Specialty drug approvals trend upward; biobetters gain momentum

Within the next 5 years approximately $100 billion will be lost on previous blockbuster brands that will lose (or lost) patent protection. For manufacturers to enhance their return on investment, they are refocusing on researching and marketing specialty drugs, according to a specialty pharmaceuticals pipeline update at the Academy of Managed Care Pharmacy’s 23rd Annual Meeting and Showcase, Minneapolis.

There is an upward trend of specialty drug approvals, including those that are shifting for use as the mainstays of treatment. In the last 3 years, specialty approvals have increased from 8 in 2008 to 17 in 2010. From 2012 and beyond, approvals are expected to increase while some large pharmaceutical manufacturers have begun to partner or purchase biotech companies to enhance their product pipelines.

Other trends include research and approval of oral specialty agents with new mechanisms of action for the management of rheumatoid arthritis (RA), multiple sclerosis (MS), and hepatitis C virus (HCV) infection. There also will be more oral options for patients with moderate-to-severe and refractory disease, such as tofacitinib (RA); BG-12 or laquinimod (MS); and telaprevir or boceprevir (HCV) genotype 1. There is also a focus on creating biosimilars or "biobetters," which are modifications of biotechnology products such as better dosing, improved administration (such as from intravenous (IV) infusion to subcutaneous (SC) administration; pegylating products for every 2-week/4-week administration vs weekly administration), or more targeted therapies.

It is anticipated that specialty pharmaceuticals will continue to dwarf traditional pharmaceuticals in approvals, and costs, with the per member per year (PMPY) costs in 2013 being 4 times that of traditional agents at 40% of the drug spend. The top 3 specialty therapy classes in PMPY continue to be inflammatory conditions, MS, and cancer. Of the cancer agents, 20% likely will be covered under the pharmacy benefit, while 80% will be covered under the medical benefit. Inflammatory condition pipeline trends include oral biologics competing with injectable agents-some with better efficacy at potentially lower costs-as well as more therapies for gout and lupus, and potential approval of "biobetters."

Some biobetters include SC forms of abatacept and belimumab. Other potential approvals in 2011/2012 include SC canakinumab (gout), twice-daily oral apremilast (psoriasis; psoriatic arthritis), twice-daily oral tofacitinib (RA; psoriasis), and weekly SC rilonacept (gout). Trends in MS agents include more oral disease-modifying therapies, the potential use of combinations of these therapies, and expanded use of currently available injectable agents. Potential approvals in 2012 include oral twice-daily dimethyl fumarate (BG-12), oral once- daily laquinimod, IV infusion alemtuzumab, oral once-daily teriflunomide, and once-monthly SC daclizumab.

Half of all the specialty drugs in the pipeline are being developed for cancer, treating it as a chronic condition, with many of them being oral therapies. In the oncology realm, pharmacogenomic testing will be utilized to determine which patients’ diseases will be potential responders. Some of these include abiraterone (castration-resistant prostate cancer), ridaforolimus (soft tissue and bone sarcomas), ruxolitinib (myelofibrosis), as well as IV infusion brentuximab (non-Hodgkin’s lymphoma and anaplastic large cell lymphoma).

According to Aimee Tharaldson, PharmD, senior clinical consultant for emerging therapeutics at Express Scripts, "boceprevir and telaprevir . . . in addition to pegylated interferon and ribavirin in patients with hepatitis C genotype 1, have the potential to significantly improve sustained virological response in previous non-responders and treatment-naive patients. More oral therapies are on the horizon and triple oral therapy for this disease may be a reality in the coming years."