NICE ASSESSMENT OUTCOMES

Updated: Oct 21

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.

Also read: HAS assessment outcomes


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.

Also read: 2022 FDA Approvals


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.


Also read: AbbVie's JAK inhibitor, Rinvoq, was approved for UC and nr-axSpA in Europe

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.

Also: EMA Approvals in 2022


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.


Also read: FDA approved Kymriah for follicular lymphoma

 

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.

Also read: CHMP Positive Opinion


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.

Also read: 2022 FDA Approvals


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.


Also read: Spinal Muscular Atrophy News

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.


Also read: FDA approvals


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.

Also read: Health Canada Drug Approvals


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.

Also read: Pharmaceuticals Deals and Acquisitions


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.