2018 Agenda

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Oxon Hill, MD, 25 - 27 April 2018

Schedule

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Apr 2608:30
Conference pass

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

Keynotes
  • 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
Apr 2608:30
Conference pass

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

Keynotes
  • 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
Apr 2608:55
Conference pass

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

Keynotes
  •     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
Hans Bishop, President and Chief Executive Officer, Juno Therapeutics
Apr 2608:55
Conference pass

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

Keynotes
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  •     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 

Hans Bishop, President and Chief Executive Officer, Juno Therapeutics
Apr 2609:20
Conference pass

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

Keynotes
    li>
  •     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  

Andre Choulika, Chairman & Chief Executive Officer, Cellectis
Apr 2609:20
Conference pass

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

Keynotes
    li>
  •     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  

Andre Choulika, Chairman & Chief Executive Officer, Cellectis
Apr 2609:45
Conference pass

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

Keynotes
  •     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? 
Apr 2609:45
Conference pass

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

Keynotes
  •     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? 
Apr 2611:30
Conference pass

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

Clinical Development & Regulatory Track
  •     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   
Apr 2611:30
Conference pass

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

Commercial Track
  •     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 
Apr 2611:30
Conference pass

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

Global Market Access Track
  • 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
Apr 2611:30
Conference pass

Genetic testing and patient engagement: faster time to diagnosis and access to research and treatments

Gene Track
  •     Removing barriers to genetic testing can help patients secure an accurate diagnosis more quickly
  •     Genetic testing programs drive patient identification, expedite trial enrollment and accelerate time to market for new therapeutics
  •     Creates engaged patient communities that partner biopharma and patient advocacy groups through Patient Insights Networks (PIN) 
Apr 2611:30
Conference pass

Empowered educated patients influencing pre-clinical development and working with industry to optimize drug discovery

Rare Disease Advocacy World
  • Patient groups role guiding research, disease understanding, supporting pre-clinical development and follow up experiments
  • Driving translational research and the influence on formulation, routes to delivery and overall clinical development
  • Effective partnerships between advocacy and industry to expand collaborations that can support enrollment and physician engagement
Apr 2611:30
Conference pass

VC Panel: Investing in the orphan drug space – derisking commercial models and driving biotechs to successful launches

Pitch & Partner
  • Partnering with key KOLs and advocacy groups at the outset to help facilitate a physician/patient company dynamic to help inform both regulatory and commercial strategy
  • Type of work and diligence to obtain comfort with the ultimate market opportunity
  • Assessing barriers to entry including therapeutic modality and IP
  • Regulators role in incentivizing or impeding the ability to develop products both domestically and abroad. What can be different?
Apr 2611:30
Conference pass

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

Clinical Development & Regulatory Track
  •     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   
Apr 2611:55
Conference pass

The virtual opportunity in rare disease trials

Clinical Development & Regulatory Track
  •     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  
Apr 2611:55
Conference pass

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

Commercial Track
  •     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 
Apr 2611:55
Conference pass

JAPAN: An orphan drug act isn’t enough: Understanding the changing pricing landscape, knowledge gaps and key aspects to access

Global Market Access Track
  • Once a pricing heaven, now threatened by increased reimbursement scrutiny, repricing and reassessment approaches
  • Current gaps in clinical development, registries and epidemiology practices
  • Looking into use of HTAs and higher evidence thresholds to support pricing and access
Apr 2611:55
Conference pass

The virtual opportunity in rare disease trials

Clinical Development & Regulatory Track
  • 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
Apr 2611:55
Conference pass

The virtual opportunity in rare disease trials

Clinical Development & Regulatory Track
  •     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  
Apr 2612:20
Conference pass

Evolving the relationships in the orphan drug ecosystem to achieve truly patient centric clinical research

Clinical Development & Regulatory Track
  •     True integration of the patient’s voice in the drug development process, through the product lifecycle and even through failed trials 
  •     Working with patient groups that have different objectives, while staying compliant
  •     How can the patient perspective change the way companies develop a new drug? 
Apr 2612:20
Conference pass

Expediting rare disease patient identification and a genetic testing-based diagnostics ecosystem to drive orphan drug adoption

Gene Track
  •     Development of a sustainable diagnostic system to help find rare disease patients 
  •     Turning the diagnostic paradigm on its head: using genetics for screening 
  •     Helping physicians reduce the time to diagnosis of the cause of metabolic cholestasis 
Apr 2612:20
Conference pass

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

Commercial Track
  • 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
Mark Baglin, Vice President of Global Marketing, Alnylam Pharmaceuticals
Apr 2612:20
Conference pass

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

Global Market Access Track
  • 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
Apr 2612:20
Conference pass

The post market patient and patient advocacy- Acknowledging that with approval, the work is not done

Rare Disease Advocacy World
  • How can patient groups keep encouraging orphan disease research and investments after approval?
  • Rare disease clinical development when approved treatments are available.
  • Opportunities to reduce delays to access prior to approvals
  • Post-marketing engagement to improve and expand patient access – engaging with multiple new stakeholders.
  • Working synergistically with industry counterparts to educate the physician community, delivery centers, and payers.
Apr 2612:20
Conference pass

Evolving the relationships in the orphan drug ecosystem to achieve truly patient centric clinical research

Clinical Development & Regulatory Track
  •     True integration of the patient’s voice in the drug development process, through the product lifecycle and even through failed trials 
  •     Working with patient groups that have different objectives, while staying compliant
  •     How can the patient perspective change the way companies develop a new drug? 
Apr 2612:40
Conference pass

Launching a commercial function from scratch – developing capabilities to enable rare disease product launches

Commercial Track
  • 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
Alex La Croix, Senior Director, Rare Genetic Disease Marketing, Agios Pharmaceuticals
Apr 2612:40
Conference pass

GERMANY: outcomes from the AMNOG reform into a more transparent pricing negotiation process

Global Market Access Track
  •     How Germany achieved an average lower reimbursement price for new pharmaceuticals than any other country in Europe 
  •     Outlook for orphan drugs in Germany including evidence requirements
  •     Should orphan drugs go through a full assessment process? 
  •     What can other European countries learn from Germany and the AMNOG process? 
Apr 2612:40
Conference pass

Using patients’ genomic information to uncover pathogenic mutations linked to rare diseases

Gene Track
  • Leveraging next generation sequencing and genome interpretation for rare disease research
  • Combining data science and systems biology to accelerate rare disease diagnosis and therapeutic discovery
  • Searching for and decoding rare disease “genomic shields”
  • Fostering partnerships to advance rare disease therapies 
Apr 2612:40
Conference pass

Inclusion of patient reported outcomes (PROs) in rare disease trial design

Rare Disease Advocacy World
  • Capturing patients’ bona-fide needs, preferences, and value drivers through systematic investigation
  • Adoption of end- to-end patient evidence generation pathways to increase the probability of success
  • Enabling payers and providers to better understand patients’ values
  • Developing meaningful patient reported outcomes and producing further evidence to support
Apr 2613:30
Conference pass

Lunch keynote address: Advanced therapies call for advanced collaborative processes to ensure patients access and market viability

Keynotes
Apr 2614:31
Conference pass

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

Keynotes
Apr 2614:31
Conference pass

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

Keynotes
Apr 2614:32
Conference pass

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

Keynotes
Andrew McFadyen, Executive Director, The Isaac Foundation
Apr 2614:32
Conference pass

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

Keynotes
Andrew McFadyen, Executive Director, The Isaac Foundation
Apr 2614:33
Conference pass

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

Keynotes
Apr 2614:33
Conference pass

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

Keynotes
Apr 2614:34
Conference pass

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

Keynotes
Apr 2614:34
Conference pass

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

Keynotes
Apr 2614:35
Conference pass

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

Keynotes
Joseph Musumeci, Senior Advisor, BluePrint Orphan
Apr 2614:35
Conference pass

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

Keynotes
Joseph Musumeci, Senior Advisor, BluePrint Orphan
Apr 2614:36
Conference pass

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

Keynotes
Yvette Venable, International Public Affairs Lead, Kyowa Kirin International
Apr 2614:36
Conference pass

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

Keynotes
Yvette Venable, International Public Affairs Lead, Kyowa Kirin International
Apr 2614:37
Conference pass

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

Keynotes
Francesca Cook, Director, Pricing and Market Access, RegenxBio
Apr 2614:37
Conference pass

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

Keynotes
Francesca Cook, Director, Pricing and Market Access, RegenxBio
Apr 2614:38
Conference pass

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

Keynotes
Apr 2614:38
Conference pass

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

Keynotes
Apr 2614:39
Conference pass

ROUNDTABLE 9: Trial Optimization - effective sourcing and distribution of comparator drugs ancillary supplies and placebos for orphan trials

Keynotes
Leslie Morgan, Managing Director, Durbin
Apr 2614:39
Conference pass

ROUNDTABLE 9: Trial Optimization - effective sourcing and distribution of comparator drugs ancillary supplies and placebos for orphan trials

Keynotes
Apr 2614:40
Conference pass

ROUNDTABLE 10: Gene therapy education- fostering transparency to achieve collaboration between internal and external stakeholders working to advance gene therapies

Keynotes
Apr 2614:40
Conference pass

ROUNDTABLE 10: Gene therapy education- fostering transparency to achieve collaboration between internal and external stakeholders working to advance gene therapies

Keynotes
Apr 2614:41
Conference pass

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

Keynotes
Apr 2614:41
Conference pass

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

Keynotes
Apr 2614:42
Conference pass

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

Keynotes
Apr 2614:42
Conference pass

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

Keynotes
Apr 2614:43
Conference pass

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

Keynotes
Betsy Ricketts, Senior Director, Government Affairs, Ultragenyx Pharmaceutical
Apr 2614:43
Conference pass

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

Keynotes
Betsy Ricketts, Senior Director, Government Affairs, Ultragenyx Pharmaceutical
Apr 2615:40
Conference pass

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

Clinical Development & Regulatory Track
  • 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
Apr 2615:40
Conference pass

Orchestrating a successful launch in the ultra-orphan space- what needs to be taken into account

Commercial Track
  • Setting up the appropriate commercial infrastructure in North America and Europe
  • Selection of CRO and third party logistics
  • Roll out the marketing and communication strategy with KOL engagement and patient connectivity
  • Defining and implementing the appropriate evidence-based pricing
  • Partnering options for Japan and rest of world
Apr 2615:40
Conference pass

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

Global Market Access Track
  •     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
Apr 2615:40
Conference pass

Creating value in gene therapy clinical development programs, achieving trial optimization and supporting the path to commercialization

Gene Track
  • Investigating novel vector technologies that can improve efficacy while reducing immunogenicity and potential re-dosing
  • Thinking about end to end clinical development including trial design, enrollment, execution, supply chain and manufacturing
  • How can clinical development be optimized to support scalability? What are the current limitations faced by this field despite a decade of improvements
Apr 2615:40
Conference pass

Beyond “patient focused drug development” buzzwords - let’s get practical

Rare Disease Advocacy World
  • Industry and patient partnerships for clinical trial design and execution, funding discussions and community  outreach
  • Patients as board members through the product lifecycle to impact the needs of the community
  • Partnering with patients in a compliant way and achieving support from government and academic institutions to drive scientific research
Apr 2616:05
Conference pass

CHINA: Better late than never – CFDA’s efforts to define rare diseases, encourage orphan development and accelerate the review process

Global Market Access Track
  • Publication of rare disease lists and working toward a patient registration system
  • Improvements to clinical regulatory data protection
  • Implementation of a US- style clinical trial application process- can this be achieved in the China?
Apr 2616:05
Conference pass

Gene editing approaches in rare blood disorders

Gene Track
  •     Unmet need for transformative therapies in hemoglobinopathies
  •     Disrupting an erythroid specific BCL11A enhancer with zinc finger nucleases for the treatment of sickle cell disease and beta-thalassemia 
  •     The potential to reactivate fetal hemoglobin to treat patients with SCD and beta-thalassemia 
Dana Levasseur, Leader and Associate Director, Bioverativ
Apr 2616:05
Conference pass

Filling post market evidence gaps to support approval decisions and reimbursement

Clinical Development & Regulatory Track
  •     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 
Apr 2616:05
Conference pass

A holistic view to sustainability in rare disease - key acquisitions, product differentiation, innovation and geographic expansion

Commercial Track
  • Complementing key portfolios with the acquisition of preclinical gene therapy assets and exploring novel platform technologies
  • Working with global teams, patients and scientists to develop long- acting fusion proteins for patients with bleeding disorders
  • Developing treatments with patients in mind and looking into achieving a global reach
Apr 2616:05
Conference pass

The holy grail of rare disease research – Framework for effective engagement with patient groups in clinical trials

Rare Disease Advocacy World
  •     Understanding current practices in patient groups-sponsors collaborations 
  •     Filling knowledge gaps regarding best practices for patient groups engagement in every phase of drug development 
  •     From fundraising to trial operations to helping with access- understanding the ecosystem  
Apr 2616:30
Conference pass

Real World Evidence 2.0 – from basic evidence collection to its inclusion in drug review applications

Clinical Development & Regulatory Track
  •     What is usable Real World Evidence?
  •     Simplifying collection, incorporating data into drug applications, and leveraging its potential to drive drug effectiveness 
  •     What are the necessary steps to incorporate RWE in the regulatory approval process? How can these be incorporated early on? 
Alastair Macdonald, Exec Director, RWLP Strategy, Global, INC Research/inVentiv Health
Apr 2616:30
Conference pass

Commercial forecasting of orphan drugs using epidemiological data to determine market viability

Commercial Track
  •     Identifying and understanding niche patient populations to meet regulatory and payers’ criteria and drive commercial differentiation
  •     Epidemiology as a tool to determine R&D budgets and remove commercial uncertainty 
  •     Guiding clinical development and trial location  
Apr 2616:30
Conference pass

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

Global Market Access Track
  •     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  
Ayse Deniz Ozger, Vice President, Healthcare Division, Eczacibasi Healthcare
Apr 2616:30
Conference pass

Developing programs and partnerships that go beyond medicine

Keynotes
  •     Patient and caregiver community as advisors coming together to create resources for patients
  •     Outputs in the form of educational resources, scholarships, memberships to attend conferences 
  •     Initiatives that support the integration of the unaffiliated patient into rare disease communities  
Apr 2617:35
Conference pass

Keynote address: Fostering innovation in rare disease and advancing the development of drugs for the rarest of diseases

Keynotes
  •     Developing a global vision for the commercialization of rare disease therapeutics
  •     Maintaining a clinical stage portfolio and working on nurturing pre-clinical stage programs
  •     Working hand in hand with advocacy and policy makers to support rare disease development efforts  
Apr 2618:00
Conference pass

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

Keynotes
  •     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?  

Create your personal agenda –check the favourite icon

Apr 2708:35
Conference pass

Keynote address: Developing genomic medicines and the first ever in- vivo human genome editing trials using zinc finger nucleases

Keynotes
  • Gene therapy, genome editing, cell therapy, gene regulation: choosing the best therapeutic platform to address a patient’s needs
  • Editing liver cells with ZFNs to potentially cure rare diseases, including MPS I, MPS II and hemophilia B
  • Zinc finger nuclease technology vs. CRISPR-Cas9: what’s important when developing genomic medicines
  • Zinc finger nucleases as a best-in-class tool to make cell therapies for cancer – Sangamo’s goal of allogeneic cell-based immuno-therapies
  • The next frontier: what does the future of genome editing look like? Delivery, capabilities, safety, ethics, etc.
 
Apr 2709:00
Conference pass

Keynote address: Advancing innovative therapies to treat genetically-defined diseases using CRISPR-based genome editing

Keynotes
  • Momentum toward making medicines – a potentially transformative new category of medicines that aim to repair “broken” genes
  • Strategy focused on diseases with few or no available treatments and exploring a Regulatory framework for CRISPR-based medicines
  • New data demonstrating advancements for translating CRISPR technologies into medicines
  • Clinical natural history study of Leber Congenital Amaurosis to understand the disease course and potential clinical endpoints 
  • Advancing toward the clinic with potential to transform therapy for retinal disease LCA10-CEP290
 
Apr 2709:25
Conference pass

Keynote address: Translating CRISPR/Cas9 technology into transformative gene-based medicines for patients with serious diseases

Keynotes
  • In vivo and ex vivo treatments using CRISPR/Cas9 for different therapeutic indications
  • CRISPR/Cas9 potential to correct DNA changes in somatic (non germ line) cells in patients with serious disease and the road to clinical trials
  • Partnerships with other pharmaceutical companies to develop gene-editing-based therapeutics
 
Apr 2709:50
Conference pass

Keynote address: The required evolution (revolution) of the healthcare system to realize the promise of genome editing medicines

Keynotes
  • Looking at the rapid acceleration of genomics and how the healthcare community can transform from care to cure
  • Societal needs, policies and new healthcare infrastructure requirements to make CRISPR therapies rapidly available to patients
  • Ethical considerations of genome editing technologies
Apr 2711:15
Conference pass

Proposed Value based frameworks for pricing and reimbursement of orphan drugs - can a new value framework help ease friction over orphan drug prices?

Clinical Development & Regulatory Track
  • Methods to recommend fair prices to allow for broader insurance coverage for innovative new treatments
  • Different proposals for gene therapy pricing and possible routes for implementation
  • Impact of willingness to pay thresholds in the US and the orphan market space
 
Apr 2711:15
Conference pass

Ultragenyx and Takeda Collaboration to Develop and Commercialize therapies for rare genetic diseases

Commercial Track
  • Leveraging Takeda’s early pipeline of molecules
  • Ultragenyx’s strong patient centric development and regulatory capabilities in the rare disease space
  • Outputs of successful partnerships including the commercialization of licensed products in different geographies
 
Apr 2711:15
Conference pass

THE PHILIPPINES: How a newly passed Rare Diseases Act is unlocking an emerging market’s commercial potential

Global Market Access Track
  • Two years after the introduction of a Rare Disease Act covering registries, patient care, R&D, patient care and screenings
  • Introduction of a national strategy specifically for rare disease management facilitating coverage and access 
  • How the Philippine society for orphan disorders pushed for the approval of this key legislation
 
Apr 2711:15
Conference pass

The era of novel technologies including CAR-T-cell, stem cells and gene therapy

Gene Track
  • Leveraging potentially curative short/single treatment duration vs rarely curative and long-term duration treatments
  • European vs. US perspectives on clinical development, pricing and adoption
  • Overcoming advocacy, clinical and manufacturing barriers
 
Apr 2711:15
Conference pass

Educating patient groups and communities on clinical development and gene therapies

Rare Disease Advocacy World
  • Tools for educating patient groups and community
  • Communicating trial expectations, exclusion criteria, and on goings with patient groups
  • Gaps in gene therapy understanding
 
Apr 2711:15
Conference pass

Maximizing the impact of public investment to accelerate rare disease therapeutic development

Pitch & Partner
  • Private public partnerships with NCATS to bridge translational gaps – funding and technical expertise
  • Establishing platform technologies at TRND to catalyze rare disease therapeutic development
  • Exploring innovative approaches to unblock regulatory bottlenecks
 
Apr 2711:40
Conference pass

Hemophilia – how to navigate the increasingly competitive market of therapies for rare blood disorders and ensure market share

Commercial Track
  • Competing with other marketed treatments – what are key aspects to achieve differentiation?
  • Fostering innovation in rare blood disorders through partnerships and exploring scientific innovation
  • Increasing access and relationships with government entities to achieve successful visibility and commercialization
 
Tom Sabia, Vice President, Haemophilia Marketing and Operations, Sobi
Apr 2711:40
Conference pass

BRAZIL: Qualifying and standardizing the legal process for access to non-approved orphan drugs in Brazil

Global Market Access Track
  • Working towards better access for orphan drugs to surpass the common route of legal action by patients
  • Navigating Brazil’s complex payer landscape and leveraging SUS’ guidelines for improved diagnosis and treatment
  • Strategies for launching orphan drugs in LatAm’s most promising orphan drug market
 
Luiz Fernando Lima Reis, Director of Education and Research, Hospital Sirio-Libanes
Arnaldo Hossepian, Member, Conselho Nacional de Justiça
Apr 2711:40
Conference pass

Pioneering partnerships between industry and patient groups to advance gene therapy research to treat inherited retinal diseases

Gene Track
  • Supporting research initiatives like My Retina Tracker registry and studies to identify barriers that prevent patients use of genetic testing
  • Leveraging partnerships and funding opportunities to increase gene therapy clinical trial enrollment
  • Working toward common objectives and maximizing patient groups’ and industry’s capabilities
 
Apr 2711:40
Conference pass

Cross-functional education of internal stakeholders while maximizing patients’ opportunities through the product lifecycle

Rare Disease Advocacy World
  • Creating a strategic plan to address patient advocacy across the spectrum of pharmaceutical functions
  • Presenting opportunities for patients to engage with regulatory   agencies at preclinical and clinical stages
  • Incorporating the patient perspective to influence endpoints, recruitment, retention, etc
Apr 2711:40
Conference pass

Case Study: What Vtesse’s acquisition by Sucampo Pharmaceuticals tells about investing in ultra-rare diseases with unmet medical needs

Pitch & Partner
Apr 2711:40
Conference pass

Drug pricing in Europe, improving the role of HTA agencies and looking into the sustainability of managed access agreements

Clinical Development & Regulatory Track
  • Looking into voluntary price joint price negotiations as a means to assess highly priced drugs and increase access
  • Can HTA assessment be improved across European countries?
  • What will be the future of managed access agreements as more drugs require them and more resources are needed? Can this model be replicated around Europe?
 
Apr 2712:05
Conference pass

Developing new payment and alternative financing models to ensure higher value and better outcomes for the money spent in highly priced medical products

Clinical Development & Regulatory Track
  • Adapting health system reimbursement models to encourage innovation in curative gene therapies and other innovative therapies
  • Obstacles and opportunities for the increased implementation of value-based payment arrangements in the United States
  • Gene therapies vs. other highly priced treatments- looking into the sustainability of the healthcare system
Apr 2712:05
Conference pass

Designing your distribution channel to optimize your orphan drug commercialization strategy

Commercial Track
  • Taking the right steps to maintain inventory control
  • Ensuring access to patient site of care
  • Increase speed to market through strong channel footprint
  • Gain valuable business insights through data and analytics
 
Apr 2712:05
Conference pass

LATAM: Orphan drugs and rare diseases in Latin America

Global Market Access Track
  • New legislations and trends addressing the health care system sustainability of the region
  • From product centric-to patient-centric
  • Is LATAM still a key player for rare disease companies? Yes, but…
  • Navigating a complex healthcare landscape with hybrid payer systems
 
Apr 2712:05
Conference pass

Scaling up viral vector development and manufacturing for cell and gene therapies

Gene Track
  • Quality, analytical, and validation characteristics unique to gene therapy products
  • Early stage commercial planning to enable scale up
  • In-house, outsourcing or hybrid models: adapting viral vector developing and manufacturing to your gene therapy program needs
  • Selecting  and managing external manufacturers effectively in a suppliers’ shortage scenario
 
 
Apr 2712:05
Conference pass

Patient-centered qualitative research to inform potential outcome measures for Angelman syndrome clinical trials

Rare Disease Advocacy World
  • Identifying and developing the best outcome measures and biomarkers for future clinical trials
  • Working with doctors and patients globally to determine what to measure, how to measure it, and how to interpret findings
  • The Angelman Biomarkers and Outcome Measures Alliance (A-BOM)
 
Apr 2715:10
Conference pass

The value of computer vision, machine learning and robotic automation to expedite rare disease research

Commercial Track
  • Running experiments and performing analysis on hundreds of diseases simultaneously using powerful software and analytics
  • Finding new indications for existing drugs using augmented high-throughput drug research and technology
  • Creating high-resolution, comprehensive biological data sets
  • Academia-industry partnerships to foster rare disease research
 
Apr 2715:10
Conference pass

35 Years: Where have we come and where are we going

Rare Disease Advocacy World
  • A look at the progress the rare disease community has made over the last 35 years, since the founding of NORD and the passage of the Orphan Drug Act
  • NORD's POV on the current state of affairs in the United States in health care, policy, research, and awareness
  • Where are science and medicine heading, and where does the rare disease community need to focus its attention and efforts to ensure sustainability for the next 35 years
 
Apr 2715:10
Conference pass

Evidence generation in rare diseases – thinking creatively to generate evidence that effectively supports products

Clinical Development & Regulatory Track
  • Evidence requirements throughout the product lifecycle – where to look during each stage
  • Partnering with patients, caregivers and physicians to support evidence generation, trial success and reimbursement
  • Understanding of the breadth of real-world evidence options
 
Apr 2715:35
Conference pass

Health outcomes driven research in rare disease to demonstrate value

Clinical Development & Regulatory Track
  • Identifying a rare disease and developing a product that gives value
  • Evaluating the value of the new drug to that disease vs. the clinical benefit
  • What about RWE? Can it be incorporated?
 
 
Apr 2715:35
Conference pass

Machine Learning – a personalized drug discovery and biomarker development bioinformatics platform targeting therapies for ALS

Commercial Track
  • Advancing the discovery of new drugs and repurpose existing ones for Amyotrophic Lateral Sclerosis (ALS) and personalized treatment for ALS patients
  • Providing transcriptomic data from ALS individuals to accelerate translational programs relevant to both the patient population and specific individuals suffering from the disease
  • Opportunities for the applications of deep learned biomarkers for the other rare diseases to seek new, breakthrough and transformative therapies
 
Apr 2715:35
Conference pass

Scaling up rare disease patient engagement in Europe

Rare Disease Advocacy World
  • The EURORDIS new structured approach on patients' engagement in research, drug development and life cycle and healthcare
  • Creating value for patients and working through partnership to create value to all stakeholders
 
last published: 13/Dec/17 18:25 GMT