Mapping the pathway

If you want a drug to be reimbursed in country with publically funded healthcare, you’ve got to win approval from the local Health Technology Assessment (HTA) body. One of the biggest challenges in this process is developing the economic models for these submissions because every HTA body has different requirements. At least that is the theory. In preparation for the ISPOR 18th Annual European Congress, Quintiles conducted research to determine how different these requirements really are, and whether a universal economic model for all HTA submissions could one day be a reality.

To conduct this research, we reviewed submission guidelines from HTA agencies in UK, Germany, France, Sweden, Netherlands, Australia and Canada to identify the similarities and differences among their requirements for economic evaluations. We compiled and reviewed the key differences, and created a set of recommendations that manufacturers should consider when developing their market access planning and economic model development plans.

Regarding the question of whether a universal economic model for all HTA submissions is possible, the short answer is no. Our research shows that while there are many similarities between HTA economic requirements, it is unlikely that there will be a single economic model for all submissions due to the varied needs in resources and costs in different countries. But it’s not all bad news. Our data also show that the many similarities between HTA data requirements offer an opportunity for manufacturers to significantly streamline the HTA approval process by creating a core submission then customizing it for each region.

We consistently found broad agreements between HTA bodies regarding types of economic data used. For example, every agency considers direct medical costs but few considered indirect, societal, or patient costs. There was also agreement over the timelines, with life-time being the preferred option for all HTA bodies. However whilst the types of data are similar, the values vary locally. The major differences between national HTA submissions were the requirements for unit costs and treatment pathways to reflect local clinical practice. Also the preferred methods used to derive quality-of-life varied between HTA agencies.

Key takeaway: Build a core submission

Whilst our study did not provide a panacea for a single-solution HTA economic model, it does offer a roadmap for manufacturers on how to streamline their submission process. While there is no one set of data that will appease all HTA bodies, manufacturers can cut time and cost by creating a core HTA submission using the evidence that is consistently sought by all HTA bodies, then customizing it for specific countries.

We suggest that manufacturers choose one country to develop a base-case model then adapt this to other countries. The UK is a preferred choice for the base-case as it has high levels of evidence requirements for economic models. When locally adapting the model, manufacturers should include local input from HTA bodies, payers and affiliates to capture the relevant data necessary for reimbursement approval.

To achieve greater speed and efficiency, this HTA submission strategy should be taken into consideration during the research planning phase to ensure all relevant economic data that is necessary for each market approval process is collected in good time. By starting early, building a core model, and incorporating local feedback to adapt the submission for each market, manufacturers may lessen the burden of the HTA approval process, and increase their competitive advantage.

Key similarities and differences between HTA economic models:

  • Cost-utility analysis (CUA) is the preferred method of assessing cost-effectiveness for most countries.
  • Good practice for conducting network-meta analysis and indirect comparisons is broadly similar, with the UK’s NICE providing the greatest detail.
  • Mapping Quality of Life (QoL) is widely accepted, but there is a preference for patient reported outcomes (PROs) collected during randomized clinical trials using standardized instruments to measure health outcome.
  • Statistically significant and clinically meaningful improvement in overall survival remains the gold standard to demonstrate the clinical benefit of new cancer treatments.
  • If practically and ethically possible, it is best to avoid treatment switching in oncology as it may lead to underestimation of OS benefits and not all HTA agencies accept the statistical adjustments methods that are currently available.
Topics in this blog post: Biopharma, Evidence, EU, HTA, Market Access