Time to define common European criteria for therapeutic added value

Abbreviations

COVID

coronavirus disease

EMA

European Medicines Agency

EUnetHTA

European Network for Health Technology Assessment

HTA

Health Technology Assessement

Determining the price and reimbursement of a new medicinal product is of national competence within the Member States of the European Union that is carried out by health authorities. The European Commission plans to make cooperation in Health Technology Assessment (HTA) between EU countries mandatory from 2022 [1]. The aim of this cooperation, leading to a common assessment of the therapeutic added value of medicines after authorization by the European Medicinal Agency (EMA), would be to eliminate redundant national assessments, inform the bodies responsible for setting prices and reimbursement, and speed up market access for the most promising medicines. This could also facilitate joint drug purchasing, particularly for innovative drugs or vaccines, as in the case of coronavirus disease (COVID) vaccines.

In 2009, a European Network for Health Technology Assessment (EUnetHTA) has been set up, based on the voluntary collaboration of the national HTA Agencies, with the objective of drafting joint assessment reports. However, this European cooperation has so far been a failure: between 2017 and 2020, 15 joint reports have only been produced [2]. There are three reasons for the reluctance to carry out a collaborative evaluation, particularly if it becomes mandatory.

The first is that each country has developed its own drug evaluation policy with criteria that take into account both the efficacy and safety of the drugs, and a medico-economic aspect considering in particular its impact on the health budget. The definition and share of each of these two components in national pricing is neither homogeneous nor always very clear [3].

The second reason is more related to the fear of a loss of sovereignty for each Member State over its health policy and expenditures, which is reflected in the lack of transparency of the cost of medicines in each Member State.

The third reason is that opinions on the added therapeutic value can be very divergent. In our article comparing this concept applied in France and Germany [4], one medicine out of two resulted in a divergent assessment between the 2 Agencies in charge of this evaluation. From a scientific point of view, the main reasons for major inconsistency were related to the choice of comparators in controlled clinical trials, a different appreciation of results from subgroup analyses of efficacy data and the use of surrogate endpoints.

It is therefore urgent that the Member States of the European Union draw inspiration from what is being done at the EMA level to develop precise guidelines that will enable a common assessment of the therapeutic added value. We make 3 proposals to develop a common approach:

• •it seems essential to work on a consensual and unambiguous definition of the concept of significant therapeutic added value, pillar of expectations from both patients and health professionals witnessing the marketing of new medicinal products. Such definition deserves to be subject to a wide public debate beyond experts, encompassing all interested parties, including patients, manufacturers and bodies responsible for setting prices at national level;
• •several countries have adopted a classification of therapeutic value such as France, Germany or Italy, with therapeutic added value considered as “major”, “significant”, “minor” or “lack of proof of benefit” compared to existing treatments. This approach seems interesting because it allows justifying economical rewards (prices) and reimbursement rates correlated to these scores;
• •the therapeutic added value expected from a medicinal product is not the same depending on targeted diseases, whether it is used to treat acute or chronic diseases, potentially debilitating or serious, life-threatening conditions… Elaboration of precise European guidelines on measures of minimal clinical significant differences depending on diseases, could be of great help, with applications from designing adequate clinical trials to harmonizing evaluation of clinical data at HTA stage.

This common European assessment of the therapeutic added value could then be exploited at the National level for medico-economic assessment purposes, which would then take into account local budgetary and epidemiological specificities (number of patients concerned, burden of the specific disease in National health expenditures, local health system particularities, etc.) to define prices and levels of reimbursement of specific medicines. Such common approach would also contribute to provide greater transparency in pricing mechanisms at a time of increasing costs of medicines.

Disclosure of interest

The authors declare that they have no competing interest.