FDA Approves Record 45 New Drugs in 2015, Led by Breakthrough Cancer Immunotherapy Opdivo
Key Insights
The FDA approved 45 new drugs in 2015, marking a significant rise in R&D productivity, with Bristol-Myers Squibb (search)'s immunotherapy Opdivo leading the pack with five new indications for various cancers.
Novel treatments like Vertex's Orkambi for cystic fibrosis (search) and Novartis's Entresto for heart failure demonstrated remarkable clinical innovation, though pricing concerns remained central to industry discussions.
The year saw major developments in drug pricing debates, with the emergence of ICER as an independent US cost-effectiveness watchdog and increased scrutiny of pharmaceutical pricing practices.
The pharmaceutical industry demonstrated renewed vigor in innovation during 2015, with the FDA approving 45 new chemical and biological entities, surpassing 2014's total of 40 approvals. This surge in R&D productivity brought forth several groundbreaking treatments that promise to reshape therapeutic landscapes across multiple diseases.
Immunotherapy Leads Cancer Treatment Revolution
Bristol-Myers Squibb (search)'s Opdivo emerged as the standout therapy of 2015, securing five new indications throughout the year. Following its initial approval for advanced melanoma (search) in December 2014, the immunotherapy gained additional approvals for both squamous and non-squamous non-small cell lung cancer (search) (NSCLC), expanded use in melanoma, and breakthrough status in renal cell carcinoma.
The drug's impressive clinical performance came with significant cost implications - priced at $12,500 monthly or $150,000 annually. When combined with Yervoy for melanoma (search) treatment, the annual cost could reach $256,000, raising concerns among oncologists despite demonstrated clinical benefits.
Innovation in Rare Diseases and Chronic Conditions
Vertex Pharmaceuticals' Orkambi marked a significant advance in cystic fibrosis (search) treatment. The combination therapy expanded treatment options to approximately 8,500 US patients with two copies of the F508del mutation, priced at $259,000 annually - notably lower than its predecessor Kalydeco's $311,000 price tag.
Novartis's Entresto represented a major breakthrough in chronic heart failure (search) treatment, demonstrating a 20% reduction in cardiovascular death or hospitalization risk over 27 months. Priced at $4,600 annually in the US, the drug pioneered innovative "pay-for-performance" pricing models, where reimbursement would be tied to reduced hospital admissions.
Pricing Debates and Market Access
The year marked a turning point in drug pricing discussions with the emergence of the Institute for Clinical and Economic Review (ICER) as America's first independent cost-effectiveness watchdog. ICER's assessments significantly influenced pricing negotiations, particularly for new cholesterol-lowering PCSK9 inhibitors like Amgen's Repatha and Sanofi's Praluent.
Biosimilars Enter US Market
In a historic development, Sandoz's Zarxio became the first biosimilar approved in the US market. While launched at a modest 15% discount to Amgen's Neupogen, the approval signaled the beginning of a new era in biological medicine accessibility, with Express Scripts projecting potential savings of $250 billion from just 11 biosimilars through 2024.
Innovation in Drug Delivery
The industry saw notable advances in drug delivery systems, including Aprecia Pharmaceuticals' Spritam, the first FDA-approved 3D-printed medication. The technology enables more potent formulations through enhanced pill porosity, potentially revolutionizing pharmaceutical manufacturing processes.
Multiple Myeloma Treatment Landscape Expands
The year concluded with significant progress in multiple myeloma (search) treatment, highlighted by the approval of Bristol-Myers Squibb (search) and AbbVie's Empliciti. The immunotherapy demonstrated a 30% reduction in disease progression or death risk when combined with standard treatments, though its $142,000 annual cost added to ongoing pricing discussions.