National Employer Initiative on

Specialty Drugs & Biopharma

Employer Focused, Employer Driven

Specialty Drug Approvals: Review of 2014 and Forecast for 2015

The impact of specialty drugs continues to increase in both drug utilization and spend. In 2012, specialty drug spend accounted for approximately $87 billion, or about 3.1% of national health spend in the United States. In 2020, the forecasted specialty drug spend is expected to be $400 billion, or about 9.1% of national health spend. In addition, within 4 years, analysts predict that 7 of the top 10 drugs in terms of sales will be specialty products.1 Given this expected growth, the specialty drug pipeline will be of particular interest to patients, prescribers, payers, and pharmacies. 

New specialty drug approvals

As of December 3, 2014, FDA had approved 39 new molecular entities and new therapeutic biologics this year. While there is no universal definition of a specialty drug, approximately 19 of this year’s approvals fit commonly used definitions of specialty products (Table 1).6,7 Of these 19, oncology and rare diseases represented the majority of approvals, a trend that is expected to continue through 2015

In the pipeline


Looking ahead to the remainder of 2014 and 2015, the pipeline is again forecasted to have the highest number of approvals in oncology, with approximately one dozen expected approvals for a variety of cancers (Table 2).3 Programmed cell death–1 (PD-1) drugs stand out as an especially promising class of therapy. Pembrolizumab (Keytruda—Merck) was the first of this class to earn FDA approval for the treatment of unresectable or metastatic melanoma. Pembrolizumab, along with fellow experimental PD-1 drug nivolumab (Opdivo—Bristol-Myers Squibb), have earned breakthrough designations from FDA and are likely to see success expanding into other indications.

Trials are currently under way with PD-1 agents for a number of cancers, including lung, liver, brain, and solid tumors. Other pipeline drugs are in late stages of development for the treatment of breast, melanoma, non–small cell lung, ovarian, and pancreatic cancers, as well as hematologic malignancies such as leukemia and multiple myeloma.3

Hepatitis C

Hepatitis C (HCV) continues to have one of the most intriguing pipelines of any disease state. Within the last year, the approvals of sofosbuvir (Sovaldi—Gilead) and simeprevir (Olysio—Janssen) as individual agents have led to major changes in the treatment of HCV. Gilead’s Harvoni, the combination of sofosbuvir and ledipasvir, was approved in October 2014. In addition, sofosbuvir and simeprevir used together was approved in November 2014. Both of these combination therapies show high sustained viral response rates and are interferon-free, oral regimens. 

Competition should come from the AbbVie “3D” HCV combination therapy composed of ombitasvir, paritaprevir, and ritonavir (Viekirax) used in combination with dasabuvir (Exviera). An FDA decision for this combination is expected by December 22, 2014. In addition, Merck’s HCV combination of MK-5172 and MK-8742 is also showing good effectiveness in clinical trials. 

Tough competition from fast-paced innovation in the HCV market has taken a toll on other pipeline agents. Faldaprevir, a late-stage drug from Boehringer Ingelheim, was abandoned. Bristol-Myers Squibb’s daclatasvir and asunaprevir, taken together as a two-drug combination therapy, was discontinued. However, studies continue with daclatasvir as a component of other drug combinations, including a three-drug combination of daclatasvir with asunaprevir and BMS-791325. A two-drug combination of daclatasvir paired with sofosbuvir is also being evaluated in clinical trials.3


In the immunology pipeline, top agents of interest include the IL-17 class of drugs: secukinumab (Novartis), brodalumab (AstraZeneca, Amgen), and ixekizumab (Eli Lilly). These agents are showing promise particularly for the treatment of psoriasis. In clinical trials, some IL-17 inhibitors have demonstrated efficacy that is superior to etanercept (Enbrel—Amgen), a commonly used drug in the treatment of psoriasis. These drugs are also being investigated for the treatment of rheumatoid arthritis, ankylosing spondylitis, and psoriatic arthritis. Sarilumab (Sanofi—Regeneron) is an additional late-stage pipeline agent being evaluated for the treatment of rheumatoid arthritis.3

Rare diseases

Opportunities in an area with limited competition have made rare disease treatments an attractive category for many manufacturers. Examples of these types of products include the recently approved drugs for idiopathic pulmonary fibrosis, pirfenidone (Esbriet—InterMune) and nintedanib (Ofev—Boehringer Ingelheim), as well as the experimental drugs eteplirsen (Sarepta) and drisapersen (Prosensa), which are currently being studied for the treatment of Duchenne muscular dystrophy. These drugs treat diseases where currently available therapies are limited. 

Growing trend

This trend toward the development of rare disease treatments is expected to continue growing. As of 2013, more than 450 drugs were in various stages of development for rare diseases.Further evidence of the growth in rare disease drug development is shown by the fact that in 2010, of the top 100 drugs in the United States, 23 were for the treatment of diseases with 100,000 or fewer patients. In 2014, that number of rare disease drugs had increased to 41. Meanwhile, drugs targeting diseases with higher patient populations became less popular. In 2010, of the top 100 drugs, 55 treated diseases with 500,000 or more patients. By 2014, that total was reduced to 35 drugs.5

The pipeline is stocked with exciting treatments for many disease states. While we can’t be certain how many of these agents will earn FDA approval, the possibility of bringing patients better treatments makes the drug pipeline worth watching.

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