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, Wednesday 10 April 2019
last published: 19/Feb/19 22:15
Commercial, Thursday 11 April 2019
08:25
Chairperson's opening remarks
Ellen Coleman, President And Chief Executive Officer, VOZ Advisors
08:30
Keynote Address: Advancing RNAi therapeutics as a whole new class of medicines
08:55
Keynote Address: Re-thinking and reshaping “rare” – continuing the march towards curative therapies for the rare disease community
09:20
Keynote Address: Accelerating Orphan Drug development and improving outcomes, by unlocking genetic information
09:40
Keynote Panel: Advancing cell and gene therapy – considerations in manufacturing, R&D expertise, early payer engagement and continuous patient centricity
Moderator: Sophie Schmitz, Managing Partner, Partners4Access
10:30
10:30 SPEED NETWORKING FOLLOWED BY MORNING NETWORKING BREAK
Commercial
11:25An innovative business model: harnessing ‘the strength of small’ to advance orphan drugs & rare disease therapies through to commercialization
Commercial
11:50Developing a commercial business model for ultra-rare disease
Commercial
12:15Unlocking the value of genetics in drug development and commercialization
13:05
1:05 NETWORKING LUNCH
2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min) – SPONSORED ROUNDTABLES AVAILABLE
ROUNDTABLE 10: Data ownership – addressing the need for increased data sharing while also considering company/patient data "ownership" through clinical trials and beyond
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
ROUNDTABLE 13: Cross-Ecosystem collaborations – mutual value propositions and collaborations for pharma, payers and providers to collaborate in the rare diseases space
ROUNDTABLE 14: Rare in India – engaging patients with rare diseases in India with global clinical trials and resources
ROUNDTABLE 15: Federal Policy – update on rare disease federal policy and NORD initiatives to engage capital hill
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
14:30
ROUNDTABLE 18: Market Access & commercialization – how specialty services and options should vary for patient populations of 20, 2,000, and 20,000
14:30
ROUNDTABLE 19: Update on patient centricity in research – preliminary survey results from patient advocacy group leaders
ROUNDTABLE 1: Global expanded access – applying artificial intelligence and access innovation for expanded access programs
ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases
ROUNDTABLE 4: BD & forecasting – how to build forecasts based on available epidemiological information and considerations for pricing and access
ROUNDTABLE 5: Patient-focused research – new approaches to bring clinical trials directly to patients and change the patient-centric trial paradigm in rare diseases
ROUNDTABLE 6: Patient-centered Development: Early patient input, understanding patient goals in clinical trials, and furthering relationships between all stakeholders
ROUNDTABLE 7: Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians and regulators during advanced therapies clinical development
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)
Commercial
15:40Commercial analytics – how to use data to overcome challenges in rare diseases
Commercial
16:00Epidemiologic methods for rare diseases: Identifying patients using real-world data and literature
Emma Viscidi, Associate Director Of Epidemiology, Biogen
Commercial
16:20What’s next? New frontiers for rare disease epidemiology in big data, genomic sets, and FDA and EMA guidance
Commercial
16:40Gold Sponsor Slot
17:00
5:00 AFTERNOON NETWORKING BREAK
18:30
6:30 NETWORKING DRINKS RECEPTION – Sponsored by Orchard Therapeutics
last published: 19/Feb/19 22:15
Organiser
