TRAIN eNews is delivered directly to organizations participating in The Research Acceleration and Innovation Network (TRAIN). TRAIN is an affinity network of foundations interested in taking a more strategic and entrepreneurial approach to their role as funders of medical research. TRAIN convenes dozens of forward-thinking foundations to learn from one another and share their novel solutions with the rest of the medical research system. To learn more about becoming a TRAIN organization, visit the TRAIN application page.

In This Issue

News From FasterCures
TRAIN Organization Updates
News You Can Use

News From FasterCures

Become a Mentor in the ‘Rare As One’ Mentorship Program!
In partnership with the Chan Zuckerberg Initiative, FasterCures seeks experienced leaders in the nonprofit disease-research community to serve as mentors in the upcoming Rare As One (RAO) Mentorship Program cycle, from May to December 2025.

As an RAO mentor, you will help shape the next generation of patient-driven research leaders by providing professional guidance, exchanging insights, and helping mentees build key relationships. Mentors will meet with their mentees every four to six weeks and receive a $1,250 stipend for their participation.

To be considered, please complete a brief questionnaire by March 28, 2025, to share your areas of expertise and experience. The questionnaire takes about 15 minutes to complete, and responses can be saved and revisited before submission.

Please note: You do not need to be a member of a rare disease organization to be considered. Apply now and help drive progress toward treatments and cures!

Research Partnership Maturity Model at Orphan Drug World Congress
This April in Boston, FasterCures Director Raymond Puerini will lead a workshop on the Research Partnership Maturity Model at the Orphan Drug Congress. Attendees will gain
valuable insights into assessing their organization’s assets and capabilities as research partners and enhancing their ability to drive innovation and accelerate research progress.

FasterCures’ Report Highlights Provider-Led Patient Engagement
Patient engagement is essential at every component of the health-care ecosystem, from drug development to care delivery. Research consistently shows that effective patient engagement in care delivery leads to better health outcomes, reduced health-care costs, and improved patient satisfaction. FasterCures’ latest report, Patient Engagement in Care Delivery: Approaches by Health-Care Providers, explores successful, actionable, provider-led strategies to enhance patient engagement and bridge this critical gap.

FasterCures Provides Recommendations to Food and Drug Administration’s Patient Preference Information Guidance
FasterCures submitted a comment letter on the Food and Drug Administration’s (FDA) expanded guidance on Incorporating Voluntary Patient Preference Information (PPI) over the Total Product Life Cycle of medical devices. Based on our expertise in accelerating biomedical innovation, we’ve provided actionable recommendations to enhance and expedite patient engagement in medtech research and development (R&D):

1. Clarify roles: Clearly define the roles of patients, caregivers, and patient organizations in planning and designing PPI studies to ensure accurate and meaningful data.
2. Expand education: Leverage platforms to offer resources and education on patient engagement, focusing on small- to mid-sized medtech companies.
3. Empower stakeholders: Support patients, caregivers, and patient organizations as drivers of patient-centric engagement in medtech R&D and regulatory processes.

TRAIN Celebrates 20 Years
For the past two decades, TRAIN has provided resources, tools, and initiatives to help patient organizations build their capacity to advance research efforts for the patient communities they serve. To commemorate TRAIN’s 20th anniversary, we launched the TRAIN Impact Series. As part of the series, throughout 2025, we will share short articles on FasterCures’ LinkedIn account, written by TRAIN members focusing on the impact that TRAIN and patient organizations have had—and continue to have—in driving research progress.

Vital Voices: Opportunities for Patient Organizations to Engage with Centers for Medicare & Medicaid Services
On April 28, 2025, TRAIN will host an in-person workshop in Washington, DC, designed to empower patient organizations and their communities with the knowledge and strategies needed to engage effectively with the Centers for Medicare & Medicaid Services (CMS). Through expert-led panels, participants will gain insights into the existing but complex structures enabling patient organizations to engage with CMS. The deeper understanding gained may also suggest alternative perspectives to help patient organizations refine their existing strategies for CMS engagement. You can register at this link.

Help Grow TRAIN
We’re expanding our network and seeking new research-focused patient organizations to join us. Take a look at our list of participating organizations—if you don’t see patient organization partners in your network listed, encourage them to apply and be part of this impactful community!

TRAIN Organization Updates

TRAIN Welcomes New Organizations
FasterCures is pleased to announce the addition of the following organizations to TRAIN:

• CACNA1A Foundation
• Chelsea's Hope Lafora Children Research Fund
• CSNK2A1 FOUNDATION
• Cure VCP Disease
• cureCADASIL Association
• The FamilieSCN2A Foundation
• PACS1 Syndrome Research Foundation
• Primary Sclerosing Cholangitis Partners Seeking a Cure
• Progressive Familial Intrahepatic Cholestasis Advocacy and Resource Network
• Raymond A. Wood Foundation
• Smith-Kingsmore Syndrome Foundation
• The Stiff Person Syndrome Research Foundation
• Tatton Brown Rahman Syndrome Community
• The TBCK Foundation
• Team Telomere
• YWHAG Research Foundation

Susan G. Komen Foundation Launches Online Breast Cancer Registry
The Susan G. Komen Foundation launched a new patient registry, ShareForCures, that will allow breast cancer patients and survivors to contribute easily to groundbreaking research from the comfort of anywhere. Data collected through ShareForCures will be available to foundation-approved research partners and academic researchers.

Ovarian Cancer Study Utilizing Artificial Intelligence for Early Cancer Detection
An Ovarian Cancer Research Alliance-funded study is attracting attention for using AI and nanotechnology to develop a cutting-edge blood test for early ovarian cancer detection. The test has the potential to identify cancer years before symptoms occur.

The Multiple Myeloma Research Foundation Enrolled First Patient in Its Horizon Clinical Trials Program
The Multiple Myeloma Research Foundation has enrolled the first patient in its Horizon Clinical Trials Program. This innovative trial is designed to test multiple novel therapies simultaneously for relapsed and refractory multiple myeloma, allowing researchers to determine the most effective treatment combinations, sequences, and durations.

Lupus Research Alliance-Funded Research Finds Link Between Lupus and Gut Bacteria
A new study, partly funded by the Lupus Research Alliance, has revealed how certain gut bacteria can escape the gut and trigger autoimmune responses in people with lupus. The findings may pave the way for potential treatments that target these bacterial triggers, possibly reducing disease severity without heavily suppressing the immune system.

News You Can Use

Philanthropy, Advocacy, and Collaboration Drive Progress in Rare Disease Research
Advocates and foundations are driving cross-sector collaboration to accelerate therapeutic development and expand treatment options for rare disease patients. Milken Institute Strategic Philanthropy Director Caitlyn Barrett, lead of the Science Philanthropy Accelerator for Research and Collaboration, shares her firsthand experience in a Milken Institute Insights article, highlighting the impact of philanthropic investment and patient advocacy in advancing rare disease research.

FDA Releases Strategic Agenda Rare Disease Innovation Hub
The FDA recently released a strategic agenda for the Rare Disease Innovation Hub introduced in 2024. The strategic agenda was developed from input from the rare disease community and outlines the following key goals for the Hub:

• Advance regulatory science for rare-disease therapies.
• Strengthen coordination across FDA’s medical product centers, with a focus on aligning efforts between the Center for Biologics Evaluation and Research and the Center for Drug Evaluation and Research.
• Establish a centralized contact point to streamline engagement with external partners.

NIH Seeking Input for Disability Health Research Strategic Plan
The NIH invites public feedback on a draft framework for its upcoming strategic plan on
disability health research. NIH encourages input from a broad range of stakeholders, including individuals with disabilities, health-care professionals, academic and industry researchers, patient advocates, and advocacy organizations. These responses will play a key role in shaping both the framework and the final strategic plan. The deadline for submissions is March 12, 2025.

Food and Drug Law Institute Workshop for Patient Organizations Engaging with the FDA
The Food and Drug Law Institute is hosting a free virtual workshop March 4–5, 2025, designed to help patient organizations navigate working with the FDA. Law, industry, and FDA experts will discuss topics essential to patient organizations’ effective engagement with the FDA, such as the regulatory processes, the goals of the new administration, and engaging with specific centers of the agency. Beyond working with the FDA, patient advocates and organizations will learn more about navigating insurance as it relates to patients, clinical trial design and participation, and how to attain goals through avenues outside of the FDA.

EveryLife Foundation Releases Summary of Workshop Focused on Characterizing Ultra-Rare Diseases
The EveryLife Foundation for Rare Diseases released a summary paper from the 2024 Scientific Workshop, Therapy Development for Small Populations: Evidence, Implications, & Policy in Characterizing Ultra-Rare. The paper recaps the key takeaways and opportunities for action, including characteristics for defining ultra-rare, innovative trial designs, ethical considerations, shifts in the risk-sharing paradigm, concerns with regulatory flexibilities, and the importance of real-world evidence.

Rare Disease Endpoint Advancement Accepting Proposals for Pilot Program
The FDA’s Rare Disease Endpoint Advancement Pilot Program is accepting proposals for a pilot program supporting the development of novel efficacy endpoints for drugs treating patients with rare diseases. Accepted sponsors will gain enhanced interaction with interdisciplinary FDA experts and relevant review divisions to refine their endpoint development. Meeting requests can be submitted on a rolling basis and must be received by the last day of each quarter: March 31, June 30, September 30, and December 31.

Impact of Executive Actions on Nonprofit Organizations
Since taking office, President Trump has implemented executive orders impacting funding, compliance, and operations for tax-exempt organizations. Arnold & Porter released an article breaking down the implications that these presidential actions could have for the nonprofit sector.