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
Commercial
12:15

The Behind the Seizure program - an innovative, cross-company collaboration that aims to provide faster diagnosis for young children with epilepsy by making it easier to access genetic testing

Robin Sherrington, Senior Vice President Business and Corporate Development, Xenon Pharmaceuticals Inc
Alexis Braun, Diagnostics Brand Lead, North America LSD, Biomarin Pharmaceuticals Inc.
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

Scott Schliebner
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)

Vikram Karnani
Commercial
15:40

Commercial analytics – how to use data to overcome challenges in rare diseases

Vikram Karnani, Chief Commercial Officer, Horizon Pharma
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: 24/Mar/19 01:45

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

Karen Anderson

ROUNDTABLE 13: Disease burden – characterizing the burden of disease to more effectively educate regulators, physicians and payers

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?

Scott Schliebner

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

Carolyn Chapman
14:30

ROUNDTABLE 20: Compassionate Use--- Ethical considerations for pre-approval access to gene therapy products

Zizi Imatorbhebhe
14:30

ROUNDTABLE 21: Moving from n=1 to marketing application – clinical and regulatory considerations for accelerating rare disease product development

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

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

16:15

End of Conference

last published: 24/Mar/19 01:45