Our recent work for NICE
In the past months, iMTA and the HTA department of ESHPM have completed six projects as External Assessment Group (EAG) in relation to the appraisal of specific technologies under the NICE Single Technology Appraisal (STA) programme. Published guidance on these projects can be found below. External assessment groups are independent academic centres commissioned by NICE to technically review the company’s evidence submission and prepare a so-called EAG report. Our NICE team consists of Maiwenn Al, Venetia Qendri and Eline Krijkamp, from the HTA department of ESHPM, and Isaac Corro Ramos, from iMTA. In this period, the team was supported by three colleagues of the HTA department of ESHPM, Annemieke Leunis-van Dongen, Mehlika Toy and Marten Poley.
Relugolix–estradiol–norethisterone for treating symptoms of endometriosis
Relugolix–estradiol–norethisterone (relugolix combination therapy [CT]) can be used, within its marketing authorisation, as an option for treating symptoms of endometriosis in adults of reproductive age who have had medical or surgical treatment for endometriosis.
After pain relief and hormonal treatment, usual treatment options for endometriosis are gonadotropin-releasing hormone (GnRH) agonists and surgery. There is no cure for endometriosis, and there is an unmet need for long-term and non-invasive (nonsurgical and not injected) treatments for its symptoms. Clinical trial evidence shows that relugolix CT reduces pain compared with placebo. Relugolix CT has not been directly compared in a clinical trial with usual treatment. Indirect comparisons suggest that it is likely to reduce pelvic pain almost as well as GnRH agonists. But it is unclear how well relugolix CT works compared with surgery. The cost-effectiveness estimates for relugolix CT compared with GnRH agonists and surgery are within the range that NICE considers an acceptable use of NHS resources. So, relugolix CT is recommended.
Article in The Guardian
Selpercatinib for advanced thyroid cancer with RET alterations untreated with a targeted cancer drug in people 12 years and over
Selpercatinib is recommended as an option for treating advanced RET fusion-positive thyroid cancer that is refractory to radioactive iodine (if radioactive iodine is appropriate) and advanced RET-mutant medullary thyroid cancer. It is for people 12 years and over and is recommended only if the cancer has not been treated with a targeted cancer drug, and the company provides it according to the commercial arrangement.
Its evaluation focused on RET-mutant medullary thyroid cancer and RET fusion-positive thyroid cancer that has not been treated with a targeted cancer drug. People may have surgery to remove all or part of the thyroid before starting drug treatments. Usual treatments for RET-mutant medullary thyroid cancer include cabozantinib (a targeted cancer drug) and best supportive care (routine care and monitoring). Usual treatments for RET fusion-positive thyroid cancer that is refractory to radioactive iodine include targeted cancer drugs (lenvatinib or sorafenib) and best supportive care. Selpercatinib is a targeted cancer drug. The main clinical trial supporting this evaluation did not directly compare selpercatinib with usual treatment. Indirect comparisons suggest that people having selpercatinib have longer before their cancer gets worse and live longer than people having usual treatment. But this is uncertain. The most likely cost-effectiveness estimates are below what NICE considers an acceptable use of NHS resources. So selpercatinib is recommended.
Read the article HERE
Ruxolitinib for treating acute graft versus host disease refractory to corticosteroids in people aged 12 and over
Final Draft Guidance established that Ruxolitinib is recommended, within its marketing authorisation, as an option for treating acute graft versus host disease (GvHD) that has an inadequate response to corticosteroids in people 12 years and over. Ruxolitinib is only recommended if the company provides it according to the commercial arrangement.https://www.nice.org.uk/guidance/indevelopment/gid-ta11512
First-line standard care for acute GvHD is corticosteroids. If corticosteroids have not worked well enough, second-line standard care can include extracorporeal photopheresis, mycophenolate mofetil, etanercept and infliximab. Ruxolitinib is an alternative to these second-line treatments. Clinical trial evidence shows that acute GvHD is more likely to improve with ruxolitinib than with standard care. Treatment failure (that is, need for another treatment, relapse of the underlying disease that led to the need for a transplant, or death) may also be less likely in people who have ruxolitinib. The most likely cost-effectiveness estimate for ruxolitinib is within the range that NICE considers an acceptable use of NHS resources. So, ruxolitinib is recommended.
Read the article HERE
Efanesoctocog alfa for treating and preventing bleeding episodes in haemophilia A in people 2 years and over
Efanesoctocog alfa is recommended as an option for treating and preventing bleeding episodes in people 2 years and over with haemophilia A (congenital factor VIII deficiency), only if they have a factor VIII activity level of less than 1% (severe haemophilia A) and the company provides it according to the commercial arrangement.
For this evaluation, efanesoctocog alfa was only considered for people with severe haemophilia A, in line with the evidence provided by the company. This does not include everyone who it is licensed for. Current treatment options for severe haemophilia A include ongoing treatment with factor VIII replacement therapies (including standard half-life and extended half-life therapies) or emicizumab to prevent bleeding. On-demand factor VIII replacement therapies are used to treat bleeding. The results from a clinical trial suggest that there may be fewer bleeding episodes with ongoing efanesoctocog than with previous ongoing factor VIII replacement therapy, but this is uncertain. There is limited clinical-effectiveness evidence directly comparing efanesoctocog alfa with currently available treatments for severe haemophilia A, and there are substantial limitations with the available indirect comparisons. So, it is uncertain how well efanesoctocog alfa works compared with other haemophilia A treatments. Because of uncertainties in the clinical-effectiveness evidence and the economic model, the cost-effectiveness estimates are uncertain. But, when considering all the available evidence and economic analyses, efanesoctocog alfa is a cost-effective use of NHS resources. So, it is recommended.
Read the article HERE
Earliers updates on the NICE projects: Read our newsletter September 2024