In This Issue
News From FasterCures
Provide Feedback on Medtech Patient Engagement Framework
On June 11, FasterCures hosted a virtual convening, “Defining the Value and ROI of Patient Engagement in Medtech Product Development,” to gather public comments on a draft framework that outlines how and where patient engagement can drive impact across the total product lifecycle of medtech research and development (R&D) activities—from early-stage development to post-market decisions. This convening also featured a group of medical device and patient organization presenters sharing their experiences of engaging patients in their R&D activities and the impact of doing so. Read the draft version of the “Framework on the Value of Patient Engagement for Medtech Product Developers." Public comment submission on the draft framework is open until June 30. All interested parties can send their input to [email protected].
Patient Engagement with CMS Workshop Summary and Webinar Series
In April, FasterCures hosted an in-person workshop to provide patient organizations with the tools and insights needed to effectively engage with the Centers for Medicare & Medicaid Services (CMS). The event featured expert panels discussing strategies for CMS engagement, real-world case studies from patient organizations, and an interactive group discussion with panelists. If you could not attend or want to revisit sessions, recordings of the panel discussions will be available soon on the FasterCures website.
Over the next few months, FasterCures will continue this work through a series of webinars on key topics related to CMS engagement that were of particular interest to workshop attendees. Each session will dive into a critical topic—building effective coalitions, collecting patient experience data, and navigating state-level policy—offering real-world insights and actionable strategies to elevate the patient voice in CMS activities and decisions.
TRAIN Webinar Recording on Drug Repurposing Available
The TRAIN webinar, "New Horizons for Accelerating Drug Repurposing," is now available on the FasterCures website. This dynamic discussion explores how existing drugs can be harnessed for new therapeutic purposes. Speakers highlighted the critical role of patient advocacy, innovative funding strategies, and the growing impact of artificial intelligence (AI) in uncovering repurposing opportunities. Watch the webinar recording to learn how cross-sector collaboration opens new treatment innovation pathways.
Milken Institute Global Conference 2025
In May, the Milken Institute hosted its 28th Annual Global Conference in Los Angeles, CA. Centered around Towards a Flourishing Future, this year’s conference convened leaders from across sectors to explore solutions to the world’s most pressing challenges and identify opportunities to build a more sustainable, equitable, and resilient future for all. All public session recordings are available to view online.
We especially encourage you to watch “Breaking New Ground in the Fight Against Cancer,” a compelling discussion on the future of cancer care featuring TRAIN’s LeadersLink participant, Anjee Davis, CEO, Fight Colorectal Cancer, as a panelist.
TRAIN Impact Stories
In celebration of TRAIN’s 20th anniversary, each month we are sharing TRAIN Impact Stories featuring reflections from members across our network. Emily Kramer-Golinkoff (cofounder, Emily's Entourage), Wen Hwa Lee (CEO and chief scientist, Action Against AMD), Debi Brooks (CEO and cofounder, the Michael J. Fox Foundation for Parkinson's Research), and Kari Luther Rosbeck (president and CEO, TSC Alliance) have each shared how participating in TRAIN has helped advance their organizations' missions and amplify their impact.
TRAIN Organization Updates
TRAIN Welcomes New Organizations
FasterCures is pleased to announce the addition of the following organizations to TRAIN:
TRAIN Organizations Provide Input to CMS Town Hall
CMS hosted a livestreamed town hall meeting in April as part of the Medicare Drug Price Negotiation Program. The town hall provided an opportunity for patients, caregivers, patient organizations, health-care providers, and other interested parties to share input relevant to the drugs selected for negotiation. Representatives from several TRAIN organizations, including Asthma and Allergy Network, Alliance for Aging Research, and COPD Foundation, provided comments on several of the selected drugs. CMS stated that the goal of the session is to reach a fair price for the selected drugs, and input from these organizations will be considered as part of the negotiation process.
CURE Epilepsy Helps Fund SCN8A-Related Disorders Natural History Study
Supported in part by the CURE Epilepsy Rare Epilepsy Partnership award, researchers at Children’s Hospital of Philadelphia’s Epilepsy Neurogenetics Initiative completed the most comprehensive natural history study to date of SCN8A-related disorders. A gene variant causes SCN8A-related disorders and is linked to a broad spectrum of neurological conditions, including epilepsy, developmental delays, autism, and movement disorders. The study revealed that SCN8A patients face a significantly higher risk of developing severe seizures, including bilateral tonic-clonic seizures, beginning in infancy. Understanding the progression of SCN8A-related symptoms is a key step toward developing targeted therapies.
LeadersLink Alumna Discusses Inspiration for Founding Organization
Emily Kramer-Golinkoff spoke to the AP about her rare form of cystic fibrosis (CF). Emily explains that while most CF patients have seen improvements in their health with “CFTR modulator” therapies, those with uncommon mutations have been left with less effective options. She describes how her motivation to co-found Emily’s Entourage was to help accelerate research for patients with rare mutations. Emily’s Entourage now supports experimental “mutation-agnostic” gene therapies, which could help all CF patients regardless of their genetic mutation. One such treatment, funded partly by her nonprofit, began human trials in late 2023.
Friends of Cancer Research CEO Discusses Policy with BIO
Jeff Allen, CEO of Friends of Cancer Research, joined a Biotechnology Innovation Organization (BIO)-hosted discussion on the Food and Drug Administration’s (FDA) Accelerated Approval Pathway and its role in accelerating treatments for serious conditions like cancer, rare diseases, and neuromuscular disorders. A key concern raised during the conversation was the ongoing coverage challenges. While Medicaid covers most FDA-approved drugs, private insurers and Medicare Advantage plans often delay or deny access. These barriers can harm patients waiting for critical care. Continued advocacy is essential to ensure access keeps pace with innovation.
Bill Nye and Andrew Rosen Discuss the Importance of Science and Research
At STAT’s Breakthrough Summit East, Bill Nye and Andrew Rosen, CEO, National Ataxia Foundation, spoke on a panel about the importance and necessity of research and science. Nye emphasized the importance of science advocacy, especially amid the federal cuts to research funding. The panel also highlighted growing interest in ataxia research, spurred in part by Nye’s efforts. Nye warned against ongoing scientific misinformation and urged renewed commitment to evidence-based policymaking.
News You Can Use
ARC Launches LEADER 3D
As part of the Accelerating Rare disease Cures Program, the FDA’s Center for Drug Evaluation and Research launched the LEADER 3D initiative—Learning and Education to ADvance and Empower Rare Disease Drug Developers. The initiative aims to address the unique challenges of rare disease drug development, including small patient populations, genetic and clinical variability, and difficulties in establishing appropriate study endpoints. LEADER 3D will create targeted educational content to support drug developers in navigating the complex process of demonstrating safety and efficacy for treatments of serious and often life-threatening rare diseases.
FDA Introduces Agency-Wide AI Tool
The FDA has launched Elsa, a generative AI tool designed to enhance efficiency for agency employees. Elsa aims to enable staff to summarize documents, compare drug labels, review clinical protocols faster, and generate code. This launch marks the beginning of the FDA’s larger AI integration goals.
NIH Establishes New Public Access Policy
The new National Institutes of Health (NIH) Director has announced that the updated NIH Public Access Policy will go into effect on July 1. The revised policy eliminates the previous 12-month embargo, ensuring immediate public access to NIH-funded research findings. By making research freely and rapidly available through platforms like PubMed Central, NIH seeks to demonstrate the value of taxpayer-funded research and support reproducible, impactful science.
Funding Opportunity for AI Validating Drug Repurposing
Cures Within Reach is accepting proposals for investigator-initiated, proof-of-concept, Phase I, or Phase IIA clinical trials to validate AI-driven drug repurposing for any unsolved disease, preferably for rare diseases and off-patent therapies. Trials must use AI-generated data as preclinical evidence, and the drugs must already be approved for other uses. Submit your proposal to Cures Within Reach by August 25, 2025.
Share Your Rare Disease Experience with New NORD Study
The National Organization for Rare Disorders (NORD) is collecting data for the Living Rare Study, the first large-scale effort to better understand how the experiences and challenges of individuals affected by rare diseases change over time. Although rare diseases represent a public health concern, there is a lack of comprehensive data capturing their true impact. This study seeks to identify the unmet needs of the rare disease community.
New Funding Opportunities from the American Brain Foundation
The American Brain Foundation has launched three new funding opportunities totaling more than $9 million to support cutting-edge, interdisciplinary research. Nearly all grants are open to applicants worldwide:
- Neuroinflammation Research Grants: $5 million available
Supports research aimed at deepening our understanding of neuroinflammation across brain diseases.
Pre-proposal deadline: July 15 - Lewy Body Dementia Biomarker Grants: $2 million available
Focuses on advancing the development of biomarkers for Lewy body dementia.
Pre-proposal deadline: July 15 - Next Generation Research Grants: 14 grants for early career researchers
Designed to help early-career investigators generate preliminary data for future funding opportunities.
Application deadline: September 9
Register for Global Genes’ RARE Drug Development Symposium
The RARE Drug Development Symposium, hosted by Global Genes and Boston Children’s Hospital, empowers rare disease patient advocates to lead early-stage research efforts. Taking place September 3–4, 2025, in Boston, MA, the 1.5-day event features expert-led sessions, breakout discussions, and hands-on workshops designed to build knowledge, skills, and strategic connections. The symposium offers practical tools to accelerate progress and drive impact in rare disease research.