Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries
Background: The experience of Kymriah® and Yescarta® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. Objective: To provide an overvie...
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| Format: | Article |
| Language: | English |
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MDPI AG
2020-01-01
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| Series: | Journal of Market Access & Health Policy |
| Subjects: | |
| Online Access: | http://dx.doi.org/10.1080/20016689.2020.1715536 |
| _version_ | 1797227105888501760 |
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| author | Jesper Jørgensen Eve Hanna Panos Kefalas |
| author_facet | Jesper Jørgensen Eve Hanna Panos Kefalas |
| author_sort | Jesper Jørgensen |
| collection | DOAJ |
| description | Background: The experience of Kymriah® and Yescarta® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. Objective: To provide an overview of the reimbursement schemes used for Kymriah® and Yescarta® in France, Germany, Italy, Spain, and the UK (EU5) as per the final quarter of 2019; to identify challenges and derive learnings for future product launches. Methodology: Secondary research, complemented by primary research with key market access stakeholders. Findings: Kymriah® and Yescarta® have relatively uniform list prices across the EU5, and are reimbursed according to their marketing authorisations. In France and the UK, reimbursement is on the condition of collecting additional data (at the cohort level) and subject to future reassessments; elsewhere, rebates (Germany) or staged payments (Italy and Spain) are linked to individual patient outcomes. Conclusions: The experience of Kymriah® and Yescarta® shows an increased appetite for outcomes-based reimbursement (OBR) in the EU5, with notably novel approaches applied in Italy and Spain (outcomes-based staged payments). Thus, real-world evidence (RWE) has become an increasingly powerful lever for demonstrating the value of health benefits in the clinical setting. |
| first_indexed | 2024-04-24T14:35:31Z |
| format | Article |
| id | doaj.art-8aa6054ae94a4c32955596128ead4211 |
| institution | Directory Open Access Journal |
| issn | 2001-6689 |
| language | English |
| last_indexed | 2024-04-24T14:35:31Z |
| publishDate | 2020-01-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Market Access & Health Policy |
| spelling | doaj.art-8aa6054ae94a4c32955596128ead42112024-04-03T00:39:41ZengMDPI AGJournal of Market Access & Health Policy2001-66892020-01-018110.1080/20016689.2020.17155361715536Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countriesJesper Jørgensen0Eve Hanna1Panos Kefalas2Cell and Gene Therapy CatapultCreativ-CeuticalCell and Gene Therapy CatapultBackground: The experience of Kymriah® and Yescarta® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. Objective: To provide an overview of the reimbursement schemes used for Kymriah® and Yescarta® in France, Germany, Italy, Spain, and the UK (EU5) as per the final quarter of 2019; to identify challenges and derive learnings for future product launches. Methodology: Secondary research, complemented by primary research with key market access stakeholders. Findings: Kymriah® and Yescarta® have relatively uniform list prices across the EU5, and are reimbursed according to their marketing authorisations. In France and the UK, reimbursement is on the condition of collecting additional data (at the cohort level) and subject to future reassessments; elsewhere, rebates (Germany) or staged payments (Italy and Spain) are linked to individual patient outcomes. Conclusions: The experience of Kymriah® and Yescarta® shows an increased appetite for outcomes-based reimbursement (OBR) in the EU5, with notably novel approaches applied in Italy and Spain (outcomes-based staged payments). Thus, real-world evidence (RWE) has become an increasingly powerful lever for demonstrating the value of health benefits in the clinical setting.http://dx.doi.org/10.1080/20016689.2020.1715536health technology assessment (hta)reimbursementmarket accessgene therapychimeric antigen receptor (car) t-cell therapyoncologyhaematology |
| spellingShingle | Jesper Jørgensen Eve Hanna Panos Kefalas Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries Journal of Market Access & Health Policy health technology assessment (hta) reimbursement market access gene therapy chimeric antigen receptor (car) t-cell therapy oncology haematology |
| title | Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries |
| title_full | Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries |
| title_fullStr | Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries |
| title_full_unstemmed | Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries |
| title_short | Outcomes-based reimbursement for gene therapies in practice: the experience of recently launched CAR-T cell therapies in major European countries |
| title_sort | outcomes based reimbursement for gene therapies in practice the experience of recently launched car t cell therapies in major european countries |
| topic | health technology assessment (hta) reimbursement market access gene therapy chimeric antigen receptor (car) t-cell therapy oncology haematology |
| url | http://dx.doi.org/10.1080/20016689.2020.1715536 |
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