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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

Female Reproductive Cancer

Atezolizumab with paclitaxel and carboplatin for previously untreated advanced endometrial cancer

Atezolizumab in addition to paclitaxel and carboplatin is in clinical development for recurrent or primary advanced endometrial cancer (EC). EC (uterine cancer) is cancer of the lining of the womb. Recurrent cancer is cancer that has come back after treatment. Advanced cancer is cancer that cannot normally be cured or controlled. EC is characterised by […]

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Urological Cancer

Enfortumab vedotin with pembrolizumab for treating bladder cancer

Enfortumab vedotin in combination with pembrolizumab is in clinical development as aneoadjuvant treatment option for cisplatin eligible patients with non-metastatic muscle invasivebladder cancer (MIBC). MIBC is a cancer that spreads into the muscle layer of the bladder wall.The survival rates for patients with MIBC are low because this type of cancer is more likely tospread […]

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Ophthalmology

Botaretigene sparoparvovec for treating X-linked retinitis pigmentosa

Botaretigene sparoparvovec is in development for the treatment of X-linked retinitis pigmentosa. Retinitis Pigmentosa (RP) is an inherited eye condition that affects the cells that line the back of the eye in the region known as the retina. RP is one of the most complicated genetic conditions, and over 80 causative genes have been identified. […]

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Immunology

Iptacopan for treating complement 3 glomerulopathy

Iptacopan is in development for the treatment of patients with complement 3 glomerulopathy (C3G) which is a rare disease. C3 is a blood protein that plays a key role in the normal immunity. The damaging build-up of the C3 protein in kidneys of patients with C3 glomerulopathy mostly results from dysregulation of the alternative complement […]

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Cardiovascular System

Upadacitinib for treating giant cell arteritis

Upadacitinib is currently in clinical development for the treatment of giant cell arteritis (GCA) in adults aged 50 years and older. GCA is a disease of blood vessels where the arteries, particularly those in the neck and at the side of the head (the temples), become inflamed. Common symptoms include headaches with severe pain and […]

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Urological Cancer

Neoadjuvant nivolumab with gemcitabine and cisplatin followed by adjuvant nivolumab for previously-untreated muscle-invasive bladder cancer

Neoadjuvant (before surgery) nivolumab in addition to gemcitabine and cisplatin followed by adjuvant (additional) nivolumab is in clinical development for muscle-invasive bladder cancer (MIBC). MIBC is when the cancer has spread beyond the lining of the bladder and into the muscle layer. The most well-known risk factor for MIBC is smoking, and the most common […]

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Haematology

Giroctocogene fitelparvovec for haemophilia A

Giroctocogene fitelparvovec is in development for the treatment of moderately severe to severehaemophilia A in adult males. Haemophilia A is a rare bleeding disorder, caused by lack of bloodclotting factor VIII which facilitates blood clotting and reduces bleeding events. The absence offactor VIII makes patients prone to excessive and uncontrolled bleeding. In severe cases, this […]

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Rheumatology

Sarilumab for treating Polyarticular-course Juvenile idiopathic arthritis in children and adolescents

Sarilumab is currently in clinical development for the treatment of children and adolescents aged 2-17 with polyarticular Juvenile Idiopathic Arthritis (JIA) or oligoarticular extended JIA subtype. Arthritis is inflammation of the joints, and juvenile idiopathic arthritis means the arthritis of unknown origin present in children and adolescents. Symptoms of polyarticular JIA include joint pain, swelling […]

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Cardiovascular System

Sirolimus-eluting collagen implant for preventing vascular access failure in end stage renal disease patients undergoing haemodialysis

Sirolimus-eluting collagen implant is in clinical development for prevention of arteriovenous (AV) vascular access dysfunction in patients with end stage renal disease (ESRD) who are undergoing haemodialysis. ESRD, also known as stage 5 chronic kidney disease (CKD) require either a kidney transplant or dialysis to remove excess fluid and waste products from the blood when […]

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Neurological Cancer

Eflornithine with lomustine for treating recurrent anaplastic astrocytoma

An anaplastic astrocytoma is a rare cancerous brain tumour that grows from astrocyte brain cells. Anaplastic means the cells divide rapidly and don’t look like normal cells in structure or function. They affect adults and children and are often treated by neurosurgery followed by radiotherapy and sometimes chemotherapy. They’re more common in adults between the […]

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Endocrine Nutritional and Metabolic Disorders

Venglustat for treating type 3 Gaucher disease after 1 therapy

Venglustat is in clinical development for the treatment of patients with Gaucher disease type 3 (GD3) who have been effectively treated with Enzyme Replacement Therapy (ERT) for at least 3 years. Gaucher Disease (GD) is a genetic disorder caused by an enzyme deficiency where fatty substances build up in areas such as the spleen, liver, […]

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Genetic Disorders

PXT3003 for treating Charcot-Marie-Tooth disease type 1A

PXT3003 is currently in clinical development for treatment of patients aged 16-65 years with Charcot-Marie-Tooth disease type 1A (CMT1A). CMT is a group of inherited conditions that damage the peripheral nerves (reside outside the brain and spinal cord). CMT1 is characterised by muscle weakness and atrophy, which can lead to repeated ankle sprains, and changes […]

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Haematological Cancer and Lymphomas

Pirtobrutinib for chronic lymphocytic leukaemia or small lymphocytic lymphoma after BTK inhibitor therapy

Pirtobrutinib is currently in clinical development for the treatment of patients with chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL) who have previously received treatment with the current standard of care. CLL and SLL are types of cancer in which too many white blood cells (B-cells) are produced, these cells build up in the […]

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Neurology

Galcanezumab for preventing migraine in children

Galcanezumab is in clinical development for the preventative treatment of paediatric patients with episodic or chronic migraine. Migraine is one of the most common neurological conditions in the paediatric population with unique clinical characteristics that can evolve with age. These characteristics can include shorter attacks, different location of pain (bilateral in younger children), and light […]

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Neurology

Pegcetacoplan for treating amyotrophic lateral sclerosis

Pegcetacoplan is in 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 life-threatening disease. Currently, there […]

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Haematology

Ravulizumab for treating thrombotic microangiopathies following haematopoietic stem cell transplantation in children

Ravulizumab is in development for the treatment of thrombotic microangiopathies following haematopoietic stem cell transplantation (HSCT-TMA) in children, adolescents, and adults. Thrombotic microangiopathies (TMA) are clinical syndromes defined by the destruction of red blood cells, low platelets, and organ damage due to the formation of microscopic blood clots in capillaries and small arteries. Haematopoietic stem […]

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Head and Neck Cancer

Tiragolumab with atezolizumab for oesophageal squamous cell cancer

Tiragolumab in combination with atezolizumab is in clinical development to treat patients withunresectable oesophageal squamous cell carcinoma (ESCC) following chemoradiotherapy. ESCCis a type of cancer that begins in the thin, flat cells lining the food pipe (oesophagus) and mayspread to other parts of the body. Unresectable refers to cancer which cannot be removedcompletely by surgery. […]

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Endocrine Nutritional and Metabolic Disorders

Fosdenopterin for molybdenum cofactor
deficiency type A

Fosdenopterin is currently in clinical development for the treatment of paediatric patients aged 1 day to 5 years with molybdenum cofactor deficiency (MoCD) type A. Patients with MoCD type A cannot produce a substance known as cyclic pyranopterin monophosphate (cPMP). MoCD type A is a rare genetic disease that can appear shortly after birth and […]

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Urological Cancer

Pembrolizumab with Bacillus Calmette-Guerin for treating high-risk non-muscle invasive bladder cancer

Pembrolizumab in combination with Bacillus Calmette-Guerin (BCG) is in development for the treatment of high-risk non-muscle invasive bladder cancer (NMIBC) that is recurrent or persistent following BCG induction or naïve to BCG treatment. Bladder cancer is when cancerous cells develop in the lining of the bladder. NMIBC is the most common type of bladder cancer […]

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Infectious Disease

Cabotegravir (long-acting injection) for prevention of HIV-1 infection

Cabotegravir is in clinical development as an alternative method of protecting an individual from contracting the human immunodeficiency virus type 1 (HIV-1). HIV is a chronic life altering condition that requires, daily antiretroviral therapy (ART) as a regular treatment routine. If left unmanaged it can lead to serious complications, such as acquired immunodeficiency syndrome (AIDS). […]

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Dermatology

Linerixibat for treating pruritus associated with primary biliary cholangitis

Linerixibat is in clinical development for the treatment of primary biliary cholangitis (PBC). PBC is a chronic autoimmune disease where bile (digestive fluid) flow from the liver is disrupted (i.e., cholestasis), resulting in excess bile acids in circulation. Cholestatic pruritus is an itch which is internal and cannot be relieved by scratching. The cause of […]

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Ear Nose and Throat (ENT)

WP1048 for treating grass pollen allergy

WP1048 is in clinical development for the treatment of adults with grass pollen allergy. Grass pollen allergy, also known as hay fever or allergic rhinitis, is caused by an immune reaction to proteins found in pollen from various types of grass. The allergy presents symptoms such as itchy eyes, blocked and inflamed nose and persistent […]

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Genetic Disorders

Alpelisib for treating PIK3CA-related overgrowth spectrum

Alpelisib is in development for the treatment of the rare disease PIK3CA-related overgrowth spectrum (PROS). PROS is a group of genetic conditions where parts of the body become abnormally overgrown and can affect anywhere from the limbs to the brain, resulting in a wide range of symptoms which can include: pain, fatigue, or seizures depending […]

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Breast Cancer

Palbociclib with trastuzumab with or without pertuzumab and endocrine therapy for HR+/HER2+ metastatic breast cancer

Palbociclib in combination with trastuzumab with or without pertuzumab and endocrine therapy is currently in clinical development for use as a maintenance therapy for patients with hormone receptor positive (HR+) and human epidermal growth factor receptor 2 positive (HER2+) metastatic breast cancer who have had prior treatment. Breast cancer occurs when abnormal cells in the […]

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Lung and Respiratory Cancer

Telisotuzumab vedotin for previously treated c-MET overexpressing EGFR wild-type non-squamous non-small cell lung cancer

Telisotuzumab vedotin is in development for the treatment of c-Met+ non-small cell lung cancer. Non-small cell lung cancer is the most common form of lung cancer. A c-Met is a protein in humans that is encoded by the MET gene that leads to growth in cells. Smoking is the cause of most lung cancers and […]

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Cardiovascular System

Invimestrocel for Acute ischaemic stroke

Invimestrocel is in clinical development for the treatment of acute ischaemic stroke within 18-36hours of onset. Ischaemic stroke is the most common type of stroke. It happens when a bloodvessel is blocked by a blood clot, cutting off blood flow to part of the brain (ischaemia). Withoutblood supply, brain cells can be damaged or destroyed […]

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Skin Cancer

Dabrafenib with Trametinib with adjuvant therapy for treating advanced BRAF V600 mutation-positive melanoma in adolescents

Dabrafenib in combination with trametinib is currently in clinical development for the treatment of advanced BRAF V600 mutation-positive melanoma in adolescents. Melanoma is a type of skin cancer. Common signs include the appearance of a new mole or a change in an existing mole. This can happen anywhere on the body, but the most commonly […]

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Female Reproductive Cancer

Durvalumab with bevacizumab and chemotherapy for treating advanced ovarian cancers

Durvalumab in combination with bevacizumab and chemotherapy is currently in clinical development for the treatment of patients with newly diagnosed, advanced ovarian cancer, primary peritoneal cancer, and/or fallopian tube cancer. Primary peritoneal cancer, fallopian tube cancer and epithelial ovarian cancer arise from the same type of tissue and thus exhibit many similar symptoms. These include […]

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Lung and Respiratory Cancer

Amivantamab with lazertinib for previously untreated locally advanced or metastatic non-small-cell lung cancer

Amivantamab with lazertinib is in clinical development for the treatment of patients with EGFR-mutated locally advanced or metastatic non-small cell lung cancer (NSCLC). NSCLC makes up the majority of lung cancers in the UK. In locally advanced NSCLC, the cancer has spread into tissues around the lungs, and metastatic NSCLC is when the cancer starts […]

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Lung and Respiratory Cancer

Tiragolumab with atezolizumab for treating stage III non-small cell lung cancer after platinum-based chemoradiotherapy

Tiragolumab with atezolizumab is currently in development for locally advanced, unresectable stage III non-small cell lung cancer (NSCLC) in patients who have received at least two cycles of chemoradiotherapy. Stage III NSCLC describes a locally advanced disease state where the cancer has spread beyond the lung that was initially affected, such as surrounding lymph nodes, […]

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