Conference Agenda

Orphan Drugs & Rare Diseases
Conference Day 1

Day 1: Tuesday - 19th October 2021

  • Impact of new innovations to patients and caregivers
  • What difference do these recent innovations have made to their lives?
  • What must be done to accelerate innovations and broaden access at the same time?
  • What challenges still remain to increased equity for people living with a rare disease and their families?

  • What dynamic guideline recommendations will be put in the hands of the healthcare professionals?
  • Collaborations and partnerships with life science industries
  • Will rare disease patients have quicker access to new treatments and innovations?

  • Changes in regulations affecting the current market
  • R&D strategies to be implemented to accelerate market access for orphan drugs
  • Methods that orphan drug manufacturers can do to partner with governments
  • How can patient, governments, and orphan drug companies help support each other’s initiative to
    provide timely access to patients

  • Situation: Regardless of therapeutic area, many small BioPharmas share a few similarities:
  • Complication: A Research culture + informal ways of working + many distractions can result in a rapidly
    growing, small BioPharma losing focus on what is important,
  • Resolution: BioPharmas should of course continue to leverage their Research heritage, but they must
    also mature and focus as they grow

Christopher Dandrea, Vice President, Global Program Management, uniQure

  • Overview of current methods for their prevention, diagnosis, and treatment.
  • Strengths and limitation of current development pathways for new drugs, medical devices, and biologics
    for rare diseases
  • Special challenges that rare diseases create for research and product regulation
  • Current public policies relevant to product development for rare diseases

  • How is this advanced therapy fundamentally changing the way rare diseases patients are treated?
  • Improving clinical development by new-age clinical trial design and recruitment
    An effective framework for developing the delivery system for the next generation of medicine
  • Making manufacturing and commercialization viable
  • Unconventional payment models to ensure innovative treatment can be accessed by rare disease

2:00 pm - 6:00 pm - Choose to attend between the 2 topic streams


  • Need for Education Platforms generating
    Disease awareness and facilitating the path to
    early diagnosis
  • Referral to Centers of Excellence offering a
    multi-disciplinary expert management and
    collaboration with the patient advocacy.
  • Case studies will be highlighted

Haya Taitel, Senior Vice President, Chief
Commercial Officer, Kadmon

  • With data and technology taking a hold on the
    medical industry, how can we leverage this
    information in a way that truly benefits us all.
  • Capitalism is reaching its limits, in a way that is
    undermining resources, how can we flip the
    narrative to build a thriving economy.
  • Data and technology have the potential to bring
    about the largest distribution of wealth ever,
    are we ready to distribute that wealth evenly?

Christine Von Raesfeld, Founder/CEO, People
with Empathy

  • Patient Journey
  • Advocacy
  • Results of a survey of patients with
    cholangiocarcinoma cancer
  • Final thoughts

Melinda Bachini, Director of Advocacy,
Cholangiocarcinoma Foundation


  • Why Engage the FDA Early?
  • Patients First: Importance of Living the Disease
  • Creating Policy Accessing Future Treatments -
    Identify the Gaps
  • Clinical Data: Need for Prospective Studies
  • Real-World Data to Translational Science:
    Finding Drug Targets
  • Basic Science: Existing Science and How it
    Compares to Human Disease State
  • FDA - The Cycle Ends - (Resulting in better
    designed clinical trials)

Monica L Weldon, President/CEO/Founder,
Bridge The Gap - SYNGAP ERF

  • The proprietary TANGO platform can STOKE
    protein production inside a cell
  • We are advancing programs focused on
    multiple targets, including haploinsufficiency
    diseases of the central nervous system and eye
  • We are initially prioritizing the development of
    potential medicines to treat genetic epilepsies,
    such as Dravet syndrome

Barry Ticho, M.D., PhD, Chief Medical Officer,
Stoke Therapeutics, Inc.

  • Learn how to design clinical trials that answer
    patient needs while meeting scientific
  • Discussion points include ways to reduce
    amendments while increasing diverse
  • A case study will illustrate innovative
    approaches that work for companies and

Deborah Collyar, President, Patient Advocates
In Research (PAIR)

  • Oral, once-a-day inhibitor being developed for
    SCD and BTL; inhibits PDE9
  • Global Ph-2b programs underway in 14
    countries, several in developing world areas
  • Ph-2a 93 patient study data shows reductions
    in VOCs, improvement in HbF
  • 2nd year of Real Impact Grants, mechanism to
    support Community Based Organizations
    ($150K in 2021)

Rahul Ballal, PhD., President & CEO, Imara Inc.

6:00 pm - Chairperson's Closing Remarks

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