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NICE ASSESSMENT OUTCOMES | 2022 United Kingdom | Healthcare | Pharma | News | Blogs | iPharm

December 15, 2022

SOBI's Doptelet was recommended for treating refractory primary chronic immune thrombocytopenia.

NICE recommended using Doptelet (avatrombopag) for treating adults with primary chronic immune thrombocytopenia (ITP) who are refractory to other treatments.

The recommendation was based on Study 302, a 26-week, Phase 3 double-blind, placebo-controlled study. The primary endpoint is the median number of weeks of platelet response (50×10 per liter or more), measured over 26 weeks. The cumulative number of weeks with platelet response was 12.4 weeks in patients on Doptelet versus 0 weeks in patients on placebo.

Also read: FDA Drug Approvals

SOBI submitted the Markov cohort model considering four health states, active treatment, responder, no treatment no response, and death. NICE agreed that the economic model submitted by SOBI was appropriate to determine the cost-effectiveness. NICE considered that Doptelet was cost-effective for primary chronic ITP in adults.

SOBI agreed to a commercial agreement with NHS to support the recommendation.


December 20, 2022

NICE's draft guidance recommended Daiichi Sankyo's Enhertu for breast cancer.

NICE's draft guidance recommended using Daiichi Sankyo's Enhertu (trastuzumab deruxtecan) for treating adult patients with unresectable or metastatic HER2-positive breast cancer who were previously treated with anti-HER2-based regiments. The product was recommended with managed access.

There is a high unmet need in patients previously treated with trastuzumab and a taxane.

Also read: India healthcare system

Daiichi Sankyo submitted a Phase 3 trial of DESTINY-Breast03, in which patients were recruited in 169 centers across 15 countries. The primary endpoint was progression-free survival, and overall survival was the secondary endpoint. The median progression-free survival was not reached in the Enhertu arm versus 6.8 months in patients on the trastuzumab emtansine arm, demonstrating a significant improvement in the progression-free survival.

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The company submitted a partitioned survival model to demonstrate the cost-effectiveness of trastuzumab deruxtecan versus trastuzumab emtansine. The company submitted Weibull models to Kaplan–Meier curves to estimate the progression-free survival, and the overall survival was extrapolated in the base case using parametric survival models.

NICE considered that Enhertu could not be cost-effective within routine commissioning.


October 19, 2022

BeiGene's Brukinsa was recommended for Waldenstrom's macroglobulinemia.

BeiGene's Brukinsa (zanubrutinib) was recommended for treating adults with Waldenstrom's macroglobulinemia. The drug is indicated for patients treated with at least one treatment and only if bendamustine plus rituximab is suitable.

Waldenstrom's macroglobulinemia is a type of non-Hodgkin's lymphoma that usually occurs in adults, with a median overall survival of 16 years in patients with symptoms. Patients typically get treated with bendamustine plus rituximab combination, and rituximab, cyclophosphamide, and dexamethasone combination as first-line therapy. NICE agreed that there is a high unmet need for oral, effective, and well-tolerated treatment options.

BeiGene submitted the ASPEN trial, in which the efficacy of Brukinsa was evaluated versus ibrutinib. The partial response was 28.4% in Brukinsa's arm versus 19.2% in the ibrutinib arm. NICE agreed it was clinically effective; however, progression-free survival and overall survival data were immature. Further, it agreed that Brukinsa is clinically effective compared with rituximab.

Further, NICE agreed that the cost-effectiveness model is appropriate for decision-making. The cost-effectiveness was less than £30,000 per QALY if Brukinsa was administered after at least one therapy compared with the rituximab plus bendamustine combination.

BeiGene has agreed to provide a discount to NHS; the discount level is confidential.


October 17, 2022

Keytruda was recommended as an adjuvant treatment for the most common type of kidney cancer.

Keytruda was recommended as an adjuvant treatment for renal cell carcinoma. The drug is approved for patients at risk of recurrence post nephrectomy, with or without metastatic lesion resection.

Renal cell carcinoma is the most common type of kidney cancer, accounting for 80% of kidney cancer cases. NICE agreed that there is a high unmet need for adjuvant treatment options. The goal of adjuvant treatment is to avoid cancer recurrence and progression to advanced stages.

Merck has submitted KEYNOTE-564 for the recommendation. NICE agreed that Keytruda improves disease-free survival versus placebo. NICE agreed that the model was appropriate to determine cost-effectiveness. The HTA body agreed that Keytruda was cost-effective and can be used under routine use.

Keytruda is preferred to administer 400 mg every six weeks. The annual cost of treatment was £89,420 (without discounts). Keytruda is available to NHS with a discount, which is confidential.


October 05, 2022

AstraZeneca's Lynparza was not recommended for prostate cancer in the UK.

NICE has not recommended using AstraZeneca's Lynparza (olaparib) for adult patients with metastatic castration-resistant prostate cancer and BRCA1/2 mutations. NICE considered it not cost-effective at a price chosen by the company.

NICE agreed that there is a high unmet need in patients with hormone-relapsed metastatic prostate cancer. NICE considered the taxane group, cabazitaxel, radium-223 dichloride, and retreatment with docetaxel as relevant comparators.

The company has submitted a PROfound trial, in which Lynparza was compared with the investigator's choice of enzalutamide or abiraterone. NICE agreed that Lynparza was more effective than retreating with enzalutamide or abiraterone.

Because of the differences in clinical trials, NICE has not accepted the indirect comparison of Lynparza with cabazitaxel for the prior taxane group.

Overall, NICE has not recommended Lynparza, considering it is not cost-effective at a price chosen by the company.


October 05, 2022

NICE recommended BMS' Zeposia for ulcerative colitis

NICE recommended using Zeposia (ozanimod) for adult patients with ulcerative colitis who have an inadequate response or are intolerant to biologics. NICE recommended Zeposia for patients for whom infliximab is not suitable.

The annual cost of Zeposia treatment was £17,910 per person per year, considering the induction and maintenance dose.

Ulcerative colitis is a lifelong condition characterized by the inflammation of the rectal and colonic lining. It severely impacts the quality of life of a patient. NICE agreed that there is a high unmet need for patients with ulcerative colitis.

The company has submitted evidence for two patient groups, TNF naive and TNF experience patients. NICE considered all TNF inhibitors, Stelara, Entyvio, and Xeljanz, as relevant comparators. NICE commented that Zeposia is neither superior nor inferior to competitors in efficacy based on the network meta-analysis.

BMS submitted TRUENORTH to support the dossier, and NICE agreed that the population is in line with the NHS clinical practice. Further, it agreed that Zeposia improved clinical remission in biologically naive and biologically experienced patients.

NICE considered Zeposia to be cost-effective in TNF-alpha inhibitor-naive subgroup except versus infliximab. NICE agreed that the benefit of Zeposia was captured in the model.


September 28 2022

Tecentriq is recommended for stage 2 to 3a NSCLC patients with PD-L1 expression of >50%.

NICE recommended the use of Roche's Tecentriq (atezolizumab) in adult patients with stage II to IIIA non-small cell lung cancer whose tumors have programmed cell death ligand-1 (PD-L1) expression >50% and who have not progressed after platinum-based chemotherapy.

NICE agreed that there is a high recurrence rate in patients with early NSCLC. Within five years, cancer reoccurs in 17% to 29% of patinents with stage 1 cancer, 38% to 46% in patients with stage 2 cancer, and 47% to 64% of patients with stage 3 cancer, regardless of adjuvant chemotherapy.

Currently, chemotherapy is the only option for patients after complete resection, which has less benefit in overall survival.

Roche submitted Phase 3 IMpower010 trial to demonstrate the clinical effectiveness of Tecentriq. IMpower010 trial is a Phase 3 multicentre, open-label study comparing Tecentriq with active monitoring in patients who underwent resection and cisplatin-based adjuvant chemotherapy in patients with 1b to 3a NSCLC. Tecentriq reduced the relative risk of cancer recurrence or death by 57% versus active monitoring. Median disease-free survival was 35.7 months in patients on active monitoring, while it was not reached in patients on Tecentriq. NICE was not certain about the improvement of overall survival with the currently available data.

While the initial company's model was not considered to be appropriate, the revised model was accepted by NICE. NICE has criticized several factors in the economic model, including the costs considered and the company's adjustments to the disease-survival extrapolation.

NICE agreed that Tecentriq met the criteria to be included in the Cancer Drugs Fund.

September 30 2022


September 30 2022

AbbVie's Rinvoq was recommended for treating active ankylosing spondylitis.

NICE has recommended using Rinvoq (upadacitinib) for adult patients with active ankylosing spondylitis who are unsuitable or have an inadequate response to tumor necrosis factor (TNF)-alpha inhibitors.

NICE has suggested considering the least suitable treatment options among upadacitinib, secukinumab, and ixekizumab. Further, NICE recommended assessing the efficacy of Rinvoq after 16 weeks of treatment and suggested continuing only if there is evidence of response.

NICE estimated the annual cost of Rinvoq to be £10,501. AbbVie has agreed to a commercial agreement.

A cost comparison analysis with secukinumab was considered appropriate