DISCREPANCIES BETWEEN GUIDELINE RECOMMENDATIONS AND REGULATORY APPROVALS IN MULTIPLE MYELOMA - IMPORTANCE OF EVIDENCE STRATIFICATION OF A LIVING SYSTEMATIC LITERATURE REVIEW (SLR)
Author(s)
Mihaela Musat, PhD1, Jessicca Rege, PhD1, Saro Sarkisian, MD, MHA2, Rozee Liu, MSc1, Anna Forsythe, MBA, MSc, PharmD1;
1Oncoscope-AI, Miami, FL, USA, 2Frederick Health, Frederick, MD, USA
1Oncoscope-AI, Miami, FL, USA, 2Frederick Health, Frederick, MD, USA
OBJECTIVES: Treatment guidelines in oncology summarize the clinical benefit from clinical trials or real-world evidence aiming to highlight all available treatment options based on the most robust data. However, some of the guideline recommended therapies lack specific regulatory endorsement, leading to reimbursement challenges and uncertainty around safety and quality data for off-label use. We sought to understand the gap between guideline recommendations and regulatory approvals in the rapidly evolving landscape of combination therapies in multiple myeloma, through a living SLR.
METHODS: A REal-time AI-assisted Living SLR (REAL-SLR), compliant with Cochrane and PRISMA guidelines was conducted in multiple myeloma. Clinical trials published in English were identified from PubMed and major conferences proceedings and assessed using Population, Intervention/Comparators, Outcomes, and Study design (PI/COS) framework. Studies were stratified by treatment/disease pathways, interventions, outcomes and subgroups categories.
RESULTS: The daily-updated REAL-SLR, contained 709 studies in multiple myeloma as of January 5, 2026. Studies were stratified based on clinical stage (newly diagnosed-300 studies, relapsed/refractory-410 studies, smoldering-20 studies), risk/cytogenetic profile, treatment path, prior treatment exposure, transplant eligibility or treatment refractoriness, and intervention category. The stratification allowed mapping against major guidelines and regulatory approvals. Among 266 studies involving pharmacological treatments recommended by USA guidelines, only 153 studies (58%) included Food and Drug Administration approved therapies. A different trend was observed for European guidelines. Among 144 studies including treatments recommended by European Hematology Association (EHA), majority (85%, 122 studies) included European Medicines Agency approved therapies. In addition, 38 studies investigated therapies not referred to in the EHA guideline.
CONCLUSIONS: Our REAL-SLR revealed a large gap between regulatory approval status and guideline advice for multiple myeloma treatment algorithms in the US. The stratification of REAL-SLR based on clinical pathways and real-time literature monitoring are critical for providing the most relevant evidence to inform decision-making.
METHODS: A REal-time AI-assisted Living SLR (REAL-SLR), compliant with Cochrane and PRISMA guidelines was conducted in multiple myeloma. Clinical trials published in English were identified from PubMed and major conferences proceedings and assessed using Population, Intervention/Comparators, Outcomes, and Study design (PI/COS) framework. Studies were stratified by treatment/disease pathways, interventions, outcomes and subgroups categories.
RESULTS: The daily-updated REAL-SLR, contained 709 studies in multiple myeloma as of January 5, 2026. Studies were stratified based on clinical stage (newly diagnosed-300 studies, relapsed/refractory-410 studies, smoldering-20 studies), risk/cytogenetic profile, treatment path, prior treatment exposure, transplant eligibility or treatment refractoriness, and intervention category. The stratification allowed mapping against major guidelines and regulatory approvals. Among 266 studies involving pharmacological treatments recommended by USA guidelines, only 153 studies (58%) included Food and Drug Administration approved therapies. A different trend was observed for European guidelines. Among 144 studies including treatments recommended by European Hematology Association (EHA), majority (85%, 122 studies) included European Medicines Agency approved therapies. In addition, 38 studies investigated therapies not referred to in the EHA guideline.
CONCLUSIONS: Our REAL-SLR revealed a large gap between regulatory approval status and guideline advice for multiple myeloma treatment algorithms in the US. The stratification of REAL-SLR based on clinical pathways and real-time literature monitoring are critical for providing the most relevant evidence to inform decision-making.
Conference/Value in Health Info
2026-05, ISPOR 2026, Philadelphia, PA, USA
Value in Health, Volume 29, Issue S6
Code
HSD36
Topic
Health Service Delivery & Process of Care
Disease
SDC: Oncology