Global Market Access Track


Global Market Access Track, Wednesday 25 April 2018





last published: 19/Mar/18 14:25

Global Market Access 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? 


Gene Track

Global Market Access Track

Genetic Testing, Genome Sequencing

Established Markets

Trevor Richter
Global Market Access Track

CANADA: Orphan drug evaluation and decision making in Canada in the context of international HTA decisions

  • Orphan drug evaluation at CADTH to reimburse, reimburse with clinical criteria/conditions and not reimburse
  • HTA challenges when there is no regulatory framework for orphan drugs in Canada and no international consensus for OD
  • Balancing evidence requirements with treatment’s cost 
Martine Zimmermann
Global Market Access Track

ITALY: The Italian experience on market access for rare disease drugs

  • What is needed to achieve regional access? How does Italy compare to other European countries? 
  • Looking into new rules to gain ‘innovative status’ 
  • Opportunities to market access through a more formalized process for early access and compassionate use requests 
Diane Kleinermans
Global Market Access Track

BENELUXA Belgium, Netherlands, Luxembourg and Austria joining forces to improve access and to orphan drugs and paving the way for more collaborations

  • Joint collaboration to negotiate with pharmaceutical companies the price of orphan drugs
  • Looking into data exchange, sharing of registries and coordinated assessment of new technologies and innovation 
  • Advantages in patient access and dossier submission for pharmaceutical companies 
Thomas Goss
Global Market Access Track

Market access challenges and opportunities for orphan and ultra-orphan drugs

  • Looking into the global demand for an evidence-based approach toward orphan drug pricing and reimbursement
  • Current landscape on pharmaceutical practices: undertaking burden of Illness studies, focusing on baseline quality of life, and direct and indirect costs to evaluate orphan drugs and assist with reimbursement
  • Tools to overcome the challenges faced by industry when identifying relevant data sources to document the cost and burden of orphan diseases 
  • Strengths and limitations of available methods of data capture


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


Emerging Markets

Kaye Robertson
Global Market Access Track

AUSTRALIA: Orphan drug program reforms: changes in the eligibility criteria of orphan drug designations

  • Expansion of the orphan disease prevalence threshold to allow more conditions to classify as orphan
  • Changes including the validity period of orphan drug designation before seeking approval, fee waivers, and additional criteria 
  • Transition period considerations for sponsors 
Kaye Robertson, Senior Medical Officer and Medicines evaluator, Prescription Medicines Assessment Branch, Therapeutic Goods Administration
Jarbas Barbosa da Silva Jr
Global Market Access Track

BRAZIL: Accelerating the review and approval of orphan drugs in Latin America’s largest market

  • Resolution to establish a procedure for clinical trials consent and good manufacturing practices
  • Guidelines for registration of new drugs for treatment, diagnosis or prevention of rare diseases
  • What this means for patients, drug manufacturers and the healthcare system 
Ayse Deniz Ozger
Global Market Access Track

TURKEY: Access to orphan drugs in a large market outside the European Union

  • Legislative efforts to define rare diseases and promote the development and commercialization of orphan drugs 
  • Channels available to obtain unapproved drugs in Turkey but available in the EU (off-label use, named- patient imports, compassionate use)
  • Working towards establishing national networks for the prevention, surveillance, treatment of rare diseases 



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: 19/Mar/18 14:25