Panel, Conference Presentation
Smart on Science: How Today's Patients Are Driving Cures
Milken InstituteKim McCleary, Bray Patrick Lake, Joe Selby, Mace Rothenberg, Lou DiGennaro, Tony Coles, Dr. Francis Collins
- Panel Composition: The session featured five leaders driving patient-centricity:
- Kim McCleary (Director, Strategic Initiatives, Faster Cures/Milken Institute)
- Bray Patrick Lake (Director of Stakeholder Engagement, Clinical Trials Transformation Initiative; NIH Precision Medicine Advisory Committee Co-Chair)
- Joe Selby (Executive Director, Patient-Centered Outcomes Research Institute [PCORI])
- Mace Rothenberg (Chief Medical Officer, Pfizer Oncology)
- Lou DiGennaro (President/CEO, Leukemia and Lymphoma Society)
- Tony Coles (CEO, Humanity; former CEO, Onyx)
- Historical Context: The shift from a paternalistic "learned intermediary" model (1973 Patients' Bill of Rights focused on care, not research) to active patient partnership began with the digital age and patient advocacy.
- Precision Medicine Initiative (PMI): President Obama launched the PMI to enroll a cohort of 1 million patients to link electronic health records, genomes, and lifestyle data; Bray Patrick Lake serves as the NIH Advisory Council Co-Chair for this initiative.
- Pfizer Strategic Shift: Pfizer appointed Dr. Rosalind Snyder as Global Patient Officer to integrate patient perspectives early in drug development rather than at Phase 3;
- This follows the commercial failure of Pfizer's inhalable insulin, which failed due to poor device usability and failure to replace injections, despite the molecule's potential.
- Pfizer now prioritizes Patient-Reported Outcomes (PROs) and early feedback to avoid late-stage development pitfalls.
- Drug Development Case Study (Gleevec):
- In 1998, patient Susan McNamara mobilized a petition after Novartis halted STI-571 (later Gleevec) trials due to supply constraints.
- Novartis responded by reopening trials within two months; the drug eventually generated over $4 billion in sales in the most recent year and is approved for 10 indications.
- Current real-world survival for Chronic Myeloid Leukemia (CML) is ~50% versus 80-90% in clinical trials, a gap largely attributed to high costs ($90,000 annually) and compliance issues.
- PCORI Methodology: PCORI mandates that research questions and study teams include patients, resulting in:
- Hepatitis C trials where patients rejected "watchful waiting" arms in favor of immediate treatment despite insurer preferences.
- Multiple Sclerosis research focusing on patient-defined symptoms (fatigue, pain) rather than just MRI plaque reduction.
- Clinical Trial Design Evolution:
- FDA increasingly accepts single-arm, non-randomized trials for drugs targeting specific driver mutations (e.g., crizotinib) when clinical equipoise is difficult.
- Regulatory frameworks (e.g., Breakthrough Therapy designation) aim to compress time-to-approval for high-need therapies.
- Patient advocacy groups (e.g., Parent Project Muscular Dystrophy) are co-designing endpoints to ensure trials measure outcomes meaningful to patients.
- Cost and Systemic Inefficiency:
- Panelists identified fragmented data systems, non-interoperable electronic health records, and siloed research infrastructures as primary drivers of high drug development costs (estimated at $2.2 billion per drug).
- PCORnet is being developed to link EHRs and claims data for post-approval real-world evidence, potentially involving industry funding.
- Future Outlook (2020 Targets):
- Data Utilization: The PMI cohort will generate big data insights at the micro-level via wearables and real-time technology.
- Collaborative Models: Expectation of deeper coalitions between industry, academia, and patient groups (e.g., Cystic Fibrosis Foundation's investment in Kalydeco).
- Cultural Shift: A move away from adversarial relationships toward shared governance of research questions and data transparency.
- Regulatory Priorities: The 21st Century Cures Act and FDA user fee cycles are prioritizing patient perspectives as a core metric for drug approval and reimbursement.
- Patient Expectations: Patients now demand to be partners in priority-setting, not just sources of blood, consent, or funding; they are increasingly leveraging social media and independent networks to influence research agendas.