Eflornithine is in clinical development for the maintenance treatment of patients with high risk neuroblastoma. Neuroblastoma is a rare cancer which most often affects children under the age of 5 years but can also affect adults in rare instances. It develops in the nervous system from the uncontrolled growth of specialised nerve cells known as […]
Histology-Independent Therapies (HITs) for cancer, or Tumour-Agnostic Therapies are recent innovations in oncology wherein treatment is based on the cancer’s genetic and molecular alterations rather than cancer type and/or site of origin.
In this paper, we provide an overview of the current clinical trial landscape for combination therapeutics targeting COVID-19 through weekly scanning of national and international clinical trial registries. Our analysis delves specifically into dual combination therapies in what can be defined as “pivotal clinical trials” (active, randomised, controlled and at least phase II), with a focus on new and repurposed therapeutic candidates that have shown positive signals and/or been granted authorisation for emergency use based on positive efficacy and safety data.
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 […]
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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 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 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