Sanofi wins appeal against negative Final Draft Guidance for its standard-of-care multiple myeloma combination therapy opening the door for a third NICE Committee Meeting

Press Release For medical and trade media only England and Wales

 

Sanofi wins appeal against negative Final Draft Guidance for its standard-of-care multiple myeloma combination therapy opening the door for a third NICE Committee Meeting

 

• Sanofi welcomes the decision as recognition from the National Institute for Health and Care Excellence (NICE) that there are challenges with the current framework for assessing and funding innovative combination therapies and there is a need for greater flexibility in certain situations

 

• As part of this extended evaluation process, Sanofi are calling for increased flexibility so that eligible new patients do not lose an important combination treatment for their relapsed and refractory multiple myeloma (RRMM) in the future

 

• Sanofi is committed to engaging further with NICE, NHS England, healthcare professionals, patients groups and other involved stakeholders to explore all options for ensuring the continued availability of isatuximab (SARCLISA ) plus pomalidomide and dexamethasone (Isa-Pd)

 

• Isa-Pd will continue to be available to newly eligible and existing patients in England and Wales through the Cancer Drugs Fund (CDF) whilst the appraisal process is still ongoing and until NICE publishes its Final Guidance

 

Reading, UK – 12 November 2024. Sanofi announced today that NICE has confirmed a third Committee Meeting to re-assess Isa-Pd at fourth-line, a subgroup of the population within the marketing authorisation, for treating RRMM in adults who have had lenalidomide and a proteasome inhibitor, and whose disease has progressed on their last treatment.[1] NICE’s decision follows a successful appeal of the negative Final Draft Guidance published in June this year.[2] The appeal committee upheld four points submitted by Sanofi, including the fact that under NICE’s standard methodology Isa-Pd cannot be deemed cost effective even if isatuximab is provided to the NHS at zero price (£0).¹ The committee also recognised NICE’s methods for evaluating medicines have changed in recent years, meaning Isa-Pd is being re-appraised for routine NHS commissioning against different criteria to which it entered the CDF.¹

 

Isa-Pd will continue to be available to eligible new patients who might benefit in England and Wales through the CDF, as well as those currently on this therapy whilst NICE’s evaluation is still ongoing and until Final Guidance is published. The combination is available to all eligible patients in Scotland. 

 

Isa-Pd has become a standard-of-care for eligible patients in England and Wales through its availability on the CDF, with over 1,700 patients registered to have started Isa-Pd treatment in England.[3] As a relapsing cancer, access to multiple lines of treatment is vital to delay progression for as long as possible.

 

Anju Bhalla

Head of Oncology and Haematology, Sanofi UK and Ireland

 

"We are encouraged by NICE’s decision to uphold our appeal, which will be welcomed by clinicians, patient groups and patients for whom the potential loss of a multiple myeloma treatment will be devastating. We are committed to maintaining access to Isa-Pd as an important fourth line option for RRMM patients who have limited alternatives at this later stage of their cancer and will work collaboratively with all involved stakeholders to find a solution. This appraisal has been protracted and complex for several reasons, but we remain hopeful of a positive outcome at the third time of sitting around the table together.”

 

Discussions haven’t reached a conclusion for Isa-Pd to date, highlighting the challenges with the current NICE framework for assessing and funding innovative combination therapies, which is struggling to potentially keep up with the pace of innovation. The consequence is negative recommendations that may not recognise the full value and benefits of combination therapies for patients. 

 

Shelagh McKinlay

Director of Research and Advocacy, Myeloma UK

 

“We have been fighting this decision for months and we are delighted to have won our appeal. The appeal panel felt, as do we, that the reimbursement of Isa-Pd requires further consideration and that NICE should have considered showing more flexibility in light of what were unique and unprecedented circumstances. We’re urging them to reconsider their decision and make this treatment available on the NHS as soon as possible. In practice, the new system has made it much harder to get treatments to patients who are nearing the end of their life. We will continue to make the case for Isa-Pd to be made available to those who need it. Every day counts when you’re living with myeloma because only one thing is certain: myeloma will always come back. This means patients need effective treatments now.”

 

England has fallen below comparable European countries when it comes to accessing cancer medicines and cancer survival rates in the UK are lagging as much as 25 years behind other parts of Europe.[4]^,[5] In terms of access to licensed and effective combination therapies, cancer patients in England and Wales are missing out compared to their European counterparts.⁴

 

Sanofi is grateful for the strong support from the clinical and patient community throughout this process and remains hopeful a way forward can be found.

 

About isatuximab  

Isatuximab is a monoclonal antibody that targets a specific epitope on the CD38 receptor on multiple myeloma cells.[6] It is designed to work through multiple mechanisms of action including programmed tumour cell death (apoptosis) and immunomodulatory activity.⁶ CD38 is highly and uniformly expressed on the surface of multiple myeloma cells, making it a target for antibody-based therapeutics such as isatuximab.⁶ Isa-Pd has been shown to offer improvements in response rates and progression-free survival, compared to Pd alone, with a manageable side effect profile.²

 

About ICARIA-MM 

The efficacy and safety of Isa-Pd was evaluated in ICARIA-MM, a multicentre, multinational, randomised, open-label, 2-arm, phase 3 study in patients with relapsed and/or refractory multiple myeloma.⁶ In this NICE appraisal, we provided data from the final analysis of ICARIA-MM for a subgroup of people who had received 3 previous lines of treatment:

 

• Median progression-free survival (PFS) [primary efficacy endpoint] in patients receiving Isa-Pd was 12.4 months versus 6.5 months for Pd alone.² The five-month improvement in PFS represented a 47.6% reduction in the risk of disease progression or death in patients treated with Isa-Pd, versus Pd alone (HR: 0.536).²
• Median overall survival (OS) was 33.3 months for Isa-Pd compared to 17.7 months in the Pd arm.²

 

The most common adverse events for Isa-Pd were neutropenia (46.7%), infusion reactions (38.2%), pneumonia (30.9%), upper respiratory tract infection (28.3%), diarrhoea (25.7%) and bronchitis (23.7%).⁶ Serious adverse reactions occurred in 61.8% of patients receiving Isa-Pd. The most frequent serious adverse reactions were pneumonia (25.7%) and febrile neutropenia (6.6%).⁶ Permanent discontinuation of treatment because of adverse reactions was reported in 7.2% of patients treated with Isa-Pd.⁶

 

About multiple myeloma  

Multiple myeloma is a progressive, incurable cancer of the blood plasma cells.[7] It affects around 24,000 individuals in the UK at any one time.⁷ There are an estimated 5,900 new cases of multiple myeloma in the UK every year, equivalent to 16 people being diagnosed each day.⁷ Treatment options become limited as patients make their way through them, as such ensuring as many options are available at each line of therapy is vitally important.⁷ Combinations are standard-of-care in multiple myeloma and have driven recent progress.[8]

 

About Sanofi

We are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and potentially life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the centre of our ambitions.

 

Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY

 

Contacts

Quiterie Mertian, Communications Manager, Specialty Care, Sanofi UK & Ireland

+44 (0) 7740 935 217| quiterie.mertian@sanofi.com

Simon Butler, Corporate Affairs Lead, Specialty Care, Sanofi UK & Ireland

+44 (0)7921 054 943| Simon.Butler@sanofi.com

 

References

 

[1] National Institute for Health and Care Excellence (NICE). Isatuximab with pomalidomide and dexamethasone for treating relapsed and refractory multiple myeloma [Review of TA658] [ID4067] Notice of Appeal. Available at: https://www.nice.org.uk/guidance/gid-ta10979/documents/html-content-5. Last accessed: November 2024

[2] National Institute for Health and Care Excellence (NICE). Available at: https://www.nice.org.uk/guidance/gid-ta10979/documents/html-content-4. Last accessed: November 2024

[3] Jim Shannon and Andrew Stephenson (2024) Multiple Myeloma: Drugs. UK Parliament: (UIN 15638). Available at: https://questions-statements.parliament.uk/written-questions/detail/2024-02-26/15638 Last accessed: November 2024

[4] European Federation of Pharmaceutical Industries and Associations. EFPIA Patients W.A.I.T. Indicator 2023 Survey. Available: https://efpia.eu/media/vtapbere/efpia-patient-wait-indicator-2024.pdf Last accessed: November 2024

[5] Macmillan Cancer Support. Decades behind and failing patients, UK cancer care is ‘stuck in the noughties’ Available at: https://www.macmillan.org.uk/healthcare-professionals/news-and-resources/blogs/cancer-care-decades-behind Last accessed: November 2024

[6] Electronic Medicines Compendium, SARCLISA 20mg/mL concentrate for solution for infusion. Available at: https://www.medicines.org.uk/emc/product/14817/smpc/print#PHARMACOLOGICAL_PROPS. Last accessed: November 2024

[7] Myeloma UK. What is myeloma? Available at: https://www.myeloma.org.uk/understanding-myeloma/what-is-myeloma/. Last accessed: November 2024

[8] Myeloma UK. Treatment for relapsed myeloma. Available at: https://www.myeloma.org.uk/understanding-myeloma/treating-myeloma/treatment-for-relapsed-myeloma/. Last accessed: November 2024