The impact of parallel regulatory-health technology assessment scientific advice on clinical development. Assessing the uptake of regulatory and health technology assessment recommendations.

AIMS: The parallel regulatory-health technology assessment scientific advice (PSA) procedure allows manufacturers to receive simultaneous feedback from both EU regulators and health technology assessment (HTA) bodies on development plans for new medicines. The primary objective of the present study is to investigate whether PSA is integrated in the clinical development programmes for which advice was sought. METHODS: Contents of PSA provided by regulators and HTA bodies for each procedure between 2010 and 2015 were analysed. The development of all clinical studies for which PSA had been sought was tracked using three different databases. The rate of uptake of the advice provided by regulators and HTA bodies was assessed on two key variables: comparator/s and primary endpoint. RESULTS: In terms of uptake of comparator recommendations at the time of PSA in the actual development, our analysis showed that manufacturers implemented comparators to address both the needs of regulators and of at least one HTA body in 12 of 21 studies. For primary endpoints, in all included studies manufacturers addressed both the needs of the regulators and at least one HTA body. CONCLUSIONS: One of the key findings of this analysis is that manufacturers tend to implement changes to the development programme based on both regulatory and HTA advice with regards to the choice of primary endpoint and comparator. It also confirms the challenging choice of the study comparator, for which manufacturers seem to be more inclined to satisfy the regulatory advice. Continuous research efforts in this area are of paramount importance from a public health perspective.

Ten years of AMNOG from an oncological perspective: new horizons and continuing expansion.

PURPOSE: 2021 marks the tenth anniversary of the AMNOG process and brought with it a new German administration-two good reasons to take stock of where we stand today, what has been achieved so far, and how the path of early benefit assessments in Germany should continue. RESULTS: From the perspective of manufacturers of cancer drugs, the AMNOG process, as a constantly evolving system, has for the most part proved itself-which does not mean that there is no longer room for improvement. Significant improvements have been achieved in the area of early consultation of medical societies regarding the selection of the appropriate comparator therapy as well as in the reimbursement of biomarker diagnostic tests in the outpatient sector. However, there is still a need for improvement, especially in the areas of patient-relevant outcomes accepted by the G-BA, the inclusion of real-world data in evidence assessments, or the transfer of evidence from certain patient groups to others. CONCLUSION: The current AMNOG structures were developed for the most part at a time when no one saw immuno-oncology or gene and cell therapies coming, when there were no multi-tumor drug approvals, and when few imagined that within a few years, the established tumor entities would be broken down into dozens of sub-entities on the basis of molecular genetic markers. Society wants these and other advances, and the HTA process must, therefore, take this into account in a healthcare system based on solidarity.