Specialty Drugs Dominate Late-Stage Pipeline

Orphan drugs and breakthrough therapies last year comprised the highest number of new medications approved since 2001.

Specialty drug use in particular continued to dominate the research and development pipeline, with significant growth expected into the future, according to the annual drug trend report issued last week by the IMS Institute for Healthcare Informatics.

Specialty medications now comprise 42% of the late-stage pipeline, up from 33% a decade ago.

Research and development priorities are increasingly focused on specialty drugs, with specialty sales accounting for 33% of medication spending in 2014, up from 19% in 2004. Increased spending on hepatitis C products in particular powered this spending, with 4 new drugs alone accounting for $11.3 billion of the $24.5 billion in new product sales last year.

Over the last 10 years, the specialty drug share of the medication pipeline has jumped 9% as a result of new therapies for cancer, nervous system disorders, infectious diseases, diabetes, and respiratory disorders, according to IMS. The total number of specialty drug approvals more than doubled from 7 in 2004 to 15 in 2014.

Orphan drugs continued to rise in prevalence, as last year marked the largest number of orphan drugs approved in a single year, at 18. A total of 61 orphan drugs have been approved in the last 5 years—the largest number of approvals in a 5-year period since the Orphan Drug Act was passed in 1983. In comparison, 31 orphan drugs were approved between 2005 and 2009.

“Aided by expedited approval pathways and FDA incentive programs for rare and infectious diseases, a robust late-phase pipeline with more than 530 distinct research programs is expected to maintain the high number of launches seen in the past 5 years into the future,” the study authors wrote.

The total number of drugs approved with the orphan designation is now up to 230. Orphan drugs for cancer are the most common category, with 9 “ultra-orphan” drugs for populations with fewer than 10,000 patients approved last year.

Treatments for rare cancers accounted for 27 of the 61 orphan products approved over the last 5 years, including treatments for homozygous familial hypercholesterolemia, idiopathic pulmonary arterial hypertension, Gaucher’s disease, and multiple hemophilias—all with fewer than 10,000 patients.

More than 40 Breakthrough Therapy Designations were also granted last year, with 10 breakthrough drugs approved. IMS projects breakthrough drug launches to grow next year, due to the 22% increase in the number of designation requests by manufacturers.

“Based on historic approval rates and time to launch, launch trajectory is expected to peak in the next 24 months and remain elevated through the next 5 years,” the authors wrote. “The pipeline continues to be driven by innovation, supplemented by many new drug delivery methods and additional indications.”