Pharma Landscape 2020: 2013 IQ Symposium Video and Report
Pharma Landscape 2020: Key Issues Informing the Future of Pharmaceutical Development
The third annual IQ Consortium symposium, “Pharma Landscape in 2020”, was held November 19, 2013 in Alexandria, Virginia. In line with the IQ mission, the event brought participants together to address three key issues influencing the future of the pharmaceutical industry: open innovation, globalization, and patient-focused personalized medicine. In addition to an exceptional faculty of speakers and moderators from within and outside of the pharmaceutical industry, the Symposium featured breakout sessions on the symposium subtopics and live audience polling.
The Pharma Landscape 2020 Highlights Video reviews key moments from the day and reflections on the Symposium's theme from several IQ participants, including the IQ Chair and Vice Chair. We are also making videos of the plenary session talks available for viewing. You can watch these by clicking on the video thumbnails below, or go to the IQ Consortium Vimeo channel to see all videos: http://vimeo.com/channels/iqconsortium.
Keynote Address: John Crowley
John Crowley is chairman & CEO of Amicus Therapeutics. His story provided the inspiration for the major motion picture, "Extraordinary Measures".
After two of his children were diagnosed with Pompe’s disease, a rare and fatal neuromuscular disorder, John Crowley founded a company focused on developing a treatment for the disease. Over the course of more than a decade, Mr. Crowley has continued to lead the efforts to develop a treatment for this disease and has experienced firsthand the need for risk taking and innovation within pharmaceutical research.
In the development of treatments, Mr. Crowley remarked that one sells hope and promise tempered with reality and good science. The high chance of failure cannot prevent industry from moving forward. It is impossible to avoid taking risks during research, as most drugs under development will not work or will not work as originally intended. Further, the pharmaceutical industry cannot settle for small, incremental steps but must attempt to move forward with “swing for the fences” ideas.
Along his journey, Mr. Crowley realized the need for hope, humility, and persistence. Scientists and patients must have hope for progress in order to overcome challenges each day, and must recognize that today’s work is laying the foundation for iterations of success. Humility and perspective have the power to heal, to enhance, and to save lives. Persistence is required to overcome the clinical and business setbacks that are common throughout R&D.
Mr. Crowley noted his experience has been a long process of trying, failing, adjusting course, and trying again. Research is a fight to bring needed treatments to patients, and Mr. Crowley emphasized the importance of maintaining a sense of urgency and fire.
Dr. Christopher Haskell leads Bayer’s U.S. Science Hub, based in the company’s U.S. Innovation Center located at Mission Bay, San Francisco.
The pharmaceutical industry is facing significant challenges today, and is increasingly looking to new models of interaction and collaboration, including aspects of open innovation. Dr. Chris Haskell discussed two primary forms of open innovation in his talk, offering examples of each within the pharmaceutical industry.
The first approach to open innovation addresses common challenges in pre-competitive space. Examples include the IQ Consortium, Innovative Medicines Initiative (IMI), TransCelerate, and Enlight Biosciences.
The second approach is through strategic partnerships among stakeholders such as pharmaceutical and biotechnology companies, academia, and others in regions known as “Innovation Ecosystems”. This format is especially common in the area of early discovery.
While many open innovation initiatives are still relatively new and frequently focus on concepts that apply to the early stages of pharmaceutical development, Dr. Haskell emphasized that open innovation can be applied throughout the entire R&D process.
Patient Focused Personalized Medicine
At Pfizer Oncology, Dr. Sweetman leads the US Medical Affairs team for Thoracic and Hematologic Malignancies. His focus over the last few years has been dedicated to development of Xalkori (crizotinib) which culminated in the accelerated approval by the FDA on 26 August 2011.
Patient focused personalized medicine is a multi-faceted concept that that has become increasingly common in recent years. It is viewed by IQ Consortium participants as an approach that encompasses precision medicine, proactive care, stratified medicine, individualized medicine, and targeted therapy, and it holds promise for individuals facing a variety of diseases. Dr. Sweetman and Richard Heiman’s on-stage discussion about Richard’s decade-long experience battling cancer brought the concept of personalized medicine to life for IQ Consortium participants. During their dialogue, Richard related the story of his initial diagnosis with lung cancer, subsequent treatments, remission, and numerous recurrences. He and Dr. Sweetman discussed how enrollment in a clinical trial for Xalkori, a medicine intended to treat a subset of cancer patients with specific genes – a subset that included Richard – offered new hope for Richard, and has significantly prolonged his life. Dr. Sweetman and Richard also discussed the challenges of participation in clinical trials, and emphasized the need for awareness among patients and doctors of the ever-evolving field of personalized medicine.
Gabor Fari is a founding team member of the Health & Life Sciences Industry Unit at Microsoft, starting in 2006. He has been involved with the biopharmaceutical industry throughout his professional career. Gabor plays a key role in defining and executing Microsoft’s Life Sciences solutions and business strategy.
Mr. Fari opened his presentation stating that globalization is more than just technology and requires cultural changes to be successful. Pharmaceutical companies have often been late adopters of certain new technologies, a sentiment that polling results showed was reflected by a large percentage of audience members.
“The cloud” and its impact on globalization was the main focus of Mr. Fari’s presentation. The cloud allows for seamless connectivity and sharing of data and computing resources, independent of their location, operating system, or device. The security of data stored in the cloud and moving to/from the cloud in maintained through the use of standards and various encryption technologies. Cloud computing is the new paradigm and will affect the way the pharmaceutical industry conducts business and operations in the future.
This year’s symposium featured six breakout sessions - three for each subtopic (Open Innovation, Personalized Medicine, Globalization), with a CMC or Biology perspective. Participants engaged in discussion about the challenges each posed to the pharmaceutical industry, and brainstormed possible solutions IQ could explore.
Open Innovation Breakout Sessions
Chemistry, Manufacturing and Control Breakout
Challenge to Innovation: The short-term focused societal norm.
Potential Solutions to Facilitate Innovation: Establish an IQ postdoctoral fellowship program in collaboration with the National Institute of Standards and Technology (NIST) and/or the National Center for Advancing Translational Sciences (NCATS) at NIH, to create an open cloud space for IQ to exchange ideas, knowledge and data, to define and raise awareness of open innovation, to collaborate and drive change proactively.
Challenges to Innovation: Legal barriers and IP concerns, company bureaucracy, budgets, identification of the right individuals to implement ideas, risk-averse culture, and the fear of lowering barriers for competitors.
Consequences if Challenges are Not Addressed: Slowing of drug development processes, increased costs, and contraction of the industry leading to a reduced ability to address the needs of patients.
Potential Solutions to Facilitate Innovation: Better define the pre-competitive space, building strong business case for open innovation initiatives, and obtain greater managerial buy in to expedite the decision-making process.
Personalized Medicine Breakout Sessions
Chemistry, Manufacturing and Control Breakout
Challenges to a Personalized Medicine Approach to Drug Development: An accelerated timeline, and the need for a shift from high production to smaller scale manufacturing processes.
Consequences if Challenges are Not Addressed: Validation failures and approval, commercial, and post-approval issues.
Potential Solutions to Facilitate a Personalized Medicine Approach: Shift in mentality toward defining target product profiles more in line with personalized medicine, development of small-scale predictive modeling tools and use of small scale processes, design of more efficient experiments, development of better methodologies for accelerated production, and sharing of manufacturing capabilities. From a regulatory perspective, more frequent and productive exchange with regulatory agencies and adaptation of regulatory review and approval approaches will be key to the management of these challenges.
Challenges to a Personalized Medicine Approach to Drug Development: Potentially profit margins due to smaller patient populations; insufficient knowledge of certain diseases, phenotypes and the magnitude of benefit from treatment; difficulty in identification of markers and target populations, and complexities in clinical trials.
Consequences if Challenges are Not Addressed: Costly drug development, treatment resistance, and difficulty in retaining good patient candidates.
Potential Solutions to Facilitate a Personalized Medicine Approach: Increase understanding of disease mechanisms and biology; consider biomarkers in earlier stages; and develop better animal models, negative predictive biomarkers, and other disease modeling tools. The discussion group proposed the establishment of a Translational Medicine/Science Leadership group, sharing of early development techniques to identify good proof-of-concept study designs, sharing of lessons learned from failures, broader collaborations within IQ and with organizations such as NCATS and the Critical Path Institute.
Globalization Breakout Sessions
Chemistry, Manufacturing and Control Breakout
Challenges Associated with Globalization: Variance in regulations, customer expectations, manufacturing requirements and IP concerns in different regions. Communication and knowledge transfer globally represent another significant challenge.
Consequences if Challenges are Not Addressed: A potential loss of competitive advantage – higher costs, product delays, and an increase in quality concerns about the supply chain.
Potential Solutions to Challenges Associated with Globalization: To address these issues, participants stressed the importance of global harmonization of regulatory requirements as well as formulation and packaging practices. IQ should try to recruit more member companies and individual participants based in other countries, establish more collaborations with worldwide organizations, share best practices in different world regions, and develop strategies for the effective use of the Cloud.
Challenges Associated with Globalization: Increased costs, communication difficulties, variations in government regulation, material import issues, and IP protection concerns.
Consequences if Challenges are Not Addressed: The industry will miss valuable opportunities to do business and to benefit patients.
Potential Solutions to Challenges Associated with Globalization: Sponsorship of international educational programs and increased relationship building and collaboration were presented as activities that IQ is uniquely positioned to undertake to enable globalization.