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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

Atezolizumab with cabozantinib for advanced renal cell carcinoma after 1 therapy

Atezolizumab in combination with cabozantinib is in clinical development for the treatment of advanced renal cell carcinoma (RCC) after prior treatment. RCC (also called kidney cancer) is a disease that affects the lining of tiny tubes within the kidney which filter waste from the blood, making urine. In many cases of RCC there are no […]

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Gastrointestinal Cancer

Nivolumab-ipilimumab for previously untreated advanced hepatocellular carcinoma

Nivolumab and ipilimumab is in development for the first-line treatment of advanced hepatocellular carcinoma (HCC). HCC is the most common type of primary liver cancer, which affects men more than women, and is more likely to develop the older a person gets. Advanced HCC means the cancer is incurable. Symptoms include weight loss, jaundice (yellowing […]

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Rheumatology

Otilimab in combination with conventional synthetic DMARDs for treating rheumatoid arthritis

Otilimab in combination with conventional synthetic disease-modifying antirheumatic drugs (DMARDs) is in clinical development for the treatment of adult patients with moderately to severely active rheumatoid arthritis (RA), who have had an inadequate response to conventional synthetic DMARDs and/or targeted therapies (biologic DMARDs and Janus Kinase inhibitors). RA is a chronic autoimmune condition where the […]

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Urological Cancer

Durvalumab with gemcitabine-cisplatin neoadjuvant treatment and durvalumab adjuvant treatment for muscle-invasive bladder cancer

Neoadjuvant treatment of durvalumab in combination with gemcitabine-cisplatin and adjuvant treatment of durvalumab alone are currently in clinical development for patients with muscle-invasive bladder cancer (MIBC). Bladder cancer is where a growth of abnormal tissue, known as a tumour, develops in the bladder lining. When the cancerous cells spread beyond the lining, into the surrounding […]

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Male Reproductive Cancer

Niraparib with abiraterone acetate and prednisone for metastatic castration resistant prostate cancer

Niraparib with abiraterone acetate and prednisone (AAP) is in clinical development for patients with prostate cancer which has spread from its original site (metastatic) and is untreatable via testosterone suppression therapy (castration resistant). Prostate cancer is a cancer of the prostate gland (a small organ in a man’s pelvis) and is the most common cancer […]

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Infectious Disease

Letermovir for preventing cytomegalovirus disease in kidney transplant patients

Letermovir is in clinical development for adult patients who have received a kidney transplant and are at risk of developing cytomegalovirus disease (CMV). CMV is normally a mild infection that causes flu-like symptoms but can be life-threatening in certain groups. Once infected with CMV, the virus stays in the body throughout a person’s life with […]

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Haematology

Efanesoctocog alfa for treating and preventing bleeding in people with haemophilia A who have been previously treated

Efanesoctocog alfa is currently in clinical development for the treatment and prevention (prophylaxis) of bleeding episodes in patients with haemophilia A. Haemophilia A is a genetic bleeding disorder caused by a deficiency or absence of a blood clotting factor, called factor VIII (eight). This causes poor blood clotting, which results in difficulty in stopping the […]

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Gastrointestinal Cancer

Ivosidenib for previously treated advanced cholangiocarcinoma

Cholangiocarcinoma is a rare type of cancer that affects the bile ducts. Different subtypes of cholangiocarcinoma are classified by the location at which they develop in the biliary tract. Genetic changes (mutations) to the metabolic enzyme isocitrate dehydrogenase 1 (IDH1) plays a central role in development of cholangiocarcinoma by contributing to the growth of abnormal […]

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Male Reproductive Cancer

Enzalutamide for treating non-metastatic prostate cancer after radical prostatectomy and/or radiotherapy

Enzalutamide is currently in clinical development for the treatment of high-risk non-metastatic prostate cancer (cancer that has not spread beyond the prostate) that has been previously treated with a radical prostatectomy (surgery to remove the prostate gland) and/or radiotherapy. Prostate cancer develops in the prostate gland and is the most common cancer in men in […]

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Renal Cancer

Nivolumab with ipilimumab adjuvant therapy for renal cell carcinoma

Nivolumab in combination with ipilimumab is in clinical development as adjuvant therapy for adult patients with localised renal cell carcinoma (RCC) who underwent partial or entire removal 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 […]

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Neurology

Mirabegron for neurogenic detrusor overactivity in people aged 3 to 17 years

Mirabegron is currently in clinical development for treatment of neurogenic detrusor overactivity(NDO) in children and adolescents aged 3 to 17 years old on clean intermittent catheterisation(CIC). NDO is a bladder dysfunction and can cause urinary symptoms such as frequency, urgencyand urinary incontinence. Other symptoms of NDO include: pelvic pain, painful urination,incontinence, increased spasticity, autonomic dysreflexia, […]

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Lung and Respiratory Cancer

Tislelizumab with etoposide and carboplatin or cisplatin for treating small-cell lung cancer

Tislelizumab in combination with chemotherapy is currently in clinical development for the first-line treatment of extensive stage small-cell lung cancer (SCLC). Extensive stage SCLC is whencancer cells form in the tissues of the lung and spread beyond the lung to other places in the body.Symptoms include chest discomfort or pain, a cough that does not […]

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Lung and Respiratory Cancer

Pembrolizumab with stereotactic body radiotherapy for unresected stage I or II non-small cell lung cancer

Pembrolizumab with stereotactic body radiotherapy (SBRT) is currently in development for patients withunresected stage I or II (stage IIB N0, M0) non-small cell lung cancer (NSCLC). NSCLC is the most commontype of lung cancer. Stage I NSCLC is small; contained inside the lung and has not spread to lymph nodes;and stage IIB is up to […]

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Gastrointestinal Cancer

Tislelizumab with chemotherapy for previously untreated unresectable or metastatic gastric cancer or gastro-oesophageal junction adenocarcinoma

Tislelizumab in combination with chemotherapy is being developed for the first-line treatment oflocally advanced, unresectable or metastatic gastric cancer (GC) or gastroesophageal junction(GEJ) adenocarcinoma. GC is a cancer that starts in the stomach. GEJ adenocarcinoma is cancerthat starts at the gastro-oesophageal junction, where the food pipe (oesophagus) joins thestomach. Risk factors of GC and GEJ […]

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Dermatology

Dersimelagon for previously untreated erythropoietic protoporphyria and X-linked protoporphyria

Dersimelagon is being developed for the treatment of erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP). EPP and XLP are rare diseases that cause intolerance to light. Inpatients with EPP or XLP, exposure to light can lead to symptoms such as pain and swelling of theskin, which prevent patients from being able to spend time outdoors […]

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Haematological Cancer and Lymphomas

Acalabrutinib with bendamustine and rituximab for treating previously untreated mantle cell lymphoma

Acalabrutinib in combination with bendamustine and rituximab is in clinical development for thefirst line treatment of mantle cell lymphoma (MCL). MCL is a rare and aggressive form of nonHodgkin lymphoma. It affects the B-cells (a type of white blood cells) and develops in the lymphnodes. The abnormal B-cells start to collect in the lymph nodes […]

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Breast Cancer

Ribociclib with endocrine therapy for HR+/HER2- early breast cancer – adjuvant treatment

Ribociclib with endocrine therapy (ET) is currently in clinical development for patients with earlybreast cancer with hormone receptor positive (HR+)/human epidermal receptor 2 negative(HER2-) subtype. Early breast cancer is where the disease is limited to the breast region and hasnot spread to other parts of the body. Symptoms include swelling of breast regions, breast ornipple […]

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Genetic Disorders

Bardoxolone Methyl for the treatment of chronic kidney disease (CKD) caused by Alport Syndrome

Bardoxolone methyl is in clinical development for the treatment of patients aged 12 years andolder with Alport syndrome. Alport syndrome is a genetic condition characterized by kidneydisease, hearing loss, and eye abnormalities. It is caused by a defect (mutation) in a gene for aprotein in the connective tissue, called collagen. There are currently no licensed […]

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Breast Cancer

Atezolizumab with chemotherapy for treating recurrent triple-negative breast cancer

Atezolizumab with chemotherapy is currently in clinical development for the treatment of earlyrelapsing, recurrent triple-negative breast cancer (TNBC). TNBC is an uncommon form kind ofbreast cancer where the tumour cells do not have any of the receptors that are commonly foundin breast cancer. TNBC is often more aggressive and harder to treat compared to other […]

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Cardiovascular System

Sacubitril-valsartan for previously untreated chronic heart failure with reduced ejection fraction in children

Sacubitril-valsartan is currently in clinical development for the treatment of paediatric chronicheart failure (HF) with reduced ejection fraction. HF is a clinical syndrome of symptoms and signsthat suggest the efficiency of the heart to pump blood around the body is impaired. The classicalpresenting symptoms of HF in young children are feeding difficulties, growth failure, irritability,and […]

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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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