Session 3.1 - RISE Together: Data Sharing Across the Rare Disease Ecosystem

Why It Matters

Standardizing and openly sharing digital health data unlocks regulatory pathways and accelerates therapeutic development for ALS and other rare diseases, delivering faster, evidence‑based treatments to patients.

Key Takeaways

• ALS stakeholders demand standardized digital health data sharing protocols.
• Actigraphy datasets reveal longitudinal patient activity but require extensive cleaning.
• Vendor-owned data limits accessibility for regulatory and trial use.
• Multi‑stakeholder working groups define gaps and prioritize biomarker development.
• Lessons from ALS aim to accelerate data sharing across rare diseases.

Summary

The RISE Together Session 3.1 convened experts to explore data‑sharing strategies across the rare‑disease ecosystem, using amyotrophic lateral sclerosis (ALS) as a pilot. Panelists Colin Hovinga of the Critical Path Institute and Natanya Kerper of the Cystic Fibrosis Foundation highlighted why sharing real‑world and device‑generated data matters for patients, sponsors, and regulators.

Stakeholders identified several unmet needs: patients seek portable, diagnostic tools that respect limited mobility; sponsors view digital health technologies (DHTs) as exploratory and hesitate to submit them for regulatory approval; and vendors often retain ownership of raw sensor data, restricting broader access. The team secured a large actigraphy dataset from LSTDI, encompassing weeks‑long limb‑sensor recordings across multiple years, but faced months of data‑scrubbing, sensor‑identification, and alignment challenges before the information could be analyzed.

To address these hurdles, a multi‑stakeholder working group was formed, bringing together patients, researchers, vendors, and regulators. The group conducted surveys of global sites, revealing wide variation in sensor placement and sampling frequency, and began drafting a prospective, standardized protocol for longitudinal actigraphy collection. Visualization tools are being built to let investigators explore the high‑dimensional data and generate hypotheses, while the consortium maps gaps toward defining biomarkers and clinical‑outcome assessments.

The initiative demonstrates how coordinated data sharing can transform fragmented device data into actionable evidence, accelerating biomarker qualification and informing trial design. By codifying best practices in ALS, the Critical Path Institute aims to replicate this model across other rare neuro‑degenerative diseases, potentially shortening development timelines and improving patient outcomes.

Original Description

Session 3.1: Data Sharing in Practice Objective: This session will feature three examples of sharing different types of data. Panelists will discuss reasons for choosing to share data, the type of data to share, challenges encountered, and outcomes of sharing data.

Moderator: Rachele Hendricks-Sturrup, Duke-Margolis Institute for Health Policy

Presentations:

Collin Hovinga, Critical Path Institute

Natanya Kerper, Cystic Fibrosis Foundation

This public workshop, co-convened by the Duke-Margolis Institute for Health Policy and the U.S. Food and Drug Administration (FDA) Rare Disease Innovation Hub, is designed for all stakeholders in the rare disease community to explore data sharing as it pertains to informing development and regulatory review for rare disease therapies. The small patient populations and often heterogenous nature of rare diseases results in a paucity of data, further intensified when multiple sponsors are working to develop medical products for the same disease state. As a result, opportunities for rigorous, high quality data collection would have a significant impact in this space. There may be opportunities for the rare disease community to support and encourage broader access through the sharing of existing data to inform certain areas of rare disease medical product development including disease progression modeling, endpoint selection, inclusion/exclusion criteria, and safety. Normalizing data sharing could help to inform clinical trial protocols, safety monitoring, and risk-benefit assessments throughout the development and post-marketing processes.

The workshop focuses on clarifying possible avenues for data sharing and the types of data that can be shared (e.g., safety information, real-world evidence, and deidentified patient data). The workshop also discusses: the promotion of data sharing practices and structures for facilitating data sharing among rare disease medical product development stakeholders; examples of the impact of data sharing in regulatory submissions; a priori considerations for the collection and sharing of high quality data; some of the logistical and legal challenges encountered in data sharing; and whether there are ways that FDA might support data sharing, within the bounds of its authority.

This project is supported by the Food and Drug Administration (FDA) of the U.S. Department of Health and Human Services (HHS) as part of a financial assistance award U19FD006602 totaling $5,192,495 with 100 percent funded by FDA/HHS. The contents are those of the author(s) and do not necessarily represent the official views of, nor an endorsement, by FDA/HHS, or the U.S. Government.