Role of HEOR in Accelerated Drug Approvals

Health Economics and Outcomes Research (HEOR) plays a crucial role in ensuring that therapies approved under expedited regulatory pathways demonstrate not only clinical benefit but also real-world value. Programs such as the FDA’s Accelerated Approval Program, Breakthrough Therapy Designation, and Priority Review allow treatments for serious or rare conditions to reach the market based on preliminary evidence, such as surrogate endpoints. However, these expedited approvals often come with uncertainties regarding long-term safety, effectiveness, and economic impact, creating challenges for payers and healthcare decision-makers. HEOR helps address these gaps through real-world evidence (RWE), economic modeling, and patient-centered outcomes research.

Key Contributions of HEOR in Accelerated Approvals

1.Addressing Uncertainty with RWE

Many drugs approved through expedited pathways rely on surrogate endpoints (e.g., tumor shrinkage, biomarker reduction) rather than definitive clinical outcomes such as overall survival or disease remission. This creates challenges for payers when determining appropriate coverage policies. HEOR helps mitigate this uncertainty by:

• Generating RWE to assess how the drug performs in broader, real-world populations beyond clinical trials.
• Examining long-term effectiveness and safety using claims databases, electronic health records (EHRs), and patient registries.
• Conducting comparative effectiveness research (CER) to understand whether the drug offers meaningful improvements over existing treatments.

Example: A gene therapy for hemophilia may show factor level improvements in clinical trials but lacks long-term data on bleeding risk reduction. HEOR studies tracking hospitalization rates, emergency visits, and quality of life improvements help payers make informed coverage decisions.

2. Informing Cost-Effectiveness and Budget Impact Assessments

Payers often hesitate to cover high-cost drugs, especially when long-term benefits remain uncertain. HEOR provides critical insights by:

• Developing cost-effectiveness models that assess whether a new therapy provides value relative to its cost.
• Performing budget impact analyses to evaluate the financial strain a new therapy may place on healthcare systems.
• Conducting early health technology assessments (HTA) to anticipate payer concerns before launch.

Example: An oncology drug approved based on progression-free survival may cost $150,000 per year, but HEOR models assessing total healthcare costs, including hospitalizations, adverse events, and productivity loss can help justify (or challenge) its reimbursement.

3. Addressing Payer Concerns Through Innovative Access Strategies

Due to the uncertainty associated with accelerated approvals, many payers implement access restrictions such as step therapy or prior authorization. HEOR supports strategies that enhance market access, including:

• Designing performance-based risk-sharing agreements (RSAs) where reimbursement is tied to patient outcomes.
• Developing value-based pricing models that adjust cost based on a drug’s real-world effectiveness.
• Providing indirect treatment comparisons (ITCs) when head-to-head clinical trials are unavailable.

Example: For a CAR-T therapy priced at $400,000 per patient, an outcomes-based agreement could link reimbursement to one-year survival rates or reduced relapse rates, ensuring that payers only pay for successful treatments

4. Enhancing Patient-Centered Outcomes Research (PCOR)

Beyond clinical efficacy, regulators and payers increasingly emphasize patient-reported outcomes (PROs) such as quality of life, symptom burden, and treatment satisfaction. HEOR strengthens this aspect by:

• Conducting PRO studies to highlight the therapy’s impact from a patient perspective.
• Assessing treatment adherence and persistence to ensure real-world effectiveness aligns with trial results.
• Exploring disparities in treatment access among different patient populations.

Example: In multiple sclerosis, a newly approved oral therapy may show improved adherence compared to injectable alternatives, supporting its value proposition.

5. Supporting Post-Marketing Regulatory Requirements

Drugs approved under accelerated pathways often require post-marketing studies (Phase IV trials) to confirm their clinical benefit. HEOR contributes by:

• Designing observational studies that track long-term safety and effectiveness.
• Using patient registries and real-world databases to fulfill regulatory commitments.
• Evaluating treatment sequencing and utilization patterns to optimize guidelines

Example: The FDA required confirmatory trials an Alzheimer’s drug, after its approval based on amyloid plaque reduction rather than cognitive outcomes. HEOR studies assessing real-world cognitive function and healthcare resource utilization are essential in this case. If post-marketing studies of a new Alzheimer’s therapy fail to show significant cognitive improvement, payers may reconsider reimbursement restrictions or update step therapy policies.

Summary

While accelerated approvals provide critical early access to life-saving therapies, they also introduce uncertainties regarding clinical and economic value. HEOR plays a vital role in mitigating these concerns by generating robust real-world evidence, informing cost-effectiveness analyses, supporting payer negotiations, and ensuring long-term patient outcomes are met. By integrating HEOR strategies early in drug development, manufacturers can strengthen their value proposition, navigate regulatory challenges, and ultimately improve patient access to innovative treatments.

HEOR serves as a critical tool in addressing these challenges by providing:

• Robust real-world evidence to support formulary decisions.
• Cost-effectiveness and budget impact models to justify pricing.
• Risk-sharing strategies to mitigate financial exposure.
• Utilization management insights to ensure appropriate patient access.