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BACKGROUND: Eli Lilly and the China Primary Health Care Foundation are currently implementing a patient assistance program (PAP) in China, which allows first-line nonsquamous non-small-cell lung cancer (NSCLC) patients who complete four cycles of pemetrexed induction therapy to receive free, continuous pemetrexed maintenance therapy. OBJECTIVE: To estimate the cost-effectiveness of pemetrexed maintenance therapy vs basic standard care (BSC) and the economic impacts of providing a PAP for pemetrexed maintenance therapy to NSCLC patients who have completed pemetrexed induction therapy in a Chinese health care setting. METHODS: We developed a novel decision-analytic model to evaluate the long-term costs and clinical efficacy of pemetrexed plus BSC vs BSC alone. We utilized a three-state (progression-free survival, progressed disease, and dead) partition survival model for both the clinical and economic aspects of the analysis. Cost and health utility estimates were derived from the literature. We performed a scenario analysis to estimate the real-world impact of introducing the PAP in China by comparing the use of the PAP vs non-PAP. Model uncertainty was evaluated using one-way and multivariate probabilistic sensitivity analysis. RESULTS: Compared to BSC, pemetrexed plus BSC resulted in a gain of 0.22 years of life (95% credible range [CR]: 0.04-0.46) and 0.13 quality-adjusted life years (95% CR: 0.04-0.26) per patient, at an increased cost of $28,105 (95% CR: -$22,720 to $48,646) without a PAP and $3,068 (95% CR: -$1,263 to $9,163) with a PAP. The incremental cost-effectiveness ratio for pemetrexed plus BSC vs BSC alone was cost-prohibitive at $222,700 for non-PAP, but cost-effective at $24,319 with a PAP. CONCLUSION: Our study suggests that maintenance pemetrexed therapy following pemetrexed induction for patients with advanced NSCLC is likely to be highly non-cost-effective in the absence of a PAP, but the pending implementation of the PAP promises to make it cost-effective, with a >90% probability of cost-effectiveness at a Chinese willingness-to-pay threshold per quality-adjusted life year.
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The "big data" era represents an exciting opportunity to utilize powerful new sources of information to reduce clinical and health economic uncertainty on an individual patient level. In turn, health economic outcomes research (HEOR) practices will need to evolve to accommodate individual patient-level HEOR analyses. We propose the concept of "precision HEOR", which utilizes a combination of costs and outcomes derived from big data to inform healthcare decision-making that is tailored to highly specific patient clusters or individuals. To explore this concept, we discuss the current and future roles of HEOR in health sector decision-making, big data and predictive analytics, and several key HEOR contexts in which big data and predictive analytics might transform traditional HEOR into precision HEOR. The guidance document addresses issues related to the transition from traditional to precision HEOR practices, the evaluation of patient similarity analysis and its appropriateness for precision HEOR analysis, and future challenges to precision HEOR adoption. Precision HEOR should make precision medicine more realizable by aiding and adapting healthcare resource allocation. The combined hopes for precision medicine and precision HEOR are that individual patients receive the best possible medical care while overall healthcare costs remain manageable or become more cost-efficient.
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INTRODUCTION: Postherpetic neuralgia (PHN), a type of peripheral neuropathic pain (pNeP), is the most common complication of herpes zoster. The objective of this analysis was to determine the cost-effectiveness of pregabalin compared with gabapentin in pNeP and PHN in China. METHODS: We developed a China-localized 12-week simulation model to determine the cost-effectiveness of pregabalin compared to gabapentin in 1000 patients with pNeP and PHN. We utilized a questionnaire of Chinese key opinion leaders to estimate the pre-treatment distribution of pain scores for pNeP and PHN. Treatment outcomes for pregabalin and gabapentin were acquired from the published literature. RESULTS: Treatment with pregabalin lead to 12-week decreases in pain scores of 0.6 (pNeP) and 0.7 (PHN) when compared to patients receiving gabapentin, at an incremental cost per additional day of mild/no pain of $45. The difference in mean days of no or mild pain, moderate pain, and severe pain was 8.8, -5.7, and -3.1, when comparing pregabalin and gabapentin, respectively. Pregabalin had more mean days with a >30% (7.71 days), 40% (8.97 days), and 50% reduction (9.97 days) in pain when compared with gabapentin. In the pNeP scenario, pregabalin was associated with a lower average pain score compared with gabapentin (3.91 vs. 4.55). The difference in mean days of no or mild pain, moderate pain, and severe pain was 9.39, -5.56, and -3.82, when comparing pregabalin and gabapentin, respectively. Pregabalin had more mean days with a >30% (8.77 days), 40% (9.81 days), and 50% reduction (10.55 days) in pain when compared with gabapentin. CONCLUSION: Pregabalin is an effective treatment for PHN and even for pNeP extensively, but at increased cost. It leads to improved outcomes including lower pain scores and more days with no or mild pain. FUNDING: Pfizer, China.