March 2022 decisions news release
The Scottish Medicines Consortium (SMC), which advises on newly licensed medicines for use by NHSScotland, has today (Monday March 7) published advice on seven new medicines.
Three of the medicines accepted, sacituzumab govitecan (Trodelvy), sotorasib (Lumykras) and lorlatinib (Lorviqua), were awarded Innovation Passports within the new Innovative Licensing and Access Pathway (ILAP). ILAP aims to provide safe, early and financially sustainable access to innovative new medicines to patients across the UK. It is a unique partnership approach that harnesses expertise from the Medicines and Healthcare products Regulatory Agency, the Scottish Medicines Consortium, the National Institute for Health and Care Excellence (NICE) and the All Wales Therapeutics and Toxicology Centre (AWTCC). Through this partnership ILAP helps to optimise the clinical development, licensing and access processes for new medicines. Further information on ILAP can be found on our website.
Sacituzumab govitecan was accepted for the treatment of patients with advanced triple negative breast cancer (TNBC). TNBC is an aggressive type of breast cancer that often affects women at a stage of their lives where they have significant family and work commitments. The medicine was considered through SMC’s Patient and Clinician Engagement (PACE) process, which is used for medicines for end of life and rare conditions. PACE participants highlighted that a diagnosis of TNBC can overwhelm patients and their families, as it has the poorest prognosis of all breast cancer subtypes. Patients may experience fatigue, bone pain and breathlessness. There are few treatment options and they have limited efficacy. Patients often respond for only a few cycles of treatment with the duration of response progressively declining with each subsequent therapy. Sacituzumab govitecan is used in patients who have had at least two prior lines of therapy. It may offer more time until the disease progresses and increased overall survival, which may allow patients a better quality of life and valuable additional time with family and friends.
Following SMC’s inclusion of medicines with ILAP status in its interim decision process in September 2021, the committee was able to accept sotorasib (Lumykras) for the treatment of patients with advanced non-small cell lung cancer (NSCLC) with a specific gene mutation (known as KRAS G12C-mutated), subject to ongoing evaluation and future reassessment. Sotorasib was assessed through the PACE process, for medicines for end of life and rare conditions. In the PACE meeting, participants highlighted that advanced NSCLC is incurable, with progressive debilitating symptoms including breathlessness, fatigue, weight loss, and pain. These symptoms have a significant impact on patients’ daily living and their quality of life. Treatment options for patients are limited and sotorasib offers the first targeted therapy for this particular genetic mutation. The clinical evidence in the company submission was based on a single-arm clinical trial, which means there is no comparison with placebo or another treatment, leading to uncertainty in the evidence for decision making. The committee therefore accepted sotorasib on an interim basis subject to ongoing evaluation and will consider further evidence on its effectiveness once this is available. Further information on interim acceptance is available on our website.
Lorlatinib (Lorviqua) has been accepted when used on its own as a first line treatment for NSCLC with a specific gene mutation (known as ALK positive) through the SMC abbreviated submission route.
Dostarlimab (Jemperli) was accepted for the treatment of patients with advanced endometrial cancer where the disease has progressed despite treatment with platinum-based chemotherapy. Endometrial cancer is a rare, life limiting condition and this diagnosis can be extremely difficult for patients and their families. There are limited treatment options, and, as dostarlimab is a targeted treatment, it may be better tolerated than chemotherapy. The clinical evidence in the company submission for dostarlimab was also based on a single-arm clinical trial leading to uncertainty in the evidence for decision making. The committee therefore accepted dostarlimab on an interim basis subject to ongoing evaluation and will consider further evidence on its effectiveness once this is available. Further information on the interim acceptance process is available on our website.
Berotralstat (Orladeyo) was accepted for the routine prevention of frequent severe attacks of hereditary angioedema (HAE) in adults and adolescents. HAE is a rare inherited condition, causing swelling that can affect any part of the body including the hands, feet, arms, legs, intestines, face and throat. Attacks are unpredictable and can be life threatening. Through the PACE process, patient groups and clinicians emphasised that HAE is a lifelong condition that can cause significant anxiety to patients and their carers. Currently available medicines that prevent HAE are given by injection which can be difficult for some patients and may not always be feasible. Berotralstat is the first licensed oral preventative treatment, providing a convenient treatment option that may be better tolerated than current treatments.
The committee was unable to accept solriamfetol (Sunosi) for the treatment of obstructive sleep apnoea (OSA) to improve excessive daytime sleepiness and wakefulness. OSA is a condition where people suffer from frequent interruptions to their breathing during sleep. The main current treatments for OSA is continuous positive airway pressure (CPAP), which uses an air pump to apply positive air pressure through a mask to the patient’s airway, keeping it open. The committee was unable to accept solriamfetol as the company’s evidence around the clinical and cost effectiveness of the treatment was not sufficiently clear.
The committee was also unable to accept a new modified release oral formulation of hydrocortisone (Efmody) for the treatment of congenital adrenal hyperplasia (CAH), a group of rare inherited conditions where the adrenal glands are larger than usual. People with CAH are unable to make enough of a hormone called cortisol so need to take medication every day to manage the symptoms of their disease. The committee was unable to accept this formulation of hydrocortisone as the company’s evidence around the clinical and cost effectiveness of the medicine when compared to other currently available treatments was not strong enough.
SMC chairman Mark MacGregor said:
“We are pleased to be able to accept these five medicines for use by NHSScotland.”
“Sacituzumab govitecan, sotorasib and lorlatinib are the first medicines the SMC Committee has considered after the award of an Innovation Passport in the new ILAP process. We’re delighted that this exciting new collaboration with partners in MHRA, NICE and AWTTC is progressing well and patients in Scotland can now access these three medicines.”
“A diagnosis of triple negative breast cancer can be devastating for patients and their families, particularly as the prognosis is so poor. We hope that our decision to accept sacituzumab govitecan, will offer patients with this form of breast cancer the prospect of a better quality of life and valuable extra time with family and friends.”
“We were also able to accept sotorasib (Lumykras) for NSCLC, the first targeted therapy for patients with this specific type of NSCLC, and from our PACE participants we know that this decision will be welcomed. While evidence on its effectiveness is limited, the committee accepted sotorasib subject to future reassessment.”
“There are very few treatment options for endometrial cancer so we know that our decision on dostarlimab will be welcomed by patients and clinicians. Dostarlimab may be a useful additional treatment but as the evidence considered was limited, the committee has accepted its use subject to reassessment once further evidence is available.”
“For patients with HAE, our decision on berotralstat offers an oral treatment option which may be easier to tolerate than current intravenous treatments.”
“The committee was unable to accept solriamfetol (Sunosi) and new modified release oral formulation of hydrocortisone as the evidence provided by the companies on on the clinical and cost effectiveness of both of these treatments was not clear.”
“We were also unable to accept the new formulation of hydrocortisone as the evidence provided by the company on the benefits of using this medicine instead of the current treatment options was not strong enough to justify its cost.”