Disease Landscape and Forecast Insights

Nearly 35 years after the Orphan Drug Act of 1983 (ODA) became law in the United States, private investment in the development of drugs for orphan diseases continues unabated. Although the growing…

Progress in R&D and technology, regulatory and monetary incentives for advancing the development of treatments for rare diseases, and ongoing focus by the pharmaceutical market on this space are…

Dravet syndrome (DS) is a rare, severe, pediatric-onset form of epilepsy characterized by developmental delay or regression and multiple seizure types. Only Diacomit (Biocodex’s stiripentol) is…

This detailed, expanded analysis explores key access and reimbursement topics in the rare disease space in the United States, such as the approaches managed care organizations take to control both…

    Pharma’s interest in treatments for rare diseases has continued to intensify as the orphan sector sees rapid growth and attractive profit margins. Developers of orphan drugs can…

By definition, rare diseases affect relatively few individual patients, but as a group they encompass up to 8,000 recognized rare diseases affecting approximately 10% of the U.S. population. The…

A Survey of Medical Oncologists and Interviews with Payers in the EU5 Hepatocellular carcinoma (HCC), thyroid cancer, gastrointestinal stromal tumor (GIST), and neuroendocrine tumors (NETs) fall…

With Few Good Treatment Choices, Which Emerging Therapies Hold the Greatest Potential for Managing AML and What Types of Therapies Will Receive Reimbursement? Despite the introduction and use of…

With Few Good Treatment Choices, Which Emerging Therapies Hold the Greatest Potential for Managing AML and What Types of Therapies Will Receive Reimbursement? Despite the introduction and use of…