Journal of market access & health policy最新文献

Introduction: A non-medical switch is a change to a patient's medication regimen for reasons other than lack of clinical response, side-effects or poor adherence. Specialist physicians treat complex patients who may be vulnerable to non-medical switching. Objectives: To evaluate specialist physicians' perceptions regarding the frequency of non-medical switch requests, and the impact on their patients' outcomes and healthcare utilization. Methods: An online survey of randomly sampled physicians spending ≥10% of time providing patient care and having received ≥1 non-medical switch request during the prior 12-months. Results: Among 404 specialist physicians surveyed, non-medical switch requests were reported as very frequent or frequent by 35.0% of oncologists (for injectable cancer agents) and up to 80.3% of endocrinologists (for injectable anti-hyperglycemics). Respondents reported decreased medication effectiveness (25.0% of oncologists to 75.0% of dermatologists) and increased side-effects (32.5% of oncologists to 66.7% of psychiatrists). Most specialists reported very frequent or frequent increases in non-office visits (52.5% of oncologists to 75.3% of endocrinologists) and calls with pharmacies (57.5% of oncologists to 80.5% of rheumatologists) due to non-medical switching. Conclusions: Receipt of non-medical switching requests were common among specialist physicians. Non-medical switching may lead to negative effects on patient care and require increased healthcare utilization.

Objective: To understand the different methodologies used to elicit willingness to pay for health and the value of a statistical life year through surveys. Methodology: A systematic review of the literature was undertaken to identify studies using surveys to estimate either willingness to pay for health or the value of a statistical life year. Each study was reviewed and the study setting, sample size, sample description, survey administration (online or face to face), survey methodology, and results were extracted. The results of the studies were then compared to any published national guidelines of cost-effectiveness thresholds to determine their accuracy. Results: Eighteen studies were included in the review with 15 classified as willingness to pay and 3 value of a statistical life. The included studies covered Asia (n = 6), Europe (n = 4), the Middle East (n = 1), and North America (n = 5), with one study taking a global perspective. There were substantial differences in both the methodologies and the estimates of both willingness to pay and value of a statistical life between the different studies. Conclusion: Different methods used to elicit willingness to pay and the value of a statistical life year resulted in a wide range of estimates.

Background: Dementia has become a growing health-care problem in the rapidly ageing Japanese population. This study assesses the impact of dementia on quality of life, economic burden, and productivity loss. Objective: The objective of this study was to assess the impact of dementia on the Quality of Life (QoL), economic burden, and productivity loss among families living with dementia. Methods: An online survey was conducted among families who lived with relatives with dementia. Demographic data and information about health condition and costs of long-term care and treatment were collected. Participants were asked to answer the EuroQol (EQ-5D-5L) questionnaire, Zarit Burden Interview (ZARIT-8), and Work Productivity and Activity Impairment Questionnaire (WPAI). Multivariate analyses were conducted to assess factors associated with burden by families living with dementia. Results: Six hundred and thirty-five participants completed the survey. Of these participants, 50.5% were primary caregivers. Overall, 78.7% of dementia patients suffered from Alzheimer, and 43.9% needed long-term care. Compared to non-primary caregivers, primary caregivers had lower health utility scores (0.896 vs 0.873; p = 0.02), higher burden of caregiving (ZARIT-8: 21.1 vs 24.5; p < 0.0001), and higher overall work impairment (40.2% vs 20.8%; p < 0.0001), absenteeism (15.3% vs 5.7%; p < 0.0001), and presenteeism-related impairment (33.2% vs 17.3%; p < 0.0001). Conclusion: Families living with dementia caring for a person with dementia experience increased burden. Health policies related to dementia need to be considered not only for patients, but also for their families living with dementia to improve their QoL.

Background:Cost-effectiveness analysis (CEA) is the economic analysis method most commonly applied today in the context of replacing one treatment with a new one in a developed healthcare system to improve efficiency. CEA is often requested by local healthcare decision-makers to grant reimbursement. New preventative interventions, such as new vaccines, may however have much wider benefits inside and outside healthcare, when compared with treatment. These additional benefits include externalities on indirect clinical impact, reallocation of specific healthcare resources, improved quality of care, better productivity, better disease control, better fiscal revenues, and others. But these effects are sometimes difficult to integrate into a meaningful CEA result. They may appear as specific benefits for specific stakeholders, other than the stakeholders in healthcare. Objective: Based on a historical view about the application of economic assessments for vaccines our objective has been to make the inventory of who was/is interested in knowing the economic value of vaccines, in what those different stakeholders are likely to see the benefit from their perspective and how  were/are we able to measure those benefits and to report them well. Results: The historical view disclosed a limited interest in the economic assessment of vaccines at start, more than 50 years ago, that was comparable to the assessment of looking for more efficiency in new industries through optimization exercises. Today, we are exposed to a very rich panoply of different stakeholders (n= 16). They have their specific interest in many different facets of the vaccine benefit of which some are well known in the conventional economic analysis (n=9), but most outcomes are hidden and not enough evaluated and reported (n=26). Meanwhile we discovered that many different methods of evaluation have been explored to facilitate the measurement and reporting of the benefits (n=18). Conclusion: Our recommendation for future economic evaluations of new vaccines is therefore to find the right combination among the three entities of stakeholder type selection, outcome measure of interest for each stakeholder, and the right method to apply. We present at the end examples that illustrate how successful this approach can be.

Background: The experience of Kymriah® and Yescarta® provides real-world examples of how health-care systems approach and manage the reimbursement of one-off, high-cost, cell, and gene therapies, and the decision uncertainty and affordability challenges they present. Objective: To provide an overview of the reimbursement schemes used for Kymriah® and Yescarta® in France, Germany, Italy, Spain, and the UK (EU5) as per the final quarter of 2019; to identify challenges and derive learnings for future product launches. Methodology: Secondary research, complemented by primary research with key market access stakeholders. Findings: Kymriah® and Yescarta® have relatively uniform list prices across the EU5, and are reimbursed according to their marketing authorisations. In France and the UK, reimbursement is on the condition of collecting additional data (at the cohort level) and subject to future reassessments; elsewhere, rebates (Germany) or staged payments (Italy and Spain) are linked to individual patient outcomes. Conclusions: The experience of Kymriah® and Yescarta® shows an increased appetite for outcomes-based reimbursement (OBR) in the EU5, with notably novel approaches applied in Italy and Spain (outcomes-based staged payments). Thus, real-world evidence (RWE) has become an increasingly powerful lever for demonstrating the value of health benefits in the clinical setting.

Background: Access to biologic medicines (including biosimilars) across Europe is largely governed by a process of tendering conducted by health authorities. Over-reliance on treatment costs in awarding tenders has the potential to hinder competition and undermine the long-term sustainability of biosimilars. Objective: To assess the extent and impact of consideration of 'value-added services' (VAS) in tendering for biosimilars, we conducted a narrative review of published literature. Results: Findings from survey-based publications indicated that tendering practices for biosimilars are widely used, with cost being the main determinant of success and little detail being available on other criteria where these apply. Criteria (of therapeutic and technical interest) beyond price were included in one tendering specification for infliximab (originator and biosimilars), while a separate tender for the same product included VAS in the form of therapeutic drug monitoring, measurement of antibodies and calprotectin. Conclusions: Published evidence concerning inclusion of VAS in tendering for biosimilars is lacking. Development and implementation of standardized criteria and methods of assessment for tenders may avoid manufacturers facing segmented markets, encourage competition and the longer-term sustainability of biosimilars, and realize the healthcare system and patient benefits these treatments can bring.

Background: In many countries, Budget Impact (BI) informs reimbursement decisions. Evidence has shown that decision-makers have restricted access based on high BI estimates but studies show that BI estimates are often inaccurate. Objective: To assess the accuracy of BI estimations used for informing access decisions on oncology drugs in the Netherlands. Study Design: Oncology products for which European Medicines Agency Marketing Authorisation was granted between 1-1-2000 and 1-10-2017 were selected. Observed BI data were provided by FarmInform. BI estimates were extracted from the reimbursement dossiers of the Dutch Healthcare Institute. Products without an estimated BI in the reimbursement dossier were excluded. Accuracy is defined as the ratio observed BI/estimated BI. Setting: General community, the Netherlands. Results: Ten products were included in the base case analysis. Mean accuracy was 0.64 and observed BI deviated by more than 40% and 100% from the estimated BI for 4 and 5 products, respectively. For all products together, €141 million BI was estimated and €82 million BI was observed, a €59 million difference. Conclusions: The findings indicate that BI estimates for oncology drugs in the Netherlands are inaccurate. The role and use of BI in reimbursement decisions for these potentially life-saving drugs should therefore be considered carefully, as well as BI estimation methodology.

Background: Non-medical switching (NMS) is defined as switching to a clinically similar but chemically distinct medication for reasons apart from lack of effectiveness, tolerability or adherence. Objective: To update a prior systematic review evaluating the impact of NMS on outcomes. Data sources: An updated search through 10/1/2018 in Medline and Web of Science was performed. Study selection: We included studies evaluating ≥25 patients and measuring the impact of NMS of drugs on ≥1 endpoint. Data extraction: The direction of association between NMS and endpoints was classified as negative, positive or neutral. Data synthesis: Thirty-eight studies contributed 154 endpoints. The direction of association was negative (n = 48; 31.2%) or neutral (n = 91; 59.1%) more often than it was positive (n = 15; 9.7%). Stratified by endpoint type, NMS was associated with a negative impact on clinical, economic, health-care utilization and medication-taking behavior in 26.9%,41.7%,30.3% and 75.0% of cases; with a positive effect seen in 3.0% (resource utilization) to 14.0% (clinical) of endpoints. Of the 92 endpoints from studies performed by the entity dictating the NMS, 88.0%were neutral or positive; whereas, only 40.3%of endpoints from studies conducted separately from the interested entity were neutral or positive. Conclusions: NMS was commonly associated with negative or neutral endpoints and was seldom associated with positive ones.

Background and objective: We previously built a weighted Depressive Health State Index (DHSI) based on 29 parameters routinely collected in an automated healthcare database (AHDB). We now propose a linear DHSI (L-DHSI) which is easier to use and to replicate across AHDBs. Methods: A historical cohort of patients with ≥1 episode of depression was identified in the Clinical Practice Research Datalink (CPRD). The DHSI was calculated for each treated episode of depression. Validation was performed by using validated definitions of remission (proxy and Patient Health Questionnaire 9 or PHQ-9) and comparing the L-DHSI between subgroups. Reliability was assessed using Cronbach's alpha. Results: Between 1 January 2006 and 31 December 2012, 309,279 episodes of depression were identified in the CPRD. Remission was observed in 5% of the patients with lowest L-DHSI scores and in 78% of the patients with highest L-DHSI scores. Although less sensitive than the weighted DHSI, the L-DHSI was reliable and relatively easy of use. The L-DHSI was highly correlated to the weighted DHSI (Spearman coefficient 0.790, p < 0.001). Conclusion: The L-DHSI represents a good balance between reliability, usability, and reproducibility. In addition, the linearity of this index allows for an easier interpretation than the original weighted DHSI.

Background: Outcomes-based reimbursement (OBR) can reduce decision uncertainty and accelerate patient access to cell and gene therapies, however, OBR is rarely applied in practice in England. Oncology is the therapy area with the most cell and gene therapies in late-stage development, and the Systemic Anti-Cancer Therapy (SACT) dataset and The European Society for Blood and Marrow Transplantation (EBMT) registry are two data collection infrastructures that could potentially act as conduits for implementing OBR in cancer in England. Objective: To perform a gap analysis to identify the key requirements for upgrading the SACT and EBMT databases for the purposes of enabling OBR, and a top-level estimation of how much this upgrade may cost, using either a manual (staff-heavy) workaround or part automation (technology-heavy) approach. Methodology: The analysis of current data capture and gaps is informed by secondary research, while the assumptions and data used to derive the top-level cost estimates were informed by consensus-based primary research with experts in healthcare information technology (IT) systems integration and platform development, as well as experts of SACT and EBMT. Findings: In its current form, the SACT dataset in isolation is largely unfit for enabling OBR in oncology, whether through clinical, economic or humanistic outcomes. The EBMT registry has a greater potential; however, this relates to key clinical outcomes only, not economic or humanistic outcomes. Part automation requires a higher upfront investment than the manual workaround (~£1.8 million vs. ~£400k); however, lower annual costs (~£200 vs. ~£260k-£850k) mean that part automation becomes a more cost-effective approach over time. Conclusions: An appropriately automated and scalable data collection infrastructure should be implemented, with the ability to integrate clinical, economic and humanistic outcomes with healthcare cost data and payment systems, to enable OBR not only in cancer but also in other therapy areas.