Agenda

4th - 6th March 2019

ABOUT

This interactive presentation will provide you with an opportunity to learn more about how can manufacturers meet global patient demand for access to medicines prior to approval. This will also discuss some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the market. R&D strategies to be implemented to accelerate market access for orphan drugs will also be explore. And how can patient, governments and orphan drug companies help support each other’s initiative to provide market access to patients?

WHO’S IT FOR

This workshop will be extremely beneficial to those individuals involve in development, commercialization and market access of orphan drugs. Project Managers and Executives in Pharma/Biotech companies, consultancy, Patient Advocacy, interested in knowing how to successfully launch an orphan drug.

ABOUT

This interactive presentation will provide you with an opportunity to learn more about how EAP’s provide pre -launch access for drugs in advance of their Marketing Authorization (MA) for patients with life- threatening conditions and no approved treatment options. Best practices in EAP planning, Set-Up and management, and provide information on current regulatory changes

WHO’S IT FOR

This workshop will be extremely beneficial to those individuals considering an EAP as part of their drug development strategy. Project Managers and Executives in Pharma/Biotech/Advocacy interested in knowing how to set-up an Early Access Program.

What contribution have the big pharma companies to guarantee growing development of orphan drugs?
Manufacturers collaboration with governments, payers and patients to continue fulfilling unmet needs?
How are small biotech start-ups contributing to the orphan drugs market?
How can developers demonstrate value for orphan drugs?
What is left to be done to increase growth in shortest possible time?
Panelist:
Christian Girard, Co-Founder & CEO, Overlord Pharmaceuticals

  • Successful example of value-based pricing in Rare Disease
  • Potential changes in treatment paradigms to improve effectiveness and reduce cost of treatment
  • Implementing measures designed to limit their exposure to costs associated with orphan drug reimbursement.
  • Legislative incentives to encourage development of orphan rare diseases therapies
  • Impact of orphan diseases treatment on healthcare payment system

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

  • 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 into Europe

RESERVED

Why should drug repurposing considered a sustainable strategy for rare diseases?
Assessing the commercial roadblocks for growth of repurposed drugs
Is there a major role for repurposing of old drugs for new uses in orphan and rare disease space?
Examples of collaborative models to drive generic drug repurposing in rare diseases

Current technological approaches and challenges
Review of the cutting-edge research programs and clinical trials
Advances in gene therapy
What is on the horizon for gene therapy for orphan drugs?

  • The collaborative model with biotech /pharma companies,
  • Support clinical development through EU funding and
  • Creation in partnership with the french Venture capital Sofinnova Partners, of a dedicated fund to invest in early stage project in the field of rare/genetic diseases to accelerate development of new therapies.

Panelist:
Lucia Faccio, Director of Research & Development, Fondazione Telethon

  • 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

Panelist:
Pedro Franco, Director of Europe for Global Regulatory & Scientific Policy, Merck KGaA*

Review of the EMA’s orphan designation program
Range of incentives to encourage the development of medicinal products for the treatment of rare diseases
Current development on EU case law and legislations
Orphan drugs pipeline in the EU

What can stakeholder do to make orphan drugs more affordable to patients?
Stakeholders strategies to encourage progress on orphan drugs policies
How are government collaborating with stakeholders to make orphan drugs readily available to patient?
Stakeholder cooperation to overcome challenges in orphan medicine development
Panelist:
Mercedes Martínez Vallejo, Technical Advisor, Ministry of Health, Consumption and Social Welfare, Spain*
How patient engagement has evolved over the last decade
How does patient engagement benefit patients?
How does patient engagement benefit Regulatory bodies?
How does patient engagement benefit pharmaceutical companies?
Out lay of Project PARADIGM
Panelist:
Maarten Kraaijenhagen, Regional Rare Disease Medical Lead, International Developed Markets, Pfizer

  • How can manufacturers work with regulators in understanding regulatory issues and challenges to minimise risks during development?
  • How should the R&D and commercial leaders in pharma anticipate and sort out the public policy risks and benefits of adopting an orphan drug development and commercialization strategy?
  • The future of approving drugs thru orphan drugs designation pathway particularly in near and long-term changes that are coming in the future with respect to orphan drugs approval process

Strategies in patient recruitment and retention for a successful clinical trial 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?
What Are the factors and steps to be taken to ensure success of the drug development?
Panelist:
Tijana Bugarcic, Managing Director, Rarebase, Evaluator, New Therapies for Rare Diseases, European Commission
How can manufacturers meet global patient demand for access to medicines prior to approval?
How can governments and orphan drug companies help support each other’s initiative to provide market access to patients?
What are some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the market?
What R&D strategies can be implemented to accelerate the market access for orphan drugs?
Scaling up a patient group for Alkaptonuria (AKU)
Building the scientific community around the disease
Organising clinical trials of a promising treatment
Nicolas Sireau, CEO and Chair of Trustees, AKU Society
How advances in science are revolutionising the treatment of rare diseases
Putting patients first in the treatment of rare diseases requires creating equitable access solutions
Our shared responsibility to leave no patient with a rare disease behind
Owen Marks, Country Lead Rare Diseases, Pfizer

  • Improved clinical outcomes require more than just clinical development
  • Understanding the patient journey in rare diseases is critical to successful clinical development
  • Innovative communication strategies, patient journey insights and behavioural science techniques can all contribute to improved patient outcomes

RESERVED FOR COMPLETE HEALTHVIZION

Overcoming issues with finding a suitable site and qualified investigators
What are the outcome measures and researches supporting clinical development
How to create a good working relationship between researchers and patient advocacy
Paulo Fontoura, Global Head Clinical Development Neuroscience, Roche Pharmaceuticals*
What regulatory mechanisms for pricing can be put in place to ensure fast access to needed medicines for patients with rare diseases in the region?
How can payers find ways to reduce R&D costs and eventual launch prices?
What is the argument behind undertaking of assessments if it is known in advance that the drug will not be costs effective? How can this be changed?
Senior Representative from NICE*

  • How can manufacturers meet global patient demand for access to medicines prior to approval?
  • How can governments and orphan drug companies help support each other’s initiative to provide market access to patients?
  • What are some of the methods that orphan drug manufacturers can do to partner with governments to bring orphan drugs to the market?
  • What R&D strategies can be implemented to accelerate the market access for orphan drugs?

Richard Porter, Chief Operating Officer, Therachon

The changing environment for ultra orphan drugs
High hurdles with little data: examples of creative data generation to support value
Julian Trimming, EMEA Regional Commercial Director, Aegerion

  • How to close the gap between regulatory approval and funding agreement?
  • How the key HTA and payers operate?
  • Samples of high profile reimbursement battles
  • How to overcome struggles to meet the strict cost-effectiveness threshold for new therapeutics?

Market implication as a result of “Brexit”
Diverse political and economic implications which can affect payers leading to potential restriction in pricing
Impact potential if current legislation is changed