Orphan Drugs Conference Day 2

Conference Registration and Continental Breakfast

Chairperson’s Welcome and Opening Remarks

Opening Keynote Panel Discussion: The role of registries in clinical research?

- A better understanding of long-term trends in a specific patient population

- What makes data good data?

- How does one harness unstructured data to provide value?

- Can you comment on the importance of data standards and ontologies?

​Femida Gwadry - Sridhar, CEO & Founder, Pulse Infoframe Inc.

Plenary session: Value of partnering with advocacy organizations to create innovative patient-driven solutions

- The foundational value of building collaboration through trust, respect, transparency and empathy

- Not all advocacy organizations are alike: assessing the landscape

- Real world examples of innovative digital solutions driving collaboration among multiple stakeholders

- It isn’t easy so why do it? Innovative partnering yields unique benefits boosting capabilities, medicine development and commercialization

T Anthony Howell, Co-Founder & Business Development & Partnerships, rareLife solutions

Plenary Session

Topic to be confirmed Shortly

Plenary Session: Opportunities to leverage real-world data and natural history studies for targeted drug development

- Unpack the meaning and utility of real-world data (RWD) & real-real world evidence (RWE)

- Explain regulatory and legislative underpinning

- Identify disparate sources from which RWD is obtained

- Describe the collaborative ecosystems optimized to enable data

Femida Gwadry-Sridhar, CEO & Founder, Pulse Infoframe Inc.

Morning Networking & Refreshment Break

ROUND TABLE DISCUSSIONS - divided between 2 sessions of 30 mins. - Attendees have 2 round table options to attend

TABLE 1 - Orphan Drugs in Europe: current developments in a heterogeneous market
Matthias Schoenermark, Professor, President and CEO, SKC Beratungsgesellschaft mbH

TABLE 2 - Challenges and Opportunities in developing treatment for rare oncology

TABLE 3 - How can developers make research goals and use of data to be transparent

TABLE 4 - Topic to be confirmed Shortly
Lisa Dilworth, Vice President, Rare and Orphan Diseases, Synteract

TABLE 5 - Patient-centric clinical development

TABLE 6 - Challenges in clinical development of cell and gene therapy medicines

TABLE 7 - Market Access & Commercialization*
Stephan Toutain, Sr. Vice President, Operations, Anavex Life Sciences

TABLE 8 - Innovative technologies that can speed up development of orphan drugs

TABLE 9 - Understanding the patient journey in rare diseases is critical to successful clinical development

TABLE 10 - Importance of site selection enabling studies to be conducted efficiently

TABLE 11 - Creating a Successful Strategy in Drug Repurposing to drive orphan drug development

TABLE 12 - Practical strategies for repurposing drugs for rare diseases
Pankaj Agarwal, SeniorFellow, Computational Biology, GSK

Keynote Panel Discussion: Cell and gene therapy for rare disease: Challenges and Potentials

- Gene therapy challenges

- Latest regulations for cell and gene therapy medicinal products

- Better delivery system in place for efficient gene therapy transfer

- Reimbursement models for gene therapies

- Gene therapies in the pipeline

PLENARY SESSION: Innovative models to accelerate diagnosis in rare disease: Multi-partner sponsored testing programs remove barriers and speed access to a genetic diagnosis.

- Learn how the "Invitae Detect" sponsored testing model is bringing companies together in the same disease space to generate broader awareness and faster diagnosis for more patients with rare disease.

- Learn how expanding an existing program from one partner to three dramatically increased testing volumes and drove more diagnoses across all disorders tested.

- Innovative new structures are increasing utility of genetic testing for patients and clinicians while decreasing risk for biopharma sponsors.

Daniel Anderson, Head of Commercial Partnerships, Invitae

Networking Lunch

Plenary Session: RNAi Therapies: Drugs of significant medical importance

- Current state of RNAi therapeutics development

- Overcoming challenges in developmental process of RNAi treatment

- Overcoming the challenges of delivering RNAi to new cell types

- Leveraging the potential of RNAi technology

- RNAi therapies in the pipeline

Keynote Panel Discussion: Pricing and Return of Investment (ROI) of Orphan Drugs: What is the Right Price Strategy for Orphan Drugs to Continue to Treat Patients and Remain Commercially Viable for Manufacturers?

- Devising funding strategies to develop drugs for the treatment of rare diseases remains a challenge as patient groups are often too small

- Bold new ways to secure investment

- Budget impact of RD therapies create a real concern for payers

- Responsible and evidence-based pricing, and innovative contracting

- Forward thinking approach for better market access to Rare Disease therapies

Afternoon Networking & Refreshment Break

Plenary Session: Financial Impact of a Rare Eye Disease

In this dynamic presentation participants will learn:

- The tools that are critical to level the playing field for a blind person

- The direct costs involved in educating a blind child in the American public school system from Early Childhood through twelfth grade (impact on the school system)

- The lost wages for one family to raise blind children (impact on family)

- The horrifying unemployment and poverty statistics in the blind community (impact on social security, society)

Kristin Smedley, President, Curing Retinal Blindness Foundation

Closing Panel Discussion: The Importance of Patient Networks and Advocacy Groups in Designing of Clinical Trials and Patient Recruitment?

- Understanding the collective voice of the patients and represent the patients’ interest

- Patient involvement is crucial for identifying the questions to ask and the outcomes to assess

- Patient involvement is essential for achieving true translational research

- The identification and proper handling of patient needs in clinical research will yield advantages in terms of clinical and economic benefits.

Leslie Leahy, VP, Medical Sciences, Ovid Therapeutics

Han Phan, Founder And Executive Officer, Rare Disease Research

Chairperson's Closing Remarks