Key factors influencing positive Health Technology Assessment (HTA) outcomes: First-of-its-kind analysis

Pharma News: In a first-of-its-kind analysis spanning seven OECD countries, including Australia, researchers delved into a multitude of parameters to determine the factors influencing positive Health Technology Assessment (HTA) outcomes and the time it takes to achieve them. This is especially relevant as Australia navigates the current HTA Review consultation 2.

Amid escalating concerns over healthcare budgets and fiscal sustainability, the demand for healthcare continues to surge, accompanied by the emergence of expensive new technologies.

Time-to-HTA Outcome: Navigating the Path to Faster Access

The analysis reveals a strong association between financial risk-sharing agreements (RSAs) and shorter time-to-HTA outcomes, providing evidence that RSAs facilitate expedited access to novel therapies. Simultaneously, a parallel review process aligning regulatory and HTA evidentiary requirements is shown to significantly reduce the time-to-HTA outcome.

However, the analysis uncovers a counterintuitive trend, as orphan drugs are associated with a longer time-to-HTA outcome. This finding is attributed to incomplete or early-phase evidence, along with negotiations required for coverage decisions post-appraisal.

Remarkably, no statistically significant differences are detected between accelerated approval pathways and standard marketing authorisation, highlighting significant evidence uncertainties for drugs qualifying for accelerated approval pathways, especially orphan drugs.

Factors Shaping HTA Outcomes: Navigating the Balancing Act

While the majority of medicines receive positive HTA outcomes, the extent of clinical and economic restrictions varies. The analysis demonstrates a delicate balancing act, weighing micro-economic efficiency, macro-economic efficiency, and equity in decisions. Resubmitted applications face challenges, significantly less likely to achieve unrestricted listing following a rejection, emphasising evidence uncertainties as a hurdle.

Oncology and orphan drugs are shown to be less likely to secure positive HTA outcomes, indicating the influence of evidence uncertainties and country-specific criteria on decisions. Notably, orphan drugs with an oncology indication are more likely to achieve positive outcomes – despite the existence of evidence uncertainties – underscoring the importance of high unmet needs in decision-making.

Navigating Uncertainties: Clinical and Economic Perspectives

Clinical evidence uncertainties, such as the magnitude of clinical benefit and trial result generalisability, are associated with negative HTA outcomes, contributing to rejections by HTA agencies. However, not all uncertainties lead to negative outcomes, as economic modelling uncertainties can be overcome by sponsors.

While there is a significant association between high-quality clinical evidence and positive HTA outcomes, the majority of positive HTA outcomes are associated with average or low quality evidence. This highlights that HTA agencies consider additional parameters of benefit, including lower cost or unmet medical need, to arrive at positive HTA recommendations.

Special Pathways and Cost-Effectiveness: A Global Perspective

Special pathways, indicated by statistically significant social value judgments and product-specific interaction effects, highlight the existence of distinct evaluation routes in most countries. Cost-effectiveness estimates play a pivotal role in HTA outcomes, with no significant differences detected between confidential ICER, explicit ICER, or an analysis without an ICER in association with HTA outcomes.

Cross-Country Differences: A Nuanced View of Decision-Making

Country fixed effects capture nuances in HTA processes, revealing both similarities and differences across agencies and settings. Despite comparable standards for high-quality evidence, differential stances on oncology and orphan drugs, as well as approaches to overcome clinical uncertainties, contribute to varied HTA outcomes.

Policy Implications: Towards Informed Decision-Making

The analysis yields several policy implications for countries with established HTA processes and those considering adoption. While most HTAs result in positive recommendations, the majority are optimised by restricting use or calibrating prices to align with health policy objectives.

The multiplicity of criteria in decision-making calls for explicit recognition of parameters as benefits, advocating for a broader set of criteria in evaluating new medical technologies. Standardisation of assessment criteria across settings could reduce duplication of effort, offering a potential avenue for harmonisation.

The study underscores the importance of robust performance metrics, value frameworks, and the consideration of special value judgments in decision-making. Evidence uncertainties do not guarantee rejection, emphasising the role of adaptive pathways and real-world evidence in contemporary HTA processes.

As clinical evidence becomes increasingly similar to that considered by regulatory bodies, the implementation of parallel review processes is suggested to expedite HTA outcomes and market entry. The study concludes that HTA bodies have adopted an extra-welfarist approach, considering broader criteria beyond economic evaluation, especially in therapeutic areas with immature evidence such as oncology and rare diseases.

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