Amgen Perspectives on Dialysis Clinical Research

1
Amgen Perspectives on Dialysis Clinical Research

• Robert M. Brenner, M.D.
• Nephrology Therapeutic Area Head
• Medical Affairs
• Amgen

2
The Ultimate Objective of Clinical Trials Is To
Improve Patient Outcomes
Period prevalent dialysis patients adjusted for
age, gender, race, primary diagnosis.

Dialysis patients, 2001, used as reference
cohort.
3
Amgen Is Committed to Evolving the
Practice of Renal Medicine

• We use innovative science to bring vital
  medicines to patients with unmet medical needs
• And explore how these therapies can be used to
  improve the lives of the patients we serve

4
Clinical Research Can Be Subdivided Into 3 Types
5
Clinical Research Can Be Subdivided Into 3 Types
6
Amgen Supports Non-Interventional Studies and
Analyses of Pre-existing Data in CKD
7
When One Thinks About Clinical Research
The Focus Is Routinely On Interventional
Trials
8
Trials Are Either Intended For Initial Regulatory
Approval or for Subsequent Submission/Publication

Interventional Clinical Trials
Subsequent Submission
Initial Registration

• RED-HF

Sensipar Outcomes Trial
9
Sensipar Outcomes Trial A Case Study

• A hypothesis was generated based on results from
  observational non-interventional and post-hoc
  analyses
• A treatment regimen for secondary HPT including
  cinacalcet will reduce mortality and
  cardiovascular morbidity compared to a treatment
  regimen without cinacalcet
• Study rationale and protocol synopsis shared with
  experts in the field
• An external Executive Committee formed
• Protocol shared with LDOs
• Protocol shared with global health authorities

10
Sensipar Outcomes Trial A Case Study

• Site feasibility underway, investigator list
  being drafted
• Study to be conducted with oversight from Amgen,
  the Executive Committee and an independent Data
  Safety Monitoring Committee
• Endpoints independently adjudicated
• Independent confirmation of statistical analyses
  will be performed
• Publications to be coordinated by the Executive
  Committee

11
Dialysis Clinical Research Challenges and
Perspectives

• Investigator and study site identification
• Experience with trials intended for regulatory
  submission
• Have adequate dedicated resources to ensure
  robust study execution
• Investigator oversight at multiple dialysis units
• Study contracts with local investigators and
  dialysis organizations
• Variation in LDO CRO scope and activities
• Timely enrollment
• HIPAA and research compliance

12
Amgen Commitment to Research in Dialysis

• Ultimate goal of improving patient outcomes
• Support a broad range of clinical initiatives
• Analyses of pre-existing data
• Non-interventional studies
• Prospective clinical trials for both regulatory
  and non-regulatory intent
• Utilize a collaborative approach to generate
  study hypotheses and trial design with
• Physicians
• Dialysis organizations