Friedreich's Ataxia Research Alliance

What it reported doing in 2024

Clinical research infrastructurein addition to research grants, fara funds the ongoing development of domain resources in the form of vital clinical research infrastructure. Clinical research infrastructure refers to the resources needed to facilitate any type of research, including clinical trials that involve patients. These resources include programs like: friedreich's ataxia global patient registry: the friedreich's ataxia global patient registry (fagpr) is the only worldwide registry of friedreich's ataxia patients. The goals of the fagpr are to collect information on all fa patients in one registry, to develop the registry into a powerful resource for research, and to engage the fa community in studies aimed at advancing our knowledge (continued in schedule o)clinical research infrastructure (cont'd): of fa and the treatments being developed. Fara partners with international patient advocacy organizations through a governance board to ensure multi-stakeholder engagement and oversight of the fagpr. More than 1000 individuals with fa are enrolled and fagpr was used to recruit for several clinical trials and many clinical research studies. To learn more, visit curefa.net/registry. Fa global clinical consortium: fara and the fa community's dedication to collecting natural history study data over the past two decades proved instrumental in the approval of the first-ever treatment for fa. Through the fa global clinical consortium (fa gcc), fara has redoubled its investment in natural history data by enabling the transition to an industry-leading data collection platform and a unified global natural history protocol (unifai) resulting from the harmonization of two well-established natural history studies, the fa clinical outcome measures study (fa-coms) and the european friedreich's ataxia consortium for translational studies (efacts). The fa gcc and the unifai study have been expanded to make patient contributions to natural history even more powerful in understanding fa, evaluating the impact of treatment options for fa, accelerating the development of new therapies and improving outcomes for those living with fa. At each unifai study visit there are more than 1400 data elements recorded. Over the last two years, the fa gcc had active participation from 54 investigators from 33 sites representing 18 countries along with representatives from patient advocacy and research organizations. Fa gcc research activities: the fa gcc leadership identified initial scientific priorities and created workgroups to address these priorities. The following work groups, each made up of 5-10 consortium members, met regularly to address gaps in current fa research: cardiac natural history, late-stage symptoms, pediatric / presymptomatic, bio-samples, mood and cognition, and patient advocacy/advisory team. Quarterly full group meetings serve as a platform for investigators to establish common research interests, form collaborations, and share their global experiences with omaveloxolone. Impact and future direction: the consortium has a long-term objective of fostering multilateral research and collaboration across continents, unifying global opinions to regulators and industry partners, accelerating the development of new therapies, and improving outcomes for those living with fa. Additional information about fara's programs in 2024 can be accessed via the annual report at: curefa.org

Financial Efficiency

• Mission deployment rate: 95.7%; rating input score 96
• Program expense ratio: 94.4%; rating input score 94
• Fundraising efficiency: $36.69 raised per $1 spent on fundraising; rating input score 70; 70th percentile among comparable filings

Transparency & Controls

• Consistency checks: 100%; rating input score 100; 62nd percentile among comparable filings
• Pay-setting controls: 2 of 6 controls in place; rating input score 33
  • Compensation committee: Pass
  • Independent consultant: Missing
  • Other Form 990s reviewed: Missing
  • Written employment contract: Missing
  • Compensation survey or study: Missing
  • Board or committee approval: Pass
• Foreign activity oversight: 2 of 8 controls in place; rating input score 25
  • Claims monitoring procedures: Pass
  • Requires narrative reports: Limited
  • Conducts field audits: Limited
  • May suspend future funding: Limited
  • Requires financial reports: Pass
  • Conducts site visits: Limited
  • Requires grant agreements: Limited
  • May require repayment: Limited
• Grant itemization coverage: 99.9%; rating input score 100; 39th percentile among comparable filings
• Major donor itemization: Yes; rating input score 100
• Related-party transparency: Limited

Accountability

• Independent voting board: 89.5%; rating input score 89; 22nd percentile among comparable filings
• Governance controls: 9 of 9 controls in place; rating input score 100
  • Conflict-of-interest policy: Pass
  • Whistleblower policy: Pass
  • Document-retention policy: Pass
  • CEO compensation review process: Pass
  • Other executive compensation review process: Pass
  • Form 990 shared with the board: Pass
  • Board meeting minutes documented: Pass
  • Audit committee oversight: Pass
  • Independent financial-statement audit: Pass

Sustainability

• Operating margin: -1.1%; rating input score 26; 26th percentile among comparable filings
• Total-asset coverage ratio: 0.52; rating input score 17; 17th percentile among comparable filings
• Liabilities to assets ratio: 0.1; rating input score 74; 74th percentile among comparable filings
• Officer compensation ratio: 6.5%; rating input score 39; 39th percentile among comparable filings

Growth

• Program expense growth: 10.3% per year; rating input score 100; 71st percentile among comparable filings
• Revenue growth: 10% per year; rating input score 100; 74th percentile among comparable filings
• Asset growth: 7.8% per year; rating input score 100; 54th percentile among comparable filings