Orphan Drugs Continue to Struggle During Approval Process

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Average development time for orphan drugs that were first-in-class new molecular entities (NMEs) and that won U.S. Food and Drug Administration (FDA) approval between 1999 and 2012 was 18% longer than the average journey for all new drugs, says a new report from the Tufts Center for the Study of Drug Development (CSDD). Orphan NME time from first patent filing to launch was 15.1 years, as compared to 12.8 for all new drugs.

Orphan drugs focused on central nervous system or cardiovascular indications showed the greatest number of development challenges, Tufts reported. Designations and approvals for orphan oncology drugs outpaced orphan drug development in all other therapeutic areas.

“Ethnic, geographic, and gender differences make orphan conditions, on occasion, hard to diagnose, difficult to study and quantify, and complicated to follow up,” the Tufts report said.

The study, which examined 46 first-in-class, orphan NMEs approved by FDA between 1999 and 2012, found that development time for drugs to treat ultra-orphans diseases—those in the U.S. that only affect up to a few hundred patients—is even longer: 17.2 years.

Orphan drugs are defined as prescription medicines developed for rare diseases and conditions, which, in the U.S., affect fewer than 200,000 people, or, in the European Union, affect 5 per 10,000 people or fewer. Orphan diseases currently encompass more than 7,000 diseases and conditions, affecting up to 30 million people in the U.S., 50% of whom are children, according to Tufts.

“Creating new medicines to treat orphan diseases continues to pose unique challenges, not the least of which is the logistical difficulty of working with small patient populations that are, in the vast majority of cases, widely geographically dispersed,” said Christopher-Paul Milne, research associate professor and director of research at Tufts CSDD at Tufts University School of Medicine, who conducted the analysis.

Milne noted while new approaches to study design, including use of patient advocacy groups and adaptive clinical trials, are helping to mitigate development problems, orphan drug development is likely to continue to face difficulty due, in part, to a lack of animal models and biomarkers.

Author: Michael Causey