The National Institute for Health and Care Excellence (NICE) has approved Belantamab Mafodotin, marking a significant advancement in the treatment of multiple myeloma in England. This decision positions the country as the first to offer this innovative therapy for patients who have undergone one prior treatment containing lenalidomide and are either refractory to or cannot tolerate it.

Context and Background

Multiple myeloma, a chronic cancer affecting plasma cells, presents challenges with its relapsing and remitting nature. It often leads to severe complications such as infections and kidney damage. With approximately 1,500 patients in England set to benefit, the need for effective second-line therapies remains critical. Historically, treatment options at this stage included combinations like daratumumab with bortezomib and dexamethasone or carfilzomib-based regimens. However, resistance and tolerability issues have highlighted an unmet need for novel treatments.

Belantamab Mafodotin, an antibody-drug conjugate, targets the BCMA protein on myeloma cells, delivering a cancer-killing agent directly to the tumour. The DREAMM-7 trial provided compelling evidence, showing that 71% of patients treated with Belantamab Mafodotin remained progression-free after one year, compared to 51% on standard care. Furthermore, preliminary data suggest a three-year survival rate of 74% versus 60% for standard care, though long-term outcomes are still under investigation.

Key Analysis and Insights

Clinical Innovation and Patient Impact

• Belantamab Mafodotin’s targeted mechanism offers precision by focusing on BCMA, minimising off-target effects and providing a new option for patients with limited alternatives.
• The DREAMM-7 trial results are promising, with significant improvements in progression-free survival (PFS) and overall survival (OS) compared to daratumumab-based regimens. However, indirect comparisons with other second-line therapies, such as selinexor or carfilzomib combinations, introduce uncertainty due to methodological limitations.
• Ocular adverse events, such as keratopathy, are a concern, with a higher incidence in the Belantamab Mafodotin arm. While patients and clinicians report these effects as manageable, the need for ophthalmic monitoring poses logistical challenges for healthcare systems.

Economic Considerations and Market Access

• NICE’s evaluation found Belantamab Mafodotin to be cost-effective compared to daratumumab-based regimens when lenalidomide is not an option, with incremental cost-effectiveness ratios (ICERs) within the acceptable range of £20,000–£30,000 per quality-adjusted life year (QALY). However, it was less cost-effective against other comparators like selinexor or carfilzomib with lenalidomide.
• A confidential discount through a patient access scheme has made the therapy viable for NHS funding, illustrating the role of innovative pricing models in balancing access and affordability.
• The requirement for ophthalmology monitoring, particularly in the first four cycles, adds to NHS resource burdens. With existing strain on ophthalmology departments, implementation delays are likely unless community-based solutions, as proposed by the manufacturer, are scaled effectively.

Policy and System Dynamics

• NICE’s recommendation limits Belantamab Mafodotin to second-line treatment for lenalidomide-refractory or intolerant patients, raising concerns about access inequities for those at later lines or without prior lenalidomide exposure. This decision reflects the tension between cost-effectiveness evidence and broader unmet needs.
• The rapid evolution of multiple myeloma therapies, with recent approvals like daratumumab with lenalidomide at first line, complicates second-line positioning. Policymakers must anticipate shifts in treatment sequencing to ensure equitable access.
• England’s early adoption of Belantamab Mafodotin sets a precedent for other healthcare systems. However, regional differences in funding models, regulatory timelines, and healthcare infrastructure may delay uptake elsewhere, particularly in resource-constrained settings.

Implications and Recommendations

The approval of Belantamab Mafodotin has significant implications for health economics, system dynamics, and policy. For senior decision-makers, the following insights and recommendations are crucial:

• Healthcare systems must prioritise partnerships with community ophthalmology services to mitigate monitoring burdens. Investment in training and capacity building will be essential to avoid delays in treatment rollout.
• Manufacturers and payers should continue to explore innovative pricing models, such as outcomes-based agreements, to ensure therapies like Belantamab Mafodotin remain accessible while maintaining fiscal sustainability.
• NICE and similar bodies globally should adopt flexible evaluation frameworks that account for evolving treatment landscapes. Expanding recommendations beyond restricted populations, where evidence permits, could address equity concerns.
• Long-term data from the DREAMM-7 trial and real-world evidence via the Cancer Drugs Fund will be vital to confirm survival benefits and refine cost-effectiveness estimates. Stakeholders should advocate for robust data-sharing initiatives.

Conclusion

NICE’s recommendation of Belantamab Mafodotin marks a significant step forward in the treatment of multiple myeloma, offering hope to patients with limited options while showcasing the potential of targeted therapies. However, its implementation raises complex challenges around cost, resource allocation, and equitable access. By addressing these issues through strategic partnerships, innovative pricing, and adaptive policies, healthcare systems can maximise the impact of such breakthroughs. As the global healthcare community watches England’s rollout, this approval serves as a call to action for collaborative efforts to ensure that innovation translates into tangible patient benefits.

Sources

NICE leads the way in approving breakthrough treatment for multiple myeloma [Internet]. NICE website: The National Institute for Health and Care Excellence. NICE; 2025 [cited 2025 Jun 16]. Available from: https://www.nice.org.uk/news/articles/nice-leads-the-way-in-approving-breakthrough-treatment-for-multiple-myeloma.

Consultation | Belantamab mafodotin with bortezomib and dexamethasone for treating relapsed or refractory multiple myeloma after 1 or more treatments [ID6212] | Guidance | NICE [Internet]. NICE; 2025 [cited 2025 Jun 16]. Available from: https://www.nice.org.uk/guidance/indevelopment/gid-ta11203/consultation/html-content-4.

Blenrep (belantamab mafodotin) combinations approved by UK MHRA in relapsed/refractory multiple myeloma | GSK [Internet]. 2025 [cited 2025 Jun 16]. Available from: https://www.gsk.com/en-gb/media/press-releases/blenrep-belantamab-mafodotin-combinations-approved-by-uk-mhra-in-relapsedrefractory-multiple-myeloma/.