Journal of market access & health policy最新文献

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.

Objective: To update the health economic evaluation of pirfenidone in the treatment of idiopathic pulmonary fibrosis (IPF) compared to all available alternatives strategies (Best supportive care - BSC and nintedanib), based on a cost-utility model previously validated by the CEESP's (French Committee for Economic Evaluation) in 2014. Methods: A standard Markov cohort model, adapted to French methodology guidelines, was used to simulate the therapeutic management and the course of IPF patients (including potential adverse events) using the collective perspective. Cost-effectiveness was evaluated regarding life years (LY); quality-adjusted life-years (QALY); average cumulative costs; the incremental cost-effectiveness ratio (ICER) expressed in cost per QALY gained. Data were retrieved from trials, meta-analysis, literature, health insurance and hospitalisation databases, and national tariffs. Results: Over 15 years, total costs accumulated in the pirfenidone strategy were estimated at €99,477 per patient, €104,610 in nintedanib, and €14,177 in Best Supportive Care (BSC). The total number of QALYs accumulated equalled 5.20 (6.91 LYs), 4.52 (5.98 LYs), and 3.79 (4.98 LYs), respectively. Pirfenidone was estimated to be dominant over nintedanib with incremental costs of -€5,133 and 0.67 more QALYs accumulated. Incremental cost versus BSC was €85,300 and 1,404 QALY gained. The cost-effectiveness ratio was estimated at 60,738€/QALY when compared to BSC. Conclusion: Pirfenidone is likely to be a cost-effective strategy compared to BSC and seems more efficient and less costly compared to nintedanib for the treatment of patients with IPF in France.

[This corrects the article DOI: 10.1080/20016689.2019.1618661.].

The importance of understanding the impact of disease and treatment on children's Health-Related Quality of Life (HRQoL) has given rise to an increasing use of child self-report and observer or proxy instruments. In this article, we review the status quo and challenges of HRQoL measurement specific to children under five. A number of HRQoL questionnaires exist for use with children and/or proxies, and both guidelines and reviews have been published on paediatric HRQoL. However, none address the challenges of measurement for children under five, for whom proxy measures should be used. In reality, there is significant heterogeneity in the cut-off age for self-report questionnaires. Recommendations are that proxies should be used for observable concepts, but not for concepts that require interpretation. Some research has been undertaken on dimensions/concepts in paediatric HRQoL questionnaires. However, no HRQoL models have been developed specifically for children, and heterogeneity in questionnaire dimensions underlines that there is no clear grasp of what HRQoL means in paediatric populations. There is a need to carry out research in order to develop theoretical models of HRQoL that are specific to children at different developmental stages, in order to evaluate and support new and existing measures for paediatric HRQoL and their use in clinical practice as well as clinical trials.

Background: Actinic keratosis (AK) is characterized by the occurrence of thick and scaly skin areas caused by damage from ultraviolet radiation. The management of AK aims to reduce lesions and prevent their recurrence by regular monitoring. French guidelines, last updated in 2009, reflect European guidelines for the management of face and scalp AK. However, they do not address all current, available options. Objective: To assess the management of face and scalp AK in French clinical practice. Methods: A two-part online questionnaire comprising a survey among French dermatologists and an analysis of patient medical records was performed to describe AK patients treated with topical therapy, patients' profiles, and characteristics of the affected lesion areas. Results: Decisions for topical treatments for face and scalp AK made by dermatologists were mainly driven by the lesion size. According to the last 10 patients they have seen, dermatologists were prescribing physical therapy in 53% of the cases, a combination of topical and physical therapy in 27% and topical only in 20%. Patient records revealed the average surface area targeted for treatment was 139 ± 113cm². Conclusions: Discrepancies between the guidelines on the treatment of face and scalp AK and clinical practice exist. Further research may help to standardize the treatment.