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, Thursday 11 April 2019

07:00

7:00 Registration and Breakfast

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

Patient data matter - perspective of people living with a rare disease on data sharing and protection

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
14:30

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
14:30

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

14:30

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

Vipul Kashyap
14:30

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

Harsha Rajasimha
14:30

ROUNDTABLE 14: Bridging US and India - strategies to accelerate rare diseases clinical research and patient recruitment

Paul Melmeyer
14:30

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

Leone Atkinson
14:30

ROUNDTABLE 19: Update on patient centricity in research – preliminary survey results from patient advocacy group leaders

Anne Cropp
14:30

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

Carol Houts
14:30

ROUNDTABLE 21: Inhouse versus outsource – determining the right orphan drug manufacturing fit for your organization

Scott Gray
David Lapidus
14:30

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

Joseph Musumeci
14:30

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

Amy Raymond
14:30

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
14:30

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

Lisa Dilworth
14:30

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

Kate Grady
14:30

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

14:30

ROUNDTABLE 9: Developing and Commercializing Orphan Drugs: Outsourcing and CMC Strategies

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

Heather Marton
Rare Disease Advocacy World
16:40

Molecular methods to improve the selection of clinical trial participants

17:00

5:00 AFTERNOON NETWORKING BREAK

18:30

6:30 NETWORKING DRINKS RECEPTION – Sponsored by Orchard Therapeutics

last published: 10/Apr/19 04:25

Rare Disease Advocacy World, Friday 12 April 2019

07:30

7:30 Registration and Breakfast

Jeff Ajer
08:50

Keynote Address: Preparing for a new treatment paradigm - gene therapy access and affordability

Stuart Bell
09:10

Keynote address: A staged approach to European patient access

10:05

Keynote address – PLATINUM SPONSOR

10:15

10:15 MORNING NETWORKING BREAK

Amanda Bok
Rare Disease Advocacy World
11:15

The role of patient advocacy in ensuring access to effective and transformative therapies

Kristin Smedley
Rare Disease Advocacy World
11:40

Shining a light on the financial and societal burden of blindness – a patient groups’ role in demonstrating value of treatment to all stakeholders

Laura Trutoiu
Rare Disease Advocacy World
12:05

One organization, three diseases - bringing together common Cerebral Creatine Deficiency Syndromes to build a platform for research

Mike Porath
Rare Disease Advocacy World
12:30

250,000 Voices: What really matters -- data and insights from patients and caregivers

12:50

12:50 NETWORKING LUNCH

14:30

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

Tim Boyd

ROUNDTABLE 10: State Policy – continued efforts to advance rare disease policy and initiatives

Theodore Roth

ROUNDTABLE 11: Alternative to viral vectors – reprogramming human T cell function and specificity with non-viral genome targeting

Melissa Hogan

ROUNDTABLE 12: Gene therapy consortium – elevating patient groups’ collective knowledge of gene therapy and working with companies though the first ever patient group gene therapy consortium

Ashish Dugar

ROUNDTABLE 13: Post-market data – executing collection to meeting regulatory requirements and payer needs

Roser Francisco Bordas

ROUNDTABLE 14: Rare in Catalonia – Regional policies for an integrated health care in rare diseases

Samir Shaikh
Jeremy Cress

ROUNDTABLE 16: Advanced manufacturing – building a cGMP in-house cell & gene therapy facility

Christina Poschen

ROUNDTABLE 17: Gene therapy commercial readiness - how to get yourself commercially ready for gene therapy. Practicalities of access agreements, concierge services and technology stewardship

Christina Poschen, Associate Consultant, Partners4Access
Jay Weaver
14:30

ROUNDTABLE 18: Sustainable pricing – will value-based payment models be sustainable for the healthcare system?

Amy Raymond

ROUNDTABLE 1: Gene therapy development – challenges, Issues, and solutions from the new frontier of rare disease therapies

Rich Franco
14:30

ROUNDTABLE 20: Expanded Access Programs - when it’s about survival, the transition from study to EAPs can’t be overmanaged

Doug Pfaff

ROUNDTABLE 2: Rare Disease Forecasting - Key drivers of uncertainty and how to deal with imperfect data

Han Phan

ROUNDTABLE 3: Site selection – how can dedicated rare disease trial sites enable studies to be conducted on a timely and effective manner

Carolyn Chapman
14:30

ROUNDTABLE 5: Compassionate Use – Ethical considerations for pre-approval access to gene therapy products

ROUNDTABLE 5: Excessive pricing - a deep dive into US and European approaches, recent cases and possible implications for industry and pricing strategies

Trista Morrison

ROUNDTABLE 6: Patient education - The importance of educating the patient community about rare diseases and newly approved and advanced therapies

Christopher Chute

ROUNDTABLE 8: Analyzing ICD-11 – challenges and complications around coding for rare diseases in the healthcare setting and understanding if the new ICD-11 will aid rare diseases coding

Pamela Gavin
15:35

Patients role in defining Centers of Excellence & standards of care

Pamela Gavin
Rare Disease Advocacy World
15:35

Patients role in defining Centers of Excellence & standards of care

Rare Disease Advocacy World
16:05

Topic to be confirmed

16:15

End of Conference

last published: 10/Apr/19 04:25