By Patrick Cooney, for AMRC
Illustrative image generated using AI.
FEDERAL AGENCY NEWS
ARPA-H and Clinical Trials
On June 10, 2026, AMRC representatives met with staff from the Food and Drug Administration to discuss the ARPA-H wants to cut drug development from over a decade to less than four years, and it has put money and a solicitation behind that goal. On September 30, 2026, the agency launched SURPASS, a program to replace sequential Phase 1-2-3 trials with continuous, model-driven “phaseless” trials, alongside three companion infrastructure projects.
For the clinical research community, this is more than a new grant opportunity. SURPASS is designed to produce public regulatory documents and validated standards that sponsors, sites, and FDA could adopt across disease areas.
SURPASS (Simulation-augmented, Real-time Platform Adaptive Seamless Trials) is seeking proposals that combine predictive computational models, shared trial infrastructure, common control groups, and real-time analysis. Its design has three layers: a phaseless design engine built on models and digital twins, a continuous inference engine for real-time analysis, and an automation layer for trial startup and data handling. This could allow researchers to simulate trial designs before launch, analyze results as they emerge, adapt trials in response to new evidence, and reduce the administrative work involved in running them.
Three companion projects sit alongside SURPASS, rounding out a package of roughly $100 million. SURPASS is the only one open to competition; the other three were awarded directly. ARPA-H is now seeking proposals from cross-disciplinary teams specializing in statistics, AI, clinical trial design, operations and regulation.
These form part of Operation TrialBlazer, the clinical research modernization initiative HHS launched on June 22 across FDA, NIH and ARPA-H. The Trump Administration frames it as a competitiveness play: bringing trials, and the drug development that follows, back to the United States.
FDA’s main contribution is the Expedited IND Pilot, opened September 15. It pairs sponsors with Qualified Research Institutions for rolling review of pre-IND components, starting with roughly 8 to 10 pairs, while FDA keeps authority over whether trials proceed. The pieces fit together. ARPA-H builds and validates the methods, FDA tests faster regulatory engagement, and the resulting standards feed future FDA guidance.
To achieve the goals of this program, ARPA-H is seeking groundbreaking ideas from cross-disciplinary teams in statistics, AI, and clinical trial design, operations, and regulation. Teams will pursue three coordinated technical areas to build faster trials with fewer patients, develop a continuous inference engine for faster decisions, and create an agentic operations layer to reduce time and cost.
Proposers’ Day Event – November 6, 2026 (Register by Oct 28, 2026)
This is the second of two optional events for the proposer community to learn more about this opportunity, ask questions, and make connections. Registration required; see Special Notice ARPA-H-SN-26-164 for event details.
Solution Summary Due – November 30, 2026
Submitting a summary is required to move forward with this opportunity. After submitting a summary, await feedback from ARPA-H before proceeding.
Proposal Due – January 22, 2027
FDA Launches Rare Disease Framework
The Food and Drug Administration (FDA) opens a public process to make “fit-for-purpose” evidence the default for rare disease review — with a public meeting October 28 and written comments due December 11.
On October 14, 2026, the FDA launched its Rare Disease Framework Initiative, a cross-center effort to rebuild how CDER and CBER advise on and review rare-disease therapies. The goal is a process in which evidentiary expectations are consistently matched to what can realistically be generated for a given disease, population, and product — and communicated clearly from pre-IND through post-approval.
FDA released a companion white paper, A New Framework for Rare Disease Review, and opened a public docket (FDA-2026-N-12214). The agency says the effort could change regulations, guidance, and internal review processes. For AMRC members, this is an early and open window to shape the rules of the road before they are written.
FDA frames the initiative around a stark gap: roughly 30 million Americans — about one in ten — live with one of 7,000 to 10,000 rare diseases, yet only about 5% of those conditions have an FDA-approved treatment. Rare disease products already account for about half of recent novel CDER and CBER approvals, but the agency concedes progress has not kept pace with need.
The white paper also puts a price on delay. It cites a 2019 analysis estimating the U.S. economic burden of rare disease at $997 billion a year, and research linking the absence of an approved therapy to a 21.2% annual increase in per-patient costs.
FDA has set out six guiding principles. The resulting development and review process is meant to:
1. Deliver clarity and predictability — tailored guidance and transparent communication on how rare disease decisions are made.
2. Embed the patient voice — early in development and at clearly defined milestones, so sponsors and reviewers both hear patient views on disease burden, benefit-risk, and meaningful outcomes.
3. Lean on external experts — as a structural feature of review, especially for ultra-rare conditions with limited natural history.
4. Build institutional knowledge — shared resources and reviewer training to make decisions consistent across divisions.
5. Apply risk-based CMC and inspections — calibrating chemistry, manufacturing, and controls review and pre-approval inspections to support earlier access without lowering quality standards.
6. Champion novel science — innovative trial designs, statistical methods, and emerging tools such as Bayesian approaches, platform trials, and real-world evidence.
The scope runs from pre-IND engagement through post-approval obligations and covers both drugs and biologics. FDA stresses that the statutory standard of safety and effectiveness does not change; what changes is how the agency judges what evidence is reasonably attainable to meet it.
CAPITOL HILL NEWS
Congress In Focus
When the House and Senate gavel back in on November 9, 2026, six days after the midterm elections, they will face one of the most crowded year-end agendas in recent memory with very little time to work through it. Government funding runs out December 11. A long list of Medicare and public health programs expires December 31. A myriad of other important legislative initiatives also await action. And every one of those decisions will be shaped by an election result lawmakers will only just have absorbed.
Republicans currently hold narrow majorities in both chambers alongside the White House, and a lame duck may be the final stretch of unified GOP control. Forecasts heading into the final two weeks of the campaign favor Democrats to win the House. The Senate is far closer, with most estimates giving Democrats under two-in-three odds of taking control.
That outcome matters enormously for what happens in November and December. A party that knows it is about to lose a chamber has every reason to lock in its priorities before January 3, when the 120th Congress is sworn in. A party that knows it is about to gain one may prefer to wait and write the final product itself. Both instincts will be at work in this session.
