Journal of market access & health policy最新文献

Background: 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.

Methods: 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.

Results: 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 P ≤ 0.01).

Conclusions: 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.

Introduction: 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.

Method: 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.

Results: 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.

Conclusions: 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.

Background: 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.

Methods: 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).

Results: 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.

Conclusions: 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.

Background: 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.

Methods: 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.

Results: 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.

Conclusions: 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.

Objective: This study aims at investigating associations between COVID-19 mortality and SARS-COV-2 variants spread during the second wave of COVID-19 pandemic in Europe.

Methods: For 38 European countries, data on numbers of COVID-19 deaths, SARS-COV-2 variants spread through time using Nextstrain classification, demographic and health characteristics were collected. Cumulative number of COVID-19 deaths and height of COVID-19 daily deaths peak during the second wave of the pandemic were considered as outcomes. Pearson correlations and multivariate generalized linear models with selection algorithms were used.

Results: The average proportion of B.1.1.7 variant was found to be a significant predictor of cumulative COVID-19 deaths within two months before the peak and between 1 January-25 February 2021, as well as of the deaths peak height considering proportions during the second wave and the pre-peak period. The average proportion of EU2 variant (S:477 N) was a significant predictor of cumulative COVID-19 deaths in the pre-peak period.

Conclusions: Our findings suggest that spread of a new variant of concern B.1.1.7 had a significant impact on mortality during the second wave of COVID-19 pandemic in Europe and that proportions of EU2 and B.1.1.7 variants were associated with increased mortality in the initial phase of that wave.

Introduction: Outcome-based agreements (OBAs) are occasionally deployed to relieve the burden of high drug prices on healthcare budgets. However, it is not clear when manufacturers are willing to collaborate in establishing such agreements. Therefore, we explored the feasibility of OBAs from the manufacturer's point of view.

Methods: Dutch market-access experts from eight major pharmaceutical companies, globally active in the field of oncology, were interviewed. Opinions were compiled, and interviewees and their colleagues were then given the chance to review the manuscript for additional comments.

Results: Most interviewees believe that OBAs can be useful in providing access to off-label use of authorised medicines, especially when no alternative treatment is available for seriously ill patients. For the licenced indications, manufacturers seem to be more inclined to collaborate when there is a potential incentive to improve market-access (e.g., if the product is not used because of concerns regarding its effectiveness). However, manufacturers are less likely to collaborate when there are greater financial risks for the company. Further concerns were definition of outcome or performance, the impact of compliance on the effectiveness of a drug, administrative burden, uncertainty regarding revenue recognition and the challenges of reimbursing combination therapies.

Discussion: Market-access interviewees were generally positive about OBAs, however they were more reluctant towards OBAs for registered indications with low response-rate. The definition of performance or outcome and its clinical relevance and validity, the feasibility of OBAs and their administrative burden are relevant aspects that need to be addressed in advance. Ideally, countries should collaborate to share the outline of OBAs and create shared databases to accumulate evidence.