The Promise of the Cancer Moonshot

Mission Context & Authorization

• The Vice President announced the Cancer Moonshot in the Rose Garden following his decision not to run for re-election, pledging 15 months to drive progress.
• The President designated the Vice President as the head of "Mission Control" for the initiative during the State of the Union on January 12.
• The White House released a memo forming the Cancer Moonshot Task Force to establish a foundation for progress in the administration's final months.
• A proposed $1 billion funding request was submitted to Congress for FY17, with minor contributions from FY16 funds, representing a substantial increase for the NIH and NCI.

Core Objectives & Strategic Shifts

• The initiative aims to shift focus from studying cancer to ensuring humans live without being killed by it, prioritizing patient outcomes over bureaucratic processes.
• Key focus areas identified include prevention, early detection, immunotherapy, combination trials, genomics, data sharing, and pediatric oncology.
• The panel emphasized breaking "bureaucratic gravity" built since the 1950s to modernize research methods and accelerate timelines.
• The goal is to move from a culture of competition to one of collaboration and shared data, modeled after the Human Genome Project's downstream productivity surge.

Data Sharing & Interoperability

• Compliance with the 2006 law requiring public sharing of aggregate clinical trial data remains "embarrassingly low," with significant delays before data becomes available.
• Researchers are currently rewarded for withholding data until publication to secure first-author status, a disincentive that hinders early collaboration.
• A proposal was made to create a "Data Commons" where datasets are citable entities, encouraging researchers to share data without losing credit.
• The National Cancer Institute plans to launch the Genomic Data Commons on June 1 to aggregate genomic data from NCI-funded trials into a cloud-based platform.
• Barriers to interoperability persist, with the medical industry uniquely exempt from standard data-sharing requirements despite advances in technology.

Clinical Trial Reform & Patient Centricity

• Only 3–4% of adult cancer patients enroll in clinical trials, compared to significantly higher rates for pediatric cancers where mandatory enrollment was agreed upon in the 1960s.
• Pediatric oncology progress (survival rates rising from 3% to 90%) was achieved by mandating trial enrollment and data sharing among all newly diagnosed acute lymphoblastic leukemia cases.
• New trial models like i-SPY2, LungMap, and MATCH utilize master protocols to test multiple drugs simultaneously, allowing patients to move to effective treatments faster.
• Patient preferences regarding risk, toxicity, and endpoints are currently underrepresented in trial design, leading to poor enrollment and irrelevant outcomes.
• The Leukemia and Lymphoma Society launched a direct dialogue with the FDA, where patients defined their own risk-benefit tolerances to inform regulatory decisions.

Public-Private Partnerships & Funding

• The NIH and private sector are exploring a "Partnership for Advancing Cancer Therapeutics" to pool resources for generating data rapidly available to all.
• Bob Bradway (Amgen) noted that corporate entities have been more willing than academic institutions to share control arm data via Project Data Sphere.
• Lou DiGennaro highlighted the need for non-profits to utilize the Moonshot's convening power to align academic, industrial, and regulatory stakeholders.
• The panel argued that basic research investment is a marathon, noting that the CAR-T therapy breakthrough relied on 20 years of Leukemia and Lymphoma Society funding before clinical success.
• Industry partners are increasingly willing to provide compounds for combination trials, moving past previous resistance to head-to-head comparisons.

Forward-Looking Statements & Metrics

• Greg Simon stated the Blue Ribbon Panel aims to present a report to the National Cancer Advisory Board by the end of the year.
• The NCI plans to implement new requirements this summer mandating that all NCI-funded clinical trials (including Phase 1 and behavioral interventions) submit data to clinicaltrials.gov.
• The FDA is being cited as the "most innovative player" in adapting regulations to support telemedicine-based clinical trials and decentralized patient access.
• Panelists suggested that future success metrics should focus on speed from "idea to definitive experiment" rather than just dollar amounts or number of papers published.
• A specific goal was raised to drive down the cost of genomic sequencing similarly to the Human Genome Project's $1,000 goal to accelerate global productivity.

Challenges in Real-World Data & Health Equity

• Disparities in care based on geography remain a critical issue, with survival rates varying significantly by location within the US.
• Electronic Medical Records (EMRs) were developed for payment purposes, not research or patient care, creating a barrier to utilizing real-world data for treatment.
• Companies like Science 37 are piloting telemedicine-based randomized trials to bring data collection directly to patients, bypassing geographic barriers.
• A significant gap exists between clinical trial success rates (e.g., 90% remission in Chronic Myeloid Leukemia trials) and real-world outcomes (less than 45% long-term remission).
• Unpublished negative results in preclinical research ("failed notebooks") are lost, preventing the community from learning from non-replicable experiments.