Beta Thalassaemia is an indication for drug development with over 50 pipeline drugs currently active. According to GlobalData, preregistered drugs for Beta Thalassaemia have a 80% likelihood of approval (LoA) indication benchmark. GlobalData’s report assesses how phase transition success rate (PTSR) and likelihood of approval (LoA) scores for pipeline drugs in Beta Thalassaemia compared to historical benchmarks. Buy the report here.
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GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.
Beta Thalassaemia overview
Beta-thalassemia is a genetic blood disorder characterized by reduced or absent production of beta-globin chains, a component of hemoglobin, the protein in red blood cells responsible for carrying oxygen throughout the body. This deficiency leads to anemia and various complications. Beta-thalassemia is classified based on the severity of symptoms: In beta-thalassemia major (Cooley’s anemia), individuals have little or no beta-globin production, leading to severe anemia, pale skin, fatigue, bone deformities, enlarged spleen, jaundice, and growth problems. Beta-thalassemia intermedia is less severe than major but can still cause moderate to severe anemia and various complications. Beta-thalassemia is an autosomal recessive genetic disorder, meaning an affected individual must inherit two abnormal beta-globin genes (one from each parent). Carriers of one abnormal gene (thalassemia trait) generally do not have symptoms but can pass the defective gene to their children.
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For a complete picture of PTSR and LoA scores for drugs in Beta Thalassaemia, buy the report here.
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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.
GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.
