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. 2025 Apr 16:19:1089-1104.
doi: 10.2147/PPA.S496106. eCollection 2025.

Perspectives of Healthcare Providers and Patients with Relapsed/Refractory Multiple Myeloma on Treatment Priorities and Novel Therapies

Sikander Ailawadhi  1 , Yelak Biru  2 , Solène Clavreul  3 , Maite San Miguel  4 , Nicolas Cormier  5 , Yvonne Efebera  6 , Maximilian Merz  7 , Anna Sato  8 , Cathy Zeanah  9 , Jack L Watkins  10 , James Farrell  11 , Erinn Hoag Goldman  10 , Rakesh Popat  12
Affiliations

Perspectives of Healthcare Providers and Patients with Relapsed/Refractory Multiple Myeloma on Treatment Priorities and Novel Therapies

Sikander Ailawadhi et al. Patient Prefer Adherence. .

Erratum in

Abstract

Purpose: With novel therapies including chimeric antigen receptor T-cell (CAR-T) therapy and bispecific antibodies (BsAbs), healthcare providers (HCPs) face complexities managing treatment for patients with relapsed/refractory multiple myeloma (RRMM). This study, among the largest surveys on RRMM, examined unmet needs in care access, barriers to novel therapy use, and treatment decision-making.

Methods: This survey-based study (March-June 2024) enrolled 2284 participants (patients: 1301; HCPs: 983) across 7 countries. Patients with >1 relapse/progression and HCPs managing ≥3 patients were included. Data were analyzed using descriptive statistics and Χ2 tests.

Results: For patients, treatment priorities included slowing disease progression (second line [2L], 47%; third or later line [≥3L], 49%), minimizing adverse events (AEs; 2L, 43%; ≥3L, 49%), and extending life (2L, 39%; ≥3L, 38%). HCPs prioritized prolonging survival and controlling disease. Younger patients (<65 vs ≥65 years) prioritized convenience (40% vs 24%; P<0.01) and avoiding referrals to new institutions for therapies (32% vs 20%; P<0.01). Across geographies, HCPs reported logistical challenges as key reasons that CAR-T (38%) or BsAb (34%) therapy was not offered. Novel therapies were offered to patients more frequently in the US vs EU (CAR-T, 84% vs 77%, P=0.023; BsAbs, 84% vs 76%, P=0.011), with a similar trend in the US vs Japan for CAR-T; however, across all geographies, few patients recalled being offered CAR-T (17%) or BsAbs (13%). Patients receiving BsAbs prioritized efficacy-related reasons (25-35%) and nonclinical factors like less time and financial impact (27-29%), whereas those who received CAR-T prioritized patient success stories (50%), efficacy-related factors (48-50%), and minimal financial burden (43%).

Conclusion: This study revealed gaps in treatment priorities; patients valued quality of life and AE management, while HCPs focused on efficacy and delaying progression. There is a significant need to educate HCPs and patients on the impact of shared decision-making when considering novel treatments for RRMM.

Keywords: CAR-T therapy; bispecific antibodies; patient perspectives; surveys.

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Conflict of interest statement

SA reports honoraria from Cellectar; consulting or advising roles for Amgen, BeiGene, Bristol Myers Squibb, Cellectar, GSK, Johnson & Johnson, Regeneron, Pfizer, Sanofi, and Takeda; and research funding from AbbVie, Amgen, Ascentage, Bristol Myers Squibb, Cellectar, Genentech, GSK, Janssen, Johnson & Johnson, Pharmacyclics, Sanofi, and Xencor. SC reports honoraria paid to institution from Pfizer; grants paid to institution from AbbVie, Amgen, Alexion, AstraZeneca, BeiGene, Binding Site, Bristol Myers Squibb, Janssen, Kite Gilead, Novartis, Oncopeptides, Pfizer, Regeneron, Roche, Sanofi, Menarini Stemline, Takeda, Sebia, Prothena, SkylineDx, and Sandoz, outside the submitted work. MSM reports honoraria from and consulting or advising roles for AbbVie, Bristol Myers Squibb, GSK, Janssen, Novartis, Pfizer, Roche, Sanofi, Stemline, and Takeda. NC reports honoraria and research funding from Bristol Myers Squibb, GSK, Janssen, Oncopeptides, Pfizer, Sanofi, and Takeda; consulting or advising roles for Bristol Myers Squibb, Genentech, Janssen, Merck Serono, Pfizer, Roche, and Servier; and speaker bureau roles for Bristol Myers Squibb, GSK, Janssen, Oncopeptides, Pfizer, Sanofi, and Takeda. MM reports personal fees from Janssen, AMGEN, Pfizer, BMS/Celgene, Takeda, AbbVie, Sanofi, and Roche/Genentech, outside the submitted work. AS and CZ report employment with ZS Associates, which was a paid consultant to Pfizer in connection with the development of this manuscript. JW, JF, and EG report employment and stock ownership with Pfizer. RP reports honoraria from AbbVie, Bristol Myers Squibb, GSK, Johnson & Johnson, Pfizer, Roche, and Sanofi; consulting or advising roles for GSK and Pfizer; speaker bureau roles for Pfizer; and research funding from GSK and Pfizer. RP is supported by the National Institute for Health and Care Research University College London Hospitals Biomedical Research Centre. The authors report no other conflicts of interest in this work.

Figures

Figure 1
Figure 1
Patient priorities for treatment decisions. All values are expressed as percentages. EU includes France, Germany, Italy, Spain, and the UK.
Figure 2
Figure 2
HCP priorities for treatment decisions. All values expressed as a percentage. EU includes France, Germany, Italy, Spain, and the UK.
Figure 3
Figure 3
Challenging AEs for patients with RRMM according to HCPs and patients with RRMM who had experienced these treatment-related AEs.
Figure 4
Figure 4
(A) Patient awareness of novel therapies. (B) Patients eligible and offered novel therapies. (C) HCP confidence in determining RRMM patient eligibility for novel therapies.
Figure 5
Figure 5
(A) Reasons why patients accepted BsAb therapy. (B) Reasons why HCPs did not offer BsAbs to all eligible patients with RRMM. (C) Reasons why patients declined BsAb therapy.
Figure 6
Figure 6
(A) Reasons why patients accepted CAR-T therapy. (B) Reasons why HCPs did not offer CAR-T therapy to all eligible patients with RRMM. (C) Reasons why patients declined CAR-T therapy.
See this image and copyright information in PMC

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