Innovation for All: Strategies for Maximizing Impact | Future of Health Summit 2024

Parkinson's Biomarker Breakthrough (Fox Foundation)

• In late 2023/early 2024, the Fox Foundation announced the discovery of a biomarker capable of detecting alpha-synuclein in living Parkinson's patients with 90% accuracy via spinal fluid analysis.
• This capability allows for the identification of the disease biology 10 years before the emergence of clinical symptoms, specifically in cohorts with genetic risk factors or REM sleep behavior disorder.
• The finding resulted from a 15-year partnership between the patient community and philanthropists, culminating in a longitudinal natural history study.
• The study initially enrolled 600 participants and has expanded to approximately 4,000, representing the largest deeply phenotyped group of patients and controls funded by philanthropy rather than government grants.
• Approximately $750 million was invested in the study; the organization mapped a $1 billion potential value from follow-up questions generated by the assay.
• Poor smell was identified as the highest predictive risk factor for a positive biomarker test result.
• The Fox Foundation is actively engaging with 150 identified risk genes (originally starting with synuclein) to de-risk drug development, moving away from reliance solely on animal models.

Population Genetics and Drug Discovery (Amgen)

• Amgen's Reykjavik site, acquired from Decode Genetics in 2012, utilizes Iceland's isolated population and extensive medical records to identify protective and disease-causing alleles.
• The "Gipper" gene (GIPR) discovery revealed that a specific mutation protects against obesity, leading Amgen to develop a GIPR inhibitor currently in Phase 2 clinical trials for chronic weight management.
• The company confirmed that its Phase 1 data for the GIPR inhibitor showed no effect on bone mineral density, addressing prior investor concerns regarding bone side effects.
• Amgen's Repatha (PCSK9) and an emerging Lipoprotein(a) therapy were developed based on natural human genetic variations that protect against cardiovascular disease.
• Amgen is shifting focus toward scalable manufacturing at the molecule design stage, favoring bispecific T-cell engagers and small molecules over complex ex vivo gene therapies (like CAR-T) to improve global access.
• Prevention is identified as the largest scientific gap, with >95% of private sector expenditure currently directed toward treating disease rather than preventing it.

Medical Device Access and Patient Education (Abbott)

• Abbott reports that only 10 million of the estimated 500+ million global diabetes sufferers use continuous glucose monitors (CGMs), highlighting a massive access gap.
• In Stockton, California, Abbott launched a community partnership to integrate social services with healthcare to improve diagnosis and CGM adoption among underserved populations.
• For Parkinson's patients, Abbott developed the NeuroSphere Virtual Clinic, enabling remote programming of deep brain stimulators (DBS) to reduce the need for in-hospital visits.
• A significant gender disparity exists in DBS adoption, with men being twice as likely to receive the therapy compared to women.

Payment Reform and Healthcare Systems (CMMI/Government)

• The Centers for Medicare & Medicaid Services (CMS) Innovation Center is piloting an Outcomes-Based Payment Model for Sickle Cell Disease cell and gene therapies, which cost millions per patient.
• Liz Fowler (CMMI) noted that 90% of Parkinson's patients are on Medicare, yet reimbursement for physical therapy remains difficult because the progressive nature of the disease conflicts with "improvement" billing standards.
• Current CMS models are largely voluntary (approx. 90%), leading to provider attrition when financial incentives are not immediate; there is a strategic push toward mandatory participation models.
• CMMI is exploring a Quality Pathway to shift payment metrics from pure cost reduction to patient-reported outcomes and holistic quality measures, moving beyond the 1965 fee-for-service framework.
• The "Sickle Cell" access model aims to negotiate terms where payment is tied to the removal of hospitalization episodes, transforming a curative therapy from a financial burden into a long-term savings opportunity.

Systemic Barriers to Innovation

• Clinical Delivery Gaps: In Parkinson's, only 25% of patients ever see a specialist, while 50% remain in primary care where novel therapies (e.g., brain-directed inhalers) often fail due to complex administration requirements (e.g., grip strength needed to use inhaler).
• Regulatory Misalignment: There is no current billing code for injecting therapies directly into the brain, blocking the delivery of advanced neuro-degeneration treatments.
• Car T-Cell Access: Despite manufacturing improvements, only 20% of eligible cancer patients receive CAR-T therapies due to provider confusion regarding REMS (Risk Evaluation and Mitigation Strategies) and lack of community oncology support.
• Prevention Gap: The vast majority of scientists study treatment endpoints; precision prevention (identifying why some populations never develop disease) remains a neglected area of research and investment.

Future Strategic Directions

• Debbie Brooks advocates for strategic scaling of philanthropy to fund biology-driven mapping of neurodegenerative conditions (Parkinson's, Alzheimer's) where government funding is limited.
• The panel consensus emphasizes that innovation in science is no longer the primary bottleneck; the critical challenge is systemic delivery, access, and reimbursement reform.
• Amgen and the Fox Foundation are moving toward collaborative models where patient groups, manufacturers, and governments co-design therapies to ensure scalability and usability.
• Mark Samuels suggests that CMS must reform its reliance on precedent to allow for innovative payment models that account for the full patient journey and downstream cost savings.