Conference Agenda

Orphan Drugs Conference Day 1

Day 1: 10th September 2019

Opening Keynote Panel Discussion: Patient perspective on Living with Rare disease: What really matters?

- Unique challenges in living with a rare disorder

- Social implications and aspects of living with rare disease

- Consequences of living with rare disease

- Experience with healthcare system

​Patricia Weltin, CEO & Founder, Beyond the Diagnosis

Julie Gortze, Founder / President, Rare New England

Neena Nizar, Founder & Executive Director, Jansen's Foundation

Maria Picone, Co-Founder/ CEO TREND Community


08:20 - 09:00

Industry Keynote Panel Discussion: Current Orphan Drugs Landscape and What must be done by Stakeholders to accelerate access and affordability while continuously advancing in R&D and innovations

- Legislation incentivizing pharmaceutical industry to invest in therapeutics for rare diseases

- Advances in sequencing that made orphan drugs development faster and cheaper

- What can be done by healthcare providers to be proactive in monitoring side effects that can put patient safety at risk and effectively communicate results to the developer?

- Why should patients’ information about their treatment experience be collected and how can this steer the evolution of orphan drugs?

- How social media plays a role for developers and patients to communicate and how companies can utilize social media to innovate and create new drugs for patients?

Brian BRONK, Ph.D., Head of BD, Rare Diseases & Rare Blood Disorders, GlobalBusiness Development & Licensing, Sanofi

09:00 - 09:40

Regulatory Keynote Panel Discussion: Current FDA thinking regarding common issues encountered in rare disease drug development. Legislative updates

- Understanding the global rare disease policy landscape

- New steps to meet the challenges of rare diseases

- What are the key elements across diverse rare disease programs and how this can affect patients access to care

- Bridging gaps between policy and practice to guarantee access for patients

09:40 - 10:20

Risky business: success and failure in orphan drugs‘ business model

- The ODRD dilemma – population size vs. price premium

- Key success factors for a powerful and sustainable market access

- Learning from failure: avoidable roadblocks and pitfalls

- Collaborative approaches for a complex environment

Matthias Schoenermark, Professor, President and CEO, SKC Beratungsgesellschaft mbH

11:00 - 11:25

Plenary Keynote: Top 5 Game Changers for Rare Disease Treatment as We Know It Today Ø What are the top issues facing the rare disease community today and who/how will they be addressed?

- What innovative changes are coming in research, clinical development, manufacturing, registrational paths, and commercial models that will impact the rare disease community

- How will patient advocacy and healthcare policy in the US healthcare environment change

- Will the future be a place without rare diseases

Amit Rakhit, Chief Medical Officer and Head of Research & Development, Ovid Therapeutics SKC Beratungsgesellschaft mbH

11:25 - 11:50


Topic to be Confirmed shortly

David Cory, Chief Executive Officer, Eiger BioPharmaceuticals

11:50 - 12:15


Please contact:

12:15 - 12:40

Plenary Keynote: Latest Developments in and the future of the Regulatory Landscape for Approving Treatments for Orphan and Rare Diseases

- How to navigate the current regulations

- How are these regulations influence time to market for orphan drugs?

- Regulatory obstacles faced by manufacturers during the registration process

- Designing best strategies for a successful regulatory approval

Senior Representative from FDA

12:40 - 13:05

Networking Lunch

13:05 - 14:15

14:15 - Attend any of the 4 Topic Focus Streams

Regulatory, Commercial & Current Trends

Access & Collaborations

Innovations, Discovery & Clinical Development

Advance Therapy Medicinal Products

Improving the precision of precision medicine: scientific and policy challenges
- Despite the rapid growth of precision medicine, its use in the clinic is still limited.
- Technical issues, including a lack of standards and issues surrounding data reproducibility, slow the discovery and translation
of targeted therapies.
- Translation of promising therapies to the clinic requires novel approaches to regulation, payment, and patient/provider education.
- Expansion of precision medicine will rely on the development and effective use of real world data, open-access resources, and other innovations.
David Litwack,Director, Regulatory Strategy and Communications, Prevail Therapeutics

Spotlight Presentation by Silver Sponsor

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 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, Marketing, Kadmon Pharmaceuticals

Challenges around the pricing and reimbursement of EDRDs
- EDRDs are a fast growing market segment
- EDRDs are more likely to generate high sales than commonly-used drugs
- The opportunity costs for payers are significant
- Pricing and affordability are barriers to access
- The proposed Canadian pricing framework expected to make prescription drugs more affordable
Elena Lungu, Manager, Policy Development, Patented Medicine Prices Review Board, Canada

The role of patient organizations in approval, pricing and reimbursement of new therapies
- Acting as the honest broker
- Patient preferences
- Improving endpoints
Isabelle Lousada, President & CEO, Amyloidosis Research Consortium

Q & A - Orphan Drugs: Current landscape, Drivers and Future trends.   - What are the growing trends in Rare Disease treatment?   - How can governments and orphan drug companies help support each other’s initiative to provide access to patients?   - Stakeholders collaboration to continue fulfilling unmet needs   - What R&D strategies can be implemented to accelerate the market access for orphan drugs?   - Challenges in bringing orphan drugs to patients after approval   All session speakers in panel

Martine Zimmermann Global Head of Regulatory Affairs, Alexion

Global early access programs to support rareindications
This session will provide key insights into the development and execution of global pre-approval access programs, including:
- Considerations for successful program
- Runway for effective early planning
- Scope considerations -individual vs. cohort programs in global territories

The skinny of data in early access program
Anne B. Cropp, Chief Scientific Officer, Early Access Care LLC

Topic to be confirmed shortly.
Luke Rosen, Founder, KIF1A.ORG / VP, Patient Engagement & Gov't Affairs, Ovid Therapeutic

Why Do Sponsors with Orphan Disease Therapies need to be in Latin America?
- Opportunities – Landscape and Logistics
- Challenges – Culture and Complexities
- Case studies: Pediatric & Adul
Sara Tylosky, CEO and President,

Utilizing Real-world evidence to impact access to medicines
Ashish Dugar, VP, Global Medical Affairs, Sarepta Therapeutic

Topic to be confirmed shortly

Raring to Go for CHI!:Collaborations to Improve the Lives of People with Congenital Hyperinsulinism- A Patient Partnership with Clinicians, Researchers, Nonprofits, and Biotech/Pharma- Patient-Powered Natural History for New Treatments and a Cure- Access to Genetic Testing and Existing Treatments- Improved Clinical Trial Operations, Protocol Design, and Expanded Access- Pilot Research Projects-Travel funds at Centers of Excellence- Physician and Patient Education Awareness through Conferences and Social Media- Family Support ForumJulie Raskin, Executive Director,Congenital Hyperinsulinism International

Q & A Ensuring Patient access and affordability for Rare diseases
- Identifying barriers for access to orphan drugs
- Addressing the challenges associated with patient access to these medicines
- High cost associated with the medicines: Is paying for expensive treatment for a fewpatients sustainable?
- Means of ensuring access andaffordability of Orphan drugs
- Establishing a reasonable price for orphan drugs
All session speakers in panel

Martine Zimmermann Global Head of Regulatory Affairs, Alexion

Pfizer Global Commitment to Rare Disease: A pharma perspective on partnerships with external innovators in the life sciences
- Pfizer has been bringing therapies to people with rare diseases for over 30 years
- Pfizer has strategic and flexible funding vehicles to partner the most innovative science
- Our purpose is to find breakthroughs that change peoples’ lives
Aileen Healy, Exec. Director & Global Head, Rare Diseases, Worldwide R& D, Pfizer, Inc.

Rethinking clinical trial design in Duchenne Muscular Dystrophy (DMD)
- The future of DMD treatment is multimodal
- Selection of study population and study design should be fit-for-purpose
- Disease progression is an individual struggle with an increasing burden
- Participation is key to success
Samuel Agus, Chief Medical Officer, Biophytis

Sponsored Spotlight Presentation - SILVER SPONSOR

Asia – the rare disease final frontier – is this where innovation will be found in the 2020’s?
Robert (Bob) Ward Chairman & CEO, Eloxx Pharmaceuticals

Hermansky-Pudlak Syndrome, Trial Recruitment and Inclusivity
- Brief description of this rare congenital syndrome and its phenotypic presentation
- Lessons learned from past incomplete recruitments: Natural History protocols, Pulmonary Fibrosis Drug Trial, GI Trial
- Brief description of two case histories
- Inclusivity in recruitment with respect to: Cultural difference, Language, Socio-Economic difference and “Big Medicine”
- Disability
Samuel L. Seward, Jr., MD, FAAP, FACP, Professor & Chair, Dept of Medicine, Mount Sinai

Drug Development Resources for Rare Disease Advocates: from Pre-Clinical to Post-Market
- How the PMS Foundation has navigated the discovery stage of drug development
- New Resources available to rare advocacy groups trying to navigate the drug development process
- When should groups start working toward drug development? NOW
- How do they gets started? - Ask for help and check out the resources from NCATS
Megan O’Boyle, Principal Investigator, PMS International

Q & A Strategies to promote research discoveries and development of orphan products to improve the health of people with rare diseases
- 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
All session speakers in panel

Martine Zimmermann Global Head of Regulatory Affairs, Alexion

Platform Approaches to Rare Disease Therapeutics and the NIH Common Fund Somatic Cell Genome Editing Program
- Grouping multiple rare diseases by underlying molecular etiology to accelerate clinical trials: learning from oncology
- Overview of the NIH Common Fund Program on Somatic Cell Genome Editing
- The Rare Disease Clinical Research Network as a platform for clinical trials
Philip John (P.J.) Brooks, Program Director, Office of Rare Diseases Research, NCATS

Novel Approaches for Development of Hybrid Cell Therapies
- Immobilized MSCs on extracorporeal device
- Dynamic sensing of inflammatory states and release of complex pharmaceutical load
- Adaptive clinical trial design
- Dialysis-dependent acute kidney injury: high unmet medical need, fewer that 200,000 patients in US.
Pedro Huertas, Chief Medical Officer, Sentien Biotechnologies

Sponsored Spotlight Presentation - SILVER SPONSOR

Topic to be confirmed shortly

Topic to be confirmed shortly

Topic to be confirmed shortly

Q & A Advance therapies: Promises and potentials for rare disease
- How are advance therapy fundamentally changing the way rare diseases patients are treated
- How policy makers and regulators approach Advance Therapy Medicinal Products (ATMP)
- What are the new development in Advance Therapy Medicinal Products?
- Incentives Supporting Development of ATMP
All session speakers in panel

18:15 - Networking Drinks Reception

19:15 - 21:30 - Networking Gala Dinner