Rare Daily Staff

It takes 15.1 years on average to advance an orphan drug from the first patent filing to product launch—18 percent longer than the average time required for all new drugs—according to a study by the Tufts Center for the Study of Drug Development.

The analysis found that development time for drugs to treat ultra-orphan diseases was even longer at 17.2 years. The study, published in the May/June Tufts CSDD Impact Report, examined 46 first-in-class, orphan new molecular entities approved by the United States Food and Drug Administration between 1999 and 2012.

The center is an independent, academic, non-profit research center that gets no funding from the university and relies in part on corporate sponsorship to support its work.

The analysis comes as there is growing scrutiny on the Orphan Drug Act and questions are being raised as to whether drugmakers are benefitting from the act in ways that were unintended when it was crafted to incentivize companies to develop drugs to address disease populations seen as otherwise too small to justify the investment in developing therapies to address.

In the United States, orphan drugs are defined as ones that treat a condition that affects 200,000 people or less. There are more than more than 7,000 diseases and conditions, affecting up to 30 million people in the United States, half of whom are children.

The report identified many of the well-known challenges associated with orphan drug development including the small patient populations, the geographic dispersion of patients, the lack of biomarkers and animal models for many conditions, and the lack of understanding of the biological basis of a disease.

“Creating new medicines to treat orphan diseases continues to pose unique challenges,” 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 because of the challenges inherent in developing treatments for these diseases.

May 9, 2018
Photo: Christopher-Paul Milne, research associate professor and director of research at Tufts CSDD