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
Moderator:
Tara J. Britt, Founder and President, Rare Disease Innovations Institute, Inc.
Panellist:
Patricia Weltin, CEO/Founder, Beyond the Diagnosis
- How do you generate RWD and evidence in support of your initiative to ensure meaningful results?
- What data types and structure is needed in support of your research?
- The important role of patients in evidence generation and how to keep them engaged?
Dr. Femida Gwadry-Sridhar, Founder & CEO, Pulse Infoframe
- 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
Tom James Lambdin, Clasina Fields, Co-Executive Directors, Bensfriends.org
Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech. Evaluation, Independence Blue Cross
Simu Thomas PhD, Vice President, Global Head HEOR, Alexion Pharmaceuticals
- 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
Moderator:
Tara J. Britt, Founder & President, Rare Disease Innovations Institute, Inc.
Panelist:
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.
