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 "It's interesting how the rare disease space is becoming so crowded. We all have to be that much smarter about how we do business, and it puts more pressure on drug development." Russell Basser, senior VP of global clinical research and development, CSL Behring tion, 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." One of those therapies is Zemaira, an Alpha-1 proteinase inhibitor developed about 10 years ago by CSL Behring. This augmentation therapy replaces the missing protein in people who have Alpha-1 deficiency. CSL asked the Alpha-1 Foundation to participate in the design of the clinical trial protocols and to be involved with FDA discussions in getting the Zemaira trial design approved. "Having a patient advocacy group at the table brings a completely new perspective that the FDA wouldn't have otherwise," says Walsh. "We actually challenged a reviewer on the number of bronchoscopies that would be given and the number of patients to whom it would be given. And the FDA backed off. The importance of being at the table with the sponsor of a drug trial adds tremendous value because the FDA gets a completely different perspective on the potential impact of the clinical protocol." Zemaira was ultimately approved by the FDA as an injectable therapy for patients suffering from Alpha-1 and is among a handful of such therapies. "It is critically important for individuals afflicted with rare diseases to be involved with clinical research," says Walsh. "Without the patients, the research won't get done. As a whole, I think the pharmaceutical industry is realizing the importance of getting patients involved with clinical studies." In fact, the FDA is promoting opportunities for patient advocacy earlier in the policymaking process than has been the case historically. The FDA Safety and Innovation Act (enacted in mid-2012) mandates the involvement of patient representatives in roles beyond those of the advisory committees. Draft procedures for patient involvement were made public in September. Both Walsh and Basser agree that the mandate will be embraced by patients and will accelerate therapies. "In the future, we will need to think more about how we engage patients in the design of a trial program," says Basser. 44 LifeScienceLeader.com November 2013 EDUCATE THE TRIAL SITES Just as patients are essential to the success of a clinical trial, so too are the investigator sites. Basser explains that it is important that the sites are engaged and that a coordinated effort be made to ensure the investigators really understand the complicated protocols. "As our success and portfolio have grown over the years, we have had to learn to engage the sites in a much more intimate way." This, he adds, is necessary because every patient counts in studies in rare disease. "We have many studies where we expect each site to recruit only one or two patients. This has a number of implications. For starters, we need to work closely with our study investigators to ensure they identify every potentially eligible patient in their clinics. We must also cast a wide net for patients and often work with investigators who have a lot of expertise in the particular rare disease but limited experience in clinical trials. Finally, there is a very steep learning curve for the site teams as the first patient they treat might be the only one." So, CSL has learned to stay close to the study coordinators and investigators to get patients into the trials and make sure the quality of the data is adequate. For example, when investigators sign up for a trial, they must learn and understand the process and the protocol, but ultimately, the sponsor has a responsibility to ensure the trial is conducted according to good clinical practice guidelines and that the data is accurate. That's especially important considering the sponsor wants to use that data when getting approval from a federal regulatory agency. Basser explains that if investigators are going to sign up for a trial, it's up to them to learn and understand all aspects of the trial, "But in the end, we're the ones who have the most in the game. If they don't follow the protocol appropriately, we get data that we can't use. Then, the product might not get licensed. It's our responsibility to ensure investigators are trained adequately."