25-26 June 2018

  • 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

  • How is the US orphan drugs market compared to Europe and what must US do to continue driving the market landscape?
  • What challenges do developers encounter when forecasting market potential of orphan drugs?
  • Is orphan drugs the answer to Big Pharma’s needs to replenish its pipeline?

Alison Shecter, Global Project Head, Rare Disease, Sanofi-Genzyme

  • How orphan drug forecasting are currently undertaken by financial market and industry analysts?
  • How can emerging growth companies assure investors to invest in their companies?
  • What options are available for both investors and companies?
  • What are the risks associated with investing in orphan drugs?

Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures

  • What are some of the main issues related to the cost-effectiveness in rare diseases?
  • How do manufacturers debate whether to engage in R&D process?
  • Analyzing the expected length of time for regulatory approval

Panelist: Jonathan Heller, Founder and CEO, Teton Therapeutics Inc

(Contact for more information)

  • Pfizer has a dedicated organization focused on developing potential therapeutics for patients with rare diseases
  • Pfizer focuses on advancing the best and most innovative science from both internal and external sources
  • Pfizer developed a strategic and flexible model supporting external collaborations
  • Pfizer uses a wide range of agreements to establish collaborations

Aileen Healy, Executive Director, Global Head, ES&I for Rare Diseases, Worldwide R&D, Pfizer, Inc.

  • The development of anti-drug antibodies (ADAs) can compromise the efficacy and safety of biologic therapies
  • We have developed synthetic vaccine particles nanoparticles containing rapamycin (SVP-Rapamycin) to mitigate the formation of ADAs and applied it to biologic drugs to treat rare and orphan diseases
  • Preclinical and clinical data from case studies with a pegylated uricase for the treatment of severe gout, a recombinant immunotoxin for the treatment of mesothelioma, and AAV gene therapy vectors for the treatment of monogenic diseases

Kei Kishimoto, Chief Scientific Officer, Selecta Biosciences

  • Introduction
  • Drug Screening methods
  • Tools for Drug Reporposing
  • Assessing the commercial roadblocks for growth of repurposed drugs
  • Case Study

  • The use of DNA plasmids for rare and new diseases has gained momentum with the use of electroporation, and with this evolution comes some new approaches to Quality and testing of the DNA product.
  • We will look at the benefits of DNA plasmids, proof of their efficacy, and some lot testing developments and concepts for purity and potency that are new to the industry.

EJ Brandreth, VP, Quality, Inovio Pharmaceuticals

  • Rare Disease prevalence and diagnosis
  • The Undiagnosed Rare Disease Patient (URD)
  • Understanding the URD patient and making a case for their support

Romina Ortiz, Chief Operating Officer, VP Patient Advocacy, Rare Genomics Institute

  • Rare Disease Patient Landscape
  • Patients Increasing Roles: from diagnosis to biotech entrepreneurship
  • Providing examples of Patients Driving Partnerships to successful outcomes

Nicole Boice, Founder & CEO, Global Genes - Allies in Rare Disease

  • Patient recruitment and retention for a successful clinical trials in rare diseases
  • How can you prepare healthcare providers to know about the therapy and identify the right patients for your therapy?
  • How can developers make research goals and use of data to be transparent and to be informed about progress or roadblocks in order to build a relationship based on trust with patients?
  • What enhanced ongoing patient support beyond the clinic can be provided to encourage positive relationships between patients and healthcare providers?

  • How to transition from existing business models that meet the needs of supply or demand
  • Dealing with obstacles and challenges in logistics
  • Advances in technology solutions that plays an essential role in supply chain

Jason Cameron, Vice President, Global Supply Chain, Amicus Therapeutics

(Contact for more information)

  • Should drugs be available to patients as soon as possible?
  • What are some of the strategies for stakeholders to come together to get orphan drugs to patients
  • How can stakeholders work together in developing orphan drugs and make it more accessible to all patients?

Jeffrey Bloss, Chief Medical Officer, Aegerion Pharmaceuticals, a Novelion Therapeutics Company

  • What is the current situation in terms of collaboration between different stakeholders
  • Why orphan drugs need collaboration

Tijana Burgarcic, Managing Director, Rarebase

  • Patients as Key Partners in Drug Development – discuss potential framework to improve patient-centricity in drug development
  • Role of Real World Evidence in Rare Disease – discuss role & sources of Real World Evidence
  • Value of Registries in Rare Disease – pros and cons

Sonal Bhatia, Vice President, NA Regional Medical Lead, Rare Disease, Pfizer

  • Payers are becoming more aggressive in scrutinizing price and implementing restrictions to patient access that have traditionally been reserved for competitive, high budget impact drug classes.
  • Recent development in orphan drugs pricing and reimbursement and the impact of those changes
  • Addressing some of the major issues facing payers and orphan drug manufacturers, highlighting the way these have been handled by both parties.
  • Implementing measures designed to limit exposure to costs associated with orphan drug reimbursement.

  • What are the key drivers and innovation that will steer the innovation and access of orphan drugs?
  • Existing regulations and policies utilized by countries to enable access to orphan drugs
  • Critical differences between countries in terms of legislations, regulations and policies implemented