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

Updated: Jun 7


Sl No

Brand

Indication

Outcome

1

Kanuma

LAL deficiency

Recommended

2

Imfinzi

Biliary tract cancer

Recommended

3

Olaparib plus bevacizumab

Ovarian, fallopian tube, or primary peritoneal cancer

Recommended

4

Talzenna

Metastatic breast cancer

Recommended

5

Lynparza

Hormone relapsed metastatic prostate cancer

Recommended

6

Opdualag

Melanoma

Recommended

7

Belumosudil

Graft-versus host disease

Recommended

8

Litfulo

Alopecia

Recommended

9

Talzenna

Breast cancer

Recommended

10

Enhertu

Breast cancer

Not recommended

11

Jemperli

Uterine cancer

Recommended

12

Velsipity

Ulcerative Colitis

13

Jemperli

Endometrial cancer

Recommended (CDF)

14

Aquipta

Migraine

Recommended

15

Kymriah

ALL

Recommended

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.


 

NICE Recommends New Treatment for Severe Alopecia, Potentially Benefiting Up to 14,000 People

A novel one-a-day tablet has been endorsed by the National Institute for Health and Care Excellence (NICE) as the first-line treatment option for individuals grappling with severe hair loss due to alopecia areata.

Ritlecitinib (Litfulo), manufactured by Pfizer, is now recommended by NICE for addressing severe alopecia areata among individuals aged 12 and above.


Administered as a convenient daily pill at home, the treatment operates by diminishing the enzymes responsible for inflammation and subsequent hair loss at the follicle. This marks a significant milestone as it is the inaugural treatment for severe alopecia areata to receive NICE endorsement for NHS utilization.


After public consultation and Pfizer's provision of additional information coupled with an enhanced discount on its price, the treatment is now regarded as both clinically effective and cost-effective for NHS integration.


Clinical trial data indicate that ritlecitinib surpasses placebo to enhance hair regrowth, with response rates steadily improving for individuals undergoing ritlecitinib treatment for up to two years.


Alopecia areata, an autoimmune condition, prompts the body's immune system to assault the hair follicles, resulting in hair loss. In severe cases, hair loss extends beyond the scalp, affecting various body parts including eyelashes, eyebrows, nasal hair, and skin, rendering individuals more susceptible to infections and impairing their ability to regulate body temperature.


While hair loss in alopecia areata can occur suddenly and affect individuals of all ages, the hair follicles remain intact albeit dormant, facilitating the possibility of regrowth. The extent and duration of hair loss can vary significantly from one person to another.

The recommended dosage entails the ingestion of a 50mg capsule once daily.


Additionally, the company has established a confidential commercial arrangement incorporating a simple patient access scheme, facilitating NHS access to ritlecitinib at a discounted rate.


 

Talzenna was recommended for locally advanced or metastatic breast cancer

Talazoparib (marketed as Talzenna by Pfizer) is approved as a standalone therapy for adult patients diagnosed with locally advanced or metastatic breast cancer, specifically those harbouring germline BRCA1/2 mutations and HER2-negative status.


Within its authorized use, Talazoparib is recommended for the management of HER2-negative locally advanced or metastatic breast cancer in adults with germline BRCA1 or BRCA2 mutations, provided they have undergone:

  1. Prior treatment with an anthracycline or a taxane, or both, unless these options are deemed unsuitable, and

  2. Endocrine therapy if they have hormone receptor (HR)-positive breast cancer unless this is not suitable


The clinical data supporting this indication are derived from EMBRACA, a phase 3 randomized controlled trial involving 431 participants conducted globally, with a subset from the UK. Progression-free survival was the primary endpoint of EMBRACA. Findings revealed that at a median follow-up of 11.2 months as of the September 2017 data cut-off, the median progression-free survival was 8.6 months with talazoparib compared to 5.6 months with the physician's choice of treatment in the overall study population.


While overall survival was a secondary endpoint, EMBRACA was adequately powered to detect a significant difference in overall survival. As of the September 2019 data cut-off, median overall survival was 19.3 months in the talazoparib arm at a median follow-up of 44.9 months, and 19.5 months in the physician's choice of treatment arm at a median follow-up of 36.8 months.


The company employed a cohort partitioned-survival model with three states (progression-free, post-progression survival, and death) to compare talazoparib with physicians' choice of treatment in individuals with HER2-negative advanced breast cancer harbouring germline BRCA mutations.


After determining that talazoparib represents a cost-effective use of NHS resources for the treatment of HER2-negative locally advanced or metastatic breast cancer with germline BRCA mutations, the committee recommended its routine use within the NHS.


 

NICE said NO to Enhertu for patients with HER2-low breast cancer


NICE issues final draft guidance concerning Enhertu post-commercial discussions conclude sans an agreed price rendering it conducive for NHS resource utilization.

NICE has released ultimate draft counsel advising against the utilization of Enhertu (alternatively known as trastuzumab deruxtecan, developed by Daiichi Sankyo) in the management of advanced HER2-low breast cancer in adult patients.


This verdict ensued subsequent to negotiations between NHS England and the manufacturer to forge a commercial agreement facilitating the affordability of Enhertu for the NHS. The evaluation of Enhertu by NICE had been on hold since December 2023 while these discussions unfolded.


This development transpired subsequent to NICE's preliminary guidance in September, which refrained from recommending Enhertu for individuals afflicted with this subtype of breast cancer post-chemotherapy. This decision stemmed from uncertainties in the evidence, leading to cost-effectiveness projections surpassing the threshold NICE deems appropriate for NHS resource allocation.

Approximately 1,000 individuals could have been eligible for trastuzumab deruxtecan therapy had NICE endorsed its utilization.


All registered stakeholders, inclusive of the manufacturer and patient advocacy groups, retain the option to contest NICE's final draft recommendations.


 

March 05, 2024

Jemperli was recommended by NICE for uterine cancer

A novel therapeutic approach accompanies chemotherapy, endorsed for specific forms of advanced or recurrent uterine cancer in the latest NICE guidance unveiled today.

Dostarlimab (also recognized as Jemperli and produced by GSK) is made accessible to 540 adults. Functioning as a monoclonal antibody, Dostarlimab aids the immune system in combatting cancer. Administered intravenously over a 30-minute period within a hospital setting.


Clinical trial data demonstrates its efficacy alongside platinum-based chemotherapy, enhancing life expectancy and delaying disease progression. Nonetheless, due to the limited duration of the study, the long-term advantages remain uncertain.


Consequently, dostarlimab is earmarked for inclusion in the Cancer Drugs Fund (CDF). This designation ensures its availability within the NHS for treating this category of uterine cancer while further evidence accrues.


Uterine cancer encompasses two primary types (endometrial cancer and uterine sarcoma), with approximately 9,000 fresh diagnoses recorded annually in the UK. Endometrial cancer constitutes the most prevalent form, with roughly 23% classified as a subtype featuring high microsatellite instability or mismatch repair deficiency. Dostarlimab garners recommendation for these subtypes characterized by advanced stage or recurrence post-prior treatment.


The impact of advanced or recurrent endometrial cancer, indicating the spread beyond the uterus or recurrence post-treatment, significantly affects life expectancy and quality of life. This marks the fourth endorsed treatment option for endometrial cancer, either for routine NHS provision or CDF utilization, since the inception of NICE in 1999.


 

March 13, 2024

Dupixent was not recommended for prurigo nodularis

Dupilumab's use in addressing moderate to severe prurigo nodularis among adults, within its authorized marketing scope, isn't advocated when systemic intervention is deemed appropriate.


Prurigo nodularis, a rare and persistent dermatological condition, manifests through dense, hardened nodules on the skin's surface. Though its etiology remains elusive, it correlates with abnormal levels of nerve fibers, neuropeptides, and cytokine-producing immune cells.


Standardized treatment protocols for prurigo nodularis are lacking, with therapeutic strategies often adopting a progressive escalation model. This approach entails introducing increasingly potent therapies, albeit potentially accompanied by severe side effects, as the condition exacerbates. Initial interventions typically involve emollients, topical corticosteroids, and calcineurin inhibitors. Subsequent options encompass phototherapy, oral corticosteroids, and antihistamines, with considerations extending to immunosuppressants, antidepressants, pregabalin, and gabapentin. In severe cases, neurokinin-1 receptor antagonists, mu-opioid antagonists, and thalidomide may be contemplated.


Clinical data primarily stem from two phase 3 randomized trials, PRIME and PRIME2, comprising adults with inadequately controlled prurigo nodularis despite prior topical therapies. Participants were randomized into either the dupilumab or placebo cohorts, alongside best supportive care, across a 24-week treatment period with 12 weeks of subsequent observation. The primary endpoint focused on a 4-point or greater reduction in the Worst Itch-Numerical Rating Scale (WI-NRS), supplemented by quality-of-life assessments and severity grading using the Investigator's Global Assessment for Prurigo Nodularis-Stage (IGA PN-S) tool.


Pooling data from both trials revealed a notable uptick in response rates among dupilumab recipients vis-à-vis best supportive care. The committee acknowledged dupilumab's efficacy in eliciting a favorable response.


The company's economic analyses incorporated decision trees and Markov models, yet all cost-effectiveness estimates surpassed NICE's acceptable threshold. Moreover, substantial uncertainties encircled both economic modeling and clinical data, precluding a recommendation for dupilumab's use in moderate to severe prurigo nodularis.


 

Pfizer's Velsipity was recommended for moderate to severe ulcerative colitis

Etrasimod has gained endorsement within its approved marketing parameters as a viable choice for managing moderately to severely active ulcerative colitis in individuals aged 16 years and older under the following circumstances:

  • Conventional or biological therapies are not viable due to intolerance.

  • Inadequate response or loss of efficacy to prior treatments has been observed.


Etrasimod, marketed as Velsipity by Pfizer, is specifically designated for individuals aged 16 and above grappling with moderately to severely active ulcerative colitis, provided they have demonstrated inadequate response, loss of efficacy, or intolerance to either conventional therapeutic modalities or biological agents.


 

Tepkinly was recommended for treating adult patients with relapsed or refractory diffuse B-cell lymphoma

Epcoritamab, marketed as Tepkinly by AbbVie, is designated for adult patients grappling with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) subsequent to undergoing two or more lines of systemic therapy.


Recommendation for epcoritamab's utilization in treating relapsed or refractory DLBCL among adults post two or more systemic treatments is conditional upon prior administration of polatuzumab vedotin.


In cases where polatuzumab vedotin is either contraindicated or not well-tolerated.

Clinical data substantiating epcoritamab's efficacy emanates from the ongoing single-arm phase 1 to 2 trial expansion segment known as EPCORE TM NHL-1. This trial encompasses three cohorts, one of which includes adults with DLBCL and other forms of large B-cell lymphoma (LBCL) who experienced relapse or inadequate response following at least two prior systemic treatments. The trial, however, is limited to individuals with ECOG scores ranging from 0 to 2 and those ineligible for autologous stem cell transplants or for whom such transplants failed. Although this doesn't fully encompass the decision-making scenario, clinical experts suggest that most candidates for epcoritamab in clinical practice fall within the ECOG score range of 0 to 2. Nonetheless, they argue against excluding those with higher ECOG scores, estimating that about 5% to 10% of epcoritamab-eligible individuals may have an ECOG score of 3. Notably, most epcoritamab-eligible individuals would be ineligible for autologous stem cell transplants. Encouragingly, a significant proportion of trial participants achieved complete disease remission with epcoritamab, although exact figures remain confidential.


The company employed a partitioned survival model to gauge the cost-effectiveness of epcoritamab, incorporating three health states: progression-free, progressed disease, and death. The model, structured upon OS and PFS curves derived from MAICs, garnered acceptance from the committee for decision-making purposes.


Given the analogous clinical effectiveness, the committee solely considered the cost differential between epcoritamab and polatuzumab-BR, with epcoritamab proving considerably pricier. The deterministic ICER, factoring in the committee's preferred assumptions, positioned epcoritamab favorably compared to R-based CIT within the cost-effectiveness range deemed acceptable for NHS resource utilization. Although epcoritamab incurred lower costs yet yielded higher QALYs than axicabtagene ciloleucel, the committee surmised that R-based CIT or axicabtagene ciloleucel would be preferred subsequent to polatuzumab vedotin administration or in cases where the latter is contraindicated or intolerable.


Consequently, epcoritamab's recommendation for NHS deployment in treating relapsed or refractory DLBCL in adults, post two or more systemic treatments, is contingent upon prior exposure to polatuzumab vedotin or contraindication/intolerance to the same.


 

25,000 individuals poised for improvement post NICE endorsement of fresh ulcerative colitis drug, Velsipity

Novel once-daily tablet suggested simultaneously with medication obtaining approval from MHRA


NICE’s ultimate counsel on etrasimod (also recognized as Velsipity and produced by Pfizer) is unveiled on the same date that the medication was officially endorsed by the Medicines and Healthcare Products Regulatory Agency (MHRA).


Slightly over 25,000 individuals in England are qualified to acquire the innovative treatment, which underwent assessment using a more straightforward technology evaluation process. Consequently, the ultimate counsel was accessible up to eight weeks earlier than would have been expected under the regular procedure.


Etrasimod is proposed for individuals aged 16 and older grappling with moderately to severely active ulcerative colitis who have exhibited an unsatisfactory reaction, lost response, or were intolerant to either traditional therapy or biological treatment.

Ulcerative colitis, a chronic ailment impacting the colon and rectum, can induce recurrent diarrhea, arthritis, and osteoporosis.


The remedy aids in diminishing inflammation in the colon by managing the concentration of immune cells in the bloodstream.


Evidence from clinical trials indicates that etrasimod surpasses placebo in efficacy for managing moderately to severely active ulcerative colitis. Indirect comparisons propose it may outperform adalimumab (an immunotherapy treatment) and could be comparably effective to other customary therapies for moderately to severely active ulcerative colitis. It is approximated that about 300,000 individuals throughout the UK have been diagnosed with ulcerative colitis. While the ailment can manifest at any age, it is frequently diagnosed in individuals aged 15 to 25.


The corporation has secured a confidential business agreement ensuring etrasimod's availability to the NHS at a discounted rate.


 

April 03, 2024

GSK's Jemperli was recommended under CDF for advanced or recurrent endometrial cancer

Dostarlimab combined with platinum-based chemotherapy is suggested, along with managed access as an alternative, for the management of primary advanced or recurring endometrial cancer in adults exhibiting high microsatellite instability or mismatch repair deficiency and who are suitable candidates for systemic therapy. Adherence to the terms outlined in the managed access agreement for dostarlimab is essential for this recommendation to be implemented.


It's important to note that this recommendation does not seek to alter the ongoing dostarlimab treatment with platinum-based chemotherapy initiated within the NHS prior to the release of this guidance. Dostarlimab (marketed as Jemperli by GSK) is specifically indicated for use "in combination with platinum-containing chemotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/ microsatellite instability-high (MSI-H) primary advanced or recurrent endometrial cancer (EC) and who are candidates for systemic therapy".


The conventional approach to managing recurrent or advanced endometrial cancer involves platinum-containing chemotherapy (PCC), typically carboplatin plus paclitaxel. Immunotherapy may be offered to individuals whose cancer progresses following chemotherapy. Pembrolizumab with lenvatinib is an available option for individuals previously treated for endometrial cancer.


The clinical basis for this recommendation stems from an interim analysis of RUBY-1, a phase 3, randomized, double-blind, multicenter, placebo-controlled trial involving individuals with advanced or recurrent endometrial cancer. The data revealed a significant improvement in progression-free survival (PFS) with dostarlimab compared to placebo. At the time of data analysis (with PFS data maturity at 56%), dostarlimab demonstrated a 72% reduction in the risk of disease progression or death compared to placebo. Overall survival (OS) was analyzed as a prespecified subgroup in the dMMR/MSI-H population, showing a 70% reduction in the risk of death with dostarlimab compared to placebo at OS maturity of 26%.


To evaluate the cost-effectiveness of incorporating dostarlimab into PCC, the company employed a partitioned survival model with three health states: PFS, disease progression, and death. Data for health-state utilities and baseline characteristics were derived from the dMMR/MSI-H population in RUBY-1. Additionally, the committee considered the substantial unmet clinical need, the sustained benefits observed in dMMR/MSI-H populations, and the data from RUBY-1 available thus far. Given the prevailing uncertainty, the committee determined that the maximum acceptable incremental cost-effectiveness ratio (ICER) would be £25,000 per quality-adjusted life year (QALY) gained, acknowledging that this threshold might evolve with additional data.


Recognizing that further data collection within the Cancer Drugs Fund could alleviate some of the uncertainty surrounding the company's estimations, the committee concluded that dostarlimab holds potential for cost-effectiveness under their preferred assumptions. Consequently, dostarlimab qualifies for consideration for inclusion in the Cancer Drugs Fund.


 

April 10. 2024

Cabometyx plus Opdivo recommended for treating advanced renal cell carcinoma 

Cabometyx (cabozantinib, Ipsen) along with Opdivo (nivolumab, from Bristol Myers Squibb) is specified for use as the initial treatment for advanced renal cell carcinoma in adults.


Cabozantinib in combination with nivolumab is suggested as a viable choice for treating advanced renal cell carcinoma in adults who have not undergone prior treatment, provided that:

  • Their condition falls within the intermediate or poor risk categories, as outlined in the criteria established by the International Metastatic Renal Cell Carcinoma Database Consortium.

  • If nivolumab combined with ipilimumab or lenvatinib combined with pembrolizumab would typically be recommended.

  • Cabozantinib and nivolumab are made available by the respective companies based on their commercial agreements.


The primary evidence supporting the use of cabozantinib alongside nivolumab in renal cell carcinoma comes from the CheckMate 9ER trial, a study that was single-blind and randomized. This trial compared the efficacy of cabozantinib plus nivolumab with that of sunitinib. In total, 651 individuals across all risk categories participated in the trial, with a final median follow-up period of 44 months. The combination of cabozantinib and nivolumab demonstrated a median overall survival of 49.5 months, as opposed to 35.5 months with sunitinib. Median progression-free survival was 16.6 months compared to 8.4 months with sunitinib.


The committee evaluated the modelling methodology employed by the EAG (Economic Analysis Group). It concluded that the overall approach was suitable and could be utilized in decision-making processes.

Due to confidentiality surrounding commercial agreements involving cabozantinib, nivolumab, and other comparator drugs, the results of cost-effectiveness analyses cannot be disclosed here. However, the committee took into account the cost-effectiveness outcomes based on the EAG's baseline scenario and the preferred assumptions of the respective companies.


NICE affirmed that the combination of cabozantinib and nivolumab constitutes an effective treatment option for renal cell carcinoma. Feedback from both patients and clinical experts emphasized the need for additional treatment alternatives. While the estimated cost-effectiveness for treating all-risk and favourable-risk cancers exceeded what is deemed acceptable by NICE (National Institute for Health and Care Excellence) for all comparator drugs, the estimates for treating intermediate- and poor-risk cancers (with nivolumab plus ipilimumab or lenvatinib plus pembrolizumab as comparators) were within acceptable limits.


Consequently, cabozantinib plus nivolumab is recommended for untreated advanced renal cell carcinoma in adults categorized as intermediate- or poor-risk, when nivolumab plus ipilimumab or lenvatinib plus pembrolizumab would otherwise be considered.


 

April 11, 2024

NICE recommended AbbVie's Aquipta for migraine

In a recent publication of final guidance, NICE announced a development in migraine treatment, offering expanded options to approximately 170,000 individuals across England.


The recommended treatment, atogepant, produced by AbbVie under the brand name Aquipta, represents a pioneering oral medication designed to prevent both chronic and episodic migraines. NICE's endorsement paves the way for eligible individuals to access this innovative therapy through the NHS.


Atogepant emerges as a viable option for adults experiencing chronic and episodic migraines, characterized by a minimum of 4 migraine days per month and a history of unsuccessful treatment with at least 3 prior preventive measures.

Migraines, impacting an estimated 4.5 million individuals in England, exert a substantial toll on daily functioning while also imposing significant strain on healthcare resources and the broader economy.


Chronic migraine manifests as a condition where sufferers endure a minimum of 15 headache days monthly, with at least 8 exhibiting migraine symptoms. Conversely, episodic migraine entails fewer than 15 headache days per month.


Administered orally as a tablet, atogepant operates by inhibiting the calcitonin gene-related peptide (CGRP) receptor within the body. CGRP, a protein situated in the sensory nerves of the head and neck, triggers vasodilation, leading to inflammation and the onset of migraine-related pain.


 

NICE recommended Kymriah for ALL relapsed after a stem-cell transplant, or relapsed for a second or later time

NICE's recent recommendation heralds a breakthrough in blood cancer treatment, promising significant benefits for children and young adults. Specifically, the endorsement pertains to personalized immunotherapy, a cutting-edge approach that harnesses the body's own immune cells to combat leukemia.


Kymriah emerges as the focal point of NICE's final draft guidance. This groundbreaking therapy is proposed for children and young adults up to the age of 25 afflicted with B-cell acute lymphoblastic leukemia (ALL). Eligibility extends to those who have not responded to conventional treatments, experienced relapse following a stem-cell transplant, or encountered recurrent relapses. Although Kymriah has been accessible through the NHS's Cancer Drugs Fund since December 2018, the latest guidance advocates for its routine integration into healthcare protocols.


ALL, characterized by the rapid proliferation of certain white blood cells, poses a grave threat to the immune system. The unchecked accumulation of malignant, immature white blood cells, known as lymphoblasts, within the bone marrow disrupts normal hematopoiesis, leading to deficiencies in white blood cells, red blood cells, and platelets.


Individuals grappling with ALL endure a spectrum of debilitating symptoms, including heightened susceptibility to infections, weight loss, fatigue, bruising, and hemorrhaging. Left unchecked, the disease can prove fatal, severely impairing both patients and their caregivers' ability to carry out daily activities or pursue education and employment opportunities.


Kymriah represents a paradigm shift in cancer treatment, classified as a CAR-T (chimeric antigen receptor T-cell) therapy. This innovative approach involves the extraction and modification of a patient's immune cells, empowering them to recognize and eradicate cancerous cells. Administered via a singular infusion into the bloodstream, this therapy offers a potent weapon against leukemia.


Approximately 40 children and young adults in England meet the criteria for this groundbreaking treatment, a group that constitutes a fraction of the roughly 300 individuals aged 25 and under diagnosed annually with acute lymphoblastic leukemia.

Extensive data, including real-world evidence from NHS utilization and clinical trial findings, underscores Kymriah's efficacy. Patients receiving this therapy exhibited prolonged survival rates, with fewer instances of disease recurrence or progression compared to conventional treatments. Notably, clinical trials revealed a median overall survival of 48 months with Tisagenlecleucel, a stark contrast to the median survival of 7.5 months with blinatumomab and three months with salvage chemotherapy, two established treatments.


With NICE's validation of its effectiveness, Kymriah is slated for routine provision within the NHS, obviating the need for inclusion in the Cancer Drugs Fund. Priced at £282,000 per infusion, the therapy benefits from a commercial arrangement, ensuring accessibility through discounted rates negotiated with the manufacturer.


 

NICE recommended Finlee plus Spexotras combination for children and adoloscents ith glioma

NICE recommended a treatment combination for children and adolescents grappling with an aggressive type of brain tumor, marking a significant stride in care. The recommended therapy involves dabrafenib, branded as Finlee, in conjunction with trametinib, known as Spexotras, both manufactured by Novartis.


This targeted medication blend is poised to enhance both the duration and quality of life for patients, offering a notable departure from conventional hospital-centered treatments.


Characterized as a 'step-change in care,' the dabrafenib-trametinib duo receives a nod from NICE for addressing BRAF V600E mutation-positive glioma in its final draft guidance. Gliomas, prevalent among children and young individuals, arise from glial cells supporting the nerve cells of the brain and spinal cord. The presence of the BRAF gene mutation prompts aberrant protein production, culminating in brain tumor formation.


Gliomas manifest as either low-grade glioma (LGG), typified by slow or non-progressive tumor growth, or high-grade glioma (HGG), marked by aggressive growth and often fatal outcomes. Current treatment modalities encompass surgical intervention, radiotherapy, chemotherapy, and palliative care. Nonetheless, patient advocates emphasize the disruptive impact of glioma treatment on children's education, social interactions, and emotional well-being, citing adverse effects like weight loss, seizures, and headaches associated with chemotherapy.


Approximately 30 children in England stand to benefit from dabrafenib with trametinib, administered as tablets twice daily and an oral solution once daily, respectively. Functioning as 'cancer growth inhibitors,' these medications target BRAF gene-affected proteins, impeding tumor progression. Notably, the drug combination has already demonstrated efficacy across various cancer types.


Eligible recipients include LGG patients eligible for systemic therapy and HGG patients who have undergone prior radiation or chemotherapy. Clinical trials underscore the treatment's efficacy, indicating a halt in tumor growth for over two years on average in LGG cases—tripling the duration compared to existing therapies.


To facilitate NHS access, the pharmaceutical company has established a confidential commercial arrangement with a discounted patient access scheme for each medication. The development of this therapeutic approach received backing from the NIHR Clinical Research Network, underscoring its promising prospects in addressing a critical medical need.


 

May 03, 2024

Revised Pricing Facilitates NICE's Endorsement of Oxbryta for Sickle Cell Treatment

Approximately 4,000 individuals diagnosed with sickle cell condition stand to gain from a fresh therapeutic approach endorsed by NICE in its ultimate draft advice.

Voxelotor addresses the issue of haemolytic anaemia stemming from sickle cell disorder (SCD) and is put forth for adoption within the NHS following a markdown in its price.


This pricing adjustment has alleviated some of the ambiguities highlighted in the data by the autonomous evaluation committee in its preceding preliminary guidance, allowing the therapy to be advocated as a prudent employment of NHS assets.

NICE's ultimate draft counsel (issued Friday 3 May 2024) suggests voxelotor (alternatively known as Oxbryta and manufactured by Pfizer) either alone or in conjunction with another SCD treatment named hydroxycarbamide.


Voxelotor's financing will commence promptly via NHS England's Innovative Medicines Fund, potentially benefitting around 4,000 individuals grappling with SCD in England right away.


The committee reached a consensus that, given the significant adversities faced by individuals with sickle cell disorder, a higher tolerance for uncertainty in the efficacy projections underpinning voxelotor's economic modeling could be justified, permitting a deviation from the customary cost-effectiveness estimates.

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