Day 2 Congress

• What are the key drivers and innovation that will steer the innovation and access of orphan drugs?
• Existing regulations and policies utilized by countries to enable access to orphan drugs
• Overcoming challenges in getting rare diseases’ patient access to orphan drugs
• Multi-disciplinary approach and patient centered support to improve accessibility and further advances which will impact the next generation aiding patients of the future.

• Identifying barriers for access to orphan drugs
• Addressing the challenges associated with patient access to these medicines
• High cost associated with the medicines: Is paying for expensive treatment for a few patients sustainable?
• Means of ensuring access and affordability of Orphan drugs
• Establishing a reasonable price for orphan drugs

• Patient recruitment and retention for a successful clinical trial in rare diseases
• Innovations proven to streamline the conduct of clinical studies
• Preparing healthcare providers to know about the therapy and identify the right patients for your therapy?

• How to improve knowledge and recognising a rare condition for patients to gain a sense of ease and trust
• Addressing issues on practical challenges
• Improving access and supporting patients

• How can stakeholders work together to remove barriers to patient access
• How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
• What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?
• Effective collaboration contributing to faster and wider access of medicines to patients

• What are some of the strategies for stakeholders to come together to get orphan drugs to patients especially in emerging countries?
• How can stakeholders work together to promoted international collaborations in developing orphan drugs and make it more accessible to all patients?

• Using initiative to drive market access to products for rare diseases
• Challenges surrounding funding for orphan drugs specifically for HTA
• What manufacturers should consider after successfully overcoming the hurdles to achieve market access

• Key concerns payers have about an orphan drugs
• What Set of Criteria Should be Considered for Orphan Drugs to Reach Patients with Rare Diseases in a Shorter Period of Time?
• How can reimbursement agencies deal with the uncertainty on efficacy of drugs but yet allow drugs to be available to patients as soon as possible?
• Vital strategies in maximizing the chances of success

• How can manufacturers meet global patient demand for access to medicines prior to approval?
• How can governments and orphan drug companies help support each other’s initiative to provide market access to patients?
• What are some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the market?
• What R&D strategies can be implemented to accelerate the market access for orphan drugs?

• Payers are becoming more aggressive in scrutinizing price and implementing restrictions to patient access that have traditionally been reserved for competitive, high budget impact drug classes
• Recent development in orphan drugs pricing and reimbursement and the impact of those changes
• Addressing some of the major issues facing payers and orphan drug manufacturers, highlighting the way these have been handled by both parties.
• Implementing measures designed to limit exposure to costs associated with orphan drug reimbursement.

• Successful example of value-based pricing in Rare Disease
• Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
• Implementing measures designed to limit their exposure to costs associated with orphan drug reimbursement.
• Legislative incentives to encourage development of orphan rare diseases therapies
• Impact of orphan diseases treatment on healthcare payment system

End of Day 1