Conference Agenda

18 Orphan Drugs & Rare Diseases 2022 Americas - Day 2

Day 2: Friday - 23rd September 2022

  • 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

  • NC 1st RDAC & Most Effective
  • One Size Doesn’t Fit All
  • How the RDACs Benefit States and the Rare Disease Community


Tara J. Britt, Founder and President, Rare Disease Innovations Institute, Inc.


Patricia Weltin, CEO/Founder, Beyond the Diagnosis


  • TBC

Anne B. Cropp, Pharm.D., Chief Scientific Officer, Early Access Care LLC

  • TBC

Philip J. Brooks, PhD, Program Director, Office of Rare Diseases Research, NCATS, NIH

  • LHON, a rare disease
  • Lumevoq clinical development and regulatory pathways
  • Main results of the pivotal studies
  • Key learnings

Magali Taiel, M.D., Chief Medical Officer, GenSight-Biologics

2:00 - 2:30, 2:35 - 3:05 pm - ROUND TABLE DISCUSSIONS - divided between 2 sessions of 30 mins. (5 mins. to change table) - Attendees have 2 round table options to attend

  • Briefly discuss Patient Reported Outcomes
  • Discuss barriers to engagement and trial participation
  • Discuss pre-trial PRO engagement as a strategy
  • Wearables and PRO’s
  • Overcoming HIPAA and other barriers

Samuel L. Seward, Jr., MD, FACP, Prof & Chair, Mount Sinai Morningside & West, Dept of Medicine

  • Lack of ICD-10 code impact on rare disease patients
  • The current process
    A clean slate for rare disease patients
  • Codes for all rare diseases or
  • Insight from the cancer community on the value of ICD-10 codes


Tara J. Britt, Founder & President, Rare Disease Innovations Institute, Inc.


Khrystal K Davis, JD, Founding President, Texas Rare Alliance

  • 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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