The number of new drug approvals for 2014 by the European Medicines Agency (EMA), and by the US Food and Drug Administration (FDA), has increased significantly over 2013 figures. According to industry analysis, the EMA approved 82 new medicines last year, up from 79 in 2013, while the FDA approved 41 new drugs, up from 29 approvals in 2013. The increased pace of approvals reflects a significant innovation drive by the commercial sector, in addition to a willingness by the regulatory agencies to progress “breakthrough” therapies in a range of indications – 9 breakthrough drugs were approved by the FDA in 2014, compare to 3 in 2013.
In respect of the FDA metrics, 2014 Is understood to be the second highest year on record for the approval of new chemical entities (NCEs), the highest record being 53 approvals in 1996. Importantly, the 41 approvals by the FDA in 2014 is well ahead of the 2000-2010 average of 24 approvals and ahead of the 1990-2000 average of 31 approvals. The 2014 output may indicate that both sides of the drug development industry – developers and regulators – are reaching an improved understanding across a range of levels in respect of what works and what doesn’t. Following years of often bitter argument between industry and regulatory officials, most notably around what are acceptable levels of risk, a consensus on how to do business may be emerging that should ultimately benefit consumers.
Notwithstanding such, even as the number of approvals has increased, there appears also to be a significant increase in the price of some medications. Amgen ‘s pricing for Blincyto (blinatumomab), indicated for acute lymphoblastic leukemia, is understood to come in at $178,000 for a course of treatment, while Aegerion’s Myalept (metreleptin) for treating metabolic disorders with generalised lipodystrophy, is priced at $325,000 a year. Most significantly, Gilead’s Harvoni (ledipasvir/sofosbuvir), for treatment of hepatitis C, priced at $94,500 per standard 12-week treatment, will not be included in Express Scripts’ 2015 formulary. Its failure to negotiate inclusion will put pressure on forecast revenues and performance for the coming year and beyond, the last thing a company needs having successfully navigated the regulatory path to approval.