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BOSTON, December 16, 2024 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative clinical effectiveness of tabelecleucel (“tab-cel”®, Pierre Fabre) for the treatment of Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD).
ICER’s report on this therapy was the subject of the November 2024 public meeting of the New England CEPAC, one of ICER’s three independent evidence appraisal committees.
Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations
“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 do not respond 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.”
ICER’s Virtual Public Meeting: Voting Results on Clinical Effectiveness and Contextual Considerations
ICER assessed, and the independent appraisal committee voted on the evidence for people with relapsed/refractory EBV+ PTLD, who have received at least one prior therapy:
- All panelists (13-0) found that current evidence is adequate to demonstrate a net health benefit for tabelecleucel when compared to usual care.
Panel members also weighed potential benefits and disadvantages beyond the direct health effects and weighed special ethical priorities. Voting highlighted the following as particularly important for payers and other policymakers to note:
- There is substantial unmet need despite currently available treatments.
- The treatment is likely to produce substantial improvement in caregivers’ quality of life.
- The treatment offers a substantial opportunity to improve access to effective treatment by means of its mechanism of action or method of delivery.
ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money
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.
Consistent with ICER’s process, because there was no firm estimate of a potential launch price during the public meeting, the panel did not take a vote on the treatment’s long-term value for money.
Key Policy Recommendations
ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including clinical experts, patient advocates, and representatives from US payers — convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include:
- Manufacturers should develop and maintain robust patient assistance programs for treatments such as tabelecleucel, as the high cost of such treatments can lead to decreased access.
- Manufacturers should endeavor to include less frequent HLA types in tabelecleucel banks, paying particular attention to historically underrepresented minorities. The banks should aim to include enough HLA types to cover at least 95% of the population.
- All payers, particularly state Medicaid programs, should ensure that their referral networks are adequate for timely access to testing for EBV+ PTLD and treatment with tabelecleucel.
- The manufacturer and funding agencies should support research to investigate broader uses for tabelecleucel, including the optimal place in therapy for EBV+ PTLD.
ICER’s detailed set of policy recommendations is available in the Final Evidence Report and in the standalone Policy Recommendations document.
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