THE EVOLVING BUSINESS MODEL FOR

Transcription

1 THE EVOLVING BUSINESS MODEL FOR CLINICAL RESEARCH CHRIS BEARDMORE CHIEF EXECUTIVE OFFICER 6100 CENTER DRIVE, SUITE 600 LOS ANGELES, CA (424)

2 Personalized Medicine THE BLOCKBUSTERS OF THE FUTURE

3 Translational Research MOVING BASIC RESEARCH FINDINGS INTO CLINICAL PRACTICE Promotion of concept implies that research is conducted within a learning system. Integrated Into Clinical Care Multi-disciplinary Data Cloud Based (shared clinical data for public good) Trusted Scientific Broker

4 National Cancer Institute ANNUAL PLAN AND BUDGET FOR THE NATIONAL CANCER PROGRAM TH Anniversary of the NCI NCI Budget for FY 2013 was $4.8 Billion Additional Cancer Research Funded by NIH, the CDCP and the DoD IOM Report 15APR2010 Restructuring of Cooperative Group System

5 Biopharmaceutical Companies THE MARKET Top 20 Capitalized at Over 1.3 Trillion 2013 Global R&D Spending $135.9 Billion (projected) Oncology Leading Market Segment Accounting for 23.9 Billion R&D Costs and Delays Threaten Shareholder Returns

6 Study Conduct Process HOW DOES IT WORK TODAY?

7 Study Conduct Process A PROCESS FOR 2020?

8 The Federal Approach A ROBUST STANDING CANCER CLINICAL TRIALS NETWORK IS ESSENTIAL Reconfigure/Consolidate Cooperative Groups (speed and efficiency) Alliance for Clinical Trials in Oncology (CALGB, NCCTG, ACOSOG) Children s Oncology Group (was POG and CCG) ECOG-ACRIN Cancer Research Group (ACRIN and ECOG) NRG Oncology Group (NSABP, RTOG, GOG) Incorporate Innovative Science Biorepositories / Novel Designs / Standards for New Technologies Improve Prioritization/Selection/Support/Completion of Clinical Trials More Process Driven Phase IIs Fewer Large Phase IIIs Incentivize Participation $2,000 Per-Case Reimbursement a Non-Starter.

9 TransCelerate Biopharma, Inc. THE MARKET Risk Based Monitoring Common Tools and Triggers to Identify Risk Categorization Criteria for Low, Medium and High Risk Trials Site Qualification and Training Common Criteria and Recognition of GCP-Training Common Forms to Collect Generic Information About Study Sites Clinical Data Standards Acceleration of Standards Development CDISC Partnership Comparator Drugs New Model Permits Direct Shipment Between Companies Master Service Agreements Common Investigator Portal

10 Association of Community Cancer Centers ANNUAL SURVEY REVEALS MISALIGNED INCENTIVES community oncologists are being marginalized despite the expectation that these same community providers will enthusiastically support, contribute to, and ultimately implement these programs. Michael A. Kolodziej, M.D. Medical Director US Oncology J National Comprehensive Cancer Network 2011; 9:

11 84% Unaware of Clinical Trials Only 2-4% Participate in Clinical Trials Spending/Marketing Does Not Help Participation Improves Outcomes (Phase I) 17.8% Objective Response Rate 34.1% Stable Disease or < Partial Response Toxic Death Rate.54%

12 Focus on Value Add Activities ADDRESS CHALLENGES BY BEING A PART OF THE SOLUTION Local Efforts Establish Proper Payer Mix - Federal vs. Industry Match Studies to Patient Population/Treatment Standards Bill for Routine Patient Care Costs Prepare Flexible and Reusable Administrative Infrastructure Commit to More Flexible Use of Central IRBs Prepare Flexible and Reusable Clinical Infrastructure Fight Regulatory Fundamentalism Collaborative Efforts Clinical Trial Agreement Language Preparing for Just-In-Time Accrual

13 Determining Payer Mix PROGRAM FINANCIAL HEALTH HELPS PATIENTS Cooperative Group Trials $2,000 per-case Industry Sponsored Trials $16,499 per-case (oncology) Phase I - $19,282 Phase II - $12,962 Phase III - $11,649 N OTE: Bill for routine patient care costs.

14 Selecting Studies MATCHING STUDIES IN THE ERA OF PERSONALIZED MEDICINE Build Base of Studies Open to Larger Number of Patients Enroll 15 or More Patients Per Year Gravitate Toward Multiple Populations in Inclusion/Exclusion HER2, HRG, TGF-β2 in Multiple Disease Types Focus on Earlier Phase Studies (Phase I, IB or II) Add Studies Focused on Certain Lines of Therapy Enroll 5 or More Patients Per Year Understand Patient Population/Treatment Standards Accept Studies of Novel/Exciting Compounds Enroll 2 or 3 Patients Per Year Participate in Cooperative Group and Site Management Organization

15 Routine Patient Care Costs REDUCES COSTS FOR SPONSORS AND INCREASES RETURNS FOR SITES Current State Laws Require Insurance Companies to Pay for Routine Patient Care Costs for Patients Participating in Cancer Clinical Trials (highlighted in blue) Patient Protection and Affordable Care Act (HR3590, Section 2709) Requires All Insurance Companies to Pay for Routine Patient Care Costs in Approved Clinical Trials. Source:

16 Affordable Care Act Coverage FOR HEALTH PLANS NEWLY ISSUED OR RENEWED AFTER JANUARY 1, 2014 Approved Clinical Trial means a phase I, phase II, phase III or phase IV clinical trial that is conducted in relation to the prevention, detection, or treatment of cancer or other lifethreatening disease or condition and is described in any of the following subparagraphs: Federally Funded Trials (NIH, CDC, AHCRQ, CMMS, Cooperative Group, DOD/DVA) Conducted Under Investigational New Drug Application Reviewed by the FDA. Inclusions - All Items and Services Typically Provided for Individuals Not in a Clinical Trial. Exclusions Investigational Item, Device or Service Itself. Items and Services Provided Solely for Data Collection and Analysis Needs Services Clearly Inconsistent with Accepted/Established Standards of Care.

17 Administrative Infrastructure ACCOUNT FOR STUDY COMPLEXITY Research Department Program Design Ancillary Service Agreements Study Identification Site Qualification Contract/Budget Negotiation Site Initiation Regulatory Research Billing/Collection Clinical Trials and Support Services Phase II, III and IV Clinical Trials Phase I/II and Utilization of Ancillary Support Services Community Physicians

18 Example Administrative Pitfall EVOLUTION IN INSTITUTIONAL REVIEW BOARD SYSTEM Local Institutional Review Board Strength Local Context and Oversight Weakness Speed and Efficiency National Cancer Institute Central Institutional Review Board Strength Reduces Administrative Burden Weakness Loss of Local Context and Oversight Deferring to an Independent IRB Strength Reduces Administrative Burden Weakness Loss of Local Context and Oversight Improvement Defer to Accredited Central IRBs

19 Clinical Infrastructure ACCESS TO PATIENTS AND FLEXIBILITY FOR PAYERS Pharmacy Hub Facilities 1 Laboratory 1 Interventional Industry Sponsor 1 1 Ophthalmology Cardiology

20 Conflicting Expectations/Demands ITS NOT THE REGULATIONS ITS HOW WE PREPARE FOR COMPLIANCE Regulatory Fundamentalists Individuals who demand strict adherence to regulatory requirements. Characterized by unwavering attachment to a set of irreducible beliefs. Faultless in their actions because they fully complied with and enforced all regulations. Regulatory Rationalists Individuals who exercise judgment in aiming to fulfill the intent of regulations while attempting to facilitate progress. Actions characterized by an understanding of law/regulation, guidance and policies/procedures. Stewart, D., 2010, Equipoise Lost: Ethics, Costs and the Regulation of Cancer Clinical Trials, Journal of Clinical Oncology, v. 28, pp

21 Example # FORM - FDA STATEMENT OF INVESTIGATOR Form has two purposes: 1. To provide the sponsor with information about the investigator s qualifications and the clinical site that will enable the sponsor to establish and document that the investigator is qualified and the site is an appropriate location at which to conduct the clinical investigation, and 2. To inform the investigator of his/her obligations and obtain the investigator s commitment to follow pertinent FDA regulations.

22 Example #1 (cont) 1572 FORM - FDA STATEMENT OF INVESTIGATOR Information Sheet Guidance for Sponsors, Clinical Investigators and IRBs May 2010

23 Example #1 (cont) 1572 FORM - FDA STATEMENT OF INVESTIGATOR The Problem Guidance document expands the definition of clinical laboratories engaged in research to include Ancillary Service Providers (regardless of whether they are engaged in the conduct of research or not). Ancillary Service Providers Laboratories Cardiology Radiology Interventional Radiology/Surgery Centers, Ophthalmology Note - Facilities could be used a single time for a single patient. Question - How can we qualify these facilities?

24 Example #1 (cont) 1572 FORM - FDA STATEMENT OF INVESTIGATOR Regulatory Conflict - FDA Information Sheets previously stated that institutions whose employees or agents perform commercial or other services for investigators are not engaged in the conduct of a clinical trial if all of the following conditions are met: The services performed do not merit professional recognition or publication privileges; The services performed are typically performed by those institutions for nonresearch purposes; and The institution s employees or agents do not administer any study intervention being tested or evaluated under the protocol.

25 Example #1 (Recommended Action) 1572 FORM - FDA STATEMENT OF INVESTIGATOR Solution - Limit inclusion of Ancillary Service Providers in Box # 4 of FDA 1572 Form to clinical laboratories testing biological specimens collected during the conduct of a clinical trial. Justification for Action: Addition of other Ancillary Service Providers will not make research participation safer for patients or improve data quality. Makes compliance less complicated/costly.

26 Need for Site Collaboration CHANGE THROUGH AGREEMENT Society of Clinical Research Sites Founded in 2012 Advocate Connect Educate Mentor White Paper Better Payment Terms for Sites Change Through Networking Certified Sites Master Clinical Trial Agreements Consistent Budget and Payment Terms Improving Compliance Through Common Order Sets Change Through Networking Infrastructure Supports Just-In-Time

27 Just-In-Time IMPROVE ACCRUAL AND REDUCE COST/WORK Patient Centric Approach to Clinical Research No Pre-Study/Site Qualification Visits Master Clinical Trial Agreement/Budget Central Site (Site Survey, Data Use Agreements, Investigator Background Form, etc.) and Investigator Essential Documents (CV, Medical License, Training Certificates, etc.) Central Infrastructure and CRO/Sponsor Negotiate Essential Document Requirements (e.g., core labs on FDA 1572 form) Routing of Essential Documents When Patient Identified Use of Common Systems (EDC, Core Imaging, Core Laboratory) Workload Reduction on Sites Sponsor Directed to Active/Interested Sites with Patient Population. NOTE: Patient Care Trumps JIT in Some Cases

28 Thank You! CHRIS BEARDMORE CHIEF EXECUTIVE OFFICER 6100 CENTER DRIVE, SUITE 600 LOS ANGELES, CA (424)