Congenital Hyperinsulinism International

What it reported doing in 2024

CHI supports research and development to better understand, treat, manage, and hopefully cure HI. CHI shares the patient perspective with researchers and members of the biopharmaceutical industry to accelerate the development of patient-centered treatments. CHI is a part of the Million Dollar Bike Ride grant program with the University of Pennsylvanias Center for Orphan Diseases; a pilot research grant is offered each year for an innovative, preclinical or clinical study with the potential to lead to a better HI treatment, a cure for HI, or improvement in the quality of life for those affected by HI. In 2024, CHI was able to award two pilot research grants through this program.In 2024, the CHI Research Staff worked with a lead epidemiologist to publish The Birth Prevalence of Congenital Hyperinsulinism: A Narrative Review of the Epidemiology of a Rare Disease. This publication, published in Hormone Research in Pediatrics, reviews epidemiological data on HI as reported in the literature, identifying the strengths and limitations of each HI prevalence study.CHI conducts the HI Global Registry (HIGR) research project. HIGR provides a convenient online platform for the HI patient community to share their experiences of living with HI. With permission from the patient families, MaxHIGR provides complementary data from the physician perspective. By participating in the HI Global Registry, the patient community helps themselves and researchers better understand HI to advance better treatments, a potential cure, and more timely and accurate diagnoses. A HIGR report is published each year by the CHI staff. Scholars and research may request deidentified data for their research projects. In 2024, CHI continued to lead the Collaborative Research Network (CRN), a project of CHI dedicated to the development of faster and more accurate diagnoses, new evidence-based treatments and cures, standardized clinical guidelines, and increased and improved access to treatment, medication, devices, and supplies. This ambitious project includes working groups focused on different aspects of a prioritized research agenda (PRA) developed by the CRNs members including 60 leading researchers, clinicians, and patient advocates from 17 countries The CRN members have been focused on the top priorities identified in the PRA: Newborn Screening (Diagnose all babies with congenital hyperinsulinism in a timely manner); Glucose as a Vital Sign (Increase awareness and timely diagnosis of HI); Care Guidelines (Create and disseminate continually evolving global care guidelines); Natural History (Build a robust registry that collects patient-reported, physician, and real-world data to provide a foundation for HI natural history); Continuous Glucose Monitoring (Create principles for Draft guidelines on CGM use in HI patients). Since the launch of the CRN in 2020, the CRN members have published a total of 194 articles on congenital hyperinsulinism covering a wide range of topics including genetics, treatment, disease management, monitoring and technology, screening and diagnostics, clinical guidelines, education, patient experience, quality of life, policy, and access to care. In 2024, the CRN published an Advocacy Statement and List of Essential Medical Care, Medication, Supplies, and Services for people with congenital hyperinsulinism (HI). The CRN also published "Continuous glucose monitoring for children and young people with hyperinsulinism: a practical guide for families & professionals and CGM: a tool to support HI families". These guides are a collaboration of work by HI experts around the world, including many who are part of the CHI CRN and CHI staff. Patient and caregiver representatives of the CHI CRN provided the patient perspective. CHI continues to support the Open Hyperinsulinism Genes Program in partnership with the University of Exeter in the UK. This program is the first point-of-need international genetic testing service for HI and is also accelerating

Financial Efficiency

• Mission deployment rate: 75.1%; rating input score 75
• Program expense ratio: 88.1%; rating input score 88
• Fundraising efficiency: $3.48 raised per $1 spent on fundraising; rating input score 11; 11th percentile among comparable filings

Transparency & Controls

• Consistency checks: 86.7%; rating input score 87; 3rd percentile among comparable filings
• Pay-setting controls: Limited
• Foreign activity oversight: 1 of 8 controls in place; rating input score 12
  • Claims monitoring procedures: Pass
  • Requires narrative reports: Fail
  • Conducts field audits: Fail
  • May suspend future funding: Fail
  • Requires financial reports: Fail
  • Conducts site visits: Fail
  • Requires grant agreements: Fail
  • May require repayment: Fail
• Grant itemization coverage: 93.5%; rating input score 93; 41st percentile among comparable filings
• Major donor itemization: Yes; rating input score 100
• Related-party transparency: Limited

Accountability

• Independent voting board: 100%; rating input score 100; 63rd percentile among comparable filings
• Governance controls: 7 of 9 controls in place; rating input score 78
  • Conflict-of-interest policy: Pass
  • Whistleblower policy: Fail
  • Document-retention policy: Fail
  • 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: 11.7%; rating input score 64; 64th percentile among comparable filings
• Total-asset coverage ratio: 1.93; rating input score 64; 64th percentile among comparable filings
• Liabilities to assets ratio: 0.15; rating input score 36; 36th percentile among comparable filings
• Officer compensation ratio: 26.5%; rating input score 9; 9th percentile among comparable filings

Growth

• Program expense growth: 21% per year; rating input score 100; 84th percentile among comparable filings
• Revenue growth: 19% per year; rating input score 100; 84th percentile among comparable filings
• Asset growth: 20.3% per year; rating input score 100; 82nd percentile among comparable filings