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.

Inherited Metabolic Disorders overview

Genetic defects causing metabolic problems are referred to as inherited metabolic disorders. These are rare genetic conditions also known as inborn errors of metabolism. Hunter syndrome, Krabbe disease, Maple syrup urine disease, Wilson’s disease, porphyria, Hunter syndrome, phenylketonuria is some of the common inherited metabolic disorders. Recent advances in these cases recommend gene replacement therapies followed by dietary restriction and even liver transplant in severe cases.

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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.