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BOSTON, October 31, 2024 – The Institute for Clinical and Economic Review (ICER) today posted its revised Evidence Report assessing the comparative clinical effectiveness and value of tabelecleucel (“tab-cel”®, Pierre Fabre) for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD).
“EBV+ PTLD is a rare and often fatal cancer associated with solid organ transplant and stem cell transplant,” said ICER’s Vice President of Research Foluso Agboola, MBBS, MPH. “Unfortunately, approximately half of EBV+ PTLD cases are refractory to initial treatment or relapse, resulting in a poor prognosis. The limited evidence on tabelecleucel suggests that it provides important clinical benefits in patients with relapsed refractory EBV+ PTLD, extending survival for patients who otherwise do not usually survive beyond a few weeks to months, with few harms.”
This Evidence Report will be reviewed at a virtual public meeting of the New England CEPAC on November 14, 2024. The New England CEPAC is one of ICER’s three independent evidence appraisal committees comprising medical evidence experts, practicing clinicians, methodologists, and leaders in patient engagement and advocacy.
Register here to watch the live webcast of the virtual meeting.
A draft version of this report was previously open for a four-week public comment period. The updated Evidence Report and voting questions reflect changes made based on comments received from patient groups, clinicians, drug manufacturers, and other stakeholders. Detailed responses to public comments can be found here.
Key Clinical Findings
Without treatment, relapsed/refractory EBV+ PTLD has a poor prognosis. Treatment with tabelecleucel appears to induce partial or complete remission in a substantial proportion of patients with relapsed/refractory EBV+ PTLD, and extends survival for patients who otherwise do not usually survive beyond a few weeks to months.
Additionally, the safety profile is reassuring, particularly with respect to severe adverse events, though longer-term safety data are still needed. Thus, despite data limitations, given the poor prognosis with usual care and the magnitude of benefits of tabelecleucel and few reported harms, we have a high certainty of substantial net health benefit (“A”) compared with usual care.
Key Cost-Effectiveness Findings
Tabelecleucel has not yet been approved by the FDA for EBV+ PTLD, and the manufacturers have not announced a US price for the therapy if approved.
ICER has calculated a health benefit price benchmark (HBPB) to be between $143,900 to $273,700 per treatment cycle.
ICER’s HBPB is a price range suggesting the highest US price a manufacturer should charge for a treatment, net of all rebates and discounts, based on the amount of improvement in overall health patients receive from that treatment, when a higher price would cause disproportionately greater losses in health among other patients in the health system due to rising overall costs of health care and health insurance. In short, it is the top price range at which a health system can reward innovation and better health for patients without doing more harm than good.
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