RARE DISEASE ADVOCACY WORLD - DAY ONE

 

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Rare Disease Advocacy World - Conference Day One

08:15

REGISTRATION AND NETWORKING BREAKFAST

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 scientists through the ‘Rare Diseases Research Consortium’ 
  • Gene therapy as the catalyst for innovation in rare disease therapeutics 
  • Powering rare disease therapeutics and patient engagement through the product lifecycle
  • 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?
Peter Saltonstall
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

INFLUENCING DRUG DEVELOPMENT

Birgitte Volck
11:20

“Focus on the Patient Program” at GSK: strengthening patient leadership to accelerate and improve clinical trials, drug development and access strategies

  • Working with patients to better understand the disease burden and adjust aspects of drug delivery methods, time-frames for data collection, target population and study endpoints 
  • Providing evidence to payers by developing patient case studies to show the human impact of the disease and treatment
  • Helping patients prepare to participate in scientific, ethical and regulatory committees with pharmaceutical and government stakeholders 
Irene Aquino
11:40

Case Study: How early is early? Enabling rare disease diagnosis

  • Misdiagnoses and delay in diagnosis: patient case study
  • Impact of genetic screening and counseling program on the patient journey
  • Cross-functional value delivered to achieve corporate objectives
Terrie Livingston
12:00

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

  • Developing meaningful patient reported outcomes by gathering information about their disease journey through an independent social network 
  • Will PROs be used to run 3b trials to can produce further evidence to support approval?
  • Strategies and tools to enhance patient engagement and patient experience 
Ms Michelle Berg
12:20

Taking on the challenges of enrolling patients in gene therapy clinical trials

  • Understanding barriers and hesitations for clinical trial participation
  • Creating patient awareness to join trials- weighing the benefit-risk to participate
  • Educating patients to make informed decisions

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 observational studies
  • 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’?
  • Roundtable 1: Building a successful launch – strategies and tactics to achieve clinical and commercial success

    Moderated by Cello Health
    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)

    Roundtable 3: Pre-launch strategies – finding and engaging with rare diseases KOL early to support prescription, commercialization, patient identification and the whole product life-cycle

    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

    Michael Murphy

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

    Michael Murphy, Chief Medical & Scientific Officer, Worldwide Clinical Trials

    Roundtable 9: Vials are vile – how intelligent device solutions improve patient adherence while dosing reliably to prevent relapses and influence P&R’?

    Moderated by Medicom
    Eric Pauwels

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

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

    Moderated by Marken
    Professor 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

    13:20 Networking Lunch sponsored by Sigma-Tau Pharmaceuticals

    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

    14:10 Networking Lunch Resumes

    ADVOCACY IN ACTION

    Pat Furlong
    14:40

    Advocacy’s influence in regulations – how united patient groups can impact policymaking

    • Increasing patient advocacy groups’ participation in congressional caucuses and how their voice can be used in PDUFA and FDASIA meetings
    • Submission of Patient-centered benefit-risk assessment studies to the FDA to include patient preference information into regulatory decision-making
    • Patients’ influence in regulatory guidelines, new research, and product approvals
    Mary Frances Harmon
    15:00

    Patient-industry partnerships to influence orphan drug development and the product lifecycle

    • Raising awareness to help with clinical trial enrollment in patient communities 
    • Helping patients understand their disease and available treatment options, as well as serving as hubs for education and support 
    • Working with patients and research institutions to accelerate translational research by providing firsthand information on the disease 
    Vignesh Ganapathy
    15:20

    Newborn Screening Bill in California – saving babies through early diagnosis

    • The new movement - homogenization of newborn screening laws to standardize the number of rare diseases screened at birth 
    • Improving early diagnosis of rare diseases and subsequent treatment through early disease screening
    • Optimizing data collection, early identification of patients for clinical trials and natural history studies 
    15:40

    AFTERNOON NETWORKING BREAK

    PRICING & REIMBURSEMENT

    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?
    •  
    Luca Pani
    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?

    Networking cocktail reception sponsored by Sigma-Tau Pharmaceuticals

    last published: 23/Mar/17 21:05 GMT

     

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    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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