Patent-to-Launch Time for Orphan Drugs is 2.3 Years Longer vs. Other Drugs, According to the Tufts Center for the Study of Drug Development
BOSTON – May 9, 2018 – Orphan drug development, which faces special challenges in recruiting sufficient numbers of patients for clinical trials, on average takes 15.1 years to go from first patent filing to product launch, 18% longer than the average time required for all new drugs, according to a study recently completed by the Tufts Center for the Study of Drug Development.
The study, which examined 46 first-in-class, orphan new molecular entities approved by the United States Food and Drug Administration (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.
"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.
He 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.
The analysis, summarized in the May/June Tufts CSDD Impact Report, released today, also found that:
Designations and approvals for orphan oncology drugs outpaced orphan drug development in all other therapeutic areas.
Orphan drug developers encountered an average of 4.2 special challenges in the course of research and development conducted during 1999-12.
Orphan drugs focused on central nervous system or cardiovascular indications experienced the greatest number of development challenges.
ABOUT THE TUFTS CENTER FOR THE STUDY OF DRUG DEVELOPMENT
Established in 1976, the Tufts Center for the Study of Drug Development (http://csdd.tufts.edu) is a multidisciplinary academic research group that provides data-driven analyses and strategic insight to help developers, regulators, and policy makers improve the efficiency and productivity of pharmaceutical R&D. Tufts CSDD also offers CME-accredited professional development courses, hosts workshops and public forums, and publishes the Tufts CSDD Impact Report, a bimonthly newsletter focusing on critical drug development issues.
Contacts: Tufts Center for the Study of Drug Development
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