Tufts CSDD Report Finds Orphan Drug Approvals Have Increased, But Challenges Exist
Although the pace of approvals for new orphan drugs have increased in the United States and Europe in recent years, patients are facing growing challenges accessing those drugs.
Orphan drugs are defined as those developed for rare diseases and conditions that affect fewer than 200,000 people in the U.S., or five per 10,000 or fewer people in the European Union. Although the pace of approvals for new orphan drugs have increased in the United States and Europe in recent years, patients are facing growing challenges accessing those drugs, a newly completed study by the Tufts Center for the Study of Drug Development at Tufts University has concluded.
During the 14-year period 2000-2013, 86 orphan drugs were approved in the U.S., up from 65 during the prior 18-year period 1983-2000, while in Europe 96 orphan drugs were approved in 2000-2013, more than double the 44 approved in the earlier period, according to Tufts CSDD. Among the challenges that limit patient access to orphan drugs in the U.S., relative to Europe, is higher cost-sharing by patients, which can lead to increased levels of non-compliance.
Read the full release here.
Unifying Industry to Better Understand GCP Guidance
May 7th 2025In this episode of the Applied Clinical Trials Podcast, David Nickerson, head of clinical quality management at EMD Serono; and Arlene Lee, director of product management, data quality & risk management solutions at Medidata, discuss the newest ICH E6(R3) GCP guidelines as well as how TransCelerate and ACRO have partnered to help stakeholders better acclimate to these guidelines.
Phase III PROMINENT Trial Initiated to Evaluate Felzartamab for Primary Membranous Nephropathy
June 30th 2025The global Phase III PROMINENT trial has begun dosing patients to evaluate felzartamab in treating primary membranous nephropathy, a serious autoimmune kidney disorder with no FDA-approved therapies.
Funding Cuts Threaten Diversity in Clinical Research
June 27th 2025In this video interview, Kyle McAllister, co-founder, CEO, Trially, discusses how recent federal funding cuts are likely to undermine research focused on underrepresented populations, and why long-term investment in community-based studies is essential to closing persistent health equity gaps.
