Critical Path Institute
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Critical Path Institute (C-Path) is a nonprofit, public-private partnership with the Food and Drug Administration (FDA) created under the auspices of the FDA’s Critical Path Initiative program in 2005. C-Path’s aim is to accelerate the pace and reduce the costs of medical product development through the creation of new data standards, measurement standards, and methods standards that aid in the sc
07/24/2026
Got Data? Evaluating Real-World Data Suitability to Inform Regulatory Decision-Making | Monday, Sept. 14 | 9 a.m. - 12:30 p.m.
Real-world data offers an opportunity to inform regulatory decision-making, particularly in rare diseases. However, variability in data sources and inconsistent data quality often limit the utility of RWD for regulatory decision-making. A central challenge is determining whether a given RWD source is fit-for-purpose before substantial analytical investment.
Critical Path Institute's Rare Disease Cures Accelerator-Data and Analytics Platform team will be hosting a free, in-person workshop, Got Data? Evaluating Real-World Data Suitability to Inform Regulatory Decision-Making. This interactive workshop will introduce a structured framework for evaluating whether real-world data is fit for regulatory use. Participants will assess theoretical RWD sources across key dimensions, including relevance, observability, comparability, and reliability, and discuss opportunities to improve RWD quality and standardization, particularly for rare diseases.
Register now: https://f.mtr.cool/brpmgtyrwo
07/22/2026
When rare disease data are collected across patient populations, the question that matters most is: how do we maximize the value of such data?
On Sept. 16 at the C-Path Global Impact Conference in Washington, D.C., a session titled "Leveraging Rare Disease Data to Inform Drug Development Tools for Rare Neurodegenerative Diseases" will address exactly that, from theory to regulatory action.
Presenters Alexandre Betourne and Mingyuan Wang, joined by Mark Gordon of Arvinas and Mohamed Hamdani of Larimar Therapeutics, will walk through how C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP®) is helping reshape how clinical trials for rare neurodegenerative diseases are designed and measured.
Session highlights include:
• Advanced analytics: An update on a pioneering computational model to optimize trial design for Friedreich’s ataxia, currently under FDA review
• Patient-focused drug development: Analysis of how different versions of the progressive supranuclear palsy rating scale predict survival and track disease progression
• Data sharing and integration: A look at how integrated rare disease datasets can address longstanding challenges in drug development
• Actionable impact: Discussion of how these approaches could extend to rare dementias and similar diseases where gaps in trial design and outcome measurement persist
This is evidence-generation work with direct implications for patients, researchers and regulators alike, and a concrete example of the paradigm shift C-Path is driving across the drug development landscape.
Early bird pricing closes June 30. Register today and save: https://f.mtr.cool/ckhppjspcb
ICYMI | Meeting Patients Where They Are: Validating Remote Digital Tools for Multi-Indication NMD Research
As clinical research continues to evolve, so must the tools used to measure outcomes particularly in neuromuscular disease studies, where accessibility and patient burden remain important considerations.
During the 2026 C-Path Rare and Orphan Disease Program webinar, "Meeting Patients Where They Are: Validating Remote Digital Tools for Multi-Indication NMD Research", Dr. Tina Duong joined a panel of experts from industry and regulatory science to discuss the future of digital measurement in neuromuscular disease research.
The webinar explored the scientific, clinical, and regulatory considerations involved in validating video-based assessments and other digital tools across multiple NMD indications. Through presentations and panel discussion, speakers examined how these technologies can help make clinical research more accessible to patients regardless of geographic location, reduce the burden of trial participation, and generate reliable, regulatory-grade data.
A recurring theme throughout the discussion was the importance of balancing innovation with patient-centered research principles. As digital tools become increasingly integrated into clinical trials, ensuring they are meaningful, accessible, and fit for purpose will be critical to advancing drug development and improving outcomes for people living with neuromuscular diseases.
The session highlighted the growing potential of remote digital assessments to expand participation in clinical research while maintaining the rigor needed to support regulatory decision-making and therapeutic development.
Watch the recording now: https://f.mtr.cool/hahrtshzmb
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