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Exclusive Life Science Feature Package integrity testing revolutionized six years, he says, competition has picked up, and the market is now worth about $10 billion. A similar story is unfolding in the HAE space. According to Basser, CSL held the title of being the only company in the world to offer a product to treat HAE, and things remained that way for 15 years. According to the Orphan Druganaut Blog, there are now a handful of similarly licensed products for HAE, a space valued at about $113.8 million in 2011 and expected to reach $385 million in 2019. "It's interesting how the rare disease space is becoming so crowded," reflects Basser. "We all have to be that much smarter about how we do business, and it puts more pressure on drug development as we are a few players vying for the same patients and skilled personnel." EMBRACE THE SPACE With a feeding frenzy under way in the rare disease drug-development sector, staying ahead of the competition requires a focused commitment. "Considering we are now surrounded by competitors in HAE, we have had to strengthen our focus even more," says Basser. To remain focused, CSL Behring has formed close relationships with patient advocacy groups. These organizations educate patients and family members about a given rare disease: how to seek a diagnosis, and how to gain access to treatment. Some groups are diseasespecific while others are umbrella organizations, such as the European Organization for Rare Diseases (EURODIS) and the National Organization for Rare Disorders (NORD). CSL works with both types of organizations to ensure patients have access to appropriate therapies. "The secondary gain becomes obvious in that we have the therapies the patients need, and we want them to use them," explains Basser. Engaging with patient groups also helps ensure that diseases don't go underappreciated or misdiagnosed, he says. "We understand that having one of these diseases can be isolating, but a misdiagnosis can lead to death. We are working with the groups to improve the rates of accurate diagnosis and the proper choice of treatments." CSL also uses social media to spread its message about treating rare diseases. Basser explains that tapping into online groups enables the company to provide education about treatments as well as information regarding what trials are occurring in a patient's region. The goal is to empower the patients. CSL has also set up specific Web pages and YouTube videos to do the same. "Engaging with patient advocacy groups isn't unique, but I think we do it as well as anyone who competes in our disease areas," says Basser. "We give support to patients beyond getting them to use our product. It's about embracing the entire opportunity. That's why we've been successful." ENLIST PATIENTS New technology uses an optical fuorescence reader and platinum chemistry to noninvasively measure O2 headspace, dissolved oxygen and obtain data to determine shelf life. Get accurate and repeatable results with the touch of a button. Watch Video www.mocon.com/optech.php Patient advocacy groups are also playing a larger role in designing clinical trials. These groups help to make sure the drugs are valuable for the patients from a regulatory and medical perspective as well as from a patient perspective. The challenge with rare diseases is that only a small number of individuals are available to participate in a clinical study. In order for a disease to be qualified in the U.S. as "rare," the afflicted patient pool needs to be fewer than 200,000 individuals. So, a patient advocacy group can really help recruit patients. This has not gone unnoticed by CSL. The company has teamed up with several advocacy groups, including the Alpha-1 Foundation, a not-for-profit Florida corporation cofounded by John Walsh in 1995, for individuals diagnosed with Alpha-1 antitrypsin deficiency, also known as genetic COPD. When someone is diagnosed with Alpha-1, that person is automatically asked to enroll in the Alpha-1 research registry and connected with a clinical resource center doing trials and studies. In fact, the Alpha-1 Foundation was the result of a seven-year Alpha-1 progression study being conducted by the NIH. "Those of us who participated in that study realized the value of participating in clinical research," explains Walsh, who is also president and CEO of the foundation. "As a foundaNovember 2013 Instruments and Testing Services Test. Measure. Analyze. 763.493.6370 Email: info@mocon.com www.mocon.com LifeScienceLeader.com 43