2011_个体化用药_基因检测_-_医保政策(现实障碍和解决之道).pdf

Reimbursement for Personalized Medicine Diagnostics: Current Limitations and Evolving Strategies Kristin Pothier, Partner Gary Gustavsen, Manager Health Advances, LLC 9 Riverside Road Weston, Massachusetts 02493 USA tel: +1 781.647.3435 fax: +1 781.392.1484 www.healthadvances.com CONFIDENTIAL June 27, 2011 Reimbursement Workshop CONFIDENTIAL June 27, 2011 1 Agenda 8:30-8:45 Introductions 8:45-9:15 Health Advances Personalized Medicine Overview 9:15-10:15 Reimbursement Landscape Overview 10:15-11:15 Breakout Sessions 11:15-11:30 Summary and Wrap-up Reimbursement Workshop CONFIDENTIAL June 27, 2011 2 Agenda Health Advances Introduction Reimbursement Landscape Limitations Real-World Impact Proposed Solutions Breakout Sessions Summary and Wrap-up Reimbursement Workshop CONFIDENTIAL June 27, 2011 3 Strategy Consulting for Industry Leaders: Diagnostics and Life Sciences Practice Turn technological insight, clinical understanding, market knowledge and competitive intelligence into compelling commercial and clinical development strategies Efficiently gather market data from providers, patients and payers employing a large, established, international network of experts to provide a robust fact base Management team of five partners and five vice presidents, each with their own expertise and practice areas Leverage 65 professionals in Boston and San Francisco to detect patterns, evaluate strategic trade-offs, and present logical alternatives Integrate therapeutics, devices and diagnostics into a comprehensive industry perspective Build consensus among management teams for recommended actions Market assessment Product strategy Portfolio prioritization Business and corporate development Pricing and reimbursement Diligence and M Heightens Company's Focus on Tests for Cancer Drug Personalization Life Technologies and Ignite Institute Partner to Create Largest NGS Facility in North America SureGene and Medco Enter Collaboration to Evaluate the Ability of Genetic Biomarkers to Enhance Drug Selection for Serious Mental Health Illnesses. bioMerieux and GSK Sign Theranostics Agreement to Develop Predictive Test for Selecting Breast Cancer Treatment Nestle Buys Prometheus Laboratories to Expand in Health On-Q-ity Announces Strategic Alliance with LabCorp Reimbursement Workshop CONFIDENTIAL June 27, 2011 9 A Personalized Approach Personalized Medicine The right treatment for the right patient at the right time Factors Driving Need for Personalized Medicine Aging population More complex diseases Desire to control costs Desire to provide better treatment with fewer side effects Source: Health Advances analysis. Reimbursement Workshop CONFIDENTIAL June 27, 2011 10 0100,000200,000 Non-Hodgkin Lymphoma Melanoma Urinary Bladder Colorectal Breast Prostate Lung and Bronchus Number of Patients per Year 2010 US Incidence 2010 US Deaths Continued Unmet Needs in Cancer Care The investment in cancer biomarkers and drug development has only begun to address this family of diseases. 2010 US Cancer Incidence and Deaths Highest Incidence Source: Health Advances analysis, American Cancer Society Cancer Facts and Figures, SEER Cancer Statistics. 0% 25% 50% 75% 100% 1975198519952005 5 Year Survival Rate Year Prostate Melanoma Breast Colorectal Ovary Stomach Esophagus Lung Liver Pancreas US Cancer 5 Year Survival Rates Progression Over Time Reimbursement Workshop CONFIDENTIAL June 27, 2011 11 DX and TX Paradigms Becoming More Complex Observation Stage IV/ Metastatic Breast Cancer Chemo +/- Targeted Therapy Progression Herceptin +/or Tykerb +/or Chemo Recurrence Neoadjuvant Stage I,II and III Surgical Resection Hormonal treatment alone or in combo PI3K inhibitors +/or Akt inhibitors MET inhibitors IGF-1 inhibitors +/or +/or +/or Herceptin Tykerb Avastin* +/or +/or HER3 inhibitors VEGF TKIs EGFR inhibitors +/or +/or +/or PARP inhibitors mTOR inhibitors +/or +/or HSP inhibitors HDAC inhibitors +/or +/or * On December 16, 2010 the FDA notified Genentech of its Proposal to Withdraw Marketing Approval of Avastin in breast cancer. Note: Graphic depicts current approved targeted agents plus Phase III and Phase II agents. Source: Health Advances interviews and analysis, NCCN, PharmaProjects. As the number of targeted therapies continues to grow, physicians will need biomarkers and methods of measuring these biomarkers (i.e. diagnostics) to guide effective treatment. Reimbursement Workshop CONFIDENTIAL June 27, 2011 12 Oncology Clinical Development Pipeline PM Drivers: Biomarkers in Oncology The increase in complex targeted drugs in the pipeline is driving the need for novel biomarkers aimed at identifying patients expressing a given target. Note: Targeted therapies are defined as affecting specific molecular targets such as EGFR, HER-2, BRAF, etc. Non-targeted therapies include traditional chemotherapeutic agents, hormonal therapies, cell based therapies, non-specific anti-apoptotic therapies, and general immunomodulatory therapies. Source: Health Advances analysis, Pharmaprojects. Complex signaling mechanisms Simple targeted therapies Co-developed combinations Single agents Widespread advanced biomarker use Moderate biomarker use Future Today 0% 25% 50% 75% 100% LaunchedPhase IIIPhase IIPhase I Percent of Compounds Non-Targeted Targeted 14% 36% 37% 45% Reimbursement Workshop CONFIDENTIAL June 27, 2011 13 Crizotinib With only three years separating Phase I and Phase III initiation, the development of crizotinib highlights the importance of incorporating molecular profiling within early- phase trials and the role of partnering for commercialization. Source: Windhover SIS, company press releases, ASCO abstracts, Health Advances analysis. Crizotinib First Synthesized PF-02341066 first synthesized as a small molecule inhibitor of cMET and ALK Phase I Initiated Originally focused on tumors with cMET activation Phase I Recruitment Expansion After the key NSCLC report during the dose escalation phase, lung cancer patients with proven ALK gene rearranged tumors were also recruited Impressive Phase I Data at ASCO In 76 heavily pretreated ALK+ NSCLC patients 64% objective response rate Companion Dx Deal with Abbott Deal with Abbott to develop FISH-based companion Dx to detect ALK gene rearrangements ALK in NSCLC Reported Report of ALK gene rearrangements occurring in NSCLC Phase III Initiated 318 ALK+ NSCLC patients Pfizer, Abbott Plan Filing Pfizer, Abbott plan parallel filing for FDA approval of crizotinib and FISH test 2006 2009 2007 2005 2010 2008 2011 Reimbursement Workshop CONFIDENTIAL June 27, 2011 14 Personalized Medicine Applications: Oncology Personalized medicine has impacted the management of oncology across the continuum of care. Source: Health Advances analysis. Description Diagnostic tests to complement traditional risk factors Applied to high-risk patients to identify disease early Used for definitive diagnosis and general cancer typing Assess severity and/or risk of recurrence Inform adjuvant chemo decision Used to predict efficacy or safety response to specific treatments Recurrence monitoring Monitoring for treatment efficacy Test Examples BRACAnalysis® COLARIS® MELARIS® deCODE Prostate Cancer PreGen-Plus EarlyCDT- Lung CancerTYPE ID® C-kit OVA1 PathFinderTG® miRview Oncotype DX® Breast and Colon Previstage MammaPrint Prezeon Adjuvant! Online HER2/neu TheraScreen® K-RAS and EGFR Mutation Kits ResponseDX VeriStrat® OnDose ChemoFx® CellSearch BCR/ABL Monitoring Therapy Selection Staging and Prognosis Diagnosis Screening Risk Assessment Novel Diagnostics Applications: Cancer Reimbursement Workshop CONFIDENTIAL June 27, 2011 15 Personalized Medicine Catalysts Personalized medicine has transformed oncology due to a convergence of several catalysts. These same catalysts suggest the next frontiers for PM. Cancer Personalized Medicine Established cancer biomarkers Clear Genetic Basis High consequences to patient of over/under treatment Consequence of Treatment Urgency to switch to optimal treatment to prevent growth and metastasis Treatment Switching Urgency Emerging subsets of disease that respond differently to therapeutics Disease Heterogeneity Paradigm of treating with targeted agents, potentially in combination Targeted Therapy Cannot afford to use ineffective treatments with high priced therapies High Priced Therapies Personalized Medicine In Other Indications Infectious disease Cardiovascular CNS Autoimmune Source: Health Advances analysis. Reimbursement Workshop CONFIDENTIAL June 27, 2011 16 Personalized Medicine Development Personalized Medicine Strategy Research and Development Regulatory Pricing and Reimbursement Business Strategy Sales and Marketing Regardless of the therapeutic area, PM diagnostics face a series of commercialization challenges. Todays workshop will focus on pricing and reimbursement. Source: Health Advances Reimbursement Workshop CONFIDENTIAL June 27, 2011 17 Represented Today In This Workshop Reimbursement Workshop CONFIDENTIAL June 27, 2011 18 Agenda Health Advances Introduction Reimbursement Landscape Limitations Real-World Impact Proposed Solutions Breakout Session Summary and Wrap-up Reimbursement Workshop CONFIDENTIAL June 27, 2011 19 Limitations of the Current Reimbursement System Health Advances and Reimbursement Reform Potential Reform Solutions and Next Steps Health Advances released an important report through BIO in 2011 which details the evolving reimbursement landscape for personalized medicine diagnostics in the US. Current System Impact on Personalized Medicine Reimbursement Workshop CONFIDENTIAL June 27, 2011 20 Review published literature In-depth analysis of historical product reimbursement experience Debrief with BIO Health Advances Project Methodology Primary interviews Focus on payers, coders, industry executives, advocacy Secondary Research Primary Research Windhovers database analysis for deal research Company-specific financial documents Primary interviews Focus on industry executives, investors, physicians, payers Synthesize proposed solutions from all stages of project research to evaluate strengths and weaknesses Primary interviews Reengagement of all stakeholders for solutions discussions Update and Working Session with BIO Update and Working Session with BIO Update and Working Session with BIO Write White Paper Project Methodology Source: The Reimbursement Landscape for Novel Diagnostics; Health Advances and BIO 2011. Reimbursement Workshop CONFIDENTIAL June 27, 2011 21 Primary Interview Program Key Decision Maker Example Organizations (not a complete list) Private Payers and Coders Sr. level medical directors, and coverage decision-makers in small/large US plans Sr. level coders in US institutions that frequently code novel diagnostics Blue Cross Blue Shield MagnaCare Medco NHIC (CMS contractor) Predictive Health CMS/ Government Sr. level diagnostics coverage decision-makers recently at CMS Sr. level representatives from government agencies Physicians Key opinion leader physicians with experience in personalized medicine Physician leaders involved in developing practice guidelines Brigham Health Advances and BIO 2011. Reimbursement Workshop CONFIDENTIAL June 27, 2011 22 Agenda Health Advances Introduction Reimbursement Landscape Limitations Real-World Impact Proposed Solutions Breakout Session Wrap-up and Next Session Reimbursement Workshop CONFIDENTIAL June 27, 2011 23 Innovative Test Examples FDA Cleared Positive Coverage Policies Aetna Regional CMS Cigna Regional BCBS AlloMap Yes Oncotype DX (breast cancer) No MammaPrint Yes Pathworks CUP Yes BRACAnalysis No OVA1 Yes KRAS (colorectal cancer) No Coverage Inconsistencies Variations in decision processes across payers result in inconsistent coverage determinations. Coverage decisions are made at the payers discretion and are typically reviewed annually. Coverage Inconsistencies for Example Novel Diagnostics As of 5/18/10 Denotes Positive Coverage Policy Limitations Source: The Reimbursement Landscape for Novel Diagnostics; Health Advances and BIO 2011. Reimbursement Workshop CONFIDENTIAL June 27, 2011 24 Coding Puzzles: Oncotype DX Genomic Health used a miscellaneous code to achieve value-based pricing for its test. Test Example: Oncotype Dx Code Stacking New Code Miscellaneous Code Molecular diagnostics RNA extraction Reverse transcription Gene amplification Interpretation and report CPT Codes 83891 83902 83898/901 83912 Rate $5.85 $20.72 $24.47 $5.85 Stack x1 x1 x21 x1 CPT Codes XXXXX Rate $XX CPT Codes 84999 Rate $3450 Total $546.29 Total $XX Total $3450 Pros Precedent set already Legitimate formal coding option Possibility of higher payment Cons Potentially lower reimbursement Viability unpredictable in long run Can be penalized if audited Long process and timeframe Reimbursement may be limited initially No guarantee of payer to agree to higher payment Risk of obtaining a lower category code with investigational connotation Long process and timeframe Reimbursement may be limited initially No guarantee of payer to agree to higher payment Result for Genomic Health $3450+/test through miscellaneous code option Source: The Reimbursement Landscape for Novel Diagnostics; Health Advances and BIO 2011. Reimbursement Workshop CONFIDENTIAL June 27, 2011 25 Coding Puzzles: OVA1 Vermillion also used a miscellaneous code to achieve value-based pricing for its test. Test Example: OVA1 Code Stacking New Code Miscellaneous Code Immunoassays Transthyretin Apolipoprotein A-1 Beta-2 Microglobulin Transferrin CA 125 Interpretation and report CPT Codes 82784 82172 82232 84466 86304 83912 Rate $13.32 $22.19 $23.17 $18.29 $29.81 $5.85 Stack x1 x1 x1 x1 x1 x1 CPT Codes XXXXX Rate $XX CPT Codes 84999 Rate $650 Total $112.63 Total $XX Total $650 Result for Vermillion $650/test through miscellaneous code option Payers express frustrations over the growing trend of miscellaneous codes The miscellaneous codes certainly make our lives more difficult from a payment and tracking perspective. I dont think this model is sustainable indefinitely. Small private payer They are going to have to set up a new coding system for PM tests soon otherwise all these tests will be miscellaneous codes and it will be a very chaotic. Coding expert Limitations Source: The Reimbursement Landscape for Novel Diagnostics; Health Advances and BIO 2011. Reimbursement Workshop CONFIDENTIAL June 27, 2011 26 Economic Arguments: Oncotype Dx Genomic Health wisely dedicated resources to developing and publishing a rigorous economic argument for Oncotype DX. Total Adjuvant Treatment Costs Chemotherapy Administration Complications x x = Costs