As Rare Disease Day 2023 is approaching, on behalf of the Chinese Organization for Rare Disorders, it is my honor to invite you to attend the Global Workshop on Access to Rare Disease Diagnosis and Treatment, on 19:00-22:30 Beijing Time, 28th February 2023.Founded in 2013 by Kevin HUANG, the Chinese Organization for Rare Disorders (CORD) functions as a platform, hub and advocacy NGO focusing on rare diseases. It is one of the largest NGOs in the realm of rare diseases in China. With rare disease patients’ interests as our first concern, CORD works to promote information exchange and cooperation among rare disease patients and organizations, medical specialists, pharmaceutical companies and governmental agencies. It is committed to enhancing public understanding of rare diseases, improving patient access to orphan drugs, fostering formulation of rare disease policies, and initiating international exchange and cooperation, to empower and help rare disease patients to live with hope, dignity and equity in our society.CORD is one of the global partners of Rare Disease Day. This year, we would like to hold a live workshop via Zoom. Focusing on access to RD treatment and diagnosis, it intends to attract panelists and listeners from various countries with diverse background and proficiency in the area of RD. We hope to provide an opportunity for stakeholders to discuss the latest global experience in improving patient access to RD therapies.For a detailed agenda of the workshop, please refer to the picture below.Please see the link below to book the eventbrite:https://www.eventbrite.com/e/global-workshop-on-access-to-rare-disease-diagnosis-and-treatment-tickets-542495868747 We look forward to meeting you at our Global Workshop.Chinese Organization for Rare Disorders (CORD)Host: Eileen Li, Senior Advisor, CORDSpeakers: Kevin Huang, President, CORD; Secretary General, Hope for Rare Foundation19:10 Current Headwinds in Reimbursement of Cell and Gene Therapy DrugsSpeakers: Alvin Luk, PhD, MBA, CCRA,Description: Reimbursement of drugs from pharmaceutical companies today occurs on a per-unit basis, which spreads out the costs of drugs for chronic conditions over years. However, the cost of a cell or gene therapy would be much complicated as it is potentially in a single payment with the unknown of benefits and risks in a long term. This presentation will explore the payment challenges for payers and healthcare providers (such as Medicare, Medicaid, BlueCross/BlueShield, etc.) as well as several alternative payment mechanisms.19:35 Value Assessment and Payment of Gene Therapy: Challenges and SolutionsSpeakers: Yang Xie, PhD, Vice President, Head of Real World Solutions, Asia Pacific & Head of HEOR/RWE, IQVIA Greater China.Description: In recent years, the development and approval of gene therapies have brought hope for patients with complex diseases. However, the value assessment and payment of those gene therapies involve with many challenges due to the high cost and uncertainty in research and development. This presentation will explore the challenges of value assessment and payment for gene therapies, the special assessment, access, and payment methods exclusively designed for gene therapies internationally. By analyzing cases of gene therapies for rare diseases, this presentation will also provide insights into improving accessibility for gene therapies in China.20:00 Access to Rare Disease Treatment in ChinaSpeakers: Linguo Li, Researcher, Public Policy Research Center, CORDDescription: This presentation will look into national and local practices of improving access to rare disease treatment in China. Examples include such efforts as The First Catalog of Rare Diseases in China, NRDL policies covering orphan drugs, and multiple national and local reformation on orphan drug importation and approval.20:25 How to Improve Access to Better Treatment through Digital Methods + Q&ASpeakers: Catherine Lamy & Sandra Bouarroudj, Co-president, Let’s Cure ACCDescription: This presentation will provide window into how rare disease patients can develop an international patient community and advocate for better treatment. Based on their own experiences, the panelists will discuss how to use digital methods to connect patients and experts from different countries, and how to fight for better healthcare and research resources in daily life and in academia/industry.21:00 Sanofi’s Commitment in Rare DiseasesSpeakers: Laurence Roux, Global Rare Diseases Market Lead, SanofiDescription: This presentation will briefly introduce Sanofi’s devotion to rare disease diagnosis and therapy, as well as their accessibility.21:05 Reducing Diagnostic Delay in Rare Diseases in France: AccelRare®+Q&ASpeakers: Anne-Sophie Chalandon, Global Public Affairs for RD/RRB, SanofiDescription: This presentation will introduce AccelRare® - an IA pre-diagnostic solution that Sanofi developed in partnership with multiple stakeholders (under Unir in France) to accelerate RD diagnosis. It is still in a pilot phase in France, but the ambition is that the tool is extended in Europe and globally.21:25 Building the 'Casa dos Raros' in BrazilSpeakers: Antoine Daher, President, FEBRARARASDescription: The project “Casa dos Raros” is conceived by Antoine Daher and Dr. Roberto Giugliani, who aspire to design and construct RD specialized health care centers in different parts of Brazil. This presentation will explore the designs of the project, the contexts and processes of the first center’s construction, as well as the project’s future development.21:50 The Rare Disease Trust Fund in Singapore (tbd.)Speakers: Rajev Rajakanth, Executive Director, Rainbow Across Border22:00 Systematic Solution to Accessibility and affordability of Rare Disease Treatment: Case study of JapanSpeakers: Yukiko Nishimura, Board Member, ASridDescription: Based on its special health and welfare systems, as well as specific measures on diagnosis, drug approval and reimbursement, the Japanese government offers systematic support for rare disease patients. This presentation will scrutinize those rare disease policies in Japan and how they have been working for patients. The presentation will also discuss how the systematic solution can be developed in the future from patient’s perspective.Speakers: Kevin Huang, President, CORD
