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

Michael A. Swit, Esq., Legal Counsel, Law Offices of Michael A. Swit

  • 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

In this talk we shall explore:

  • The key attributes of a successful orphan drug launch
  • Examples of a successful orphan drug launch

Anthony J Arleth, Managing Director, Consultancy Practice, Pennside Partners Ltd

  • 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

Carl K. Edwards, III, Chief Scientific Officer, DNX Biopharmaceuticals, Inc.
Jonathan Heller, Founder and CEO, Teton Therapeutics Inc
Justin Bingham, Vice President Business Development North America, Centogene AG
Radhika Tripurareni, VP, Medical Affairs, Prothena Biosciences

  • Current situation in Europe from a regulatory and a market access perspective
  • Pricing and reimbursement: key success factors
  • Case studies: stories of success and failure
  • Strategic implications for biopharma companies, planning to expand in Europe

Matthias P. Schönermark, Managing Director, SKC

  • The Biology
  • The Approval
  • The Investor

Jonathan Heller, Founder and CEO, Teton Therapeutics Inc

  • 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

    • Screening strategies and logistics to reach your patients
    • Biomarker development and implementation as a diagnostic
    • Understanding your at-risk populations for genetic testing

Nevine Shalaby, Head of Scientific Collaborations, Centogene AG

  • 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

  • What are some success stories that transitioned from orphan drugs into non-orphan indications?
  • How will this affect the orphan market?

Michael A. Swit, Esq., Legal Counsel, Law Offices of Michael A. Swit

  • 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

  • Describe the process for developing patient-reported outcome (PRO) instruments
  • Identify the challenges of developing PRO instruments
  • Appreciate the benefits and risks of using data and input from patient communities (including those online)

Experience with an Observational Registry in a Rare Disease (MF-CTCL)
Badri Rengarajan, Medical Affairs Lead , ASPIRE Unit, Action/J&J

  • 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

  • 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.

Raquel Cabo, Vice President, Global Market Access, Ovid Therapeutics

  • Review the impact and status of legislation
  • Delineate trends in FDA approvals & pipeline
  • Discuss economic implications on healthcare
  • Compare contemporary channel strategies
  • Pose an argument for the reinvention of Specialty Pharmacy

Gordon J. Vanscoy, Chairman & CEO, PANTHERx Specialty Pharmacy

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

Tijana Burgarcic, Managing Director, Rarebase

  • What are the key drivers and innovation that will steer the innovation and access of orphan drugs?
  • What are innovative approaches that needs to be used in order to obtain regulatory approval
  • How could Advocacy groups and Pharma companies collaborate to develop novel therapies for Rare and Ultra Rare diseases
  • Critical differences between countries in terms of legislations, regulations and policies implemented
  • How stakeholders can take an orphaned innovation and ignore the ROI but instead focus on impacting people's lives?

Ken Kengatharan, CEO, Auxesia Orion and Managing Partner, Atheneos Ventures
Matthias P. Schönermark, Managing Director, SKC
Gordon J. Vanscoy, Chairman & CEO, PANTHERx Specialty Pharmacy