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
08:25
Chairperson's opening remarks
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
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
12:15The 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, Executive Vice President, Xenon Pharmaceuticals
Alexis Braun, Diagnostics Brand Lead, North America Lsd, Biomarin
13:05
1:05 NETWORKING LUNCH
2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min) – SPONSORED ROUNDTABLES AVAILABLE
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
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
14:30
ROUNDTABLE 13: Cross-Ecosystem collaborations – mutual value propositions and collaborations for pharma, payers and providers to collaborate in the rare diseases space
14:30
ROUNDTABLE 14: Bridging US and India - strategies to accelerate rare diseases clinical research and patient recruitment
14:30
ROUNDTABLE 15: Federal Policy – update on rare disease federal policy and NORD initiatives to engage capital hill
14:30
ROUNDTABLE 16: Internal organization and policy - data sharing and ownership, internal structure and funding, scientific advisors and more
Alexander Kosenko, Union of Patients' and Patients' Organizations with Rare Diseases Russia
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
14:30
ROUNDTABLE 1: Global expanded access – applying artificial intelligence and access innovation for expanded access programs
14:30
ROUNDTABLE 21: Inhouse versus outsource – determining the right orphan drug manufacturing fit for your organization
14:30
ROUNDTABLE 3: Epidemiology & forecasting – value, challenges, and methods for rare diseases
14:30
ROUNDTABLE 4: BD & forecasting – how to build forecasts based on available epidemiological information and considerations for pricing and access
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
14:30
ROUNDTABLE 6: Patient-centered Development: Early patient input, understanding patient goals in clinical trials, and furthering relationships between all stakeholders
14:30
ROUNDTABLE 7: Meaningful endpoints – navigating the differences in what it means to patients, caregivers, physicians and regulators during advanced therapies clinical development
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
14:30
ROUNDTABLE 9: Planning for orphan drug supply – the importance of manufacturability
ROUND 2 (select a second roundtable to attend from the options above)
Commercial
15:40Generating commercial insights and addressing data gaps across the patient journey using machine learning
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
18:25
Keynote Closing Remarks
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
08:45
Chairperson’s opening remarks
08:50
Keynote Address: Preparing for a new treatment paradigm - gene therapy access and affordability
09:10
Keynote address: A staged approach to European patient access
09:30
Keynote Panel: Improving access and affordability of new orphan drugs while ensuring continuous investment in rare disease innovation and R&D
Moderator: Jim Lang, Chief Executive Officer, EVERSANA
Daniel Faga, Chief Business Officer, Spark Therapeutics
10:05
Keynote address – PLATINUM SPONSOR
10:15
10:15 MORNING NETWORKING BREAK
Commercial
11:15Commercial insights during early product development
Commercial
11:40How to approach global expansion after initial approval and how strategy may change over time
Commercial
12:05Industry 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
Commercial
12:30From 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
ROUNDTABLE 10: State Policy – continued efforts to advance rare disease policy and initiatives
ROUNDTABLE 11: Alternative to viral vectors – reprogramming human T cell function and specificity with non-viral genome targeting
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
ROUNDTABLE 13: Post-market data – executing collection to meeting regulatory requirements and payer needs
ROUNDTABLE 14: Rare in Catalonia – Regional policies for an integrated health care in rare diseases
ROUNDTABLE 15: FDA Patient Affairs - Learn new ways to share your perspective with the FDA
Andrea Furia-Helms, Director, Patient Affairs Staff, U.S. Food and Drug Administration (FDA)
ROUNDTABLE 16: Advanced manufacturing – building a cGMP in-house cell & gene therapy facility
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
14:30
ROUNDTABLE 18: Sustainable pricing – will value-based payment models be sustainable for the healthcare system?
14:30
ROUNDTABLE 19: Real World Evidence – updates from FDA
ROUNDTABLE 1: Gene therapy development – challenges, Issues, and solutions from the new frontier of rare disease therapies
14:30
ROUNDTABLE 20: Expanded Access Programs - when it’s about survival, the transition from study to EAPs can’t be overmanaged
14:30
ROUNDTABLE 21: Moving from n=1 to marketing application – clinical and regulatory considerations for accelerating rare disease product development
Brad Gillespie, Principal Consultant, PAREXEL International
ROUNDTABLE 2: Rare Disease Forecasting - Key drivers of uncertainty and how to deal with imperfect data
ROUNDTABLE 3: Site selection – how can dedicated rare disease trial sites enable studies to be conducted on a timely and effective manner
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
ROUNDTABLE 6: Patient education - The importance of educating the patient community about rare diseases and newly approved and advanced therapies
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
14:30
ROUNDTABLE 9: Updates from Office of Orphan Product Development
15:35
Patients role in defining Centers of Excellence & standards of care
16:15
End of Conference
last published: 10/Apr/19 04:25
Organiser
