Advanced Therapy Medicinal Products (ATMPs) are novel therapies based on cells, genes or tissues with the potential to address the underlying cause of the diseases. ATMPs come with potentially significant implementation challenges, high costs and regulatory & assessment uncertainties.
In response to the COVID-19 pandemic the Innovation Observatory has collated a comprehensive global dataset of SARS-CoV-2 diagnostic tests, supporting stakeholder in assessing priorities and mitigation options in response to testing challenges. Using manual and automated horizon scanning approaches, our diagnostic landscape has provided key insights into the evolution of technological innovations, their regulation and […]
This workshop was delivered to HTAi members with varying degrees of knowledge and experience in horizon-scanning (both beginners and experienced). It shares the experiences, insights and case studies from IO’s horizon-scanning activities to help participants gain an understanding of horizon scanning in the context of HTA, provide an overview of key elements for consideration in a […]
Emicizumab is currently in development for the treatment of congenital mild or moderate haemophilia A, without factor VIII inhibitors. Haemophilia A is a genetic bleeding disorder caused by insufficient levels of a blood clotting factor, called factor VIII (eight). This causes poor blood clotting, which results in difficulty in stopping the flow of blood from […]
Dasiglucagon is in clinical development for treating hypoglycaemia due to congenital hyperinsulinism (CHI) in children. CHI is an inherited disorder in which the body releases insulin even when it is not needed. Insulin is a hormone that helps control blood glucose (sugar) levels by driving glucose into the cells of the body. In hyperinsulinism, the […]
Dupilumab is in clinical development for the treatment of chronic spontaneous urticaria for people who still have symptoms despite previous treatment. Urticaria is a skin disorder where red, raised, itchy rashes appear on the skin. Urticaria is considered chronic once symptoms have been present for six weeks or longer and appear almost daily. Spontaneous urticaria […]
Pembrolizumab in combination with gemcitabine plus cisplatin is being developed for patients with advanced/unresectable biliary tract cancer (BTC). BTC is a rare type of cancer that affects bile ducts and the gallbladder. Bile ducts are tubes that connect the gallbladder and liver to the small bowel. Bile is produced in the liver and stored in […]
Nivolumab in addition to docetaxel is in clinical development for the treatment of adult males with advanced, castration-resistant prostate cancer. Prostate cancer is a common cancer caused by the growth of a tumour on the prostate gland. Advanced prostate cancer is a form of prostate cancer where the tumours have spread to other areas of […]
Dupilumab is in clinical development for the treatment of prurigo nodularis (PN) for people who have not responded to or cannot have corticosteroid treatment. PN is a chronic skin disorder in which people have firm, raised bumps on the skin known as nodules. The nodules are very itchy and itching the affected area can worsen […]
Pembrolizumab in combination with pemetrexed and cisplatin is in clinical development for thetreatment of adult patients with advanced malignant pleural mesothelioma (MPM). MPM is a rarevariation of cancer that affects the internal chest wall and the outer linings of the lungs.Mesothelioma is often diagnosed at an advanced stage and surgery is not always possible.Treatment is […]
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Interested groups * Achondroplasia Activated PI3K delta syndrome Acute hepatic porphyria (AHP) Acute ischaemic stroke Acute lymphoblastic leukaemia (ALL) Acute myeloid leukaemia (AML) Adrenoleukodystrophy (ALD) Adrenomyeloneuropathy (AMN) Advanced dedifferentiated liposarcoma Age-related macular degeneration (AMD) Alagille syndrome Alcohol use disorder Alkaptonuria Alopecia areata Alport Syndrome Alzheimer's disease Amyloid light chain amyloidosis Amyloidosis Amyotrophic lateral sclerosis Anaemia Anaemia due to myelodysplastic syndromes (MDS) Anaemic myelofibrosis Anaesthesia Anal cancer Anaplastic astrocytoma Anaplastic large cell lymphoma (ALCL) Aneurysmal subarachnoid haemorrhage Angioimmunoblastic T-cell Lymphoma (AITL) Ankylosing spondylitis Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) Aromatic L-amino acid decarboxylase (AADC) deficiency Arterial venous access Articular cartilage defects Asthma Atopic dermatitis Atypical haemolytic uraemic syndrome (aHUS) Axial spondyloarthritis B-cell non-Hodgkin lymphoma Back pain Benign prostatic hyperplasia (BPH) Beta thalassemia Biliary tract cancer Bipolar disorder Bladder cancer Bladder pain syndrome Blastic plasmacytoid dendritic cell neoplasm (BPDCN) Bleeding Bone and Cartilage Cancer Bone sarcoma Breast cancer Calciphylaxis Canagliflozin (Invokana;JNJ-28431754) Cancer pain Cardiovascular disease (CVD) Cardiovascular events Cardiovascular System Cervical cancer Charcot-Marie-Tooth disease Chemoradiation-induced oral mucositis Chemotherapy-induced neutropenia Chemotherapy-induced thrombocytopenia Cholangiocarcinoma Choroideremia Chronic heart failure Chronic hepatitis D Chronic inducible cold urticaria Chronic kidney disease (CKD) Chronic lymphocytic leukaemia (CLL) Chronic myeloid leukaemia (CML) Chronic myelomonocytic leukaemia (CMML) Chronic obstructive pulmonary disease (COPD) Chronic spontaneous urticaria (CSU) Chronic thromboembolic pulmonary hypertension (CTEPH) Clostridioides difficile infection Cognitive impairment Cold agglutinin disease (CAD) Colon cancer Colorectal cancer Common cold Community-acquired pneumonia Complement 3 glomerulopathy Complex regional pain syndrome (CRPS) congenital ADAMTS-13 deficiency Congenital heart disease Congenital hyperinsulinism COVID-19 Critical limb ischaemia (CLI) Crohn's disease Cutaneous T-cell lymphoma (CTCL) Cystic fibrosis Cystinuria Cytomegalovirus (CMV) Delayed graft function (DGF) Dental and Oral Health Depression Dermatology Diabetic foot ulcer (DFU) Diabetic macular oedema Diabetic nephropathy Diabetic retinopathy Differentiated thyroid cancer (DTC) Diffuse large B-cell lymphoma (DLBCL) Distal renal tubular acidosis Duchenne muscular dystrophy (DMD) Dupuytren's disease Dystrophic epidermolysis bullosa (DEB) Ear Endometrial cancer Endometriosis-associated pain Enthesitis-related arthritis Eosinophilic granulomatosis Eosinophilic granulomatosis with polyangiitis Eosinophilic oesophagitis Epilepsy Epithelial ovarian cancer Epithelial ovarian cancers Erectile dysfunction Erythropoietic protoporphyria Exocrine pancreatic insufficiency (EPI) Fabry disease Facial angiofibroma Fallopian tube cancer Familial adenomatous polyposis (FAP) Familial primary hypoalphalipoproteinemia Fatal Female Reproductive Cancer Fibrodysplasia ossificans progressiva Focal segmental glomerulosclerosis (FSGS) Follicular lymphoma Friedreich's ataxia Gastric cancer Gastro-oesophageal junction adenocarcinoma Gastro-oesophageal junction cancer Gastroenteropancreatic neuroendocrine tumour Gastroesophageal adenocarcinoma Gastrointestinal Cancer Gastrointestinal stromal tumour (GIST) Gaucher disease Generalised pustular psoriasis Generalised pustular psoriasis flares Genetic Disorders Geographic atrophy Giant cell arteritis Glaucoma Glioma Glucose transporter type 1 deficiency syndrome Glycogen storage disease type 1a GM1 gangliosidosis GM2 gangliosidosis Graft versus host disease (GvHD) Granulomatosis with polyangiitis (GPA) Grass pollen allergy Gynaecology and Obstetrics Haemophilia A Haemophilia B Hand dysfunction Head and neck cancer Heart failure (HF) Heavy menstrual bleeding Helicobacter pylori Hepatocellular carcinoma (HCC) Hereditary angioedema (HAE) Heterozygous familial hypercholesterolaemia (HeFH) Hidradenitis suppurativa (HS) High grade B cell lymphoma (HGBCL) HIV infection HIV-1 infection Hodgkin lymphoma Homozygous familial hypercholesterolemia Hospital-acquired pneumonia Hot flushes Huntington's disease Hyperargininemia Hypereosinophilic syndrome (HES) Hyperlipidaemia Hyperphagia Hypersomnia Hypertrophic cardiomyopathy (HCM) Hypoglycaemia Hypoparathyroidism Hypophosphataemia Hypoplasminogenaemia Idiopathic chronic cough Idiopathic inflammatory myopathies Idiopathic thrombocytopenic purpura IgA nephropathy Immune thrombocytopenia (ITP) Immunodeficiency Influenza Inherited epidermolysis bullosa (EB) Injured gluteal musculature Insomnia Interstitial cystitis Interstitial lung disease (ILD) Invasive candidasis Iron overload Irritable bowel syndrome with predominant constipation (IBS-C) Ischaemic stroke Juvenile idiopathic arthritis Juvenile psoriatic arthritis Leber's hereditary optic neuropathy (LHON) Lennox-Gastaut syndrome Leukocyte adhesion deficiency Long chain fatty acid oxidation disorders (LC-FAOD) Lower limb spasticity Lupus nephritis Lymphoblastic lymphoma (LBL) Lymphocytic colitis Major adverse cardiovascular events (MACE) Major cardiovascular events Major depressive disorder (MDD) Malignant hyperthermia Malignant pleural mesothelioma (MPM) Mantle cell lymphoma Marginal zone lymphoma (MZL) Mature T-cell lymphoma Medullary thyroid cancer (MTC) Melanoma Merkel cell carcinoma (MCC) Metabolic acidosis Metabolic Disorders Metachromatic leukodystrophy (MLD) Methotrexate toxicity during cancer therapy Microscopic polyangiitis (MPA) Migraine Mixed dyslipidaemia Molybdenum cofactor deficiency Type A Monocarboxylate transporter 8 deficiency (Allan-Herndon-Dudley syndrome) Motor neurone disease (MND or ALS) Mucopolysaccharidosis type IIIB (Sanfilippo B syndrome) Mucopolysaccharidosis type VII Multiple myeloma (MM) Multiple sclerosis (MS) Muscle spasticity Myasthenia gravis (MG) Myelodysplastic syndrome (MDS) Myelofibrosis Myeloproliferative Neoplasms Myofibroblastic tumour Myotonia Myotonic dystrophy Myxoid round cell liposarcoma Narcolepsy Nasal polyps Nasopharyngeal cancer Nasopharyngeal carcinoma Neovascular age-related macular degeneration Neuroblastoma Neurofibromatosis Neurogenic detrusor overactivity Neuromyelitis optica Neuromyelitis optica spectrum disorders Niemann-Pick Disease Nocturia Non-alcoholic steatohepatitis (NASH) Non-gBRCA mutated high-grade serous ovarian cancer Non-Hodgkin lymphoma (NHL) Non-malignant diseases Non-metastatic prostate cancer Non-small-cell lung cancer (NSCLC) Nose and Throat Obesity Obesity due to heart-failure Obesity due to Leptin receptor deficiency Obesity due to pro-opiomelanocortin deficiency Obesity-related heart failure Ocular hypertension Ocular inflammatory pain Oesophageal cancer Oesophageal squamous cell cancer Oesophageal squamous cell carcinoma Opioid dependence Opioid overdose Oral ulcers Orthostatic hypotension Osteoarthritic pain Osteomalacia Osteoporosis Ovarian cancer Ovarian function Overactive bladder Painful bladder syndrome Pancreatic cancer Parkinson's disease Paroxysmal nocturnal haemoglobinuria Paroxysmal nocturnal haemoglobinuria (PNH) Peanut allergy Pemphigus vulgaris Peripheral arterial disease (PAD) Peripheral T-cell lymphoma (PTCL) PIK3CA-related overgrowth spectrum (PROS) Plaque psoriasis Plexiform neurofibromas Polycythaemia vera Pompe disease Post-acute myocardial infarction (MI) Post-menopausal vasomotor symptoms Post-operative inflammation Post-operative pain Post-transplant lymphoproliferative disease (PTLD) Postmenopausal osteoporosis Postnatal depression Postpartum depression Pouchitis Prevention of skeletal related events in multiple myeloma Primary biliary cholangitis (PBC) Primary generalised tonic-clonic seizures Primary haemophagocytic lymphohistiocytosis (HLH) Primary hypercholesterolaemia Primary hyperoxaluria Primary mediastinal large B-cell lymphoma (PMBCL) Primary open-angle glaucoma primary peritoneal cancer Primary sclerosing cholangitis (PSC) Procedural sedation Progeria Progressive familial intrahepatic cholestasis (PFIC) Progressive fibrosing interstitial lung disease (PF-ILD) Progressive supranuclear palsy (PSP) Prostate cancer Prurigo nodularis (PN) Pruritus Psoriatic arthritis Pulmonary arterial hypertension (PAH) Pyruvate kinase deficiency Regeneration of cartilage Renal anaemia Renal cell carcinoma (RCC) Renal disease Respiratory dysfunction Retinal Vein Occlusion Retinitis pigmentosa Retinopathy of prematurity Rett syndrome Rheumatoid arthritis Rhinosinusitis Schizophrenia Schnitzler syndrome Serious Trouble Short bowel syndrome Sialorrhea Sickle cell anaemia Sickle cell disease (SCD) Small lymphocytic lymphoma (SLL) Small-cell lung cancer (SCLC) Soft tissue sarcoma Solid tumours Spasmodic torticollis Spinal cord injury Spinal muscular atrophy (SMA) Spondyloarthritis Squamous cell carcinoma (SCC) Stroke Sudden sensorineural hearing loss Super-refractory status epilepticus (SRSE) Synovial sarcoma Systemic lupus erythematosus (SLE) Systemic mastocytosis Systemic sclerosis Tendon injury Tenosynovial giant cell tumour (TGCT) Thromboembolism Thrombotic microangiopathies Thrombotic thrombocytopenic purpura Thyroid cancer Transthyretin amyloid cardiomyopathy Transthyretin-mediated amyloid polyneuropathy Traumatic brain injury (TBI) Tree pollen allergy Tuberous sclerosis Type 1 diabetes Type 2 diabetes Ulcerative colitis Ulcerative proctitis Uncontrolled bleeding Upper limb spasticity Urinary incontinence Urothelial cancer Uterine Fibroids Uveal melanoma Venous leg ulcers Visual impairment Vitiligo von Willebrand disease Weight management WHIM syndrome Wilson's disease Wiskott-Aldrich syndrome (WAS) X-linked adrenoleukodystrophy (X-ALD) X-linked protoporphyria Check All
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