Conference Presentation, Panel
Sustaining the New 'Golden Age' of Medical Research
Milken InstituteMargaret Anderson, Francis Collins, Kathy Giusti, Michael Milken, Kevin Yoder, Elias Zerhouni
- The Precision Medicine Initiative aims to enroll one million participants by 2019, with a potential public enrollment opening in September or October if ten million people express readiness, though accumulating necessary laboratory data will be a costly process requiring new systems and carrying risks of security breaches that could damage public confidence.
- A blue ribbon panel intends to outline specific scientific goals for the Cancer Moonshot in FY17, while a targeted meeting scheduled for June involving the Prostate Cancer Foundation and other major foundations seeks to establish new business models and incentives to resolve data sharing obstacles.
- Financial commitments face critical uncertainty; while a recent $2 billion (7%) fiscal year increase is noted, a lack of decade-long investment plans makes long-term planning difficult due to annual budget volatility, and experts warn that failing to maintain U.S. commitment could cede bioscience leadership to China, which has allocated over $300 billion, and Japan, where companies like Fujifilm have pivoted to bioscience.
- Global competitive pressure is intensifying as the U.S. lacks a monopoly on discoveries, with international rivals investing heavily, and major technology firms such as Facebook, Google, Apple, and Microsoft are expected to move quickly to capture medical data value if domestic progress lags.
- Clinical trial success rates are projected to double if participation is doubled, yet current enrollment remains critically low at less than 1.5% for Parkinson's and 5 to 6% for cancer, prompting calls for social movements and greater patient involvement to accelerate progress, as evidenced by the Match trial which was overwhelmed by enrollment speed.
- Technological capabilities are expected to expand dramatically, with future computers capable of processing trillion-by-trillion files and performing tens of trillions of calculations per second, while artificial intelligence and global data analysis by mathematicians and computer scientists will eventually dictate medical decision-making.
- Scientific productivity is anticipated to shift from single-therapeutic discovery models, which could take a century to resolve current challenges, toward parallel processing and Accelerating Medicine Partnerships that aim to reduce industry failure rates from 95% to 90%, effectively doubling productivity.
- Disease categorization and prevention strategies are evolving, with expectations that current classifications like diabetes will be viewed as outdated within 50 years as 50 distinct subtypes are identified, and that studying specific diseases like multiple myeloma can lead to prevention rather than just treatment.
- Regulatory and operational frameworks are shifting to support faster innovation, including FDA willingness to engage researchers early regarding drug combinations and approval on phase 2 trials, though high industry turnover necessitates continuous training for pharmaceutical teams on data usage.
- Patient engagement is identified as the critical path for research, as clinicians in academia lack time to analyze data sets due to patient care duties, and future progress depends on patients understanding and actively participating in their tissue and data contributions through social media and open discussions.
- The biomedical investment landscape faces demographic challenges where young investigators may not secure their first grant until age 42, despite 95% of scientists having made their primary contributions by that age, and training programs with a 16% success rate are discouraging scientists aged around 34.
- Framing biomedical research as a national security issue comparable to the $700 billion defense budget is proposed as a strategy to secure conservative political support, drawing parallels to the national response if 600,000 people were killed in a terrorist attack.
- The overall outlook emphasizes that a decade or more of sustained commitment is required to turn medical research into a long-term investment capable of reducing healthcare costs associated with cancer and Alzheimer's and maintaining U.S. leadership.