In This Newsletter
News From FasterCures
TRAIN Organization Updates
News You Can Use
News From FasterCures
FasterCures Vital Voices Webinar Series
On August 5, FasterCures hosted the first webinar of our three-part webinar series. This webinar, “Strength in Numbers: Coalitions to Amplify Patient Voices in CMS Decision-Making,” focused on engaging coalitions to make a favorable impact on the Centers for Medicare & Medicaid Services (CMS) decision-making. We are excited to continue this momentum with our second webinar, “Just the Facts: Patient Experience Data to Inform CMS Decision-Making,” on September 30 at 1–2 p.m. EST. This webinar will provide practical guidance on how patient organizations can effectively collect, structure, and present patient-experience data to support CMS decisions. Our third webinar, “Where the Rubber Meets the Road: Navigating State-Level Coverage and Payment Decisions,” will be held in November. We will send updates when the date and registration are available.
FasterCures’ Vital Voices Workshop Recording/Resources
On April 28, FasterCures hosted the “Vital Voices Workshop: Opportunities for Patient Organizations to Engage CMS” at our Washington, DC, office, bringing together former CMS leaders, patient advocates, and policy experts to explore how patient organizations can play a more active role in shaping CMS access and coverage decisions.
Milken Institute Future of Health Summit 2025
On November 4–6, the Milken Institute will host the annual Future of Health Summit in Washington, DC. Each year, this meeting convenes many of the world’s brightest minds to confront the toughest challenges in health by matching human, financial, and scientific resources with the most innovative and impactful ideas to drive better health outcomes for everyone in every community. Organizations participating in The Research Acceleration and Innovation Network (TRAIN) will receive further details in the coming weeks.
TRAIN 20th Anniversary Impact Stories
In celebration of TRAIN’s 20th anniversary, each month in 2025, we are sharing TRAIN Impact Stories featuring reflections from members across our network on the impact of patient organizations in advancing research and how TRAIN has supported their efforts. Kari Luther Rosbeck, president and CEO, TSC Alliance; David Panzirer, trustee, Helmsley Charitable Trust; Kapila Viges, CEO, MPN Research Foundation); and Michael Hund (CEO, EB Research Partnership) are four of our most recent impact stories. Visit the links above to learn more about their efforts!
TRAIN Organization Updates
TRAIN Welcomes New Organizations
FasterCures is pleased to announce the recent addition of the following organizations to TRAIN:
the American Brain Foundation, Food Allergy Fund, and Lymphatic Education and Research Network.
Friends of Cancer Research: New Framework Proposes Efficient Pathway to Advance Cell-Based Gene Therapies
Friends of Cancer Research, in partnership with the Parker Institute for Cancer Immunotherapy, has introduced a new framework to accelerate the development of cell‑based gene therapies. The framework is detailed in an article published in the Journal for ImmunoTherapy of Cancer on June 17, 2025, titled “Intentional Heterogeneity in Autologous Cell‑Based Gene Therapies: Strategic Considerations for First‑in‑Human Trials,” to accelerate the development of cell‑based gene therapies. The framework supports the inclusion of multiple genetically modified T‑cell subpopulations within a single investigational product, allowing simultaneous evaluation of different edits and reducing the time needed to test each one individually. It includes technical guidance on trial design, manufacturing and control standards, and nonclinical considerations, and builds on input from a public Food and Drug Administation‑inclusive meeting held in May 2024. Find more information.
Michael J. Fox Foundation and Alzheimer’s Association: Bridge Funding for Disrupted Neurodegenerative Research
The Michael J. Fox Foundation and the Alzheimer’s Association have jointly launched the Bridge Funding for Disrupted Neurodegenerative Research Grant Program, a collaborative initiative to support scientists whose research into Parkinson’s, Alzheimer’s, and related neurodegenerative diseases has been unexpectedly interrupted due to institutional or funding changes.
The program offers short-term, flexible funding to help sustain critical research efforts and prevent the loss of valuable progress while investigators work to secure long-term support. Learn more.
David Fajgenbaum Shares Powerful TED Talk
We're proud to share a powerful TED talk by David Fajgenbaum, MD, cofounder and president of the Castleman Disease Collaborative Network. Inspired by his personal journey—saving his own life with a repurposed drug and advancing research and treatment for Castleman disease— Fajgenbaum also cofounded Every Cure to unlock the greater, often life-saving potential of existing medicines. We hope you'll watch and be inspired by Fajgenbaum's TED talk and the work Every Cure is driving forward.
Genetic Alliance: Resilience Project and Webinar
On August 13, 2025, Genetic Alliance hosted a webinar as part of the Resilience Project, introducing a new drug discovery platform. The project, led by Stephen Friend and Eric Schadt, aims to identify drug targets by studying rare individuals who carry disease-causing mutations yet do not develop symptoms.
The approach uses large genomic datasets and advanced artificial intelligence/machine learning models to detect protective genetic factors—candidate suppressors—that could inform new therapies. The August 13 webinar outlined the project’s goals and invited rare-disease advocacy organizations to codesign the platform. A recording of the session is available for those interested in learning more or becoming founding partners.
News You Can Use
US FDA: Orphan Grants Program
The Food and Drug Administration’s Orphan Products Grants Program provides funding to support the clinical development of medical products for rare diseases and conditions. By offering grants to academic and industry researchers, the program aims to advance innovative therapies that might otherwise lack commercial incentives due to the small patient populations affected.
Since its inception, the program has helped bring numerous treatments to market, particularly for diseases with unmet medical needs. Applicants can receive funding for both early- and late-stage clinical trials, promoting faster development timelines and broader patient access. Learn more.
TargetCancer Foundation Launches “Paying With Our Lives”: Advocacy Campaign to Save Rare Cancer Research
TargetCancer Foundation has launched “Paying With Our Lives,” a campaign to raise awareness about the potential impact of recent federal funding cuts on rare cancer research. The campaign highlights the possible consequences of decreased support, including delayed clinical trials and slowed progress in developing new treatments. Individuals affected by rare cancers are encouraged to share their stories, engage with policymakers, and use the campaign’s resources, such as advocacy toolkits and social media materials, to support continued investment in rare cancer research. Find out more information.
Apply for the NORD Rare-Disease Research Grants Program
The National Organization for Rare Disorders (NORD) has announced the availability of $245,000 in research seed grants to support scientific discovery in five rare-disease areas: alkaptonuria, fibrodysplasia ossificans progressiva, nontuberculous mycobacterial lung disease, primary ciliary dyskinesia, and stiff person syndrome. These grants are intended to enable early-stage research projects that can lead to larger studies and ultimately to new treatments. NORD is now accepting proposals and encourages researchers to apply by the October 21, 2025, deadline. Find more information.
MDIC Patient Engagement Estimation Tool and User Guide
The Medical Device Innovation Consortium (MDIC) has launched the Patient Engagement Estimation Tool and User Guide to help medical device developers incorporate patient perspectives early in development. The tool uses a 10-question assessment with scoring and graphic results to recommend engagement strategies. This resource supports patient-centered decision-making to improve product design and regulatory alignment. Find more information.
CIVR Fall Policy Summit: Centering Patient Values in Drug Policy: Transparency, Value, and Access
On October 7, 2025, the Center for Innovation & Value Research (CIVR) will host an event to examine the implications of International Reference Pricing and spotlight patient-informed solutions for sustainable, equitable drug pricing reform. Learn more and register for the event—available both in-person and virtual.
Global Genes Guide to Comprehensive Fundraising Strategic Documents
On June 23, Global Genes published the “Guide to Comprehensive Fundraising Strategic Documents,” a quick guide offering a clear framework for rare‑disease nonprofits to secure sustainable funding. It lays out essential components for a well‑structured fundraising strategy, including an executive summary, organizational overview, goals that are specific, measurable, achievable, relevant, and time-bound, donor segment analysis, and detailed fundraising methods—ranging from individual giving and corporate partnerships to digital campaigns and legacy giving. The guide also covers financial projections, implementation timelines, roles and responsibilities, metrics for monitoring success, risk mitigation plans, and stakeholder communications. Learn more.
BIO Patient Advocacy Changemakers Event
On October 26–28, 2025, the Biotechnology Innovation Organization Patient Advocacy Changemakers Event will bring together advocates, policymakers, and industry leaders to break down barriers to access and advance patient-centered innovation. Hosted at the Hamilton Hotel in Washington, DC, the event will feature patient stories, policy insights, and new initiatives driving progress across the health-care system. To learn more and register, please visit the event page.