Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case
Abstract Early access programs (EAPs) generally refer to patient access to medicines/indications before marketing authorization, possibly extended to price and reimbursement approval. These programs include compassionate use, which is usually covered by pharmaceutical companies, and EAPs reimbursed...
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| Format: | Article |
| Language: | English |
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Taylor & Francis Group
2023-05-01
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| Series: | Journal of Pharmaceutical Policy and Practice |
| Subjects: | |
| Online Access: | https://doi.org/10.1186/s40545-023-00570-z |
| _version_ | 1827360441575145472 |
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| author | Alice Tarantola Monica Hildegard Otto Patrizio Armeni Francesco Costa Francesco Malandrini Claudio Jommi |
| author_facet | Alice Tarantola Monica Hildegard Otto Patrizio Armeni Francesco Costa Francesco Malandrini Claudio Jommi |
| author_sort | Alice Tarantola |
| collection | DOAJ |
| description | Abstract Early access programs (EAPs) generally refer to patient access to medicines/indications before marketing authorization, possibly extended to price and reimbursement approval. These programs include compassionate use, which is usually covered by pharmaceutical companies, and EAPs reimbursed by third-party payers. This paper aims at comparing EAPs in four European countries (France, Italy, Spain, UK) and providing empirical evidence on EAPs in Italy. The comparative analysis was conducted through a literature review (including scientific and grey literature), complemented by 30-min semi-structured interviews with local experts. The Italian empirical analysis employed data available on the National Medicines Agency website. Although EAPs are very different across countries, they exhibit some common features: (i) eligibility criteria refer to the absence of valid therapeutic alternatives and a presumed favourable risk–benefit profile; (ii) payers do not allocate a pre-determined budget to these programs; (iii) total spending on EAPs is unknown. The French EAPs seem to be the most structured, financed through social insurance, covering pre-marketing, post-marketing and pre-reimbursement phases and providing for data collection. Italy’s approach to EAPs has been varied, with several programs covered by different payers, including the cohort-based 648 List (for both early access and off-label use), the nominal-based 5% Fund, and Compassionate Use. Most applications to EAPs are from the Antineoplastic and immunomodulating drug class (ATC L). Some 62% of indications in the 648 List are either not under clinical development or have never been approved (pure off-label use). For those subsequently approved, most approved indications coincide with those covered through EAPs. Only the 5% Fund provides data on economic impact (€ 81.2 million in 2021; average cost per patient € 61.5K). Diverse EAPs are a possible source of inequalities in access to medicines across Europe. A harmonization of these programs, though difficult to achieve, could be modelled on the French EAPs and provide key advantages, not least of which a common effort to collect real-world data in parallel with clinical trials and clear separation between EAPs and off-label use programs. |
| first_indexed | 2024-03-08T06:46:19Z |
| format | Article |
| id | doaj.art-8d6401fd725a4f5fb756bbf97b1bb5c0 |
| institution | Directory Open Access Journal |
| issn | 2052-3211 |
| language | English |
| last_indexed | 2024-03-08T06:46:19Z |
| publishDate | 2023-05-01 |
| publisher | Taylor & Francis Group |
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| series | Journal of Pharmaceutical Policy and Practice |
| spelling | doaj.art-8d6401fd725a4f5fb756bbf97b1bb5c02024-02-03T07:54:50ZengTaylor & Francis GroupJournal of Pharmaceutical Policy and Practice2052-32112023-05-0116111310.1186/s40545-023-00570-zEarly access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian caseAlice Tarantola0Monica Hildegard Otto1Patrizio Armeni2Francesco Costa3Francesco Malandrini4Claudio Jommi5SDA Bocconi School of Management, Centre for Research on Health and Social Care Management (CERGAS)SDA Bocconi School of Management, Centre for Research on Health and Social Care Management (CERGAS)SDA Bocconi School of Management, Centre for Research on Health and Social Care Management (CERGAS)SDA Bocconi School of Management, Centre for Research on Health and Social Care Management (CERGAS)SDA Bocconi School of Management, Centre for Research on Health and Social Care Management (CERGAS)Department of Pharmaceutical Sciences, Università del Piemonte OrientaleAbstract Early access programs (EAPs) generally refer to patient access to medicines/indications before marketing authorization, possibly extended to price and reimbursement approval. These programs include compassionate use, which is usually covered by pharmaceutical companies, and EAPs reimbursed by third-party payers. This paper aims at comparing EAPs in four European countries (France, Italy, Spain, UK) and providing empirical evidence on EAPs in Italy. The comparative analysis was conducted through a literature review (including scientific and grey literature), complemented by 30-min semi-structured interviews with local experts. The Italian empirical analysis employed data available on the National Medicines Agency website. Although EAPs are very different across countries, they exhibit some common features: (i) eligibility criteria refer to the absence of valid therapeutic alternatives and a presumed favourable risk–benefit profile; (ii) payers do not allocate a pre-determined budget to these programs; (iii) total spending on EAPs is unknown. The French EAPs seem to be the most structured, financed through social insurance, covering pre-marketing, post-marketing and pre-reimbursement phases and providing for data collection. Italy’s approach to EAPs has been varied, with several programs covered by different payers, including the cohort-based 648 List (for both early access and off-label use), the nominal-based 5% Fund, and Compassionate Use. Most applications to EAPs are from the Antineoplastic and immunomodulating drug class (ATC L). Some 62% of indications in the 648 List are either not under clinical development or have never been approved (pure off-label use). For those subsequently approved, most approved indications coincide with those covered through EAPs. Only the 5% Fund provides data on economic impact (€ 81.2 million in 2021; average cost per patient € 61.5K). Diverse EAPs are a possible source of inequalities in access to medicines across Europe. A harmonization of these programs, though difficult to achieve, could be modelled on the French EAPs and provide key advantages, not least of which a common effort to collect real-world data in parallel with clinical trials and clear separation between EAPs and off-label use programs.https://doi.org/10.1186/s40545-023-00570-zEarly access programsOff-labelMedicinesFranceItalySpain |
| spellingShingle | Alice Tarantola Monica Hildegard Otto Patrizio Armeni Francesco Costa Francesco Malandrini Claudio Jommi Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case Journal of Pharmaceutical Policy and Practice Early access programs Off-label Medicines France Italy Spain |
| title | Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case |
| title_full | Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case |
| title_fullStr | Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case |
| title_full_unstemmed | Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case |
| title_short | Early access programs for medicines: comparative analysis among France, Italy, Spain, and UK and focus on the Italian case |
| title_sort | early access programs for medicines comparative analysis among france italy spain and uk and focus on the italian case |
| topic | Early access programs Off-label Medicines France Italy Spain |
| url | https://doi.org/10.1186/s40545-023-00570-z |
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