Specialty Pipeline: New Approvals Pending, More Management Awaits

Aimee Tharaldson, PharmD, senior clinical consultant, emerging therapeutics, Express Scripts, gave the health plan, PBM, and insurer attendees insight into the direction of specialty pharmacy approvals over the next few years: “There will be many more therapeutic options for several disease areas than there were 10 years ago,” she said, pointing to $13.1 billion in specialty generic opportunities and $31.8 billion in biosimilar opportunities in the next 5 years alone. However, the progress on formalizing the 351(k) pathway for the approval of biosimilars will result in “more biologic license applications (BLAs) being filed than 351(k) applications in the near term.” She expects a cost differential of 20% on biosimilar products compared with innovator agents, but this could be greater, depending on the interchangeability issue: this will be the key to greater cost savings, according to Dr Tharaldson, but she also expects plenty of legal barriers to be raised by branded manufacturers, delaying as much as possible the introduction of these biosimilars.
In the pipeline, however, there seems to be hope for better treatments—but likely at premium costs. She noted that orphan drug development, a key pipeline trend, may yield blockbuster status for up to 30% of these infrequently prescribed agents. “The actual annual cost of an orphan drug treatment last year was $185,000,” reported Dr Tharaldson.
Another important trend is the increasing number of pipeline products designated as “breakthrough agents” by the Food and Drug Administration (FDA). Drugs designated as breakthrough therapies will be indicated for serious or life-threatening conditions, and must demonstrate at least some level of evidence of efficacy. Of the 133 requests by the biopharmaceutical industry for breakthrough designation, FDA has granted 40 of these requests.
Currently, specialty drugs account for 28% of the total spend, according to Express Scripts Drug Trends Report. But only 2% of patients are using these medications. Dr Tharaldson foresees that the FDA will continue the recent annual trend of approving more specialty medications than conventional drugs (over half of all drugs approved in 2013 were specialty drugs).
Notable 2013 approvals include Adempas (riociguat) from Bayer, a 3-times-daily oral therapy to treat pulmonary arterial hypertension (PAH); Opsumit (macitentan) from Actelion and Orenitram (treprostinil) from United Therapeutics, both also oral PAH drugs.
There were 2 important hepatitis C virus (HCV) approvals that are by now well known—Olysio (simeprevir) from Janssen and Sovaldi (sofosbuvir) from Gilead—with several more oral, interferon-free treatments in the pipeline. 
For 2014 and beyond, approvals will likely include a number of costly specialty drugs aimed at treating small numbers of patients, like a sleep/wake disorder drug that could reach a cost of $100,000 per year. Vimzim (elosulfase alfa) from BioMarin was just approved in February, and targets a disease that affects only around 3000 patients worldwide; Northera (droxidopa) from Chelsea Therapeutics, for treatment of neurogenic orthostatic hypotension; Myalept (metreleptin for injection) from Bristol-Myers Squibb/AstraZeneca for treatment of generalized lipodystrophy; are all expected to cost over $100,000 per year.
Dr Tharaldson said that going forward, the FDA may also be approving expanded indications for current  drugs as well as more oral therapies and new injectables, among others, along with several new orphan drugs in therapeutic areas such as hemophelia A and B, multicentric Castleman disease, angioedema, and hypoparathyroidism.

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