Value in Health最新文献

{"title":"Evidence Quality and Health Technology Assessment Outcomes in Reappraisals of Drugs for Rare Diseases in Germany.","authors":"Lea A Wiedmann, John A Cairns, Ellen Nolte","doi":"10.1016/j.jval.2024.07.012","DOIUrl":"10.1016/j.jval.2024.07.012","url":null,"abstract":"<p><strong>Objectives: </strong>Evidence on reappraisals of health technologies in Germany is limited, and for rare disease treatments (RDTs), the Federal Joint Committee follows different processes (limited or regular), depending on whether an annual revenue threshold has been exceeded. Our objective is to better understand (re)appraisal processes and their outcomes for RDTs in Germany.</p><p><strong>Methods: </strong>We analyzed appraisal documents of 55 RDT indications for which an initial appraisal and a reappraisal were conducted between 2011 and 2023. We extracted information for the type of evidence, the risk of bias, the availability of additional evidence, and the change in the maturity of survival data as proxies for evidence quality. Specifically, we reviewed the reasons for conducting reappraisals, examined how evidence quality and the clinical benefit rating (CBR) differed between initial appraisals and reappraisals, and explored the association between evidence quality and (1) the CBR and (2) the change in the CBR after reappraisal.</p><p><strong>Results: </strong>Most reappraisals were conducted because the annual revenue threshold was exceeded or the initial appraisal resolution was time limited. Almost all initial appraisals used the limited process, whereas the majority of reappraisals used the regular process. The CBR increased in only 9 and decreased in 21 of 55 reappraisals. There was some evidence that reappraisals with an accepted randomized controlled trial were significantly more likely to achieve a higher CBR.</p><p><strong>Conclusions: </strong>Findings confirmed that reasons and processes for conducting reappraisals of RDTs in Germany differ. Further, high CBRs in reappraisals were not common and evidence quality in initial appraisals and reappraisals was limited.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879522","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Informing the United States Medicare Drug Price Negotiation for Apixaban and Rivaroxaban: Methodological Considerations for Value Assessments Many Years After Launch.","authors":"Marina Richardson, Abigail C Wright, Jeffrey A Tice, David M Rind, Matt Seidner, Sarah Emond, Steven D Pearson","doi":"10.1016/j.jval.2024.07.011","DOIUrl":"10.1016/j.jval.2024.07.011","url":null,"abstract":"<p><strong>Objectives: </strong>To demonstrate how health technology assessment methods can be used to support Medicare's price negotiations for apixaban and rivaroxaban.</p><p><strong>Methods: </strong>Following the statutory outline of evidence that will be considered by Medicare, we conducted a systematic literature review, network meta-analyses, and decision analyses to evaluate the health outcomes and costs associated with apixaban and rivaroxaban compared with warfarin and dabigatran for patients with nonvalvular atrial fibrillation. Our methods inform discussions about the therapeutic impact of apixaban and rivaroxaban and suggest price premiums above their therapeutic alternatives over a range of cost-effectiveness thresholds.</p><p><strong>Results: </strong>Network meta-analyses found apixaban resulted in a lower risk of major bleeding compared with warfarin and dabigatran and a lower risk of stroke/systemic embolism compared with warfarin but not compared with dabigatran. Rivaroxaban resulted in a lower risk of stroke/systemic embolism versus warfarin but not dabigatran, and there was no difference in major bleeding. Decision-analytic modeling of apixaban suggested annual price premiums up to $4350 above the price of warfarin and up to $530 above the price for dabigatran at cost-effectiveness thresholds up to $200 000 per equal value of life-years gained. Analyses of rivaroxaban showed an annual price premium of up to $3920 above warfarin and no premium above that paid for dabigatran.</p><p><strong>Conclusions: </strong>Although health technology assessment is typically performed near the time of regulatory approval, with modifications, we produced comparative clinical and relative cost-effectiveness findings to help guide negotiations on a \"fair\" price for drugs on the market for over a decade.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879523","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"The Health Benefits, Costs, and Cost-Effectiveness of Ultraorphan Drugs.","authors":"Katherine A Clifford, A Alex Levine, Daniel E Enright, Peter J Neumann, James D Chambers","doi":"10.1016/j.jval.2024.07.005","DOIUrl":"10.1016/j.jval.2024.07.005","url":null,"abstract":"<p><strong>Objectives: </strong>To examine ultraorphan drugs in terms of incremental health, costs, and cost-effectiveness compared with more prevalent disease drugs.</p><p><strong>Methods: </strong>We identified Food and Drug Administration drug approvals from 1999 to 2019. For drugs approved for multiple indications, we considered each drug-indication pair separately. Utilizing Food and Drug Administration's orphan drug designation and US disease prevalence, we categorized drug-indication pairs as: ultraorphan (<10 000 patients), \"other\" orphan (≥10 000 and <200 000), and nonorphan (≥200 000). We searched the PubMed database for cost-effectiveness analyses and comparative effectiveness studies. We excluded manufacturer-funded studies. We extracted estimates of incremental health gains in terms of quality-adjusted life-years (QALYs) and incremental costs associated with drug-indication pairs compared with the standard of care at the time of their approval. We compared QALY gains, added costs, and incremental cost-effectiveness ratios (ICERs) using the Kruskal-Wallis, Mann-Whitney U (MWU), and Kolmogorov-Smirnov (KS) tests.</p><p><strong>Results: </strong>Median incremental QALYs, costs, and ICERs differed across nonorphan, \"other\" orphan, and ultraorphan categories (Kruskal-Wallis P < .01). Compared with nonorphan drugs, ultraorphan drugs had larger QALY gains (0.700 vs 0.050, MWU P < .01, KS P < .01), larger costs ($172 231 vs $3360, MWU P < .01, KS P < .01), and larger ICERs ($1 216 184/QALY vs $114 061/QALY, MWU P < .01, KS P <.01). Compared with \"other\" orphan drugs, ultraorphan drugs had larger QALY gains (0.700 vs 0.310, MWU P =.65, KS P =.32), larger costs ($172 231 vs $69 308, MWU P = .03, KS P = .03), and larger ICERs ($1 216 184/QALY vs $223 472/QALY, MWU P <.01, KS P <.01).</p><p><strong>Conclusions: </strong>Novel ultraorphan drugs typically offer larger incremental health gains than drugs for more prevalent diseases, but because of their substantial added costs, are typically less cost-effective.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Choice Consistency in Discrete Choice Experiments: Does Numeracy Skill Matter? (VIH-2023-0494.R2).","authors":"Mesfin G Genie, Nabin Poudel, Francesco Paolucci, Surachat Ngorsuraches","doi":"10.1016/j.jval.2024.07.001","DOIUrl":"https://doi.org/10.1016/j.jval.2024.07.001","url":null,"abstract":"<p><strong>Objectives: </strong>This study investigated the relationship between numeracy skills and choice consistency in discrete choice experiments (DCEs).</p><p><strong>Methods: </strong>A DCE was conducted to explore patients' preferences for kidney transplantation in Italy. Patients completed the DCE and answered three-item numeracy questions. A Heteroskedastic Multinomial Logit (HMNL) model was used to investigate the effect of numeracy on choice consistency.</p><p><strong>Results: </strong>Higher numeracy skills were associated with greater choice consistency, increasing the scale to 1.63 (p<0.001), 1.39 (p<0.001), and 1.18 (p<0.001) for patients answering 3/3, 2/3, and 1/3 questions correctly, respectively, compared to those with no correct answers. This corresponded to 63%, 39%, and 18% more consistent choices, respectively. Accounting for choice consistency resulted in varying willingness-to-wait (WTW) estimates for kidney transplant attributes. Patients with the lowest numeracy (0/3) were willing to wait approximately 42 months [95% CI: 29.37, 54.68] for standard infectious risk, compared to 33 months [95% CI: 28.48, 38.09] for 1/3, 28 months [95% CI: 25.13, 30.32] for 2/3, and 24 months [95% CI: 20.51, 27.25] for 3/3 correct answers. However, WTW differences for an additional year of graft survival and neoplastic risk were not statistically significant across numeracy levels. Supplementary analyses of two additional DCEs on COVID-19 vaccinations and rheumatoid arthritis, conducted online, supported these findings: higher numeracy skills were associated with more consistent choices across different disease contexts and survey formats.</p><p><strong>Conclusions: </strong>The findings suggested that combining patients with varying numeracy skills could bias WTW estimates, highlighting the need to consider numeracy in DCE data analysis and interpretation.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879518","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Linking Reimbursement to Patient Benefits for Advanced Therapy Medicinal Products and Other High-Cost Innovations: Policy Recommendations for Outcomes-Based Agreements in Europe.","authors":"Tuba Saygın Avşar, Jamie Elvidge, Claire Hawksworth, Juliet Kenny, Bertalan Németh, Marcelien Callenbach, Johanna Ringkvist, Dalia Dawoud","doi":"10.1016/j.jval.2024.07.007","DOIUrl":"10.1016/j.jval.2024.07.007","url":null,"abstract":"<p><strong>Objectives: </strong>Health technology assessment (HTA) of advanced therapy medicinal products (ATMPs), such as high-cost and one-time cell and gene therapies, is particularly challenging. Outcomes-based agreements (OBAs) are a potential solution to mitigate the risks while providing access to patients but are not widely used across Europe. This study aimed to develop policy recommendations to support the acceptability and implementation of OBAs in Europe.</p><p><strong>Methods: </strong>A policy sandbox approach was used to engage with stakeholders and explore how HTA organizations can support reimbursement decisions regarding OBAs for ATMPs. A panel of 38 experts from across the European region was convened in 2 workshops, representing payers, HTA organizations, patients, registries, and an industry trade body.</p><p><strong>Results: </strong>Policy recommendations were developed to support the appropriate consideration of OBAs for reimbursing highly uncertain technologies, such as ATMPs. If a positive HTA recommendation cannot be made at the proposed price, then a simple price discount reflecting the uncertainty is preferred over complex solutions such as OBAs. If an OBA is pursued, it should be designed collaboratively with all stakeholders to understand data collection feasibility and minimize burden to patients and providers. Payers are encouraged to approach OBAs as a tool for informed decision making, including a readiness to make negative reimbursement decisions based on unfavorable evidence.</p><p><strong>Conclusions: </strong>The study presents a policy framework for using OBAs in reimbursement decisions. OBAs must be carefully designed, focusing on appropriateness and the burden of implementation. The relevant authorities should be committed to making decisions in light of the resulting evidence.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141879524","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Examining the Impact of Structural Uncertainty Across 10 Type 2 Diabetes Models: Results From the 2022 Mount Hood Challenge.","authors":"James Altunkaya, Xinyu Li, Amanda Adler, Talitha Feenstra, Adam Fridhammar, Mi Jun Keng, Mark Lamotte, Phil McEwan, Andreas Nilsson, Andrew J Palmer, Jianchao Quan, Harry Smolen, An Tran-Duy, William Valentine, Michael Willis, José Leal, Philip Clarke","doi":"10.1016/j.jval.2024.06.010","DOIUrl":"10.1016/j.jval.2024.06.010","url":null,"abstract":"<p><strong>Objectives: </strong>The Mount Hood Diabetes Challenge Network aimed to examine the impact of model structural uncertainty on the estimated cost-effectiveness of interventions for type 2 diabetes.</p><p><strong>Methods: </strong>Ten independent modeling groups completed a blinded simulation exercise to estimate the cost-effectiveness of 3 interventions in 2 type 2 diabetes populations. Modeling groups were provided with a common baseline population, cost and utility values associated with different model health states, and instructions regarding time horizon and discounting. We collated the results to identify variation in predictions of net monetary benefit (NMB) and the drivers of those differences.</p><p><strong>Results: </strong>Overall, modeling groups agreed which interventions had a positive NMB (ie, were cost-effective), Although estimates of NMB varied substantially-by up to £23 696 for 1 intervention. Variation was mainly driven through differences in risk equations for complications of diabetes and their implementation between models. The number of modeled health states was also a significant predictor of NMB.</p><p><strong>Conclusions: </strong>This exercise demonstrates that structural uncertainty between different health economic models affects cost-effectiveness estimates. Although it is reassuring that a decision maker would likely reach similar conclusions on which interventions were cost-effective using most models, the range in numerical estimates generated across different models would nevertheless be important for price-setting negotiations with intervention developers. Minimizing the impact of structural uncertainty on healthcare decision making therefore remains an important priority. Model registries, which record and compare the impact of structural assumptions, offer one potential avenue to improve confidence in the robustness of health economic modeling.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-08","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141580983","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EQ-5D-5L Population Scores in Mainland China: Results From a Nationally Representative Survey 2021.","authors":"Qiang Yao, Fei Yang, Xiaodan Zhang, Jiale Qi, Haomiao Li, Yibo Wu, Chaojie Liu","doi":"10.1016/j.jval.2024.06.012","DOIUrl":"10.1016/j.jval.2024.06.012","url":null,"abstract":"<p><strong>Objectives: </strong>There is a lack of monitoring changes in the population scores of the most recent version, EQ-5D-5L, in mainland China. This study aimed to address this knowledge gap by assessing the EQ-5D-5L scores in mainland China using a nationally representative sample.</p><p><strong>Methods: </strong>Data were extracted from the 2021 Survey of Health Index of Chinese Families, which covered 31 provinces/autonomous regions/municipalities in mainland China. The survey used a multistage quota sampling strategy encompassing 120 prefecture-level cities. Quotas were allocated to each prefecture-level city in accordance with the 2020 China Population Census. This approach resulted in a final sample of 11 030 eligible questionnaires. The utility index (UI) and EuroQol Visual Analog Scale (EQ VAS) scores were reported for the entire sample (age-gender-urban/rural weighted) and by the characteristics of the study participants.</p><p><strong>Results: </strong>The study participants had a weighted mean UI of 0.939 (SD 0.135) and EQ VAS score of 80.19 (SD 18.39). The most commonly reported problem was anxiety/depression (26.37%), whereas self-care was the least reported problem (6.18%). Those who were male, were younger, lived without chronic conditions and disabilities, had higher levels of education, earned higher monthly household income, and were covered by basic medical insurance for urban employees had higher scores in both the UI and EQ VAS.</p><p><strong>Conclusion: </strong>This study revealed slightly lower UI scores despite a much higher drop in EQ VAS scores whereas China maintained minimum cases of COVID-19 in 2021 compared with the population norms recorded in 2019. Further studies are warranted to unveil the full impacts of COVID-19 outbreaks.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559833","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Do Behavioral Characteristics Influence the Breast Cancer Diagnosis Delay? Evidence From French Retrospective Data.","authors":"Christine Le Clainche, Antoine Marsaudon, Lise Rochaix, Baptiste Haon, Jean-Christophe Vergnaud","doi":"10.1016/j.jval.2024.06.008","DOIUrl":"10.1016/j.jval.2024.06.008","url":null,"abstract":"<p><strong>Objectives: </strong>This study aimed to analyze the behavioral determinants of breast cancer (BC) diagnosis delays in France. To do so, we investigated whether time discounting, risk tolerance, and personality traits influenced the BC diagnosis delay of patients.</p><p><strong>Methods: </strong>We used original retrospective data collected on 2 large online patient networks from 402 women diagnosed of BC. The BC diagnosis delay was measured by the difference between the date of diagnosis and the date of first symptoms. Time discounting and risk tolerance are measured with both self-reported questions and hypothetical lotteries. Personality traits are measured with the 10-item Big Five indicator. Ordinary least square and probit models were used to analyze whether these behavioral characteristics influenced the BC diagnosis delay.</p><p><strong>Results: </strong>Results showed that risk tolerance and time discounting were not significantly associated with the BC diagnosis delay. However, we found a longer diagnosis delay for women with a neuroticism personality trait (standardized coefficients ranged from 0.104 [P-value = .036] to 0.090 [P-value = .065]).</p><p><strong>Conclusions: </strong>Overall, our findings underline the need for an increased consideration of cancer screening public health policy for women with mental vulnerabilities since such vulnerabilities were found to be highly correlated with a neuroticism personality trait.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559831","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Generating Utilities for the Château-Santé Base: A Novel, Generic, and Patient-Centered Health-Outcome Measure.","authors":"Xin Zhang, Karin M Vermeulen, Nic J G M Veeger, Ruslan Jabrayilov, Paul F M Krabbe","doi":"10.1016/j.jval.2024.06.013","DOIUrl":"10.1016/j.jval.2024.06.013","url":null,"abstract":"<p><strong>Objectives: </strong>We have developed a new patient-centered, preference-based generic health-outcome measure, Château-Santé Base (CS-Base), which is based on a novel multiattribute preference response (MAPR) measurement framework. This study aimed to generate a first utility set for the CS-Base, making it suitable for use in health-economic evaluations.</p><p><strong>Methods: </strong>CS-Base comprises 12 health attributes: mobility, vision, hearing, cognition, mood, anxiety, pain, fatigue, social functioning, daily activities, self-esteem, and independence, each with 4 levels. Our methodology to generate utilities for the CS-Base was 2-fold. First, we derived coefficients from patient MAPR data to calculate CS-Base values. Subsequently, these were normalized to a 0.0 to 1.0 utility scale, in which 0.0 signifies dead. The dead position was estimated using general population data from a discrete choice experiment (discrete choice experiment + dead), using a division-value strategy, which localize the position of states better or worse than dead.</p><p><strong>Results: </strong>We analyzed MAPR data from 3222 patients and discrete choice experiment + dead data from 1995 respondents. All MAPR coefficients were negative, logically ordered, and significantly different from the reference level. The dead position was denoted by a division value of -148.385. Utility values spanned from -0.071 to 1.0, and only 53 of 16 777 216 states were deemed worse than dead.</p><p><strong>Conclusions: </strong>This study introduced the first CS-Base utility set, underlining a 2-step utility derivation method. This method, blending societal and patient views, surpasses traditional preference-based approaches, yielding firmer results. However, improvement of the normalization procedure is expected. Estimating CS-Base utilities is an ongoing process that gains precision over time.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559834","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Predicting the Population Health Economic Impact of Current and New Cancer Treatments for Colorectal Cancer: A Data-Driven Whole Disease Simulation Model for Predicting the Number of Patients with Colorectal Cancer by Stage and Treatment Line in Australia.","authors":"Koen Degeling, Yat Hang To, Karen Trapani, Sophy Athan, Peter Gibbs, Maarten J IJzerman, Fanny Franchini","doi":"10.1016/j.jval.2024.06.006","DOIUrl":"10.1016/j.jval.2024.06.006","url":null,"abstract":"<p><strong>Objectives: </strong>Effective healthcare planning, resource allocation, and budgeting require accurate predictions of the number of patients needing treatment at specific cancer stages and treatment lines. The Predicting the Population Health Economic Impact of Current and New Cancer Treatments (PRIMCAT) for Colorectal Cancer (CRC) simulation model (PRIMCAT-CRC) was developed to meet this requirement for all CRC stages and relevant molecular profiles in Australia.</p><p><strong>Methods: </strong>Real-world data were used to estimate treatment utilization and time-to-event distributions. This populated a discrete-event simulation, projecting the number of patients receiving treatment across all disease stages and treatment lines for CRC and forecasting the number of patients likely to utilize future treatments. Illustrative analyses were undertaken, estimating treatments across disease stages and treatment lines over a 5-year period (2022-2026). We demonstrated the model's applicability through a case study introducing pembrolizumab as a first-line treatment for mismatch-repair-deficient stage IV.</p><p><strong>Results: </strong>Clinical registry data from 7163 patients informed the model. The model forecasts 15 738 incident and 2821 prevalent cases requiring treatment in 2022, rising to 15 921 and 2871, respectively, by 2026. Projections show that over 2022 to 2026, there will be a total of 116 752 treatments initiated, with 43% intended for stage IV disease. The introduction of pembrolizumab is projected for 706 patients annually, totaling 3530 individuals starting treatment with pembrolizumab over the forecasted period, without significantly altering downstream utilization of subsequent treatments.</p><p><strong>Conclusions: </strong>PRIMCAT-CRC is a versatile tool that can be used to estimate the eligible patient populations for novel cancer therapies, thereby reducing uncertainty for policymakers in decisions to publicly reimburse new treatments.</p>","PeriodicalId":23508,"journal":{"name":"Value in Health","volume":null,"pages":null},"PeriodicalIF":4.9,"publicationDate":"2024-07-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141559815","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":2,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}