The Role of a Rare Disease Organization in Promoting Patient Advocacy
Based on current reports, nearly 80% of rare diseases are genetic, with approximately 70% appearing in childhood.
About 95% of these diseases lack approved treatments, and the average time for an accurate diagnosis is 4.8 years. Sadly, around 30% of children with a rare disease pass away before the age of five. There is currently no global standard definition for rare diseases.
In the United States, a condition is categorized as rare when it affects less than 200,000 people, whereas in the European Union (EU), a rare disease impacts fewer than 1 in 2000 individuals. Despite their rarity on an individual basis, rare diseases collectively affect over 300 million people around the world.
Despite their infrequency, these diseases profoundly impact the lives of those affected, presenting unique challenges in diagnosis, treatment, and everyday living. In this situation, rare disease organizations come into play.
This is the story of a three-year-old Ravi, diagnosed with muscular dystrophy, experienced worsening symptoms that left him wheelchair-bound.
His sister Geetha showed similar signs at 2.5 years old, prompting their doctor’s parents to seek a diagnosis. Despite extensive testing, including a negative test for Spinal Muscular Atrophy (SMA), they remained undiagnosed for 13 years.
Ravi and Geetha excelled academically, with Ravi pursuing law and Geetha aiming to be a programmer. Their story underscores the challenges of diagnosing rare diseases and the importance of support networks like IndoUSrare.
These dedicated patient advocacy groups are active patient advocates, not just names on a list. They ensure that the voices of those with rare diseases are heard, their needs are met, and their rights are protected.
Rare disease organizations are making a tangible difference in the lives of individuals with rare conditions through a comprehensive approach that includes awareness campaigns, support services, policy advocacy, and research initiatives.
Their work is improving healthcare and fostering a more inclusive and responsive healthcare environment.
The Role of IndoUSrare in Promoting Patient Advocacy
In this blog, we will explore IndoUSrare’s multifaceted role in championing patient rights, fostering community, and driving research and policy changes forward.
Boosting the Voice of Patients
One of the primary roles of IndoUSrare is to amplify the voices of patients and their families. It acts as a foundation for rare diseases providing platforms for patients to share their stories, experiences, and challenges. They create a collective voice that is harder to ignore by bringing together individuals with similar conditions.
For example, patient advocacy groups like the National Organization For Rare Diseases (NORD)’s advocacy led to the passage of the Orphan Drug Act in 1983, which provides incentives for developing treatments for rare diseases. This legislation has approved numerous drugs that might not have been developed otherwise due to the small market size for rare diseases.
These groups often use social media, online forums, and traditional media channels to amplify patient stories, educate the public and policymakers, and mobilize support for research and treatment access.
Patient registries and patient-centered research initiatives also empower individuals to contribute directly to scientific understanding and treatment development for their conditions. These efforts collectively enhance visibility, support, and resources for patients facing rare diseases.
Engagement in clinical trials and drug development
IndoUSrare is pivotal in engaging patients in clinical trials and drug development, ensuring that the patient perspective is incorporated at every process stage. We serve as a bridge between researchers, pharmaceutical companies, and patients, facilitating recruitment, retention, and the design of patient-centric studies.
Regular communication between the biotech company, researchers, advocacy groups, and patients throughout the trial helps maintain transparency and trust. Patients provide valuable feedback on their experiences, influencing aspects of the trial such as protocol adjustments and developing patient-reported outcome measures.
Ultimately, the collaboration results in a successful trial outcome, demonstrating the treatment’s efficacy and safety. This success story advances scientific knowledge and treatment options and strengthens the community’s trust and engagement in future research efforts for rare diseases.
Driving Research and Innovation
Research is a cornerstone of progress in the realm of rare diseases. Rare disease organizations often fund and support research initiatives to better understand these conditions and develop new treatments.
We, at IndoUSrare, collaborate with academic institutions, pharmaceutical companies, researchers, and government agencies to drive innovation.
Patient registries and biodatas are among the essential resources managed by these organizations. Collecting and sharing data accelerates research efforts and facilitates clinical trials.
Conclusion
Rare disease organizations are vital advocates for a population that often goes unheard of. By amplifying patient voices, providing crucial support and resources, driving research, and influencing policy, they make a tangible difference in the lives of those affected by rare diseases.
Their work improves individual patient outcomes and contributes to a broader understanding and awareness of rare conditions within the healthcare system and society.
As we strive for a more inclusive and equitable healthcare system, the role of rare disease foundations in promoting patient advocacy cannot be overstated. Their dedication, passion, and relentless advocacy pave the way for a brighter future for those with rare diseases. Their efforts ensure no patient is left in the shadows and every voice is heard.
Also Read: Why Patient Advocacy Organizations are Essential to Families Coping with Rare Diseases
