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La valutazione farmacoeconomica nel contesto regolatorio italiano

Analisi quali-quantitativa dei dossier di richiesta del prezzo e della rimborsabilità

Pharmacoeconomic evaluations in the Italian regulatory context: a quali-quantitative analysis of pricing and reimbursement dossiers

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PharmacoEconomics Italian Research Articles

Summary

Objective

In the last decade, pharmacoeconomics has gained an increasing influence on the decisional process in the regulatory context of several European countries. The main aim of this analysis was to assess the quality of pharmacoeconomic evaluations enclosed by pharmaceutical companies in the pricing and reimbursement dossiers presented to the Italian Medicines Agency (AIFA).

Methods

A survey of pricing and reimbursement dossiers presented to AIFA between August 2004 (date of the agency’s institution) and November 2007 was conducted. According to Italian regulations concerning the submission of pharmacoeconomic information to national authorities, only those dossiers regarding the authorization of new chemical entities (never marketed before), orphan drugs for the treatment of rare diseases and those requiring the extension of therapeutic indications of already marketed drugs were selected for the survey. Data on both methodological features of pharmacoeconomic evaluations and overall features of clinical trials used as source of efficacy data for the economic analyses were collected. Some descriptive statistics of incremental cost-effectiveness ratios reported in the dossiers were also provided.

Results

In the time-span of about three years of AIFA activity, 109 dossiers have been reviewed and for 34 of them pharmaceutical companies provided also pharmacoecononomic analyses. Overall the production rate was 36% (39 out of 109 dossiers); as a matter of fact, a dossier may contain more than one pharmacoeconomic analysis (i.e. one for each therapeutic indication).

As expected, cost-effectiveness analysis is the most frequent type of economic evaluation (53.8%), followed by cost-utility analyses (20.5%). Cost-minimization analyses represent 25.6% of all economic evaluations, which is notably higher than the observed frequency in the international literature. Most economic evaluations were conducted for the assessment of new treatments for oncologic diseases or biotechnological products for rheumatic diseases, followed by drugs for infectious diseases (mostly AIDS) or neurological conditions.

The overall qualitative features of pharmacoeconomic evaluations in the dossiers appear to be highly variable, ranging from analyses meeting the highest methodological standards (especially in dossiers concerning medicines authorized via centralized procedure) to a dossier not providing basic information and describing the pharmacoeconomic results only in qualitative terms.

Based on drug prices submitted by pharmaceuticals manufacturers to AIFA, mean values of incremental cost-effectiveness ratios obtained after bootstrap resampling resulted € 17,907 per QALY (95% CI: 7,000–25,912) and € 41,676 per life-year saved (95% CI: 16,300–71,431).

Conclusions

Pharmacoeconomic techniques for health technology assessment of drug therapies need further development in the Italian context. In this perspective, the current survey of pharmacoeconomic evaluations enclosed in pricing and reimbursement dossiers could provide several suggestions.

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  1. Agenzia Italiana del Farmaco, Via della Sierra Nevada 60, 00144, Roma, Italia

    P. Russo

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I contenuti del presente articolo riflettono la libera espressione e opinione dell’autore e non esprimono in alcun caso la posizione ufficiale dell’Agenzia Italiana del Farmaco.

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Russo, P. La valutazione farmacoeconomica nel contesto regolatorio italiano. Pharmacoeconomics-Ital-Res-Articles 10, 59–75 (2008). https://doi.org/10.1007/BF03320643

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