RARE DISEASE ADVOCACY WORLD

 

Rare Disease Advocacy World will feature advocacy program, patient groups, companies & agencies discussing some critical topics including patient data considerations, genetic testing and genomic sequencing and the evolving role of patient groups.

 

Key Speakers

 

Rare Disease Advocacy World, Wednesday 10 April 2019

last published: 15/Jan/19 21:25

Rare Disease Advocacy World, Thursday 11 April 2019

Ellen Coleman
08:25

Chairperson's opening remarks

Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
John Maraganore
08:30

Keynote Address: Advancing RNAi therapeutics as a whole new class of medicines

Christopher Austin
08:55

Keynote Address: Re-thinking and reshaping “rare” – continuing the march towards curative therapies for the rare disease community

Arndt Rolfs
09:20

Keynote Address: Accelerating Orphan Drug development and improving outcomes, by unlocking genetic information

10:30

10:30 SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK

Anne Pariser
Rare Disease Advocacy World
11:25

RADAR, the NIH registry initiative – Clinical trial readiness through stronger natural history data

Yann Le Cam
Rare Disease Advocacy World
11:50

Patients Data Matters - key learnings from the first ever qualitative and quantitative study on data sharing for people living with a rare disease

Jennifer Thornton
Rare Disease Advocacy World
12:15

Owning your own data – lessons learned from establishing a direct-to-participant data platform

Oodaye Shukla
Rare Disease Advocacy World
12:40

Integrating social determinants of health to increase adherence

13:05

1:05 NETWORKING LUNCH

2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min) – SPONSORED ROUNDTABLES AVAILABLE

Tiina Urv

ROUNDTABLE 10: Data ownership – addressing the need for increased data sharing while also considering company/patient data "ownership" through clinical trials and beyond

Tara Mathiesen

ROUNDTABLE 11: Educating HCPs – when considering the absence of newborn screening lessons learned from Metachromatic Leukodystrophy (MLD)

Walter Kowtoniuk

ROUNDTABLE 12: Chilling investment? – the potential implications of increased public scrutiny of rare disease drug prices in the US

Vipul Kashyap

ROUNDTABLE 13: Cross-Ecosystem collaborations – mutual value propositions and collaborations for pharma, payers and providers to collaborate in the rare diseases space

Harsha Rajasimha

ROUNDTABLE 14: Rare in India – engaging patients with rare diseases in India with global clinical trials and resources

Paul Melmeyer

ROUNDTABLE 15: Federal Policy – update on rare disease federal policy and NORD initiatives to engage capital hill

Susan Robinson
14:30

ROUNDTABLE 18: Market Access & commercialization – how specialty services and options should vary for patient populations of 20, 2,000, and 20,000

Anne Cropp

ROUNDTABLE 1: Global expanded access – applying artificial intelligence and access innovation for expanded access programs

David Lapidus

ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases

Joseph Musumeci

ROUNDTABLE 4: BD & forecasting – how to build forecasts based on available epidemiological information and considerations for pricing and access

Scott Schliebner

ROUNDTABLE 5: Patient-focused research – new approaches to bring clinical trials directly to patients and change the patient-centric trial paradigm in rare diseases

Jamie Arnott

ROUNDTABLE 6: Patient-centered Development: Early patient input, understanding patient goals in clinical trials, and furthering relationships between all stakeholders

Lisa Dilworth

ROUNDTABLE 7: Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians and regulators during advanced therapies clinical development

Kate Grady

ROUNDTABLE 8: Driving diagnosis – steps industry can take to improve the diagnosis of rare disease

14:30

ROUNDTABLE 9: Sponsored by Catalent Pharma Solutions

Matthew Silva
14:31

ROUNDTABLE 20: Combining imaging and genomics analysis – integration of multi-modal data analysis to accelerate and de-risk drug development in rare diseases

Matthew Silva, Executive Vice President, Scientific Applications, Invicro Llc
Brigette Tippin Davis, Senior Vice President, R&D, Ambry Genetics

ROUND 2 (select a second roundtable to attend from the options above)

Annie Kennedy
Rare Disease Advocacy World
15:40

Decode Duchenne: how free genetic testing, counseling and a strong patient registry are supporting advances in Duchenne and Becker Muscular Dystrophy diagnosis, research and education

Kimberly Leblanc
Rare Disease Advocacy World
16:00

The role of genetic counselors in educating rare disease patients and families about genetic testing, data and diagnosis

Linn Parrish
Rare Disease Advocacy World
16:20

Bringing an end to the diagnostic odyssey through genetic testing and partnership - Outcomes from the Global Commission partnership

17:00

5:00 AFTERNOON NETWORKING BREAK

18:30

6:30 NETWORKING DRINKS RECEPTION

last published: 15/Jan/19 21:25