London Summit 2015 - Tech Rx: Harnessing Big Data to Cure Disease

Event Context & Strategic Vision

• The session was hosted by Margaret Anderson, Executive Director of Faster Cures (a Milken Institute center), focusing on leveraging big data to accelerate disease cures.
• The central narrative has shifted from the theoretical "magical day" of personalized medicine to observable benefits driven by big data utilization.
• The core ethos of Faster Cures is to unify all ecosystem stakeholders—industry, government, patients, and researchers—to move biomedical research efficiently.

European Perspective: Economics, Demographics, and Real-World Data

• Speaker: Mary Baker, European Brain Council.
• Europe spends approximately €800 billion annually on brain diseases, a figure exceeding combined spending on cancer, diabetes, and cardiovascular diseases.
• Current data from the European Medicines Agency (PSURs) is described as "non-comprehensible," prompting a shift toward utilizing real-world data (RWD).
• Demographic trends indicate that two-thirds of all people who have reached age 65 in human history are currently alive, exacerbating the challenge of comorbidity and polypharmacy.
• The "health is wealth" mantra emphasizes that while interventions are costs, their societal outcomes (e.g., returning patients to work) define them as investments.
• Preventing deterioration is identified as a key strategy for an aging population, contingent on gathering standardized, trusted big data.

Mayo Clinic: Data Infrastructure and Clinical Utility

• Speaker: Gianrico Ferrugia, VP of Mayo Clinic and CEO of Mayo Clinic Florida.
• Mayo Clinic operates a $9 billion not-for-profit ecosystem employing 60,000 people, investing $900 million annually in research and education.
• Four major areas for big data application were identified: comparative effectiveness, behavior/policy, regional treatment variations, and treatment response.
• Case Study: Analysis of millions of data points revealed a critical age threshold at 65 for choosing blood thinners, where the cost-benefit analysis shifts between warfarin and newer agents.
• The primary challenge is no longer data volume or velocity, but veracity; ensuring data quality without over-cleaning data that loses context.
• A paradigm shift is required to determine whether big data informs clinical decisions directly or serves solely as a hypothesis generator for Randomized Control Trials (RCTs).
• Initiative: OptumLabs, a partnership with UnitedHealth Therapeutics and 20+ partners (including Harvard, Novartis, Merck), combines 150 million claims, 37 million health records, and 38 million consumer records.

Wellcome Trust: Privacy, Trust, and Public Policy

• Speaker: Bethany (Wellcome Trust).
• The Wellcome Trust funds £800 million annually in biomedical research, viewing big data as a tool to ask questions impossible to address via traditional trials (e.g., in pregnant women or rare diseases).
• Success Story: Analysis of GP records definitively disproved the link between MMR vaccines and autism.
• Trust Crisis: Public trust in the UK was damaged by the failed launch of the "Care.data" program due to poor communication and lack of safeguards.
• Rebuilding trust requires complete transparency regarding data usage, commercial ownership, and the implementation of robust opt-out mechanisms.
• Clinical trial data is deemed easier to manage due to existing consent frameworks, whereas GP records require new governance to allow commercial access.

Pfizer: Industry Application and Regulatory Challenges

• Speaker: Albert (Pfizer).
• Pfizer is investing $7.5 billion in R&D, viewing big data as essential for moving from prolonging life to curing diseases like cancer.
• Four Drivers: Genomic sequencing, digitization of medical records, wearable device technology, and social media lifestyle data.
• Case Study (Lupus): Collaboration with 23andMe combines DNA records and lifestyle data from 5,000 consented participants to identify subgroups where a pipeline drug may be most effective.
• Case Study (Lung Cancer): Big data identified non-smokers with ALK gene mutations who respond extremely well to specific targeted therapies, despite lacking traditional risk factors.
• Compliance Risk: Pharmacovigilance regulations require reporting every adverse event within 24 hours; integrating unstructured big data creates a compliance nightmare for tracking side effects.
• Legal Obstacle: The European Court decision prohibiting data transfer to the US creates a significant barrier for global research collaboration.

UK Government: Infrastructure and Accelerated Access

• Speaker: Nicola (Office for Life Sciences, UK Dept. of Health).
• The UK is building a "data for research" ecosystem integrating NHS records, the 100,000 Genome Project, and biobanks to support RWD studies.
• Accelerated Access Review: Aims to enable conditional reimbursement where products reach patients earlier, with pricing determined later based on real-world evidence collection.
• The government plans to increase patient access to personal records via smartphones to facilitate integration with data from wearables (e.g., Fitbits).
• The strategy aims to generate economic growth by making the UK a hub for health analytics and digital health technologies.

Challenges: Data Quality, Wearables, and "Normal" Baselines

• Wearable data (e.g., Fitbits) is currently insubstantial in isolation; value is derived only when linked with clinical data to identify patterns (e.g., efficacy of obesity drugs in exercising populations).
• Risk: Linking diverse data sets increases the probability of individual re-identification, necessitating stronger anonymization protocols.
• Physician Anxiety: Real-time monitoring of retirement home residents via sensors revealed high rates of ectopic heartbeats, causing physician burnout due to an inability to distinguish noise from pathology.
• The industry must shift from "large data" to "big data" that provides a 360-degree patient context, requiring new algorithms to sift information without interfering with daily life.
• A critical friction point exists between patient autonomy and family surveillance rights regarding the health data of elderly relatives.

Patient Engagement, Education, and Societal Shifts

• Behavioral Insight: A trial showed that patients using apps to track blood pressure achieved better outcomes than those receiving standard GP appointments alone.
• Communication Gap: Society remains largely unaware of precision medicine benefits; experts warn that ignoring public understanding risks backlash similar to the Luddite movement.
• Patient Empowerment: Patients at Mayo Clinic have expressed a strong desire to access their own genomic data immediately, often knowing more than their providers about their conditions.
• Physician Education: The burden is shifting to educating healthcare providers on big data tools, as patients are increasingly more informed than their doctors regarding their specific disease markers.
• Strategic Reframing: Technology should promote "wellness" rather than "sickness" to avoid stigma and increase adoption among younger generations.
• Legal Evolution: The right for patients to know their diagnosis was only legally established in 1975, indicating a slow institutional adaptation to modern data transparency.

Forward-Looking Statements and Action Items

• Infrastructure: The industry must build a common data infrastructure capable of moving information as efficiently as the financial sector.
• Outcome Measurement: Future metrics must focus on tangible patient outcomes (e.g., return to work) to prove the economic value of medical interventions beyond simple cost.
• Collaboration: Success depends on industry, government, charities, and researchers pooling diverse data sets to solve complex disease mechanisms.
• Optimism vs. Reality: While the technology and computing power exist, legal barriers (like data transfer restrictions) and the need for societal buy-in remain significant hurdles.
• Long-term Goal: The ultimate objective is to democratize access to cures and prevention strategies globally, ensuring benefits extend beyond the current room or continent.