WORLD ORPHAN DRUG CONGRESS USA - DAY ONE

 

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World Orphan Drug Congress USA 2017 - Conference Day One

REGISTRATION AND NETWORKING BREAKFAST

Chris Garabedian
08:50

Chairperson’s Opening Remarks

FUTURE OF ORPHAN DRUGS

Michael Goettler
09:00

Keynote address: The potential of big pharma in accelerating rare disease breakthroughs

  • Joint drug discovery program between researchers and industry scientists
  • Gene therapy as the catalyst for innovation in rare disease therapeutics 
  • Powering rare disease therapeutics and patient engagement through the product life-cycle
  • Where will the orphan drug market be in 2020?
Sy Pretorius
09:20

Keynote CEO panel: Driving the future of the orphan drug market – adapting to change, fostering innovation and transforming patients’ lives

  • Building a long-term vision- bringing leadership and past experiences to guide the direction of a rare disease company 
  • Nurturing and growing a business while staying responsive to patients’ needs 
  • What does the future of orphan drugs look like and what can patients and other stakeholders expect to see in the next 5 years?
James Greenwood
09:50

Keynote panel: Prescription Drug User Fee Act (PDUFA VI) and 21ST Century Cures – recent and pending legislative efforts to make drugs better for patients

  • Making the drug development process better and more efficient for patients through biomarker development, precision medicine, patient reported outcomes and RWE for regulatory uses 
  • Engagement of patients in regulatory decision-making, streamlined drug development and faster applications review - what this means for drug manufacturers 
  • How can legislative pieces be improved to continue getting support from Congress? 

10:20 SPEED NETWORKING

10:40 MORNING NETWORKING BREAK

PATIENT-CENTRIC RESEARCH

M&A & COMMERCIALIZATION

11:20

Is the FDA patient-centric? Patient-focused drug development (PFDD) as a systematic approach to gather input from patients

  • An overview of Patient-Focused Drug Development (PFDD) initiatives under PDUFA V to support drug development and evaluation
  • Using the evidence gathered from patients to conduct benefit-risk assessments and advise sponsors on their drug development programs
  • Providing consultation to stakeholders developing PRO tools through the Clinical Outcomes Assessment Program at CDER/FDA
11:20

A pharma perspective on partnerships with external innovators in the life sciences

  • Why and how pharma pursues external partners
  • How to prepare for “successful” partnership discussions with pharma – It is a matter of fit
  • What you should expect from your pharma counterpart during and after diligence
11:40

Creating significant shareholder value: turning around NPS, two rare diseases products launched and a $5.2B sale to Shire

  • Defining a new business model and a values-based culture
  • Repurposing two products from large indications to niche orphans with no competition
  • Managing the adversities along the way
12:00

Orchestrating successful pre-launch strategies for orphan drugs: overcoming challenges, optimizing approaches

  • Leveraging insights – knowing your market, understanding your patients
  • Demonstrating value – scientific differentiation, clinical needs, commercial relevance
  • Honing strategies – assessing market dynamics and potential, optimizing your path to market
  • Telling the story – multi-channel initiatives to engage and educate stakeholders-
                                        

ACCESS

12:20

Commitment to access: a key element of development and availability of rare disease treatments

  • Looking to guarantee access to available medicines through the humanitarian donation of protein factor for hemophilia in countries where there is no access
  • Using donations to open a dialogue to allow the treatment to be included in the regular healthcare system in developing countries
  • Setting the foundation to create a sustainable model for humanitarian support in the rare disease field
12:20

The other side - effective licensing and collaboration agreements with rare disease biotechnology companies

  • Expanding operations while retaining the essence of patient engagement and tight relationships with the patient community
  • Sharing of scientific information and protocols between companies to find different ways of looking at diseases 
  • When is the right time to engage in partnerships?

12:40 ROUNDTABLES

  • Building a successful launch – strategies and tactics to achieve clinical and commercial success
  • Clinical development- overcoming operational challenges in rare disease trials
  • Expanded access programs – patient services helping patients navigate programs that can lead to early access to experimental treatments
  • Gene therapy – designing clinical development programs that support approval, reimbursement and commercialization
  • New scientific platforms – scientific innovation to bring new treatments for rare disease (mAbs, gene therapy, mRNA-based therapeutics, synthetic biology, antisense)
  • Orphan launches – addressing unique challenges of small biotech organizations and orphan drug launches and leveraging your healthcare communications partner
  • Patient centricity – fundamental collaborations with patient groups to advance orphan drug development
  • Pre-launch strategies – finding and engaging with rare diseases KOL early to support prescription, commercialization, patient identification and the whole product life-cycle
  • PROs – defining the patient-centered experience of painful crises in Sickle Cell disease through the development of patient reported outcomes
  • RWD – Using real-world data to find rare disease patients for long-term
  • Trials Innovation – clinical solutions for rare disease clinical trials
  • Vials are vile – how intelligent device solutions improve patient adherence while dosing reliably to prevent relapses and influence P&R’?
  • Paul Eccles, PhD

    Roundtable 1: Building a successful launch – strategies and tactics to achieve clinical and commercial success

    Aoife M Brennan

    Roundtable 2: New scientific platforms – scientific innovation to bring new treatments for rare disease (mAbs, gene therapy, mRNA-based therapeutics, synthetic biology, antisense)

    Kaye Spratt

    Roundtable 3: Gene therapy commercialization- advances in delivery, development and regulatory to move products into approval and commercialization

    Andreas Pleil

    Roundtable 5: PROs – defining the patient-centered experience of painful crises in Sickle Cell disease through the development of patient reported outcomes

    Barbara Wuebbels

    Roundtable 7: Expanded access programs – patient services helping patients navigate programs that can lead to early access to experimental treatments

    Leslie Wetherell

    Roundtable 8: Clinical development- overcoming operational challenges in rare disease trials

    Eric Pauwels

    Roundtable 10: Patient centricity – fundamental collaborations with patient groups to advance orphan drug development

    Sean O'Bryan

    Roundtable 12: Gene therapy – designing clinical development programs that support approval, reimbursement and commercialization

    Sascha Sonnenberg

    Roundtable 13: Direct to Patient – how DTP services are playing an essential role in the continued growth of the orphan drug market

    Prof Matthias Schönermark

    Roundtable 14: Orphan drugs in Germany – market access for orphan drugs and lessons learned from the AMNOG process

    Jeff Ceitlin

    Roundtable 15: Next-generation commercial strategy – disrupting rare/orphan disease commercial strategy through proven, predictive modeling

    Joseph Musumeci

    Roundtable 17: BD & Forecasting- pricing and access considerations and understanding how to build the patient forecast based on available epidemiological information

    Alastair Macdonald

    Roundtable 18: Integrated medical plans – begin with the end in mind to develop an integrated medical plan to maximize your drug’s potential

    13:20 Networking Lunch sponsored by Leadiant Biosciences

    Gayatri Rao JD., MD,
    13:50

    Lunch Keynote: Fostering natural history studies in rare disease – identifying subpopulations, developing and validating clinical outcome measures, biomarkers and companion diagnostics

    • Collecting data on rare diseases’ progression over time to inform and support product development and approval
    • Impact of natural history studies in clinical trial design and endpoint identification
    • Bridging funding gaps to conduct natural history studies and complement existing efforts to bring new therapies to rare disease patients

    14:10 Networking Lunch Resumes

    REAL WORLD EVIDENCE

    MARKETING & COMPETITIVENESS

    14:40

    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?
    14:40

    Transforming a R&D biotech into a fully vertically integrated company in the ultra-orphan space

    • Setting up a commercial infrastructure (>100 people) for the launch of a treatment for acquired TTP in North America and Europe
    • Implementing a supply strategy, from commercial manufacturing to last-mile distribution
    • Rolling out a marketing and communication strategy and defining the appropriate evidence pricing
    • Preparing out-licensing options for Japan and rest of world
     
    15:00

    Improving the use of RWE for regulatory decisions – steps to support new indications and label expansions

    • Is the regulatory environment conducive to the effective incorporation and use of RWE?
    • Recent policy proposals and discussions regarding use of RWE by FDA
    • opportunities for leveraging RWE to improve evidence development for regulatory decisions in rare diseases.  
    • Approaches for public and private partnerships to support a national data and evidence resource for developing RWE
    15:00

    Marketing strategies to stay competitive during post-launch, ensure brand loyalty and market share

    • Leveraging patient associations, patient services, remote engagement and physician relationships to drive the ongoing use of treatments
    • How to innovate while keeping launched products competitive? How to do this in therapeutic categories with multiple treatment options?
    • Effective life cycle management of orphan drugs and the integrated work with medical affairs and advocacy
    15:20

    How can patients build better registries themselves?

    • De-risking patient’s data collection, increasing clinical trial participation and supporting translational research 
    • Applications of registry data in industry incentives, clinical trial design and recruitment, surveys and focus groups and patient focused drug development meetings with regulators, and payers
    • Overcoming challenges related to funding, type of nature of the data collected, utilization and distribution and access and location
    15:20

    Doing digital right: raising awareness of MPS trough the launch of digital paper planes

    • Partnering with the International MPS Society and The US MPS Society to raise the profile of MPS (mucopolysaccharide) diseases
    • Enabling people to share information about MPS globally aiming to shorten the path to diagnosis
    • Using a social media platform to amplify messages and post awareness messages simultaneously 
    15:40

    15:40 AFTERNOON NETWORKING BREAK

    PRICING & REIMBURSEMENT

    Katie Verb
    16:30

    Evolving access challenges for one of the first orphan drugs- the hemophilia case

    • Payer restrictions for hemophilia therapies despite a history of open access
    • Why traditional payer management mechanisms are inappropriate for rare diseases like hemophilia
    • The role of a patient advocacy organization in protecting access to lifesaving therapies and the importance of Hemophilia Treatment Centers in providing Managed Care for this population over the last 40 years
    Luca Pani
    16:50

    Keynote address: How can different stakeholders pay for successes in gene therapy?

    • How did GSK and AIFA reach a price for Strimvelis and what other pricing models were considered?
    • Applicability of pay-for-performance to other parts of the world that don’t have a single payer system
    • Will pay-for-performance foster gene therapy commercialization and change the evaluation of premium pricing for other drugs?
    Anna Bucsics
    17:10

    Keynote payers’ panel: Effectively assessing the value of orphan drugs in a time of increased scrutiny of drug pricing

    • How can payers reward industry for true innovation without breaking the bank? 
    • Effectively assessing international referencing pricing, differential pricing, pay-for-performance and HTA to increase access for patients
    • How will pay for performance models impact the reimbursement of all orphan drugs?
    • Will the changing political landscape affect orphan drug reimbursement? 
    • If robust data is not yet available, why should payers reimburse at premium levels?

    17:40 Chairperson's Closing Remarks

    17:45 Networking cocktail reception sponsored by Leadiant Biosciences

    18:45 END OF DAY 1

    last published: 20/Apr/17 19:45 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.

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