Reimbursement of licensed cell and gene therapies across the major European healthcare markets

Abstract

ObjectiveThe aim of this research is to identify the pricing, reimbursement, and market access (P&R&MA) considerations most relevant to advanced therapy medicinal products (ATMPs) in the Big5EU, and to inform their manufacturers about the key drivers for securing adoption at a commercially viable reimbursed price.MethodologyThe research was structured following three main steps: 1) Identifying the market access pathways relevant to ATMPs through secondary research; 2) Validating the secondary research findings and addressing any data gaps in primary research, by qualitative interviews with national, regional, and local-level payers and their clinical and economic advisors; 3) Collating of primary and secondary findings to compare results across countries.ResultsThe incremental clinical benefit forms the basis for all P&R&MA processes. Budget impact is a key consideration, regardless of geography. Cost-effectiveness analyses are increasingly applied; however, only the United Kingdom has a defined threshold that links the cost per quality-adjusted life year (QALY) specifically and methodologically to the reimbursed price. Funding mechanisms to enable adoption of new and more expensive therapies exist in all countries, albeit to varying extents. Willingness to pay is typically higher in smaller patient populations, especially in populations with high disease burden. Outcomes modelling and risk-sharing agreements (RSAs) provide strategies to address the data gap and uncertainties often associated with trials in niche populations.ConclusionsThe high cost of ATMPs, coupled with the uncertainty at launch around their long-term claims, present challenges for their adoption at a commercially viable reimbursed price. Targeting populations of high disease burden and unmet needs may be advantageous, as the potential for improvement in clinical benefit is greater, as well as the potential for capitalising on healthcare cost offsets. Also, targeting small populations can also help reduce both payers’ budget impact concerns and the risk of reimbursement restrictions being imposed.

Advanced therapy medicinal products: value judgement and ethical evaluation in health technology assessment

“…In the ATMPs product development, to achieve the market access, developers need to first have a clear understanding of the markets and their reimbursement requirements before initiating clinical studies, namely to have a clear understanding of the market environment for the target indication, the disease burden, the target patient type, the alternative treatments already available, the reimbursement profiles of alternative products and the reimbursement policy of the relevant payers [8]. Additionally, the minimum efficacy thresholds necessary to support a commercially viable reimbursed price, will need to be the identified [9]. In summary, to achieve success in the HTA and market access strategy, it will be essential to understand whether there is room for accommodating the innovation and to establish a solid positioning strategy [8].…”

“…On one hand, the focus of the analysis is typically the healthcare budget isolated, and savings in the social care budget (e.g., rehabilitation or long-term social care) are not adequately captured in the benefits assessment. On the other hand, the time horizon of the analysis is often 1-2 years, which implies that long-term benefits, such as in the case of ATMPs, are also inadequately captured [9].…”

“…Over the years, the assessment of price and reimbursement for innovative therapies has shifted towards valuebased models [8,9]. However, the methods by which added value is captured and translated into a reimbursed price vary in different geographies.…”

“…The clinical benefit is measured in comparison to a suitable comparator. This comparator can differ between countries, since clinical practice may be different in different settings, which raises the need to perform indirect comparisons [9].…”

The potential price and access implications of the cost-utility and budget impact methodologies applied by NICE in England and ICER in the US for a novel gene therapy in Parkinson’s disease

“…Furthermore, its use has gradually become more widespread in EU countries where it has historically seen limited use, e.g. France, and Spain [7]. …”