Quantification of the societal value of cultured human corneal endothelial cell transplantation

Introduction As a new therapeutic approach, regenerative medicine is expected to provide novel tools for the treatment of diseases that cannot be adequately addressed via the use of conventional therapies, and it has now been introduced into treatment strategies for ophthalmic diseases where its efficacy (i.e., improvement of not only corneal endothelial cell [CEC] density, but also best-corrected visual acuity [BCVA]) and safety for the treatment of corneal endothelial failure has recently been reported. While the value of new keratoplasty methods has traditionally been assessed primarily in terms of clinical outcomes and healthcare costs, successful treatment in the clinical setting using corneal regenerative medicine products may generate broader benefits for society. For example, restoration of BCVA may reduce the need for disability pension expenditure. With that said, such societal benefits have received relatively limited attention in the overall evaluation of regenerative medicine products. Hence, using a neltependocel (i.e., a human allogeneic CEC-processed product) as a case study, the purpose of this present study was to evaluate the potential societal value of cultured human CEC transplantation through cost offsets from avoided disability pension and break-even life expectancy. Methods In this retrospective study, the transplantation therapy-related BCVA improvement, disability pension grade change, benefits amount, medical cost, break-even life expectancy, abridged life tables, and potential cost savings were evaluated through a scenario-based analysis using publicly available data sources. Results As part of the scenario-based analysis, in 2 of 12 cases in a phase 3 clinical trial, BCVA improvement corresponding to a disability pension grade change from Grade 1 to non-eligibility criteria was observed. The annual Grade 1 disability basic pension benefit amount of the Japan Pension Service was ¥1,059,125 Japanese yen (i.e., $6746 U S. dollars). Initial therapy costs were equivalent to ¥10,153,730 ($64,673). The follow-up costs at 2-weeks and 1-, 3-, and 6-months postoperative were ¥10,967 ($70), ¥11,534 ($73), ¥12,101 ($77), and ¥13,274 ($85), respectively. The annual follow-up costs after 6-months postoperative were estimated to be ¥26,548 ($169). The break-even life expectancy was estimated to be 9.88 years. In cases where the disability pension grade improved, even at 65 years of age, which corresponds to the old-age (elderly) population, the potential cost savings were estimated to be ¥9,902,668 ($63,074) for male and ¥14,972,621 ($95,367) for female. Conclusions The findings in this study through a scenario-based analysis reveal that the surgical transplantation of neltependocel, a novel regenerative-medicine treatment for corneal endothelial failure, is estimated to potentially result in reductions in disability pension expenditures that exceed the initial and subsequent follow-up costs.