Conference Agenda

Orphan Drugs & Rare Diseases - Conference Day 2

Day 2: Thursday - 2nd December 2021

8:00 am - Conference Registration and Continental Breakfast

8:30 am - Chairperson's Opening Remarks and Summary of Day 1

8:40 am - Keynote Panel Discussion: Implications of Covid-19 Pandemic to Rare disease community

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?

9:20 am - Plenary Session: Postcards from the Trenches: Lessons Learned from a Crises

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

9:50 am - Panel Discussion: Designing Effective Rare Disease Advisory Councils

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

10:30 am - Spotlight Presentation: What does generating evidence and real-world data (RWD) really mean?

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

11: 00 am - Morning Networking & Refreshments

11:30 pm - Spotlight Presentation: Global early access programs to support rare indications

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

12:00 am - Moving from clinical trials for ‘one disease at a time' to ‘many diseases at a time’

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

12:30 pm - Spotlight Presentation: Lumevoq gene therapy in Leber Hereditary Optic Neuropathy

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

1:00 pm - Networking Lunch Break

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

TABLE 1 - Challenges and Opportunities in developing a treatment for rare oncology

TABLE 2 - How to involve patients in research trials and developing Natural History

Tom James Lambdin, Clasina Fields, Co-Executive Directors, Bensfriends.org

TABLE 3 - Understanding the changes in approvals and the adaptabilities of deviations from standards for orphan drugs and rare diseases

Eugean Jiwanmall, Sr Research Analyst, Med. Policy & Tech. Evaluation, Independence Blue Cross

TABLE 4 - Rare Disease Value Assessments – are we ready?

Simu Thomas PhD, Vice President, Global Head HEOR, Alexion Pharmaceuticals

TABLE 5 - Strategy and Value of Early Access in the US and EU

3:10 pm - Afternoon Networking Break

3:40 pm - PRO’s—early pre-trial engagement of patients in understanding what’s important

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

4:10 pm - Panel Discussion: ICD-10 Codes for all Rare Diseases

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

4:40 pm - Closing Panel Discussion: Global collaborations to improve diagnosis and care for persons living with a rare disease

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.

5:20 pm - Chairperson's Closing Remarks