The document outlines the agenda for a pre-conference seminar on payer perspectives regarding orphan drugs to be held from April 20-22, 2016 at the Washington Hilton in DC. The seminar will include presentations from representatives of CMS, Medicaid Health Plans of America, Aetna, NICE, HAS, and research institutions on national and international orphan drug coverage policies, challenges assessing orphan drugs, and approaches to funding and reimbursement. Panel discussions will address US and European payer perspectives, the use of real-world data for orphan drugs, rare diseases in Eastern Europe, and Australia's approach to orphan drug reimbursement through the Pharmaceutical Benefits Scheme. The seminar aims to provide insights into payer requirements and processes

1. Washington Hilton, DC
April 20-22, 2016
Pre-conference Seminar
Wednesday, April 20, 2016
Payers’ Seminar
9:00 Introductory remark
Adam Barak, Managing Director, PPi Healthcare Consulting
US PAYER PERSPECTIVE
9:30 US national medicaid coverage and payment policy for orphan drugs
 CMS National Medicaid Program – prescription drug benefit overview
 State Medicaid Program Requirements
o Drug coverage and payment
 Prior Authorization Requirements (if any)
o Payment methodologies (current and future)
 Manufacturer requirements
o Medicaid Drug Rebate Program enrollment
o Rebate payment requirements; NDA, BLA and ANDA Approvals
John Coster, Director, Division of Pharmacy, Center for Medicare and Medicaid Services (CMS)
10:00 Medicaid state overview
 Drug utilization review
 Establishing clinical step edits and prior authoriztion criteria
 Supplemental rebates for “preferred” position
Jeff Myers, President & CEO, Medicaid Health Plans of America
10:30 Networking coffee break
11:00 Private payer perspective
Ed Pezalla, Vice President, National Medical Director for Pharmaceutical Policy and Strategy, Aetna
11:30 Panel discussion
12:30 Networking lunch break
EUROPEAN PAYER PERSPECTIVE
1:30 Market access for orphan drugs – an English perspective
 A brief outline of the NICE highly Specialised Technology (HST) Evaluation process
 Topic selection and NICE HST Prioritisation Criteria
 Working with stakeholders : including patient support organisations
Sheela Upadhyaya, Associate Director Highly Specialised Technologies, Centre for Health Technology
Evaluation, National Institute for Health and Care Excellence, UK
2:00 Health Technology Assessment of Orphan Drug in Europe

2. Washington Hilton, DC
April 20-22, 2016
 The situation in France
 Adaptive pathways, managed entry agreements and other initiative
 The growing role of Real World Data
 European cooperation between HTA bodies
 Cooperation with EMA ( scientific Advice and post – launch studies)
Francois Meyer, Advisor to the President, International Affairs, HAUTE AUTORITE DE SANTE, France
2:30 Rare diseases, orphan drugs and real world data: how they can fit?
 Need for good quality clinical trials
 How Italy is using data from “real world” registries
 Strong collaboration with patients/advocates and regulatory agencies
 A new perspective of “consistent value assessment” from an International panel group
Carlo Tomino, Research and Development Coordinator, San Raffaele Institute of Reseach, Rome (Former
Head of Research and Clinical Trials, Italian Medicines Agency)
3:00 Networking coffee break
3:30 Rare diseases and orphan drugs in Eastern Europe: the case of Bulgaria and cross-border access
 Mapping of centres of expertise for rare diseases in Bulgaria
 Generating and collecting rare disease data at local level
 Funding for rare disease therapies in deficit spending context
 Multi-criteria decision analysis in assessment and appraisal of orphan drugs
Rumen Stefanov, Director, Institute for Rare Diseases, Bulgaria
4:00 Orphan drugs - the Australian approach to reimbursement
 A brief outline of the Australian Pharmaceutical Benefits Scheme (PBS) and the operation of the
Pharmaceutical Benefits Advisory Committee (PBAC)
 Particular requirements for data for listing orphan drugs on the PBS
 Suggestions for overcoming data deficiencies for orphan drugs, including types of statistical analysis
to account for issues such as crossover
 Other approaches to funding of orphan drugs - existing and potential ideas
Diana Macdonell, former Head, Secretariat, Pharmaceutical Benefits Advisory Committee, Australia
4:30 Panel discussion
5:30 End of seminar followed by a Welcome Drinks Reception