Development of the ASH Research Collaborative Multiple Myeloma Program: A Multicenter, Real-World Evidence Generation Network

Background: Contemporary questions about treatment and outcomes of patients (pts) living with multiple myeloma (MM) demand efficient and timely evidence generation methods, where real-world/ everyday practice data can play an important role. Relevant potential applications include understanding and addressing disparities in treatment access, mitigating toxicities of novel therapies while maintaining quality of life, appropriately sequencing treatments within long-term management strategies, and disseminating and appropriately using surrogate endpoints in practice, such as minimal residual disease. However, existing real-world data (RWD) repositories are limited by an inability to implement interventions in response to issues identified through retrospective data analysis, which is pivotal for the use of newer therapies. The ASH Research Collaborative (ASH RC) MM Data Hub Program was developed to address these gaps.

Methods: We developed a multicenter network of US-based tertiary academic medical centers, engaging site clinicians, patients, biostatisticians, informaticians, and data scientists. Initial activities included constructing a shared real-world MM data model comprised of consensus core data elements. Pt data from each site, dating back to at least 2019, is currently being entered through electronic case report forms, batch uploaded utilizing existing institutional data repositories, and soon direct electronic health record import in standard data acquisition formats. Data are aggregated in the Data Hub and shared in a confidential Site Portal with each site. The Portal allows sites to track their own pts, analyze outcomes, and identify ways to improve the quality of care provided to individuals living with MM. We also developed an innovative evidence generation learning community of site clinicians who meet monthly and collaborate on the “COVID-19 OutcomeS in Myeloma and the Impact of VaCcines (COSMIC)” study, a prospective, decentralized observational study that rapidly accrued pts and collects relevant practice information. The group also shares best practices in study conduct to accelerate study enrollment and execution.

Results: The MM Data Hub Program now contains twelve centers: Memorial Sloan Kettering Cancer Center, Mayo Clinic, Icahn School of Medicine at Mount Sinai, University of California San Francisco, University of California Davis, Medical College of Wisconsin, Barbara Ann Karmanos Cancer Institute, University of North Carolina Medical Center at Chapel Hill, Emory University Hospital, Washington University School of Medicine in St. Louis, Dana-Farber Cancer Institute, and Atrium Health. From this Network, six centers have contributed data from 8,824 pts (2,825 with sufficiently complete data are included in this analysis). The median age at time of MM diagnosis is 63, and of the 627 deceased pts, the median age at death is 70.6 years, with median time to death from first MM diagnosis of 6 years. The proportions of pts by decade of diagnoses are 34.9% from 2020-2024; 57.6% from 2010-2019; 7.3% from 2000-2009; and 0.9% prior to 2000. 83.1% of pts had a diagnosis of plasma cell myeloma (non-light chain), and 14.4% of pts had a diagnosis of light chain myeloma. 42.3% of pts are female, and the self-reported patient races are: 85.0% white; 9.2% black or African; and 4.3% all other races (race was not documented in 1.5% of pts). 2.8% of pts are of Hispanic ethnicity. Participant data files are currently undergoing completion and cleaning procedures; the analysis will include updated results at the meeting, and a Network-wide analytic data set will be available for initial retrospective data analyses in early 2025. The COSMIC study opened for enrollment on January 22, 2024, and completed enrollment of 200 pts on May 17, 2024.

Conclusions: The ASH RC Data Hub MM Program launched in 2023 and is comprised of 12 sites that collectively have contributed participant files for 8,824 pts thus far. Utilizing the structure of the Network, we successfully implemented and established the feasibility of collecting data directly from pts electronically to monitor SARS-CoV-2 vaccines and COVID-19 related outcomes. The Program serves as a successful proof-of-concept model, providing an enduring RWD resource and a prospective evidence generation platform for the myeloma community.

Kumar:Abbvie: Membership on an entity's Board of Directors or advisory committees, Research Funding; Oncopeptides: Other: Independent review committee participation; Takeda: Membership on an entity's Board of Directors or advisory committees, Research Funding; Celgene: Membership on an entity's Board of Directors or advisory committees, Research Funding; Adaptive: Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi: Research Funding; Roche: Research Funding; Novartis: Research Funding; Merck: Research Funding; MedImmune/AstraZeneca: Membership on an entity's Board of Directors or advisory committees, Research Funding; KITE: Membership on an entity's Board of Directors or advisory committees, Research Funding; Janssen: Membership on an entity's Board of Directors or advisory committees, Research Funding. Wood:Teledoc Health: Consultancy; Koneksa Health: Consultancy, Current equity holder in publicly-traded company; Genetech: Research Funding; Pfizer: Research Funding; ASH Research Collaborative: Consultancy. Anderson:Dynamic Cell Therapies: Membership on an entity's Board of Directors or advisory committees; C4 Therapeutics: Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy; Pfizer: Consultancy; Amgen: Consultancy; AstraZeneca: Consultancy; Window: Membership on an entity's Board of Directors or advisory committees; Genentech: Consultancy; Starton Therapeutics: Membership on an entity's Board of Directors or advisory committees. Neuberg:Madrigal Pharmaceutical: Current equity holder in publicly-traded company. Schroeder:Incyte: Honoraria; Advarra: Honoraria; Kura Oncology: Honoraria. Rosenberg:Biomea: Research Funding; Pfizer: Consultancy; Sanofi: Membership on an entity's Board of Directors or advisory committees; BMS: Consultancy; Kangpu Pharmaceuticals: Research Funding. Zonder:BMS, Janssen, RLL: Research Funding; Regeneron: Consultancy; BMS (employment of spouse): Current Employment. Rubinstein:LAVA Therapeutics: Consultancy; Sanofi: Consultancy; Johnson & Johnson: Consultancy; Bristol Myers Squibb: Membership on an entity's Board of Directors or advisory committees, Other: Abemca. D'Souza:Abbvie: Research Funding; Bristol Myers Squibb: Consultancy, Membership on an entity's Board of Directors or advisory committees; Caelum: Research Funding; Janssen: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Kedrion: Membership on an entity's Board of Directors or advisory committees; Novartis: Research Funding; Pfizer: Membership on an entity's Board of Directors or advisory committees; Prothena: Consultancy, Membership on an entity's Board of Directors or advisory committees, Research Funding; Regeneron: Research Funding; Takeda: Research Funding. Martin:Roche: Honoraria; GSK: Honoraria; AMGEN: Research Funding; Janssen: Research Funding; BMS: Research Funding; Sanofi: Research Funding; Pfizer: Honoraria. Chari:Abbvie, Adaptive, Amgen, Antengene, Bristol Myers Squibb, Forus, Genetech/Roche, Glaxo Smith Klein, Janssen, Karyopharm, Millenium/Takeda, Sanofi/Genzyme: Consultancy; Janssen: Research Funding. Vij:Sanofi, BMS, Takeda: Other, Patents & Royalties; Janssen, Pfizer, GSK, Regeneron, Karyopharm: Other, Patents & Royalties. Nooka:Janssen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Sanofi: Honoraria, Membership on an entity's Board of Directors or advisory committees; Cellectis: Research Funding; Amgen: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; AstraZeneca: Honoraria, Membership on an entity's Board of Directors or advisory committees; Pfizer: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Bristol-Myers Squibb: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Aduro Biotech: Research Funding; Adaptive Biotechnologies: Honoraria, Membership on an entity's Board of Directors or advisory committees; Cellectar Biosciences: Honoraria, Membership on an entity's Board of Directors or advisory committees; GSK: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; ONK Therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Arch Oncology: Research Funding; Sebia: Honoraria, Membership on an entity's Board of Directors or advisory committees; Takeda: Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Genentech: Research Funding; K36 Therapeutics: Honoraria, Membership on an entity's Board of Directors or advisory committees; Karyopharm: Research Funding; Kite Pharma: Research Funding; Merck: Research Funding. Bhutani:Janssen: Research Funding; Amgen: Research Funding; BMS: Research Funding; Takeda: Research Funding; Caribou Biosciences: Research Funding; Abvvie: Research Funding. Voorhees:Abbvie: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; BMS: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; AstraZeneca: Consultancy; Karyopharm: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Regeneron: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees, Research Funding; Lava Therapeutics: Consultancy; Sanofi: Consultancy, Honoraria, Membership on an entity's Board of Directors or advisory committees; Janssen: Consultancy, Research Funding; GSK: Consultancy, Research Funding. Sperling:Novartis: Consultancy. Usmani:EdoPharma: Consultancy; Merck: Research Funding; TeneoBio: Consultancy; Bristol-Myers Squibb: Consultancy, Research Funding; Bristol-Myers Squibb - Celgene:: Consultancy, Research Funding; Sanofi: Consultancy, Research Funding; Oncopeptides: Consultancy; Abbvie: Consultancy, Research Funding; SkylineDX: Consultancy, Research Funding; GSK: Consultancy, Research Funding; Pharmacyclics: Research Funding; Gracell: Consultancy; Sanofi: Consultancy, Research Funding; Pfizer: Consultancy; Genentech: Consultancy; Gilead: Research Funding; Amgen: Consultancy, Research Funding; Array Biopharma: Research Funding; SeaGen: Consultancy, Research Funding; SecuraBio: Consultancy; Takeda: Consultancy, Research Funding; Bristol-Myers Squibb - Celgene: Consultancy, Research Funding; Johnson & Johnson - Janssen: Consultancy, Research Funding.