2017 EVENT AT A GLANCE

• The role of referencing and how this is carried out in selected regional regulatory agencies, including Singapore’s HAS
• Considerations and limitations of using the referencing approach for expedited approval tracks
• The role of patients in strengthening regulatory systems for referencing approach

• Orphan biologic candidates in biosimilar development
• Understanding the barriers to orphan biosimilar development
  • Regulatory considerations
  • Clinical development and operational challenges
• Key stakeholders’ influence on orphan biosimilars

·         Partnering with pharma

·         Navigating regulatory requirements

·         Clearing with customs

• Overview of orphan drugs in ASEAN countries – availability and accessibility
• Review of some industry examples implemented in various countries within the region – patient access schemes, shared-contribution model
• How strategic partnerships between industry, healthcare providers, government and patient groups can be leveraged for expanded access

• Heterogeneous perception of OD across countries from the APAC region
• Challenges & opportunities in patient access
• Patient advocacy efforts

• Korea - rare disease landscape
• The evolution of high quality sites & patient advocacy groups driving rare disease research in Korea
• A collaborative approach: the only way to address the challenges of rare diseases clinical research – Patient Access, Site Engagement & Innovative platforms  

• No-fail patient group power and campaigning for 500,000 signature to pass a senate bill on rare disease and orphan drugs
• Ten key provisions of RA 10747
• Report and updates on the Technical Working Group for the implementation of the law

• Past: doom and gloom
• Present: The DART story
• Future: accessible and affordable treatments

• Comparing the US orphan drug and rare disease environment with Asia
• Balancing the management of a foundation, family and advocacy
• Helping Duchenne patients in Malaysia

• Regulatory approval and market access policies in each of the countries
• What are the predominant diseases in the country? What are the known rare disease indications in the country? 
• Issues on rare disease awareness, affordability and political will
• Are rare diseases and orphan drug being discussed at all? What does the government think of it and what could be a potential next step towards a Rare Disease Act?
• What can patients, drug manufacturers and other stakeholders do to help?

• Present status and future concerns of newborn and expanded newborn screening in Asia
• Collaborating and cooperative networking to facilitate the development of sustainable newborn screening systems
• Identify creative funding mechanisms such as inclusion in maternal/child health packages and health/social insurance