Orphan Drugs & Rare Diseases - Conference Day 2

• Major challenges that each stakeholder encountered during the pandemic
• Lessons learned from Covid-19 and how we can benefit from it?
• How to build specific needs, support, and recommendations for sustainable healthcare systems that
  provide directions for better preparedness in the future?

• This presentation will address the challenges to rare disease drug development, the hurdles facing drug
  discovery and development, and frontiers for potential solutions.

Neena Nizar, President & Founder, The Jansens Foundation

• Describe FDA Patient Affairs Staff and patient engagement at FDA
• Share opportunities for patients, caregivers and advocates to engage with the Agency and get their
  perspectives heard
• Provide resources available to help patient communities connect with and navigate the Agency

Andrea Furia-Helms, Director of the Patient Affairs Staff, Office of Clinical Policy and Programs, Office of
the Commissioner, U.S Food and Drug Administration

• How to improve patient engagement during studies while collecting standardized, regulatory-grade data
• clinical and hybrid studies to improve real-world data collection for drug development

Dr. Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe

A better understanding of long-term trends in a specific patient population,
What makes data good data?
How does one harness unstructured data to provide value?
Can you comment on the importance of data standards and ontologies?

• How to close the gap between regulatory approval and funding agreement?
• How do the key HTA and payers operate?
• Samples of high profile reimbursement battles
• How to overcome struggles to meet the strict cost-effectiveness threshold for new therapeutics?

1. TABLE 1 - Challenges and Opportunities in developing a treatment for rare oncology
2. TABLE 2 - Value of partnering with advocacy organizations to create innovative patient-driven
   solutions
3. TABLE 3 - Patient-Centric Clinical Development: The importance of patient education for rare
   disease and advanced therapy trials
4. TABLE 4 - Building a meaningful partnership in rare disease communities
5. TABLE 5 - Strategy and Value of Early Access in the US and EU

• What mobile research nursing is all about.
• Utilizing mobile research nursing in pediatric trials.
• Giving the child control over their participation in the trial.

Senior Representative, Illingworth Research

• The foundational value of building collaboration through trust, respect, transparency, and empathy
• Not all advocacy organizations are alike: assessing the landscape
• Real-world examples of innovative digital solutions driving collaboration among multiple stakeholders

• Breaking down barriers for access
• Strengthening health systems around the world and accelerate pathways to diagnosis
• Stakeholders to address the rare diseases' ecosystem gaps to collaboratively build a sustainable roadmap
  for better health and a brighter future for the patients suffering from rare diseases.
• Challenges and opportunities in creating sustainable healthcare for all.