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PMU93 ASSESSING THE OUTCOMES FOR ORPHAN STATUS OR ACCELERATED ASSESSMENT EMA APPROVED MEDICINES: A COMPARISON OF HEALTH TECHNOLOGY ASSESSMENT IN DENMARK, FINLAND, NORWAY AND SWEDEN

      Objectives

      Whilst the aims of national health technology assessment (HTA) bodies in Denmark, Finland, Norway and Sweden are similar, each agency has specific evidentiary requirements, with no joint decision making. This study contrasted the HTA requirements in these Nordic countries and subsequent HTA outcomes of selected European Medicines Agency (EMA) approved treatments.

      Methods

      A search for Nordic HTA assessments of EMA-approved orphan status or accelerated assessment treatments from 2016-2017 was carried out using the IQVIA HTA Accelerator (HTAA). The analysis was limited to treatments where an HTA was available for at least three of the Nordic countries. The outcomes of the HTAs were validated by conducting a targeted search of agencies also involved in reimbursement negotiations, but not included in the HTAA database e.g. the National Therapeutics Council in Sweden. Finally, a report of hospital/pharmacy sales data was obtained from IQVIA and national sources, to understand the availability and uptake of these treatments in each country, outside of the reimbursement status.

      Results

      Forty-nine orphan/ accelerated assessment treatments were approved by the EMA in 2016-17. Six were selected for analysis, as they were assessed in a minimum of three countries: inotuzumab ozogamicin, cabozantinib, daratumumab, glecaprevir/pibrentasvir, nonacog beta pegol and nusinersen. Reimbursement was approved for 50%, 83%, 83% and 100% of treatments in Denmark, Finland, Norway and Sweden respectively. Negative or ‘no recommendation’ were either due to a perceived lack of benefit or due to cost. In Finland, Norway and Sweden, a negative/no recommendation due to cost did not appear to restrict sales; indicating that pricing negotiation ensued.

      Conclusions

      Findings from this study suggest that the evidence requirements for reimbursement of orphan/accelerated medicines varies greatly across the Nordic countries. It highlights the need for harmonised processes to enable faster access to patients. This may be partly addressed by the Finose collaboration.