Definition: Managed Entry Agreement

| Presentation Title | Presenter Name | Date | Subject | Business Use Only 1

Luca Pani, AIFA, 2014

§ It is an arrangement between a manufacturer and payer/provider that enables access to (coverage/reimbursement of) a health technology subject to specified conditions.

§  These arrangements can use a variety of mechanisms to address uncertainty about the performance of technologies or to manage the adoption of technologies in order to maximize their effective use, or limit their budget impact.

| Presentation Title | Presenter Name | Date | Subject | Business Use Only 2

| Presentation Title | Presenter Name | Date | Subject | Business Use Only 3

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Key Goal: Leverage your PSP to demonstrate real world effectiveness value to gain access to payers

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§ Rare diseases present unique challenges for: - Diagnosis, Testing,

- Reimbursement, Financial assistance,

- Ongoing treatment

- Monitoring

§  PSPs for Rare Diseases are: -  Product focused, patient centric;

- Gather valuable real life data extremely useful for payers – useful for potential Adaptive Listing initiatives;

- Manufacturer funded

- Opportunity to become more centralized to support overall Rare Disease Strategy

Ultimate objective is to build a build a different framework for Orphan Drugs which will shorten the timelines to access

Health Outcomes Research

Value to Payers

Patient Support Program

Demonstrate

Real World

Effectiveness and

Value

Capitalize on PSP to generate real world data

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Patient Support Programs — Objectives §  Allow patients to access commercial therapy §  Assist in the navigation of multidisciplinary services §  Coordinate patient logistic services

(F/A, Pharmacy, Distribution, Nursing)

Post – Marketing Surveillance and Real World Data - Objectives §  To track outcomes in real world setting §  To illustrate and prove value of drug

Reimbursement – Objectives §  To gain coverage and funding with payers §  To gain coverage on behalf of patients Clinical Research

§  To prove clinical efficacy and safety (Phase I–III)

SAP

NOC pre-launch post-launch

Managed Access: To demonstrate real world effectiveness and value to accelerate access for patients

Health Outcomes Research

Value to Payers

Patient Support Program

Demonstrate Real World

Effectiveness and

Value

Our ultimate objective is to build a different framework for Orphan Drugs which will ultimately prove their value, and shorten the timelines for

access 6