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Medicine Industry's Gateway to NICE

The National Institute for Health and Care Excellence (NICE) TA programme provides UK healthcare guidance through Technology Appraisals and Highly Specialised Technologies assessments. A NICE recommendation is crucial for market access to the NHS in England and Wales. The Innovation Observatory is the first step in the process and we work closely with our pharmaceutical industry partners. 

We provide the 'gateway' to NICE by submitting potential topics that meet the NICE Topic Selection criteria for medicines as set out in the NICE Topic Selection Manual. We follow the development of new and repurposed medicines from at least two years before estimated UK licence to provide a timely notification to NICE. This early notification triggers the NICE TA process and allows NICE to provide new guidelines as close to the licence date as possible. 

The Innovation Observatory has a strict confidentiality agreement which means we only share confidential information with selected policymakers. 

Latest Technology Briefings

Renal Cancer

Nivolumab adjuvant therapy for renal cell carcinoma

Nivolumab is in clinical development as adjuvant therapy for adult patients with localised renal cell carcinoma (RCC) who underwent surgical removal of partial or entire of a kidney. RCC (the most common type of kidney cancer in adults) is a disease that affects the lining of tiny tubes within the kidney which filter waste from […]

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Female Reproductive Cancer

Pembrolizumab with chemotherapy for platinum-resistant recurrent ovarian cancer after 1 or 2 therapies

Pembrolizumab in combination with paclitaxel with or without bevacizumab is in clinical development for the treatment of platinum-resistant recurrent ovarian cancer after one or two prior therapies. Ovarian cancer occurs when abnormal cells in the ovary begin to grow and divide in an uncontrolled way to eventually form a growth (tumour). Some of the symptoms […]

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Lung and Respiratory Cancer

Tarlatamab for relapsed/refractory small-cell lung cancer

Tarlatamab is in clinical development for the treatment of adults with refractory (unresponsive to treatment) or relapsed small cell lung cancer (SCLC) who have progressed or recurred following one platinum-based (chemotherapy) regimen and at least a prior line of therapy. SCLC is a disease in which malignant (cancer) cells form in the tissues of the […]

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Women's Health

Elinzanetant for the treatment of moderate to severe vasomotor symptoms (hot flashes) associated with the menopause

Elinzanetant is in clinical development for the treatment of vasomotor symptoms (hot flashes) in women undergoing the menopause. The menopause occurs between 45 and 55 years of age and is caused by declining hormone levels. The declining hormone levels mean that neurons (nerve cells) in the hypothalamus (small part in the brain) become hyperactive and […]

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Ear Nose and Throat (ENT)

Tezepelumab for treating severe chronic rhinosinusitis with nasal polyps

Tezepelumab is in clinical development for the treatment of severe chronic rhinosinusitis with nasal polyps. Chronic rhinosinusitis is a condition in which the lining of the sinuses (air-filled spaces behind the nose, eyes, and cheeks) becomes inflamed for more than 12 weeks. People with the condition may have nasal polyps which are growths inside the […]

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Female Reproductive Cancer

Mirvetuximab soravtansine for advanced, epithelial ovarian, fallopian tube, or primary peritoneal cancer with high folate receptor-alpha expression

Mirvetuximab soravtansine is in clinical development for the treatment of adults with advanced, platinum-resistant, high-grade epithelial ovarian cancer, primary peritoneal cancer and fallopian tube cancer with high folate receptor-alpha expression. Epithelial ovarian cancer means the cancer started in the surface layer covering the ovary. The main symptoms are frequent swollen or bloated tummy, pain or […]

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Genetic Disorders

Vanzacaftor / tezacaftor / deutivacaftor for the treatment of cystic fibrosis

Vanzacaftor/tezacaftor/deutivacaftor is in development for the treatment of cystic fibrosis in patients with at least one F508del mutation (faulty gene). Cystic fibrosis is a rare, chronic, and life-shortening genetic disease caused by a faulty gene that affects the movement of salt and water in and out of cells. It is a progressive, multi-system disease that […]

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Cardiovascular System

Evolocumab for preventing major cardiovascular events in adults with a high risk of cardiovascular events

Evolocumab is currently in clinical development for the prevention of major cardiovascular events in adults without prior heart attack or stroke who are at high risk of a cardiovascular event. Cardiovascular disease (CVD) is an umbrella term for all diseases of the heart and circulation. It includes everything from conditions that are inherited or that […]

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Dermatology

Apremilast for treating moderate to severe plaque psoriasis in children and young people

Apremilast is being investigated for the treatment of moderate to severe plaque psoriasis in children and adolescents (aged 6 to 17 years old). Psoriasis is a skin disease that causes a rash with itchy, scaly patches, most commonly on the knees, elbows, trunk, and scalp. Plaque psoriasis is the most common, and is characterised by […]

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Haematological Cancer and Lymphomas

I-131-Apamistamab for acute myeloid leukaemia

I-131-Apamistamab is in clinical development for the treatment of adult patients with relapsed, or refractory acute myeloid leukaemia (AML), prior to bone marrow transplant. AML is a type of blood cancer where excess immature white blood cells are made by the bone marrow, these immature cells do not function like healthy blood cells in fighting […]

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Genetic Disorders

Omaveloxolone for the treatment of Friedreich's ataxia

Omaveloxolone is in development for the treatment of patients with Friedreich’s Ataxia (FA). FA is an inherited disease that causes a range of symptoms including difficulty walking, inability to coordinate movements, muscle weakness, speech problems, damage to the heart muscle and diabetes. Patients with FA do not have enough frataxin, a protein that regulates iron […]

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Gastroenterology

Pylera with omeprazole for treating helicobacter pylori associated ulcers

Pylera in combination with omeprazole is currently in development for the treatment of adults who test positive for Helicobacter pylori (H. pylori) with upper gastrointestinal symptoms. H. pylori infection occurs when H. pylori bacteria infect the stomach, which typically happens during childhood. The bacteria are usually passed from person to person through direct contact with […]

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Breast Cancer

Vepdegestrant for previously treated locally advanced or metastatic ER+/HER2– breast cancer

Vepdegestrant is in development for the treatment of patients with oestrogen receptor-positive (ER+) and human epidermal growth factor receptor 2-negative (HER2-) metastatic breast cancer, who previously received endocrine-based treatment. A locally advanced breast cancer is a stage 3 breast cancer where the cancer has spread from the breast to lymph nodes close to the breast […]

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Lung and Respiratory Cancer

Pembrolizumab-vibostolimab with etoposide and platinum chemotherapy for previously untreated extensive-stage small cell lung cancer

Pembrolizumab-vibostolimab with etoposide and platinum chemotherapy is in clinical development for the first-line treatment of extensive stage small cell lung cancer (SCLC). Extensive stage SCLC is when cancer cells form in the tissues of the lung and spread beyond the lung to other places in the body. Symptoms include chest pain, a cough that does […]

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Breast Cancer

Datopotamab deruxtecan for previously treated unresectable or metastatic HR+ HER2- breast cancer

Datopotamab deruxtecan is in clinical development for the treatment of inoperable or metastatic HR-positive HER2-negative breast cancer after one or two lines of systemic chemotherapy. Breast cancer is the growth of abnormal cells in the breast which divide uncontrollably to eventually form a tumour. Breast cancers can also be hormone receptor positive (HR+), meaning that […]

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Breast Cancer

Trastuzumab deruxtecan neoadjuvant therapy for high-risk HER2-positive early stage breast cancer

Trastuzumab deruxtecan is in clinical development for the neoadjuvant treatment of high-risk HER2-positive early-stage breast cancer. Breast cancer is when abnormal cells in the breast begin to grow and divide in an uncontrolled way and eventually form a growth (tumour). Breast cancer that has not spread beyond the breast or the axillary lymph nodes is […]

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Haematological Cancer and Lymphomas

Obecabtagene autoleucel for treating refractory or relapsed B cell acute lymphoblastic leukaemia

Obecabtagene autoleucel is an advanced therapy medicinal product (gene therapy product) known as ‘Chimeric Antigen Receptor’ (CAR) T cell therapy which is in clinical development for relapsed/refractory B-cell acute lymphoblastic leukaemia (B-ALL) in adults. B-ALL is an aggressive type of leukaemia (cancer of the blood and bone marrow), that can progress rapidly without treatment and […]

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Lung and Respiratory Cancer

Serplulimab with chemotherapy for treating previously untreated extensive stage small-cell lung cancer

Serplulimab with chemotherapy is currently in clinical development as a first-line treatment for extensive-stage small-cell lung cancer (SCLC). SCLC is a very aggressive form of cancer that forms in the tissues of the lungs and often goes undiagnosed until it’s more advanced, so the survival rate tends to be low. Extensive stage SCLC (ES-SCLC) is […]

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Lung and Respiratory Cancer

Niraparib with pembrolizumab maintenance therapy for metastatic stage III or IV non-small cell lung cancer

Niraparib with pembrolizumab is in clinical development for the maintenance therapy of patients with later stages of non-small cell lung cancer (NSCLC) after first-line platinum-based chemotherapy with pembrolizumab. Lung cancer is the third most common cancer in the UK and about 47,000 people are diagnosed with it each year. When cancer starts in the lungs, […]

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Genetic Disorders

Eplontersen for hereditary transthyretin-mediated amyloid polyneuropathy

Eplontersen is currently in development for the treatment of hereditary transthyretin-mediated amyloid polyneuropathy (hATTR-PN). Hereditary transthyretin amyloidosis with polyneuropathy (formerly known as Familial Amyloid Polyneuropathy) is a rare disease caused by mutations in the gene that codes for transthyretin and is characterised by a multisystem extracellular deposition of amyloid (abnormal fibrous, extracellular, proteinaceous deposits found […]

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Musculoskeletal System

Xeomin (Botulinum neurotoxin type A) for lower limb and/or upper limb spasticity

Xeomin (Botulinum neurotoxin type A) is in clinical development for the treatment of adults with lower limb and/or upper limb spasticity. Spasticity is a long-term symptom described as a pathological increase in muscle tension (hypertonicity) resulting from damages to the brain, spinal cord, or motor nerves associated with conditions such as stroke, traumatic brain injury […]

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Dermatology

Remibrutinib for treating chronic spontaneous urticaria inadequately controlled by H1-antihistamines

Remibrutinib is currently in clinical development for the treatment of chronic spontaneous urticaria (CSU) in adult patients inadequately controlled by H1-antihistamines. CSU is a distressing skin condition that is characterised by itchy and sometimes painful raised rash or patches for at least six weeks with no known obvious trigger. It may also be accompanied by […]

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Cardiovascular System

Acoramidis for symptomatic transthyretin amyloid cardiomyopathy

Acoramidis is in clinical development for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM). ATTR-CM is an underdiagnosed, progressively debilitating, rare disease associated with high mortality. ATTR-CM occurs when a protein called transthyretin (TTR) becomes unstable and aggregates, forming amyloids, and deposits primarily in the heart, as well as other organs, known as transthyretin amyloidosis. This […]

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Haematological Cancer and Lymphomas

Zanubrutinib plus obinutuzumab for treating relapsed or refractory follicular lymphoma

Zanubrutinib, combined with an already licensed medicine, obinutuzumab, is in clinical development for the treatment of patients with relapsed or refractory B-cell follicular lymphoma. Follicular lymphoma is a slow-growing blood cancer that affects white blood cells. In follicular lymphoma, the affected white blood cells multiply abnormally and aggregate in certain parts of the body, such […]

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Hepatology

Resmetirom for non-alcoholic steatohepatitis

Resmetirom is currently in clinical development for the treatment of non-alcoholic steatohepatitis (NASH). NASH is caused by an accumulation of fat in the liver that results in inflammation and damage to the liver. It belongs to a class of diseases known as non-alcoholic fatty liver disease (NAFLD); it is an advanced form of the disease. […]

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Haematological Cancer and Lymphomas

Tazemetostat for treating relapsed or refractory
follicular lymphoma after two therapies

Tazemetostat is in clinical development for the treatment of adult patients with relapsed orrefractory follicular lymphoma who have received at least two prior treatments. Follicularlymphoma is a cancer of a type of white blood cell called B lymphocytes, or B cells. In follicularlymphoma, B cells multiply quickly and live for too long, so there are […]

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Breast Cancer

Capivasertib with paclitaxel for previously untreated locally advanced or metastatic triple-negative breast cancer

Capivasertib with paclitaxel is currently in clinical development for previously untreated locallyadvanced (inoperable) or metastatic triple-negative breast cancer (TNBC). TNBC is a rare form ofbreast cancer where the tumour cells do not have receptors for oestrogen, progesterone, or thehuman epidermal growth factor receptor 2 (HER2) protein. Symptoms of TNBC include: a newlump or thickening in […]

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Breast Cancer

Capivasertib with fulvestrant for HR+/HER2- locally advanced or metastatic breast cancer

Capivasertib with fulvestrant is in development for the treatment of patients with locally advanced (inoperable) or metastatic Hormone Receptor-Positive (HR+) and Human Epidermal growth factor Receptor 2-Negative (HER2-) breast cancer. A locally advanced breast cancer is stage 3, where cancer has spread from the breast to areas close to the breast or to the chest […]

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Genetic Disorders

Givinostat for Duchenne muscular dystrophy

Givinostat is in clinical development for Duchenne muscular dystrophy (DMD). DMD is a geneticdisease that gradually causes weakness and atrophy (wasting) of the muscles. It mainly affectsboys, and usually starts before the age of six years. Patients with DMD lack normal dystrophin, aprotein found in muscles. Because this protein helps to strengthen and protect muscles […]

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Endocrine Nutritional and Metabolic Disorders

Leriglitazone for X-linked andrenoleukodystrophy

Leriglitazone is currently being developed to treat male patients with X-linked adrenoleukodystrophy (X-ALD). Adrenoleukodystrophy (ALD) is a degenerative condition where there is a build-up of substances known as “very long chain fatty acids” in tissues around the body, particularly in the brain, spinal cord and adrenal glands. There are three types of ALD: a cerebral […]

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