Chasing Cures: Reinventing the Business of Medicine

Current State of Medical Research

• Translating a scientific discovery to patient care currently takes approximately 15 years and costs roughly $1 billion.
• Only 1 out of every 10,000 academic discoveries successfully reaches patients.
• Of the roughly 7,000 human diseases, cures or treatments exist for only about 500.
• The National Institutes of Health (NIH) funds the biomedical research enterprise with approximately $30 billion annually.

Strategic Shift to Open Innovation and Collaboration

• The panel advocates moving from a "golf" (individual competition) model to a "basketball" or "jazz" (team collaboration) model across the industry.
• Accelerating Medicines Partnership (AMP): An unprecedented pre-competitive collaboration involving 10 pharmaceutical companies, NIH, and FDA to address Alzheimer's, type 2 diabetes, rheumatoid arthritis, and lupus.
  • Funding is split 50/50 between NIH and participating companies.
  • All data generated is open access to enable other researchers to utilize findings.
• GSK and Novartis Transaction: A creative deal separating R&D portfolios to consolidate scale in vaccines and consumer health while transferring oncology access to Novartis, without GSK abandoning oncology R&D.
  • GSK retains full R&D operations in oncology but partners with Novartis to optimize commercial access for approved medicines.
• Johnson & Johnson Innovation: Established Janssen Labs to embed innovation clusters globally, providing early-stage biotech companies with access to wet labs, equipment, and business services to lower the barrier to entry for life science startups.

Evolving Role of the Patient

• Patient organizations are increasingly funding research, defining natural disease history, and selecting clinically meaningful outcome measures.
• Patient-Reported Outcomes (PROs): NIH is implementing PROMIS to systematically capture patient perspectives on intervention success, shifting the definition of clinical endpoints from purely biomarker-based to patient-centric.
• PCORI: The Patient-Centered Outcomes Research Institute, funded by health plan taxes and appropriations, manages a network covering 20 million lives to conduct real-world, comparative effectiveness research.
• Data Transparency: Companies like J&J and GSK are moving toward publicizing clinical trial data, arguing that transparency accelerates collective progress despite concerns over competitive disadvantage.

Reinventing Clinical Trials and Data

• Digital and mHealth Integration: GSK envisions using wearable sensors to collect granular, continuous patient data in real-time, replacing infrequent clinic visits for more discriminative efficacy assessment.
• Real-World Evidence (RWE): Mayo Clinic partnered with Optum to create a lab analyzing claims data for over 149 million Americans to replicate large-scale NIH trials (costing ~$300 million) using observational data at a fraction of the cost.
• Francis Collins notes that while randomized controlled trials remain necessary for causality, observational studies are becoming nearly cost-free once infrastructure is established.

Value-Based Healthcare and Sustainability

• Value Equation: The industry must redefine value as (Quality + Safety + Service + Patient-Defined Outcomes) divided by Cost over time.
• Rare Disease Challenges: While orphan drugs offer market exclusivity, high per-patient costs face increasing scrutiny; Mark Rothera suggests a renegotiation with regulators to allow faster approval based on weaker initial evidence, with post-approval real-world data collection to verify utility.
• Innovation Deficit: NIH funding purchasing power has dropped 25% in the last decade, reducing the success rate for young investigators from 1 in 3 to 1 in 6, creating a bottleneck for talent.
• Global Access Responsibility: GSK commits to selling malaria vaccines to developing nations at cost plus a 5% margin, reinvested into neglected disease research.

Forward-Looking Statements (Next 10 Years)

• Bio-electronic Medicine: Monsef Slaoui predicts a shift from chemical molecules to electrical signals delivered via nanochips to modulate organ function through the nervous system.
• Neuroscience Breakthroughs: Francis Collins points to the imminent "Wild and wonderful ride" of the Brain Activity Mapping Initiative to decode neural circuits and treat conditions like Alzheimer's and schizophrenia.
• Precision Medicine: Francis Collins highlights the potential for a universal influenza vaccine, an HIV cure, and cancer treatments tailored to individual tumor profiles.
• Regulatory Evolution: Mark Rothera anticipates a fundamental shift in how "enough evidence" is defined for orphan drug approval to reduce development timeframes.
• Industry Consolidation: Monsef Slaoui and John Noseworthy emphasize that "follow the science" will dictate market positioning, leading to more non-traditional partnerships where companies commercialize medicines they did not discover if it maximizes patient access.