featured image

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

Dermatology

Secukinumab biosimilar for treating plaque psoriasis

Secukinumab biosimilar is in clinical development for the treatment of moderate to severe plaque psoriasis in adults. Plaque psoriasis is a long-lasting autoimmune disease that causes skin cells to reproduce very quickly. Plaque psoriasis is characterised by thick, scaly patches called plaques on the skin. These plaques most commonly effect elbows, the back, knees, and […]

Download
Endocrine Nutritional and Metabolic Disorders

Olezarsen for treating familial chylomicronaemia syndrome

Olezarsen is in clinical development for the treatment of patients with familial chylomicronaemia syndrome (FCS). FCS is a rare genetic disorder that causes very high levels of triglycerides, which is a kind of fat, in the blood. This can lead to serious health problems, including acute and chronic pancreatitis and type 3c diabetes. Symptoms of […]

Download
Hepatology

Odevixibat for treating biliary atresia in children following Kasai hepatoportoenterostomy

Odevixibat is in clinical development for the treatment of children with biliary atresia (BA) following a Kasai hepatoportoenterostomy (HPE). BA is a rare gastrointestinal disorder where either all or a portion of the bile duct on the outside of the liver is absent or destroyed. As a result, bile acids accumulate in the liver causing […]

Download
Endocrine Nutritional and Metabolic Disorders

Ferric maltol for treating iron deficiency anaemia in children and adolescents aged 1 month to 17 years

Ferric maltol is in clinical development for the treatment of children and adolescents with iron deficiency anaemia. Iron deficiency anaemia is diminished red blood cell production due to low iron stores in the body, and can develop because of malnutrition, malabsorption, or blood loss. Iron deficiency, with or without anaemia, adversely affects overall health and […]

Download
Breast Cancer

Inavolisib with palbociclib and fulvestrant for treating HR+/HER2-, PIK3CA mutated locally advanced or metastatic breast cancer

Inavolisib in combination with palbociclib and fulvestrant is in clinical development for the treatment of patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-), PIK3CA-mutated locally advanced or metastatic breast cancer. Breast cancer occurs when abnormal cells in the breast begin to grow and divide in an uncontrolled way and […]

Download
Haematological Cancer and Lymphomas

Brentuximab vedotin with chemotherapy (BrECADD) for previously untreated advanced classical Hodgkin lymphoma

Hodgkin lymphoma (HL) is an uncommon cancer that develops in the lymphatic system, which is a network of vessels and glands spread throughout the body. About 95% of all HL cases are classical HL. Lymphomas are cancers that occur if white blood cells of the immune system start to multiply uncontrollably. Symptoms of classical HL […]

Download
Haematological Cancer and Lymphomas

Elranatamab monotherapy or with daratumumab for previously treated relapsed or refractory multiple myeloma

Elranatamab, alone or in combination with daratumumab, is in clinical development for the treatment of relapsed or refractory multiple myeloma (MM). MM is a type of bone marrow cancer that is characterised by an abundance of abnormal immune cells, known as plasma cells, in the bone marrow. In the early stages, MM may not cause […]

Download
Cardiovascular System

Inhaled treprostinil for treating pulmonary hypertension with interstitial lung disease including combined pulmonary fibrosis and emphysema

Pulmonary hypertension is a rare and serious condition that occurs when blood pressure within the lungs becomes abnormally high. It can be caused by thickening of the pulmonary artery walls, heart failure, lung disease and clots within the lungs' blood vessels. Symptoms of pulmonary hypertension include shortness of breath, tiredness, feeling faint or dizzy, chest […]

Download
Urological Cancer

Durvalumab and enfortumab vedotin with or without tremelimumab for muscle-invasive bladder cancer

Durvalumab and enfortumab vedotin with or without tremelimumab is currently in clinical development for patients with muscle invasive bladder cancer (MIBC), who are ineligible or refuse cisplatin therapy (a type of chemotherapy) and are receiving a radical cystectomy (removal of bladder and surrounding lymph nodes). MIBC is a cancer that spreads into the muscle layer […]

Download
Neurology

Zavegepant for treating acute migraine

Zavegepant is in clinical development for the acute treatment of migraine. Migraines is a headache that is usually characterised by throbbing in one side of the head. In addition to this, other symptoms include sensitivity to light and sound, nausea and vomiting. The cause of migraines is not known but some triggers include tiredness, anxiety […]

Download
Endocrine Nutritional and Metabolic Disorders

Setmelanotide for treating acquired hypothalamic obesity in people 4 years and older

Setmelanotide is currently in development for the treatment of acquired hypothalamic obesity. Acquired hypothalamic obesity refers to excess weight gain that may follow from an injury to the hypothalamus, a brain region which coordinates the endocrine system (controls body functions). The hypothalamus affects energy intake, by regulating how much we eat and how much energy […]

Download
Respiratory System

Brensocatib for people aged 12 and over with non-cystic fibrosis bronchiectasis

Brensocatib is currently in clinical development for the treatment on non-cystic fibrosis bronchiectasis in patients aged 12 and older. Bronchiectasis is a lung condition where inflammation from lung infections causes irreversible damage to the lungs which can get worse over time. During bronchiectasis, the airways are wider and become inflamed with thick phlegm (sputum). This […]

Download
Haematological Cancer and Lymphomas

Blinatumomab with chemotherapy for frontline consolidation treatment of Philadelphia chromosome negative, measurable residual disease negative, B-lineage acute lymphoblastic leukaemia

Acute lymphoblastic leukaemia (ALL) is a rare and aggressive blood cancer, characterised by proliferation of immature and abnormal white blood cells responsible for the immune system (lymphocytes), in the bone marrow and blood. These immature/abnormal lymphocytes dominate bone marrow, resulting in a decrease of red blood cells, normal white blood cells and platelets. ALL can […]

Download
Nephrology

DMX-200 for treating focal segmental glomerulosclerosis in people receiving angiotensin II receptor blocker therapy

DMX-200 is currently in clinical development for treating focal segmental glomerulosclerosis (FSGS) among patients who are also receiving an angiotensin II receptor blocker (ARB). FSGS is a rare disease in which scar tissue develops on the parts of the kidneys that filter waste from the blood (glomeruli). When the glomeruli become damaged or scarred, proteins […]

Download
Musculoskeletal System

Vosoritide for treating achondroplasia in children

Vosoritide is in clinical development for the treatment of achondroplasia in children aged 0 to 59 months. Achondroplasia is a rare genetic bone growth disorder and the most common form of disproportionate short stature. Achondroplasia is caused by a change in the structure of a gene called the fibroblast growth factor receptor 3 (FGFR3) gene, […]

Download
Dermatology

Guselkumab for treating chronic plaque psoriasis in children aged 6 to 17 years

Guselkumab is in clinical development for the treatment of chronic plaque psoriasis in children aged 6 to 17 years. Psoriasis is a skin condition that causes flaky patches of skin which form scales. Chronic plaque psoriasis is the most common form of psoriasis. The ‘plaques’ are formed due to the build-up of skin cells and […]

Download
Female Reproductive Cancer

Dostarlimab with paclitaxel and carboplatin for recurrent or primary advanced endometrial cancer

Dostarlimab in combination with paclitaxel and carboplatin is in clinical development for the treatment of primary advanced or recurrent endometrial cancer. Endometrial cancer is cancer of the lining of the womb. Advanced cancer is when the cancer has spread to other parts of the body; recurrent cancer means the cancer has come back after treatment. […]

Download
Dermatology

Delgocitinib for treating moderate to severe chronic hand eczema after 1 previous therapy

Delgocitinib is currently in clinical development for the treatment of adults with moderate to severe chronic hand eczema. Hand eczema is one of the most common types of eczema. It mainly affects the palms but can also affect other parts of the hand. Chronic hand eczema is hand eczema that lasts for more than three […]

Download
Haematological Cancer and Lymphomas

Subcutaneous isatuximab with pomalidomide and dexamethasone for treating relapsed and/or refractory multiple myeloma

Subcutaneous isatuximab in addition to pomalidomide and dexamethasone is in clinical development for the treatment of patients with relapsed and/or refractory multiple myeloma (RRMM). Multiple myeloma (MM) is a rare, incurable cancer of the plasma cells in the bone marrow. In MM, large amounts of abnormal plasma cells are produced which fill the bone marrow […]

Download
Lung and Respiratory Cancer

Lurbinectedin with atezolizumab maintenance therapy for extensive-stage small-cell lung cancer

Lurbinectedin in combination with atezolizumab is in clinical development for patients with extensive stage small-cell lung cancer (ES-SCLC) after first-line induction therapy. Small-cell lung cancer (SCLC) is a type of lung cancer that usually develops in the central part of the lungs, and in which the cancer cells are small compared with other types of […]

Download
Haematological Cancer and Lymphomas

Pirtobrutinib for untreated chronic lymphocytic leukaemia or small lymphocytic lymphoma

Pirtobrutinib is currently in clinical development for untreated chronic lymphocytic leukaemia (CLL) or small lymphocytic lymphoma (SLL). CLL is a rare type of cancer that affects the blood and bone marrow (the spongy inner part of the bone that produces red and white blood cells). CLL and SLL are the same disease, but whereas in […]

Download
Lung and Respiratory Cancer

Cobolimab with dostarlimab and docetaxel for advanced non-small-cell lung cancer after anti-PD-L1 therapy and chemotherapy

Cobolimab in combination with dostarlimab and docetaxel is in clinical development for the treatment of advanced non-small cell lung cancer (NSCLC) in patients who have progressed on prior treatment with anti-PD-L1 therapy and chemotherapy. NSCLC is the most common type of lung cancer. Symptoms include a cough, repeated chest infections, breathlessness, unexplained pain, weight loss […]

Download
Haematology

Efgartigimod PH20 SC for adults with primary immune thrombocytopenia

Efgartigmod PH20 SC is currently in phase 3 development for the treatment of adults with primary immune thrombocytopenia (ITP). ITP is a rare disorder that makes the blood clot poorly due to low levels of platelets, the cells that plug wounds. ITP can cause bleeding under the skin, in the mouth, nose or other organs. […]

Download
Genetic Disorders

Mexiletine hydrochloride for treating myotonia in children and adolescents with myotonic disorders

Mexiletine is currently in clinical development for the treatment of children and adolescents with myotonic disorders. Myotonic disorders comprise both myotonic dystrophy (DM1 and DM2) and non-dystrophic myotonia (NDM). A common symptom of both conditions is myotonia, during which muscles relax slowly and with difficulty after a voluntary contraction. Muscles affected can include those in […]

Download
Lung and Respiratory Cancer

Pembrolizumab with or without olaparib following pembrolizumab with chemoradiation therapy for previously untreated, advanced, stage III non-small-cell lung cancer

Pembrolizumab with or without olaparib following pembrolizumab with chemoradiation therapy is currently in development for treatment of adults with unresectable, locally advanced, stage III non-small-cell lung cancer (NSCLC). NSCLC is the most common form of lung cancer. Advanced cancer often refers to cancer that cannot be cured. Stage III NSCLC is considered “locally advanced,” meaning […]

Download
Neurology

Efgartigimod PH20 SC for treating chronic inflammatory demyelinating polyradiculoneuropathy

Efgartigmod PH20 SC are currently in clinical development for the treatment of adults with chronic inflammatory demyelinating polyradiculoneuropathy (CIDP). CIDP is a neurological disorder that leads to progressive weakness and reduced senses in the arms and legs. It is caused by damage to the fat protecting the nerves. Symptoms of CIDP include tingling or no […]

Download
Haematology

Mim8 for prophylaxis of haemophilia A

Mim8 (NNC0365-3769) is in clinical development for haemophilia A with or without inhibitors.Haemophilia is a rare condition that affects the blood's ability to clot. It is usually inherited. Aninhibitor is a type of antibody that prevents factor replacement treatment from working.Normally, when you cut yourself, substances in your blood known as clotting factors mix withblood […]

Download
Neurological Cancer

Vorasidenib for treating residual or recurrent grade 2 glioma

Vorasidenib is in clinical development for the treatment of residual or recurrent grade 2 glioma. Glioma is a type of brain tumour that begins in glial cells (the cells that surround and support nerve cells). There are three main types of gliomas: astrocytoma, oligodendroglioma, and glioblastoma. These are categorised by the isocitrate dehydrogenase (IDH) gene […]

Download
Lung and Respiratory Cancer

Pembrolizumab with olaparib and chemoradiation for previously untreated limited-stage small-cell lung cancer

Small cell lung cancer (SCLC) is an aggressive (fast-growing) cancer that forms in tissues of thelung and can spread to other parts of the body. Limited stage SCLC (LS-SCLC) means the cancercells can be seen in one lung and in nearby lymph nodes. SCLC can affect anyone, but it typicallyaffects people who have a long […]

Download
Dermatology

Deucravacitinib for treating active psoriatic arthritis

Deucravacitinib is in clinical development for the treatment of active psoriatic arthritis (PsA). PsA is a long-term disabling condition causing stiff, inflamed, or painful joints (especially the small joints of hands and feet). The severity of symptoms tends to fluctuate. Characteristic symptoms include one or more swollen toes or fingers, perhaps with damaged nails, and […]

Download
Medicines
Medicine Industry's Gateway to NICE
Support for Industry
Outputs & Publications