Home » Agenda » Day 1

Day 1

22nd Orphan Drugs and Rare Diseases Global Congress 2024 Americas

Enhance Information Sharing, Improve Collaboration to Increase Drug Approval for Rare Diseases

Boston, MA, USA

Conference Agenda

Day 1: Thursday - 14th November 2024

MACRO OUTLOOK AND CURRENT TRENDS

  • What innovative changes are coming in research, clinical development, manufacturing, registrational paths, and commercial models that will impact the rare disease community
  • Regulatory Framework for Drugs for Rare Diseases
  • Insights and recommendations for those preparing for their go-to-market
  • 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

Moderator:

Panelist:

  • After FDA approval, what are the accessibility challenges that rare disease patients face (esp those on state programs)?
  • What are some policy changes that could increase the ability for patients to access treatments in a timely manner?
  • How are states grappling with the sustainability challenges in the orphan drug space?

Erica Barnes, Executive Director of the Minnesota Rare Disease Advisory Council

  • Understand the impact of advanced therapies on commercial models
  • Examine innovative ways in patient finding
  • Discuss ideas for getting patients to the right treatment center

Bennett M. Smith, SVP, GM, North America, Orchard Therapeutics

  • This presentation will focus on the role of RWD in natural history and patient registries.
  • How data can be utilized to help inform study design
  • The use of RWD as an external control and to find eligible patients for trials
  • Using RWD with prior clinical trial data to better understand patient outcomes.

Dr. Femida Gwadry-Sridhar, Founder and CEO, Pulse infoframe Inc.

  • Determining accurate patient population estimates for rare diseases is critical at all stages of drug development from R&D through to securing orphan drug status and commercialisation.
  • Standard methods pose significant limitations due to the paucity of data, which presents risks to decision-making processes.
  • The availability of genetic databases has enabled a new “bottom-up” approach to establishing accurate data on patient populations, and mitigating such risks.
 

Mohamed H. Ladha, President & GM, North America, RECORDATI RARE DISEASES

  • Overcoming hurdles to getting innovative therapies to more rare diseases patients around the world
  • 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
  • Unconventional payment models to ensure innovative treatment can be accessed by rare disease patients
  • Technologies that improve scalability, decrease the cost of goods, enhance overall safety, or increase durability
  • Making manufacturing and commercialization viable

Moderator:
Alison Schecter, Chief Executive Officer, Molecules to Medicine Advisory Group

Panellist:

  • What is FDA’s Project Optimus Oncology Initiative and draft Optimal Dosage Regimen (ODR) guidance for oncology?
  • How does one apply FDA’s Project Optimus and ODR guidance to developing drugs for orphan oncology indications?
  • For orphan oncology indications, does the requirement of having an acceptable balance between benefit and risk for approval conflict with the requirements of Project Optimus and the ODR guidance?

  David Young, President, Research & Development, Processa Pharmaceuticals, Inc

  • In vivo phenotypic screens can identify therapeutic targets underlying common pathological problems in neurological and rare disorders
  • KCC2 potentiating therapies for neurological and rare disorders involving excitation/inhibition imbalance and neuronal circuit disinhibition in the CNS
  • Orphan drug indication prioritization and commercialization: Portfolio of KCC2 therapies to match target-product-profile (TPP) and modality to patient needs

Shane Hegarty, Chief Scientific Officer & Co-Founder, AXONIS Therapeutics, Inc.

  • Definition of Orphan and Ultra-orphan Indications in Oncology
  • Challenges of Development: The Regulatory and Developmental Environment
  • Role of Big Pharma: Recent Trends
  • Prospective Strategies to Overcome the Challenges

Sanjeev Luther, President & CEO, Cornerstone Pharmaceuticals

  • 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
  • Drug repurposing for rare diseases
  • Special challenges that rare diseases create for research and product regulation
  • Current public policies relevant to product development for rare diseases

END OF DAY 1

Scroll to Top