Journal of cancer policy · 2026
International Regulatory Status and Reimbursement Recommendations of Haematological Malignancy Drugs Withdrawn from the FDA Accelerated Approval Pathway.
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Mulsant Sophie S, Kesselheim Aaron S, Naci Huseyin
Аннотация
BACKGROUND: The US Food and Drug Administration (FDA) can grant earlier access to promising therapies through its accelerated approval pathway, conditional on post-authorisation trials confirming clinical benefit. Between 1 January 2010 and 31 December 2024, the FDA granted accelerated approval to 59 haematological malignancy drug indications, 13 of which were subsequently withdrawn because of unconfirmed clinical benefit or safety concerns.
METHODS: We assessed the regulatory status and reimbursement recommendations for these 13 FDA-withdrawn drug indications in Australia, Canada, and England using publicly available regulatory and health technology assessment (HTA) data.
RESULTS: Eight of the 13 FDA-withdrawn drug indications were approved in at least one comparison jurisdiction, resulting in 14 marketing authorisations overall. Three of 14 were subsequently withdrawn. As of June 2025, 11 authorisations remained active, of which only five had received positive reimbursement recommendations.
CONCLUSION: Many haematological malignancy drug indications withdrawn from FDA accelerated approval remain authorised in other jurisdictions, with a minority continuing to receive positive reimbursement recommendations. Differences in regulatory and reimbursement decisions across jurisdictions may reflect variation in pathway use, post-authorisation evidence requirements, authorised indications, and interpretation of evidence.
POLICY SUMMARY: The continued authorisation of haematological malignancy drug indications in Australia, Canada, and England following FDA withdrawal of comparable indications has important implications for patients and health systems. Continued approval for the indication may facilitate use of the drug in patients with refractory disease and few alternatives, but may also expose patients to treatments with no clear evidence of clinical benefit or for which safety concerns altered the benefit-risk assessment. Continued public reimbursement under such conditions could also lead to unnecessary spending for health systems. Major new evidence or regulatory action in a comparable jurisdiction should prompt proportionate reassessment of local authorisation and reimbursement decisions, with consideration of differences in the authorised indication and applicable evidence.
METHODS: We assessed the regulatory status and reimbursement recommendations for these 13 FDA-withdrawn drug indications in Australia, Canada, and England using publicly available regulatory and health technology assessment (HTA) data.
RESULTS: Eight of the 13 FDA-withdrawn drug indications were approved in at least one comparison jurisdiction, resulting in 14 marketing authorisations overall. Three of 14 were subsequently withdrawn. As of June 2025, 11 authorisations remained active, of which only five had received positive reimbursement recommendations.
CONCLUSION: Many haematological malignancy drug indications withdrawn from FDA accelerated approval remain authorised in other jurisdictions, with a minority continuing to receive positive reimbursement recommendations. Differences in regulatory and reimbursement decisions across jurisdictions may reflect variation in pathway use, post-authorisation evidence requirements, authorised indications, and interpretation of evidence.
POLICY SUMMARY: The continued authorisation of haematological malignancy drug indications in Australia, Canada, and England following FDA withdrawal of comparable indications has important implications for patients and health systems. Continued approval for the indication may facilitate use of the drug in patients with refractory disease and few alternatives, but may also expose patients to treatments with no clear evidence of clinical benefit or for which safety concerns altered the benefit-risk assessment. Continued public reimbursement under such conditions could also lead to unnecessary spending for health systems. Major new evidence or regulatory action in a comparable jurisdiction should prompt proportionate reassessment of local authorisation and reimbursement decisions, with consideration of differences in the authorised indication and applicable evidence.