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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 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

Odevixibat for cholestatic liver disease in Alagille syndrome patients

Odevixibat is currently in clinical development for the treatment of cholestatic liver disease in Alagille syndrome (ALGS) patients. ALGS is an inherited developmental disorder, caused by a mutation in the JAGGED1 gene or in the NOTCH2 gene. The mutation causes problems with early embryonic development leading to abnormalities in various parts of the body. ALGS […]

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Haematological Cancer and Lymphomas

ASTX727 for acute myeloid leukaemia

ASTX727 is in clinical development for patients with de novo or secondary acute myeloid leukaemia (AML), who are not candidates for standard induction chemotherapy. De novo AML is where there has been no past medical history of the condition or exposure to the drugs which commonly treat it, secondary AML is where patients have had […]

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Neurology

Natalizumab biosimilar for treating relapsing-remitting multiple sclerosis

Natalizumab biosimilar is being developed as a biosimilar medicine to the approved monoclonal antibody natalizumab and is proposed to be used to treat adult patients relapsing remitting multiple sclerosis (RRMS). Multiple sclerosis is an autoimmune condition where the body’s immune system attacks the brain and nerves. RRMS is characterised by attacks of new or increasing […]

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Haematological Cancer and Lymphomas

REGN5458 for relapsed or refractory multiple myeloma

REGN5458 is in clinical development for the treatment of adults with multiple myeloma (MM) whose disease has returned following treatment. MM is a form of cancer that occurs in immune cells found in bone marrow. The disease occurs due to uncontrolled duplication of these immune cells, known as plasma cells. Symptoms of MM can include […]

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Cardiovascular System

Empagliflozin for treating chronic kidney disease

Empagliflozin is in clinical development for the treatment of adult patients with chronic kidney disease (CKD). In CKD, the kidneys do not function as well as they should. This leads to the leakage of blood and protein into the urine. CKD can be developed due to age, but the main risk factors are diabetes, hypertension, […]

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Genetic Disorders

Fordadistrogene movaparvovec for Duchenne muscular dystrophy

Fordadistrogene movaparvovec is a medicinal product in clinical development for the treatment of ambulatory boys greater than or equal to 4 years of age to 7 years with a confirmed genetic diagnosis of Duchenne muscular dystrophy (DMD). DMD is a severe and rare progressive neuromuscular disorder caused by a gene mutation (change). DMD is caused […]

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Dermatology

Bimekizumab for Hidradenitis Suppurativa

Bimekizumab is in clinical development for the treatment of Hidradenitis Suppurativa (HS) in adults. HS is a painful, long-term skin condition that causes abscesses and scarring on the skin. The exact cause of hidradenitis suppurativa is unknown, but it occurs near hair follicles where there are sweat glands, usually around the groin, bottom, breasts and […]

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Gastrointestinal Cancer

Tislelizumab for treating unresectable, previously untreated hepatocellular carcinoma

Tislelizumab is being developed for the first-line treatment of unresectable hepatocellular carcinoma (HCC). HCC is the most common type of liver cancer and the leading cause of cancer related death worldwide. This type of cancer develops from the main liver cells, called hepatocytes. Liver cancer is more common in people who have long‐term damage to […]

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Genetic Disorders

Leniolisib for previously untreated activated phosphoinositide 3-kinase delta syndrome

Leniolisib is in clinical development for patients with Activated Phosphoinositide 3-kinase Delta Syndrome (APDS). APDS is an inherited disorder where the patient is unable to fight infections because the immune system (the body's natural defences) does not work properly. APDS is caused by defects in the genes that control the production of a protein called […]

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Oncology

Glucarpidase for methotrexate toxicity

Glucarpidase (Voraxaze), previously known as carboxypeptidase G2, is a carboxypeptidase G2 enzyme that hydrolyses the terminal glutamate residue from methotrexate.2 Glucarpidase hydrolyses methotrexate rapidly to the inactive metabolites DAMPA (4-[[2,4-diamino-6-(pteridinyl)methyl]-methylamino]-benzoic acid) and glutamate in patients with methotrexate-induced nephrotoxicity and delayed methotrexate excretion.3 It has been shown that the conversion of methotrexate to DAMPA relieves stress […]

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Gastroenterology

Teduglutide for short bowel syndrome in infants

Teduglutide is in clinical development for infants aged four to twelve months with short bowel syndrome (SBS) who are dependent on parenteral support (PS). SBS is a complex disease that occurs due to the physical loss or the loss of function of a portion of the small and/or large intestine. Consequently, individuals with SBS often […]

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Lung and Respiratory Cancer

Amivantamab with chemotherapy for previously untreated advanced EGFR exon 20 insertion mutations positive non-small cell lung cancer

Amivantamab in combination with carboplatin-pemetrexed chemotherapy is in development forthe treatment of epidermal growth factor receptor (EGFR) exon 20 insertion mutations positivenon-small cell lung cancer (NSCLC). NSCLC is one of the most common types of cancer. EGFR isa protein found on certain types of cells that is involved in cell signalling pathways that controlcell division […]

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Mental and Behavioural Health

Dexmedetomidine film for agitation associated with bipolar disorder

Dexmedetomidine sublingual film is in clinical development for the treatment of agitationassociated with bipolar disorder. Bipolar disorder is mental health condition characterised byperiods of mania and severe depression. Agitation is a disruptive, and comorbid complication ofmany chronic mental illnesses, including bipolar disorders. Agitation is described as excessivemotor activity associated with a feeling of inner tension. […]

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Mental and Behavioural Health

Dexmedetomidine film for agitation in schizophrenia

Schizophrenia is a severe long-term mental condition which can affect a person’s perception andinterpretation of the world around them. Symptoms include hallucinations (hearing and seeingthings), delusions (beliefs not based on reality) and agitation. It is not known what causesschizophrenia, but it is likely a combination of genetic and environmental factors. It affects bothmen and women […]

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Nephrology

Ravulizumab for treatment of neuromyelitis optica spectrum disorder

Ravulizumab is in clinical development for the treatment of neuromyelitis optica spectrumdisorder (NMOSD) NMOSD is a rare autoimmune disease where the immune system attacks thenerves in the eyes and central nervous system (CNS), which can result in weakness, paralysis, pain,blindness, and premature death. The condition often has frequent relapses which can result inprogressive disabilities for […]

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Lung and Respiratory Cancer

Lenvatinib with pembrolizumab for previously treated metastatic non-small cell lung cancer

Lenvatinib with pembrolizumab is in development for the treatment of metastatic non-small celllung cancer (NSCLC). NSCLC is the most common form of lung cancer. Metastatic NSCLCdescribes tumours that have spread from the lungs to other parts of the body. Smoking tobaccois the cause of most lung cancers and the biggest risk factor. Other risk factors […]

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Gastrointestinal Cancer

Tislelizumab with chemotherapy for previously
untreated advanced oesophageal squamous cell
carcinoma

Tislelizumab with chemotherapy is in clinical development to treat patients with advancedoesophageal squamous cell carcinoma (ESCC). ESCC is a type of cancer that begins in the thin,flat cells lining the food pipe (oesophagus) and may spread to other parts of the body. Symptomsinclude difficulty swallowing, persistent indigestion or heartburn, weight loss, pain in the throat,and […]

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Gastroenterology

Dupilumab for treating eosinophilic oesophagitis in people aged 12 years and older

Dupilumab is in clinical development for the treatment of eosinophilic oesophagitis (EoE) in adultsand adolescents. EoE is a condition that affects the gullet (oesophagus). There is a build-up of aparticular type of immune cell (eosinophils) in the lining of the gullet causing inflammation orirritation. This affects how the gullet works, causing symptoms such as difficulty […]

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Lung and Respiratory Cancer

Canakinumab adjuvant therapy for treating completely
resected non-small-cell lung cancer

Canakinumab is in clinical development as an adjuvant therapy for stage II-III non-small cell lungcancer (NSCLC) that has been surgically completely removed. NSCLC accounts for most lungcancers that are diagnosed and can originate anywhere in the lungs but may spread to surroundingtissues as the disease progresses. Adjuvant therapy is a treatment given after the primarytreatment, […]

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Lung and Respiratory Cancer

Durvalumab with tremelimumab for limited stage small-cell lung cancer

Durvalumab in combination with tremelimumab is in clinical development for patients with limitedstage small cell lung cancer (LS-SCLC). SCLC is a fast-growing cancer that forms in the tissues ofthe lung and may have spread to the area between the lungs or to the lymph nodes above thecollarbone. SCLC is described as limited if the cancer […]

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Endocrine Nutritional and Metabolic Disorders

Teplizumab for the delay of clinical type-1 diabetes in at-risk patients aged 8 years and over

Teplizumab is in clinical development for the delay of clinical type-1 diabetes (T1D) in individuals at risk of developing the disease. T1D is a condition which usually starts early in life where the immune system (blood cells which usually defend the body from infection) attacks an organ called the pancreas. The pancreas usually makes a […]

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Haematology

Danicopan with eculizumab or ravulizumab for treating paroxysmal nocturnal haemoglobinuria

Danicopan is in clinical development as an add-on therapy to eculizumab or ravulizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH). PNH is a rare, long-term and life-threatening blood disease in which red blood cells (RBCs) are attacked by a group of proteins in the body’s immune system, which are collectively known […]

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Haematological Cancer and Lymphomas

Zanubrutinib for previously untreated chronic lymphocytic leukaemia or small lymphocytic lymphoma

Chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL) are types of cancer in which too many white blood cells (B-cells) are produced, these cells build up in the lymph nodes, blood, and bone marrow. As these cells develop abnormally, they are unable to function, fight infection and reduce the production of healthy blood cells. […]

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Female Reproductive Cancer

Olaparib maintenance therapy for non-gBRCA mutated high-grade serous ovarian cancer after platinum-based chemotherapy

Olaparib is in clinical development for the maintenance treatment of adults with platinum-sensitive non-germline breast cancer susceptibility gene (non-gBRCA) mutated high-grade serous ovarian cancer, including fallopian tube cancer or primary peritoneal cancer. These cancers occur in the female reproductive system and are often grouped together as they are treated in the same way. Tumours can […]

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Neurology

Oxybutynin hydrochloride for treating neurogenic detrusor overactivity

Oxybutynin hydrochloride is in clinical development for the treatment of neurogenic detrusor overactivity (NDO), in patients managing bladder emptying by clean intermittent catheterisation. Damage to the spinal cord, via spina bifida, multiple sclerosis or injuries, can disrupt communication between the spinal cord and bladder causing NDO. NDO increases pressure in the bladder and decreases the […]

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Haematological Cancer and Lymphomas

Zanubrutinib for relapsed or refractory chronic lymphocytic leukaemia or small lymphocytic lymphoma

Chronic lymphocytic leukaemia (CLL) and small lymphocytic lymphoma (SLL) are types of cancer in which too many white blood cells (B-cells) are produced, these cells build up in the lymph nodes, blood, and bone marrow. As these cells develop abnormally, they are unable to function, fight infection and reduce the production of healthy blood cells. […]

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Female Reproductive Cancer

Durvalumab add-on therapy for previously untreated locally advanced cervical cancer

Durvalumab is in clinical development as an add-on treatment of female adults with previously untreated, locally advanced cervical cancer. Durvalumab will be added to the current treatment of concurrent chemotherapy and radiation therapy (cCRT). Cervical cancer arises when abnormal cells lining the cervix begin to grow uncontrollably and create tumours. Cervical cancer may not present […]

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Haematological Cancer and Lymphomas

Zanubrutinib for treating marginal zone lymphoma

Zanubrutinib is currently in clinical development for treating relapsed or refractory marginal zone lymphoma (MZL). MZL is a rare group of non-Hodgkin’s lymphomas that affect a type of immune cell called B-cells. These MZLs are slow growing and do not always cause symptoms. Broad symptoms that do sometimes appear in MZL patients include unexplained weight […]

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Haematological Cancer and Lymphomas

Duvelisib for treating relapsed and/or refractory peripheral T-cell lymphoma

Duvelisib is in development for relapsed or refractory (R/R) peripheral T-cell lymphoma (PTCL), a group of lymphomas (cancer that begins in cells of the immune system), that develop from mature T-cells, which are a type of white blood cell. Relapsed means that the disease has reappeared after a period of remission; refractory is used to […]

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Breast Cancer

Giredestrant with palbociclib for previously untreated advanced or metastatic breast cancer

Giredestrant combined with palbociclib is in clinical development for previously untreated oestrogen receptor (ER)-positive, human epidermal growth factor receptor-2 (HER2)-negative locally advanced (recurrent or progressed) or metastatic breast cancer. Breast cancer arises from the tissues of the breast and most commonly originates in the cells that line the milk ducts. This kind of ER-positive cancer […]

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