Abstract
Even for centrally approved products, each European country is responsible for the effective national market access. This step can result in inequalities in terms of access, due to different opinions about the therapeutic value assessed by health technology assessment (HTA) bodies. Advanced therapy medicinal products (ATMPs) represent a major issue with regard to the HTA in order to make them available at a national level. These products are based on genes, tissues, or cells, commonly developed as one-shot treatment for rare or ultrarare diseases and mandatorily authorized by the EMA with a central procedure. This study aims to provide a comparative analysis of HTA recommendations issued by European countries (France, Germany, and Italy) following EMA approval of ATMPs. We found a low rate of agreement on the therapeutic value (in particular the “added value” compared to the standard of care) of ATMPs. Despite the differences in terms of clinical assessment, the access has been usually guaranteed, even with different timing and limitations. In view of the importance of ATMPs as innovative therapies for unmet needs, it is crucial to understand and act on the causes of disagreement among the HTA. In addition, the adoption of the new EU regulation on HTA would be useful to reduce disparities of medicine’s assessment among European countries.
Highlights
The centralized procedure adopted by the European Union (EU) and mandatory for some drugs category since 2004 (The Council of the European Communitie, 1993; European Parliament, Council of the European Union, 2001; European Parliament and the Council of the European Union, 2004) enables a rapid, EU-wide authorization of medicinal products based on a benefit/risk assessment, which requires the evaluation of quality, nonclinical, and clinical data on safety and efficacy submitted by the applicant
This study aimed to provide a comparative analysis of health technology assessment (HTA) recommendations issued by European countries following European Medicines Agency (EMA) approval of Advanced therapy medicinal products (ATMPs)
Identification of the reimbursement status and the HTA of ATMPs currently approved in Europe by the EMA performed by EU national authorities (France, Germany, and Italy); selection of countries was based on the availability of assessments for public consultation and on the clear definition of therapeutic values through comparable rating scales; 3)
Summary
The centralized procedure adopted by the European Union (EU) and mandatory for some drugs category since 2004 (The Council of the European Communitie, 1993; European Parliament, Council of the European Union, 2001; European Parliament and the Council of the European Union, 2004) enables a rapid, EU-wide authorization of medicinal products based on a benefit/risk assessment, which requires the evaluation of quality, nonclinical, and clinical data on safety and efficacy submitted by the applicant. Despite the unification of the procedures for drug approval, each country is Health Technology Assessment of ATMPs responsible for an effective national market access, which needs the pricing and reimbursement decision to be adopted This can result in patient access inequalities among European countries, due to differences in terms of willingness to pay and opinion about the therapeutic value assessed by health technology assessment (HTA) bodies. The HTA evaluates the added therapeutic benefits and risks for covering a new technology in the context of local standard of care (van Nooten et al, 2012), based on clinical (efficacy and safety), economic, ethical, and organizational aspects in support of policy decision-making about price and reimbursement decisions (Angelis et al, 2018). The heterogeneity of HTA recommendations, thereafter probably reflected in national reimbursement decisions and pricing agreements, is related to differences in assessment methodologies and healthcare systems’ organization and to the available evidence and, above all, willingness to accept uncertainty (Jommi et al, 2020)
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