Orphan Drugs & Rare Diseases - Conference Day 2
Day 2: Wednesday - 20th October 2021
- 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
- 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?
- TABLE 1 - Challenges and Opportunities in developing a treatment for rare oncology
- TABLE 2 - Value of partnering with advocacy organizations to create innovative patient-driven
- TABLE 3 - Patient-Centric Clinical Development: The importance of patient education for rare
disease and advanced therapy trials
- TABLE 4 - Building a meaningful partnership in rare disease communities
- 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.