RARE DISEASE ADVOCACY WORLD - DAY TWO

 

For an easier view of the complete agenda, click here >

Rare Disease Advocacy World - Conference Day Two

08:15

REGISTRATION AND NETWORKING BREAKFAST

Scott Gray
08:50

Chairperson’s Opening Remarks

GENE THERAPY

Sven Kili
09:00

Keynote address: Development of lifesaving gene therapies through collaboration between academic discovery, scientific research, medical practice and product development

  • How can cell and gene therapy programs be developed through collaborative efforts with academia and lead to more approvals?
  • Designing a trial when using a patient’s own gene modified stem cells to correct immunodeficiency’s 
  • Overcoming challenges of small population sample, skepticism of the community and high pricing 
Nick Leschly
09:20

Keynote address: Transforming the life of children on a genetic death sentence by giving them a bluebird day

  • Overview of the clinical development program for cerebral adrenoleukodystrophy and beta-thalassemia
  • Overcoming challenges in the design and interpretation of the human clinical trials, immunogenicity, and the selection of the best primary efficacy end point to progress clinical development 
  • How to balance the science and medicine being developed with the expectations of payers and regulators? 
Matt Kapusta
09:40

Keynote address: Adeno-associated virus (AAV)-based gene therapies for rare, chronic and degenerative diseases

  • Innovative and modular technology platform for hemophilia gene therapies 
  • Using therapeutic miRNAs for the development of a gene therapy for Huntington’s Disease
  • Strategic collaboration with Bristol-Myers Squibb to advance gene therapies for cardiovascular disease
Jeffrey D. Marrazzo
10:00

Keynote address: Driving towards success in gene therapy development: pioneering a different approach for differentiated outcomes in rare diseases

• Inherited Retinal Disease (IRD): a collaborative effort between scientists, patients and regulators during a decades-long journey 
• Building a fully-integrated company to prepare for a potential first-ever U.S launch
• Considering potential new value propositions for patients, payers and the health care system
• Gene therapy platform development: lessons learned
10:20

SPEED NETWORKING

10:40

MORNING NETWORKING BREAK

ACCESS

Andrew McFadyen
11:20

Compassionate use: patient advocacy organization helping patients get access to medication through Compassionate Use Programs

  • Navigating the various compassionate use and overcoming obstacles in clarity of the programs, inclusion criteria and lack of transparency
  • Educating the community on expanded access programs to ensure safe access to experimental treatments
  • Liaising with pharmaceutical stakeholders to help patients gain access to potentially lifesaving treatments
Panel discussion
11:40

Panel: Early access to experimental drugs - is ‘Right to Try’ the solution for patients with no treatment options?

  • Are these laws bypassing the FDA and exposing patients to dangerous experimental treatments? 
  • Is building robust compassionate use programs in small, medium sized and big pharma the answer to strengthen the responsible access to experimental treatments?
  • The impact of RTT on clinical trial recruitment and the drug development programs of small and medium sized biotechs
  • Are changes to FDA’s compassionate use program needed? 

12:20 ROUNDTABLES

  • Access – How can rare disease companies, insurers and patients/families work together to ensure access to transformative and curative therapies?
  • Drug repurposing – finding scientific rationale and relying on strong safety data for orphan drug repurposing
  • Enhanced collaboration – collaboration between patients, researchers and government institutions to support patient-centered research
  • Ethical considerations – patients’ rights when participating in rare disease trials
  • Gene therapy – partnering to build capabilities in Gene Therapy
  • Global procurement – effective optimization of rare disease clinical processes to improve efficiencies and support commercialization
  • Multi-stakeholder collaboration to accelerate rare disease clinical development
  • Pricing – price gouging and the dangerous new breed for pharma companies
  • RWD – Using real-world data to find rare disease patients for long-term observational studies
  • Steering committees – reaching the goal of patient committees that can drive trial design and meaningful data
  • Strategy – successfully managing, and implementing drug development programs for domestic and international commercialization of orphan drugs
  • Diane White

    Roundtable 2: Strategy – successfully managing, and implementing drug development programs for domestic and international commercialization of orphan drugs

    Edmund Pezalla

    Roundtable 3: Pricing – price gouging and the dangerous new breed for pharma companies

    Christopher Missling

    Roundtable 4: Partnerships- successful collaboration between a rare disease and a pharmaceutical company to advance preclinical and clinical research in Rett Syndrome

    Dr Cynthia Jackson

    Roundtable 5: Drug development- simplifying the complexities of rare disease drug development

    Donna Armentano

    Roundtable 6: Gene therapy – partnering to build capabilities in Gene Therapy

    Jayne Gershkowitz

    Roundtable 7: Steering committees – reaching the goal of patient committees that can drive trial design and meaningful data

    Rashmi Gopal-Srivastava

    Roundtable 8: Enhanced collaboration – collaboration between patients, researchers and government institutions to support patient-centered research

    Keith DeMatteo

    Roundtable 9: Global procurement – using your procurement function to drive innovation and not just three bids and a buy

    Claudia Hirawat

    Roundtable 10: Ethical considerations – patients’ rights when participating in rare disease trials

    Francesca Cook

    Roundtable 11: Access: It takes a Village- How can rare disease companies, insurers and patients/families work together to ensure access to transformative and curative therapies

    Professor Matthias Schönermark

    Roundtable 12: European market access – effective strategies for setting drug prices and achieving reimbursement of orphan drugs

    Horacio Plotkin

    Roundtable 13: Virtual trials - Can we make them a reality in orphan drug development?

    John Boland

    Roundtable 14: Trial optimization – unique challenges in small population/rare disease studies vs. large population trials

    Ms Katherine Brandt

    Roundtable 15: Orphan drugs manufacturing – overcoming CMO selection, auditing strategies, risk management and other outsourcing challenges

    Steven Anderson

    Roundtable 17: Genomics & biomarkers – solutions for rare diseases, implications for natural history and patient recruitment

    If you are interested in sponsoring a roundtable contact André Singer at andre.singer@terrapinn.com or +1 646 619 1797

    13:20 NETWORKING LUNCH

    Gayatri Rao JD., MD,
    13:30

    Lunch Keynote: Scientific rationale – FDA’s updates and considerations to orphan drug approval

    • Rigorous collection of natural history data
    • Rigorous development of assets prior to start of clinical trials, particular biomarker assets
    • Adoption of randomization to control groups in trials
     
    13:50

    NETWORKING LUNCH RESUMES

    NATIONAL PATIENT ORGANIZATIONS

    Gudrun Helga Haroardottir
    14:30

    Creating rare disease awareness in a country with a population of 330,000 people

    • Guaranteeing government coverage and overcoming access problems
    • How to determine gray areas in the government that affect rare disease patients?
    • Improving local diagnostics and data collection of rare diseases in Iceland
     
    Kelly Du Plessis
    14:40

    Overcoming the lack of medical infrastructure, research and support to improve the quality of life of rare disease patients in South Africa

    • Efforts to move rare disease policy forward in Africa
    • Creating awareness and establishing a network to connect all stakeholders that can help rare disease patients 
    • Facilitating access and supportive care for patients and families
    Amanda Samanek
    14:50

    Working with governments, researchers, clinicians and industry to promote research, diagnosis, treatment and services for all rare diseases in Australia

    • Raising awareness of issues faced by the rare disease community to promote understanding, motivation and empowerment
    • Educating physicians and other healthcare professionals to promote early diagnosis and optimal treatment
    • Promoting optimal care outcomes for people living with rare diseases
    Angela Chaves
    15:00

    Influencing public policy to achieve a true recognition and support for population with Rare Diseases in Colombia and Latin America

    • Working to overcome challenges of disparity in rare disease public policies in Latam, in order to improve access and medical care
    • State of the art in Colombian public policies in the recognition of rare disorders- can this be a pathway for other countries in Latam?
    • Supporting the patient ecosystem to achieve accurate diagnosis and recognition of rare diseases by the professional health community 
    Letitia O'Dwyer
    15:10

    Changing the paradigm of access, recognition and collaboration in rare diseases in New Zealand

    • Working with rare disease groups to ensure their concerns and interests are communicated to health officials during consultation processes
    • Liaising with pharmaceutical companies and PHARMAC to facilitate access
    • Providing solutions to connect patients to specialists
    Yukiko Nishimura
    15:20

    Making rare and intractable diseases an Asian health priority

    • Japanese ‘Specified Disease Treatment Research Program’ and Orphan drug legislation to encourage orphan drug research
    • J-Rare and ODOD (Open Discussion for Orphan Drug Discovery programs) to foster rare disease identification, registries and diagnosis
    • Connecting Asian rare disease stakeholders to accelerate the path to treatment 
    Ms Pamela Gavin
    15:30

    Unifying the voices of many rare disease organizations to advance new diagnostics, treatments and cures for rare diseases

    • The NORD’s research grant program: working with patient groups for translational and clinical studies
    • Campaigns to promote the awareness of rare diseases and raise funds to assist undiagnosed patients
    • Educating patients in the process and eligibility for accessing unapproved therapies 
    Harsha Rajasimha
    15:40

    Challenges of patients with rare diseases in India and recent progress

    • Awareness, Education and Advocacy in India
    • Connecting patients and physicians in India with international research and clinical trials
    • Facilitating coordinated care and multidisciplinary approach for proper diagnosis and treatment of patients with rare diseases in India
    Yann Le Cam
    16:00

    Scaling up rare disease patient engagement in Europe

    • 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

     

    16:20 CLOSING REMARKS AND END OF CONFERENCE

    last published: 24/Mar/17 14:35 GMT

     

    WORLD ORPHAN DRUG CONGRESS HAS IT ALL...

    World Orphan Drug Congress USA is bringing together the worlds of Scientific Innovation and Commercialization, with 2 tracks in the main conference. By exploring trends in Digital Health, Biosimilars, Gene Therapy, and more, we will uncover the next scientific breakthrough. Examinations of Mergers, Commercialization, Marketing and the Regional Markets will provide insight into this billion-dollar business.

    Download the brochure to find out more!

    DOWNLOAD BROCHURE