Agenda

12th - 14th September 2017

About

This interactive presentation by WEP Clinical, will provide you with an opportunity to learn more about how Expanded Access Programs, are now a growing regulatory expectation, of companies with innovative medicines that fulfil unmet clinical need. You will learn from the experiences gained in delivering multiple projects, across many therapy areas. We will share best practices in EAP planning, Set-Up and management, and provide information on current regulatory changes such as recent legal acts in the US and EU.

The first part of the session will focus on the legal frameworks, that allow (and encourage) expanded access in the US, selected EU countries and other important ROW territories. The second part, looks at the fundamentals of good project planning and key considerations for Expanded Access. This workshop will be extremely beneficial to those individuals considering an EAP as part of their drug development strategy.

Who's it for?

Project Managers and Executives in Pharma/Biotech/Advocacy interested in knowing how to set-up an Expanded Access Program

Agenda

9:00 – 10.30am - Interactive presentation: Legislation & Expanded Access
10:30 – 11:00am - Networking break
11:00 – 12:00pm - Interactive presentation: Key considerations for Expanded Access

Workshop Leader: Jas Khera, Managing Director, WEP Clinical
Workshop Leader: Amandip Sidhu, Senior EAP Director, WEP Clinical

Assuring success from regulatory to global market access of Rare Disease Treatments

Expertise shared from Mapi Group: Mapi is the leading Patient-Centered Research company serving academia, life science researchers, and the pharmaceutical industry for over 40 years. Mapi’s commitment to patient-focused research is demonstrated not only by our expertise and nearly four decades of service but also through our direct contribution back to the industry. Mapi is among only a handful of global organizations that is capable of engaging with a patients’ complete ecosystem, their HCPs, Patient Communities, payers, and even regulators. And only Mapi has the unique history to make us the most experienced at interacting with all of them.
This workshop will be covering topics on compassionate use, Real World evidence, global regulatory strategies and market access.

1pm - 2pm - PARTNERING WITH PATIENTS FOR CLINICAL STUDY SUCCESS - Kelly Franchetti, Vice President Global Patient Insights and Engagement

This interactive workshop will introduce participants to a 360° approach to obtaining patient insights and applying them for the successful design and execution of clinical studies. Through engaging activities, we will address how to get to the heart of the patient’s decision-making process, assess their physical/emotional/social needs, and identify their motivators, barriers and influencers as they relate to clinical study participation. Participants will then learn how insights are analyzed and applied in order to:

  • Design studies that appeal to the targeted patients
  • Determine effective and motivating recruitment methods
  • Engage and communicate with patients and caregivers
  • Identify retention methods that meet the needs of a specific patient population
  • Develop materials that resonate with patients and caregivers

2pm-3pm - An introduction to the science of endpoint design in clinical trials in the current regulatory and HTA environment in the US and Europe - Rory Graham

  • Regulatory Strategies for Early Scientific Advice: FDA & EMA Scientific Advice, PRIME & Adaptive Pathways Link to Scientific Advice
  • The creation, categorization and hierarchy of endpoints will be described, and contrasts identified between the approaches and requirements of different stakeholder groups, including Regulatory Agencies, Clinicians, HTA bodies, Payors, and increasingly active patient groups.
  • real-life examples of endpoints used in selected therapeutic area
  • techniques for the development and validation of new endpoints
  • highlight guidances and publications that will be of use to a newcomer to the area
  • discuss some areas of non-alignment between different agencies
  • geographical differences in approach.

3:30pm - 4:30pm - Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies - Dr Will Maier

  • How is expanded access used to provide treatment for patients with rare diseases?
  • Compassionate Use Programs in the window between submission and commercialization
  • What are the benefits of conducting clinical studies and registries for rare diseases?
  • What are the types of studies conducted during different phases of orphan drug development?
  • How are patient registries used to generate real-world data for rare diseases and orphan drugs?
  • Ethical Patient Warehousing – working with Advocacy to identify populations in advance of research
  • What are the unique challenges of conducting rare disease/orphan drug registries?
  • How can proactive study management strategies successfully address these challenges?
  • Potential risk mitigation strategies, and alternative registration strategies will be discussed.
  • Engaging patient groups and highlighting their role with – patient groups to be confirmed
  • Globalization – challenges in introducing a drug to different European markets and making sure there are appropriate translational efforts in place
  • Methodologies in developing Real World Data: Engaging stakeholders in developing evidence strategies
  • Market Access Challenges for Rare Diseases: Recent Developments in HTA and Reimbursement Decision-Making Considerations for Orphan Drugs in Europe
  • Global regulatory strategies: The emerging global Regulatory Pathway for Rare Disease treatments

Dr Will Maier, Chief Scientific Officer, Mapi Group

Kelly Franchetti, Vice President Global Patient Insights and Engagement, Mapi Group

Rory Graham, Senior Director, Regulatory Affairs, Europe & International Region, Mapi Group

  • Pricing and reimbursement and the current landscape.
  • Explore accusations and investigations around orphan drugs

Panelist: Robert Derham, Co-Founder, CheckOrphan

  • Regional specifics for orphan drug development and approval
  • Successfullly aligning input from regulatory authorities
  • Maximising utility of national rare disease programs

Mike Page, Executive Director, Global Regulatory Affairs Portfolio Products, Alexion Pharmaceuticals

  • A review of recent drug launch success
  • Current Trends affecting Rare Disease, including the implications of the 21st Century Cures Act
  • Challenges affecting Rare Disease
  • Overcoming the challenges with Real World Evidence
  • Building an Integrated Medical Plan to prepare for success: “Begin with the End in Mind”

Alastair MacDonald, Executive Director Real World & Late Phase, INC Research

  • Sharing information about rare conditions is difficult when doctors generally want to learn more about conditions they commonly treat rather than ones they do not. How can we change that mindset?
  • Orphan drug companies are constantly struggling, with limited budgets, to reach new doctors with new information. How can companies reach and educate those doctors (and not break the bank)?
  • Rare disease patients are often the most knowledgeable about a condition. Are doctors, researchers, and regulators really listening to what they have to say?

James Radke, PhD, Vice President, Editorial, Rare Disease Report

  • Criteria for Designation
  • Regulatory Review Process
  • Expedited Reviews & Vouchers

Timothy R. Cote MD, MPH, Principal & Chief Executive Officer, COTE ORPHAN

  • Introduction to the science of endpoint design in clinical trials in the current regulatory and HTA environment in the US and Europe.
  • The creation, categorization and hierarchy of endpoints will be described
  • A look at the varying approaches and requirements of different stakeholder groups, including: Regulatory Agencies, Clinicians, HTA bodies, Payors, and patient groups.
  • Real-life examples of endpoints used in selected therapeutic areas and Techniques for the development and validation of new endpoints.
  • Guidances and publications to assist newcomers to the area and Non-alignment between different agencies and geographical differences in approach.
  • Potential risk mitigation strategies and Alternative registration and agency interaction strategies

Rory Graham, Senior Director EU regulatory Services, MAPI Group

  • The complex molecular biology of many rare diseases is poorly understood, making traditional target-based drug discovery approaches difficult
  • Target agnostic approaches, which circumvent the lack of molecular understanding, may be a useful alternative strategy
  • A number of target-agnostic approaches will be discussed, along with a detailed discussion of work being done at Recursion Pharmaceuticals to use complex and subtle signatures at the level of individual cells as the basis for broad drug discovery approaches

Christopher Gibson PhD, Co-Founder and CEO, Recursion Pharmaceuticals

  • Key challenges to improve cost-efficiency in clinical development of orphan drugs
  • Regulatory evolution in the review process of Orphan Drugs
  • After examples like Sarepta’s Exondys51 approval, how will the evaluation of clinical benefits change based on endpoints?
  • What aspects of commercially planned clinical trials can contribute to cash-burn during orphan drug development?
  • How do you plan for scale up while improving cost efficiency?
  • What role does multi-stakeholder collaboration in rare disease research play in improving cost efficiency of clinical trials?

Dr. Carlos R. Camozzi, Group Chief Medical Officer (CMO), Simbec Orion

  • What factors enable success in rare disease drug development?
  • Disease and modality selection criteria
  • Embedding patients in discovery and development
  • Accelerating the path to pivotal data
  • Innovation beyond the laboratory

Omar Khwaja, MD PhD, Global Head of Rare Diseases, F. HOFFMANN-LA ROCHE
Anja Harmeier, PhD, MBA, Project Leader Discovery Rare Disease, Roche

  • Identify key aspects of rare disease patients’ mindset and views on logical elements of clinical trial participation
  • Recognize potential implications of patients’ views on study design, as well as clinical trial recruitment, retention and compliance
  • Actions that sponsors, investigator sites and clinical research organizations can take to make clinical studies more patient centric

Leone Atkinson, MD, PhD, Executive Director, Neuroscience Medical and Scientific Services, Covance

  • Challenges and hurdles in developing treatment for ultra-rare diseases
  • Current research, innovation and creative solutions to discover new treatments
  • Ways to ensure that patients have access to these new therapies
  • Decision makers need to recognise the significance of challenges and the need for innovative solutions

Eduardo Bruno Martins, MD, DPhil, Sr. VP, Liver & Infectious Diseases Drug Development, Eiger BioPharmaceuticals

  • Key considerations when determining atypical approval end points and path to obtaining acceptance by regulators and payers
  • Obtaining insights from a rare disease patient community to help design pivotal clinical trials
  • How do you go about engaging rare disease patients as partners in medical research from a practical perspective?
  • Do you have any evidence that rare disease patients are willing research partners?
  • How can we create sustainable research-focused partnership models with rare disease patients?
  • How to increase the industry’s adoption of patient-centric initiatives in clinical development?

Moderator: Dr. John P. Cooke, Chair, Department of Cardiovascular Sciences, Houston Methodist Research Institute
Panelist: Fabrice Chartier, Phd, Group Chief Operating Officer, Simbec Orion Group
Panelist: Leone Atkinson, MD, PhD, Executive Director, Neuroscience Medical and Scientific Services, Covance
Panelist: Kelly Franchetti, Vice President Global Patient Insights and Engagement, Mapi Group

Peter Milner, Chairman, Armetheon Inc
Dr. Ken Kengatharan, President & iCEO, Armetheon Inc

Deep analysis of genomic data to yield genetic diagnoses is the first step in finding potential molecular targets for rare disease therapies.

  • The RG research addresses the need for combined in-depth analysis of the genetic and medical data for each patient which is not provided by physicians, genetic counselors, or sequencing services.
  • Our research model involves crowd-sourcing ideas from our global network of scientists, cloud collaboration, adoption of state of the art computational systems, and utilizing the unique expertise of our in house scientific team to provide actionable insights into each case.
  • Our research model is helping to meet a crucial need for rare disease patients and demonstrates that exome sequencing coupled with in depth analysis leveraging medical history and global collaboration can be deeply insightful.

Rajini Haraksingh, Vice President, Science 2.0, Rare Genomics Institute

Mastering the process of finding, engaging and activating patients is a key success factor for all biopharmaceutical companies developing new treatments for rare diseases. Patients have transitioned online to seek disease, drug and clinical trial information. Thus, if our industry is looking to intersect patients for clinical trials, patient registries or other efforts, social media cannot be overlooked. In fact, a social media strategy must become a basic component of every clinical trial recruitment plan. This talk will explore how to effectively use social media to find and engage patients with rare diseases.

Sandra Shpilberg, CEO, Seeker Health

  • At which stage of the drug development does in-licensing occur and what is the reason for this trend?
  • What criteria do partners look for when partnering for in and out-licensing?

Immo Zadezensky, Senior Director, Head US for Global Regulatory and Scientific Policy, EMD Serono, Inc

  • At which stage of the drug development does in-licensing occur and what is the reason for this trend?
  • What criteria do partners look for when partnering for in and out-licensing?

Immo Zadezensky, Senior Director, Head US for Global Regulatory and Scientific Policy, EMD Serono, Inc

  • Starting with the patient (understanding the patient needs, size and market dynamics)
  • Decoupling the supply chain, building efficiency and agility
  • Challenges / opportunities of small volumes
  • Ending with the patients (managing the last mile)

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

  • Key reasons for tremendous interest from private and public markets in companies with orphan drugs related programs
  • What company profile private and public equity investors looking for when it comes to financing companies with orphan drug programs
  • Could the new RegA+ path to financing companies be a critical driver for more orphan drug companies accessing start-up and growth capital
  • Role of corporate partnership and investors in enabling orphan drug development and the future of M&A in this space.
  • Are there key determinants that differentiate successful orphan drug companies from unsuccessful ones

Moderator: Dr. Ken Kengatharan, President & iCEO, Armetheon Inc.
Panelist: Rob Hopfner, Managing Director, Bay City Capital
Panelist: Paul Dorfman, Managing Director, NYSE
Panelist: Michael Henderson, VP, Asset Acquisition, Strategy, and Operations, Bridgebio

Michael Henderson, VP, Asset Acquisition, Strategy, and Operations, BridgeBio

  • How can reimbursement agencies deal with the uncertainty on efficacy of drugs but yet allow drugs to be available to patients as soon as possible?
  • How to overcome the weaknesses of the current model in market access
  • What are the innovative approach to speed up development and access

Panelist: Dr Will Maier, Chief Scientific Officer, Mapi Group