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AIXIAL Review #9

Joint European clinical assessment of health products: what place remains for national Health Technology Assessment agencies?

 

An initiative of the European Commission foresees that the National Health Technology Assessment (HTA) institutions of the members of the European community jointly assess, via a coordination committee, the added value of health products with a view to their access to the market.

Rational project! one might say, because the scientific evaluation of this added value carried out so far by each of the Community countries, on its own account, with its own criteria, from a body of largely identical clinical data is somewhat redundant. A single assessment would save resources and perhaps time, and even give greater legitimacy in terms of robustness to this assessment. For its defenders, – note that it received the joint support of representatives of the German and French pharmaceutical industry Leem-vfa[1] – the heavy repetitive administrative burden, sometimes dissuasive, of the filing of files to be formatted according to local requirements, would thus be reduced. Finally, an advantage eagerly awaited by manufacturers, the joint assessment would allow better predictability.

However, such an initiative firstly raises the question of compliance with the principle of subsidiarity and would have significant direct and indirect consequences, it would directly modify the regulations of the Parliament and the Council and indirectly the missions of national HTA institutions. Knowing that the work provided via the European coordination committee would cover a large part of that carried out by the national HTA organizations: what place would it then leave to these institutions such as the Haute Autorité de Santé (HAS) – High Authority of Health – in France[2]?

To ensure respect for subsidiarity, the Commission stipulates that the joint evaluation would only describe and synthesize the clinical facts resulting from the R&D programs without making a value judgment on how each state should use them. The new regulation which would be imposed would also leave the possibility to the Member States to carry out additional clinical analyzes on the data which allowed its work. Likewise, the latter could carry out non-clinical evaluations at any time and would have the possibility of asking manufacturers for additional information, analyzes and other evidence, if necessary.

Thus described, it will be assumed that the joint evaluation would replace, at least partially, that part of the assessment carried out in France by the Transparency Commission (TC) which allows the level of added value of a drug to be graded, the ASMR (Improvement of the Rendered Medical Service) or by the CNEDIMTS for medical devices which uses the ASA (Improvement of the Expected Service).

How closely would the joint assessment overlap with that of ASMR or ASA? Details are awaited. However, we can already try to bring out the limits of such a criterion in order to deduce what would be lacking to enlighten decision-makers. This would leave us to assume the space that the national HTA organizations could retain.

How closely would the joint assessment overlap with that of ASMR or ASA? Details are awaited. However, we can already try to bring out the limits of such a criterion in order to deducedraw what would be lacking to enlighten decision-makers. This would leave us to assume the space that the national HTA organizations could retain.

Take medecine as an example. The ASMR is essentially based on a comparison of the clinical value of one drug generally against another. In good cases, the comparison is made face to face in a randomized, blinded controlled trial with an active product, known as an acceptable comparator, that is used optimally. It can be delivered either with respect to this comparator, in terms of efficacy and / or sometimes tolerance, or in the therapeutic strategy, often unspecified. For ASMR to be quantified between I and V (no ASMR), the amount of effect in terms of clinical efficacy, quality of life and safety is decisive. Indeed, only a clinical and / or quality of life benefit fully deserves the qualification of progress; those assessed on surrogate endpoints remote from clinical benefit being much more often hypothetical and uncertain. What about the progress brought about by a drug that improves the 6-minute walk test in a rare disabling disease?

In its doctrine dated December 2020[3], the TC mentioned weighting elements such as the notion of medical need and the quality of the demonstration. It assesses the effect size and its clinical relevance, in particular „with regard to the medical need in the indication evaluated“. „For example, a small amount of effect in terms of morbidity and mortality could lead to an ASMR V when the medical need is covered or to an ASMR greater than V when the medical need is not.“

Suppose the joint assessment synthesizes the clinical facts used to date to assess the ASMR, showing the theoretical clinical performance of one product compared to one or more others; that it can also, like TC, assess the quality of the demonstration, estimate the degree of uncertainty or even introduce the notion of medical need in a given disease. What would be missing then as information on the progress to be expected from a drug in order to inform decision-makers?

It will be assumed that in order to make decisions about a health product on behalf of a community, decision-makers need to know what it specifically brings to that community. However, the clinical data used to assess the ASMR or the ASA are insufficient to fully document this question, which requires collecting and taking into account purely national complementary elements, the assessment of which is a matter of national prerogatives. In order to estimate the specific contribution of a health product for its home community, it would therefore legitimately be up to the national HTA organizations to evaluate them and to adapt the added value evaluation to these national particularities.

How to achieve this adaptation? I will not be bold enough to make rules for the HTA organizations. I would just like to remind that in France, there is a criterion called “Public Health Interest” (PHI) which appears in the decree of October 1999. This criterion has been the subject of two successive studies to which I have had the pleasure to participate[4]. It contains, it seems to me, the missing elements which could usefully complement the joint evaluation as we have supposedly described it. Indeed, the very principle of PHI is to adopt a population perspective in real life for a given community. It feeds on the evaluation of the clinical added value of a health product without being limited to it. The health public impact of a product on a target population, the dependence of its effect on a given health system and its impact on it, the public health burden concerning its indication in a particular territory, the importance of the need it represents in this territory, taking into account the epidemiology of the disease in question, are PHI criteria which should make it possible to adapt the European assessment of added value to the particularities of each Member State.

HTA organizations should therefore not only retain a role in the evaluation of health products for market access but also develop their own methods for evaluating the improvement of the service provided by health products for their own. community.

Finally, such an assessment aimed at decision-makers could promote consistency between reimbursement and pricing decisions, which, however periodic and limited they may be, have a national public health impact – with public health policies of each country, be it public health plans, health system administration or biomedical and health research orientations …

— Written by Jacques Massol, MD PhD,  AIXIAL consultant

[1] https://www.leem.org/publication/position-commune-leem-vfa-sur-la-cooperation-europeenne-en-matiere-devaluation-des

[2] We will not discuss here the consequences in terms of medico-economic evaluation

[3] https://www.has-sante.fr/upload/docs/application/pdf/2021-03/doctrine_ct.pdf

[4] The first conceptualization work was carried out at the request of Lucien Abenhaim, then Director General of Health under the chairmanship of Bernard Bégaud. It gave rise to a first unpublished report: Evaluation of the impact of a drug on the health of populations and public health in 2002 C. Le Gales, J. Massol, B. Bégaud. The second is the result of the work of the ISP group, created at DGS and then transferred to HAS, a group that I have had the pleasure of leading for many years. It resulted in a report to the President of the TC entitled „Principles and Methods of Health Public Impact Assessment“. Its content was partially published in an article: The public health benefit of medicines: how it has been assessed in France? The principles and results of five years‘ experience. Maison P, Zanetti L, Solesse A, Bouvenot G, Massol J; ISPEP group of the French National Authority for Health.Health Policy. 2013 Oct; 112 (3): 273-84. doi: 10.1016 / j.healthpol.2013.04.007. Epub 2013 May 10

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