Budget Impact Models for New Drug Funding Best Practices
- R.M. Boylan
- Jan 21
- 4 min read
Introduction:
The International Society for Pharmacoeconomic and Outcomes Research outlines best practices for constructing a Budget Impact Model for a new drug within an existing drug class. These practices align with the Patented Medicine Prices Review Board Canada's guidelines for Budget Impact Modelling. The task involves identifying common best practices and integrating recommendations by clearly distinguishing the differences and similarities between the approaches in the US and Canada.
The International Society for Pharmacoeconomics and Outcomes Research (ISPOR) provides guidelines for developing budget impact models (BIMs) to assess the economic implications of new drugs. These practices are crucial for decision-makers in healthcare, particularly for patented manufacturers and regulatory bodies like the Pricing Review Board in Canada. Below, we outline the best practices from ISPOR and compare them with the recommendations from the Canadian context, highlighting similarities and differences.
ISPOR Best Practices
Define the Scope: Clearly outline the perspective of the analysis (e.g., healthcare system, societal) and the time horizon.
Target Population: Identify the specific patient population that will benefit from the new drug.
Comparative Intervention: Select appropriate comparators, typically existing treatments within the same drug class.
Data Sources: Utilize reliable data sources for clinical effectiveness, resource utilization, and costs.
Model Structure: Choose an appropriate model structure (e.g., cohort, patient-level simulation) that reflects the disease progression and treatment pathways.
Uncertainty Analysis: Incorporate sensitivity analyses to assess the robustness of the model results.
Stakeholder Engagement: Involve stakeholders throughout the model development process to ensure relevance and applicability.
Key resources you may want to use:
Canadian Budget Impact Modelling Best Practices
Regulatory Context: Understand the specific requirements of the Canadian Pricing Review Board, including the need for transparency and justification of pricing.
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Real-World Evidence: Emphasize the incorporation of real-world data to validate model assumptions and results.
Budget Impact Thresholds: Consider the thresholds for budget impact that are relevant to Canadian health systems.
Equity Considerations: Address potential equity implications of the new drug and its budget impact on different populations.
Collaboration with Payers: Engage with payers early in the process to align on expectations and data requirements.
Key resources:
chrome-extension://efaidnbmnnnibpcajpcglclefindmkaj/https://www.pmprb-cepmb.gc.ca/cmfiles/bia-may0738lvv-5282007-5906.pdf
Common Best Practices
Clear Definition of Scope: Both ISPOR and Canadian guidelines stress the importance of defining the scope and perspective of the analysis.
Target Population Identification: Both frameworks emphasize the need to identify the relevant patient population.
Use of Reliable Data: Both advocate for the use of high-quality data sources to inform the model.
Uncertainty Analysis: Sensitivity analysis is highlighted as a critical component in both contexts.
Stakeholder Engagement: Engaging stakeholders is recognized as vital for ensuring the model's relevance.
Differences Between US and Canada
Regulatory Environment: The US has a more fragmented healthcare system, while Canada has a two-tiered model which has an umbrella single-payer model that influences budget impact assessments. In Canada a public payer model is required for each public payer and then for private payers depending on the private payer.
Pricing Justification: Canadian models require explicit justification of pricing to the Pricing Review Board as separate to the model, whereas US models may focus more on market access strategies.
Data Availability: The availability and types of real-world evidence may differ, impacting model assumptions and outcomes.
Equity Considerations: Canadian models may place more emphasis on equity and access issues compared to US models.
Other Elements in Health Technology Assessment to Consider
Long-term Outcomes: Assess long-term health outcomes and their impact on quality of life.
Cost-Effectiveness Analysis: Integrate cost-effectiveness analysis alongside budget impact modeling for a comprehensive assessment.
Patient Preferences: Consider incorporating patient preferences and values in the assessment process.
Implementation Challenges: Address potential challenges in the implementation of the new drug within the healthcare system.
Health Equity: Evaluate the implications of the new drug on health equity and access across different populations.
In conclusion, while ISPOR and Canadian best practices for budget impact modeling share several commonalities, they also reflect distinct regulatory and contextual differences. By merging these insights, manufacturers can develop more robust models that effectively address the needs of healthcare decision-makers in both regions.
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