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

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