Clinical Development

Clinical Development & Regulatory Track, Wednesday 25 April 2018





last published: 19/Mar/18 14:25

Clinical Development & Regulatory 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

Clinical Development & Regulatory Track

Genetic Testing, Genome Sequencing

Access & Trials

Beverly Harrison
Clinical Development & Regulatory Track

The evolution of a partnership to help decision making and improve patient access to investigational medicines

  • Exploring ethical issues surrounding access to investigational products before FDA approval 
  • Working with patient groups to help demystify pre-approval access and create community-specific resources
  • Developing a group approach to ethical decision medicine – pharma and ethicists working together  
Karen Kaucic
Clinical Development & Regulatory Track

The virtual opportunity in rare disease trials

  • Solving specific challenges with virtual trials and their supporting technologies 
  • Reducing patient burden through siteless data capture and evidence generation 
  • Exploring the promise and pitfalls of telemedicine 
Angi Robinson
Clinical Development & Regulatory Track

Early development planning for orphan drugs: tasks, timelines, and takeaways

  • Organizing orphan drug designations: Where do they fit in and how long do they take?
  • Understanding natural history studies: purpose, design, and obtaining meaningful data
  • Preparing for an IND/IMPD and related clinical studies

Christopher Austin
Clinical Development & Regulatory Track

IRDiRC goals 2017-2027 – new research goals to accelerate rare disease therapies and diagnostics globally

  • Spearheading patient diagnosis by having currently undiagnosable individuals enter a globally coordinated diagnostic and research pipeline 
  • Working toward the approval of 1000 new rare diseases therapies focused mainly on diseases without approved options
  • Development of methodologies to assess the impact of diagnoses and therapies on rare disease patients
Clinical Development & Regulatory Track

Sponsored Talk


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

Data, Big Data and Real-World Evidence

Jacqueline Corrigan-Curay
Clinical Development & Regulatory Track

From real world data to real world evidence: FDA’s perspective on the enhanced use of RWE

  • RWE goals to have regulatory decisions incorporate data/evidence from settings that more closely reflect clinical practice
  • RWE expectations within 21st Century Cures and PDUFA VI 
  • FDA’S experience with RWE, collaborations and the assessment of data fitness and standards 
  • Impact of the inclusion of RWE in regulatory decision-making and the assessment of orphan drugs
Ashish Dugar
Clinical Development & Regulatory Track

Filling post market evidence gaps to support approval decisions and reimbursement

  • Thinking about post approval data collection before approval
  • Designing new studies and looking into real world evidence, patient reported outcomes and natural history studies 
  • Developing an evidence strategy that assesses the current reimbursement, regulatory and commercial landscape
Tiina Urv
Clinical Development & Regulatory Track

From helping trial recruitment to developing natural history studies – creating patient registries that are meaningful in drug development

  • Patients, industry and other stakeholders’ contribution to produce better quality registry data 
  • What technology developments exist to optimize data collection, analysis and curation?
  • What are the characteristics of a high quality registry?
Tiina Urv, Program Director, Office of Rare Disease Research, National Center for Translational Sciences, National Institute of Health



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