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Hearing for HTA-method developers December 9th 2022

Pharmaceuticals Pricing Board (HILA), Finnish Medicines Agency (Fimea), Social Insurance Institution (KELA) and Finnish Coordinating Center for Health Technology Assessment (FinCCHTA) organize a hearing on methods used for Health Technology Assessment (HTA) on Friday December 9th 2022. In the event, the developers are given an opportunity to present their methods potentially used in the upcoming reimbursement submissions or appraisals to Finnish authorities involved in HTA. The deadline for applying as a presenter was October 14th 2022 and there were five applications. Three presenters were selected based on the applicability to authorities’ work while trying to avoid excessive similarities between presentations.

In their presentation Medaffcon reviewed which real world data is available from registries and how readily so. Medaffcon introduced ”virtual control arms” based on patient records, as well as methods used to account for differences between clinical trials and observational data. According to data Analysis Lead Iiro Toppila, these methods differ from each other regarding the data requirements, but even if only the aggregated patient characteristics are available from the clinical trial, the patients from register data can be weighted to match them. Medaffcon had created simulated data from a hypothetical clinical trial and patient register, which they employed to demonstrate examples of results from different mathing and weighting methods.

Professor Janne Martikainen from the University of Eastern Finland presented a summary of the publications and teaching of the research group he leads. A member of the group, adjunct professor Piia Lavikainen gave a presentation on the utilization of electronic patient records as a source for RWD with an example of type 2 diabetes. Lavikainen described the data available within the patient registry of Siun Sote Hospital district, known as Mediatri, which can be among other things used to calculate treatment costs. In her example, Lavikainen presented an example of the data being used to identify HbA1c-trajectories for type 2 diabetes patients and their differences between treatment complications and costs. In the second example, interrupted time series was used to analyze the effect of change in reimbursement level and covid lockdown on the treatment balance. The third example was about the correlation structure of patient characteristics predisposing to type 2 diabetes. Researcher Aku-Ville Lehtimäki compared cohort and microsimulation models in his presentation. According to Lehtimäki microsimulation facilitates straightforward coding of differences between subgroups at the expense of increased computational time.

Senior lecturer Howard Thom from the University of Bristol with Professor Gianluca Baio introduced the R for HTA –collaboration and the meetings and courses hosted by it. Thom described Excel-software as computationally slow and limited in terms of statistical capabilities and transparency. According to Thom, R facilitates improvements to all of these aspects, and can be further expanded with packages. The presentation comprised a comparison between Excel and R in running the same model, demonstrating the speed advantage for the latter. Thom also regarded the possibility of commenting the code as a transparency advantage. The presentation comprised examples of results from a cost-effectiveness model plotted using the BCEA-package and with RShiny graphical user interfaces. Furthermore, other packages used in cost-effectiveness modeling were listed. Thom shared R code for a decision tree model via GitHub, which the attendees executed themselves.