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NICE Assessment Outcomes | Untied Kingdom | Drug News | Updates | 2024 | iPharmaCenter

Sl No






LAL deficiency




Biliary tract cancer



Olaparib plus bevacizumab

Ovarian, fallopian tube, or primary peritoneal cancer




Metastatic breast cancer




Hormone relapsed metastatic prostate cancer








Graft-versus host disease


January 10, 2024

NICE recommended Alexion's Kanuma for LAL deficiency

Sebelipase alfa, marketed as Kanuma by Alexion, holds an indication for the 'long-term enzyme replacement therapy (ERT) in patients of all ages with lysosomal acid lipase (LAL) deficiency.' Specifically, its recommendation as an option for long-term enzyme replacement therapy is made in the context of Wolman disease (rapidly progressive lysosomal acid lipase deficiency [LAL-D]), provided the treatment initiates when individuals are 2 years or younger. This recommendation is contingent upon the company supplying sebelipase alfa in accordance with the established commercial arrangement.

The supporting clinical evidence for sebelipase alfa is derived from two single-arm, open-label, multicenter trials that included individuals diagnosed with LAL deficiency, some of whom were from the UK. LAL-CL08, a phase 2 trial involving 10 individuals diagnosed at 8 months old or younger, primarily focused on safety outcomes related to severe treatment-emergent adverse events during up to 3 years of sebelipase alfa administration.

Meanwhile, LAL-CL03, a phase 2/3 trial with 9 individuals diagnosed at 2 years old or younger, assessed the proportion of people alive at 12 months old as its primary outcome during up to 5 years of treatment. Additionally, the company utilized a natural history study (LAL-1-NH01) involving 35 people diagnosed with Wolman disease between 1985 and 2012 to estimate outcomes for clinical management without sebelipase alfa.

The committee recognized the limitations in clinical trial inclusion criteria based on the age of diagnosis or symptom onset. Clinical experts explained that diagnosis may occur up to 2 years old, emphasizing a similar poor prognosis in the older population. Despite acknowledging the limited trial size and differences in inclusion criteria, the committee revised the cost-effectiveness estimates, considering plausible assumptions, and deemed sebelipase alfa an acceptable use of NHS resources for treating Wolman disease, specifically for individuals aged 2 years or under at the start of treatment.

Wolman disease, characterized by a complete loss of lysosomal acid lipase (LAL) enzyme activity, manifests as a rapidly progressing multisystem disease primarily affecting babies and children under 2 years old. Diagnosis is achieved through genetic testing or identifying variants in the LIPA gene, and the condition typically results in death within the first 6 months of life due to multiple organ failure. The evaluation scope pertains exclusively to Wolman disease within the company's marketing authorization for sebelipase alfa.


January 10, 2024

Imfizi was recommended for metastatic biliary tract cancer

Durvalumab, commercially known as Imfinzi by AstraZeneca, in conjunction with gemcitabine and cisplatin, is authorized for the initial treatment of adults facing locally advanced, unresectable, or metastatic biliary tract cancer. The endorsement for durvalumab plus gemcitabine and cisplatin is granted as an option for managing locally advanced, unresectable, or metastatic biliary tract cancer in adults, contingent on the company's adherence to the commercial arrangement. The clinical evidence stems from the ongoing TOPAZ-1, a phase 3, double-blind, randomized controlled trial comparing durvalumab plus gemcitabine and cisplatin with placebo plus gemcitabine and cisplatin.

In this trial, individuals received either durvalumab plus gemcitabine and cisplatin or placebo plus gemcitabine and cisplatin every 3 weeks (up to 8 cycles), followed by durvalumab or placebo monotherapy every 4 weeks until disease progression. The study encompassed adults with previously untreated unresectable advanced or metastatic biliary tract cancer or whose cancer recurred more than 6 months post-surgery or completion of adjuvant therapy. The exclusion of ampullary cancer aimed to reduce heterogeneity. The trial included 341 individuals in the intervention group and 344 in the comparator group. The trial data, as of February 2022, showed an increase in median overall survival from 11.3 to 12.9 months and median progression-free survival from 5.7 to 7.2 months for durvalumab plus gemcitabine and cisplatin compared to the placebo group. The committee acknowledged the proportional hazard assumption's non-compliance and considered piecewise hazard ratios, recognizing the treatment benefit appearing several months after randomization due to durvalumab's immunotherapeutic mechanism.

The economic model, reflecting TOPAZ-1, utilized a partitioned survival model, adopting a 20-year time horizon with costs discounted at 3.5% annually. The committee found the model structure appropriate and accepted the company's model for decision-making. The preferred ICERs for durvalumab plus gemcitabine and cisplatin, ranging from £20,000 to £30,000 per QALY gained, were deemed acceptable, given the substantial unmet need. Noting uncertainties, the committee concluded that the most likely cost-effectiveness estimates fell within NICE's acceptable range and recommended durvalumab plus gemcitabine and cisplatin as an option for unresectable or advanced biliary tract cancer.

Recognizing its status as the first immunotherapy for such conditions, the committee considered the treatment innovative but found no additional benefits unaccounted for in economic modelling, affirming that all benefits were duly considered.


January 17, 2023

Olaparib plus bevacizumab recommended for ovarian, fallopian tube, or primary peritoneal cancer

Olaparib in combination with bevacizumab has received a recommendation for use within its authorized market scope. This recommendation is specifically for the maintenance treatment of high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer in adults meeting the following criteria:

  1. The cancer has completely or partially responded following first-line platinum-based chemotherapy with bevacizumab.

  2. The cancer is at an advanced stage (International Federation of Gynecology and Obstetrics [FIGO] stages 3 and 4).

  3. The cancer is positive for homologous recombination deficiency (HRD).

The supporting evidence for the clinical effectiveness of olaparib with bevacizumab is derived from the PAOLA‑1 trial, a phase 3, double-blind, randomized controlled trial involving 806 individuals with advanced ovarian cancer (stages 3 and 4). In this trial, olaparib (300 mg twice daily, n=537) was compared to a placebo (n=269), with all participants receiving bevacizumab (15 mg per kg every 3 weeks) as maintenance treatment.

The primary endpoint in PAOLA‑1 was the investigator-assessed progression-free survival (PFS). In the course of the review, the company provided more mature PFS data, which consistently demonstrated a statistically significant advantage in PFS for the HRD-positive subgroup treated with olaparib and bevacizumab compared to the placebo with bevacizumab. The median PFS was significantly higher in the olaparib with the bevacizumab group (46.8 months) than in the placebo with the bevacizumab group (17.6 months).

While overall survival (OS) was a secondary endpoint in PAOLA‑1, the data for the HRD-positive subgroup were considered promising but uncertain due to their immaturity. The median OS had not been reached at the data cut-off point. The committee acknowledged the company's modelling approach as suitable for decision-making but highlighted the persistent uncertainty regarding the proportion of individuals who might achieve a 'cure.'

The committee also noted certain unaccounted benefits, such as the impact of HRD testing on the necessity for somatic BRCA testing in UK clinical practice. Additionally, broader benefits of HRD testing, including enhanced understanding of genetic cancer drivers for prognosis and optimal management, were not factored into the model. Given the high level of uncertainty in cost-effectiveness estimates, the committee agreed that an acceptable Incremental Cost-Effectiveness Ratio (ICER) would be below £20,000 per Quality-Adjusted Life Year (QALY) gained.


January 19, 2023

NICE's draft guidance recommended Talzenna for advanced or metastatic breast cancer

In the recently published final draft guidance, NICE endorses the use of talazoparib for a specific category of locally advanced or metastatic breast cancer.

This recommendation represents a reversal of NICE's initial draft decision, which did not favour the use of talazoparib for adults with BRCA 1 or 2 mutated HER2-negative locally advanced or metastatic breast cancer post-chemotherapy.

Talazoparib is positioned as an alternative to chemotherapy for this particular patient population. Currently, the NHS lacks targeted treatments for this advanced type of breast cancer, with limited alternative options available. Standard treatments include chemotherapy, primarily taxanes, along with best supportive care.

Clinical trial evidence indicates that talazoparib extends the progression-free survival compared to chemotherapy, although there was no observed difference in overall survival. Classified as a PARP inhibitor, talazoparib operates by reducing or impeding the growth of specific types of cancer cells.


February 07, 2024

Lynparza is recommended for hormone-relapsed metastatic prostate cancer adult patients who are unsuitable for chemotherapy

Lynparza (olaparib) combined with abiraterone and prednisone or prednisolone has been endorsed as a viable option for untreated hormone-relapsed metastatic prostate cancer in adults who are unsuitable for chemotherapy, within its authorized usage. Its recommendation is contingent upon the company's compliance with commercial agreements.

The clinical validation for olaparib with abiraterone stems from a rigorous double-blind phase 3 randomized controlled trial known as PROpel. This trial compared olaparib (300 mg) alongside abiraterone (1,000 mg) and prednisone or prednisolone (5 mg) against a placebo combined with abiraterone (1,000 mg) and prednisone or prednisolone (5 mg) in adult patients yet to receive treatment for hormone-relapsed metastatic prostate cancer. NICE concluded that olaparib with abiraterone demonstrates improvements in overall survival (OS) and progression-free survival (PFS) compared to abiraterone alone.

The company provided a 3-state partitioned survival model to assess the cost-effectiveness of olaparib with abiraterone relative to enzalutamide and abiraterone. These states encompassed progression-free, post-progression, and mortality phases.

Utilizing an independent parametric curve fitting approach, the company's economic model utilized generalised gamma curves to forecast outcomes for the olaparib with abiraterone, enzalutamide, and abiraterone treatment arms. After thorough consideration of available data and perspectives, the committee determined that the generalised gamma curve was the most suitable for projecting OS.

NICE agreed that an acceptable Incremental Cost-Effectiveness Ratio (ICER) would lean towards the higher spectrum of the usual range considered cost-effective for National Health Service (NHS) resource utilization (£20,000 to £30,000 per Quality-Adjusted Life Year [QALY] gained).

Ultimately, the committee deemed its favoured cost-effectiveness evaluations to be within the bounds of acceptable NHS resource allocation. Thus, it recommended Lynparza (olaparib) with abiraterone and prednisone or prednisolone for untreated hormone-relapsed metastatic prostate cancer in adults not suited for chemotherapy based on clinical indications.


February 07, 2024

BMS' Opdualag recommended for initial management of advanced melanoma

Nivolumab–relatlimab has been endorsed as a potential treatment for untreated advanced melanoma in individuals aged 12 and above, on the condition that treatment ceases after two years or earlier if the cancer progresses. Marketed under the name Opdualag by Bristol Myers Squibb, Nivolumab–relatlimab is approved for use in adults and adolescents 12 years and older for the initial management of advanced melanoma.

The comparative efficacy data was derived from the RELATIVITY‑047 trial, where nivolumab–relatlimab (n=355) was compared with nivolumab monotherapy. Investigator-assessed Progression-Free Survival (PFS) served as an exploratory endpoint. Median PFS, as assessed by Blinded Independent Central Review (BICR), was 10.2 months in the nivolumab–relatlimab arm versus 4.6 months in the nivolumab arm. In terms of overall survival, as of the October 2022 data cutoff, median overall survival was not reached in the nivolumab–relatlimab arm, while it was 33.2 months in the nivolumab arm. Based on these findings, the committee determined that nivolumab–relatlimab exhibited superior efficacy compared to nivolumab.

The company's submission included a partitioned survival model with a 40-year outlook, comprising three health states: progression-free, disease progression, and mortality. Given the uncertainty surrounding overall survival, the committee concluded that an acceptable Incremental Cost-Effectiveness Ratio (ICER) would lie at the lower end of the typical range considered cost-effective.

While the cost-effectiveness estimates in comparison to nivolumab leaned towards the higher end, it's worth noting that pembrolizumab is the preferred monotherapy option as per section 3.1. Moreover, nivolumab is less commonly utilized within the National Health Service (NHS). Consequently, the committee deemed nivolumab–relatlimab suitable for treating advanced melanoma in individuals aged 12 and above.


February 07, 2024

NICE agreed to recommend belumosudil for GVHD

Belumosudil has garnered recommendation for managing chronic graft-versus-host disease (GVHD) in individuals aged 12 and above who have undergone two or more systemic treatments, as per its marketing authorization. This recommendation is contingent upon the company's adherence to commercial arrangements. Marketed as Rezurock by Sanofi, belumosudil mesilate is indicated for patients aged 12 years and older grappling with chronic GVHD and who have undergone at least two prior lines of systemic therapy.

Graft-versus-host disease (GVHD) typically arises following an allogeneic hematopoietic stem cell transplant (HSCT), where transplanted white T-cells target the recipient's own cells. Chronic GVHD typically manifests later post-HSCT, often within the first year when immunosuppressive medications are tapered.

The clinical evidence supporting belumosudil is derived from two trials: ROCKstar (KD025‑213) and KD025‑208. The ROCKstar study is an ongoing phase 2 trial, randomized and open-label across multiple centers, comparing the efficacy of belumosudil 200 mg once daily versus twice daily.

The primary endpoint in both ROCKstar and KD025‑208 was the best overall response rate, defined as the proportion of patients achieving complete or partial response. Data from both trials were pooled and analyzed for individuals who had undergone two or more prior lines of therapy. The overall response rate for the combined 200-mg dose was estimated at 73.1%, with 69.9% experiencing a partial response and a minority achieving a complete response (3.4%). Based on pooled efficacy data up to 3 years, the committee observed a consistent effect across all doses of belumosudil.

The company's modeling approach utilized a partitioned survival model with three states (failure-free, failure progressed, and death) to evaluate the cost-effectiveness of belumosudil compared to the best available therapy for chronic GVHD post-two lines of systemic therapy. While the committee acknowledged potential limitations with this model, they deemed it acceptable for decision-making provided other modeling assumptions were adequately addressed.

Despite considerable uncertainty surrounding cost-effectiveness estimates, the committee concluded that the most plausible estimates fell within the range typically considered cost-effective for NHS resource allocation. Consequently, belumosudil receives a recommendation for treating chronic GVHD after two or more lines of systemic therapy.


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