From The Cutting Room Floor: December 2016 Issue
What 13 Life Science Trendsetters Expect For 2017 And Beyond
When Life Science Leader magazine asked for executive-level participation for the signature 2017 Outlook issue published in December 2016, we were overwhelmed with the response. This year’s feature articleinvolved 13 executive thought leaders! One of the problems with that amount of participants is it makes it impossible to publish all of their comments in one print edition. As a solution we decided to create four Cutting Room Floor articles so we could share all of this thought leader knowledge. In part two of this series we pose the question, “What are you doing at your organization to address/capitalize on certain trends?” We hope you enjoy this supplement to the Life Science Leader magazine’s December 2016 cover feature. In addition, be sure to check out part 3 of this trend setter series: Shire CEO and Novartis CMO Weigh In On Trends For 2017: Trendsetter Series Part 3 of 4.
What are you doing at your organization to address/capitalize on certain trends?
Mark Alles, CEO, Celgene
At Celgene we hold ourselves to the highest standards of driving medical innovation that delivers meaningful and measurable clinical benefit and value to patients, healthcare systems, and society. This effort begins by consistently reinvesting approximately 30 percent of our annual revenue toward R&D. Celgene’s medicines have become the backbone of treatment strategies that have turned multiple myeloma from an incurable blood cancer into a chronic disease for many patients. Today, our pipeline contains nearly 50 compounds, most with first-in-class potential that target unmet needs in approximately 100 different disease areas. Such a commitment to medical progress must go hand-in-hand with ensuring patient access, which we do through our patient assistance programs, access to clinical trials, and support for education and charitable programs. Since inception, more than 50,000 patients have been helped by our patient support program, and several hundred thousand have participated in our clinical trials.
Christopher P. Austin, M.D., Director, National Center for Advancing Translational Sciences (NCATS) at the NIH
NCATS is approaching patient engagement as a scientific problem to determine best practices to optimize patient involvement in accelerating translation (i.e., the process of turning observations in the laboratory, clinic and community into interventions). An ongoing effort at NCATS aims to create a generalizable paradigm to address global epidemics via rapid collaborative screening of drugs from the NCATS Pharmaceutical Collection (i.e., a compendium of approved and investigational molecular entities that might be repurposed). Recently, NCATS researchers identified compounds that potentially can be used to inhibit Zika virus replication and reduce its ability to kill brain cells. While we await the development of effective vaccines, our identification of repurposed small molecule compounds may accelerate the translational process of finding a potential therapy.
Jean-Jacques Bienaimé, CEO and Chairman, BioMarin
For almost 20 years, BioMarin has been building on its expertise of discovering therapeutics for rare monogenetic diseases. Since the beginning, we have worked with patient groups and key opinion leaders (KOLs) to understand the natural history of a disease and discover interventions. In rare diseases there is very little information about disease progression, and natural history studies need to be completed to understand what clinical outcomes a drug intervention will need to prove scientifically. Rare disease patient groups can potentially speed up the drug development process by identifying the patient endpoints that are important scientifically and clinically, as well as personally.
Stanley Crooke, M.D., Ph.D., Founder, Chairman, CEO, Ionis Pharmaceuticals
The only answer to managing costs in our industry is to create disruptive new technologies that enhance production. This has been one of our goals through the 27 years we have been developing antisense technology. As evidence of our productivity around antisense technology, I point to the fact that at Ionis we have one drug in development for every 11 people who work here. The second component of the Ionis strategy is maximizing value by focusing the technology on novel targets that may fundamentally alter the course of diseases. Nusinersen, an investigational antisense oligonucleotide for the treatment of spinal muscular atrophy (SMA), is an example of this strategy in action.
Ruud Dobber, Ph.D., President AstraZeneca U.S., EVP AstraZeneca North America
Personalized approaches to care and identifying the right patient for the right treatment is one way to demonstrate value. AstraZeneca is deeply invested in an increasingly personalized approach to treatment, including development of next-generation biomarker companion diagnostic tools. We believe our achievements in immuno-oncology will hinge on our ability to successfully identify which therapy may be best for each patient. Nearly 90 percent of the projects in our oncology pipeline have a personalized healthcare strategy, and many will be launched with a companion diagnostic. We also recently launched an integrated genomics approach. Through collaborations and the creation of a genome center we intend to leverage information from up to 2 million genome sequences, including over 500,000 from AstraZeneca clinical trials.
John Maraganore, Ph.D., CEO and Board of Director Member, Alnylam Pharmaceuticals
It’s pretty simple: We need to continue to focus on innovation and product differentiation. At Alnylam our focus remains on discovery, development, and commercialization of RNAi therapeutics that have the potential to transform the treatment of disease. We have developed a set of access-related guiding principles as we move toward the launch of our first medicines. As we commercialize RNAi therapeutics, Alnylam aims to advance performance-based pricing and reimbursement schemes and work with payers and healthcare technology assessment (HTA) companies to put these innovative structures in place. We also aim to grow our business through continued innovation, and believe we can achieve our objectives for shareholders while maintaining responsible positions on the value of our medicines.
Vas Narasimhan, M.D., Global Head of Drug Development and Chief Medical Officer, Novartis
One of our main focuses at Novartis is pursuing clinical advancements in chronic disease areas causing the highest numbers of fatalities annually. Four main types of non-communicable diseases (NCD) – cardiovascular disease, respiratory disease, cancer and diabetes – account for 82 percent of all NCD deaths annually. The number of patients suffering from these illnesses is projected to grow. We are also pursuing treatments in areas of broad unmet need where patient populations are large and advancements have been few (e.g., ophthalmology, immunology) in an attempt to drive a major impact for large numbers of patients. In addition, we are also working to advance and grow our portfolio of biosimilars to increase access to lower-cost standard of care treatments.
Flemming Ornskov, M.D., M.P.H, CEO and executive committee member, Shire
An important lesson we can learn from technology companies is how much of their success resides with developing innovative products with strong consumer connectivity. As there is intimacy between rare disease patients and their treatments, we have that same opportunity. At Shire, we place great importance on collaborating with and servicing patient and physician communities. One of our collaborations involves the hemophilia community. Given that prophylaxis treatment must be individualized, we developed myPKFit, a validated software medical device that offers pharmacokinetics (PK) estimates and regimen personalization for our hemophilia therapy, Advate. We’ve launched another integrated patient service platform to improve access for Xiidra (for dry eye disease) to assist patients through the reimbursement and co-pay process.
Michel Vounatsos, EVP and Chief Commercial Officer, Biogen
In MS, Biogen has embarked on a journey to better understand the needs of key stakeholders, and find new ways to build and deepen our relationships toward a common goal of improving patient outcomes. Beginning with the patient journey, we built an engagement model that focuses on providing a seamless and differentiated experience with Biogen, regardless if people engage with us in person or digitally. This is the foundation of an integrated four-pillar approach, which includes portfolio strategy, commercial excellence, medical leadership, and building trust and value. An example of this approach is our recently launched MS Paths initiative, which is creating a standardized, high-quality data repository from a very diverse real-world patient population in the U.S. and EU.
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