Agenda

28th - 29th June 2016

Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA
• Short introduction to the history of Orphan Drugs Act and why it was set-up
• Evolution of the process for obtaining orphan designation over the years including how does one approach the agency to obtain an orphan designation and what are the critical success factors for obtaining this designation
• The misconceptions regarding orphan designation, exclusivity and orphan drugs approval process and what are the key reasons applications get rejected
• Alignment of orphan drugs designation between US, European and Japanese agencies. Critical considerations.
• The future of approving drugs thru orphan drugs designation pathway in particular near and long term changes that are coming in the future with respect to orphan drugs approval process.

Dr. Gayatri Rao, Director for the Office of Orphan Products Development, FDA
Interviewer: Dr. M. (Ken) Kengatharan

• 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?

Oved Amitay, VP - Head of Commercial, ALNYLAM PHARMACEUTICALS, USA

• 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 options are available for both investors and companies?
• What are the risks associated with investing in orphan drugs?

Moderator:
Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA

Panelists:
James McArthur, Founder and Chief Scientific Officer, CYDAN
John J. Maddox, Jr., Managing Director, Infusion Pharma
Sara M. Nayeem, M.D, Partner, NEW ENTERPRISE ASSOCIATES

• Rare disease opportunities
• Orphan drug development challenges
• Use of alternative control groups
• Importance of treatment effect size
• Adaptive trial designs
• Accelerated approval pathway

Gerald F. Cox, Vice President, Clinical Development, GENZYME, A SANOFI COMPANY

• New strategies are needed to develop therapeutic options for the 95% of rare disease patients without specific therapies today
• Precision medicines focusing on a specific type of mutation present in multiple diseases offers one approach to tackling the deficit
• The nonsense mutation read-through agent is an example of this strategy

Mark Rothera, Chief Commercial Officer, PTC THERAPEUTICS

• Key reasons why funding of diagnostics only related projects are few and far between in the last 5-10 years and how can Orphan Diagnostics Act change this landscape
• Current and future role of diagnostics in the clinical management of orphan and rare diseases
• What would be the key components of a potential Orphan Diagnostics Act compared to the Orphan Drugs Act of 1983
• What could be the next steps to make Orphan Diagnostics Act a reality: Role for key stakeholders

Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA

• Systematic understanding of disease mechanisms leading to the discovery of novel targets and pathway for identification and characterization of orphan disease targets
• Tools already available to aid in fight to prevent and treat these debilitating disorders
• What are the up-coming emerging treatment with great potentials
• Strategies to increase awareness of health care professionals to reduce the time to diagnosis and treatment to these diseases
• What bottlenecks still exist in the development and availability of oncology orphan drugs?
• How to overcome a higher than average degree of uncertainties for clinical as well as population based decision-making
• Framework study protocols in specific rare cancers
• Ways on conveying new agents early to rare cancer patients

Moderator:
Aoife Brennan, VP Research and Development, Rare Diseases, Biogen

Panelists:
Michael Hennig, CEO, leadXpro AG, Switzerland, previously from F. Hoffmann - La Roche Ltd
Prof. Arndt Rolfs, Chief Executive Officer, CENTOGENE AG
Dr.Dione T. Kobayashi, VP, Preclinical Translation, CYDAN

• Challenges and opportunities for target selection in rare disease indications
• Efficient application of biophysical methods and structure information for challenging transmembrane protein targets
• Example: generation of lead compounds for treatment of Retinitis pigmentosa

Michael Hennig, CEO, leadXpro AG, Switzerland, formerly from F. Hoffmann - La Roche Ltd

• Orphan drugs represent the most dynamic market in the next decades
• The development, market entry and long-term success of an orphan drug depend on the knowledge of the differences in the multiple geographical areas
• Clinical phenotype of the patients and the epidemiology
• Biomarkers will not only help to identify patients but will gain in longitudinal monitoring data

Prof. Arndt Rolfs, Chief Executive Officer, CENTOGENE AG

• Key strategic elements for drug repurposing
• What are the potential barriers and how to deal with it?
• Advancing the evaluation and development of product that can potentially be used in diagnosis or treatment of rare diseases

Moderator:
Dr. M. (Ken) Kengatharan, President, ARMETHEON

Panelists:
Dr. Francois Nader, Chairman, ACCELERON PHARMA
Tom Held, VP Rare Diseases Franchise Head, NOVARTIS
Aoife Brennan, VP, R&D, Rare Diseases, BIOGEN

• Key advancements in gene therapy
• Opportunities and challenges the clinical, regulatory and policy environment
• Preparing the marketplace for a different business model

Elizabeth M. White, AVP, Rare Disease Commercial Development, PFIZER

• Discuss the paradigm shift in the stakeholder landscape with specific focus on patient centricity and payers
• How an integrated medical affairs strategy and engagement can enhance medical value of orphan drugs and address complex requirements from a broad range of stakeholders (Physicians, Patients, Payer arch types)
• How medical insights through Big Data and Patient journey assessments are strategically/tactically influencing both drug development and commercialization of orphan drugs

Moderator:
Usman Iqbal, Sr. Medical Affairs Leader-Neuroscience,

Panelists:
David Boothe, Global Commercial Leader, GSK
Vanessa Rangel Miller, VP, Genetic and Data Management Services, PatientCrossroads
Mark Rothera, Chief Commercial Officer, PTC THERAPEUTICS
Oved Amitay, VP - Head of Commercial, ALNYLAM PHARMACEUTICALS, USA

Dr. M. (Ken) Kengatharan, President, ARMETHEON, INC., USA

• 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 are payers supporting patients in need of orphan drugs?
• Will policies change in the near to medium term in support of patients with rare diseases?

Dr. Francois Nader, Chairman, ACCELERON PHARMA

When planning to launch your orphan drug product into the EU market it is important that you fully understand the market requirements and the challenges you may face. In this session, Almac will discuss these factors and share with you our extensive commercial experience with Orphan Drug launches. This session will help you to be better prepared for what is ahead and help identify any areas for which you need expert support to ensure your commercial launch is successful.

Michael Rooney, Senior Business Support Manager, ALMAC GROUP

• What can be done by healthcare providers to be proactive in monitoring side effects that can put patient safety at risk and effectively communicate results to the developer?
• Why should patients’ information about their treatment experience be collected and how can this steer the evolution of orphan drugs?
• How social media plays a role for developers and patients to communicate and how companies can utilize social media to innovate and create new drugs for patients?

Moderator: Tom Held, VP Rare Diseases Franchise Head, NOVARTIS PHARMACEUTICALS CORPORATION

Panelists:
Tom Croce, Head – Global Patient Advocacy, SHIRE PHARMACEUTICALS, USA
James McArthur, Founder and Chief Scientific Officer, CYDAN
Dr. Barbara Handelin, Chief Executive Officer, BIOPONTIS ALLIANCE, USA
Patricia Ferland Weltin, CEO/Founder, RDUF
Ian Estepan, Sr. Director of Corporate Affairs, SAREPTA THERAPEUTICS

With pharmaceutical pricing having attracted new levels of legislative and payor scrutiny, we examine the key trends that make impact pricing and the implications for future orphan drug pricing

• Key drivers impacting future pharma pricing latitude
• Value vs. affordability
• Potential impact for orphan products
• Advantaged vs. disadvantaged orphan products
• Implications for development strategy
• Special situations

John J. Maddox, Jr., Managing Director, Infusion Pharma Consulting LLC

• VC- funded biotech or pharma are the traditional partners for rare disease drug development
• A new model has evolved- focused, well funded, nimble - the rare disease accelerator
• Experience of the Cydan accelerator- 3 years of data

James McArthur, Founder and Chief Scientific Officer, CYDAN

• A brief history of the Orphan Drug Act
• Why Orphans are Special
• Nitty Gritty of the Orphan Designation Process
• Specific Review Issues for Orphans

Dr. Tim Cote, Founder and CEO, COTE ORPHAN

• Patients are increasingly influential in their treatment choices and this is amplified in orphan and rare diseases
• Their experience of their disease and their treatment is rich, complex and multi-factorial and can impact brand success or failure
• Breaking down this experience and understanding these dynamics helps build product ecosystems that deliver better patient experiences, better healthcare professional experiences and support brand differentiation and success

Robbie McCarthy, Vice President, Rare Diseases and Patient Portfolio, SPHERIX GLOBAL INSIGHTS

• 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?

Moderator:

Panelist:

Tom Croce, Head – Global Patient Advocacy, SHIRE PHARMACEUTICALS, USA
Michael Rooney, Senior Business Support Manager, ALMAC GROUP
Rena Denoncourt, Director, Market Access, ALNYLAM PHARMACEUTICALS
Ian Estepan, Sr. Director of Corporate Affairs, SAREPTA THERAPEUTICS

• What are some of the strategies for stakeholders to come together to get orphan drugs to patients especially in emerging countries?
• How can stakeholders work together to promoted international collaborations in developing orphan drugs and make it more accessible to all patients?

Dr. John ZL Gong, Chief Executive Officer, 3D MEDICINES, CHINA

• Addressing payer requirements in clinical development programs for Orphan drugs
• How to distinguish and evaluate valuable propositions from government and private payer
• How to improve pharma’s comprehension of the complicated payer market environment and what it takes to improve game plan and utilization
• Evaluate market access drivers, barriers for products in development and develop recommendation for strategy and actions to undertake.

Moderator:
Dr. Tim Cote, Founder and CEO, COTE ORPHAN

Panelist:
Tom Held, VP Rare Diseases Franchise Head, NOVARTIS PHARMACEUTICALS CORPORATION
John J. Maddox, Jr., Managing Director, Infusion Pharma Consulting LLC
Rena Denoncourt, Director, Market Access, ALNYLAM PHARMACEUTICALS

• What are the key drivers that will steer the innovation and access in emerging markets?
• What are the financial, clinical and regulatory risks involved?

Moderator:
Dr. John ZL Gong, Chief Executive Officer, 3D MEDICINES, CHINA

Panelist:
Vanessa Rangel Miller, VP, Genetic and Data Management Services, PatientCrossroads
Tom Held, VP Rare Diseases Franchise Head, NOVARTIS PHARMACEUTICALS CORPORATION
Brian Dwyer, VP, Business Development, COTE ORPHAN