Conference Agenda
Orphan Drugs & Rare Diseases
Conference Day 1
Day 1: Tuesday - 19th October 2021
08:00 am - Conference Registration and Continental Breakfast
08:30 am - Chairperson's Welcome & Opening Remarks
8:40 - Patient and Caregivers perspective: Transforming Lives through innovations
- 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?
9:20 am - - Keynote Fireside Chat: Strategies that can promote rare disease product development.
- 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?
10:00 am - am - Keynote Panel Discussion: Overview of the current market landscape for orphan drugs
- 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
10:40 am - Morning Networking & Refreshments Break
11:20 am - Keynote Plenary Session: Many Biopharma’s are pursuing the same targets, what is a small Biopharma to do?
- 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
11:50 pm - - Sponsored Plenary Keynote: Strategies to promote research discoveries and development of orphan products to improve the health of people with a rare disease
- 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
12:20 pm - Keynote Panel Discussion: Expectations and Future Directions For Cell & Gene Therapies
- 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
patients
1:00 pm - Networking Lunch Break
2:00 pm - 6:00 pm - Choose to attend between the 2 topic streams
ACCESS & COLLABORATION
2:10 pm - Session Chair Opening Remarks
2:15 pm - When building a commercialization platform for an asset for the management of rare disease, In-sight Mining associated with the Patient Path, Disease Burden and Unmet Needs are critical to establishing a product value proposition*
- 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
2:45 pm - Sponsored Spotlight Presentation: Advancing Orphan Drugs Innovation and clinical development to facilitate patient access
3:15 pm - Patients as proprietors: how can we harness technology and data to end socioeconomic disparities.
- 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
3:45 pm - Afternoon Networking Break
4:30 pm - How can governments and orphan drug companies help support each other’s initiative to provide access to patients?
5:30 pm - Patient Perspective on Cancer Care
- Patient Journey
- Advocacy
- Results of a survey of patients with
cholangiocarcinoma cancer - Final thoughts
Melinda Bachini, Director of Advocacy,
Cholangiocarcinoma Foundation
6:00 pm - Session Chair's Closing Remarks
DISCOVERY & INNOVATION
2:10 pm - Session Chair Opening Remarks
2:15 pm - Reverse Engineering for Drug Development in Rare Diseases
- Why Engage the FDA Early?
- Patients First: Importance of Living the Disease
Experience - 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
2:45 pm - Sponsored Spotlight Presentation: Accelerating Scientific development for rare disease from breakthrough technologies
3:15 pm - Using TANGO to make RNA medicines for severe genetic diseases
- 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.
3:45 - 4:30 pm - Afternoon Networking Break
4:30 pm - Creating successful clinical trials for companies and patients alike
- Learn how to design clinical trials that answer
patient needs while meeting scientific
endpoints - Discussion points include ways to reduce
amendments while increasing diverse
recruitment - A case study will illustrate innovative
approaches that work for companies and
patients
Deborah Collyar, President, Patient Advocates
In Research (PAIR)
5:00 pm - Sponsored Spotlight Presentation: The use of Artificial Intelligence (AI) in Rare Disease
5:30 pm - IMR-687 Development for Sickle Cell Disease (SCD) & Beta-Thalassemia (BTL)
- 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 - Session Chair's Closing Remarks
6:00 pm - Chairperson's Closing Remarks