Medicine Industry's Gateway to NICE
The Innovation Observatory is the gateway to the NICE Technology Assessment (TA) Programme for industry, preventing delays in bringing new and repurposed medicines to the UK market through the early identification and tracking of new and repurposed medicines and health technologies.
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.
Sulthiame for treating self-limiting epilepsy with centrotemporal spikes in children
Sulthiame is in development for the treatment of self-limiting epilepsy with centrotemporal spikes in children. A self-limiting epilepsy with centrotemporal spikes is defined as an age-dependent epilepsy, with a typical onset at 5–8 years that usually subsides by the teenage years. The main symptom of the condition is seizures lasting between 1 – 3 mins. […]
DownloadRadium-223 dichloride with enzalutamide for treating hormone-relapsed metastatic prostate cancer
Radium-223 dichloride in combination with enzalutamide are in clinical development for the treatment of hormone-relapsed metastatic prostate cancer. Hormone-relapsed metastatic prostate cancer occurs when prostate cancer, which has spread to other parts of the body, stops responding to hormonal treatment. Initially, treatments can control the cancer for months or years, but eventually, the cancer grows […]
DownloadDostarlimab and docetaxel for treating advanced non-small-cell lung cancer after progression on anti-PD-L1 therapy and chemotherapy
Dostarlimab in combination with docetaxel is in clinical development for the treatment of advanced non-small cell lung cancer (NSCLC) in patients who have progressed on prior anti-PD(L)-1 therapy and chemotherapy. NSCLC is the most common type of lung cancer. Advanced cancer means it has spread around the body from where it started making it harder […]
DownloadEntrectinib for treating relapsed or refractory locally advanced or metastatic solid tumours with NTRK1/2/3 gene fusions in children and adults
Entrectinib is in clinical development for the treatment of children and adolescents with relapsed (recurrent) or refractory (treatment resistant) locally advanced or metastatic solid or primary tumours harbouring neurotropic tropomyosin receptor kinase (NTRK) 1/2/3. Solid tumours are defined as abnormal masses of tissue that usually do not contain cysts or liquid areas and may be […]
DownloadDurvalumab and tremelimumab with or without lenvatinib in combination with concurrent transarterial chemoembolisation in patients with locoregional hepatocellular carcinoma
Durvalumab and tremelimumab with or without lenvatinib in combination with concurrent transarterial chemoembolisation are in clinical development for the treatment of adults with hepatocellular carcinoma (HCC). HCC is a primary cancer arising from hepatocytes in predominantly cirrhotic liver. However, some patients may not have cirrhosis before developing HCC, especially those with chronic hepatitis B virus. […]
DownloadAtezolizumab for adjuvant treatment of muscle-invasive bladder cancer
Atezolizumab is currently in clinical development for the adjuvant treatment of circulating tumour DNA test (ctDNA) positive muscle-invasive bladder cancer (MIBC) following cystectomy. Bladder cancer is a disease in which certain cells in the bladder become abnormal and multiply uncontrollably to form a tumour. MIBC is when the cancer has spread beyond the lining of […]
DownloadTrastuzumab deruxtecan adjuvant treatment of high-risk HER2-positive, residual invasive breast cancer after neoadjuvant therapy
Breast cancer occurs when abnormal cells in the breast grow and divide uncontrollably, forming a tumour. Cancers that have high levels of a protein called human epidermal growth factor receptor 2 (HER2) on the cell surface are called HER2-positive cancers, making them more aggressive and prone to rapid spread and growth compared to HER2-negative cancers. […]
DownloadInebilizumab for immunoglobulin G4-related disease
Inebilizumab is in development for the treatment of immunoglobulin G4-related disease (IgG4-RD). Immunoglobulin G4 is a type of antibody, which are proteins essential to the immune system as they can bind to unwanted substances within the body and eliminate them. In IgG4-RD these antibodies do not function correctly, which leads to inflammation of organs. Prolonged […]
DownloadMavacamten for treating non-obstructive hypertrophic cardiomyopathy
Mavacamten is in clinical development for treatment of non-obstructive hypertrophic cardiomyopathy (HCM). HCM is often an inherited disease, where the heart muscle cells enlarge, and the walls of the heart thicken. If there is no significant blocking of blood flow, the condition is called nonobstructive HCM (nHCM). This is caused by excessive binding of myosin […]
DownloadAvatrombopag for treating immune thrombocytopenia in children
Avatrombopag is in clinical development for the treatment of immune thrombocytopenia (ITP) in children with a duration of ≥6 months that have had a poor response to a previous treatment. ITP is an autoimmune disease (where the immune system attacks the body instead of foreign invaders like bacteria and viruses), where the immune system mistakenly […]
DownloadInebilizumab for treating myasthenia gravis
Inebilizumab is currently in clinical development for the treatment of adults with myasthenia gravis (MG). MG is a rare, long-term autoimmune disorder that leads to muscle weakness and fatigue, 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 life. […]
DownloadTrastuzumab deruxtecan for treating locally advanced, unresectable, or metastatic HER2+ solid tumours
Trastuzumab deruxtecan is in clinical development for treatment of unresectable (unable to be removed with surgery), locally advanced or metastatic (i.e., the cancer has spread to other tissues or organs) HER2+ solid tumours which have not responded to a prior treatment. HER2+ means that, due to a genetic mutation, the cancer cells produce a protein […]
DownloadSibeprenlimab for treating Immunoglobulin A Nephropathy
Sibeprenlimab is in clinical development for the treatment of adults with Immunoglobulin A Nephropathy (IgAN). IgAN is a chronic kidney disease with a mean age of diagnosis of 42 years in adult patients. In IgAN a protein called immunoglobulin A (IgA) becomes trapped in the very fine filters of the kidney (glomeruli), causing damage and […]
DownloadMezigdomide with dexamethasone and carfilzomib for treating relapsed or refractory multiple myeloma
Mezigdomide is in clinical development for the treatment of relapsed or refractory multiple myeloma (RRMM) in combination with carfilzomib and dexamethasone after 1 or more prior treatments. Multiple myeloma is a type of blood cancer that is characterised by an abundance of abnormal immune cells, known as plasma cells, in the bone marrow. Symptoms include […]
DownloadDorocubicel for treating haematological malignancies in adults requiring allogeneic HSCT
Haematological cancers also known as blood cancers, occur when abnormal cells in the blood grow out of control. They are usually categorised by site according to whether cancer is first detected in the blood (leukaemias), lymph nodes (lymphomas), or bone (myelomas). The abnormal cells can crowd out normal blood cells, which have important functions in […]
DownloadAstegolimab for treating chronic obstructive pulmonary disease in people aged 40 to 80 years
Astegolimab is currently in clinical development for the treatment of chronic obstructive pulmonary disease (COPD) in patients aged 40 to 80 years who are former or current smokers and have a history of frequent exacerbations. COPD is the name for a group of lung conditions that cause breathing difficulties. It is a disease that causes […]
DownloadCabozantinib for previously treated locally advanced, unresectable or metastatic neuroendocrine tumours
Cabozantinib is currently in clinical development for treating locally advanced, unresectable or metastatic neuroendocrine tumours. A neuroendocrine tumour (NET) is a rare cancerous tumour that can develop in many different organs of the body. It affects the cells that release hormones into the bloodstream. There are several different types of NETs and the type a […]
DownloadObicetrapib or obicetrapib-ezetimibe for primary hypercholesterolaemia or mixed dyslipidaemia
Obicetrapib or obicetrapib-ezetimibe is in clinical development for treating primary hypercholesterolaemia (PH) or mixed dyslipidaemia in adults on maximally tolerated lipid-lowering therapy, that is patients that fail to reach treatment goals inspite of being on highest tolerated dose of lipid-lowering therapy. High levels of cholesterol in the blood (hypercholesterolaemia) may be caused by genetic defects […]
DownloadOrforglipron as an adjunct treatment for obesity or overweight with co-morbidities
Orforglipron is in clinical development for the treatment of adults living with obesity or overweight with weight-related comorbidities. Overweight and obesity are typically defined by a body mass index (BMI) over 25 and 30, respectively. This is commonly caused by excessive calorie intake, particularly from high-fat and high-sugar foods, along with genetic and environmental factors. […]
DownloadDurvalumab with chemoradiation for untreated locally advanced unresectable oesophageal squamous cell carcinoma
Durvalumab in combination with definitive chemoradiation is in clinical development for the treatment of locally advanced, unresectable oesophageal squamous cell carcinoma. This is a type of cancer that begins in the thin, flat cells lining the food pipe (oesophagus), that has spread into surrounding tissues in the oesophagus, and is untreatable by surgery. Symptoms include […]
DownloadTrastuzumab deruxtecan for treating previously untreated locally advanced or metastatic non-small cell lung cancer with HER2 mutations
Trastuzumab deruxtecan is in clinical development for treatment of previously untreated advanced or metastatic non-small cell lung cancer (NSCLC), with HER2 genetic mutations. Lung cancer is the third most common cancer in the UK and NSCLC accounts for approximately 85% of all lung cancer cases, with around 1-4% of these being caused by mutations in […]
DownloadBevacizumab gamma for treating neovascular age-related macular degeneration
Bevacizumab gamma is in clinical development as an intravitreal injection (given through the white part of your eye known as vitreous) for the treatment of wet/neovascular age-related macular degeneration (nAMD). nAMD is a retinal condition that can cause deterioration of vision. nAMD can affect any part of your retina, a thin layer of tissue on […]
DownloadDiltiazem hydrochloride cream for treating anal fissures
Diltiazem hydrochloride cream is in clinical development for the treatment of chronic anal fissure. An anal fissure is a small tear or cut in the lining of the skin around the back passage (anus). The most common symptoms of anal fissures include pain around the anus and bleeding when passing stools. Anal fissures often arise […]
DownloadNifedipine–lidocaine for treating chronic anal fissure
Nifedipine-lidocaine has been clinically developed for the treatment of chronic anal fissure. An anal fissure is a tear or open sore (ulcer) that develops in the lining of the large intestine, near the anus. Many fissures heal by themselves, or certain medicines can be prescribed to treat them. However, sometimes a fissure does not heal […]
DownloadPertuzumab biosimilar with trastuzumab and chemotherapy neoadjuvant and adjuvant therapy for early stage and locally advanced HER2-positive breast cancer
Pertuzumab biosimilar is in clinical development for the adjuvant and neoadjuvant treatment of invasive early stage or locally advanced breast cancer (BC) that is human epidermal growth factor 2 (HER2) positive (+). HER2 is a protein found on some cancer cells. Neoadjuvant therapy comes before primary treatment. Adjuvant therapy follows the primary treatment. BC is […]
DownloadPirtobrutinib for previously treated Bruton tyrosine kinase inhibitor naïve mantle cell lymphoma
Pirtobrutinib is in clinical development for the treatment of previously treated Bruton tyrosine kinase (BTK) inhibitor-naïve mantle cell lymphoma (MCL) in adults. MCL is a rare and aggressive blood cancer that starts in white blood cells (B cells) in the outer edge of the lymph nodes. The abnormal white blood cells start to collect in […]
DownloadSelumetinib (granule formulation) for treating neurofibromas type 1-related symptomatic, inoperable plexiform in children
Selumetinib granule formulation is currently in clinical development for the treatment of symptomatic and inoperable plexiform neurofibromas (PN) associated with type 1 neurofibromatosis (NF1) in children. NF1 is a genetic condition that causes tumours to grow along the nerves. The tumours are usually non-cancerous but may cause a range of symptoms. Some children have a […]
DownloadSavolitinib with osimertinib for treating EGFR- mutated, MET-overexpressed advanced non-small-cell lung cancer
Savolitinib in combination with osimertinib is in clinical development for treatment of acquired mesenchymal epithelial transition factor (MET)-mediated osimertinib resistance in epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC). NSCLC is the most common form of lung cancers in the UK and at the metastatic stage, the disease has already spread from the […]
DownloadSoticlestat as an adjunctive therapy in patients with Dravet syndrome
Soticlestat is in clinical development as an adjunctive therapy in patients with Dravet syndrome, a rare neurological condition characterized by treatment-resistant epilepsy, intellectual disability, and a spectrum of associated conditions such as behaviour, speech, feeding and mobility difficulties. Typically, Dravet syndrome involves multiple, frequent, and often prolonged seizures that do not respond well to treatment. […]
DownloadPembrolizumab with lenvatinib and chemotherapy for the treatment of metastatic squamous cell oesophageal carcinoma
Lenvatinib in combination with pembrolizumab and chemotherapy is in clinical development as a first line therapy for metastatic squamous cell oesophageal carcinoma (SCOC). SCOC is a type of cancer that originates in the squamous cells that line the oesophagus (sometimes referred to as the food pipe or gullet). Metastatic cancer means the cancer has spread […]
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