MI Summit 2013 - London: Funding Medical Breakthroughs, Enriching Society

Panel Composition and Context

• The session was hosted by Margaret Anderson (Executive Director, Faster Cures/Milken Institute) to discuss medical research funding, innovation, and economic impact.
• Panelists included Sir Mark Walport (UK Chief Scientific Advisor), Gary Nabel (Sanofi CSO, ex-NIH), Ruxandra Draghiakri (European Commission), and Tim Peekman (CEO, UK Biocenter).
• US FDA Commissioner Margaret Hamburg was absent due to a US government shutdown preventing travel.
• The discussion framed biomedical R&D as a critical economic driver and a societal necessity, linking it to the London Summit on financial markets.

Optimism Regarding Scientific Breakthroughs

• Water & Public Health: Sir Mark Walport identified sanitation (separating clean water from waste) as the single most effective historical public health measure.
• Genomics & Microbiology: Walport highlighted the "Power of the Genome" to track infections, monitor antibiotic resistance, and understand human microbial variation.
• Universal Flu Vaccine: Gary Nabel expressed optimism about developing a universal flu vaccine (similar to measles/mumps/rubella) to eliminate annual vaccination needs and pandemic unpredictability.
• Cancer Immunotherapy: Nabel noted rapid progress in genetically modifying T-cells to recognize and eliminate tumors, with end-stage leukemia patients showing complete tumor regression within a week.
• Scalability Challenges: Nabel warned that while immune therapies work, scaling the manufacturing and delivery of genetic modifications from "bottle drugs" to cell therapies remains a major hurdle.
• Pre-competitive Collaboration: Ruxandra Draghiakri cited the Innovative Medicines Initiative (IMI) as a model for accelerating drug development by bridging the "valley of death" between academic research and commercial application.
• Disease Taxonomy: Draghiakri outlined plans to shift disease classification from phenotypic to molecular bases to enable targeted interventions.
• Long-term Data Maturity: Tim Peekman noted the UK Biobank (500,000 participants recruited since 2002) is now yielding insights into chronic diseases like pulmonary conditions that were previously unattainable.
• Technological Cost Reduction: Peekman projected that whole-genome sequencing costs could drop to approximately 500 pounds per genome within the next three to five years.

Economic Perspectives and Strategic Frameworks

• Health as Wealth: The UK government launched a "Innovation, Health, and Wealth" strategy, asserting that health underpins economic prosperity and is a significant sector of the national economy.
• Government Investment: The UK committed £100 million to sequence 100,000 genomes within the National Health Service (NHS).
• Cost Efficiency via Informatics: Walport cited a Scottish diabetes database study that reduced lower-limb amputations by 40% through better data monitoring and adherence to standard testing protocols.
• Regulatory Reform Needs: Walport identified an asymmetry in regulatory incentives where regulators are penalized for allowing harm but not for preventing good, calling for accountability in decisions that block therapies.
• Open Innovation: Nabel stated that the cost of developing a new drug has risen to approximately $5 billion (exceeding previous $1 billion estimates), necessitating industry-wide collaboration to de-risk investments.
• European Funding Scale: Draghiakri reported the first IMI iteration involved €1 billion from the EU matched by €1 billion from industry; the proposed IMI2 (part of Horizon 2020) targets €1.725 billion from each side.
• Ecosystem Development: Nabel noted that biotech pharma generated over 27,000 new jobs in the Boston area last year, emphasizing the need to nurture ecosystems combining academia, pharma, and venture capital.

Patient Engagement and Data Ethics

• UK Biobank Participation: Peekman reported a high recruitment rate for the UK Biobank, driven by altruism and trust in the NHS, with a negligible dropout rate despite data privacy incidents elsewhere.
• Informed Consent Models: Participants in the UK Biobank understood they would not receive individual feedback on genetic risks, consenting to the "benefit of future generations" model.
• Real-World Evidence: Walport and Nabel agreed that observing populations in real-time post-launch (via informatics) provides more data than traditional Phase III trials.
• FDA Patient Perspective: Anderson described an FDA initiative involving 1,000+ patients with narcolepsy (via Skype) to inform reviewers on the lived experience of risk and benefit, aiming to humanize the regulatory process.
• Rare Disease Consortiums: Draghiakri highlighted the Rare Disease International Research Consortium, which aims to provide diagnostics for most rare diseases and 200 new therapies by 2020, heavily mobilized by patient organizations like Genetic Alliance.
• Patient Advocacy History: Nabel recalled early turbulent relationships with AIDS activists (e.g., ACT UP) evolving into strategic partnerships where advocates contributed scientific and clinical trial insights.

Identified Concerns and Obstacles

• Data Privacy vs. Science: Peekman warned that legal and ethical frameworks (e.g., data privacy laws) are failing to keep pace with medical science, citing the halted Swedish national cohort study as an example of lost opportunity.
• Implementation Inertia: Nabel expressed concern that effective innovations often fail to reach patients due to healthcare system inertia, lack of physician education, and arduous adoption pathways.
• Aging and Lifestyle Diseases: Walport identified major unmet needs stemming from aging populations, including dementia, malnutrition (obesity, starvation, micronutrient deficiencies), and infectious diseases.
• Regulatory Bottlenecks: Mark suggested that regulation costs have "ramped up astronomically," questioning the necessity of certain costs and the need to utilize informatics to reduce development expenses.
• Integration of Sectors: The panel emphasized the necessity of aligning public funding agencies (e.g., Wellcome Trust, MRC, NIHR) to create a continuum from basic research to translational medicine.