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.
Berotralstat for preventing recurrent attacks of hereditary angioedema in children aged 2 to <12 years
Berotralstat is in development for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in children aged 2 to <12 years. HAE is a rare disease, caused by a gene alteration that means an important protein in the immune system, C1 esterase inhibitor (C1-INH), is missing or dysfunctional in these patients. Lack of the […]
DownloadDatopotamab deruxtecan with durvalumab and carboplatin for previously untreated, locally advanced, unresectable non-small cell lung cancer
Datopotamab deruxtecan in combination with durvalumab and carboplatin is in clinical development for the treatment of previously untreated, locally advanced, or metastatic non-small cell lung cancer (NSCLC), without actionable genomic alterations. NSCLC is the most common lung cancer. Locally advanced cancer has grown outside the body part it started in but has not yet spread […]
DownloadVedolizumab for treating moderately to severely active ulcerative colitis in children aged 2 to 17 years
Vedolizumab is currently in clinical development for the treatment of moderately to severely active ulcerative colitis (UC) in children and teenagers (paediatric patients). UC, a type of inflammatory bowel disease, is a long-term condition where the colon and rectum become inflamed and small ulcers can develop on the colon's lining, which may bleed and produce […]
DownloadLebrikizumab for treating atopic dermatitis in children aged 6 Months to 18 Years
Lebrikizumab is being developed for paediatric patients with atopic dermatitis (AD). AD is a common, long-term (chronic) inflammatory skin condition that results in patches of redness, itchiness, and scaling of the skin. In moderate to-severe cases of AD, the patches cover a large area of the skin and can be associated with intense itch. The […]
DownloadTirzepatide for treating type 2 diabetes in children and adolescents aged 10 to 18 years
Tirzepatide is currently in clinical development for treating type 2 diabetes (T2D) in children and adolescents aged 10 to 18 years when their conditions are not being controlled with metformin and/or insulin. T2D is a lifelong condition where the body’s cells become resistant to, or stop producing enough insulin, a hormone that helps to control […]
DownloadOrforglipron for treating type 2 diabetes
Orforglipron is currently in phase III clinical development for the treatment of 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 […]
DownloadSavolitinib with durvalumab for treating unresectable locally advanced or metastatic MET-driven papillary renal cell carcinoma
Savolitinib in combination with durvalumab is currently in clinical development for the treatment of patients with unresectable and locally advanced or metastatic mesenchymal-epithelial transition (MET)-driven papillary renal cell carcinoma (PRCC). Renal cell carcinoma is a common type of kidney cancer. Unresectable means the cancer cannot be removed with surgery. Locally advanced means the cancer has […]
DownloadInclisiran for treating heterozygous and homozygous familial hypercholesterolaemia in adolescents aged 12 to 17 years
Inclisiran is currently in development for the treatment of heterozygous and homozygous familial hypercholesterolaemia in adolescents aged 12 to 17 years. Familial hypercholesterolaemia is an inherited condition where differences in certain genes affect the liver’s ability to process cholesterol. This can lead to premature cardiovascular diseases. Heterozygous familial hypercholesterolaemia occurs when one gene for familial […]
DownloadUstekinumab for treating moderately to severely active ulcerative colitis in children aged 2 to 17 years
Ustekinumab is currently in clinical development for the treatment of moderately to severely active ulcerative colitis (UC) in paediatric patients. UC, a type of inflammatory bowel disease, is a long-term condition where the colon and rectum become inflamed and small ulcers can develop on the colon's lining, which may bleed and produce pus. Symptoms of […]
DownloadClindamycin for treating bacterial vaginosis
Clindamycin phosphate vaginal gel is in clinical development for the treatment of bacterial vaginosis, which is a common cause of unusual vaginal discharge caused by a change in the natural balance of bacteria in the vagina. Around half of patients with bacterial vaginosis do not show any symptoms; when symptoms are present, the condition is […]
DownloadGanaxolone for treating tuberous sclerosis complex-related epilepsy after 2 or more treatments
Ganaxolone is in clinical development for the treatment of tuberous sclerosis complex (TSC) -related epilepsy. TSC is a rare genetic condition that causes non-cancerous tumours to grow in various organs, including the brain, skin, kidneys, heart, eyes and lungs. These tumours result from changes in the TSC1 or TSC2 genes and can lead to epilepsy, […]
DownloadUX111 for treating mucopolysaccharidosis type IIIA
UX111 is in clinical development for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA). MPS, also known as Sanfilippo syndrome, is a rare genetic condition that causes fatal brain damage and is a type of childhood dementia. MPS III is caused by the lack of an enzyme that normally breaks down and recycles a large, […]
DownloadNavepegritide for treating achondroplasia in children and adolescents aged 2 to 15 years old
Navepegritide is in clinical development for the treatment of achondroplasia. Achondroplasia is a genetic condition that causes impaired bone growth, particularly in the limbs and the spine. It is caused by a change in the fibroblast growth factor receptor 3 (FGFR3) gene which is a regulator of bone growth at cartilage growth plates (the areas […]
DownloadApraglutide for treating short bowel syndrome
Apraglutide is currently in clinical development for the treatment of short bowel syndrome with intestinal failure (SBS-IF) in patients who rely on intravenous parenteral nutrition for essential fluids and nutrients. Short bowel syndrome (SBS) is a condition where part of the small intestine is missing or does not work properly making it hard for the […]
DownloadClascoterone for acne vulgaris
Acne vulgaris is a common skin condition where the skin pores become clogged with oil and dead skin cells. Pore blockages produce blackheads, whiteheads, and other types of pimples and possible scarring. Pimples are pus-filled, sometimes painful, bumps on the skin. It primarily affects the face, upper part of the chest, and back. The causes […]
DownloadSulthiame 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 […]
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