Clinical Trial Outsourcing: A Case Study For Small Biotechnology Companies Operating Globally

Transcription

1 Clinical Trial Outsourcing: A Case Study For Small Biotechnology Companies Operating Globally Cerus Corporation Concord, CA Laurence Corash, MD Chief Medical Officer Meisa Propst, Associate Director Clinical Operations

2 Agenda The corporate environment Clinical trial environment Matrix approach Flexible outsourcing Identifying resources Managing external resources 2

3 KITS SOLD TO PRODUCE >1MILLION UNITS FOR TRANSFUSION 100 Employees Two offices: US and Europe

4 Corporate Environment Focused on INTERCEPT Blood System pathogen inactivation (PI) for blood components to improve transfusion safety Commercial Products Phase 4 studies Post marketing safety surveillance Phase 3 studies for label claim extensions Clinical development Phase 2/3 clinical trials 4

5 Test Article: Blood Component Prepared With Pathogen Inactivation (A similar treatment set is used for platelets.) 5

6 Potential Locations for Clinical Trials Over 100 centers in 18 countries Cerus Europe (Amersfoort, NL) Cerus Europe : Routine Customers Chile Réunion Canary Islands Martinique Guadeloupe French Polynesia 6

7 Complex Logistics For Transfusion Clinical Trials BLOOD CENTER RBC PRODUCTION Regular RBC Inventory (~95% of production) Rare Blood Program: RBC Inventory for Special Patients Acute Anemia: Such as Surgery and Trauma Limited number of transfusions Chronic Anemia: Sickle Cell, Thalassemia, MDS Transfusions every 2-4 weeks 7

8 Screen, randomize Screen, randomize Example: Phase III Studies to Support CE Mark: Acute & Chronic Anemia Studies INTERCEPT ACUTE ANEMIA Elective cardiovascular surgery patients, first time CABG or valve repair (n=50) 1 endpoint = Hemoglobin content per unit RBC support up to 7 days* RBC support up to 7 days* Control CHRONIC ANEMIA** Transfusion-dependent thalassemia major patients (n=70) 1 efficacy endpoint = Hemoglobin usage 1 safety endpoint = Immunogenicity with repeat exposure INTERCEPT 2 Control 4 txns Control 2 4 txns 2 4 txns 2 4 txns INTERCEPT Each patient is on study ~12 months * Day of surgery plus 6 days post-op. 8

9 Components Of The Matrix Blood Center un-blinded team Production and inventory management GMP training Data management Monitoring Hospital blinded team Patient recruitment Patient management Data management Monitoring Data Coordinating Center Specialized Assessments (6MWT)

10 Hosp. A Phase 3 Trial Logistics Hosp. B EDC/IVRS Recruit & Screen Patients Randomization Request through EDC/IVRS Randomization Inventory Management EDC Data Management Warehouse and Distribution Ship Products to BTCs Clinical Trial External Costs Site costs (Patient, Doctor, Nurses, labs) Site Management and Data Monitoring Database Management Warehouse and Distribution BTC BTC Hosp. A Hosp. B Data Entry CRO-X Monitoring Site Management Project Management DSMB Cerus Team Regulatory Project Management Statistical Analysis Programming Safety Deployment Quality

11 Additional Components Of The Matrix Regulatory submissions Biologic and Device Regulations Ethical committee interactions Licensure to manufacture a biologic Establish blood center and hospital communication pathways Understanding the culture Contractual relationships with clinical investigators & hospitals Conflict of interest Financial controls Expert legal opinion Outside counsel in foreign countries

12 Flexible Outsourcing Options Relying on CRO expertise/niche clinical areas Transfusion medicine Cardiovascular surgery Choosing a vendor of appropriate size Local presence, knowledge and experience Complementary Resources Stability of the operating team

13 Identifying Resources - Parameters Capability- Experience, Expertise, Speed, Track Record Capacity- Do they have the right resources? Quality- Quality Control Procedures and Processes, Continuous Monitoring Compliance- Understanding of applicable regulations Financial Considerations- Financial Stability, Long Term Viability, Cost of Services Culture- Ability to build lasting, mutually profitable relationships 13

14 Selection Process Send RFPs to at least three vendors Evaluate proposals and the team Project manager CRA team Present to the clinical team listing pros and cons of each CRO QA and Clinical Operations audit of top 2 CROs Budget negotiations Bid defenses by the top two potential CROs

15 Criteria For Selection Team Chemistry CRO and Project Manager Experience CRA experience Efficient Vendor SOPs Project Execution Plan Problem Solving Processes How problems are solved CRO Staff Turnover Company Size and Financial Stability Colleague referrals

16 Managing External Resources Open Communication Mutual Trust and Respect Shared risks and rewards Commitment to one another s success Co-monitoring to insure quality Ability to deal with differences in a constructive fashion A CRO/vendor is an extension of the internal team at the Sponsor. Sponsor holds the primary relationship with the site 16

17 Conclusions The clinical trial environment is a high risk environment Limited control over the clinical setting Costly Time consuming Results are critical to corporate milestones Investment community watching closely Requires a high level of interactive collaboration Responsiveness Flexibility to solve problems Ability to be proactive Connecting the dots making the matrix functional 17

18 The Environment For Blood Transfusion Can Change Rapidly: La Reunion Island 2006