Orphan drugs cost 13.8 times more per patient than mass-market drugs – a somewhat static fact that continues to impact the course of biopharma R&D.
Meaning: There’s been little change in the past few years on growth forecasts in this specialized sector, according to a new report from EvaluatePharma. But the lure of orphan drugs still outpaces the broader pharmaceutical industry.
“If anything sums up the current orphan drug market, it is steady as she goes,” EvaluatePharma says. “However, the almost 12 percent annual growth seen in this sector is something that the general drug market could only dream about.”
Notably, the report says, the biggest change is that it’s now looking as though Celgene will knock Novartis off its perch as the world’s biggest orphan drug company in sales by 2020.
Indeed, worldwide orphan drug sales are projected to hit $178 billion by 2020, the report says. Orphans are set to account for just over 20 percent of all prescription drug sales by 2020, excluding generics.
Last year was a record year for both U.S. and European orphan designations, with 291 by the FDA and 201 by the European Medicines Agency.
The benefits of orphan drug discovery are vast, of course: There’s a 50 percent tax credit on R&D costs, there are ample Phase 1 and 2 clinical trial grants to be hand, and user fees are waived.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
Orphan drug top sellers include Rituxan, Copaxone and Revlimid.
