The success of an anticancer therapy is not only dependent on the outcome of its pivotal trial, but also on that trial’s design, which is examined by the regulatory bodies, payers, and prescribers. In our analysis, we assess the influence of trial design on treatment and reimbursement decisions regarding current and emerging therapies for glioblastoma, prostate cancer, and squamous cell carcinoma of the head and neck (SCCHN). In addition, we explore payers’ and prescribers’ cost-sensitivity and preference for trial design features, such as primary end points, comparators, and enrollment size.

Table of contents

  • Clinical Trial Design - Access & Reimbursement - Detailed, Expanded Analysis (US): Glioblastoma, Prostate Cancer, Squamous Cell Carcinoma Of The Head And Neck
    • Actionable Recommendations to Optimize Market Access
      • Successes and Stumbles
        • Successes Among Glioblastoma, Prostate Cancer, and SCCHN Therapies in the United States
        • Stumbles Among Glioblastoma, Prostate Cancer, and SCCHN Therapies in the United States
        • United States: Reimbursement Successes and Stumbles
        • Key Stakeholders in the Road to Market Access
          • Key Market Access Roadblocks
            • Reimbursement Dynamics
              • United States- Commercial Managed Care Organizations
                • Number of Voting Members in MCOs' P&T Committee
                • Next Planned Meeting of P&T Committee for Review of Prostate Cancer, Glioblastoma, and SCCHN Therapies
                • Positions of Greatest Influence on Formulary Inclusion of Prostate Cancer, Glioblastoma, and SCCHN Therapies
                • Items Needed for Inclusion in P&T Committee Drug Review
                • Lessons Learned and Key Takeaways
                • Current Coverage Status of Branded Therapies for Prostate Cancer, Glioblastoma, and SCCHN
                • Impact of Clinical Trial Design on Formulary Inclusion Decisions for Prostate Cancer, Glioblastoma, and SCCHN Therapies
                • Proportion of Payers Examining Clinical Trial Design of FDA-Approved Therapies
                • Drivers of Reimbursement by MCO for Prostate Cancer, Glioblastoma, and SCCHN: Clinical Trial Design
                • Drivers of Reimbursement by MCO for Prostate Cancer, Glioblastoma, and SCCHN: Features of Pivotal Clinical Trials
                • Importance of Active Comparator in Pivotal Clinical Trials to Formulary Inclusion of Prostate Cancer Therapies
                • Importance of Active Comparator in Prostate Cancer Clinical Trials: Payers
                • Importance of Active Comparator in Pivotal Clinical Trials to Formulary Inclusion of Glioblastoma Therapies
                • Importance of Active Comparator in Glioblastoma Clinical Trials: Payers
                • Importance of Active Comparator in Pivotal Clinical Trials to Formulary Inclusion of SCCHN Therapies
                • Importance of Active Comparator in SCCHN Clinical Trials: Payers
                • Key Findings on Pricing and Reimbursement Drivers
                • Restrictions Used in Commercial Plans for Avastin in Treatment of Glioblastoma
                • Restrictions and Prior Authorizations in SCCHN
                • Restrictions Used in Commercial Plans for Glioblastoma and SCCHN
                • Restrictions and Prior Authorizations in Prostate Cancer
                • Restrictions Used in Commercial Plans for Branded Prostate Cancer Therapies
                • Restrictions Used in Commercial Plans for Prostate Cancer
                • Key Restrictions
            • Pricing and Reimbursement, Policy, and Coverage: Impact on Prescribing
              • Patient Share in Key Payer Channel
                • Patient Share of Prostate Cancer Therapies in Commercial Payer Channel
                • Most-Prescribed Agents for First- and Second-Line Metastatic Castrate-Resistant Prostate Cancer
                • Patient Share of Avastin in Glioblastoma in Commercial Payer Channel
                • Prescriber Views on Avastin as Glioblastoma Therapy
                • Current Avastin Patient Shares in Glioblastoma
                • Patient Share of Erbitux in SCCHN in Commercial Payer Channel
                • Current Erbitux Patient Shares in SCCHN
                • Key Findings
                • Top Prescribing Drivers for Prostate Cancer
                • Top Prescribing Drivers in Prostate Cancer
                • Top Prescribing Drivers for Glioblastoma
                • Top Prescribing Drivers in Glioblastoma
                • Top Prescribing Drivers for SCCHN
                • Top Prescribing Drivers in SCCHN
              • Prescriber Preference
                • Prescriber Preferences for Prostate Cancer
                • Most-Preferred Agents for First- and Second-Line Metastatic Castrate-Resistant Prostate Cancer
                • Prescriber Preferences for Glioblastoma
                • Preferred Avastin Patient Shares in Glioblastoma
                • Prescriber Preferences for SCCHN
                • Prescriber Access to Erbitux in SCCHN
                • Preferred Erbitux Patient Shares in SCCHN
              • Impact of Payer Policy on Prescribing
                • Key Findings
                • Impact of Payer Policy on Prescribing for Glioblastoma
                • Hospital-Based Oncologist-Reported Most-Important Restrictions for Glioblastoma
                • Office-Based Oncologist-Reported Most-Important Restrictions for Glioblastoma
                • Impact of Payer Policy on Prescribing for Prostate Cancer
                • Hospital-Based Oncologist-Reported Most-Important Restrictions for Prostate Cancer
                • Office-Based Oncologist-Reported Most-Important Restrictions for Prostate Cancer
                • Impact of Payer Policy on Prescribing for SCCHN
                • Office- and Hospital-Based Oncologist-Reported Most-Important Restrictions for SCCHN
                • Impact of Head-to-Head versus Single-Arm Trial Design in Different Drug-Pricing Scenarios
            • Market Access Landscape for Emerging Therapies
              • Likely Impact of Emerging Therapy
                • Likely Impact of Emerging Therapies for Prostate Cancer
                • Likely Impact of Tier Placement on Apalutamide (JNJ-927) Patient Share in Prostate Cancer
                • Likely Impact of Tier Placement on ODM-201 Patient Share in Prostate Cancer
                • Likely Impact of Emerging Therapy for Glioblastoma
                • Likely Impact of Tier Placement on DCVax-L Patient Share in Glioblastoma
                • Likely Impact of Emerging Combination Therapy for SCCHN
                • Likely Impact of Tier Placement on Durvalumab and Tremelimumab Combination Patient Share in SCCHN
              • Potential Placement of Emerging Therapy at Various Prices
                • Potential Placement of Emerging Therapies for Prostate Cancer
                • Potential Placement of Apalutamide in Prostate Cancer at Various Price Points
                • Potential Placement of ODM-201 in Prostate Cancer at Various Price Points
                • Potential Placement of an Emerging Therapy for Glioblastoma
                • Potential Placement of DCVax-L in Glioblastoma at Various Price Points
                • Potential Placement of an Emerging Therapy for SCCHN
                • Potential Placement of Durvalumab and Tremelimumab Combination in SCCHN at Various Price Points
              • Clinical Trial Enrollment
                • Key Findings
                • Percentage of Prostate Cancer, Glioblastoma, and SCCHN Patients Enrolled in Clinical Trials
                • Top Limiting Factors for Clinical Trial Enrollment
                • Limiting Factors for Clinical Trial Enrollment
                • Top Limiting Factors for Biosimilar Clinical Trial Enrollment
                • Percentage of Prescribers Who Have Enrolled Patients in Clinical Trials of Biosimilars
                • Limiting Factors for Biosimilar Clinical Trial Enrollment
              • Biosimilar Indication Extrapolation
                • Prescriber Preferences for Biosimilars' Indication Extrapolation
                • Prescriber Preferences for Biosimilars' Indication Extrapolation
              • Prescriber and MCO Opinion on Remaining Unmet Need
                • Key Findings
                • Payer Preference on Pharma R&D Spending for a Novel Treatment for Glioblastoma
                • Prescriber Preference on Pharma R&D Spending for a Novel Treatment for Glioblastoma
            • Appendix
              • Physician Survey
                • Percentage of Prostate Cancer, Glioblastoma, and SCCHN Patients Enrolled in Clinical Trials
                • Limiting Factors for Clinical Trial Enrollment
                • Reasons for Patient Refusal to Participate in Clinical Trials
                • Payers of Clinical Trial Expenses
                • Prescribers' Attitude Toward Details of Clinical Trials
                • Drivers of Reimbursement: Clinical Trial Design
                • Drivers of Reimbursement: Features of Pivotal Clinical Trials
                • Level of Concern Over Surrogate End Points
                • Prescriber Views on Regulatory Approaches
                • Effect of Cost and Trial Design on Prescribing
                • Percentage of Prescribers Who Have Enrolled Patients in Clinical Trials of Biosimilars
                • Limiting Factors for Biosimilar Clinical Trial Enrollment
                • Prescriber Preferences for Biosimilars' Indication Extrapolation
                • Minimal Acceptable Clinical Trial Phase for Regulatory Approval in Prostate Cancer
                • Minimal Acceptable Primary End Point for Regulatory Approval in Prostate Cancer
                • Minimal Acceptable Comparator for Regulatory Approval in Prostate Cancer
                • Minimal Acceptable Number of Months' Improvement for Regulatory Approval in Prostate Cancer
                • Minimal Acceptable Improvement in Response Rate for Regulatory Approval in Prostate Cancer
                • Minimal Acceptable Clinical Trial Phase for Regulatory Approval in Glioblastoma
                • Minimal Acceptable Primary End Point for Regulatory Approval in Glioblastoma
                • Minimal Acceptable Comparator for Regulatory Approval in Glioblastoma
                • Minimal Acceptable Number of Months' Improvement for Regulatory Approval in Glioblastoma
                • Minimal Acceptable Improvement in Response Rate for Regulatory Approval in Glioblastoma
                • Minimal Acceptable Clinical Trial Phase for Regulatory Approval in SCCHN
                • Minimal Acceptable Primary End Point for Regulatory Approval in SCCHN
                • Minimal Acceptable Comparator for Regulatory Approval in SCCHN
                • Minimal Acceptable Number of Months' Improvement for Regulatory Approval in SCCHN
                • Minimal Acceptable Improvement in Response Rate for Regulatory Approval in SCCHN
                • Importance of Active Comparator in Prostate Cancer Clinical Trials: Prescribers
                • Importance of Active Comparator in Glioblastoma Clinical Trials: Prescribers
                • Importance of Active Comparator in SCCHN Clinical Trials: Prescribers
                • Reasons Behind Late-Phase Glioblastoma Failures
                • Prescriber Views on Avastin as Glioblastoma Therapy
                • Current Avastin Patient Shares in Glioblastoma
                • Preferred Avastin Patient Shares in Glioblastoma
                • Hospital-Based Oncologist-Reported Most-Important Restrictions for Glioblastoma
                • Office-Based Oncologist-Reported Most-Important Restrictions for Glioblastoma
                • Preferred Timing of Investigational Immunotherapy Treatment in Glioblastoma
                • Prescriber Use of Optune Device
                • Reasons for Not Using Optune Device
                • Prescriber Views on Use of Placebo in Xofigo and Zytiga Pivotal Trials: Hospital-Based Oncologists
                • Prescriber Views on Use of Placebo in Xofigo and Zytiga Pivotal Trials: Office-Based Oncologists
                • Effect of Reimbursement on Prescribing in Prostate Cancer
                • Most-Prescribed Agents for First- and Second-Line Metastatic Castrate-Resistant Prostate Cancer
                • Most-Preferred Agents for First- and Second-Line Metastatic Castrate-Resistant Prostate Cancer
                • Hospital-Based Oncologist-Reported Most-Important Restrictions for Prostate Cancer
                • Office-Based Oncologist-Reported Most-Important Restrictions for Prostate Cancer
                • Top Prescribing Drivers in Prostate Cancer
                • Top Prescribing Drivers in Glioblastoma
                • Top Prescribing Drivers in SCCHN
                • Effect of Reimbursement on Prescribing in SCCHN
                • Current Erbitux Patient Shares in SCCHN
                • Preferred Erbitux Patient Shares in SCCHN
                • Office- and Hospital-Based Oncologist-Reported Most-Important Restrictions for SCCHN
                • Prescriber Level of Satisfaction with Apalutamide Pivotal Trial Design
                • Features with Potential for Improvement in Apalutamide Pivotal Trial Design
                • Preferred Comparator for Apalutamide Pivotal Trial
                • Preferred Primary End Point for Apalutamide Pivotal Trial
                • Preferred Number of Patients in Apalutamide Pivotal Trial
                • Likely Impact of Tier Placement on Apalutamide Patient Share in Prostate Cancer
                • Prescriber Level of Satisfaction with ODM-201 Pivotal Trial Design
                • Features with Potential for Improvement in ODM-201 Pivotal Trial Design
                • Preferred Comparator for ODM-201 Pivotal Trial
                • Preferred Primary End Point for ODM-201 Pivotal Trial
                • Preferred Number of Patients in ODM-201 Pivotal Trial
                • Likely Impact of Tier Placement on ODM-201 Patient Share in Prostate Cancer
                • Prescriber Level of Satisfaction with DCVax-L Pivotal Trial Design
                • Features with Potential for Improvement in DCVax-L Pivotal Trial Design
                • Preferred Comparator for DCVax-L Pivotal Trial
                • Preferred Primary End Point for DCVax-L Pivotal Trial
                • Preferred Number of Patients in DCVax-L Pivotal Trial
                • Likely Impact of Tier Placement on DCVax-L Patient Share in Glioblastoma
                • Prescriber Level of Satisfaction with Durvalumab and Tremelimumab Combination Pivotal Trial Design
                • Features with Potential for Improvement in Durvalumab and Tremelimumab Combination Pivotal Trial Design
                • Preferred Comparator for Durvalumab and Tremelimumab Combination Pivotal Trial
                • Preferred Primary End Point for Durvalumab and Tremelimumab Combination Pivotal Trial
                • Preferred Number of Patients in Durvalumab and Tremelimumab Combination Pivotal Trial
                • Likely Impact of Tier Placement on Durvalumab and Tremelimumab Combination Patient Share in SCCHN
                • Prescriber Preference on Pharma R&D Spending for a Novel Treatment for Glioblastoma
              • MCO Survey
                • Percentage of Beneficiaries with Prostate Cancer, Glioblastoma, and SCCHN Enrolled in Clinical Trials
                • Number of Voting Members in MCOs' P&T Committee
                • Next Planned Meeting of P&T Committee for Review of Prostate Cancer, Glioblastoma, and SCCHN Therapies
                • Positions of Greatest Influence on Formulary Inclusion of Prostate Cancer, Glioblastoma, and SCCHN Therapies
                • Items Needed for Inclusion in P&T Committee Drug Review
                • Clinical Trial Expenses Reimbursed by Payers
                • Proportion of Payers Examining Clinical Trial Design of FDA-Approved Therapies
                • Drivers of Reimbursement by MCO for Prostate Cancer, Glioblastoma, and SCCHN: Clinical Trial Design
                • Drivers of Reimbursement by MCO for Prostate Cancer, Glioblastoma, and SCCHN: Features of Pivotal Clinical Trials
                • Importance of Active Comparator in Prostate Cancer Clinical Trials: Payers
                • Importance of Active Comparator in Glioblastoma Clinical Trials: Payers
                • Importance of Active Comparator in SCCHN Clinical Trials: Payers
                • Current Coverage Status of Branded Therapies for Prostate Cancer, Glioblastoma, and SCCHN
                • Preferred Comparators for Clinical Trials in Prostate Cancer
                • Preferred Comparators for Clinical Trials in Glioblastoma
                • Preferred Comparators for Clinical Trials in SCCHN
                • Future Coverage Status of Branded Therapies for Prostate Cancer, Glioblastoma, and SCCHN
                • Pharmacy and Medical Benefit for Currently Reimbursed Therapies
                • Tier Placement of Branded Therapies for Prostate Cancer, Glioblastoma, and SCCHN
                • Payer Level of Satisfaction with Apalutamide Pivotal Trial Design
                • Features with Potential for Improvement in Apalutamide Pivotal Trial Design: Payers
                • Preferred Comparators for Apalutamide Pivotal Trials: Payers
                • Preferred Primary End Points for Apalutamide Pivotal Trials: Payers
                • Preferred Number of Patients in Apalutamide Pivotal Trials: Payers
                • Potential Placement of Apalutamide in Prostate Cancer at Various Price Points
                • Payer Level of Satisfaction with ODM-201 Pivotal Trial Design
                • Features with Potential for Improvement in ODM-201 Pivotal Trial Design: Payers
                • Preferred Comparator for ODM-201 Pivotal Trial: Payers
                • Preferred Primary End Point for ODM-201 Pivotal Trial: Payers
                • Potential Placement of ODM-201 in Prostate Cancer at Various Price Points
                • Payer Level of Satisfaction with DCVax-L Pivotal Trial Design
                • Features with Potential for Improvement in DCVax-L Pivotal Trial Design: Payers
                • Preferred Comparator for DCVax-L Pivotal Trial: Payers
                • Preferred Primary End Point for DCVax-L: Payers
                • Preferred Number of Patients in DCVax-L Pivotal Trial: Payers
                • Potential Placement of DCVax-L in Glioblastoma at Various Price Points
                • Payer Level of Satisfaction with Durvalumab and Tremelimumab Combination Pivotal Trial Design
                • Features with Potential for Improvement in Durvalumab and Tremelimumab Combination Pivotal Trial Design: Payers
                • Preferred Comparator for Durvalumab and Tremelimumab Combination Pivotal Trials: Payers
                • Preferred Primary End Points for Durvalumab and Tremelimumab Combination Pivotal Trials: Payers
                • Preferred Number of Patients in Durvalumab and Tremelimumab Combination Pivotal Trials: Payers
                • Potential Placement of Durvalumab + Tremelimumab in SCCHN at Various Price Points
                • Reimbursement of Optune Device for Glioblastoma
                • Restrictions Used in Commercial Plans for Avastin in Treatment of Glioblastoma
                • Restrictions Used in Commercial Plans for Branded Prostate Cancer Therapies
                • Payer Preference on Pharma R&D Spending for a Novel Treatment for Glioblastoma

          Author(s): Natalia Reoutova, MSc, MA

          Natalia Reoutova, M.A., M.Sc., is a senior business insights analyst in the oncology division at Decision Resources Group. Ms. Reoutova has worked on a range of oncology indications including prostate cancer and malignant melanoma. She has authored reports on non-small-cell lung cancer and colorectal cancer, and worked on publications covering both the major and emerging pharmaceutical markets.

          Natalia Reoutova holds a M.A. in natural sciences (specializing in pharmacology) from the University of Cambridge and a M.Sc. in drug discovery from the University of London. Her postgraduate research focused on developing small-molecule inhibitors of oncogenic transcription factors. Prior to working at Decision Resources, she was responsible for planning and estimating the cost of clinical trials at Pharm-Olam International.


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