A European Efficacy/Effectiveness Assessment

The session seeks to discuss the challenges in developing and using EU relative efficacy/effectiveness assessments that can inform member states’ decisionmaking processes about the clinical added value of new medicines at the point of regulatory approval (market launch). It will gather representatives from the various actors most concerned in this issue: memberstates’ Health Technology Assessment (HTA) authorities, law-makers, decisionmakers, industry, patients, medical professionals. They will debate what European member states can deliver jointly to support better and faster reimbursement decisions that are based on the concept of clinical added value, with the aim of facilitating patient access to new medicines.

The premise of the EU in its efforts to move towards joint relative efficacy/effectiveness assessments, and more broadly all joint work in aspects affecting HTA is simple. Health is a preserved authority and so each member state needs to determine where a new medicine will be placed in its health system’s clinical pathway and to determine whether it adds value to currently existing treatments. But this can lead to duplication of effort across member states as the same clinical evidence is assessed and delays in decision- making. Hence it has been a driving ethos of the European network for HTA (EUnetHTA) for the past nine years to develop agreed methods for HTA and produce EU relative effectiveness assessments that can inform national decisionmaking. Despite this, national HTA authorities still seem to use their own methods and few joint assessments have led to actual decisions. National processes still take precedence.

EUnetHTA is about to enter its third EU Joint Action of Member States and is tasked with showing how its fairly theoretical work and pilot ventures can be translated into decisions that make a real difference for patients’ access to medicines. This panel will debate the challenges of developing a rapid effectiveness assessment at the point of product launch so that it can inform the reimbursement of a new medicine. Will we see the same evolution as in the field of Europe’s regulatory framework, or have times changed too much for history to repeat itself? What role can European regulators play in ensuring clinical data is not reinterpreted 28 times across Europe? What can politicians do to foster a better understanding of the need for this cooperation? What role can medical professionals play in ensuring that the output from joint work can make a meaningful contribution to clinical practice in member states? Is industry ready to adapt to the risk inherent in joint assessments in exchange for more speed and predictability? How can the system ensure patient-driven priorities become truly part of the effort?