In This Newsletter:
News from FasterCures
TRAIN Organization Updates
News You Can Use
News from FasterCures
Resources to Strengthen Patient Organization Engagement with CMS, Now Available in the Vital Voices Collection
The FasterCures Vital Voices effort, part of our patient engagement work, equips patient organizations with the knowledge and strategies to engage effectively with the Centers for Medicare and Medicaid Services (CMS), the federal agency that plays a key role in shaping the US health care system by setting national standards for coverage, reimbursement, and value assessment.
Vital Voices offers both an understanding of CMS’s structure and mandates and practical strategies for building coalitions, collecting patient experience data, and navigating state-level policy. Together, these resources provide real-world insights and actionable approaches that strengthen patient organizations’ engagement with CMS and elevate the patient voice across their activities and decisions.
- Vital Voices Workshop—A four-part in-person workshop that brings together former CMS leaders, patient advocates, and policy experts to explore how patient organizations can play a more active role in shaping CMS decisions on access and coverage.
- Vital Voices Webinar Series—A three-part series focused on CMS engagement topics that were of particular interest to workshop attendees. Each session dives into a critical topic, offering real-world insights and actionable strategies to elevate the patient voice in CMS activities and decisions.
- Vital Voices Toolkit—Coming soon!
Support Emerging Patient Organization Leaders—Become a Rare As One Mentor
The Rare As One Mentorship Program, developed in collaboration with the Chan Zuckerberg Initiative, supports patient organizations as they build research capacity and advance progress toward treatments and cures. As a member of the TRAIN network, your leadership and experience uniquely position you to provide strategic guidance to emerging leaders at a pivotal stage of their work.
Mentors are asked to meet with their mentees every four to six weeks through December 2026 and provide tailored guidance aligned with their mentees’ organizational goals. Mentors receive a $1,250 stipend in recognition of this commitment.
Leaders from both rare and more prevalent disease organizations are encouraged to participate. If you are interested, please complete this questionnaire by March 13.
Strategies and Resources to Support Patient Engagement Across the Medtech R&D Lifecycle: Virtual Public Convening
Patient engagement in medtech continues to grow, yet it is not consistently treated as a business imperative, and practical barriers often limit adoption. To help close this gap, FasterCures is developing a Medtech Patient Engagement Toolkit, a Case Study Library, and a companion how-to guide aligned with its Medtech Patient Engagement Value Framework.
Join us on March 19, 2026, from 11 a.m. to 1 p.m. ET for an interactive virtual public convening, “Strategies and Resources to Support Patient Engagement Across the Medtech R&D Lifecycle,” to share feedback on these resources. The event will also feature device developers and patient organizations discussing real-world patient engagement strategies and their impact throughout the product lifecycle. Register.
TRAIN Organization Updates
TRAIN Welcomes New Organizations
We’re pleased to announce the recent addition of the following organizations to TRAIN:
ARRE Foundation
The ASXL Rare Research Endowment Foundation is a family-led nonprofit advancing research and support for individuals affected by ultra-rare ASXL-related genetic disorders.
UPA United Porphyrias Association is a patient advocacy nonprofit dedicated to improving the quality of life for people living with porphyria by advancing awareness, research, and therapies.
Myotonic Dystrophy Foundation Launches the 2026 Spring Pilot Grants Program
The Myotonic Dystrophy Foundation (MDF) has launched its 2026 Spring Pilot Grants Program, offering $50,000 in one‑year funding to support innovative research in myotonic dystrophy. The program enables investigators to explore new ideas, generate preliminary data, and build the foundation for larger research efforts. Applications are open to researchers at academic institutions and nonprofit research institutes worldwide. The application deadline is March 20, 2026.
MDF also announced an update to its Drug Development Pipeline chart. The resource provides an overview of active investigational programs, along with historical and inactive efforts, and serves as a reference tool for researchers, clinicians, industry partners, and community stakeholders tracking therapeutic development in myotonic dystrophy.
EB Research Partnership: Matter of Time Now Streaming on Netflix
A new documentary, Matter of Time, premiered at the Tribeca Festival in 2025 and tells the story of musician Eddie Vedder and a global community working to cure Epidermolysis Bullosa (EB), a rare and severe genetic skin disease that primarily affects children. The film follows Vedder’s 2023 Seattle benefit concerts, which raised critical funding for clinical research through the EB Research Partnership, the largest nonprofit funder of EB research, which is focused on finding a cure by 2030.
News You Can Use
FDA’s TAP Facilitates Engagement with Non-FDA Parties
The FDA’s Center for Devices and Radiological Health launched the voluntary Total Product Life Cycle Advisory Program (TAP) Pilot to accelerate the development of high-quality, safe, effective, and innovative medical devices critical to public health. TAP provides patient organizations with a structured way to engage early with companies developing new medical devices. Companies enrolled in TAP work with a TAP Advisor who helps them prepare to engage with an organization. These early interactions help TAP innovators better understand patient perspectives in medical device development, health care provider needs for market adoption, and payer needs for coverage and reimbursement. The goal is to improve patient access to high-quality, safe, effective, and innovative medical devices. Find more information on TAP stakeholder engagement.
NORD Launches a New Training Module for Rare Disease Drug Development
The National Organization for Rare Disorders (NORD) has launched a free, three-part online course designed to teach patients, caregivers, and advocates how to actively engage in the drug development process for rare diseases. Developed in partnership with the FDA and Critical Path Institute, the course explains each key step, from early research and clinical trials to FDA review, demonstrating how patient input can shape study design and treatment progress. The course is available on NORD’s RareEDU platform.
Register for Rare Disease Scientific Symposium—April 14–15
The NORD Rare Disease Scientific Symposium will bring together a dynamic community of researchers, clinicians, industry leaders, and advocates to spark the conversations and connections that drive science forward. This 1.5-day event in Arlington, VA, is designed to push boundaries and bridge gaps across the rare disease landscape.
National Health Council Science of Patient Engagement Summit—May 11–12
The Science of Patient Engagement Summit convenes innovators, health leaders, decision-makers, and patient advocates to explore how meaningful patient engagement leads to better diagnostics, more effective treatments, and stronger health system adoption. The event focuses on practical ways to involve patients early and throughout development and implementation to improve data quality, performance, and real-world health outcomes.
First FDA Approval of Rare Disease Gene Therapy Sponsore Nonprofit
The FDA has approved Waskyra (Etuvetidigene autotemcel), a new gene therapy for the rare immune disorder Wiskott-Aldrich syndrome. Waskyra is the first gene therapy approved for this rare disease, and Fondazione Telethon is the first nonprofit to secure approval for a gene therapy in the US. The treatment uses a patient’s own blood stem cells, which are genetically corrected and reinfused. In trials, it showed strong results, reducing severe infections by 93 percent and moderate-to-severe bleeding episodes by 60 percent. The approval follows a recent positive recommendation in Europe and marks a notable milestone for nonprofit-led rare disease research.
Global Genes Guide to Cooperating Among Patient Advocacy Organizations
Global Genes’ Beyond Boundaries Quick Guide demonstrates how patient advocacy groups focused on the same rare disease can enhance their impact by collaborating rather than operating separately. It provides practical strategies for cooperation to minimize duplication and better support their communities, including joint awareness campaigns, policy initiatives, and shared resources such as research, fundraising, and patient support.
The Rising Tide Foundation for Clinical Cancer Research Launches ACCESS Award
The Rising Tide Foundation for Clinical Cancer Research’s ACCESS Award funds projects that develop innovative, affordable, and scalable solutions to improve cancer care and reduce disparities, particularly in low- and middle-income countries. The program seeks practical approaches that can be implemented in real-world settings. Academic institutions, nonprofits, and research consortia are encouraged to apply, with preference for international collaborations. Awards provide up to $500,000 over five years with required co-funding.
First-of-Its-Kind Venture to Revive Promising Rare Disease Treatments
A partnership between the American Society of Gene & Cell Therapy (ASGCT) and Orphan Therapeutics Accelerator (OTXL) will launch CGTxchange, a clearinghouse and marketplace designed to revive and advance deprioritized cell and gene therapies (CGTs). Many of these therapies demonstrated patient benefit in trials but were set aside due to commercialization challenges, especially for ultra-rare diseases. Using OTXL’s AI-based platform, CGT assets and related information can be cataloged in a secure, searchable database, analyzed to generate listings and profiles of shelved therapies, and used to provide risk-based assessments.
The listed assets can be matched with prospective investors or partners in the marketplace, thereby expanding the potential to secure funding and new clinical sponsors. ASGCT and OTXL will initiate development of CGTxchange in early 2026, with a public rollout planned for mid-2026.