Expert Patient Advocates & 21st Century Therapies Forum Nov 19 - 20, 2015

1. Definition: Managed Entry Agreement
| Presentation Title | Presenter Name | Date | Subject | Business Use Only1
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.

2. | Presentation Title | Presenter Name | Date | Subject | Business Use Only2

3. | Presentation Title | Presenter Name | Date | Subject | Business Use Only3
8
15
6
2
2 7
2

4. Key Goal: Leverage your PSP to demonstrate
real world effectiveness value to gain access to payers
4
§ 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
Demonstr
ate
Real
World
Effective
ness
and
Value

5. Capitalize on PSP to generate real world data
5
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

6. 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