Panel
Regulating Medical Innovation in the Digital Age
Milken InstituteLisa Suennen, William Carson, Aneesh Chopra, Sohini Chowdhury, Lucia Savage, Jeffrey Shuren, Lisa Sunin, Jeff Sheeran, Bill Carson
- Investment and Market Growth: Digital healthcare investment surged from $1 billion in 2010 to an expected ~$11 billion in the current year, cumulatively exceeding $40 billion over the last eight years.
- Regulatory Catalysts: Three primary regulatory forces are driving capital investment and innovation:
- Medicare/Medicaid shifts to value-based payment models unlocking billions in capital.
- Regulations mandating consumer access to health data within digitized medical records.
- New legal mechanisms enabling commercial access to longitudinal claims data for 130 million lives (Medicare Advantage and upcoming Medicaid data).
- First Digital Medicine Approval: Otsuka and Proteus achieved the first approval for a digital medicine (Abilify MySite) through a collaborative, iterative process with the FDA, bypassing a requested 5,000-person trial in favor of human factor studies for patients with schizophrenia and mood disorders.
- Iterative Development Challenges: Pharma partners noted a significant cultural and procedural disconnect, contrasting the multi-year, linear pharma trial cadence against the weekly or monthly iteration cycles of software development.
- Software as a Medical Device (SaMD) Limitations: The FDA highlighted that the 40-year-old regulatory framework designed for hardware is ill-suited for SaMD, which features rapid iteration, non-deterministic outcomes, and algorithms that cannot be fully reverse-engineered post-deployment.
- AI and Deep Learning Regulation: The FDA is preparing a white paper to address machine learning modifications, aiming to avoid pre-approval for every algorithmic update while maintaining safety, particularly for "black box" deep learning models where decision logic is opaque.
- Data Quality and Context: Experts warned that without contextual metadata, real-world data is often unusable (e.g., distinguishing Parkinson's tremors from cobblestone-induced movement) or contains significant errors (e.g., incorrect patient demographics in EHRs).
- Interoperability Barriers: The marginal cost of data linkage is currently high due to a lack of industry standards; however, the FHIR API mandate has reduced Apple's connection costs to zero, highlighting the need for similar standards in therapeutic digital ecosystems.
- Pre-Certification Pilot: The FDA is piloting a "Pre-Cert" program for SaMD that focuses on certifying the organization's quality culture and processes rather than individual products, aiming to streamline low-risk device reviews.
- Pre-Cert Risks: Concerns were raised that without standardized data linking, the cost of generating real-world evidence for the pre-cert program could become 10x to 100x higher than necessary.
- Direct-to-Consumer (DTC) Regulation: The DTC space faces a fragmented regulatory landscape involving 57 state and federal regulators (FTC, AGs), creating transparency gaps regarding data privacy compared to clinical trial contexts.
- Telemedicine Licensure: Panelists identified state-based medical licensure as a major barrier to digital health innovation, advocating for federal reforms to enable cross-state practice.
- Reimbursement-Driven Evidence: The FDA cited CMS reimbursement policies as a primary driver for faster evidence generation, noting that requiring data collection for payment (e.g., trans-catheter aortic valve registries) accelerated global adoption and data availability.
- GDPR Implementation: The Michael J. Fox Foundation reported that GDPR has been integrated into US clinical studies and consent forms due to European partners, but expressed concern over the lack of harmonized global privacy standards for medical research.
- Blockchain Utility: Opinions on blockchain were mixed; one panelist saw value in pharmacovigilance for tracking unique patient reports, while another expressed skepticism regarding privacy and consumer understanding of public/private key cryptography.
- Voluntary Industry Standards: A proposal was made for a multi-stakeholder, enforceable code of conduct for unregulated consumer health apps (similar to the "Karen Alliance" education privacy pledge) to establish baseline privacy protections without immediate legislation.
- Siteless Clinical Trials: The industry is moving toward "siteless" trials (like 23andMe) which require new solutions for adverse event monitoring and ensuring patient care access when no physical site or designated physician is involved.
- Data Ownership Debate: Panelists discussed the potential for patients to own and trade their data, suggesting blockchain could facilitate provenance tracking, though concerns remained about consumer comprehension and privacy risks.