RARE ONCOLOGY

 

The Rare Oncology track will feature agencies, developers, and patient organizations who are in the trenches advancing rare cancer awareness, diagnosis, and treatment.

 

Key Speakers

 

Rare Oncology, 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

Samit Hirawat
Rare Oncology
11:25

Opportunities and challenges in developing medicines for rare cancers

Jerry Collins
Rare Oncology
11:50

National Cancer Institute initiatives to tackle rare cancer and foster drug development

Jeff Allen
Rare Oncology
12:15

Recent policy initiatives that have helped facilitate drug development for rare cancer population

Jorge Franceschi
Rare Oncology
12:40

Disruption in oncology clinical trial administration provides cost savings, efficiency and risk mitigation

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)

Denise Reinke
Rare Oncology
15:40

Uniting rare cancers patients to advance diagnosis, data collection and drug development

Denise Reinke, President and CEO, SARC
Rare Oncology
15:40

Uniting rare cancers patients to advance diagnosis, data collection and drug development

Corrie Painter
Rare Oncology
16:00

Count Me In - accelerating research for rare cancers through patient-partnered genomic studies

Rare Oncology
16:00

Panel tests – when and how to use, working with providers, physicians and patients to ensure best practices

Rare Oncology
16:00

Panel tests – when and how to use, working with providers, physicians and patients to ensure best practices

Rare Oncology
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

Rare Oncology, 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

Jaydira Del Rivero
Rare Oncology
11:15

Addressing challenges in rare cancer research to support development of new treatments: The My Pediatric and Adult Rare Tumor (MyPART) network at NCI

Jaydira Del Rivero, Medical Oncology & Clinical Endocrinology, Director CCR Rare Tumor Clinic, Rare Tumor Initiative, Medical Oncology Service/Pedia, National Cancer Institute - NIH
Michael Cox
Rare Oncology
11:40

Developing drugs against rare oncogenic targets in pediatric populations - considerations in patient identification, patient recruitment, and working with regulators

Sanjeev Luther
Rare Oncology
12:05

Identifying and developing treatment for rare cancers - orphan drug designation, and the challenges of moving into clinical development and beyond

Pascal Touchon
Rare Oncology
12:30

Bringing Kymriah to patients globally - A unique experience

Rare Oncology
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