Orphan Drugs, Breakthrough Therapy Designations, Oncology, And Statistics
As reported widely in the media and the literature, oncology indications make up a significant percentage of orphan drug indications and FDA Breakthrough Therapy (BTD) Designations. The IMS Institute for Healthcare Informatics publishes this month the “Innovation in Cancer Care and Implications for Health Systems Global Oncology Trend” report. The report reviews current trends in the global oncology market, the status of innovation in therapeutics, measures the value of treating cancer, and the pricing trends for oncology products.
Here are a few observations for the drug trends in the United States for orphan drugs and BTDs with oncology indications from the May 2014 report:
• In 2013, 17 new drugs are launched to treat orphan diseases, 8 of which are for the treatment of cancer – in other words, 47% of the new drugs launched in 2013 have an oncology indication
• 34% of FDA BTDs (only 75% of BTDs have been reported by the sponsor company) are oncology products
• 1st drug launched with an FDA BTD is Genentech’s Gazyva (Obinutuzumab) for the treatment of Chronic Lymphocytic Leukemia (CLL)
• In 2013, 2 BTDs, Gazyva and Pharmacyclics’ Imbruvica (Ibrutinib) receive approval 1 to 3 months earlier than anticipated.
The Orphan Druganaut Blog has supplied the following statistics for oncology products:
• 75% of the FDA BTDs receiving approval (3 of 4 approvals, as of 04/29/14) are for an oncology indication (CLL, MCL, and ALK+ NSCLC)
• Of the 38 BTDs reported by sponsor companies as of 04/24/14), 42% are for an oncology indication.
The FDA Breakthrough Therapy Designation has the potential of playing an important role in helping to accelerate drug development and the approval process for oncology indications.