Rare Disease Report

New Data Reinforces Difficulty in Orphan Drug Development

MAY 09, 2018
Mathew Shanley
New data revealed by the Tufts Center for the Study of Drug Development concludes that orphan drug development takes 18% longer than the average time required for other new drugs.

The study, summarized in the May/June edition of Tufts CSDD Impact Report, evaluated 46 first-in-class, orphan new molecular entities approved by the U.S. Food and Drug Administration (FDA) between 1999 and 2012.

Results showed that orphan drugs, on average, take 15.1 years to go from first patent filing to product launch. This reinforces the existing belief that, because of the challenges faced in recruiting sufficient numbers of patients for clinical trials, orphan drug development is simply more difficult than most other cases of drug development.

Additionally, it was found that development time for drugs to treat ultra-orphan diseases – those in the United States that only affect a few hundred patients – is even longer. For this classification of diseases, orphan drug development takes, on average, 17.2 years.

Christopher-Paul Milne, research associate professor and director of research at Tufts CSDD at Tufts University School of Medicine, conducted the analysis, serving as the study’s primary author. "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,” Milne said in a press release.

Milne mentioned that while new approaches to study design, including use of patient advocacy groups and adaptive clinical trials, are assisting in mitigating development problems, difficulties in orphan drug development are expected to persist due, in part, to a lack of animal models and biomarkers.

In the analysis, it was also noted that orphan drug developers encountered an average of 4.2 special challenges during research and development conducted between 1999 and 2012. Designations and approvals for orphan oncology drugs outpaced drug development in all other therapeutic areas; and orphan drugs focused on the central nervous system (CNS) or cardiovascular indications experienced the greatest number of development challenges.

On March 5, the New Jersey Rare Disease Alliance hosted its Rare Disease Day observation at the NJ State Museum in Trenton, and was joined by Paul Melmeyer, Director of Federal Policy for the National Organization for Rare Disorders (NORD). There, Melmeyer stressed the need to have patient voice orphan drugs continue to be developed.

“It’s so incredibly important for patients to get involved in state policy, but also federal policy, and for various reasons,” Melmeyer said. “One is that the patient voice is uniquely powerful. A patient and his or her loved ones can tell the story of the experiences of having a rare disease better than anyone else – including the advocates or any doctor. Having a patient or their family member actually in the room as lawmakers are considering next steps is an impactful opportunity for the legislator, regulatory individual or administrator to see things firsthand as they’re considering various different policy options.”

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