COMMERCIAL

 

How to commercialize rare disease products? This is the major question addressed on our commercial program. Hear form companies who are taking a unique approach to areas in commercialization including innovative business models, global expansion, and commercial strategies.

 

Key Speakers

 

Commercial, 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

Denise Scots Knight
Commercial
11:25

An innovative business model: harnessing ‘the strength of small’ to advance orphan drugs & rare disease therapies through to commercialization

Scott Dorfman
Commercial
11:50

Developing a commercial business model for ultra-rare disease

Daniel Anderson
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)

Chris Murphy
Commercial
15:40

Generating commercial insights and addressing data gaps across the patient journey using machine learning

Emma Viscidi
Commercial
16:00

Epidemiologic methods for rare diseases: Identifying patients using real-world data and literature

Emma Viscidi, Associate Director Of Epidemiology, Biogen
Alexander Cole
Commercial
16:20

What’s next? New frontiers for rare disease epidemiology in big data, genomic sets, and FDA and EMA guidance

Commercial
16:40

Gold Sponsor Slot

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

Commercial, 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

Mike Blum
Commercial
11:15

Commercial insights during early product development

Thomas Lester
Commercial
11:40

How to approach global expansion after initial approval and how strategy may change over time

Marshall Summar
Commercial
12:05

Industry and hospital collaboration - Preparing hospitals to administer therapies by having policies and procedures in place along with pre-positioned, highly flexible, multidisciplinary team rare dis

Oved Amitay
Commercial
12:30

From epidemiology insights to high-risk population screening: innovative genetically based approaches in a commercial framework

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

16:15

End of Conference

last published: 10/Apr/19 04:25