Journal of market access & health policy最新文献

{"title":"An integrated valuation model for payer and investor","authors":"M. Nuijten, S. Capri","doi":"10.1080/20016689.2022.2080631","DOIUrl":"https://doi.org/10.1080/20016689.2022.2080631","url":null,"abstract":"ABSTRACT Background In order to optimize positioning and associated drug price for both payer and investor, it is for a company essential to forecast the potential market access attractiveness for the new drug for different indications at the early onset of the clinical development program. This analysis must include the constraints from the perspective of the payer, but also the biotech companies, who require a minimum drug price to satisfy their investors. This paper aims to provide an Integrated Valuation Model for payer and investor, bridging concepts from health economics and economic valuation reflecting the perspectives of the payer and the investor for a drug in early clinical development phase. The concept is illustrated for a new hypothetical drug (Product X) in advanced breast cancer in 1-line, 2-line, and 3-line position. Methods The Integrated Valuation Model includes the outcomes of the budget impact model, pricing matrix model, and cost-effectiveness model reflecting the payer’s perspective. These models are interacted and linked with a discounted cash flow model in order to reflect also the economic value from the investor’s perspective. Results The maximum price in 1-line position is €269.7 for the payer and the minimum price is €14.7 for the investor, which are unit prices per administration corresponding with treatment regimens for the comparative treatments. In 2-line position, the maximum price is €274.1 for the payer and the minimum price for the investor increases to €184.5 for the investor because of the smaller market size in 2-line position, which leads to a smaller pricing corridor to satisfy both payer and investor. Consequently, Product X has market access attractiveness for both payer and investor in 1-line and 2-line position. However, the minimum price €942.7 in 3-line position for the investor is higher than the maximum price €283.3 for the payer, which means there is no market potential. Conclusion The practical strategic application of the Integrated Valuation Model is optimization of positioning and price of Product X. Hence, it can be a transparent tool in early-stage development of a compound based on upfront assessment of market access attractiveness for the payer and the investor.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-05-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"42746475","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Barriers in precision medicine implementation among Advanced Nonsquamous Cell Lung Cancer-patients: A Real-World Evidence Scenario.","authors":"Flavia A Duarte,&nbsp;Carlos Gil Ferreira,&nbsp;Rodrigo Dienstmann,&nbsp;Bruno L Ferrari,&nbsp;Matheus Costa E Silva,&nbsp;Pedro Nazareth A Junior,&nbsp;Paulo Guilherme de O Salles,&nbsp;Paulo Henrique C Diniz","doi":"10.1080/20016689.2022.2077905","DOIUrl":"https://doi.org/10.1080/20016689.2022.2077905","url":null,"abstract":"<p><strong>Background: </strong>Precision oncology has a prominent role in nonsquamous non-small cell lung cancer (nsNSCLC) treatment progress; however, its access in a real-world scenario might be limited.</p><p><strong>Objective: </strong>To investigate the time spent in nsNSCLC molecular profile evaluation and its influence on clinical decisions.</p><p><strong>Methods: </strong>nsNSCLC patients who underwent molecular testing in a private referral Brazilian center between November 2015 and February 2020 were identified. The interval from nsNSCLC diagnosis to the characterization of the molecular profile was determined. Other outcomes, focusing on the biomarker tissue journey, were also assessed.</p><p><strong>Results: </strong>In this cohort (<i>n</i> = 78), the median time between the advanced nsNSCLC diagnosis and biomarker characterization was 40.5 days (range, 29.5-68.5). The median interval between the diagnosis and the test request was longer than the interval between the request and the results (respectively 29.0 <i>versus</i> 12.0 days; <i>p</i> < 0.001). At the treatment initiation, 51% (36/71) of the patients who received any systemic therapy did not have their driver mutations panel results available. But on these, 42% (15/36) had a targetable alteration identified later on. Among patients harboring a targetable alteration, only 46% (<i>n</i> = 13/28) received a tyrosine kinase inhibitor (TKI) as first-line therapy. The median time to the TKI initiation was even longer than the median time to all treatment initiation (92.0 <i>versus</i> 40.0 days).</p><p><strong>Conclusions: </strong>Our data show a long median time from advanced nsNSCLC diagnosis and the availability of the biomarker testing in medical practice, which impacted the choice of a non-personalized therapy as the first-line.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":" ","pages":"2077905"},"PeriodicalIF":0.0,"publicationDate":"2022-05-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/1b/4d/ZJMA_10_2077905.PMC9639562.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"40454218","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Can domestic medical tourism contribute to healthcare equity? A commentary","authors":"Michelle Rydback, A. Hyder, G. Macassa, Clara Simonsson","doi":"10.1080/20016689.2022.2061241","DOIUrl":"https://doi.org/10.1080/20016689.2022.2061241","url":null,"abstract":"ABSTRACT Pupose - This essay uses service marketing concept to discuss how domestic medical tourism (DMT) can contribute to healthcare equity in developed countries. Approach - The authors take up several vital issues. First, the potential benefits of DMT are outlined from a healthcare equity perspective; second, the challenges that DMT confronts in reaching its aim are identified; and finally, a few research areas are suggested. Finding - It is suggested that increased awareness about the healthcare service and proper service delivery are required to improve healthcare equity. Practical implication - This paper raises several research issues from service marketing to deal with delivery, communication, efficiency, and insurance practices regarding healthcare. Social implication - From a societal point of view, it explores how healthcare equity can be improved by DMT.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":" ","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-04-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"41780510","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cost-saving prediction model of transfer to palliative care for terminal cancer patients in a Japanese general hospital","authors":"Yukiko Hashimoto, A. Hayashi, Takashi Tonegawa, L. Teng, A. Igarashi","doi":"10.1080/20016689.2022.2057651","DOIUrl":"https://doi.org/10.1080/20016689.2022.2057651","url":null,"abstract":"ABSTRACT Background Although medical costs need to be controlled, there are no easily applicable cost prediction models of transfer to palliative care (PC) for terminal cancer patients. Objective Construct a cost-saving prediction model based on terminal cancer patients’ data at hospital admission. Study design Retrospective cohort study. Setting A Japanese general hospital. Patients A total of 139 stage IV cancer patients transferred to PC, who died during hospitalization from April 2014 to March 2019. Main outcome measure Patients were divided into higher (59) and lower (80) total medical costs per day after transfer to PC. We compared demographics, cancer type, medical history, and laboratory results between the groups. Stepwise logistic regression analysis was used for model development and area under the curve (AUC) calculation. Results A cost-saving prediction model (AUC = 0.78, 95% CI: 0.70, 0.85) with a total score of 13 points was constructed as follows: 2 points each for age ≤ 74 years, creatinine ≥ 0.68 mg/dL, and lactate dehydrogenase ≤ 188 IU/L; 3 points for hemoglobin ≤ 8.8 g/dL; and 4 points for potassium ≤ 3.3 mEq/L. Conclusion Our model contains five predictors easily available in clinical settings and exhibited good predictive ability.","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"10 1","pages":""},"PeriodicalIF":0.0,"publicationDate":"2022-03-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"59990895","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Increased reliance on physician assistants: an access-quality tradeoff?","authors":"Bhavneet Walia,&nbsp;Harshdeep Banga,&nbsp;David A Larsen","doi":"10.1080/20016689.2022.2030559","DOIUrl":"https://doi.org/10.1080/20016689.2022.2030559","url":null,"abstract":"Overview In recent years, Physician Assistants (PAs) have become an increasingly important class of medical practioners in the USA (U.S.) healthcare system. After physicians, PAs and Nurse Practitioners are the most skilled among the commonly observed types of medical practitioners, having earned a Masters Degree from an accredited medical sciences program. Further, PAs perform many of the same tasks as physicians within the U.S. healthcare system. According to the American Association of Physician Assistants, PAs commonly: ‘Take medical histories; Conduct physical exams; Diagnose and treat illness; Order and interpret tests; Develop treatment plans; Prescribe medication; Counsel on preventive care; Perform procedures; Assist in surgery; Make rounds in hospitals and nursing homes; Do clinical research.’ [1] These tasks can either be transferred from physicians to PAs or completed in physician-PA teams. As such, PAs can act as substitutes or complements for physicians within U.S. healthcare and other healthcare systems. More specifically, PAs can work without day-to-day physician supervision while performing physician-like tasks or in teams in which they are directly supervised by physicians [2]. Given that their tasks are highly related to those of U.S. physicians, it is important to characterize trends in the role and scale of PAs in the U.S. healthcare system. The number of PAs is growing at a rapid rate in U.S. healthcare systems [3]. The number of employed PAs in the U.S. is expected to grow by 39,300 or 31.3% between 2019 and 2029. This growth rate is well above the average rate of labor growth in the healthcare industry. By comparison, the projected growth rate for U.S. physician and surgeon positions over the same time period is 3.6%, with a projected 27,300 new physician/surgeon positions over that time. Figure 1 shows the beginning of this projected trend. These projections suggest that the ratio of physicians to PAs will decrease from 6:1 in 2019 to 4.7:1 in 2029. This rapid change can be linked to structural shifts in the U.S. healthcare systems, including increased demand attributable partly to the Affordable Care Act of 2010, an increased market concentration of for-profit health institutions that seek to maximize profit partly by reducing labor costs, and a fairly-substantial average pay gap between physicians and PAs, among others. Presently, we consider whether this shift will create a tradeoff between health care access and quality within U.S. healthcare. In 2019, median physician pay in the U.S. was $208,000 compared to $115,390 for Pas [3]. Consequently, the cost savings from increasing the proportion of PAs relative to physicians are substantial. The BLS projects that the number of U.S. PAs and physicians combined will expand to 944,500 by 2029. If this expansion were to be conducted while preserving the 6:1 physician-to-PA ratio observed in 2019, it would cost approximately $1.38 trillion more systemwide at current salaries, ","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"10 1","pages":"2030559"},"PeriodicalIF":0.0,"publicationDate":"2022-01-24","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/dd/81/ZJMA_10_2030559.PMC8788342.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39865401","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Psychometric validation of a patient-reported single-item assessment of 'Good Day Bad Day' in a neurogenic orthostatic hypotension population treated with droxidopa.","authors":"Clément François, Nicola Germain, Renata Majewska, Vanessa Taieb, L Arthur Hewitt, Steven Kymes","doi":"10.1080/20016689.2021.2010961","DOIUrl":"10.1080/20016689.2021.2010961","url":null,"abstract":"<p><strong>Background: </strong>Symptoms of neurogenic orthostatic hypotension (nOH), including lightheadedness/dizziness, presyncope, syncope, and falls, can lead to impaired functional ability and reduced quality of life. Because the severity and frequency of nOH symptoms fluctuate, it may be difficult for patients to accurately quantify the effect of symptoms on their daily lives using available outcome measures. A new single-item instrument, the 'Good Day Bad Day,' was developed, and its psychometric validity was assessed in patients with nOH.</p><p><strong>Methods: </strong>Data from a 6-month, prospective, observational cohort study of patients with nOH who were newly initiating droxidopa treatment were used. Patients were asked to quantify the number of good and bad days in the previous 7 days and responded to other validated patient-reported outcomes instruments. The concurrent and discriminant validities and the stability of the Good Day Bad Day instrument were assessed.</p><p><strong>Results: </strong>A total of 153 patients were included in the analysis (mean [SD] age, 62.3 [17] years). Change in the number of good days moderately correlated with improvements in other patient-reported outcomes (rho value range, -0.38 to -0.61). When data were examined categorically (low vs high symptom severity), the mean number of good days was higher in subgroups representing low symptom severity across measures at 1, 3, and 6 months (all <i>P</i> ≤ 0.01).</p><p><strong>Conclusions: </strong>The Good Day Bad Day instrument provided good discrimination at baseline and over time and may aid in assessment of the effects of nOH symptoms on patients.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"10 1","pages":"2010961"},"PeriodicalIF":0.0,"publicationDate":"2022-01-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/2d/4e/ZJMA_10_2010961.PMC8757596.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39702402","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pricing Zolgensma - the world's most expensive drug.","authors":"Mark Nuijten","doi":"10.1080/20016689.2021.2022353","DOIUrl":"https://doi.org/10.1080/20016689.2021.2022353","url":null,"abstract":"A heated discussion has recently broken out in Europe about the price of Zolgensma, ‘the most expensive drug ever’. The National Institute for Health and Care Excellence (NICE) approved Zolgensma in March this year, which is set to become the most expensive treatment ever approved by NICE. Zolgensma is a gene therapy medicine for treating spinal muscular atrophy (SMA), a serious and rare condition of the nerves that causes muscle wasting and weakness [1]. It is estimated that the drug will cost approximately €1.9 million per course of treatment [2]. Patients with SMA have a defect in a gene known as SMN1, which the body needs to make a protein essential for the normal functioning of nerves that control muscle movements. Zolgensma is a gene therapy containing a functional copy of this gene which, after injection, passes into the nerves from where it provides the correct gene to make enough of the protein and, thereby, restore nerve function [1]. At first impression, the price level of Zolgensma raises many understandable questions, because €1.9 million sounds exorbitantly high in the public domain (often driven by emotions and lack of specialised knowledge of the costs and risk of the development of a new pharmaceutical). However, there are many factors that may justify NICE’s decision to approve the intervention for use. In the Netherlands, since the debate in 2013 about the high price of medicines for Fabry and Pompe diseases, ‘expensive’ medicines are increasingly only reimbursed after tough price negotiations with the Ministry of Health [3]. This usually concerns medicines for the treatment of rare diseases, the so-called ‘orphan drugs’ such as Zolgensma. For example, it is estimated that only one in 11,000 children is born with SMA [4]. These price negotiations have since become a permanent and important part of the market access process for new ‘expensive’ orphan drugs, where expenditure weighed against patient suffering, a difficult and ethically difficult task for all parties [3]. The current choice for ‘expensive’ medicines is based on clinical and economic criteria, whereby in The Netherlands the Ministry of Health is willing to pay a maximum of €80,000 for each extra life year gained with perfect quality of life, the so-called qualityadjusted life year” (QALY). It often concerns orphan drugs which, due to their high price, have a ‘cost per QALY’ that is much higher than the Dutch threshold value of €80,000. However, the price per patient for an orphan drug is often much higher, because the fixed research and development (R&D) costs, which are not much different than the R&D costs for non-orphan drugs, are recouped on far fewer patients. For example, the reimbursement of Spinraza, the first effective drug for SMA, was also initially denied due to an excessively high ‘cost per QALY’ of €600,000. Finally, Spinraza became available for Dutch SMA patients in 2018 after much delay due to lengthy price negotiations resulting in a heavily enfor","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"10 1","pages":"2022353"},"PeriodicalIF":0.0,"publicationDate":"2021-12-29","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/7a/ea/ZJMA_10_2022353.PMC8725676.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39904881","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Burden of illness associated with pneumococcal infections in Japan - a targeted literature review.","authors":"Ataru Igarashi,&nbsp;Maki Ueyama,&nbsp;Koki Idehara,&nbsp;Mariko Nomoto","doi":"10.1080/20016689.2021.2010956","DOIUrl":"10.1080/20016689.2021.2010956","url":null,"abstract":"<p><strong>Introduction: </strong>Pneumococcal diseases (PDs) are among the leading causes of mortality and morbidity worldwide. However, the evidence on epidemiology, health economic, and patient-reported outcomes has not been systematically reviewed and published in Japan. This study aimed to assess the burden, treatment adherence and compliance, and serotype distribution associated with PDs in Japan.</p><p><strong>Method: </strong>One hundred and eight studies were identified between January 2005 and June 2020. The identified studies were mostly regional and with a limited scale, clinical settings, and populations.</p><p><strong>Results: </strong>In 2013-2017, invasive PD incidence rates were 4.98-9.47/100,000 in <4-year-olds, 0.36/100,000 in 5-14-year-olds, 0.46/100,000 in 15-64-year-olds, and 1.50-5.38/100,000 in the elderly. The incidence of invasive PDs in children decreased from 24.6/100,000 in 2008 to 10.7/100,000 in 2013 after the introduction of PCV7 and further declined to 10.3/100,000 in 2014 after PCV13 was introduced. From 2014, the prevalence of PCV13 serotypes decreased across all age groups along with a decrease of PPV23 serotypes, but an increase of PPV23 serotypes not included in PCV13 among adults and the elderly. No study reported health-related quality-of-life data for PDs. In children, direct costs were 340,905-405,978 JPY (3,099-3,691 USD) per pneumococcal bacteraemia, 767,447-848,255 JPY (6,977-7,711 USD) per pneumococcal meningitis, and 79,000 JPY (718 USD) per pneumococcal acute otitis media episodes. In adults and the elderly, the direct cost of pneumococcal pneumonia was 348,280-389,630 JPY (3,166-3,542 USD). The average hospital stay length was 7.2-31.9 days in children, 9.0 days in adults and 9.0-28.7 days in adults and the elderly.</p><p><strong>Conclusions: </strong>The epidemiological burden of PDs remains high in Japan, especially among children and the elderly with invasive PDs accounting for a very small proportion of all PDs. A significant impact of the PCV13 vaccine program was reported, while the PPV23's impact remains unclear. A substantial decrease in quality-adjusted life years in adults and the elderly and a high economic burden may exist.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"10 1","pages":"2010956"},"PeriodicalIF":0.0,"publicationDate":"2021-12-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/f5/6b/ZJMA_10_2010956.PMC8725729.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39904880","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Does supplementary health insurance play a role in the switching behaviour of citizens in the Netherlands?","authors":"Laurens Holst,&nbsp;Anne Brabers,&nbsp;Judith de Jong","doi":"10.1080/20016689.2021.2015863","DOIUrl":"https://doi.org/10.1080/20016689.2021.2015863","url":null,"abstract":"<p><strong>Background: </strong>Several healthcare systems have elements of managed competition in which citizens can choose between multiple insurers. In order for this principle to function properly, all citizens should have equal opportunities to switch insurer. Studies, conducted around 2015, have shown that the supplementary insurance policy is perceived by citizens as a barrier to switching, which could have negative consequences for the intended goals of the system.. We aim to explore whether a supplementary insurance policy still has a restraining role on the opportunity to switch among citizens in the Netherlands from 2015 to 2020. Furthermore, we will examine if the extensiveness of the supplementary insurance policy relates to the switching behaviour of citizens. This element has not been addressed in previous studies.</p><p><strong>Methods: </strong>We obtained information on the role of the supplementary health insurance policy in the switching behaviour of citizens by sending questionnaires, yearly in February from 2015-2020, to 1,500 members of the Dutch Health Care Consumer Panel (DHCCP) each year. As such, we were able to examine whether having a supplementary insurance policy plays a role in the decision of Dutch citizens to switch insurer. The response rates were consecutively from 2015 to 2020: 60% (n = 896), 47% (n = 703), 44% (n = 659), 50% (n = 751), 48% (n = 715), and 54% (n = 806).</p><p><strong>Results: </strong>Citizens with a supplementary insurance policy switch less often than citizens without one. The extensiveness of the supplementary insurance policy is significantly associated with the decision of citizens to switch insurer; the more extensive citizens are insured, the less often they switch. Additionally, our results show that every year a small group of citizens does not switch insurer because they are concerned that they will not be accepted for a supplementary insurance policy.</p><p><strong>Conclusions: </strong>Our results indicate that having a supplementary insurance policy holds citizens back from using their opportunity to switch. This contributes to the idea that having a supplementary insurance policy could be experienced by citizens as a barrier to switch. This raises questions about the extent to which the principle of managed competition in the Dutch healthcare system works as intended.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"10 1","pages":"2015863"},"PeriodicalIF":0.0,"publicationDate":"2021-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/e6/e5/ZJMA_10_2015863.PMC8676582.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39827520","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An overview of health technology assessments of gene therapies with the focus on cost-effectiveness models.","authors":"Michał Pochopień,&nbsp;Ewelina Paterak,&nbsp;Emilie Clay,&nbsp;Justyna Janik,&nbsp;Samuel Aballea,&nbsp;Małgorzata Biernikiewicz,&nbsp;Mondher Toumi","doi":"10.1080/20016689.2021.2002006","DOIUrl":"10.1080/20016689.2021.2002006","url":null,"abstract":"<p><strong>Background: </strong>Gene therapies can treat, prevent, or cure a disease by changing the expression of a person's genes. They are an innovative strategy for treating genetic disorders; however, they are still emerging on the market access and in the healthcare system. Health technology assessment (HTA) agencies have not yet elaborated any standardised approach for assessing gene therapies; therefore, significant differences can be seen during HTAs carried out in various countries. In this review, we focused on submitted economic models of gene therapies approved for use by the US FDA and EMA with the aim to provide a comprehensive summary of how selected HTA bodies assessed the cost-effectiveness of gene therapies. An additional objective was to examine and discuss differences in the methods used in economic models across countries and drugs.</p><p><strong>Methods: </strong>We identified economic models of gene therapies from six countries (NICE, IQWiG, SMC, HAS, CADTH, ICER) and focused on nine agents (Glybera, Imlygic, Strimvelis, Yescarta, Kymriah, Luxturna, Zynteglo, Zolgensma, Tecartus). Details of cost-utility evaluations and budget impact models were reviewed and extracted.</p><p><strong>Results: </strong>Overall, 983 publications were identified, and 17 studies were included for the analysis. Reviewed evaluations of gene therapies differed in terms of the study perspective, discounting, extrapolation of outcomes based on limited and immature data, time horizon, and adequate estimation of benefits in terms of quality-adjusted life-years. Methods of economic evaluations were in line with the current recommendations; however, long-term follow-up studies are still missing.</p><p><strong>Conclusions: </strong>Discrepancies in an economic evaluation of gene therapies between different HTA bodies are rooted in a lack of general assessment frameworks specific to gene therapies. Although challenges were resolved by adjustments to the currently used value assessment framework, new methodological approaches would be useful. In addition, to improve the methods and quality of an evaluation, further research would be valuable.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"2002006"},"PeriodicalIF":0.0,"publicationDate":"2021-11-13","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/9a/1c/ZJMA_9_2002006.PMC8592603.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39722116","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}