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AIXIAL review #12

Will early access put performance contracts back on the agenda?

New, allegedly innovative drugs with uncertain performance and supposedly high costs are multiplying. In many cases, they will use the new derogatory route of early access. 

While granting access to this type of drug automatically leads to coverage and a temporarily free price, it may nevertheless raise pricing difficulties when they are brought into the ordinary law.

At the stage of their passage through the „HTA assessment“ (i.e., in France to the Transparency Commission), making an informed judgement on the medical service they are likely to provide to the community is, if not impossible, at least particularly difficult. It is therefore risky, given the expected prices, to predict their efficiency from the outset, and even more so to predict their long-term budgetary impact.

In addition to the medical risk of postponing or accepting this type of drug too early, the economic risk that they carry with them can weigh heavily in the decision-making of health authorities.

In an attempt to manage these risks, risk-sharing contracts have been developed in many countries (to a limited extent in France, but much more so in countries such as Italy[1], particularly performance contracts between payers (in France, the CEPS) and manufacturers.

Such contracts are based on real-life performance estimated on the basis of relevant and reliable clinical indicators that allow for evaluation at the population or individual level („pay to see“ or „satisfied or reimbursed“)[2] . These contracts therefore have, at least in theory, predictable consequences.

As regards early access (granted for one year, possibly renewable), the company benefiting from it is obliged to monitor the patients treated (PUT-RD)[3]. But unlike performance contracts, the pricing consequences of the results of this type of monitoring are, if not absent, at least hardly predictable. Formalized in the form of PUT-RD, just like post-registration studies, the purpose of this type of monitoring is health-related, not price-related.

In France, although the current president of the CEPS has declared himself in favour of performance contracts[4], the experience that has emerged seems to be very mixed[5] and these contracts cannot be applied to all cases, for example when the time horizons for acquiring evidence require long periods, which are incompatible with price adjustments.

Moreover, the citizen has some difficulty in assessing the outcome because their clauses are hardly accessible and it is difficult to know whether the bets made when they were signed have been kept, except when the performance is good, in which case the results will obviously be more readily published.

Based on the experience of disappointing contracts, the question arises as to whether the pricing of what are now called „disruptive innovations“ – which are fraught with uncertainty but can be expected to have a high budgetary impact – might not be more appropriately covered by more satisfactory contracts?

Drawing on the experience of many countries that use them[6] and provided that a number of key points are agreed upon:  – indisputable indicators on which to base them, (developed by the HAS?), guarantees of collection, analysis and evaluation of monitoring data, securing mechanisms for applying tariff consequences according to results – could such contracts become more attractive for France? then becoming one of the keys to the difficult financing of allegedly costly innovations.

Examples of successful contracts, useful to public health and to both contracting parties, would possibly reduce payers‘ fears and put these contracts back on the agenda. But society still needs to be reassured that decision-makers are making good use of public funds. If up to now, citizens do not seem to be concerned by the financing of innovations, leaving the administration to do the best it can, the amounts of money committed on behalf of the community for presumed innovations in the last few years will undoubtedly increase the expectations of transparency. Knowing how CEPS accepts such contracts and how they are respected, without infringing on industrial secrets, seems to be the minimum required.

[1] Outcomes-Based Contracting Experience: Research Findings from U.S. and European Stakeholders Nazareth et al. (October 2017). Accessed May 30, 2022.

[2] L’accès au marché remboursé pour les médicaments : Les contrats de partage de risque fondés sur les résultats. Gérard de Pouvourville and Louis Mongrédien (July 2012). Accessed May 30, 2022.

[3] Accès précoce et compassionnel aux médicaments : quels changements pour les industriels ? HAS Official website (July 2021). Accessed May 30, 2022.

[4] Report, « Commission d’enquête chargée d’identifier les facteurs qui ont conduit à la chute de la part de l’industrie dans le PIB de la France et de définir les moyens à mettre en œuvre pour relocaliser l’industrie et notamment celle du médicament » (November, 2021), Hearing at the National Assembly. Accessed May 30, 2022.

[5] Jean-Patrick Sales (CEPS) : « Les deux inconvénients du paiement étalé » Gilles Noussenbaum (July, 2020) in « Décision & Stratégie Santé ». Accessed May 30, 2017. Available at: https://www.decision-sante.com/medecine-pharmacie/actualites/jean-patrick-sales-ceps-les-deux-inconvenients-du-paiement-etale. Accessed May 30,  30 mai 2022

[6] Outcomes-Based Contracting Experience: Research Findings from U.S. and European Stakeholders Nazareth et al. (Octobre 2017). Accessed May 30,  30 mai 2022.

— Written by, Ryan Trehard, economist, consultant at AIXIAL Group and Jacques MASSOL, MD, PhD, Prof of therapeutics, consultant at AIXIAL Group

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