It's truly an honor to be invited to the 2024 Global Rare Disease Research Symposium & The Second China Rare Disease Research and Translational Medicine Annual Conference, a collaborative effort by Hope for Rare Foundation, the International Rare Diseases Research Consortium (IRDiRC), and Fudan University. This year's conference, held from May 23-25 in Shanghai, China, brought together over 100 speakers and around 3,000 attendees from global universities, hospitals, academic institutions, patient organizations, and pharmaceutical companies.
With 20 parallel sessions and several satellite meetings, the event covered a wide range of topics including basic research on rare diseases, gene and cell therapies, Investigator-Initiated Trials (IIT), clinical studies, drug development, and international research collaborations. This symposium highlighted the latest developments and original findings in rare diseases research, especially cell and gene therapies, with a special emphasis on China’s contributions in a global context.
There were many interesting sessions in the panel on international research collaborations. I had the chance to speak at the panel titled "Benefit-sharing: A Thorny Question for Biomedical Innovation," chaired by Dr. Yeyang Su. The session featured speakers who provided valuable insights into various aspects of benefit-sharing in biomedical innovation.
- Dr. Adam Resnick, the director of the Center for Data-Driven Discovery in Biomedicine at Children's Hospital of Philadelphia (CHOP), discussed strategies for establishing a network to facilitate children's brain tumor research.
- Prof. Dong Dong from the Jockey Club School of Public Health and Primary Care at the Chinese University of Hong Kong explored the ethical considerations surrounding care for rare disease patients.
- Prof. Wim Pinxten from Hasselt University provided insights into the ethical considerations on fair pricing and benefit sharing.
- Dr. Linguo Li, who is the director of public policy at the Chinese Organization for Rare Disorders, discussed the evolving role of patient organizations as innovators.
- I delved into the necessity for a new social contract in orphan drug development to achieve equitable returns among stakeholders, transparent pricing, and a sustainable healthcare system.
This conference underscored the importance of collaboration with patient organizations and innovation in advancing rare disease research. Our current project has investigated the growing divergence in orphan drug approvals between the EMA and FDA, highlighting the unequal availability across regions and the significant barrier posed by high prices. This situation is even more dire in middle and low-income countries. Innovation in pharmaceutical R&D is just the first step of the long journal to the patient access. Without innovative business and pricing models, patients cannot access treatments, wasting public resources since early-stage orphan drug R&D is often funded by public grants and patient organizations.
As we look ahead, the insights and connections gained from this symposium will propel our project to further develop the research on a new social contract of orphan drug development to tackle the challenges above. This new social contract will address several key issues:
Global Accessibility: Ensuring that orphan drugs are available in middle and low-income countries, not just in affluent regions.
Fair Pricing: Implementing innovative pricing models that balance affordability for patients and reasonable returns for companies.
Benefit-Sharing: Developing strategies where profits are shared more equitably among stakeholders, including patients, researchers, and pharmaceutical companies.
Public Resource Utilization: Ensuring that the public resources which often supports early-stage orphan drug R&D is effectively utilized by making treatments accessible to all and utilising the returns to address other unmet medical needs, thereby preventing wastage of resources and make the system sustainable.
I extend my sincere gratitude to the conference organizers: Hope for Rare Foundation, International Rare Diseases Research Consortium, Fudan University, and the Chinese Organization for Rare Disorders (CORD). The opportunity to exchange knowledge and insights has been invaluable.
