What does it mean to be innovative in the pharma industry?
It's common to read about the lack of true innovation in the pharmaceutical industry, but many of the current trends suggest a highly innovative, productive atmosphere -- and the numbers tell the tale.
In 2013, 27 new molecular entities (NMEs) were approved -- and one-third of them were first-in-class therapies, a hallmark of innovation. In addition, FDA programs designed to expedite the approval process facilitated the approval of nine drugs with orphan drug designations -- medications developed to treat diseases that affect 20,000 or fewer individuals. Twenty-five of the 27 NMEs approved in 2013 were designated for some type of fast track development.
Overall, 88% of newly approved molecules only had to undergo one cycle of review. Of the NMEs approved in 2013, 74% were approved first in the United States before being approved outside of the US.
As it turns out, 2012 was an even more productive year than 2013. A total of 39 NMEs were approved, with a broad range of therapeutic applications, ranging from stroke prevention (Equilis-apixiban); to parenteral nutrition for adults with short bowel syndrome (Gattex-teduglutide); to a new treatment for overactive bladder (Myrbetriq-mirabegron).
And so far in 2014, 18 NMEs and biologic license applications (BLAs) have been approved by FDA. Some of the most widely publicized approvals include Farxiga (dapglifozin) for treatment of type 2 diabetes, Zontivity (vorapaxar) to reduce the risk of heart attack and stroke; and Entyvio (vedolizumab) for treatment of severe ulcerative colitis and Crohn’s disease in adults.
A call for transformative innovation
Writing in Forbes in August 2012, industry expert Annie Chang suggested that transformative innovation is needed in the pharmaceutical industry. She encapsulated the problem as follows: “Our health landscape has shifted from infectious diseases to complex lifetime conditions. When it comes to heart disease and diabetes, there is no one single pathogenic culprit to target. Pharmaceutical companies must respond beyond the formula of infectious diseases.”
Chang’s point is valid, but most pharmaceutical companies that are developing therapies for the treatment of diabetes, for example, have patient support programs, just as they do in other therapeutic areas. It is possible to be innovative in communications and to address a problem as large and complex as diabetes from a variety of angles, but the real innovation -- the game-changing activity -- is still happening in research labs, especially in collaborative situations between academic and the pharmaceutical industry.
Progress driven by development
The Pharmaceutical Researchers and Manufacturers of America (PhRMA) makes a case for the pharmaceutical industry as one of the most innovative in the world. According to PhRMA, there are currently more than 5,000 drugs in development. In addition, investment commitment is strong, with over $500 billion invested in R&D since 2000.
It’s not just a numbers game -- it’s about transforming what it means to be alive by providing a therapeutic resources to address as many medical needs as possible. PhRMA makes the point that the endgame is developing therapies that help patients live longer, healthier lives. To that point, childhood cancer survival rates have increased from 58% in 1970 to 83% right now, and the death rate from HIV/AIDS has decreased 85% since 1995. Meanwhile, the death rate associated with heart disease declined 30% between 2001 and 2011, while the death rate from cancer declined 20% between 1999 and 2006.
Many people would consider these results a byproduct of innovative pharmaceutical research and development. And these numbers are meaningful, especially to the individuals and families, who are affected by the various acute, chronic and atypical diseases that are inevitably a part of the human condition.
- FDA New Molecular Entity Approvals for 2013
- Pharmaceutical Researchers & Manufacturers of America The Biopharmaceutical Industry is Driving Unprecedented Progress in Drug Discovery and Development
- Forbes Three Imperatives to Transform Innovation in Pharma