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

Updated: Jun 7

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.

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NICE's draft guidance recommended Daiichi Sankyo's Enhertu for breast cancer.

NICE's draft guidance recommended using Daiichi Sankyo's Enhertu (trastuzumab deruxtecan) for treating adult patients with unresectable or metastatic HER2-positive breast cancer who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.

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.

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 by NICE. AbbVie presented two Phase 3 trials, SELECT‑AXIS 1, which included patients previously not treated with biological disease-modifying antirheumatic drugs (DMARDs), and SELECT‑AXIS 2, which included patients treated with biological disease-modifying antirheumatic drugs (DMARDs). In both trials, there was a statistically significant improvement in Assessment in Spondyloarthritis International Society 40% (ASAS40) response and BASDAI 50 score versus placebo.

In the cost-comparison model, NICE commented on not including the cost of adverse events in the company's model. NICE agreed that the total costs associated with Rinvoq are lesser than those associated with secukinumab and ixekizumab.


September 30 2022

NICE said no to PTC Therapeutics's Translerna for DMD.

NICE published the draft guidance in which it did not recommend using Translarna (ataluren) for treating Duchenne muscular dystrophy (DMD). This recommendation is based on the re-evaluation to decide if Translerna should be used for routine use. It has been available for six years under managed access agreement (MAA). Nearly sixty children were administered with Translarna under this arrangement.

DMD is caused because of a lack of dystrophin. This leads to loss of muscle function, with children needing a wheelchair and eventually needing help for breathing.

Translarna costs £220,000 per patient per year. NICE has made this decision based on the data collected from MAA, new real-world evidence, and feedback from clinicians and people.

NHS and PTC Therapeutics agreed that patients currently using Translarna will be administered with Translarna until physicians consider it beneficial.


September 21, 2022

NICE recommended CSL Vifor's Tavneos for severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA)

NICE recommended the use of Tavneos (avacopan) for treating active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA). Tavneos is indicated in combination with rituximab or cyclophosphamide.

Antineutrophil cytoplasmic antibody (ANCA) associated vasculitis is characterized by blood vessel inflammation. GPA and MPA are the two most common types of ANCA-associated vasculitis. NICE agreed that the company's positioning was appropriate; the company informed that it recruited patients with GPA or MPA in the clinical trial.

The recommendation was based on ADVOCATE, a Phase 3 trial. The primary endpoint was people with disease remission at weeks 26 and 52. 72% of people on Tavneos had disease remission at week 26 versus 70% of patients on prednisone. 66% of patients on Tavneos had disease remission versus 55% of patients on prednisone.

NICE agreed that the economic model submitted by the company was appropriate for decision-making. It agreed that Tavneos with cyclophosphamide or rituximab regimen was cost-effective versus standard of care.


August 10, 2022

Opdivo was recommended for invasive urothelial cancer at high risk of recurrence.

NICE recommended the use of Bristol Myers Squibb's Opdivo (nivolumab) for treating adult patients with muscle-invasive urothelial carcinoma (MIUC) and with PD-L1 expression ≥ 1%. It was restricted to patients in which platinum‑based chemotherapy is unsuitable. BMS agreed with the commercial agreement for the recommendation.

Surgery was the aim of removing cancer. The standard of care to avoid recurrence was using platinum-based chemotherapy or best supportive care.

The recommendation was based on the Checkmate 274 study, an ongoing Phase 3 clinical trial. NICE agreed that there was an increase in disease-free survival versus placebo in patients with PD-L1≥ 1%. However, overall survival gains were not established.

NICE commented that the company had not compared the cost-effectiveness of Opdivo versus platinum-based chemotherapy; CE estimates were considered appropriate only if chemotherapy is unsuitable. It was recommended under routine commissioning.


August 10, 2022

NICE approved Janssen's Tremfya for psoriatic arthritis

NICE recommended Janssen's Tremfya (guselkumab) for treating adult patients with psoriatic arthritis who had an inadequate response or who are intolerant to a prior disease-modifying anti-rheumatic drug (DMARD) therapy. NICE recommended Tremfya for patients who have had at least one biologic DMARD or who are contraindicated to TNF inhibitors.

Janssen has submitted two trials - DISCOVER‑1 and DISCOVER‑2. In both studies, NICE agreed that the efficacy of guselkumab is higher than placebo. Further, it agreed that the population is in accordance with NHS clinical practice.

NICE broadly agreed that the model submitted by Janssen was according to the other technology appraisals.


August 10, 2022

Piqray was recommended for HER2-negative PIK3CA mutated breast cancer.

NICE has recommended the use of Novartis' Piqray (alpelisib) for hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative breast cancer. The product was positioned after a CDK4/6 inhibitor plus an aromatase inhibitor. Further, Novartis agreed to a commercial agreement with NICE.

The current standard of care for HR+, HER2-negative, PIK3CA mutated, locally advanced, or metastatic breast cancer after CDK4/6 inhibitor was everolimus plus exemestane. With the indirect comparison, NICE agreed that Piqray plus exemestane had shown superior efficacy.

The recommendation was based on the BYLieve study, a single-arm study. However, NICE has not agreed to the SOLAR-1 study, as it included only 20 relevant patients.

NICE agreed that Piqray met the NICE's criteria of being a life-extending drug at the end of life. NICE agreed that the cost-effectiveness estimates are within the acceptable limits, though they are uncertain.


May 17, 2022

NICE recommended Verzenios for HR+, HER2-negative, node-positive early breast cancer

NICE recommended Verzenios (abemaciclib) for treating patients with hormone-positive, HER2-negative, and node-positive breast cancer. Verzenios was indicated for patients with high-risk recurrence following surgery.

Further, NICE agreed that the treatment with Verzenios resulted in 30% better chances of cancer not returning compared to patients who were on hormone therapy alone.

NICE highlighted that nearly 50,000 have breast cancer in England, out of which 70% of patients have HER2-negative breast cancer. Cancer in almost 30% of patients will return after the initial treatment.


May 25, 2022

Keytruda was recommended for a rare form of TNBC

NICE has published the final draft recommendation, in which Keytruda (pembrolizumab) in combination with chemotherapy (paclitaxel or nab-paclitaxel) was recommended for treating patients with triple-negative breast cancer, which has spread to other parts of the body. Merck has agreed to provide Keytruda to the NHS with a discount.

Keytruda is recommended in patients whose tumors express PD-L1 with a combined positive score (CPS) of 10 or more and immune cell staining (IC) of less than 1%.

Merck submitted KEYNOTE-355, a double-blind placebo-controlled active-comparator trial that evaluated the efficacy of Keytruda in combination with chemotherapy versus chemotherapy alone. The study showed improvement in the progression-free survival in the Keytruda arm compared to the placebo arm. Median progression-free survival was 7.6 months and 5.6 months in the Keytruda arm and chemotherapy arm, respectively. At six months, 56.4% and 47.8% of patients on Keytruda and chemotherapy alone had no disease progression or death.

Previously, Tecentriq (atezolizumab) was recommended for treating patients with untreated PD-L1 positive, advanced or metastatic, triple-negative breast cancer.


May 24, 2022

NICE recommended Vabysmo for two forms of sight loss

NICE has recommended Roche's Vabysmo (faricimab) for treating wet age-related macular degeneration or diabetic macular edema. NICE published the final recommendation for treating two forms of sight loss.

It is administered as an eye injection in adult patients with wet age-related macular degeneration or diabetic macular edema. Vabysmo is administered with an interval of up to 16 weeks between two doses and is as effective as aflibercept which was administered every eight weeks.

Up to 300,000 patients with wet age-related macular degeneration and 28,000 people with diabetic macular edema are eligible for treatment in England.


May 13, 2022

NICE recommended Ryeqo for women with uterine fibroids

In its final draft guidance, NICE has recommended Ryeqo (relugolix / estradiol/norethisterone acetate) for treating women with moderate to severe fibroids.

The usual treatment for uterine fibroids includes hormonal contraception and non-steroidal anti-inflammatory drugs. For treating moderate to severe patients, injectable gonadotropin-releasing hormone (GnRH) agonists are used. NICE, in its statement, mentioned that nearly 4,500 women would be eligible for the new oral treatment option.

Uterine fibroids usually impact 1 in 3 16-50 years old and are a non-cancerous growth around the uterus. The symptoms include menstrual bleeding, pelvic pain, and fertility problems.


14 April 2022

NICE recommended Tepmetko for NSCLC with METex14 skipping gene alterations

NICE recommended Merck Serono's Tepmetko (tepotinib) for treating adult patients suffering from NSCLC with METex14 skipping gene alterations.

Patients with METex14 skipping gene alterations account for 1-2% of lung cancer patients in England.

Tepmetko is recommended under Project Orbis, which aims to provide access to cancer therapies faster to patients. From trial data and analyzing real-world data, it is expected that the project might increase its survival. It is expected to be recommended as first-line therapy after genomic testing.

12 April 2022

Cefiderocol and ceftazidime-avibactam will be available in the UK under an innovative subscription-style payment model

NICE evaluated cefiderocol and ceftazidime-avibactam under the project in collaboration with NHS England and NHS Improvement, and the Department of Health and Social Care (DHSC) to develop therapies for antimicrobial resistance. It is indicated for patients who develop severe infections and are not having therapeutic alternatives.

The main goal of the subscription-style payment model is to encourage innovation to address antimicrobial resistance. NICE highlighted that only 41 new antimicrobials are in clinical trials in 2020, compared to 1800 immuno-oncology drugs.

In 2019, the UK government published its five-year plan to address antimicrobial resistance. The main goal is to invest in innovation and access to antimicrobial resistance. NICE agreed developing therapies for antimicrobials is not commercially attractive.

NICE highlighted that antimicrobials are essential to ensure chemotherapy, surgery, and medical procedures go ahead, prevent the risk of infecting others, and provide multiple antimicrobials for treating antimicrobial resistance.

07 April 2022

NICE recommended Merck Serono's Bavencio for routine use in patients with urothelial cancer

NICE recommended using Bavencio (avelumab) for routine use for patients with adults with locally advanced or metastatic urothelial cancer. It is indicated in patients who were previously not treated with platinum-based chemotherapy.

The recommendation was based on new evidence submitted by the company and the additional discount provided by Merck Serono. NICE estimated that nearly 800 patients would benefit from this new treatment option.

The recommendation was based on the JAVELIN Bladder 100 trial, in which Bavencio showed improvement in the overall survival compared to the standard of care. The risk of death was reduced by 31% in the Bavencio arm compared to SoC. There is more improvement in PD-L1 positive tumors - the risk of death was reduced by 44% in the Bavencio arm compared to SoC.

Urothelial cancer is more common in men than women; the primary risk factors include age, smoke, and industrial chemicals.

07 April 2022

NICE says Trodelvy is effective but not cost-effective

In its graft guidance, NICE has not recommended the use of Gilead's Trodelvy (sacituzumab govitecan) in patients with triple-negative breast cancer.

Trodelvy is indicated for triple-negative breast cancer that cannot be removed by surgery and the breast cancer is in the advanced or metastatic stage. It is indicated in patients who were previously treated with two other systemic therapies.

Triple-negative breast cancer accounts for 15% of breast cancer in the United Kingdom, and the annual incidence is 4,500-6,750.

The list price of Trodelvy is £ 793 per 180mg vial. The draft guidance is under public consultation until 29 April 2022.

03 March 2022

NICE recommended Amgen's Lumykras for KRAS positive NSCLC

NICE has recommended using Amgen's Lumykras (sotorasib) for KRAS 12C gene mutation of non-small-cell lung cancer. It is indicated for patients who have progressed on, or are intolerant to, platinum-based chemotherapy and/or immunotherapy.

Nearly 500 patients in England will benefit from the new recommendation. KRAS mutation is the most common mutation in the NSCLC.

Platinum-based chemotherapy and/or immunotherapy is the standard of care for NSCLC patients and is indicated after chemotherapy.

08 February 2022

Wegovy recommended for people living with obesity.

Wegovy (semaglutide) is recommended for patients living with obesity by NICE. It is recommended in patients with a BMI of 35 kg/m2, and exceptionally, to people with a body-mass index (BMI) of 30.0 kg/m2 to 34.9 kg/m2.

Wegovy is recommended for a maximum duration of two years.

Wegovy acts like a glucagon-like peptide-1 hormone, a hormone released after eating. In STEP 1 clinical trial, patients on Wegovy lost 12% more weight than those on placebo.

The 2019 Health Survey of England estimated that 28% of people in England are obese and 36% are overweight. The current cost because of obesity is £6.1 billion.

04 February 2022

NICE recommended £2.8 million drug for metachromatic leukodystrophy

NICE has recommended using Libmeldy (atidarsagene autotemcel), a gene therapy from Orchard Therapeutics for metachromatic leukodystrophy (MLD). NICE also recommended Libmeldy for patients who have symptoms yet can walk.

The one-time gene therapy costs around £2.8 million. Previously, the drug was not recommended in the draft guidance; however, the drug is recommended after giving confidential discounts.

NICE agreed that Libmeldy helps improve motor and cognitive function in the short term, and there is a probability of correcting the enzyme deficiency.

08 February 2022

GSK's Jemparli recommended by NICE for endometrial cancer

NICE recommended using GSK's Jemparli (dostarlimab) for treating adult patients with endometrial cancer within the Cancer Drugs Fund.

Jemparli is recommended as second-line therapy in patients with endometrial cancer with high microsatellite instability or mismatch repair deficiency.

NICE agreed that there are no standard second-line treatments; there is a high unmet need in these patients.

GSK submitted the GARNET trial, phase 1 single-arm trial. The primary endpoint are objective response rate and duration of response. NICE commented that the data is immature. However, it agreed that Jemperli improves survival, but outcomes are uncertain. GSK submitted partitioned-survival model. NICE has not recommended for routine commission, but is recommended under Cancer Drugs Fund.

Endometrial cancer originates in the womb lining, and the incidence is 2,162 in the UK. NICE commented that chemotherapy administration takes a day; however, dostarlimab administration can be done within 30 minutes.

16 December 2021

NICE guidance recommended Roche's Evrysdi for SMA

NICE has published its draft guidance on Roche's Evrysdi, and it has recommended it for patients with 5q spinal muscular atrophy (SMA). It has recommended Evrysdi in 2 months of age and older patients diagnosed with either Type 1, Type 2, or Type 3 SMA or with one to four SMN2 copies.

Roche has submitted the results from the SUNFISH and FIREFISH, which NICE considered appropriate for decision-making. SUNFISH included patients with type 2 or type 3; FIREFISH included patients with type 1 SMA. NICE agreed that the evidence was sufficient to make the decision.

Based on the JEWELFISH study, NICE agreed that Evrysdi could be recommended in patients who previously had nusinersen.

NICE agreed that there was an improvement in the motor function, but there was no evidence on overall survival data for type 1 SMA.

NICE believed that the ICER value of Evrysdi was £50,000 per QALY gained. The company has agreed to provide the managed access agreement (MAA), and NICE has recommended Evrysdi with MAA.

05 November 2021

NICE draft guidance recommended AstraZeneca's Forxiga for CKD

In the draft guidance, NICE recommended AstraZeneca's Forxiga (dapagliflozin) for treating chronic kidney disease in adults. The drug is recommended as an add-on to the standard of care (ACE inhibitors or ARBs unless they are contraindicated). Further, patients whose eGFR is in the range of 25 ml/min/1.73 m2 to 75 ml/min/1.73 m2 and uACR of 22.6 mg/mmol or more are eligible for receiving the treatment.

NICE estimated that nearly 91,000 patients in England would benefit from the treatment.

Forxiga is a sodium-glucose co-transporter-2 (SGLT2) inhibitor and is the first SGLT2 inhibitor recommended by NICE. The drug acts by inhibiting SGLT2 protein in the kidney, thereby reducing the inflammation and pressure on the kidney.

The addition of the drug to the therapeutic strategy is expected to reduce the risk of end-stage renal disease and death.

AstraZeneca has submitted the DAPA-CKD trial in which dapagliflozin plus standard of care was compared with placebo plus standard of care. Further, the company submitted a de novo health economic model, which included a cohort-level state transition approach with six health states. NICE considered the drug cost-effective in patients included in the DAPA-CKD trial and patients with a uACR of 3 mg/mmol or more and with type 2 diabetes.

October 28 2021

NICE recommended Janssen's Erleada for prostate cancer

NICE recommended the use of Erleada for treating high-risk hormone relapsed non-metastatic prostate cancer and hormone-sensitive metastatic prostate cancer.

In order to support the recommendation for patients with high-risk hormone-relapsed non-metastatic prostate cancer, the company has submitted SPARTAN trial. SPARTAN trial was a Phase-3, placebo-controlled study, in which apalutamide plus ADT was compared with placebo plus ADT. NICE agreed that the patients included in the study reflected people in NHS clinical practice. The median overall survival was 73.9 months in apalutamide plus ADT group versus 59.9 months in placebo arm. Metastasis-free survival in apalutamide plus ADT was 40.5 months versus 15.7 months in placebo arm. NICE agreed with the model submitted and considered that the drug to be cost effective.

To support the recommendation for patients with hormone-sensitive metastatic prostate cancer, the company submitted TITAN Phase 3 results. In the trial, apalutamide plus ADT was compared with placebo plus ADT. Median radiographic progression-free survival was not reached in apalutamide plus ADT, it was 22.1 months in placebo arm. The company submitted a partition survival model, and NICE has considered it to be cost-effective.

October 5 2021

NICE says yes to Novartis' Adakveo for sickle cell disease

NICE accepted Adakveo for recurrent sickle cell crises (vaso-occlusive crises) in patients with sickle cell disease. The drug is indicated for patients of age 16 years and above.

Hydroxycarbamide is the standard of care for patients with SCD. Blood transfusions are recommended for patients for whom hydroxycarbamide cannot be administered.

The recommendation was based on the SUSTAIN trial, a double-blind, placebo-controlled study. The primary endpoint is the annual rate of sickle cell-related

pain crises; the median sickle cell-related pain crises reduced significantly in the Adakveo arm versus placebo arm. However, there was uncertainty over the long-term clinical effectiveness.

NICE has initially not recommended Adakveo considering it not to be cost-effective, however, the company has provided managed access agreement. Considering the access agreement, NICE recommended the use of Adakveo. Novartis proposed considering the data under managed access agreement, to address uncertainties of long-term clinical effectiveness.

The STAND trial and National Haemoglobinopathy Registry will be considered for collecting the additional data.


May 16 2021

NICE recommended the use of Opdivo in advanced, recurrent, or metastatic esophageal squamous cell carcinoma patients

NICE recommended using Opdivo in patients with advanced, recurrent, or metastatic esophageal squamous cell carcinoma. The drug is recommended after fluoropyrimidine and platinum-based therapy.

The company submitted ATTRACTION‑3 trial in its dossier; people were administered with either nivolumab or taxane chemotherapy. Opdivo showed an improvement in the median overall survival of 2.4 months versus chemotherapy. But NICE observed that the progression-free survival and overall response rate were lower in the Opdivo arm.

NICE commented that there is uncertainty over the method used for extrapolating overall survival. Considering the commercial access agreement, NICE agreed that Opdivo is cost-effective.

NICE said OK to Kesimpta to treat RRMS as first-line therapy

NICE recommended the use of Kesimpta (ofatumumab) for relapsing-remitting multiple sclerosis. The HTA body recommended using the drug as first-line therapy.

NICE agreed that Kesimpta has the advantage of administrating at home, which increases the patient’s comfort compared to other intravenous dosage forms.

The recommendation was based on Phase III ASCLEPIOS I (n=927) and ASCLEPIOS II (n=955); Kesimpta was compared versus teriflunomide. Kesimpta demonstrated better efficacy than teriflunomide. Further, the committee mentioned that Kesimpta reduces annualized relapse rates and slow disability progression than most comparators except monoclonal antibodies.

The list price of Kesimpta is £1,492.50 per unit.


May 06 2021

NICE draft guidance says Bavencio was not cost-effective for urothelial cancer

NICE issued draft guidance not recommending the use of Bavencio (avelumab) in patients with urothelial cancer after platinum-based chemotherapy.

NICE agreed that the evidence demonstrated that people with urothelial cancer live longer with Bavencio and take longer for cancer to worsen. However, NICE has commented that the drug is not cost-effective.

NICE further commented that there are no ongoing studies to address the critical uncertainties; the drug was not considered to include in the Cancer Drugs Fund.

Currently, in England, atezolizumab, docetaxel, paclitaxel, or best supportive care is indicated for treating urothelial cancer patients whose disease progressed after platinum-containing chemotherapy.

In Europe, Bavencio was approved in January 2021 for urothelial cancer in patients prior treated with platinum-containing chemotherapy.

April 2021

NICE said OK for Ultomiris to treat PNH patients

In its draft guidance, NICE recommended the use of Ultomiris (ravulizumab) to treat patients with paroxysmal nocturnal hemoglobinuria (PHN).

Currently, Soliris is available to treat PNH patients. The drug is administered as an intravenous infusion every two weeks.

The patients in England are eligible for Ultomiris, which is administered every eight weeks. Experts in the UK said that a reduction in the number of doses increases the quality of life of PNH patients.

The recommendation is based on the fact that treatment with Ultomiris reduced the episodes of breakthrough hemolysis and clinically as effective as Soliris.

PNH is a rare blood disorder caused by the breakage of red blood cells, which leads to anemia, kidney problems, and blood clots in the blood vessels.

March 2021

NICE accepted Opdivo as an adjuvant treatment for wholly resected melanoma with lymph node involvement or metastatic disease

NICE said OK for Opdivo as an adjuvant treatment for completely resected melanoma in adults with lymph node involvement or metastatic disease.

Lymph node resection is considered the standard of care for stage 3 melanoma and some patients with stage 4 melanoma. Until recently, continuous monitoring is recommended after the resection of melanoma. The committee believed that there is a need for adjuvant treatment for the complete removal of the melanoma. The adjuvant therapy helps in preventing the recurrence of cancer. Keytruda (pembrolizumab) was recommended for patients who are at high risk of recurrence.

The recommendation was based on the CheckMate 238 trial. The study was a Phase-3 multi-center, randomized trial comparing the efficacy of Opdivo versus Yervoy. Patients with complete resection in either stage 3B, 3C, or four were enrolled in the trial. Significant improvement in recurrence-free survival was observed in the Opdivo arm versus the Yervoy arm. However, the overall survival data were immature.

Further, the committee was of the opinion that there are several treatment options available in case of recurrence. The data available from the Cancer Drugs Fund is limited for patients who were treated with Opdivo; only 14% of patients had subsequent treatments. Clinical experts believed that combination treatment could be provided if the patient is tolerant. NICE agreed that there is a higher degree of certainty in trial than the data available with CDF. NICE agreed that the subsequent treatment of the Opdivo arm in the CheckMate 238 trial is per the clinical practice.

BMS submitted a partitioned survival model and a state transition model for the appraisal.


NICE draft guidance recommended Novartis’ £1.79 million Zolgensma for SMA

NICE published draft guidance, which recommended the use of Zolgensma (also called onasemnogene abeparvovec) for patients of age less than 12 months.

Zolgensma is used for treating patients with SMA 1, a rare genetic disorder. Patients with SMA have a life expectancy of less than two years.

NICE stated that even though the product is highly expensive, it recommends using it within the NHS as it benefits young babies significantly.

Further, NICE suggested using Zolgensma after an initial discussion with a national multidisciplinary clinical team. This enables the patients to get the treatment who are eligible as per market authorization of the drug but were not included in the trial.

SMA is a rare neuromuscular condition caused by a mutation that results in the inadequate expression of survival motor neuron (SMN) protein. Previously NICE recommended Biogen’s Spinraza for SMA type 1, 2, or 3 patients.

It is estimated that nearly 65 children are born each year in England with SMA, 60% of who have type 1 SMA.


February 2021

NICE OKs Novartis Kisqali for routine use

Improvement in the overall survival certainty and improved progression-free survival are critical drivers for the recommendation.

NICE has announced that Kisqali (ribociclib) will be available for routine use after its draft guidance. Previously the drug is recommended under Cancer Drugs Fund (CDF).

The draft guidance recommended the combination of Kisqali and fulvestrant for treating patients with hormone receptor-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced metastatic breast cancer.

The recommendation was based on the fact that Kisqali and fulvestrant combination increase the progression-free survival and overall survival compared to fulvestrant alone.

Kisqali is the only CDK4/6 inhibitor recommended for routine use; the other two products (palbociclib, abemaciclib) are recommended under the Cancer Drugs Fund.


Roche’s Rozlytrek recommended as monotherapy for solid tumors expressing a neurotrophic tyrosine receptor kinase gene fusion

The drug is recommended Rozlytrek (entrectinib) in adult and pediatric patients (12 years and older) patients who have:

  • Locally advanced metastatic cancer or where surgical resection is likely to result in severe morbidity

  • Were not previously treated with NTRK inhibitor

  • No satisfactory treatment options are available

The company has agreed to a commercial agreement that enables NHS to obtain entrectinib with a discount. At list price, the cost of treatment with entrectinib for one year is nearly £ 62,000.

England’s cost watchdog has commented that the drug targets a specific genetic mutation, which posed challenges in appraising the drug candidate. The drug target a neurotrophic tyrosine receptor kinase (NTRK) gene fusion. Entrectinib is placed as a final option for treating cancer; it can be administered if the patient was prior treated with all the available NHS commissioned treatments.

The drug has been recommended for use within the Cancer Drugs Fund.


Roche’s Rozlytrek recommended for ROS1-positive advanced non-small-cell lung cancer (NSCLC)

NICE has recommended Rozlytrek (entrectinib) for ROS1-positive advanced non-small-cell lung cancer (NSCLC) as a first-line treatment. NICE has recommended the product only if the company agrees for the commercial access agreement.

NICE has agreed that patient experts would prefer to have an oral dosage form ROS1 positive NSCLC. ROS1 positive mutations are rare in patients with NSCLC, with only 2% of NSCLC patients have ROS1 positive mutations.

The company has submitted two Phase 1 studies (ALKA and STARTRK‑1) and one Phase 2 study (STARTRK‑2) for the appraisal. NICE agreed that entrectinib shows a high overall response rate and slows disease progression.

NICE accepted that Rozlytrek is cost-effective, the drug is likely to have an ICER value of less than £50,000 per QALY gained versus PEM+PLAT. It was considered to be acceptable for end-of-life treatments.


NICE recommended Astellas Xospata for FLT3 positive AML

NICE has recommended Xospata (gilteritinib) for FLT3mutation-positive acute myeloid leukemia. The drug is administered as monotherapy. NICE recommended Xospata not to be administered as maintenance therapy for patients who underwent hematopoietic stem cell transplant.

The company has agreed to provide a commercial agreement to have a positive recommendation.

FLT3 mutations are associated with poor outcomes and a high relapse rate. NICE agreed that new treatment options are welcome, which improve the survival and quality of life. NICE commented that oral dosage form improves the quality-of-life benefits for patients. Also, patients who were on Xospata live longer compared to patients who were on chemotherapy.

The company has submitted the ADMIRAL study for the assessment. NICE considered salvage chemotherapy as an appropriate comparator; the median overall survival was increased from 5.6 months to 9.3 months compared to salvage chemotherapy.

NICE commented on the lack of robust evidence in patients who underwent transplantation.

The ICER value was less than £50,000 per QALY compared to the salvage chemotherapy. NICE has accepted that this is within the range, at which it is considered to be acceptable for life-extending treatment.


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