GLOBAL MARKET & PATIENT ACCESS

 

The Global Market & Patient Access track will feature authorities, companies and patient organizations disucssing market specific regulatory updates, hurdles, and additional access frameworks for advances therapies.

 

Key Speakers

 

Global Market and Patient Access, 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

Philippe Auvaro
Global Market and Patient Access
11:25

JAPAN: leveraging a comprehensive value creation platform to successfully enter and navigate the Japanese rare disease market

Amin Hussain Al Amiri
Global Market and Patient Access
11:50

UAE: updates on market access regulation, rare disease clinical trial enrollment, and patient populations

André Liamas
Natalia Fernandez Fruttero
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)

Ailsa Brown
Global Market and Patient Access
15:40

SCOTLAND: Improved Access - allowing three years of patient access to medicines for ultra-rare conditions in NHS Scotland and creating the opportunity to collect real-world data for reimbursement

Alba Ancochea Díaz
Global Market and Patient Access
16:00

SPAIN: Opportunities to engage authorities, regional agencies and patient organizations to improve access in Spain

Anant Murthy
Global Market and Patient Access
16:20

EU & CANADA: Working with international authorities on novel genetic medicines

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

Global Market and Patient Access, 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

Janet Rae
Global Market and Patient Access
11:15

US, EUROPE, CANADA: Working with regulatory bodies through the pre-approval stage for gene therapies

Kevin Huang
Global Market and Patient Access
11:40

CHINA: Patient access, working with companies, expanding regional access to clinical trials and the evolving relationship between patient groups, companies and regulatory authorities

Yukiko Nishimura
Global Market and Patient Access
12:05

JAPAN: Patient Reported Outcome efforts to help clinical development and company-patient relationships in Japan

Global Market and Patient Access
12:05

Working with international regulators through an ever-changing cell and gene therapy regulatory and approval landscape

Martine Zimmermann
Global Market and Patient Access
12:30

AUSTRALIA: Experience with the regulatory framework for orphan drugs

Global Market and Patient Access
12:30

Gold Sponsor Slot

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