1. Industry Perspective: Expanded Access Programs
Barbara Klencke, M. D. Senior Vice President, Development
Onyx Pharmaceuticals, an Amgen Subsidiary
Introduction:
Your 2 decades of patient care experience
Medical oncologist at UCSF
Firsthand experience treating patients who have run out of treatment options and the impact on families
Industry experience
Strong interest in advances that have potential to transform lives of patients with cancer

2. Disclaimer
The views expressed herein represent those of the presenter and do not necessarily represent the views or practices of the presenter’s employer or any other party.

3. Approved medicines Clinical Trials Expanded Access Types of Access
Regulatory approval provides broadest access
Clinical Trials
Evaluation of safety and efficacy of medicines to support approvals
Expanded Access
Single patient and
treatment protocols
Primary goal: access
Ultimate goal: to provide broadest access possible to all patients who could benefit from a medicine
Fastest way to provide broadest access: through approval mechanism, so robust regulatory strategy needs to be priority
FDA has programs/pathways in place to accelerate regulatory process for important new medicines
Breakthrough therapy designation
Accelerated approval pathways
Surrogate endpoints
Importance of partnering with FDA, Advocating w/ FDA for early approvals
Clinical trials:
Awareness, i.e., Clinicaltrials.gov
Patient groups: Advocate w/ companies to perform clinical trials that enroll patients with the greatest unmet needs
Expanded Access:
Can provide patients with access to a medicine w/ a proven benefit/risk profile, while regulatory strategy in place
Single patient programs
Clinical trials with primary goal, or primary endpoint, of access

4. Goals of Expanded Access/Compassionate Use
Provide seriously ill patients, who have no other treatment options, access to investigational new drugs that are not yet approved, based on a request from the treating physician
Set context:
Ultimate goal: to provide broadest access possible to all patients who could benefit from a medicine
Fastest way to provide broadest access: through approval mechanism, so robust regulatory strategy needs to be priority
Role of Expanded Access:
Can provide patients with access to a medicine w/ a proven benefit/risk profile, generally once regulatory strategy is clear

5. 1 2 Expanded Access Unapproved Therapies Approved Therapies
Access to a product not yet approved anywhere in the world, in any indication
Access in a region beyond where a product is currently approved
Access for an unapproved indication
Various models for expanded access

6. Expanded Access Considerations
Sufficient Benefit/Risk Data
Sufficient clinical evidence of safety and effectiveness established
Defined Approval Pathway
Medicine is under active development in the indication
Regulatory strategy determined and underway
Program Viability
Sufficient medicine to ensure adequate supply for clinical trials
Sufficient staff to support program
Regulatory mechanism in the region to support expanded access
Patient Need
The patient has a serious/ life threatening disease or condition
No comparable or satisfactory alternative therapy
Discuss four considerations for expanded access programs

7. Case Study: Partnering for Patient Access
Collaboration with Multiple Myeloma Research Foundation
Patient access prior to FDA accelerated approval
Access for more than 300 patients
CMAP: example of successful expanded access program
Multiple myeloma: rare blood cancer, new treatments needed
Carfilzomib Myeloma Access Program (CMAP) established to bring carfilzomib to patients who had exhausted available treatment options
Keys to success
Program launched in 2011 after benefit/risk profile for carfilzomib understood, US regulatory process defined and underway
Partnership with Multiple Myeloma Research Foundation (MMRF) advocacy group which simultaneously advocated w FDA for Accelerated Approval of Carfilzomib
Results
More than 300 patients received carfilzomib through CMAP before cafilzomib achieved US accelerated approval in 2012
Onyx provided patient support and service program to help patients transition from CMAP clinical trial to commercial Kyprolis, once approved

8. Shared goal: Broad patient access to important medicines
Next Steps
Shared goal: Broad patient access to important medicines
Consider all types of access:
Rapid regulatory approval of safe, effective new medicines
Clinical trials
Expanded Access
Partnership and support of FDA, clinical trials
In summary, patients, regulators and companies share a common interest in bringing important new medicines to the patients that need them the most once sufficient evidence of safety and effectiveness is established
Informed patients and patient advocacy groups can influence the process through partnership and support of FDA and the regulatory process, and through participation in important clinical trials.