In This Newsletter

News From FasterCures
TRAIN Organization Updates
News You Can Use

News From FasterCures

• Apply to Join the Next Cohort of the FasterCures LeadersLink Program

  Applications for the next cohort of the FasterCures LeadersLink program are open until Friday, February 16, 2024. LeadersLink is a program to support the growth and development of a cadre of promising leaders of nonprofit, patient-driven organizations that are changing the landscape of biomedical R&D. The 18-month program focuses on building participants’ leadership skills and professional network and addressing their top priorities through mentorship, a capstone project (for this cohort, centered on “Engaging Diverse Patients across the Spectrum of R&D”), a series of convenings, virtual collaboration, and access to Milken Institute events and networks. Apply here for consideration to join the 2024–2025 cohort!

• Register for TRAIN Webinar on Building Impactful, Transparent, and Reciprocal Relationships with Industry Partners

  You’re invited to join TRAIN for the “Building Impactful, Transparent, and Reciprocal Relationships with Industry Partners” webinar on Wednesday, January 31, from 2 to 3 p.m. ET. In this webinar, presenters will share their perspectives and experiences navigating building patient organization and industry partnerships and lessons learned for both stakeholders. Register here to attend the webinar.

• FasterCures to Host Research Partnership Maturity Model Interactive Workshop on the East Coast

  FasterCures held the inaugural Research Partnership Maturity Model Interactive Workshop in March 2023 in California. Nonprofit patient foundation leaders explored strategic growth using the Research Partnership Maturity Model, a tool developed by FasterCures. In Q1 of 2024, we are bringing the workshop to the East Coast (location TBD). The workshop will feature collaborative exercises to assess research readiness and identify alignment with key domains within the model. Workshop registration details will be sent to TRAIN organizations and other members of the FasterCures community soon.

• FasterCures Communities of Practice Update

  FasterCures launched Communities of Practice (CoP) for members of TRAIN and Rare As One Mentorship to discuss areas of challenge and need and share promising practices, strategies, and ideas to progress in those areas. The four communities are centered around the expertise, patients, money, and relationships needed to advance R&D, and have been proceeding with energizing and fruitful discussions. To join one of the CoPs, please reach out to [email protected].

• New FasterCures Publication on The Impact of Insight: Patient Preferences in Novel Screening Technologies for Cancer

  In September, FasterCures published The Impact of Insight: Patient Preferences in Novel Screening Technologies for Cancer, a report exploring patient preferences in cancer screening modalities and early interventions. The report found that patients’ top preferences for screenings include accuracy; administration by primary care physicians; causing minimal harm, discomfort and prep; and convenience. Patients also expressed being open to new technologies. Download the report infographic. We encourage you to share with your network!

TRAIN Organization Updates

• TRAIN Welcomes Two New Member Organizations

  CureSHANK

  Global Lyme Alliance

• Ontario Brain Institute Partners to Launch the Canadian Neuroanalytics Scholars Program

  The Ontario Brain Institute, in partnership with Campus Alberta Neuroscience, the Hotchkiss Brain Institute, and the Tanenbaum Open Science Institute at The Neuro, with support from the Hilary & Galen Weston Foundation, has launched the Canadian Neuroanalytics Scholars Program. The program will support and train up to 20 postdoctoral scholars in advanced analytics to cultivate a world-class talent pool that can effectively utilize the existing open neuroscience data and meet the growing demand for neuroscience research in artificial intelligence and machine learning. Kirk Nylen, a 2022–2023 participant in the FasterCures LeadersLink program, developed this new scholars program.

• Cure HHT to Sponsor Clinical Trial of Self-Manufactured Investigational Drug Product

  Cure HHT has become one of the few patient advocacy organizations worldwide to sponsor a clinical trial of a self-manufactured investigational drug product. When Pazopanib, a cancer medication, was sold to a new company that discontinued hereditary hemorrhagic telangiectasia- (HHT) related clinical trials, Cure HHT decided to invest in buying and manufacturing the drug itself. Through fundraising and Department of Defense and Food and Drug Administration (FDA) grants, Cure HHT built a clinical research organization and developed a Phase II/III clinical trial of Pazopanib to treat HHT-related bleeding. Learn more about Cure HHT’s journey and process.

• Foundation Fighting Blindness Partners with Verana Health to Integrate Genetic Testing Data into Real-World Evidence Research

  Foundation Fighting Blindness (FFB) and Verana Health have partnered to work on providing the life sciences community with deeper data to optimize and expedite clinical trials. Through this partnership, FFB will provide Verana with de-identified genomics data from its My Retina Tracker Registry to support clinical trial research surrounding patients with inherited retinal degenerative diseases. This groundbreaking partnership exemplifies how genomics data can be critical for the next frontier in real-world evidence.

News You Can Use

• FDA Launches START Pilot Program to Accelerate Development of Rare Disease Therapies

  FDA announced an opportunity for a limited number of development programs to participate in the Support for clinical Trials Advancing Rare disease Therapeutics (START) Pilot Program. Program participants will receive more frequent advice and increased communication with FDA staff to address product-specific development issues. The Center for Drug Evaluation and Research will accept applications between January 2 and March 1, 2024.

• FDA Releases ‘Rare Diseases: Considerations for the Development of Drugs and Biological Products’ Guidance Document

  In December 2023, the FDA released “Rare Diseases: Considerations for the Development of Drugs and Biological Products” guidance document for industry. The guidance intends to assist rare disease drug and biologic product developers in conducting successful drug development programs. The guidance finalizes the draft guidance issued in 2019.

• Chan Zuckerberg Initiative Science in Society Program Launches Third Cycle of Rare As One Network

  The Chan Zuckerberg Initiative’s Science in Society Program opened applications for the third cycle of the Rare As One (RAO) Network. In cycle 3 of the program, nonprofit/charitable patient-led rare disease organizations will receive a five-year grant opportunity to strengthen their organizational capacities, develop a collaborative research network, convene their patient and research communities, and align them along shared research priorities. Learn more and apply by Thursday, February 22, 2024.

• Rare Disease Week on Capitol Hill in Washington, DC, February 25–28, 2024

  Rare Disease Week on Capitol Hill, hosted by the Rare Disease Legislative Advocates, a program of the EveryLife Foundation for Rare Diseases, is taking place in Washington, DC, February 25–28, 2024. During this multiday event, rare disease advocates are brought together to be educated on policy proposals impacting the rare disease community and provided opportunities to advocate for policy changes directly with their Members of Congress.

• Rare Disease Day at NIH, February 29, 2024

  On February 29, 2024, the National Institutes of Health (NIH) will host its annual Rare Disease Day to raise awareness for rare disorders and advance research for new treatments. The event will be hosted on the NIH Main Campus and virtually live streamed, and will feature panel discussions, rare disease stories, and more.

• Global Genes Rare Drug Development Symposium, April 29–May 1, 2024

  The RARE Drug Development Symposium, hosted by Global Genes, will take place in Philadelphia April 29–May 1, 2024. This year’s theme is Innovative Ideas from Next Generational Change-Makers and will focus on equipping advocates with the knowledge, skills, and connections they need to advance therapy development for their communities.