Beyond borders: unlocking value
Products and pipeline
In our 2013 report, we cheered what appeared to be the start of a more sustained upward trend in the number of new products approved as 2012 US FDA approvals reached levels not seen since 1997. That trend wasn’t sustainable, and in 2013, only 27 new products were approved. As in 2012, first-in-class products, products with novel mechanisms of action and products for orphan indications were well represented in the 2013 class of new molecular entities.
In part, this is because companies are recognizing that new therapies must demonstrate value to succeed with payers and in the marketplace. The good news is regulators are keen to prove they will support novel treatments in areas of high unmet medical need.
In 2013, the FDA refined its process for expediting drug reviews and approvals adding the Breakthrough Therapy Designation program. In March 2014 the EMA launched a pilot program to allow certain medicines to utilize a staggered approval process that authorizes the earlier use of medicines but only in restricted patient populations. (Based on further evidence collection, the drug’s use could be expanded to a broader patient population at a future date.)
It isn’t necessarily the case that the ebb and flow of the market has little impact on biopharma drug development. Improved market conditions in 2013 helped to create the most favorable investment climate in over a decade, and biotech’s commercial leaders responded by reinvesting in their R&D pipelines.
Given the complexity of human disease and the capriciousness of drug discovery, all biopharma companies must continue to use tactics that enable them to create more value from their R&D efforts. As our opening chapter “Perspectives” explains, strategies such as adaptive clinical trials and biomarker approaches allow companies to perform smarter, more cost-effective R&D while simultaneously collecting evidence important to regulators and payers.
A three-year upward trajectory in the number of approved new drug applications (NDAs) and biologic license applications (BLAs) came to a halt in 2013. The FDA approved 25 NDAs, down from 33 in 2012, and just two BLAs, down from six. That said, the 2013 figures were in line with the average number of approvals from 2004 to 2012. Meanwhile, the number of new applications dropped from 41 to 36 in 2013, which is consistent with the historical number of submissions.
Of the newly approved products, many are on their way to blockbuster status, including Biogen Idec’s Tecfidera, an oral therapy for relapsing forms of multiple sclerosis, and Gilead Sciences’ Sovaldi, an interferon-free oral treatment option for hepatitis C. Sovaldi, which was approved in late 2013, generated revenue in excess of US$2 billion in its first quarter on the market in 2014, partly because physicians were so excited about the drug’s upcoming launch they refrained from putting new patients on existing therapies.
In addition to Sovaldi and Tecfidera, noteworthy new products developed by biotechs included Imbruvica (developed by Pharmacyclics and partnered with Johnson & Johnson), Actelion’s Opsumit and Celgene’s multiple myeloma therapy Pomalyst.
Nine of the 27 new products were first-in-class; another nine are treatments for orphan indications. In addition, three products — Imbruvica, Sovaldi and Roche’s Kadcyla — were approved via the FDA’s new Breakthrough Therapy expedited review process. In all, more than 50% of the drugs approved in 2013 took advantage of one of the FDA’s expedited review processes. Meanwhile, the Center for Drug Evaluation and Research (CDER) met its PDUFA goal dates for 100% of the NMEs approved in 2013.
Based on the FDA’s novel new drugs summary, GlaxoSmithKline topped the table for most new drugs approved — five in all, including the approval of its metastatic melanoma therapies Mekinist and Tafinlar and Viiv Healthcare’s HIV treatment Tivicay. GlaxoSmithKline also won approval for its COPD treatment Breo Ellipta, which it developed in conjunction with Theravance.
Biotech partners played important roles in the creation of other big pharma drugs approved in 2013: the cancer drug Kadcyla utilized key technology from Immunogen, and Algeta played such a pivotal role in the development of Xofigo that Bayer HealthCare bought out its smaller partner to get full rights to the prostate cancer medicine.
Cancer treatments accounted for eight of the 2013 approvals, including the only two biologics on the list — Roche’s Gayza and Kadcyla. It was also a big year for infectious disease products, most notably drugs for hepatitis C: the aforementioned Sovaldi and Johnson & Johnson’s Olysio.
FDA product approvals, 1996–2013
Source: EY and FDA.
US product approvals are based on CDER approvals only.