The rare disease field is experiencing remarkable scientific progress in research and drug development. With over 50 new rare disease drugs and more than 10 potentially curative gene therapies on the horizon—many offering the first-ever treatment options—there’s real hope for countless patients.
However, despite these advancements and the hard work of advocates to amplify the diagnosis, care, and treatment of rare patients, far too many conditions are under-researched.
Rare disease is finally on the public health radar, thanks to the tireless efforts of rare disease patient advocacy groups (PAGs). These groups have helped open the door for R&D and commercial organization innovation that addresses unmet patient needs.
At IndoUSrare, these voices are the cornerstone of our mission to bridge care gaps, accelerate research, and foster a more inclusive healthcare ecosystem.
Advocacy-Driven Research
Rare disease advocacy has significantly influenced research priorities and methodologies. Patient advocates have pushed for increased funding, establishing patient registries, and adopting innovative research models such as adaptive, modern, and AI-driven clinical trials.
For instance, the efforts of the Global Genes, driven by patient advocacy, led to the development of breakthrough therapies that have transformed the lives of those living with this condition.
Advocates participate in study design and data collection to ensure that research is patient-centered and addresses pressing unmet needs.
Such collaborations have paved the way for personalized medicine, opening doors to treatments tailored to individual genetic profiles.
Policy Change and Access to Care
Advocacy is also a powerful driver of policy change. Patients and advocacy groups have successfully lobbied for rare disease legislation, such as the Orphan Drug Act in the United States and similar initiatives globally.
These policies incentivize pharmaceutical companies to invest in developing treatments for rare diseases that might otherwise be deemed unprofitable.
In India, patient advocacy has spurred the creation of National Rare Disease Policy 2021 to improve access to diagnosis and treatment. For example, organizations like the IndoUSrare Indian advocacy groups have brought patient voices to the forefront, leading to policy improvements.
Yet, significant challenges remain, particularly in ensuring equitable access to care for marginalized communities. This is where cross-border collaborations, like those facilitated by IndoUSrare, play a critical role.
By sharing best practices and resources, we aim to address disparities and ensure that rare disease patients worldwide receive the care they deserve.
Building Communities of Support
Rare diseases can be isolating, not only due to their medical complexities but also because of the lack of community understanding. Advocacy helps to bridge this gap by fostering communities where patients and families can connect, share experiences, and find support.
At IndoUSrare, we have seen the power of these connections through our patient support groups and events like Indo US Bridging Rare Summit 2024.
This recent Summit has played a vital role in tackling global disparities in rare disease research, clinical trials, and treatment access.
“Key areas like patient advocacy, robust patient registries, decentralized trials, and regulatory innovation are essential to involving India and its diaspora in the orphan drug revolution,” says Harsha Rajasimha, PhD, Founder and Executive Chairman of IndoUSrare.
The Summit will alternate in Washington, DC, in Nov 2025. For speaking and sponsorship opportunities, visit https://summit.indousrare.org.
Advocacy and the Future of Rare Diseases
The future of rare disease advocacy is intertwined with technological advancements, big data, and precision medicine. Digital tools enable patients to document and share their health journeys more effectively, while data analytics uncover patterns that accelerate diagnoses and treatment development.
However, the human element remains irreplaceable. Advocacy ensures that technology serves patients, not the other way around.
By keeping patient needs and experiences at the forefront, advocates help to shape ethical and inclusive innovations that benefit all.
A Call to Action
As we reflect on the incredible strides made by patient advocates, we are reminded of the work still ahead. Advocacy is not a solo endeavor; it requires a collective effort from patients, families, researchers, healthcare providers, policymakers, and organizations like IndoUSrare.
Join Us in advocacy efforts in breaking down barriers, challenging stigmas, and create a world where rare disease patients are seen, heard, and supported.
To the advocates leading this charge: your voices are catalysts for change. You are speaking for yourselves and countless others who share your journey.
IndoUSrare is proud to stand with you, amplifying your voices and working alongside you to transform the landscape of rare diseases.
Let us continue to listen, learn, and act. Together, we can ensure that no rare disease patient feels forgotten and that every voice has the power to inspire change.
Read More: How Rare Disease Advocacy Groups Are Driving Research and Innovation
