A Conversation with Francis Collins, Director, National Institutes of Health

Diversity and Inclusion in Healthcare Leadership

• The evolution of inclusion efforts has shifted from "tolerance" (conferring superiority/inferiority) and "diversity" (separating people) to "inclusion and diversity" as the current norm, emphasizing that defining people by "otherness" hinders cohesion.
• Despite 50% of the healthcare workforce, clients, and providers being women, only 3% of CEOs are women, representing a regression to pre-existing levels in Fortune 500 rankings.
• Organizations with greater diversity on boards and in leadership consistently deliver better financial results, making inclusion a business imperative rather than just an HR benefit.
• The Institute has launched the "Equality Pledge" to request data transparency from Fortune 500 organizations regarding gender demographics to inform curative strategies.
• McKinsey & Company's 2015 study found that companies in the top quartile for gender diversity were 15% more likely to have financial returns above the national industry median.
• Companies in the top quartile for racial and ethnic diversity were 35% more likely to achieve financial returns above the national industry median.
• Since 2015, companies featured in the McKinsey study have seen only a 2% increase in gender representation and a 1% increase in ethnic diversity on executive teams.
• Horizon Pharma has formed an Inclusion Leadership Council and established three Business Resource Groups (Hispanics at Horizon, HAN, Women at Horizon) to align diversity initiatives with business strategy.
• Horizon CEO Tim Wahlberg signed the CEO Action Pledge for Diversity and Inclusion, joining over 500 other CEOs in the largest CEO-driven commitment to advance workplace diversity.
• Horizon's core belief is that assembling people with distinct technical, professional, and personal differences drives innovation, nimble problem-solving, and faster cures for patients.

NIH "All of Us" Research Program

• The NIH "All of Us" initiative is a 10-year, $255 million program supported by the 21st Century Cures Act aimed at moving from "one-size-fits-all" medicine to individualized health maintenance.
• The program targets the enrollment of 1 million or more participants, representing the largest cohort study ever contemplated by the NIH.
• The program explicitly aims for 50% of participants to be from racial and ethnic minority groups, a significantly higher proportion than typical clinical studies.
• Enrollment launched in May with 115,000 sign-ups in a short timeframe, with 49% of those participants coming from racial and ethnic minorities.
• Participation is voluntary and reciprocal; participants provide health behaviors and biospecimens (including whole genome sequencing) and receive access to their own health data and genomic results.
• The database is encrypted end-to-end and will be accessible to researchers with approved ideas, provided all individual identifiers are stripped.
• The program creates a "pre-consented" platform to accelerate future clinical trial enrollment, potentially offering tens of thousands of patients with specific conditions (e.g., diabetes) for immediate recruitment.
• The program faces challenges regarding trust due to historical research abuses (e.g., Tuskegee), prompting the hiring of a specific engagement officer and the provision of complete openness to build confidence.
• Approximately 50% of those approached agree to participate regardless of prior research experience.
• Whole genome sequencing for all participants is currently actionable for only 2-3% of individuals regarding immediate clinical interventions, though pharmacogenomics utility is expected to grow as data becomes standard in medical records.
• Participants may opt out of the program at any time, including stopping the receipt of emails or data updates.

NIH Brain Initiative and Alzheimer's Research

• The NIH Brain Initiative, a 10-year effort partially funded by the 21st Century Cures Act, aims to map the activity of 86 billion neurons and their 1,000 connections each to understand real-time brain circuitry.
• The first 3.5 years of the Brain Initiative have focused on technology development, with the next five years planned to accelerate toward understanding applications for Alzheimer's, Parkinson's, schizophrenia, autism, and addiction.
• Researchers characterize the original plan as potentially "under-ambitious" given recent technological progress, suggesting the initiative can move faster than initially projected.
• Alzheimer's disease costs the U.S. economy approximately $300 billion annually, a figure projected to reach $1 trillion without effective interventions.
• NIH funding for Alzheimer's research has more than tripled over the last five years, with a shift toward a "systems biology" approach via the Accelerating Medicines Partnership (AMP) to identify new drug targets beyond the amyloid hypothesis.
• While amyloid is confirmed as a driver in genetic cases, NIH is now heavily investigating other proteins like tau and other druggable mechanisms.
• Congress has increased NIH funding by 30% over the last four years, reversing a 20% loss in purchasing power experienced between 2003 and 2015.
• In FY18, NIH funded 1,208 early-stage investigators who had never previously been a Principal Investigator, the highest number ever recorded.
• Despite increased funding, NIH can currently fund only about one out of every four to five grant applications, leaving significant high-quality science unfunded.
• Dr. Collins noted that while the U.S. leads in biomedical research, sustained investment is required to maintain dominance against rising research capabilities in China.

Emerging Therapeutic Technologies

• Cancer immunotherapy is currently achieving cures for metastatic cancers previously considered untreatable, a breakthrough recognized by the Nobel Prize.
• Sickle cell disease is expected to be cured within the next five years via gene therapy and gene editing strategies currently in clinical trials.
• Development is advancing toward new vaccines for HIV/AIDS and a universal flu vaccine.
• Progress is being made in neurological conditions including schizophrenia and autism through better understanding of brain circuitry and function.