The vision of Life Science Leader is to be an essential business tool for life science executives. Our content is designed to not only inform readers of best practices, but motivate them to implement those best practices in their own businesses.
Issue link: https://lifescienceleadermag.epubxp.com/i/201345
Exclusive Life Science Feature COMPETING IN THE RARE DISEASE SPACE BY CINDY DUBIN, CONTRIBUTING EDITOR T "As a foundation, we can recruit for a Phase 3 pivotal study within 6 to 10 weeks. That's why we have been able to get companies like CSL Behring to focus on developing therapies for Alpha-1," says John Walsh, president and CEO of the Alpha-1 Foundation. here are approximately 7,000 different types of rare diseases and disorders affecting an estimated 350 million people worldwide. Because these diseases are so diverse and complex, there are inherent gaps that exist in patient treatment. The Rare Disease Impact Report illustrates that it takes an average of seven years for a patient with a rare disease in the United States to receive a proper diagnosis. On the journey to diagnosis, a patient typically visits up to eight different physicians and receives two to three misdiagnoses. CSL Behring began carving out its niche in the rare disease space about 25 years ago, focusing on plasma-derived proteins (PDPs). PDPs are used to treat rare and serious diseases 42 LifeScienceLeader.com November 2013 that include coagulation disorders such as hemophilia and immune deficiencies, Alpha-1 antitrypsin deficiency, hereditary angioedema (HAE), and hemolytic disease in newborns. At that time, CSL was the lone fish in a small pond. Today, the global provider of plasmaderived and recombinant factor products is up against a handful of competitors in a space estimated to be worth more than $15 billion annually. As previously reported by Life Science Leader magazine (see September 2009 issue, "Lessons Learned From PDP Market Success"), industry experts expect the size of the market to continue to steadily grow and potentially exceed $32 billion per year by 2016. "In the rare disease arena, there is generally a lower cost of development and the promise of patent protection, so it becomes evident that a company can realize a certain degree of profitability under the right circumstances," says Russell Basser, senior VP of global clinical research and development at CSL Behring. "This tends to attract more companies to the space." Consider the hemophilia sector. Basser explains that there was a flurry of recombinant factor development and launches after the contamination issues in the late 1980s with several recombinants hitting the market. Yet, prior to that, little or no technological progress had been made for 25 years. But, in the last five or