(A-024) Evaluating Clinical Benefit and Regulatory Approval Patterns of Newly Approved Therapies for Multiple Myeloma: Is Brazil Keeping Pace with Global Advances?

Background: Multiple myeloma (MM) is a rare and incurable hematological malignancy. Despite recent therapeutic advances improving survival, uncertainties remain regarding the clinical benefits and safety of new treatments.

Objectives: This study evaluates the clinical benefits of newly MM therapies, analyzing drug characteristics, regulatory approval patterns in Brazil and internationally, and the quality of supporting evidence.

Methods: This was a retrospective cohort study that included MM therapies approved between January 2003 and December 2024 by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Brazilian National Health Surveillance Agency (Anvisa). Therapies were identified through structured searches in PUBMED/MEDLINE and publicly available regulatory documents. Data collection included drug classification by Anatomical Therapeutic Chemical (ATC) code, origin, innovation status, regulatory designations, and incorporation into the Brazilian public health system. The European Society for Medical Oncology-Magnitude of Clinical Benefit Scale: Hematology version 1.1 (ESMO-MCBS: H v 1.1) was used to evaluate the clinical benefit of these therapies at the time of approval. Descriptive statistical analysis was performed using SPSS® 25.0. Kaplan-Meier survival curves were analyzed to compare approval times across agencies using the log-rank (Mantel-Cox) test.

Results: In Brazil, Anvisa approved 12 (70.6%) of the 17 internationally available MM therapies. Among them, 7 (58.3%) were biological, 5 (41.7%) were first-in-class, and 6 (50%) held Breakthrough Therapy designation. However, only 4 (23.5%) demonstrated substantial clinical benefit at approval in non-curative settings (ESMO-MCBS: H v1.1 score: 4)—two proteasome inhibitors (PI) and two bispecific monoclonal antibodies (BsMAb). Of these, only the proteasome inhibitors were incorporated into the public health system in Brazil. Compared to other agencies, the FDA and EMA scored BsMAb similarly (ESMO-MCBS: H v1.0 score: 4), while PI received lower scores. Anvisa approvals lagged behind FDA and EMA approvals by 19 and 11 months, respectively, with shorter review times in Brazil for biological drugs (p=0.008), monoclonal antibodies (p=0.035), and Breakthrough Therapy–designated treatments (p=0.031).

Conclusions: Brazil’s newly approved therapies reflect advancements in MM treatment, with increased immunotherapy use, but the incorporation into the public health system remains limited. Therapies demonstrating substantial benefit warrant prioritization for reimbursement to optimize resource allocation and reduce disparities in access.