and VRd. NIHO recommended not extending the indication restriction for Darzalex in the DaraRd combination for newly diagnosed MM patients unsuitable for ASCT unless the reimbursed price is adjusted. Therapeutic combination DaraRd was added to reimbursement list in the assessed indication by appraisal committee.

CONCLUSIONS: Clinical experts in Slovakia are accustomed to declaring an unmet needs and lack of innovative treatment options. While the clinicians' desire to provide the best possible treatment is understandable, the urgency was mitigated by the availability of two out of the three guideline-recommended regimens. In health technology assessment, professional opinions are invaluable but should be viewed considering possible bias.