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
ACCESS & COLLABORATION
- Need for Education Platforms generating
Disease awareness and facilitating the path to
- 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
- Patient Journey
- Results of a survey of patients with
- Final thoughts
Melinda Bachini, Director of Advocacy,
DISCOVERY & INNOVATION
- 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