Commercial Track

Commercial Track, Wednesday 25 April 2018

last published: 23/Mar/18 19:55

Commercial Track, Thursday 26 April 2018



Katherine High

Keynote address: A new era in medicine- introducing gene therapy

  • What we’ve learned – lessons science has taught us as we create the path for the first gene therapy for an inherited disease in the U.S.
  • Staying true to our roots while preparing the patient, medical and policy maker communities for a new chapter in the retinal disorders textbook
  • Assessing the impact of potential one-time treatments like gene therapies for patients with rare diseases
Hans Bishop

Keynote address: Cell and gene therapy innovation and their role in the future of rare disease research

  • Current state of cell and gene therapies and why this field is becoming more and more significant 
  • Looking into scalability and centralized approaches for cell and gene therapy development and commercialization
  • Novel approaches and how these therapies benefit the continuum of research for rare diseases
Andre Choulika

Keynote address: Next- generation CAR T-cells to cure cancer - paving a new way for R&D, value-assessment and pricing and reimbursement of treatments for genetic diseases

  • Learnings from CAR-T development that can be applied to rare disease drug development 
  • Will CAR therapy pricing impact pricing schemes of other orphan drugs and gene therapies? 
  • Exploring aspects of these different therapeutic areas that share common goals, personalization and pricing challenges 
Panel discussion

Keynote CEO Panel: Gene therapy - a new paradigm for patients, new challenges for industry

  • Looking back at the commercial performance of the first generation of gene therapies- from proof of concept to the road to approval 
  • How will gene therapy innovation look in the next 10 years?
  • Considering potential new value propositions for patients, payers and the health care system
  • Strengthening clinical development, exploring novel technologies while maintaining safety- what needs to be improved? 


Commercial Track

Business Model

Lara Sullivan
Commercial Track

Collaborative mission-driven approach to developing innovative potential new treatments for unserved and underserved patient communities

  • Addressing industry challenges of too much good science and insufficient resources to fully advance pipelines 
  • Aligning biopharma innovators with investigators, patient groups, and funders to advance promising investigational therapies 
  • Concept in Action: Big Pharma expertise and capabilities leveraged to enable company launch pipeline of four mid- and late -stage clinical assets, enabling strong Series A funding
Gregory Fond
Commercial Track

Navigating the rare disease innovation ecosystem and partnering for the long-term

  • Looking into new technology platforms and transformative new products
  • Nurturing relationships with academia, incubators, startups and biotechs to develop fruitful partnerships 
  • From due diligence to alliance management, realizing the potential of scientific innovation, and going beyond ROI
Mark Baglin
Commercial Track

Development and execution of commercial strategies for clinical and commercial stage RNAi products for serious, life-threatening diseases

  • Pre-launch strategies and building a rare disease commercial team from scratch
  • Launching successfully: different challenges, different approaches, same result
  • Achieving market access by placing a focus on prescribers, patients and payers
  • Global vs. local strategies
Alex La Croix
Commercial Track

The role of commercial in early development through launch

  • Leading market access strategies, KOL development, engagement and mapping out all scenarios for a drug’s entire life cycle prior to launch
  • Driving lifecycle expansion into other indications and geographies 
  • Managing and growing global commercial teams in rare disease


Yann Le Cam

Lunch keynote address: Breakthrough therapies call for breakthrough processes to ensure sustainable patient access


2:30 ROUNDTABLES (1-hour roundtable sessions split between 2 rounds of 30min – attendees can select two roundtables to attend)

If you are interested in sponsoring a roundtable contact André Singer at or +1 646 619 1797

ROUNDTABLE 1: Pediatric trials - reducing the financial, logistical and emotional burden for patients and families participating in pediatric trials

Andrew McFadyen

ROUNDTABLE 2: Compassionate Use Programs - helping patients get access to medication, educating the community, physicians and investors to ensure safe access to experimental treatments

Scott Schliebner

ROUNDTABLE 3: Virtual Trials - enabling clinical trials to be more virtual, reducing distance times, overcoming geographic barriers and increasing data collection through the use of wearables

Jayne Gershkowitz

ROUNDTABLE 4: Expanded access - incorporating the patient advocacy perspective to develop a review process and a formalized procedure on expanded access

Joseph Musumeci

ROUNDTABLE 5: BD & Forecasting - pricing and access considerations and understanding how to build the patient forecast based on available epidemiological information

Yvette Venable

ROUNDTABLE 6: Strategy - patient engagement and pipeline building for the commercialization and access of rare disease products

Francesca Cook

ROUNDTABLE 7: Gene therapy – new payment and financing models are needed to facilitate access

Michael Murphy

ROUNDTABLE 8: Clinical development – When the sum is greater than the parts through academic, pharmaceutical and CRO collaboration in orphan disease drug development

Robert Donnell

ROUNDTABLE 9: Managed Access Programs - How MAPs (pre-commercial supply to patients with unmet medical needs) can be a valuable component of launch strategy for orphan drugs

Samantha Parker

ROUNDTABLE 10: Gene therapy - Optimizing gene therapy clinical development through education, collaboration, patient input and involvement in advisory boards

Philip J. Brooks

ROUNDTABLE 11: Scientific innovation - platform approaches to evaluate multiple genetic diseases at the same time

David Lapidus

ROUNDTABLE 12: Epidemiology and forecasting - using epidemiological data to determine market viability, niche populations and commercial differentiation

Betsy Ricketts

ROUNDTABLE 13: Government affairs - developing a company’s footprint and contributions to impact policy, reimbursement and patient engagement

Anne Cropp

Roundtable 15: Expanded Access - drug development strategy considerations for open label extension or expanded access protocols

Ari Brettman

ROUNDTABLE 16: VC funding – successfully addressing investors interests and challenges when assessing companies of all sizes to guarantee funding of orphan drug biotechs

Trista Morrison

ROUNDTABLE 17: Disease awareness - best practices and tips for smaller companies to tell a corporate or rare disease story and raise awareness

Francesca Mcbride

ROUNDTABLE 18: Facility design – critical aspects to consider for clinical and commercial production of cell and gene therapy products

Francesca Mcbride, Director Regulatory Compliance, Jacobs


Emerging Markets


Walter Colasante
Commercial Track

Value assessment is different for Rare Diseases, requiring new thinking to build consensus around access decisions

  • Addressing divergent priorities from stakeholders in response to potentially curative gene and cell therapies, including physicians, regulators, patients, payers and manufacturers 
  • Unblocking the affordability conundrum by bringing stakeholders together to rethink the measurement of value and how this impacts wider societal and economic factors
  • The need for tailored planning that differs from more mainstream disease areas given the very distinct role that patients and advocacy groups play in rare diseases
Gary Geipel
Commercial Track

Spotlight on ultra-rare: commercializing therapies for very small patient populations

  • Lessons from the development and launch of Mepsevii for a disease affecting <200 patients
  • Accelerating approval, finding the patients, building a value story, and assuring access
  • Commercialization challenges posed in the ultra-rare- versus rare-disease space.
Adam Sohn
Commercial Track

Orphan drug launches – what drives success? How to best prepare for an orphan drug launch.

  • Launch excellence: how orphan drugs performance is associated with pre-launch preparedness and developed commercial awareness 
  • Performance: the impact of multiple levels of orphan drug differentiation and unmet medical needs
  • Not all orphan drugs are the same – how to leverage pre-approval initiatives, forecasting and epidemiology for commercial success




Douglas Ingram

Keynote panel: How rare disease companies are ramping up innovation to boost orphan drug development and patient centricity

  • Incorporating patients in the drug development process and working towards commercial viability- how to fully engage? 
  • How do you view disruptive therapies such as (gene therapy/gene editing) impacting the broader orphan disease development paradigm? How are you innovating within your company?
  • Responding to the changes in the political, regulatory and reimbursement landscape while guiding the direction of rare disease companies 
  • How to achieve industry collaboration and idea exchanges that can better serve the patient community? 

6:20 NETWORKING COCKTAIL PARTY – Reserved for Gold Sponsor

last published: 23/Mar/18 19:55