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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

Haematological Cancer and Lymphomas

Zanubrutinib for previously treated mantle cell lymphoma

Zanubrutinib is in clinical development for the treatment of mantle cell lymphoma (MCL) in adultswho have received at least one prior treatment. MCL is a rare type of B-cell non-Hodgkinlymphoma (NHL), a cancer of the lymphatic system. The lymphatic system branches through allparts of the body carrying a liquid called lymph. The lymph contains a […]

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Haematological Cancer and Lymphomas

Favezelimab-Pembrolizumab for treating relapsed or refractory classical Hodgkin Lymphoma after 2 or more therapies

Co-formulated favezelimab-pembrolizumab is in clinical development for patients with relapsed orrefractory classical Hodgkin Lymphoma (cHL) who have exhausted all available treatment options andwhose disease has progressed on treatment with an anti-PD-(L)1 monoclonal antibody. Hodgkinlymphoma is an uncommon cancer that develops in the lymphatic system, which is a network of vesselsand glands spread throughout your body. […]

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Breast Cancer

Tucatinib with trastuzumab and pertuzumab for maintenance treatment of unresectable locally advanced or metastatic HER2-positive breast cancer

Tucatinib in combination with trastuzumab and pertuzumab is in clinical development for the maintenance treatment of unresectable locally advanced, or metastatic HER2-positive (HER2-POSITIVE) breast cancer. Breast cancer is cancer that starts in the breast tissue. HER2-positive breast cancer means that the cancer cells express more of the HER2 protein on their surface. HER2 encourages cell […]

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Haematological Cancer and Lymphomas

Tafasitamab with lenalidomide as add-on therapy for previously treated follicular lymphoma or marginal zone lymphoma

Tafasitamab with lenalidomide (as an add-on to rituximab) is in clinical development for treating relapsed or refractory follicular lymphoma (FL) of any grade except 3B, or marginal zone lymphoma (MZL). These are slow-growing types of cancer that develop when white blood cells, called B lymphocytes, grow out of control. Common symptoms include painless swellings in […]

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Gastroenterology

Ustekinumab for the treatment of moderately to severely active Crohn's disease in children

Ustekinumab is in clinical development for the treatment of moderately to severely active Crohn’s disease in children. Crohn’s disease is an autoimmune disease, where the immune system attacks the body, and in Crohn’s disease this causes ulcers and inflammation in the gut wall. Symptoms include diarrhoea, stomach aches and cramps, blood in the faeces, fatigue […]

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Haematology

Rilzabrutinib for treating persistent or chronic immune thrombocytopenia

Rilzabrutinib is currently in clinical development for the treatment of persistent or chronic immune thrombocytopenia (ITP) in adult. ITP is an auto-immune disease that arises when the body’s immune system attacks platelet cells resulting in a decreased number of platelets (platelet count) and impairs platelet production. Platelets are a specialised type of blood cell that […]

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Breast Cancer

Sacituzumab govitecan for treating previously untreated locally advanced or metastatic triple negative breast cancer with negative PD-L1 status

Sacituzumab govitecan is in clinical development for the treatment of adult patients with previously untreated locally advanced or metastatic triple negative breast cancer (TNBC) with PD-L1- status. TNBC is a rare form of breast cancer where the tumour cells do not have hormone response receptors such as oestrogen or progesterone. PD-L1 is a protein involved […]

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Neurology

Tolebrutinib for treating relapsing and progressive forms of multiple sclerosis

Tolebrutinib is currently in clinical development for the treatment of relapsing and progressive forms of multiple sclerosis (MS). MS is a chronic autoimmune condition that affects the brain and spinal cord. It can lead to increased mortality and is one of the most common causes of disability in younger adults. Relapsing remitting multiple sclerosis (RRMS) […]

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Musculoskeletal System

Nipocalimab for Generalised Myasthenia Gravis

Nipocalimab is currently in clinical development for the treatment of adults with generalised myasthenia gravis (gMG). gMG is a rare, long-term autoimmune disorder that leads to muscle weakness and tiredness, which can be seriously debilitating and life-threatening, affecting eye alignment, swallowing, speech, mobility and respiratory function. These symptoms can significantly impair independence and quality of […]

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Male Reproductive Cancer

Apalutamide for treating high-risk, localised or locally advanced prostate cancer

Apalutamide in combination with androgen deprivation therapy (ADT) is in clinical development for patients with high-risk, localised or locally advanced prostate cancer who are candidates for radical prostatectomy. Prostate cancer is a cancer of the prostate gland (a small organ in a man’s pelvis) and is the most common cancer in older men. Locally advanced […]

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Gastroenterology

Golimumab for treating moderately to severely active ulcerative colitis in children aged 2 to 17 years

Golimumab is currently in clinical development for the treatment of moderately to severely activeulcerative colitis (UC) among paediatric patients aged 2 to 17 years. UC is a long-term conditionwhere the colon and rectum become inflamed and small ulcers can develop on the colon's lining,which may bleed and produce pus. Symptoms of UC include recurring diarrhoea, […]

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Immunology

Ruxolitinib for grades II-IV treatment-naïve or steroid-refractory acute graft versus host disease

Ruxolitinib is in clinical development for the treatment of patients aged 28 days to 17 years with a clinically confirmed diagnosis of grades II-IV treatment-naïve or steroid-refractory acute graft versus host disease (aGvHD) and who have undergone allogeneic stem cell transplantation (alloSCT) from any donor source using bone marrow, peripheral blood stem cells, or cord […]

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Ophthalmology

NVK-002 for myopia in children

NVK-002 is currently in clinical development for the treatment of myopia in children. Myopia, also known as short-sightedness, is an eye condition that means a person is unable to see objects that are far away very clearly due to changes in the shape of the eye structure. The condition usually starts in children aged between […]

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Female Reproductive Cancer

Pembrolizumab with chemotherapy for treating advanced or recurrent endometrial cancer

Pembrolizumab with carboplatin and paclitaxel is in development for the treatment of endometrial cancer (cancer of the womb lining) when it has started to spread to other parts of the body (advanced) or has come back after initial treatment (recurrent). Symptoms of endometrial cancer can include abnormal vaginal bleeding, pelvic pain, painful urination or pain […]

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Haematological Cancer and Lymphomas

Isatuximab induction therapy with bortezomib, lenalidomide and dexamethasone for previously untreated multiple myeloma

Isatuximab in addition to bortezomib, lenalidomide and dexamethasone is currently in clinical development as an induction therapy option for patients with newly diagnosed multiple myeloma (MM)who are eligible for autologous stem cell transplant (ASCT). MM is a type of bone marrow cancer that often affects several areas of the body. MM has premalignant or asymptomatic […]

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Neurology

SAR443820 for treating amyotrophic lateral sclerosis (ALS)

SAR443620 is in phase II clinical development for the treatment of patients with amyotrophic lateral sclerosis (ALS), a form of motor neurone disease. ALS is a progressive disease where the nerve cells responsible for sending instructions to the muscles gradually deteriorate, leading to weakness, muscle wasting and paralysis. ALS is a debilitating and fatal disease. […]

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Neurology

Botulinum toxin type A for preventing episodic migraine

Botulinum toxin type A (an active constituent in BOTOX) is currently in clinical development for the prevention of episodic migraine. Migraine is a debilitating neurological condition that commonly causes extremely painful headaches. Episodic migraine is defined as a headache which occurs on less than 15 days per month. Migraines may be triggered by any of […]

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Endocrine Nutritional and Metabolic Disorders

IcoSema for treating adults with type 2 diabetes

IcoSema is currently in phase 3 clinical development for type 2 diabetes. Type 2 diabetes is a lifelong condition that develops when the body becomes resistant to or does not produce enough insulin – a hormone produced in the pancreas that enables sugar to enter body cells. Signs and symptoms of type 2 diabetes often […]

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Neurology

Opicapone adjuvant for the treatment of idiopathic Parkinson's disease

Parkinson’s disease (PD) is a chronic, progressive neurodegenerative condition resulting from the loss of dopamine nerve cells in the brain. This leads to a reduction in a chemical called dopamine in the brain, which plays a vital role in regulating movement of the body. The diagnosis and treatment of PD typically occur when the disease […]

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Lung and Respiratory Cancer

Vibostolimab-pembrolizumab for previously untreated PD-L1-positive metastatic non-small cell lung cancer

Vibostolimab-pembrolizumab is currently in clinical development for previously untreated programmed cell death 1 ligand 1 (PD-L1) positive metastatic non-small cell lung cancer (NSCLC). NSCLC is the most common form of lung cancer. Elevated levels of PD-L1 expression are observed on the cell surface of different types of cancer cells, including NSCLC. It is believed that […]

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Lung and Respiratory Cancer

Tiragolumab with atezolizumab and pemetrexed and carboplatin or cisplatin for previously untreated advanced non-small-cell lung cancer

Tiragolumab in combination with atezolizumab, pemetrexed and carboplatin or cisplatin is currently in development for patients with previously untreated locally advanced unresectable or metastatic programmed death-ligand 1 (PD-L1) selected non-small cell lung cancer (NSCLC). NSCLC makes up most lung cancers in the UK. Metastatic NSCLC is when the cancer has spread beyond the lung that […]

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Gastrointestinal Cancer

Zanidatamab for treating advanced or metastatic HER2-positive biliary tract cancer in adults

Zanidatamab is being developed for adult patients with advanced or metastatic human epidermal growth factor-2 (HER2)-positive biliary tract cancer (BTC). BTCs are a rare and diverse group of aggressive cancers arising from the gallbladder and bile ducts. The bile ducts are tubes that link the gallbladder, liver, and pancreas together, all of which are used […]

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Gastroenterology

Budesonide suspension for treating eosinophilic oesophagitis in children and adolescents

Budesonide suspension is currently in clinical development for treating eosinophilic oesophagitis in children and adolescents. Eosinophilic oesophagitis is inflammation of the oesophagus (the passage that leads from the mouth to the stomach), which causes symptoms such as dysphagia (difficulty swallowing) and blockage of the oesophagus. It is caused by a large build-up of white blood […]

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Urological Cancer

Pembrolizumab and chemoradiation for treating muscle-invasive, non-metastatic bladder cancer

Pembrolizumab in combination with chemoradiation is currently in clinical development for muscle-invasive, non-metastatic bladder cancer in adults. Bladder cancer occurs when a tumour develops in the bladder lining and if the cancer grows into a deeper, muscular layer of the bladder; this is known as muscle-invasive bladder cancer. The most common symptom of the condition […]

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Gynaecology and Obstetrics

Aflibercept for previously untreated retinopathy of prematurity in children

Retinopathy of prematurity (ROP) is a condition that can occur when a baby is born prematurely, which affects blood vessels in a part of the eye called the retina. The retina is at the back of the eye. It detects light and sends messages to the brain, which allows us to see. In severe ROP, […]

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Cardiovascular System

Semaglutide for treating overweight or obesity with cardiovascular disease

Semaglutide is in clinical development for the treatment of overweight or obesity in patients with cardiovascular disease (CVD). Obesity is a long-term (chronic) health condition that progresses over time and is commonly defined by excess body fat (adipose tissue) that may impair health. Obesity is associated with a higher risk of developing serious diseases, including […]

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Lung and Respiratory Cancer

Alectinib adjuvant therapy for ALK-positive non-small cell lung cancer

Alectinib is in clinical development for the adjuvant treatment of patients with anaplasticlymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) that have undergonecomplete resection. NSCLC makes up most lung cancer cases. In patients with ALK-positiveNSCLC, part of the ALK gene is mutated which results in the production of an abnormal form ofthe ALK protein which […]

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Cardiovascular System

Aprocitentan for treating resistant hypertension

Aprocitentan is in clinical development for the treatment of resistant (difficult to control) hypertension (high blood pressure). The causes of high blood pressure are not always clear, but the risks are increased by factors such as being overweight, eating too much salt and lack of exercise. Resistant hypertension is defined as blood pressure that remains […]

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Hepatology

Semaglutide for non-cirrhotic non-alcoholic steatohepatitis

Non-alcoholic steatohepatitis (NASH) is a more serious, progressive form of non-alcohol-related fatty liver disease (NAFLD) that involves the accumulation of fat and inflammation in the liver. The word ‘steato’ in NASH refers to fat while hepatitis refers to inflammation and damage to the liver. This inflammation can lead to scarring of liver tissue, which is […]

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Immunology

Obinutuzumab with mycophenolate mofetil for treating lupus nephritis

Obinutuzumab in combination with mycophenolate mofetil is in clinical development for thetreatment of class III or class IV lupus nephritis (LN) in adults. LN is a manifestation of a diseasecalled systemic lupus erythematosus (SLE). In LN, the immune system (the body’s naturaldefences) attacks the kidneys, causing inflammation and kidney damage. LN remains a substantialcause of […]

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