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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

Neurology

Inebilizumab for neuromyelitis optica spectrum disorders

Inebilizumab is in clinical development for the treatment of adults with neuromyelitis optica spectrum disorder (NMOSD). NMOSD is a rare autoimmune condition in which the immune system attacks and damages one’s own spinal cord and optic nerves (nerves in the eyes). Autoantibodies (proteins that attacks own cells) against a cell surface protein found in neural […]

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Haematological Cancer and Lymphomas

Brentuximab vedotin with cyclophosphamide, prednisone and doxorubicin for treating peripheral Tcell lymphoma

Brentuximab vedotin in combination with cyclophosphamide, prednisone and doxorubicin is in clinical development for the treatment of peripheral T-cell lymphoma (PTCL). Peripheral T-cell lymphomas (PTCLs) are a group of rare blood cancers. They develop in T-cells and affect the lymphatic system. PTCLs happen when the T-cells mutate and become cancerous cells that multiply uncontrollably. PTCL […]

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Gastroenterology

Linaclotide acetate for treating Chronic idiopathic constipation

Linaclotide acetate is currently in clinical development for the treatment of adults with chronic idiopathic constipation (CIC). Constipation is defecation that is problematic because of infrequent and/or hard stools, difficulty passing stools (often involving straining), or the sensation of incomplete emptying or anorectal blockage. Chronic is when the symptoms are present for at least three […]

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Breast Cancer

Datopotamab deruxtecan for previously untreated locally recurrent inoperable or metastatic triple-negative breast cancer

Datopotamab deruxtecan is in development for the treatment of patients with previously untreated, locally recurrent inoperable or metastatic triple negative breast cancer (TNBC), who are not suitable candidates for PD-1/PD-L1 inhibitor therapy. TNBC is a subtype of breast cancer which lacks hormone receptor expression. The risk factors for TNBC include a history of breast disease […]

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Infectious Disease

Sarconeos for treating severe COVID-19 in people aged 45 years and over

Sarconeos is currently in clinical development for treating adults aged 45 or over who are hospitalised with COVID-19. COVID-19 is an infectious respiratory disease caused by the severe acute respiratory syndrome-coronavirus 2 (SARS-CoV-2). The main symptoms of COVID-19 are fever, a new continuous cough, and loss of taste or smell. In some patients, COVID-19 may […]

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Male Reproductive Cancer

Talazoparib with enzalutamide for previously untreated homologous recombination repair-mutated metastatic hormonal-sensitive prostate cancer

Talazoparib in combination with enzalutamide is in clinical development for the treatment of homologous recombination repair (HRR)-mutated metastatic hormonal-sensitive prostate cancer (mHSPC). Prostate cancer is a cancer of the prostate gland and is the most common type of cancer in men in the UK. The cancer is called metastatic when it has spread to other […]

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Renal Cancer

Nivolumab for treating advanced clear cell renal cell carcinoma after systemic treatment

Subcutaneous (SC) Nivolumab is currently in clinical development for treating advanced or metastatic clear cell renal cell carcinoma (ccRCC) in adult patients who previously received systemic treatment. Metastatic cancer is when cancer cells have spread to other parts of the body and advanced cancer is when the cancer cannot be cured. ccRCC is the most […]

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Immunology

Teprotumumab for thyroid eye disease

Teprotumumab is currently in development for treatment of thyroid eye disease (TED). TED, also known as Graves’ orbitopathy or ophthalmopathy, is an autoimmune condition. It occurs when the body’s immune system attacks the tissue surrounding the eye causing inflammation in eye muscles, eyelids, tear glands and fatty tissues behind the eye. This can cause the […]

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Breast Cancer

Retifanlimab with chemotherapy for previously untreated metastatic non-small-cell lung cancer

Retifanlimab is currently in clinical development for previously untreated metastatic non-small-cell lung cancer (NSCLC). NSCLC is the most common lung cancer. There are three main types: adenocarcinoma, squamous cell carcinoma and large-cell lung cancer. Stage IV NSCLC is also called metastatic (the most advanced stage) lung cancer. Smoking tobacco is the cause of most lung […]

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Dermatology

Secukinumab biosimilar for treating plaque psoriasis

Secukinumab biosimilar is in clinical development for the treatment of moderate to severe plaque psoriasis in adults. Plaque psoriasis is a long-lasting autoimmune disease that causes skin cells to reproduce very quickly. Plaque psoriasis is characterised by thick, scaly patches called plaques on the skin. These plaques most commonly effect elbows, the back, knees, and […]

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Endocrine Nutritional and Metabolic Disorders

Olezarsen for treating familial chylomicronaemia syndrome

Olezarsen is in clinical development for the treatment of patients with familial chylomicronaemia syndrome (FCS). FCS is a rare genetic disorder that causes very high levels of triglycerides, which is a kind of fat, in the blood. This can lead to serious health problems, including acute and chronic pancreatitis and type 3c diabetes. Symptoms of […]

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Hepatology

Odevixibat for treating biliary atresia in children following Kasai hepatoportoenterostomy

Odevixibat is in clinical development for the treatment of children with biliary atresia (BA) following a Kasai hepatoportoenterostomy (HPE). BA is a rare gastrointestinal disorder where either all or a portion of the bile duct on the outside of the liver is absent or destroyed. As a result, bile acids accumulate in the liver causing […]

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Endocrine Nutritional and Metabolic Disorders

Ferric maltol for treating iron deficiency anaemia in children and adolescents aged 1 month to 17 years

Ferric maltol is in clinical development for the treatment of children and adolescents with iron deficiency anaemia. Iron deficiency anaemia is diminished red blood cell production due to low iron stores in the body, and can develop because of malnutrition, malabsorption, or blood loss. Iron deficiency, with or without anaemia, adversely affects overall health and […]

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Breast Cancer

Inavolisib with palbociclib and fulvestrant for treating HR+/HER2-, PIK3CA mutated locally advanced or metastatic breast cancer

Inavolisib in combination with palbociclib and fulvestrant is in clinical development for the treatment of patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-), PIK3CA-mutated locally advanced or metastatic breast cancer. Breast cancer occurs when abnormal cells in the breast begin to grow and divide in an uncontrolled way and […]

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Haematological Cancer and Lymphomas

Brentuximab vedotin with chemotherapy (BrECADD) for previously untreated advanced classical Hodgkin lymphoma

Hodgkin lymphoma (HL) is an uncommon cancer that develops in the lymphatic system, which is a network of vessels and glands spread throughout the body. About 95% of all HL cases are classical HL. Lymphomas are cancers that occur if white blood cells of the immune system start to multiply uncontrollably. Symptoms of classical HL […]

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Haematological Cancer and Lymphomas

Elranatamab monotherapy or with daratumumab for previously treated relapsed or refractory multiple myeloma

Elranatamab, alone or in combination with daratumumab, is in clinical development for the treatment of relapsed or refractory multiple myeloma (MM). MM is a type of bone marrow cancer that is characterised by an abundance of abnormal immune cells, known as plasma cells, in the bone marrow. In the early stages, MM may not cause […]

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Cardiovascular System

Inhaled treprostinil for treating pulmonary hypertension with interstitial lung disease including combined pulmonary fibrosis and emphysema

Pulmonary hypertension is a rare and serious condition that occurs when blood pressure within the lungs becomes abnormally high. It can be caused by thickening of the pulmonary artery walls, heart failure, lung disease and clots within the lungs' blood vessels. Symptoms of pulmonary hypertension include shortness of breath, tiredness, feeling faint or dizzy, chest […]

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Urological Cancer

Durvalumab and enfortumab vedotin with or without tremelimumab for muscle-invasive bladder cancer

Durvalumab and enfortumab vedotin with or without tremelimumab is currently in clinical development for patients with muscle invasive bladder cancer (MIBC), who are ineligible or refuse cisplatin therapy (a type of chemotherapy) and are receiving a radical cystectomy (removal of bladder and surrounding lymph nodes). MIBC is a cancer that spreads into the muscle layer […]

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Neurology

Zavegepant for treating acute migraine

Zavegepant is in clinical development for the acute treatment of migraine. Migraines is a headache that is usually characterised by throbbing in one side of the head. In addition to this, other symptoms include sensitivity to light and sound, nausea and vomiting. The cause of migraines is not known but some triggers include tiredness, anxiety […]

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Endocrine Nutritional and Metabolic Disorders

Setmelanotide for treating acquired hypothalamic obesity in people 4 years and older

Setmelanotide is currently in development for the treatment of acquired hypothalamic obesity. Acquired hypothalamic obesity refers to excess weight gain that may follow from an injury to the hypothalamus, a brain region which coordinates the endocrine system (controls body functions). The hypothalamus affects energy intake, by regulating how much we eat and how much energy […]

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Respiratory System

Brensocatib for people aged 12 and over with non-cystic fibrosis bronchiectasis

Brensocatib is currently in clinical development for the treatment on non-cystic fibrosis bronchiectasis in patients aged 12 and older. Bronchiectasis is a lung condition where inflammation from lung infections causes irreversible damage to the lungs which can get worse over time. During bronchiectasis, the airways are wider and become inflamed with thick phlegm (sputum). This […]

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Haematological Cancer and Lymphomas

Blinatumomab with chemotherapy for frontline consolidation treatment of Philadelphia chromosome negative, measurable residual disease negative, B-lineage acute lymphoblastic leukaemia

Acute lymphoblastic leukaemia (ALL) is a rare and aggressive blood cancer, characterised by proliferation of immature and abnormal white blood cells responsible for the immune system (lymphocytes), in the bone marrow and blood. These immature/abnormal lymphocytes dominate bone marrow, resulting in a decrease of red blood cells, normal white blood cells and platelets. ALL can […]

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Nephrology

DMX-200 for treating focal segmental glomerulosclerosis in people receiving angiotensin II receptor blocker therapy

DMX-200 is currently in clinical development for treating focal segmental glomerulosclerosis (FSGS) among patients who are also receiving an angiotensin II receptor blocker (ARB). FSGS is a rare disease in which scar tissue develops on the parts of the kidneys that filter waste from the blood (glomeruli). When the glomeruli become damaged or scarred, proteins […]

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Musculoskeletal System

Vosoritide for treating achondroplasia in children

Vosoritide is in clinical development for the treatment of achondroplasia in children aged 0 to 59 months. Achondroplasia is a rare genetic bone growth disorder and the most common form of disproportionate short stature. Achondroplasia is caused by a change in the structure of a gene called the fibroblast growth factor receptor 3 (FGFR3) gene, […]

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Dermatology

Guselkumab for treating chronic plaque psoriasis in children aged 6 to 17 years

Guselkumab is in clinical development for the treatment of chronic plaque psoriasis in children aged 6 to 17 years. Psoriasis is a skin condition that causes flaky patches of skin which form scales. Chronic plaque psoriasis is the most common form of psoriasis. The ‘plaques’ are formed due to the build-up of skin cells and […]

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Female Reproductive Cancer

Dostarlimab with paclitaxel and carboplatin for recurrent or primary advanced endometrial cancer

Dostarlimab in combination with paclitaxel and carboplatin is in clinical development for the treatment of primary advanced or recurrent endometrial cancer. Endometrial cancer is cancer of the lining of the womb. Advanced cancer is when the cancer has spread to other parts of the body; recurrent cancer means the cancer has come back after treatment. […]

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Dermatology

Delgocitinib for treating moderate to severe chronic hand eczema after 1 previous therapy

Delgocitinib is currently in clinical development for the treatment of adults with moderate to severe chronic hand eczema. Hand eczema is one of the most common types of eczema. It mainly affects the palms but can also affect other parts of the hand. Chronic hand eczema is hand eczema that lasts for more than three […]

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Haematological Cancer and Lymphomas

Subcutaneous isatuximab with pomalidomide and dexamethasone for treating relapsed and/or refractory multiple myeloma

Subcutaneous isatuximab in addition to pomalidomide and dexamethasone is in clinical development for the treatment of patients with relapsed and/or refractory multiple myeloma (RRMM). Multiple myeloma (MM) is a rare, incurable cancer of the plasma cells in the bone marrow. In MM, large amounts of abnormal plasma cells are produced which fill the bone marrow […]

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Lung and Respiratory Cancer

Lurbinectedin with atezolizumab maintenance therapy for extensive-stage small-cell lung cancer

Lurbinectedin in combination with atezolizumab is in clinical development for patients with extensive stage small-cell lung cancer (ES-SCLC) after first-line induction therapy. Small-cell lung cancer (SCLC) is a type of lung cancer that usually develops in the central part of the lungs, and in which the cancer cells are small compared with other types of […]

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Haematological Cancer and Lymphomas

Pirtobrutinib for untreated chronic lymphocytic leukaemia or small lymphocytic lymphoma

Pirtobrutinib is currently in clinical development for untreated chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL). CLL is a rare type of cancer that affects the blood and bone marrow (the spongy inner part of the bone that produces red and white blood cells). CLL and SLL are the same disease, but whereas in […]

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