RWE complements the overall picture of the authorities

In health economic studies, real world evidence (RWE) can produce additional knowledge, especially on the patient characteristics, the use of health services and the differences between current and comparison treatments.

Real world evidence has its role in supporting the work of authorities, states Senior Specialist at Pharmaceuticals Pricing Board Hila, Kalle Aaltonen, at Medaffcon’s customer evening.

According to Aaltonen, Hila considers carefully the internal and external validity of a study, that is, generalisability.

– RWE can bring more generalisability to the study, to a limited extent. The data produced through clinical trials is always primary.

In health-economic studies, RWE can produce more information on the patient characteristics, the use of health services and the differences in the current and comparison treatments. For products that have been in the market for longer, we can receive information related to the treatment effect.

Contributes to improving generalisability

According to Aaltonen, RWE related to patient characteristics complements the overall picture especially if the patient group of a clinical trial cannot be generalised to Finland.

– It is very case-specific when the patient group cannot be generalised to Finland. This is influenced, for example, by the differences in the healthcare systems and funding in different countries.

On the basis of the RWE material, one can also model RWE data, such as predict a treatment response for another patient group and thus aim at improving generalisability.

– It is necessary to have correctly parametrised models. It is also important to note that if improving the generalisability of the results of clinical trials is up to the applicant of reimbursement, it enables opportunism. An applicant can improve the generalisability of the results if it benefits them and ignore it if the results are not beneficial for the applicant.

RWE and renewal application

According to Aaltonen, it could be useful to use RWE to ensure the long-term results of a medicinal product, if the follow-up time of a clinical trial is short.

– This can, of course, be difficult. If a medicine requires a long follow-up in a clinical trial, it also requires that in the everyday life of healthcare.

The participants at Medaffcon’s customer evening were interested in the situations where Hila requires RWE in connection with a re-application and whether this will increase in the future.

According to Aaltonen, this is case-specific. RWE can be required if the conclusion depends on whether cost savings are realised, and the information can be achieved with register data in a certain period.

– The need and possibility for further evidence impact the decision on requesting the data.

Currently, the Pharmaceuticals Pricing Board has the right to ask the applicant to provide further information in connection with a renewal application. Aaltonen demonstrated two examples of this:

Hila’s Senior Specialist Kalle Aaltonen talked about the use of RWE in health economic reviews at Medaffcon’s customer evening organised at Espoo Museum of Modern Art Emma on 14 Sep 2022.