Panel
Precision Medicine, Custom Cures
Milken InstituteMargaret Anderson, Mike Polini, Jim Greenwood, George Church, Retta Berry, Chris Austin
- The industry expects a rapid acceleration in precision medicine, with the capability to deploy personalized pills and custom cures becoming a reality, driven by a million-fold decrease in the cost of genomics and microbial genomics.
- Predictions indicate that within five years, every patient in the United States newly diagnosed with non-routine cancers will have both their specific genome and the cancer's genome sequenced, shifting cancer treatment from tissue-specific to molecular pathway-based approaches.
- Plans include expanding data sharing through initiatives like the Personal Genome Project and the Million Veterans Project, where 96% of volunteers expressed a desire to learn their genomic data, while also integrating phenotype and clinical manifestations to bridge the gap between genotype and treatment.
- Significant challenges remain in interpreting sequence data and matching it to clinical practice, as the current system is not suited for precise sequencing, with a typical diagnostic odyssey lasting two to four years and a lack of treatments for approximately 6,500 of the 7,000 known rare diseases.
- Regulatory and reimbursement frameworks present major risks, including the FDA's requirement for companion diagnostics, the high cost of individualized treatments (potentially $200,000 to $400,000 annually), and the inability of 10-year budget cycles and private insurers to account for long-term preventative savings despite potential annual savings of 17,000 strokes from optimized drug dosing.
- While gene therapy is advancing toward precise insertion or removal of genetic material in neurons, retina, and muscle cells, current FDA regulations treat distinct mutations for conditions like Duchenne muscular dystrophy as separate drugs, requiring significant regulatory changes to enable personalized nucleic acid treatments for varying mutations.
- Economic opportunities exist in repurposing existing drugs to avoid the 10- to 15-year billion-dollar development cycle, yet the sector faces inertia, siloed data, and the risk that payers may reject high-cost therapies if the translation from molecular discovery to tangible health interventions does not improve.
- Clinical outcomes are showing promise, exemplified by a 100% response rate in a trial for a gene fusion in non-small cell lung cancer identified just 14 months prior, demonstrating that the process from discovery to clinical trial reporting can be rapid if all parties collaborate.