RAPID_PXP_Day_Special Notice_FINAL.pdf

Special Notice (SN) ARPA-H-SN-25-127

Patients’ Day Announcement for Advanced Research Projects Agency for

Health (ARPA-H)/Proactive Health Office (PHO)

Opportunity: Rare Disease AI/ML for Precision Integrated Diagnostics

(RAPID)

I. KEY INFORMATION

Patients’ Day February 25, 2025: 1:30 PM to 4:00PM EST

Location Virtual

Website for Registration

Patients’ Day - Patient Experience (PX) Partner Event Registration

Registration Opens December 19, 2024, at Noon ET

Registration Closes February 21, 2025, at Noon ET

Technical Point of Contact

Scott Gorman, Program Manager, ARPA-H / PHO Office

Program Email RAPID@ARPA-H.gov

Program Solicitation ARPA-H-SOL-25-119 (Final Anticipated in mid-January 2025, Draft currently available)

II. INTRODUCTION

This Special Notice is intended to alert the patient advocacy and engagement community to a newly approved ARPA-H Program and notify potential Patient

Experience (PX) Partner applicants of an associated ARPA-H event: RAPID

Patients’ Day. Under the cognizance of PHO, ARPA-H intends to release a

Innovative Solutions Opening (ISO) for the RAPID Program in mid-January

2025 (ARPA-H-SOL-25-119) on SAM.gov. The draft ISO release can be found here: Draft ISO on SAM.gov

Potential applicants are encouraged to attend the optional RAPID Patients’ Day.

While not mandatory, ARPA-H is hosting the Patients’ Day to achieve the following:

• Provide an overview of ARPA-H, its mission, programs, and funding structure.

• Provide information about the RAPID Program, including its goals, scope, timeline, and the forthcoming ISO.

https://solutions.arpa-h.gov/events/RAPID mailto:RAPID@ARPA-H.gov https://sam.gov/opp/c79e234252324d459a3081566d3bf3f8/view

• Detail the expected role and selection of the RAPID Patient Experience

(PX) Partners.

• Outline the role of the Independent Verification and Validation (IV&V) partner and their collaboration with ARPA-H and RAPID PX Partners.

RAPID Patients’ Day will be a virtual event. Attendance at this event is not a requirement for application to the PX Partner Network in support of the RAPID program. ARPA-H will collect participant questions throughout the event and publish answers on the RAPID Program page. Event slides will be made available on the RAPID Program page and on SAM.gov.

Advance registration is required. Approval of registration requests are at the discretion of ARPA-H. Registration does not guarantee acceptance to attend.

Accepted attendees will be notified via email with further event details.

Patients’ Day Participation is intended for the patient advocacy and engagement community, and is not intended for press, or a general interest audience.

III. RAPID – PROGRAM INFORMATION

The following is a brief description of the RAPID Program. Full programmatic details will be provided in the ISO.

RAPID – What if we could end the rare disease diagnostic odyssey?

RAPID aims to end the prolonged diagnostic odysseys affecting millions of rare disease patients worldwide. RAPID is designed to accelerate the development of novel diagnostic support tools for population-scale rare disease detection, systematically identifying and diagnosing rare disease patients efficiently and accurately. By aggregating and harmonizing fragmented data and employing advanced AI/ML techniques, RAPID aims to drive the development and deployment of comprehensive and highly accurate rare disease detection models.

The program aims to democratize access to advanced diagnostic support tools, ensuring that underserved and marginalized communities benefit from these innovations. By prospectively validating models in diverse clinical settings, RAPID seeks to transform the landscape of rare disease diagnosis, enabling timely and effective care for millions of patients.

While the primary goal of RAPID is to aid clinical diagnosis for rare diseases, the program is also expected to uncover new disease insights and biomarkers, support drug development, and impact the broader healthcare ecosystem.

Technical Approach

The RAPID program aims to systematically identify undiagnosed rare disease patients at scale, leveraging novel training data, AI/ML, and real-world validation. The program will be conducted over two consecutive phases.

Initially, ARPA-H plans to solicit innovative solutions for Phase I and Phase II that address the following Technical Areas (TAs): TA1, TA2, and TA3. A subsequent solicitation for TA4 will be issued separately at a later date. TA4 performers will only participate in Phase II. An Independent Verification and Validation (IV&V) partner will be selected separately from the RAPID ISO and will provide continuous support and oversight throughout the program, including oversight of PX Partners.

• TA1 – Massive-Scale Rare Disease Dataset: Develop the largest curated dataset of longitudinal rare disease patient health data by integrating information from a fragmented data landscape. This extensive resource, spanning thousands of rare and ultra-rare diseases, is purpose-built to accelerate the development and validation of advanced detection algorithms and diagnostic tools. Sustainable systems will ensure the continuous aggregation and curation of patient data, driving transformative advancements in rare disease detection and diagnosis.

• TA2 - Novel Diagnostic Indicators and Population-Scale Discovery:

Leverage cost-effective, widely accessible, and remotely deployable digital tools to collect data directly from patients, creating a comprehensive, multimodal dataset of rare disease patient health information. This dataset is designed to drive the discovery of novel diagnostic indicators and enable scalable detection of rare diseases in previously undiagnosed individuals, with an emphasis on reaching underrepresented and underserved populations.

• TA3 – Sustainable Platform for AI Diagnostic Development: Establish an enduring Rare Disease Data Commons (RDDC) with a benchmark dataset, enabling the accelerated development and empirical evaluation of rare disease diagnostic models. This dynamic, AI/ML-optimized platform will support interoperable data collection, sharing, analysis, and evaluation among key stakeholders, driving advancements in diagnostic decision support systems for rare diseases.

• TA4 - Effective Deployment and Real-World Validation: Develop, deploy, and prospectively validate AI diagnostic support models and applications in real-world settings to ensure they are accurate, reliable, and effective for identifying rare disease patients at scale. Support deployment directly to patients at home and integration into clinician workflows, ensuring the solutions are effective, trusted, and impactful for patients and providers.

IV. PATIENTS’ DAY – ABOUT PATIENT EXPERIENCE PARTNERS

Patients’ Day will detail the expected role and selection process of the RAPID

Patient Experience (PX) Partners. PX Partners will provide support to the program and performers across all four technical areas (TAs) within RAPID. It is expected that several patient advocacy groups will be funded to support

RAPID activities and ensure patient perspectives are integrated throughout the program to achieve the following:

PX Partner Objectives:

• Amplify Patient Voices: Ensure patient feedback, perspectives, and experiences are integrated into program decisions and technology development.

• Improve Data Relevance: Align data collection, aggregation, and diagnostic models with the real-world conditions, symptoms, and challenges faced by rare disease patients.

• Promote Engagement Actively engage populations underrepresented in rare disease research and historically marginalized in healthcare, including racial and ethnic minorities and rural communities, to ensure their perspectives are reflected in data collection and analysis.

• Build Patient Trust: Foster transparency and trust between the program and patients through active, ongoing engagement, championing responsible and ethical program practices.

For more details, please reference the RAPID Program Special Notice.

The Patients’ Day - PXP Network Event will be a one-day event including presentations by ARPA-H and our IV&V Partner covering various topics relevant to the PXP participation alongside RAPID technical awardees.

https://sam.gov/opp/9f2829bc90334594974c63674aca430f/view

Registration Information

There is no registration fee for the Patients’ Day event. Participants must register in advance through the registration website. Registration will close as specified in Section I. An online registration form and various other meeting details can be found at the registration website. Full event details along with an agenda will be provided upon registration confirmation.

All inquiries regarding the event should reference “Patients’ Day (ARPA-H-SN-

25-127)” in the subject line of the email. Prior to submitting an e-mail inquiry, please check out the Questions and Answers already posted on the ARPA-H website to see if your question is answered there.

V. DISCLAIMER

This SN is issued solely for information and potential new program planning purposes; the SN does not constitute a formal solicitation for solutions.

Participation is voluntary and is not required to respond to subsequent ISOs (if any) or research solicitations (if any) on this topic. ARPA-H will not provide reimbursement for costs incurred in responding to this SN. Further, this announcement is not a request for solutions; any so sent will not be reviewed.

Respondents are advised ARPA-H is under no obligation to acknowledge receipt of the information received or provide feedback to respondents with respect to any information submitted under this SN.

https://arpa-h.gov/research-and-funding/programs/rapid