The International Gaucher Alliance (IGA) marks its 30th anniversary with a five-year strategic plan to advocate for Gaucher patients globally. The plan underscores a commitment to advocacy and collaboration, aiming to drive tangible advancements in patient access, research, and community empowerment, according to GlobalData, a leading data and analytics company.

Gaucher disease, which is caused by defects in the GBA1 gene, results in the harmful accumulation of glucocerebroside, which may affect the liver, spleen and nervous system.

Sulayman Patel, MSci, Pharma Analyst at GlobalData, comments: “As we celebrate this significant milestone, the unveiling of the five-year strategic plan marks a pivotal moment in the journey to improve outcomes for Gaucher disease patients. This plan serves as a pledge to drive tangible improvements in outcomes for Gaucher disease patients globally.”

Key elements of the IGA’s strategic plan include initiatives to enhance patient access to diagnosis, treatment, and care. This involves the creation of online resources to pinpoint diagnostic centers worldwide, as well as the development of diagnostic pathways in underserved regions such as Africa. This will be further supported by collaborations with healthcare organizations to educate doctors on identifying signs and symptoms.

The IGA’s strategy encompasses a robust awareness campaign dubbed “Gaucher Red Flags”, aimed at alerting clinicians to key disease indicators. Collaborations across healthcare disciplines will ensure global coverage, thereby improving the timeliness and accuracy of Gaucher disease diagnosis.

Patel continues: “The strategic imperative extends to influence the Gaucher disease research agenda, addressing key unmet needs by promoting early engagement with pharmaceutical companies and advocating for accessible treatment options. By actively involving stakeholders in discussions, the IGA seeks to steer research towards addressing critical patient needs.”

Empowering and supporting IGA members and the wider Gaucher community worldwide forms the cornerstone of the final strategic imperative. This involves exploring the development of regional hub networks to overcome language and time zone barriers, strengthening governance structures, and expanding volunteer programs to enhance project delivery.

The unveiling of the IGA’s strategic plans harmonize with the upcoming Rare Diseases Day on 29 February 2024, underscoring the organization’s commitment to enhancing understanding, support, and resources for rare disease communities worldwide.

Patel concludes: “As we look ahead, the IGA remains steadfast in its commitment to drive meaningful change for Gaucher patients through advocacy, collaboration, and innovation. By supporting engagement amongst relevant stakeholders, the IGA has highlighted its pivotal role in improving patient outcomes around the world.”