• Wednesday, 09/13/17
    3:00 PM  -  7:00 PM
    Registration & Information Desk
  • Thursday, 09/14/17
    8:00 AM  -  8:30 AM
    Welcome by Co- Chairs - Setting the Stage
    8:35 AM  -  9:45 AM
    Innovation Company Creation Models for Rare Diseases
    This discussion will explore the evolving landscape of rare disease drug discovery and development, with a focus on innovative investment strategies. We’ll identify some successes and pitfalls of new rare disease funding models, and concentrate on emerging drivers and trends in a field that has received a growing attention from pharmaceutical companies, venture investors, government agencies, and patient advocates.
    8:45 AM  -  9:05 AM
    Cures Within Reach
    9:15 AM  -  9:35 AM
    Cure Duchenne
    9:45 AM  -  10:05 AM
    9:50 AM  -  10:30 AM
    Overcoming the Development Challenges of a Rare Disease Therapeutic
    Developing therapeutics for rare diseases pose specific challenges because of the limited population of patients with these conditions, the variation in the way a disease may manifest itself, and the range in severity of the disease. Among the challenges drug developers face is identifying validated biomarkers, having robust natural histories, and determining appropriate clinical trial endpoints. How can you design a rare disease clinical trial that ensures the best chance of success? How can you work with regulators to understand endpoints are meaningful for a disease that may not be well understood? What role can patient groups play in helping design and enroll a clinical trial?
    10:15 AM  -  10:35 AM
    10:35 AM  -  11:15 AM
    How to Ensure a Successful Commercial Launch of a Rare Disease Therapeutic
    Marketing rare disease therapies pose specific challenges because of the limited population of patients with these conditions. Drugmakers must educate patients, doctors, and payers about a disease and new therapy with which they may not be familiar. They must also justify what may seem like a high price tag for a new therapy. And, to make the economics of a rare disease therapy, they may need to have a commercial strategy that targets multiple international markets where many of the patients that may benefit from the drug may live. How can companies best find patients? Should they find a partner for international markets or build their own team? How should they prioritize markets and navigate the regulatory and reimbursement hurdles in different countries? What role can named patient programs play? What about the role of patient assistance programs?
    10:45 AM  -  11:05 AM
    11:15 AM  -  11:35 PM
    Project Violet
    11:20 AM  -  12:30 PM
    Alternatives to Standard Newcos: Spin-offs, Build-to-Buys, Joint Ventures and Alliances
    Increasingly, entrepreneurs and investors involved in forming rare disease-focused companies are seeking more innovative structures to translate scientific discoveries and create shareholder value rather than pursuing the traditional angel- and VC-backed financing of startups. This session will discuss the advantages and potential pitfalls of corporate spin-offs, strategic alliances and joint ventures, as well as build-to-buy models that include attractive, non-dilutive cash support combined with milestone-based options to acquire the entity.
    11:45 AM  -  12:05 PM
    Think Genetic
    12:15 PM  -  12:35 PM
    Portable Genomics
    12:40 PM  -  1:50 PM
    Networking Lunch

    Sponsored by:



    2:00 PM  -  2:20 PM
    Children's Tumor Foundation
    2:00 PM  -  3:30 PM
    Rare Disease Partnering Between Advocates, Academics, Startups and Global Pharmas
    Rare disease drug development is a team sport. This session will explore current and future partnering models between rare disease stakeholders including research institutions, industry, and patient organizations. Participants will discuss essential elements of successful partnering, how to align the differing interests of the participants, and how to overcome cultural differences to align everyone to work together towards common goals. Hear new ways that organizations are collaborating to discover, develop, and bring life-saving therapies to market.
    2:30 PM  -  2:50 PM
    TREND Community
    3:00 PM  -  3:20 PM
    3:45 PM  -  5:00 PM
    RARE Battle of the Brains
    In partnership with and Takeda Tak-celerator.
    5:30 PM  -  7:30 PM
    Backyard BBQ

    Come out to unwind and network with fellow attendees at our Backyard BBQ. Enjoy food and drink while playing a variety of fun interactive games in this one-of-a-kind RARE space.