Seventy-eighth World Health Assembly – Daily update: 24 May 2025

Rare Diseases: A New Era of Global Health Priority

Imagine a world were over 300 million people, many of them children, are no longer left behind. That’s the promise of a new, landmark resolution declaring rare diseases a global health priority.

A Turning Point for millions

For too long, rare diseases have been overlooked, leaving patients and their families to navigate a complex and often isolating journey. This resolution marks a pivotal shift, recognizing the significant physical, emotional, and financial hardships faced by those living with these conditions.

The Scope of the Challenge

Did you know? There are over 7,000 identified rare diseases, most of which begin in childhood. These conditions can range from genetic disorders to autoimmune diseases,each presenting unique challenges for diagnosis and treatment.

Speedy Fact: In the United states, a disease is considered rare if it affects fewer than 200,000 people.

What Does the Resolution Entail?

This isn’t just a symbolic gesture.The resolution urges countries to take concrete steps to integrate rare diseases into national health planning. This includes improving diagnosis and care through global health coverage, promoting inclusive policies, and accelerating innovation, research, and access to affordable treatment.

Key Actions for Member States

• Integration into National Health Planning: Ensuring rare diseases are considered in healthcare strategies.
• Improved Diagnosis and Care: Leveraging universal health coverage to provide necessary support.
• Inclusive Policies: Creating supportive environments for patients and families.
• Accelerated Innovation and Research: Investing in the development of new treatments and therapies.

The WHO’s Role: A 10-Year Global Action Plan

Perhaps the most significant aspect of the resolution is the mandate for the World Health Organization (WHO) to develop a thorough 10-year global action plan on rare diseases. This plan will serve as a roadmap for countries worldwide, guiding their efforts to address the unique challenges posed by these conditions.

What to Expect from the Action Plan

The 10-year global action plan is expected to address key areas such as:

Expert tip: Look for the WHO to collaborate with patient advocacy groups, research institutions, and pharmaceutical companies to develop a holistic and effective action plan.

• Early Diagnosis: Implementing strategies for faster and more accurate diagnosis.
• Treatment Development: Incentivizing research and development of new therapies.
• patient Support: Providing resources and support for patients and their families.
• Data Collection and Surveillance: Improving data collection to better understand the prevalence and impact of rare diseases.

Impact on the United States

For Americans living with rare diseases, this resolution could mean significant improvements in access to care, research funding, and policy changes. The U.S. already has the Orphan Drug Act,which incentivizes the development of treatments for rare diseases,but this global effort could further accelerate progress.

Real-World Examples in the U.S.

Consider the case of cystic fibrosis (CF), a rare genetic disorder affecting the lungs and digestive system.Thanks to research and advocacy efforts, there have been significant advancements in CF treatment. This resolution could pave the way for similar progress in other rare diseases.

Challenges and Opportunities

While the resolution is a major step forward, there are still challenges to overcome. these include:

Potential Roadblocks

• Funding: Securing adequate funding for research and treatment development.
• Data Collection: improving data collection and surveillance to better understand the prevalence and impact of rare diseases.
• Access to treatment: Ensuring equitable access to affordable treatments, especially in developing countries.

Opportunities for Innovation

Despite these challenges,there are also significant opportunities for innovation. Advances in genomics, personalized medicine, and digital health technologies could revolutionize the diagnosis and treatment of rare diseases.

The Future of rare Disease Care

this resolution is more than just a declaration; it’s a call to action. It’s a promise to the millions of people living with rare diseases that they will no longer be forgotten. By integrating rare diseases into global health priorities, we can create a future where everyone has access to the care and support they need to thrive.

What are your thoughts on this resolution? Share your comments below and let’s continue the conversation.

Call to Action: Share this article to raise awareness about rare diseases and the importance of global action.

Landmark Resolution Declares Rare Diseases a Global Health Priority: An Expert Weighs In

New Hope for Millions: Understanding the Impact of the WHO’s Rare Disease Resolution

Millions of people worldwide live with rare diseases, often facing diagnostic delays, limited treatment options, adn a lack of awareness. A recent resolution declaring rare diseases a global health priority marks a meaningful turning point. To delve deeper into this crucial development, we spoke with Dr. Alistair Humphrey, a leading researcher in rare genetic disorders and professor of human genetics at the University of california, San Francisco.

Time.news: Dr. Humphrey, thank you for joining us. This resolution seems like a game-changer. Can you explain why it’s so significant?

Dr. Humphrey: Absolutely. For too long, rare diseases have been on the periphery of global health conversations. This resolution acknowledges that collectively, these diseases affect a massive number of people – over 300 million globally. It’s about legitimizing the challenges faced by patients and families while mandating concrete action from member states. This formal recognition is critical to driving focus and resources towards diagnosis, treatment development for rare diseases, and – importantly – rare disease patient support.

Time.news: The article highlights the sheer number – over 7,000 – of identified rare diseases. That’s overwhelming. How does this resolution practically address such a vast and diverse landscape?

Dr.Humphrey: The key lies in the framework it establishes. Instead of focusing on each disease individually,which is incredibly challenging given the diversity,the resolution promotes a broader approach. This includes integrating rare diseases into national health planning, leveraging worldwide health coverage to improve rare disease diagnosis and care, advocating for inclusive policies, and, of course, accelerating research and innovation.The WHO’s 10-year global action plan is precisely designed for this approach. The hope is to address the common challenges across all types of these afflictions.

Time.news: This 10-year action plan seems central to the resolution’s success. What specific outcomes can we expect from this initiative within the next decade?

Dr. Humphrey: We can anticipate progress across several critical areas.Firstly,the plan should focus on early diagnosis of rare diseases. This involves improving awareness among healthcare professionals, developing better diagnostic tools, and implementing screening programs where feasible. Secondly, we hope to to see more robust incentives for rare disease treatment development, encouraging pharmaceutical companies and research institutions to invest in this area. Thirdly, expanded resources and support networks for patients and their families, addressing not just medical needs but also the emotional, social, and financial burdens. improved data collection and surveillance to get a better handle on the true prevalence and impact of rare diseases.

Time.news: The article mentions the U.S. already has the Orphan Drug Act. How will this global resolution complement existing efforts in countries like the U.S.?

Dr. Humphrey: The Orphan Drug Act has been incredibly successful in stimulating rare disease research funding and treatment development. However, a global resolution can further strengthen these efforts by promoting international collaboration, standardizing diagnostic criteria, and facilitating the sharing of data and best practices. This international collaboration will be vital in areas like genetic testing standards and cross-border research collaboration.

Time.news: What are the biggest challenges in implementing this resolution effectively,and what can individuals or organizations do to help?

Dr. Humphrey: Funding is undoubtedly a significant challenge. Research, drug development, and patient support all require ample investment. Another challenge is data collection and surveillance. Many rare diseases are poorly understood, and accurate data on their prevalence and impact is lacking. Access to treatment, notably in developing countries and even marginalized communities within developed countries, is another hurdle. To help, individuals can support patient advocacy groups and research foundations dedicated to specific rare diseases. They can also advocate for policies that prioritize rare disease research and treatment. organizations can facilitate collaboration, share best practices, and invest in innovative technologies.

Time.news: What excites you most about this resolution, and what opportunities do you see for the future of rare disease care?

Dr. Humphrey: What excites me most is the recognition that rare diseases are not isolated problems but a global health priority that demands our attention and resources. With advances in genomics, personalized medicine, and digital health, we have unprecedented opportunities to revolutionize the diagnosis and treatment of these conditions. This resolution is a call to action, and if we work together, we can make a real difference in the lives of millions.

Time.news: Dr. Humphrey, thank you for your valuable insights.

Dr. Humphrey: My pleasure. this is an important conversation to keep having.