The chronic lymphocytic leukemia (CLL) and chronic myeloid leukemia (CML) markets are becoming increasingly crowded, with recent marketing authorizations expanding therapy options for patients. As more therapies are added to the armamentarium for these indications, spiraling healthcare costs mean payers must make increasingly economical reimbursement decisions. In addition, oncologists are faced with increasing challenges because they must balance superior efficacy with the rising cost for patients.

Questions answered:

  • Reimbursement of CML and CLL therapies depends on both clinical and cost-related factors. What is the single most influential factor for formulary placement of CML and CLL therapies? How widespread is the use of pharmacoeconomic models for measuring the cost and efficacy impact of CML and CLL therapies and which cost- effectiveness models do payers favor? How much of an impact do payer strategies have on prescribing of key current CML and CLL therapies?
  • Generic versions of Gleevec have been available for over a year. How does the coverage status of Gleevec brand differ from that of imatinib generics? How aware are the MCO directors of existing copay assistance programs for both generic imatinib and branded Gleevec and what impact do copay programs have on formulary inclusion decisions?
  • Acalabrutinib and ublituximab are emerging CLL therapies that are expected to become available in the US in the next two years. What are the most important attributes that payers and prescribers believe the pharmaceutical companies should focus on when developing new CML and CLL therapies? How will the pricing of emerging therapies impact their tier placement? If both acalabrutinib and ublituximab were both to be placed on a preferred tier, would patient shares of Venclexta and Imbruvica suffer?

Scope:

  • Markets covered: United States
  • Methodology: Surveys of 100 hematologist-oncologists and 30 managed care company officials, including 15 pharmacy directors and 15 medical directors, in March 2017
  • Indication coverage: chronic lymphocytic leukemia and chronic myeloid leukemia
  •  Key drugs covered: Rituxan, Zydelig, Arzerra, Imbruvica, Gazyva, Venclexta, Gleevec, Sprycel, Tasigna, Bosulif, Iclusig, Synribo
  •  Key companies mentioned: Roche/Genentech, Novartis

Table of contents

  • High-Cost Hematological Malignancies - Access & Reimbursement - Detailed, Expanded Analysis Hematological Malignancies (US)
    • Key Updates
      • September 2017
    • Introduction
      • Market Access Overview
    • Actionable Recommendations to Optimize Market Access
      • Successes and Stumbles
        • Overview of Successes & Stumbles for CML and CLL Therapies
        • Access: Physicians
          • Successes Among CML Therapies in the United States: Physicians
          • Physicians: Perceived Best-Performing Therapies for Chronic Phase CML on Market Access Factors
          • Successes Among CLL Therapies in the United States: Physicians
          • Physicians: Perceived Best-Performing Therapies for CLL on Market Access Factors
          • Stumbles Among CML Therapies in the United States: Physicians
          • Stumbles Among CLL Therapies in the United States: Physicians
        • Reimbursement: MCOs
          • Successes Among CML Therapies in the United States: MCOs
          • Stumbles Among CML Therapies in the United States: MCOs
          • Successes Among CLL Therapies in the United States: MCOs
          • Stumbles Among CLL Therapies in the United States: MCOs
      • Key Stakeholders in the Road to Market Access
        • Physicians: Influential Stakeholders in Prescribing for CML and CLL
        • United States: Stakeholder Dynamics in the Road to Reimbursement
        • Healthcare Coverage in the United States
        • Healthcare Coverage in United States
        • Major Commercial Insurers in the United States
      • Key Market Access Roadblocks
        • Reimbursement Dynamics
          • United States: Commercial Managed Care Organizations
            • Payers: Items Needed for P&T Committee Review of CML and CLL Therapies
            • Lessons Learned and Key Takeaways
            • Payers: Coverage Status of Key CML Therapies
            • Payers: Coverage Status of Key CLL Therapies
            • Payers: Expected Coverage Status of CML Therapies in 12 Months' Time
            • Payers: Expected Coverage Status of CLL Therapies in 12 Months' Time
            • OCM Pilot and Implications for Industry
            • Pricing and Reimbursement Drivers
            • Key Findings
            • Payers: Most Influential Factors for Formulary Placement of CML and CLL Therapies
            • Value Assessment and Pharmacoeconomics
            • Payers: Preferred Cost-Effectiveness Models for Emerging CML Therapies
            • Payers: Preferred Cost-Effectiveness Models for Emerging CLL Therapies
            • Contracting for CML and CLL Therapies
            • Payers: Contracting Agreements for CML Therapies
            • Payers: Contracting Agreements for CLL Therapies
            • Cost-Sharing for Key CLL and CML Therapies
            • Copay and Coinsurance Rates for CLL and CML Therapies
            • Restrictions Affecting Treatment of CML and CLL
            • Restrictions Used in Commercial Plans for CML
            • Restrictions Used in Commercial Plans for CLL
            • Impact of Prior Authorization on CML Therapies
            • Impact of Prior Authorization on CLL Therapies
        • Pricing and Reimbursement, Policy, and Coverage: Impact on Prescribing
          • Patient Share in Key Payer Channel
            • Key Findings for High-Cost Therapies for CML and CLL
            • Physicians: Patient Shares of Key CML Therapies by Line of Therapy
            • Physicians: Patient Shares of Key CLL Therapies by Line of Therapy
            • Treatment Decision Drivers
            • Physicians: Factors That Most Strongly Influence Choice of Therapy for CML
            • Physicians: Factors That Most Strongly Influence Choice of Therapy for CLL
          • Prescriber Preference
            • Prescriber Preferences for CML and CLL
            • Physicians: Impact of Payer Strategies on Prescribing of Key CML Therapies
            • Physicians: Impact of Payer Strategies on Prescribing of Key CLL Therapies
          • Impact of Restrictions on Prescribing
            • Impact on Prescribing for CLL and CML
            • Physicians: Market Access Issues That Restrict Prescribing of CLL Therapies
            • Physicians: Reimbursement of Therapies for CLL by Line of Therapy
            • Physicians: Market Access Issues That Restrict Prescribing of Therapies for Chronic Phase CML
            • Physicians: Reimbursement of Therapies for CML by Line of Therapy
        • Market Access Landscape for Emerging Therapies
          • Impact of Emerging Therapies on Prescribing
            • Likely Impact of Emerging Therapies for Second-Line High-Risk CLL
            • Physicians: Anticipation of Performance Superiority for Emerging CLL Therapies
            • Physicians: Anticipated Patient Shares of Current and Emerging Therapies for Second-Line High-Risk CLL
          • Potential Placement of Emerging Therapy at Various Prices
            • Potential Reimbursement of Emerging Therapies for CLL
            • Payers: Ublituximab's Likely Tier Placement Based on Price
            • Payers: Acalabrutinib's Likely Tier Placement Based on Price
          • Prescriber and MCO Priorities for Emerging Therapy Evaluation
            • Key Findings on Prescriber and MCO Priorities for Emerging Therapy Evaluation
            • Payers: Most Compelling Pharmacoeconomic/Health Economic Outcomes Data for a New CML Therapy
            • Payers: Most Compelling Pharmacoeconomic/Health Economic Outcomes Data for a New CLL Therapy
            • Payers: Perception of Most Important Attributes for Development of Emerging Therapies for CML and CLL
            • Physicians: Perception of Most Important Attributes for Development of Emerging Therapies for CML and CLL
        • Methodology
          • Primary Market Research Methodology: Physicians
            • Years in Practice Postresidency
            • Average Number of CML and CLL Patients Treated per Month
            • Distribution of Medical Practice by Region
            • Distribution of Medical Practice by State
            • Insurance Coverage of CML and CLL Patients
            • Agents Prescribed Outside of a Clinical Trial for CLL
            • Agents Prescribed Outside of a Clinical Trial for CML
            • Practice Location Within Community
          • Primary Market Research Methodology: MCOs
            • Distribution of MCO Respondents by Job Title
            • Distribution of MCOs by Region
            • Distribution of MCOs by State
            • Operations of MCOs: National, Regional, or Single-State
            • Types of Health Plans Offered by MCOs
            • Distribution of MCOs by Number of Covered Lives
            • Average Number of Lives Covered by MCOs
        • Appendix
          • Physician Survey
            • Physicians: Awareness of Gleevec and Imatinib Generics' Copayment Assistance Programs and Estimated Percentage of CML Patients Who Benefit
            • Physicians: Perceived Best-Performing Therapies for Chronic Phase CML on Clinical Factors
            • Physicians: Perceived Best-Performing Therapies for CLL on Clinical Factors
            • Physicians: Payer-Imposed Cost Controls That Restrict Prescribing of CML Therapies
            • Physicians: Payer-Imposed Cost Controls That Restrict Prescribing of CLL Therapies
            • Physicians: Patient Shares of Key CML Therapies by Line of Therapy
            • Physicians: Average Duration of Treatment with Key Therapies in the First Line of CML
            • Physicians: Average Duration of Treatment with Key Therapies in the Second and Subsequent Lines of CML
            • Physicians: Percentage of Physicians Who Experience Duration of Treatment Restrictions When Prescribing Key Chronic Phase CML Therapies
            • Physicians: Most Common Duration of Treatment Restrictions Imposed by Payers for Key CML Therapies
            • Physicians: Factors That Most Impact Patient Adherence to Chronic Phase CML Treatment with TKI Therapies
            • Physicians: First-Line Chronic Phase CML Therapies Most Commonly Discontinued by Patients Due to Long Treatment Duration and Associated Costs
            • Physicians: Second- and Subsequent-Line Chronic Phase CML Therapies Most Commonly Discontinued by Patients Due to Long Treatment Duration and Associated Costs
            • Physicians: Willingness to Prescribe Bosulif in First-Line Chronic Phase CML
            • Physicians: Level of Agreement with Statements Regarding TKIs Used in Treatment of CML
            • Physicians: Agreement with Statements Regarding Generic Imatinib
            • Physicians: Agreement with Statements Regarding Copay Assistance Programs for Gleevec and Generic Imatinib
            • Physicians: Most Convincing Reasons to Prescribe a New CML Agent
            • Physicians: Patient Shares of Key CLL Therapies by Line of Therapy
            • Physicians: Treatment Until Disease Progression vs. Fixed Treatment Duration in Relapsed/Refractory CLL
            • Physicians: Reasons for Not Treating Relapsed/Refractory CLL Patients Until Disease Progression or Unacceptable Toxicity
            • Physicians: Percentage of Imbruvica, Zydelig, and Venclexta Prescriptions Denied by Insurers in Relapsed/Refractory CLL
            • Physicians: Main Reasons for Imbruvica, Zydelig, and Venclexta Prescription Denial
            • Physicians: Level of Agreement with Statements Regarding CLL Therapies
            • Physicians: Impact of Biosimilar Rituximab Entry on Choice of Anti-CD20 Therapy
            • Physicians: Reasons to Prescribe Gazyva Over Biosimilar Rituximab
            • Physicians: Factors That Will Restrict Prescribing of Acalabrutinib Monotherapy to High-Risk CLL Patients
            • Physicians: Factors That Will Restrict Prescribing of Ublituximab + Imbruvica to High-Risk CLL Patients
            • Physicians: Level of Agreement with Statements Regarding Future Prescribing Acalabrutinib and Ublituximab for CLL
          • MCO Survey
            • Payers: P&T Committee Members with Greatest Influence on Formulary Decisions for CML and CLL
            • Payers: Interim Status Applied to FDA-Approved Premium-Priced CML and/or CLL Therapies Pending Complete Formulary Review
            • Payers: Time Between CML and/or CLL Therapy Launch and P&T Committee Review and Decision on UM Criteria
            • Payers: Entities Responsible for Formulary Development and Maintenance and for Contracting with Pharmaceutical Manufacturers
            • Payers: Type of PBM Formulary Used by MCO
            • Payers: Degree of Formulary Customization
            • Payers: CLL Therapies Reimbursed as a Pharmacy Benefit Also Covered as a Medical Benefit
            • Payers: CML Therapies Reimbursed as a Pharmacy Benefit Also Covered as a Medical Benefit
            • Payers: Reasons CLL Therapies Are Not Reimbursed
            • Payers: Reasons CML Therapies Are Not Reimbursed
            • Payers: Most Common Coinsurance for CLL Therapies Covered as a Pharmacy Benefit
            • Payers: Most Common Copayment for CLL Therapies Covered as a Pharmacy Benefit
            • Payers: Most Common Coinsurance for CML Therapies Covered as a Pharmacy Benefit
            • Payers: Most Common Copayment for CML Therapies Covered as a Pharmacy Benefit
            • Payers: Cost-Share Separate from Office Visit Charge for Key CLL Therapies Covered Under Medical Benefit
            • Payers: Cost-Share Separate from Office Visit Charge for Key CML Therapies Covered Under Medical Benefit
            • Payers: Strategies Used to Manage Utilization and/or Cost of Key CLL Therapies
            • Payers: Strategies Used to Manage Utilization and/or Cost of Key CML Therapies
            • Payers: Prior Authorization Restrictions for Key CLL Therapies
            • Payers: Prior Authorization Restrictions for Key CML Therapies
            • Payers: Number of Steps Required Before Receiving CLL Agents Subject to Step Therapy Control
            • Payers: Number of Steps Required Before Receiving CML Agents Subject to Step Therapy Control
            • Payers: Distribution Channels for Branded CLL Therapies
            • Payers: Distribution Channels for Branded CML Therapies
            • Payers: Agreement with Statements Regarding Branded CML Therapies
            • Payers: Agreement with Statements Regarding Branded CLL Therapies
            • Payers: Perceived Best-Performing Therapies for Chronic Phase CML
            • Payers: Perceived Best-Performing CLL Therapies
            • Payers: Duration Limits for First- and Subsequent-Line CML Therapies Subject to Duration Restrictions
            • Payers: Duration Limits for Second- and Subsequent-Line CLL Therapies Subject to Duration Restrictions
            • Payers: MCO Participation in Clinical Pathway Programs for CML and CLL
            • Payers: CLL Therapies Included in Clinical Pathways Program
            • Payers: CML Therapies Included in Clinical Pathways Program
            • Payers: Mean Targeted Compliance for CML and CLL Clinical Pathways
            • Payers: Incentives for Reaching Targeted Compliance Rate in 12 Months' Time
            • Payers: Controls to Minimize Branded Gleevec Prescribing
            • Payers: Awareness of Copayment Assistance Programs for Gleevec and Imatinib Generics
            • Payers: Likelihood of Gleevec and Imatinib Generics' Formulary Inclusion Based on Copay Assistance Program Availability
            • Payers: Emerging Therapy for High-Risk CLL with Greatest Anticipated Impact
            • Payers: Potential Formulary Inclusion of Biosimilar Rituximab
            • Payers: Strategies to Encourage Use of Biosimilar Rituximab
            • Payers: Measures to Control Rising Cancer Treatment Costs
            • Payers: Companies to Submit Pharmacoeconomic/Health Economic Outcomes Data for CLL Therapies
            • Payers: Companies to Submit Pharmacoeconomic/Health Economic Outcomes Data for CML Therapies
            • Payers: Quality of Pharmacoeconomic/Health Economic Outcomes Data Submitted for CLL Therapies
            • Payers: Quality of Pharmacoeconomic/Health Economic Outcomes Data Submitted for CML Therapies
            • Payers: Impact of Pharmacoeconomic/Health Economic Outcomes Data on Reimbursement of CLL Therapies
            • Payers: Impact of Pharmacoeconomic/Health Economic Outcomes Data on Reimbursement of CML Therapies

      Author(s): Niamh Buckley

      Niamh Buckley Ph.D. is a senior director in the oncology team at Decision Resources Group where she manages a team of analysts in producing syndicated and custom primary and secondary market research on a wide range of oncology indications. She also provides sales and client support for all oncology products.

      Dr. Buckley has extensive experience in market access and forecasting across oncology and hematology-oncology drug markets She also has considerable experience in conducting primary research with physicians and payers throughout the major pharmaceutical markets. Prior to joining Decision Resources, Dr. Buckley worked as a postdoctoral scientist focusing on translational breast cancer research. She has a B.Sc. in biotechnology and obtained her Ph.D. in molecular oncology from Trinity College in Ireland.