Conference Agenda

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
    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
  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.

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