top of page


Updated: 6 days ago

Camzyos (mavacamten) has been recommended as a viable option for treating symptomatic obstructive hypertrophic cardiomyopathy (oHCM) in adults who fall within New York Heart Association (NYHA) class 2 to 3. This recommendation is contingent on Camzyos being utilized as an additional component to individually optimized standard care, which should include beta-blockers, non-dihydropyridine calcium-channel blockers, or disopyramide unless contraindicated.

Camzyos is specifically indicated for managing symptomatic obstructive hypertrophic cardiomyopathy in adult patients classified as NYHA class II-III. When used alongside standard care versus standard care alone, the supporting evidence for the effectiveness and safety of mavacamten primarily derives from the pivotal clinical trial known as EXPLORER-HCM.

EXPLORER-HCM was a phase 3, double-blind, randomized, placebo-controlled, multicenter study involving 251 individuals afflicted with obstructive HCM. Longer-term safety data were obtained from the EXPLORER-LTE cohort, comprising 231 participants who had previously enrolled in EXPLORER-HCM and continued into the long-term extension study MAVA-LTE.

The primary endpoint established in EXPLORER-HCM encompassed a composite measure. This endpoint was designed to incorporate a physiological assessment of exercise capacity determined by peak oxygen consumption and a physician-assessed component that evaluated symptoms based on NYHA class.

EXPLORER-HCM effectively met its primary endpoint, with mavacamten demonstrating clinically meaningful enhancements in NYHA class and peak oxygen consumption. A more significant proportion of individuals in the mavacamten group reached the primary endpoint compared to those in the placebo group, with percentages of 37% and 17%, respectively.

When considering the committee's preferred assumptions and the company's revised commercial arrangement, the Incremental Cost-Effectiveness Ratio (ICER) for mavacamten used alongside standard care versus standard care alone fell within the range that NICE deemed a cost-effective use of NHS resources.

Retsevmo (selpercatinib) is recommended for treating RET fusion-positive advanced non-small-cell lung cancer (NSCLC) in adults who have not previously undergone treatment and is recommended under managed access.

The established protocol for addressing untreated RET fusion-positive advanced NSCLC encompasses two primary options: pemetrexed combined with platinum-based chemotherapy and pembrolizumab combined with pemetrexed and platinum-based chemotherapy. Retsevmo represents an alternative avenue for treatment.

Based on findings from clinical trials, Retsevmo exhibits potential effectiveness in the context of untreated RET fusion-positive advanced NSCLC. However, the current state of evidence is somewhat uncertain due to the absence of direct comparative analysis and the ongoing nature of the trial itself. Eli Lilly submitted LIBRETTO-001 trial results; objective response rate (ORR) was primary endpoint; progression-free survival and overall survival (OS) were secondary endpoints. The overall response rate was 84%, and the progression-free survival was 22 months.

Given the ambiguity surrounding the clinical effectiveness data, the estimates regarding cost-effectiveness remain similarly uncertain. The anticipated figures for cost-effectiveness surpass the threshold deemed cost-effective by NICE, even when the severity of the condition and its impact on life quality and duration are taken into account. Consequently, Retsevmo cannot be advocated for routine utilization.

Retsevmo's cost-effectiveness could improve if supplementary evidence substantiates its ability to extend individuals' lifespans during treatment. A more conclusive understanding of survival outcomes can potentially emerge from direct comparisons facilitated by the ongoing trial. As a result, managed access is recommended for the employment of Retsevmo.

Lynparza (olaparib) was recommended as a maintenance treatment option for adults with relapsed, platinum-sensitive ovarian, fallopian tube, or peritoneal cancer of high-grade epithelial nature.

This recommendation applies to patients whose cancer has shown a positive response to platinum-based chemotherapy and meets the following criteria: they have a BRCA1 or BRCA2 mutation and have undergone at least two rounds of platinum-based chemotherapy.

During the evaluation process, it was noted that when olaparib was initially included in the Cancer Drugs Fund, comprehensive survival data from the SOLO2 clinical trial, which compared olaparib with placebo in individuals with a BRCA mutation after platinum-based chemotherapy, was not fully available. Instead, data from Study 19 was used, but caution was advised when considering its relevance.

The committee acknowledged that the overall survival data from the SOLO2 trial is now complete and more applicable to the target population of this assessment. Therefore, the committee carefully reviewed the baseline characteristics of the participants in the SOLO2 trial.

Based on unadjusted data from the SOLO2 trial, it was observed that olaparib significantly prolongs the time to disease progression after the second round of chemotherapy and improves median overall survival. The clinical expert mentioned that approximately 20% of patients responded positively to the treatment.

In the evaluation process, the company employed a three-state partitioned survival model, similar to TA620, incorporating data from SOLO2 instead of Study 19. Additionally, the time horizon was extended from 30 to 50 years, aligning with the committee's preference. Consequently, the committee deemed that the maximum acceptable ICER (incremental cost-effectiveness ratio) would fall towards the higher end of the range typically considered cost-effective for utilizing NHS resources (between £20,000 and £30,000 per QALY gained). The ICER figures are confidential due to commercial agreements concerning subsequent treatments in the treatment pathway, so they cannot be disclosed. However, after accounting for confidential discounts, the corrected company base-case ICER, determined by the ERG (Evidence Review Group), fell within the cost-effective range. Therefore, the committee recommended routine use of olaparib following two courses of platinum-based chemotherapy within the NHS.

In conclusion, considering the ICER falls within NICE's definition of cost-effective utilization of NHS resources, olaparib is recommended as a maintenance treatment for adults with relapsed, platinum-sensitive, high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who have responded to two courses of platinum-based chemotherapy and possess a BRCA1 or BRCA2 mutation. Thus, olaparib is advised for routine use within the NHS.

Pfizer's Vydura (rimegepant) is prescribed to prevent episodic migraines in adults experiencing at least four monthly migraine attacks. NICE recommends Rimegepant as a preventative option if at least three other failed treatments. If the frequency of migraine attacks does not decrease by at least 50% after 12 weeks of treatment, rimegepant should be discontinued.

Migraines are severe headaches lasting 4 to 72 hours, often accompanied by nausea, vomiting, dizziness, and sensitivity to light, sound, and smells. They can significantly impact the quality of life and daily activities.

To evaluate rimegepant's effectiveness, a clinical trial called BHV3000‑305 was conducted, involving adults with a history of migraines. The trial demonstrated rimegepant's positive effect in reducing the number of moderate-to-severe migraine days.

NICE assessed the cost-effectiveness of rimegepant compared to other standard treatments. After considering the evidence and adjusting the price, NICE concluded that Rimegepant is cost-effective compared to two out of three common migraine treatments.

NICE recognized migraines' substantial burden on individuals' lives and noted that Rimegepant is likely to be a clinically effective treatment. It also recommended discontinuing rimegepant if the desired reduction in migraine frequency is not achieved after 12 weeks. NICE advised healthcare professionals and patients to discuss the advantages and disadvantages of available treatments, considering factors such as administration costs, dosage, and price per dose, and to opt for the least expensive suitable option.

The National Institute for Health and Care Excellence (NICE) committee has stated that more evidence is required on the clinical and cost-effectiveness of Mounjaro (tirzepatide), a new treatment option for individuals with type 2 diabetes before it can be recommended for use in the NHS. In draft guidance released on June 27, NICE did not recommend tirzepatide, which is developed by Eli Lilly, for treating type 2 diabetes in adults alongside diet and exercise.

Tirzepatide is administered once weekly through injection. The company has positioned tirzepatide as an alternative option further along the treatment pathway, offering the NHS an alternative to glucagon-like peptide 1 (GLP-1) receptor agonists such as dulaglutide, liraglutide, and semaglutide (Ozempic/Rybelsus), which are already recommended for NHS use.

Recognizing the significance of new treatment options, the independent NICE committee noted that fewer than two-thirds of adults with type 2 diabetes achieve adequate glucose control with current treatments.

Clinical trial evidence submitted to the committee demonstrated that tirzepatide, at any dose, resulted in better glucose control and lower weight than semaglutide or insulin therapy. The weight reduction was more pronounced with higher doses of tirzepatide, while the impact on glucose levels appeared less dependent on the dose. The company's network meta-analysis showed similar effects compared to all GLP-1 receptor agonists, but the certainty of these findings was uncertain.

The committee has requested additional data from the company to address uncertainties in the clinical evidence, particularly in comparison to all appropriate alternative treatments. Furthermore, the company needs to provide further analyses and clarifications regarding their submitted economic model.

Additionally, it remains unclear how accurately the model predicts the long-term health benefits, such as preventing diabetes complications, associated with tirzepatide compared to other GLP-1 receptor agonists. Therefore, whether tirzepatide offers good value for money is yet to be determined.

The committee has also requested evidence demonstrating how the company's model results compare to other economic models for diabetes.

The pricing of the pre-filled disposable injection pens for tirzepatide is commercially confidential until the final guidance is published.

Sotyktu (deucravacitinib) is recommended for adults with moderate to severe plaque psoriasis if they meet specific criteria. These criteria include having a Psoriasis Area and Severity Index (PASI) score of 10 or higher and a Dermatology Life Quality Index (DLQI) score of over 10. Additionally, the condition should not have responded to other systemic treatments such as ciclosporin, methotrexate, or phototherapy, or these options should be contraindicated or not tolerated.

The recommendation for deucravacitinib is supported by data from two double-masked, randomized controlled trials: POETYK-PSO-1 and POETYK-PSO-2. These trials included 1,686 participants and compared deucravacitinib (6 mg daily) with placebo over 16 weeks and with apremilast (30 mg twice daily) over 24 weeks. The primary endpoints were the percent of patients achieving a PASI 75 score and a static Physician Global Assessment (sPGA) score of 0 or 1. Secondary outcomes included the DLQI score and adverse events. Deucravacitinib demonstrated significantly better results than placebo and apremilast in achieving PASI 75 or sPGA 0 or 1 scores at week 16.

When considering the cost-effectiveness of deucravacitinib, various comparators were evaluated. It was found that deucravacitinib was not cost-effective compared to adalimumab, bimekizumab, or tildrakizumab, as it was considered less effective and more expensive. However, compared to apremilast, dimethyl fumarate, and most other biological treatments, deucravacitinib was deemed a cost-effective option and a practical use of NHS resources.

Considering its effectiveness, monitoring guidelines, and cost-effectiveness with other treatment options, deucravacitinib presents a valuable addition to the treatment arsenal for plaque psoriasis.


Recent Posts

See All


bottom of page