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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

Neurology

Masitinib with Riluzole for amyotrophic lateral sclerosis

Masitinib in combination with riluzole is in clinical development for the treatment of patients withthe amyotrophic lateral sclerosis (ALS), form of motor neurone disease (MND). ALS is aprogressive disease of the nervous system, where nerve cells in the brain and spinal cord thatcontrol voluntary movement gradually deteriorate, causing loss of muscle function and paralysis.ALS is […]

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Haematology

Crovalimab for treatment of paroxysmal nocturnal haemoglobinuria

Crovalimab is currently in clinical development for patients with paroxysmal nocturnalhaemoglobinuria (PNH). PNH is a rare, acquired, life-threatening disease of the blood. The diseaseis characterised by destruction of red blood cells, blood clots, and impaired bone marrow function.It is a blood condition where blood cells are prone to be attacked by part of the body’s […]

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Cardiovascular System

Macitentan-tadalafil for treating pulmonary arterial hypertension

Macitentan and tadalafil as a fixed-dose combination (FDC) is in clinical development for thetreatment of pulmonary arterial hypertension (PAH). PAH is a rare disorder in which there issevere narrowing of the arteries of the lungs. More pressure is needed to force blood through thenarrowed artery which this leads to high blood pressure in the lungs

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Female Reproductive Cancer

Pembrolizumab with chemoradiotherapy for treating high-risk locally advanced cervical cancer

Pembrolizumab in combination with chemoradiotherapy is in clinical development for thetreatment of patients with high-risk, locally advanced cervical cancer. Cervical cancer ariseswhen abnormal cells lining the cervix begin to grow uncontrollably and create tumours. Cervicalcancer may not present symptoms, however the most common symptoms seen are unusualvaginal bleeding, pain during sex, vaginal discharge and pain […]

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Haematological Cancer and Lymphomas

Momelotinib for the treatment of symptomatic and anaemic myelofibrosis

Momelotinib is currently in clinical development for the treatment of symptomatic myelofibrosiswith anaemia. Myelofibrosis is a rare blood cancer, where the bone marrow is too active so scartissue builds up inside the bone marrow and blood cells cannot develop properly. The reductionin blood cells can result in anaemia, amongst other symptoms such as tiredness, shortness […]

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Endocrine, nutritional and metabolic disorders

Ranibizumab port delivery system for treating
diabetic macular oedema

The port delivery system with ranibizumab (PDS) is in clinical development for the treatment ofdiabetic macular oedema (DMO). DMO is swelling of the macula caused by diabetes or byblockage of the veins behind the retina and it is the most common cause of sight loss in peoplewith diabetes. DMO occurs when blood vessels damaged by […]

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Gastrointestinal Cancer

Atezolizumab with bevacizumab for patients with resected or ablated hepatocellular carcinoma who are at high risk for disease recurrence

Atezolizumab and bevacizumab in combination is in clinical development for treating adults with high riskof hepatocellular carcinoma (HCC) coming back (recurring), after complete surgical removal(resection/ablation) of the affected tissue. HCC is the most common type of primary liver cancer, whichaffects men more than women, and is more likely to develop the older a person gets.

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Haematological Cancer and Lymphomas

Idecabtagene vicleucel for treating relapsed or
refractory multiple myeloma after 2 therapies

Idecabtagene vicleucel is in clinical development for the treatment of adult patients with relapsedor refractory multiple myeloma (MM) who have received at least 2 but no greater than 4 priorMM regimens. MM is a rare, incurable blood cancer that forms in the plasma calls in the bonemarrow, inside some of the large bones of the […]

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Rheumatology

Baricitinib for treating juvenile idiopathic arthritis in paediatric patients

Baricitinib is in clinical development for children aged 1 to 17 years with juvenile idiopathicarthritis (JIA). JIA is defined as arthritis of unknown causes that manifests itself before the age of16 years and persists for at least 6 weeks, while excluding other known conditions. JIA occurswhen the body's immune system attacks its own cells and […]

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Endocrine, nutritional and metabolic disorders

Semaglutide for treating overweight and obesity in adolescents ages 12 to 17 years old

Semaglutide is in clinical development for the treatment of overweight and obese adolescentsbetween the ages of 12 and 17. Obesity is a chronic disease and global public health challengeand the prevalence of childhood obesity rising in the UK. Obesity increases the risk of developinga range of health conditions in childhood and later life, including stroke, […]

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Gastrointestinal Cancer

Durvalumab with bevacizumab adjuvant therapy for treating newly diagnosed hepatocellular carcinoma at high risk of recurrence after curative therapy

Durvalumab with bevacizumab is intended to treat hepatocellular carcinoma (HCC), the mostcommon type of liver cancer, where there is a high risk of the cancer returning after curativetreatment. It is common that people with HCC are asymptomatic (do not show symptoms relatedto the cancer). There is increased risk of developing HCC if you have underlying […]

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Haematology

ABP 959 for treating paroxysmal nocturnal
haemoglobinuria

ABP 959 is in development for the treatment of paroxysmal nocturnal haemoglobinuria (PNH).PNH is a rare condition, which occurs due to a genetic mutation within stem cells in the bonemarrow. In PNH, red blood cells lack specific proteins on their surface which normally protectthem from being destroyed (a process called haemolysis) by the body's normal […]

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Neurology

Cenobamate as an adjunctive therapy for primary generalised tonic-clonic seizures

Cenobamate is in clinical development as an adjunctive therapy for adults with primarygeneralized tonic-clonic (PGTC) seizures. A generalised tonic-clonic seizure is the type of epilepticseizure which is defined as a seizure that has a tonic phase (stiffening of the muscles) followed byclonic muscle contractions (rhythmical jerking).

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Immunology

AR101 for peanut allergy in children aged 1 – 3 years

A food allergy is when the body’s immune system reacts to proteins from specific foods, as if itwere a threat. Common foods that cause an allergic reaction are milk, eggs, peanuts, tree nuts,fish, and shellfish. Most food allergies affect younger children under the age of 3 years old,however peanut and tree nut allergies are longer-lasting […]

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Neurology

Gantenerumab for Alzheimer’s disease

Gantenerumab 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, the cells that carry signals or information messages to and from the brain and the restof the body. It is the most common type of dementia.

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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 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 by an absence of functional dystrophin, a protein that helps keep muscle […]

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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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