ADVANCED THERAPIES - CLINICAL

 

On the Advanced Therapies- Clinical track you will hear from companies, regulators and researchers currently developing call and gene therapies to treat rare diseases.

 

Key Speakers

 

Advanced Therapies - Clinical, Wednesday 10 April 2019

last published: 13/Feb/19 21:55

Advanced Therapies - Clinical, 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

Charlie Albright
Advanced Therapies - Clinical
11:50

Further defining the path to the clinic for in vivo gene editing

Deborah Gill
Advanced Therapies - Clinical
12:15

Getting to the clinic through strategic partnership – combining scientific, clinical development, and manufacturing skills to develop new treatments for Cystic Fibrosis patients in Europe

Mariah Baltezegar
Advanced Therapies - Clinical
12:40

Use of real world data to support the path to approval for advanced therapies in rare diseases

Michael Murphy
Advanced Therapies - Clinical
13:00

When predictable execution trumps brilliant planning: the interplay of strategic and transactional relationships in orphan disease drug development

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)

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

Clive Whitcher

ROUNDTABLE 17: Patient Access in Europe & Beyond - Enabling access for rare disease therapies exploring alternative pathways for patients in need

Clive Whitcher, Vice President Head of Global Patient Access, Inceptua Commercial Products
Kelly Fearn, Principal Consultant, Inceptua Medicines Access
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

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

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

Suyash Prasad
Advanced Therapies - Clinical
15:40

Advancing the Clinical Program Development of AAV-Based Gene Therapy for Rare Diseases

Barrett Katz
Advanced Therapies - Clinical
16:00

Advancing the clinical program development of gene therapy for ultra-orphan disease: finding the patients, getting them enrolled

Ilan Irony
Advanced Therapies - Clinical
16:20

FDA gene therapy draft guidance documents – disease specific guidances and long-term follow up

Advanced Therapies - Clinical
16:40

Unique challenges for expanded access in gene therapies – considerations for companies, patients

17:00

5:00 AFTERNOON NETWORKING BREAK

18:30

6:30 NETWORKING DRINKS RECEPTION – Sponsored by Orchard Therapeutics

last published: 13/Feb/19 21:55