Key Takeaways
- NICE is consulting on draft guidance that currently does not recommend elacestrant for advanced breast cancer due to uncertainty in clinical evidence.
- The consultation focuses on treating oestrogen receptor-positive, HER2-negative breast cancer with an ESR1 mutation after disease progression.
- NICE is working closely with Menarini Stemline to resolve the uncertainty ahead of the next review in November 2024.
The National Institute for Health and Care Excellence (NICE) has initiated a public consultation on its draft guidance regarding elacestrant (KORSERDU), a new targeted therapy for advanced breast cancer. The draft, issued on October 1, 2024, outlines concerns about the uncertainty of the clinical evidence supporting the use of elacestrant for treating oestrogen receptor-positive, human epidermal growth factor receptor 2-negative (HER2-negative), locally advanced or metastatic breast cancer with an activating ESR1 mutation. The guidance is open for consultation until October 22, 2024, giving stakeholders an opportunity to provide input.
Elacestrant, developed by Menarini Stemline, is the first targeted treatment for patients with ESR1-mutated advanced breast cancer, a group that has seen limited options following progression after hormone therapy and CDK 4/6 inhibitors. Despite early promising data indicating that elacestrant may delay disease progression compared to standard care, NICE has expressed concern over the reliability of the estimates, prompting the need for further analysis before any final recommendations can be made.
Helen Knight, Director of Health Technology at NICE, emphasized the importance of ensuring that all uncertainties are addressed: “We stand ready to work with the company to try and resolve the issues identified by the committee. This is critical in determining whether elacestrant can provide meaningful benefits for patients facing the challenges of advanced breast cancer.”
The Role of Elacestrant in Advanced Breast Cancer Treatment
Elacestrant is a selective oestrogen receptor degrader (SERD) designed to target oestrogen receptor-positive breast cancers with ESR1 mutations, which develop in up to 50% of patients whose disease has progressed after endocrine therapy. By binding to and degrading the oestrogen receptors, elacestrant interrupts the cancer’s ability to use oestrogen as a growth signal, offering a potential treatment pathway for patients whose cancers have become resistant to other hormonal therapies.
Despite the potential benefits, the NICE committee has raised concerns regarding the reliability of the evidence, particularly how long elacestrant can prevent disease progression when compared to current clinical practice. This uncertainty complicates the assessment of whether a severity modifier—an adjustment to reflect the increased importance of benefits for severe diseases—can be applied in this case.
NICE has requested additional analyses and clarification from Menarini Stemline ahead of a further committee review scheduled for November 12, 2024. This step is vital to determine whether the clinical and economic uncertainties can be resolved, allowing elacestrant to be considered a valuable treatment option for patients with advanced breast cancer.
Impact on Patients and the Path Forward
If approved, elacestrant could offer new hope to around 1,000 patients in the UK who have limited treatment options due to the presence of the ESR1 mutation, which drives resistance to conventional hormone therapies. For these patients, progression to metastatic disease often results in substantial distress and reduced quality of life, underscoring the importance of ensuring that any new treatments are effective and can meaningfully extend progression-free survival.
NICE’s rigorous assessment process ensures that new therapies not only deliver clinical benefit but also provide value for the healthcare system. With three-quarters (76%) of treatments initially rejected in draft guidance ultimately receiving approval following further evidence review, there is optimism that, through collaboration with Menarini Stemline, the uncertainties surrounding elacestrant can be addressed before final guidance is published.
Helen Knight noted the agency’s proactive approach: “During the 2023-24 period, 76% of topics initially not recommended at the draft stage turned positive before final guidance. We are committed to working closely with all stakeholders to ensure that every opportunity to resolve uncertainties is fully explored.”
Conclusion and Next Steps
As NICE continues to consult with Menarini Stemline and other stakeholders, the final decision on whether elacestrant will be recommended for use in the NHS is expected later this year. The public consultation phase will remain open until October 22, 2024, allowing for broader input, after which the evidence will be further reviewed during the November committee meeting.
While this draft guidance does not yet recommend elacestrant, there remains a strong possibility that, with additional clarification and analysis, the therapy could become a key part of the treatment landscape for ESR1-mutated advanced breast cancer. Should the uncertainties be addressed, elacestrant may offer an essential option to delay disease progression for many patients, improving both survival and quality of life.
Resource: The National Institute for Health and Care Excellence, October 01, 2024
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