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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

Genetic Disorders

Alpelisib for treating PIK3CA-related overgrowth spectrum

Alpelisib is in development for the treatment of the rare disease PIK3CA-related overgrowth spectrum (PROS). PROS is a group of genetic conditions where parts of the body become abnormally overgrown and can affect anywhere from the limbs to the brain, resulting in a wide range of symptoms which can include: pain, fatigue, or seizures depending […]

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Breast Cancer

Palbociclib with trastuzumab with or without pertuzumab and endocrine therapy for HR+/HER2+ metastatic breast cancer

Palbociclib in combination with trastuzumab with or without pertuzumab and endocrine therapy is currently in clinical development for use as a maintenance therapy for patients with hormone receptor positive (HR+) and human epidermal growth factor receptor 2 positive (HER2+) metastatic breast cancer who have had prior treatment. Breast cancer occurs when abnormal cells in the […]

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Lung and Respiratory Cancer

Telisotuzumab vedotin for previously treated c-MET overexpressing EGFR wild-type non-squamous non-small cell lung cancer

Telisotuzumab vedotin is in development for the treatment of c-Met+ non-small cell lung cancer. Non-small cell lung cancer is the most common form of lung cancer. A c-Met is a protein in humans that is encoded by the MET gene that leads to growth in cells. Smoking is the cause of most lung cancers and […]

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Cardiovascular System

Invimestrocel for Acute ischaemic stroke

Invimestrocel is in clinical development for the treatment of acute ischaemic stroke within 18-36hours of onset. Ischaemic stroke is the most common type of stroke. It happens when a bloodvessel is blocked by a blood clot, cutting off blood flow to part of the brain (ischaemia). Withoutblood supply, brain cells can be damaged or destroyed […]

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Cardiovascular System

Benralizumab for treating relapsing or refractory eosinophilic granulomatosis with polyangiitis

Benralizumab is currently in clinical development for relapsing or refractory eosinophilic granulomatosis with polyangiitis (EGPA). EGPA, formally known as Churg-Strauss Syndrome, is a rare disease characterised by blood vessel inflammation. This inflammation can restrict blood flow to organs and tissues, sometimes permanently damaging them. Relapsed refers to a disease that has come back, and refractory […]

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Lung and Respiratory Cancer

Nivolumab with ipilimumab for previously untreated locally advanced non-small-cell lung cancer

Nivolumab in combination with ipilimumab is in clinical development for the treatment of previously untreated locally advanced non-small-cell lung cancer (NSCLC). NSCLC is the most common form of lung cancer. Locally advanced NSCLC means the cancer is in more than one lobe of the lung, or it has spread to lymph nodes or nearby structures […]

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Gastroenterology

Guselkumab for treating ulcerative colitis

Guselkumab is in clinical development for the treatment of moderately to severely active ulcerative colitis (UC). UC is a long-term condition where parts of the large bowel become inflamed, causing urgent bloody diarrhoea and abdominal pain. The symptoms of UC often follow a pattern where individuals with the condition have periods of no symptoms or […]

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Respiratory System

Dupilumab for treating Chronic obstructive pulmonary disease (COPD)

Dupilumab is in clinical development for the treatment of chronic obstructive pulmonary disease (COPD) in adults aged 40 years and older. COPD is the name for a group of lung conditions that cause breathing difficulties. It is a disease that causes obstructed airflow from the lungs. It is typically caused by long-term exposure to irritating […]

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Haematological Cancer and Lymphomas

Daratumumab with bortezomib, lenalidomide and dexamethasone for previously untreated transplant eligible multiple myeloma

Daratumumab in combination with bortezomib, lenalidomide and dexamethasone is being developed for adult patients with previously untreated multiple myeloma who are eligible for high-dose chemotherapy with autologous stem cell transplant (ASCT). Multiple myeloma is an incurable type of cancer which develops when plasma cells inside the bone marrow become cancerous. This results in the excessive […]

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Gastroenterology

Guselkumab for treating moderately to severely active Crohn’s disease

Guselkumab is in clinical development for the treatment of moderately to severely active Crohn’s disease. Crohn's disease is a lifelong condition where parts of the digestive system become inflamed causing painful ulcers and inflammation most commonly in the small intestine and colon. The main symptoms of Crohn’s disease are diarrhoea, stomach aches and cramps, blood […]

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Haematological Cancer and Lymphomas

Gilteritinib maintenance therapy for FLT3-ITD mutated acute myeloid leukaemia

Gilteritinib is in clinical development as maintenance therapy for patients with FMS-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) acute myeloid leukaemia (AML) who have received a haematopoietic stem cell transplant (HSCT) and are in first complete remission (CR1). AML is a type of cancer of the white blood cells which are important in fighting […]

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Gastroenterology

Filgotinib for treating moderately to severely active Crohn's disease

Filgotinib is being developed for adults with moderately to severely active Crohn’s disease. Crohn’s disease is a type of inflammatory bowel disease which can affect any part of the digestive system. Crohn’s disease causes inflammation and ulceration, which affects food digestion, nutrient absorption, and waste elimination. Symptoms include abdominal pain, diarrhoea, weight loss and fatigue. […]

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Urological Cancer

Enfortumab vedotin with pembrolizumab for previously untreated locally advanced or metastatic urothelial cancer

Enfortumab vedotin in combination with pembrolizumab is in clinical development for the treatmentof patients with previously untreated locally advanced or metastatic urothelial cancer (UC). UC occurson the lining of the renal pelvis, ureter, bladder and urethra, and other parts of the urinary system.Metastatic UC occurs when the cancer has spread to other parts of the […]

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Lung and Respiratory Cancer

Osimertinib maintenance therapy for treating stage III unresectable EGFR mutation positive non-small-cell lung cancer after definitive platinum-based chemoradiation therapy

Osimertinib is in development for the treatment of stage III, unresectable EGFR mutation positive non-small cell lung cancer (NSCLC) following definitive chemoradiation therapy. NSCLC is the most common form of lung cancer. Stage III lung cancer means the cancer is in more than one lobe of the lung, or it has spread to lymph nodes […]

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Neurology

Donanemab for treating early symptomatic Alzheimer's disease

Donanemab is currently in clinical development for the treatment of early Alzheimer’s disease(AD). AD is a progressive neurological disease which is caused by loss of function and death ofneurones in the brain. It is the most common type of dementia. One of the early symptoms of ADis mild cognitive impairment where a person may have […]

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Oncology

Selpercatinib for previously untreated advanced RET-fusion positive thyroid cancer

Selpercatinib is in clinical development for untreated advanced RET-fusion positive thyroid cancer in adults. Thyroid cancer is a rare form of cancer that originates in the thyroid gland located in the front of the neck. RET is a type of protein called a kinase, which is involved in various cell processes. RET-fusion positive cancer is […]

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Breast Cancer

Benegrastim for treating chemotherapy-induced neutropenia in women with breast cancer

Benegrastim is being developed for the treatment of neutropenia in patients receiving chemotherapy treatment for breast cancer. Neutropenia occurs when an individual has low levels of neutrophils (a type of white blood cell), and it is a common side effect of cancer chemotherapy. Neutropenia can leave patients vulnerable to infections and cause complications such as […]

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Endocrine Nutritional and Metabolic Disorders

CAEL-101 for treating amyloid light chain amyloidosis

CAEL-101 is in clinical development for the treatment of amyloid light chain (AL) amyloidosis. AL amyloidosis belongs to a group of diseases called systemic amyloidosis in which deposits of proteins (called amyloids) accumulate and cause damage in tissues and organs, such as the kidneys, liver, gut, heart and nerves. In AL amyloidosis, the deposits are […]

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Dermatology

Nemolizumab for treating atopic dermatitis and prurigo nodularis

Nemolizumab is currently in development for atopic dermatitis and prurigo nodularis. Atopic dermatitis is the most common form of eczema, a long-term (chronic) condition that causes patches of the skin to become itchy, dry and cracked. In moderate-to-severe cases, the patches cover a large area of the skin and can be associated with an intense […]

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Sarcoma

Letetresgene autoleucel for treating synovial sarcoma and myxoid round cell liposarcoma

Letetresgene autoleucel is in clinical development for the treatment of advanced synovial sarcoma (SS) and myxoid/round cell liposarcoma (MRCLS) in children and adults. Both SS and MRCLS are two different types of soft tissue sarcomas, rare forms of cancer that develop in the tissues that connect, support, and surround other body structures and organs. Advanced […]

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Endocrine Nutritional and Metabolic Disorders

Potassium citrate-potassium bicarbonate prolonged release granules for treating cystinuria

ADV7103 is currently in clinical development for the treatment of cystinuria in patients aged 1 year and above. Cystinuria is an inherited metabolic condition where irregular changes to genes called SLC3A1 and SLC7A9 results in abnormal transport of the amino acid, cysteine, in the kidney. This results in excessive amounts of cysteine excreted in the […]

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Lung and Respiratory Cancer

Nivolumab with chemotherapy neoadjuvant followed by nivolumab adjuvant for treating early stage non-small-cell lung cancer

Neoadjuvant nivolumab in combination with chemotherapy followed by adjuvant nivolumab is currently in clinical development for early stage non-small-cell lung cancer (NSCLC). NSCLC is the most common type of lung cancer. Early-stage lung cancer is typically treated with surgery consisting of removing either part of or the whole of the lung, followed by chemotherapy and/or […]

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Haematological Cancer and Lymphomas

Zamtocabtagene autoleucel for treating relapsed or refractory diffuse large B-cell lymphoma

Zamtocabtagene autoleucel is currently in clinical development for adults with relapsed or refractory diffuse large B cell lymphoma (DLBCL), not eligible for high-dose chemotherapy and autologous stem cell transplantation. DLBCL is a cancer of B cells (a type of immune cell) and the most common type of fast-growing non-Hodgkin’s lymphoma. In DLBCL, abnormal B cells […]

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Lung and Respiratory Cancer

Durvalumab with platinum-based chemotherapy for the neoadjuvant/adjuvant treatment of resectable non-small-cell lung cancer

Durvalumab in combination with platinum-based chemotherapy is in clinical development for the neoadjuvant/adjuvant treatment of resectable non-small-cell lung cancer (NSCLC). NSCLC is the most common type of lung cancer. Early-stage lung cancer is typically treated with surgery to remove the tumour, followed by chemotherapy and/or radiotherapy. However, survival rates are still poor. Symptoms include a […]

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Gastroenterology

Risankizumab for moderate to severe ulcerative colitis

Risankizumab is in clinical development for moderate to severe ulcerative colitis. Ulcerative colitis is one of two major types of inflammatory bowel disease (IBD). It is a long-term immune-mediated inflammatory disorder where the colon and rectum become inflamed and small ulcers can develop on the colon’s lining, which can cause symptoms such as rectal bleeding […]

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Haematological Cancer and Lymphomas

Daratumumab for the treating high-risk smouldering multiple myeloma

Daratumumab is in clinical development for high-risk smouldering multiple myeloma (SMM). SMM is an early form of myeloma which usually progresses to active myeloma, but at a slow rate. SMM is a precancerous condition that changes certain proteins in blood and/or increases plasma cells in the bone marrow but does not cause symptoms of disease. […]

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Male Reproductive Cancer

Apalutamide with primary radiation therapy for treating high-risk, localised or locally advanced prostate cancer

Apalutamide with androgen deprivation therapy (ADT), is in clinical development for patients with high-risk localised or locally advanced prostate cancer who are receiving primary radiation therapy. Prostate cancer is a cancer of the prostate gland (a small organ in a man’s pelvis) and is the most common cancer in men in the UK. The symptoms […]

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Female Reproductive Cancer

Atezolizumab with platinum-based chemotherapy and bevacizumab for epithelial ovarian cancer or fallopian tube cancer or primary peritoneal cancer

Atezolizumab in combination with bevacizumab and chemotherapy is currently in clinicaldevelopment for the treatment of patients with epithelial ovarian cancer or primary peritonealor fallopian tube cancer. Epithelial ovarian cancers are the most common type of ovarian cancer.Primary peritoneal cancer and fallopian tube cancer are similar to epithelial ovarian cancer. Allthree types show signs of pain […]

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Cardiovascular System

Tirzepatide for treating obese or overweight adults

Tirzepatide is in clinical development for the treatment of obesity or overweight individuals with weight-related comorbidities (e.g., sleep apnoea, cardiovascular disease). Excess weight can place stress on both mental and physical health, leading to complications such as depression, low self-esteem, and increased risk of heart disease, stroke, and type 2 diabetes. Weight can be affected […]

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Haematological Cancer and Lymphomas

Ivosidenib with azacitidine for previously untreated acute myeloid leukaemia with an IDH1 mutation

Ivosidenib is in development with azacitidine for the treatment of patients with newly diagnosed acute myeloid leukaemia (AML) who have an isocitrate dehydrogenase 1 (IDH1) mutation and are ineligible for intensive induction chemotherapy. AML is a rare form of blood cancer that affects the white blood cells resulting in frequent infections for patients. AML also […]

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