Global Orphan Drug Market

High Pricing and Undefined Reimbursement Policies will Limit the Sales of Orphan Drugs

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The treatment of rare diseases requires the use of specialized drugs. Patient populations for rare diseases are most often quite small, consequently the products treating them earn the designation orphan drugs. These drugs must undergo the research, development, and clinical trials that all drugs do, yet the investment into the development is great, while the promise for recouping financial costs is not guaranteed. This study covers the spectrum of orphan diseases and highlights drugs already marketed as well as those in the development pipeline for them. Regional highlights, market opportunities, companies to watch, and legislative timelines are provided. This snapshot captures the global orphan drug landscape as of June 2016.

Table of Contents

Executive SummaryScope and SegmentationKey Questions this Snapshot will AddressKey Takeaways—Orphan Drug Market Executive Summary—Market SnapshotExecutive Summary—Market Snapshot (continued)Commercialization Analysis 8 Big Market Themes for Orphan DrugsA Vibrant EcosystemCancer Therapies Remain the Largest Orphan Drug Segment Global Overview and AnalysisRegional HighlightsGame-changing Companies Companies to WatchNew Market Opportunities—Orphan DrugsKey 2016 Predictions—Orphan Drug MarketTimeline of Orphan Drug LegislationDisruptive Technologies and Advances in this FieldOverall Environmental Impact FactorsOrphan Drug Payer EnvironmentA Snapshot of Global Drug Price Control Methods Conclusion and Take Home PointsKey ConclusionsThe Last Word—3 Big PredictionsLegal DisclaimerAppendix A—Orphan Diseases with FDA-approved TherapiesOrphan Diseases with FDA-approved TherapiesOrphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Orphan Diseases with FDA-approved Therapies (continued)Appendix B—Pipeline Analyses for Select Rare DiseasesTop Indications by Activity and Disease Overview—Blood and Lymphatic System DiseasesSCD—Orphan Drugs on the MarketSCD—Orphan Drugs in DevelopmentSCD—Orphan Drugs in Development (continued)SCD—Orphan Drugs in Development (continued)MDS—Orphan Drugs on the MarketMDS—Orphan Drugs in DevelopmentTTP—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Cardiovascular DiseasesTop Indications by Activity and Disease OverviewPAH—Orphan Drugs on the MarketPAH—Orphan Drugs in Development PAH—Orphan Drugs in Development (continued)Calciphylaxis—Orphan Drugs in Development and on the MarketPipeline Analyses for Select Rare Diseases—Lung DiseasesTop Indications by Activity and Disease OverviewCF—Orphan Drugs on the MarketCF—Orphan Drugs in Development CF—Orphan Drugs in Development (continued) CF—Orphan Drugs in Development (continued) CF—Orphan Drugs in Development (continued) IPF—Orphan Drugs on the MarketIPF—Orphan Drugs in Development IPF—Orphan Drugs in Development (continued) Pipeline Analyses for Select Rare Diseases—Congenital and Genetic DiseasesTop Indications by Activity and Disease OverviewFragile X Syndrome—Orphan Drugs in Development HIBM—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Connective Tissue/Skin DiseasesTop Indications by Activity and Disease OverviewSSc—Orphan Drugs in DevelopmentEB—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Digestive/Nutritional DiseasesTop Indications by Activity and Disease OverviewPed CD—Orphan Drugs on the MarketPed CD—Orphan Drugs in DevelopmentPed CD—Orphan Drugs in Development (continued)Ped UC—Orphan Drugs on the MarketPed UC—Orphan Drugs in DevelopmentPed UC—Orphan Drugs in Development (continued)Pouchitis—Orphan Drugs in Development Pipeline Analyses for Select Rare Diseases—Endocrine DiseasesTop Indications by Activity and Disease OverviewT1D with Residual Beta-cell Function—Orphan Drugs in Development T1D with Residual Beta-cell Function—Orphan Drugs in Development (continued)Pipeline Analyses for Select Rare Diseases—Exposure/ToxicityTop Indications by Activity and Disease OverviewARS—Orphan Drugs in Development ARS—Orphan Drugs in Development (continued)Pipeline Analyses for Select Rare Diseases—Eye DiseasesTop Indications by Activity and Disease OverviewRP—Orphan Drugs in DevelopmentRP—Orphan Drugs in Development (continued)Stargardt Disease—Orphan Drugs in DevelopmentUveitis—Orphan Drugs on the MarketUveitis—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Immunological/Inflammatory DiseasesTop Indications by Activity and Disease OverviewBehcet's Disease—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Infectious/Parasitic DiseasesTop Indications by Activity and Disease OverviewCMV Infection—Orphan Drugs on the MarketCMV Infection—Orphan Drugs in DevelopmentMalaria—Orphan Drugs on the MarketMalaria—Orphan Drugs in DevelopmentLeishmaniasis—Orphan Drugs on the MarketLeishmaniasis—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Metabolic DiseasesTop Indications by Activity and Disease OverviewTop Indications by Activity and Disease Overview (continued)MPS—Orphan Drugs on the MarketMPS—Orphan Drugs in DevelopmentMPS—Orphan Drugs in Development (continued)Acromegaly—Orphan Drugs on the MarketAcromegaly—Orphan Drugs in DevelopmentUrea Cycle Disorders—Orphan Drugs on the MarketUrea Cycle Disorders—Orphan Drugs in DevelopmentNPC Disease—Orphan Drugs on the MarketNPC Disease—Orphan Drugs in DevelopmentPompe Disease—Orphan Drugs on the MarketPompe Disease—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Musculoskeletal DiseasesTop Indications by Activity and Disease OverviewDMD—Orphan Drugs in DevelopmentDMD—Orphan Drugs in Development (continued)DMD—Orphan Drugs in Development (continued)DMD—Orphan Drugs in Development (continued)SMA—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Neurologic DiseasesTop Indications by Activity and Disease OverviewALS—Orphan Drugs in DevelopmentALS—Orphan Drugs in Development (continued)ALS—Orphan Drugs in Development (continued)ALS—Orphan Drugs in Development (continued)HD—Orphan Drugs on the MarketHD—Orphan Drugs in DevelopmentFA—Orphan Drugs in DevelopmentPipeline Analyses for Select Rare Diseases—Rare CancersTop Indications by Activity and Disease OverviewPancreatic Cancer—Orphan Drugs on the MarketPancreatic Cancer—Orphan Drugs in DevelopmentPancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Pancreatic Cancer—Orphan Drugs in Development (continued)Glioma—Orphan Drugs on the MarketGlioma—Orphan Drugs in DevelopmentGlioma—Orphan Drugs in Development (continued)Glioma—Orphan Drugs in Development (continued)Glioma—Orphan Drugs in Development (continued)Glioma—Orphan Drugs in Development (continued)AML—Orphan Drugs on the MarketAML—Orphan Drugs in DevelopmentAML—Orphan Drugs in Development (continued) AML—Orphan Drugs in Development (continued) AML—Orphan Drugs in Development (continued) AML—Orphan Drugs in Development (continued) The Frost & Sullivan StoryThe Frost & Sullivan StoryValue Proposition: Future of Your Company & CareerGlobal PerspectiveIndustry Convergence360º Research PerspectiveImplementation ExcellenceOur Blue Ocean Strategy




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