Journal of market access & health policy最新文献

{"title":"Modeling long-term health and economic implications of new treatment strategies for Parkinson's disease: an individual patient simulation study.","authors":"Conor Chandler,&nbsp;Henri Folse,&nbsp;Peter Gal,&nbsp;Ameya Chavan,&nbsp;Irina Proskorovsky,&nbsp;Conrado Franco-Villalobos,&nbsp;Yunyang Yang,&nbsp;Alex Ward","doi":"10.1080/20016689.2021.1922163","DOIUrl":"https://doi.org/10.1080/20016689.2021.1922163","url":null,"abstract":"<p><p><b>Background</b>: Simulation modeling facilitates the estimation of long-term health and economic outcomes to inform healthcare decision-making. <b>Objective</b>: To develop a framework to simulate progression of Parkinson's disease (PD), capturing motor and non-motor symptoms, clinical outcomes, and associated costs over a lifetime. <b>Methods</b>: A patient-level simulation was implemented accounting for individual variability and interrelated changes in common disease progression scales. Predictive equations were developed to model progression for newly diagnosed patients and were combined with additional sources to inform long-term progression. Analyses compared a hypothetical disease-modifying therapy (DMT) with a standard of care to explore the drivers of cost-effectiveness. <b>Results</b>: The equations captured the dependence between the various measures, leveraging prior values and rates of change to obtain realistic predictions. The simulation was built upon several interrelated equations, validated by comparison with observed values for the Movement Disorder Society Unified PD Rating Scale (MDS-UPDRS) and UPDRS subscales over time. In a case study, disease progression rates, patient utilities, and direct non-medical costs were drivers of cost-effectiveness. <b>Conclusions</b>: The developed equations supported the simulation of early PD. This model can support conducting simulations to inform internal decision-making, trial design, and strategic planning early in the development of new DMTs entering clinical trials.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1922163"},"PeriodicalIF":0.0,"publicationDate":"2021-06-03","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1922163","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39089623","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":"Impact of health technology assessment on prescribing patterns of inhaled fixed-dose combination triple therapy in chronic obstructive pulmonary disease.","authors":"Jennifer Cook,&nbsp;Chloe Bloom,&nbsp;Jen Lewis,&nbsp;Zoe Marjenberg,&nbsp;Jaime Hernando Platz,&nbsp;Sue Langham","doi":"10.1080/20016689.2021.1929757","DOIUrl":"https://doi.org/10.1080/20016689.2021.1929757","url":null,"abstract":"<p><p><b>Background</b>: Evidence suggests that triple therapy for patients with chronic obstructive pulmonary disease (COPD) is being used in a broader range of patients than recommended by guidelines, which may have health and cost implications. <b>Objective</b>: To explore the relationship between national health technology assessment (HTA) agency appraisals and market penetration of two fixed-dose combination (FDC) triple therapies. <b>Study design</b>: HTAs from Q3 2017 to Q1 2020 from 10 countries were evaluated. <b>Intervention</b>: Glycopyrronium bromide/formoterol fumarate/beclomethasone (Trimbow®) and umeclidinium/vilanterol/fluticasone furoate (Trelegy™ Ellipta®). <b>Main outcome measure</b>: HTA restrictions and prescribing rates (days of therapy). <b>Results</b>: Seven countries (70%) imposed restrictions on use including prescription only for patients stable on free-combination triple therapy or not controlled on dual therapy, requirement of a specialist prescription or therapeutic plan, prescription only for patients with severe COPD, and use as second-line therapy or later. In general, countries that have imposed restrictions on the use of FDC triple therapies have seen a lower than average uptake. <b>Conclusion</b>: Payer guidance on prescribing FDC triple therapy may potentially support more appropriate prescribing in line with clinical guidelines. It is important for payers to consider which restrictions would ensure the most efficient use of scarce resources.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1929757"},"PeriodicalIF":0.0,"publicationDate":"2021-06-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1929757","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"39091054","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":"Trends in US emergency department visits and subsequent hospital admission among patients with inflammatory bowel disease presenting with abdominal pain: a real-world study from a national emergency department sample database.","authors":"Zhijie Ding,&nbsp;Aarti Patel,&nbsp;James Izanec,&nbsp;Christopher D Pericone,&nbsp;Jennifer H Lin,&nbsp;Christopher W Baugh","doi":"10.1080/20016689.2021.1912924","DOIUrl":"https://doi.org/10.1080/20016689.2021.1912924","url":null,"abstract":"<p><p><b>Background/Objective</b>: This study evaluated emergency department (ED) visit trends, subsequent inpatient admissions for patients with inflammatory bowel disease (IBD) diagnosis and IBD-related abdominal pain (AP), and hospital-level variation in inpatient admission rates in the USA (US). <b>Methods</b>: This population-based, cross-sectional study included data from Nationwide Emergency Department Sample (NEDS, 2006─2013) database. Patients ≥18 years of age with primary ED diagnosis of IBD/IBD-related AP were included. Variables included demographics, insurance information, household income, Quan-Charlson comorbidity score, ED discharge disposition, and length of hospital stay (2006, 2010, and 2013). Variation between hospitals using risk-adjusted admission ratio was estimated. <b>Results</b>: Annual ED visits for IBD/100,000 US population increased (30 in 2006 vs 42 in 2013, p = 0.09), subsequent admissions remained stable (20 in 2006 vs 23 in 2013, p = 0.52). ED visits for IBD-related AP increased by 71% (7 in 2006 vs 12 in 2013; p = 0.12), subsequent admissions were stable (0.50 in 2006 vs 0.58 in 2013; p = 0.88). Proportion of patients with subsequent hospitalization decreased (IBD: 65.7% to 55.7%; IBD-related AP: 6.9% to 4.9%). Variation in subsequent inpatient admissions was 1.42 (IBD) and 1.96 (IBD-related AP). <b>Conclusions</b>: An increase in annual ED visits was observed for patients with IBD and IBD-related AP; however, subsequent inpatient admission rate remained stable.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1912924"},"PeriodicalIF":0.0,"publicationDate":"2021-04-19","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1912924","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38965543","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":"Allocating treatment resources for hepatitis C in the UK: a constrained optimization modelling approach.","authors":"Ru Han,&nbsp;Shuyao Liang,&nbsp;Clément François,&nbsp;Samuel Aballea,&nbsp;Emilie Clay,&nbsp;Mondher Toumi","doi":"10.1080/20016689.2021.1887664","DOIUrl":"https://doi.org/10.1080/20016689.2021.1887664","url":null,"abstract":"<p><p><b>Background and objective:</b> Although the treatment of chronic hepatitis C (CHC) has significantly evolved with the introduction of direct-acting antivirals, the treatment uptake rates have been low especially among marginalized groups in the UK, such as people who inject drug (PWID) and men who have sex with men (MSM). Cutting health inequality is a major focus of healthcare agencies. This study aims to identify the optimal allocation of treatment budget for chronic hepatitis CHC among populations and treatments in the UK so that liver-related mortality in patients with CHC is minimized, given the constraint of treatment budget and equity issue. <b>Methods:</b> A constrained optimization modelling of resource allocation for the treatment of CHC was developed in Excel from the perspective of the UK National Health System over a lifetime horizon. The model was designated with the objective function of minimizing liver-related deaths by varying the decision variables, representing the number of patients receiving each treatment (elbasvir-grazoprevir, ombitasvir-paritaprevir-ritonavir-dasabuvir, sofosbuvir-ledipasvir, and pegylated interferon-ribavirin) in each population (the general population, PWID, and MSM). Two main constraints were formulated including treatment budget and the issue of equity. The model was populated with UK local data applying linear programming and underwent internal and external validation. Scenario analyses were performed to assess the robustness of model results. <b>Results:</b> Within the constraints of no additional funding over original spending in status quo and the consideration of the issue of equity among populations, the optimal allocation from the constrained optimization modelling (treating 13,122 PWID, 160 MSM, and 904 general patients with ombitasvir-paritaprevir-ritonavir-dasabuvir) was found to treat 2,430 more patients (relative change: 20.7%) and avert 78 liver-related deaths (relative change: 0.3%) compared with the current allocation. The number of patients receiving treatment increased 4,928 (relative change: 60.1%) among PWID and 42 (relative change: 35.8%) among MSM. <b>Conclusion:</b> The current allocation of treatment budget for CHC is not optimal in the UK. More patients would be treated, and more liver-related deaths would be avoided using a new allocation from a constrained optimization modelling without incurring additional spending and considering the issue of equity.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1887664"},"PeriodicalIF":0.0,"publicationDate":"2021-03-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1887664","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25568838","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 updated cost-utility model for onasemnogene abeparvovec (Zolgensma®) in spinal muscular atrophy type 1 patients and comparison with evaluation by the Institute for Clinical and Effectiveness Review (ICER).","authors":"Rebecca Dean,&nbsp;Ivar Jensen,&nbsp;Phil Cyr,&nbsp;Beckley Miller,&nbsp;Benit Maru,&nbsp;Douglas M Sproule,&nbsp;Douglas E Feltner,&nbsp;Thomas Wiesner,&nbsp;Daniel C Malone,&nbsp;Matthias Bischof,&nbsp;Walter Toro,&nbsp;Omar Dabbous","doi":"10.1080/20016689.2021.1889841","DOIUrl":"https://doi.org/10.1080/20016689.2021.1889841","url":null,"abstract":"<p><p><b>Background</b>: Recent cost-utility analysis (CUA) models for onasemnogene abeparvovec (Zolgensma®, formerly AVXS-101) in spinal muscular atrophy type 1 (SMA1) differ on key assumptions and results. <b>Objective</b>: To compare the manufacturer's proprietary CUA model to the model published by the Institute for Clinical and Economic Review (ICER), and to update the manufacturer's model with long-term follow-up data and some key ICER assumptions. <b>Study design</b>: We updated a recent CUA evaluating value for money in cost per incremental Quality-adjusted Life Year (QALY) of onasemnogene abeparvovec versus nusinersen (Spinraza®) or best supportive care (BSC) in symptomatic SMA1 patients, and compared it to the ICER model. <b>Setting/Perspective</b>: USA/Commercial payer <b>Participants</b>: Children aged <2 years with SMA1. <b>Interventions</b>: Onasemnogene abeparvovec, a single-dose gene replacement therapy, versus nusinersen, an antisense oligonucleotide, versus BSC. <b>Main outcome measure</b>: Incremental-cost effectiveness ratio and value-based price using traditional thresholds for general medicines in the US. <b>Results</b>: Updated survival (undiscounted) predicted by the model was 37.60 years for onasemnogene abeparvovec compared to 12.10 years for nusinersen and 7.27 years for BSC. Updated quality-adjusted survival using ICER's utility scores and discounted at 3% were 13.33, 2.85, and 1.15 discounted QALYs for onasemnogene abeparvovec, nusinersen, and BSC, respectively. Using estimated net prices, the discounted lifetime cost/patient was $3.93 M for onasemnogene abeparvovec, $4.60 M for nusinersen, and $1.96 M for BSC. The incremental cost per QALY gained for onasemnogene abeparvovec was dominant against nusinersen and $161,648 against BSC. These results broadly align with the results of the ICER model, which predicted a cost per QALY gained of $139,000 compared with nusinersen, and $243,000 compared with BSC (assuming a placeholder price of $2 M for onasemnogene abeparvovec), differences in methodology notwithstanding. Exploratory analyses in presymptomatic patients were similar. <b>Conclusion</b>: This updated CUA model is similar to ICER analyses comparing onasemnogene abeparvovec with nusinersen in the symptomatic and presymptomatic SMA populations. At a list price of $2.125 M, onasemnogene abeparvovec is cost-effective compared to nusinersen for SMA1 patients treated before age 2 years. When compared to BSC, cost per QALY of onasemnogene abeparvovec is higher than commonly used thresholds for therapies in the USA ($150,000 per QALY).</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1889841"},"PeriodicalIF":0.0,"publicationDate":"2021-02-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1889841","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25467146","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":"A conceptual framework to develop a patient-reported experience measure of the quality of mental health care: a qualitative study of the PREMIUM project in France.","authors":"S Fernandes,&nbsp;G Fond,&nbsp;X Zendjidjian,&nbsp;P Michel,&nbsp;C Lançon,&nbsp;F Berna,&nbsp;F Schurhoff,&nbsp;B Aouizerate,&nbsp;C Henry,&nbsp;B Etain,&nbsp;L Samalin,&nbsp;M Leboyer,&nbsp;D Misdrahi,&nbsp;P M Llorca,&nbsp;M Coldefy,&nbsp;P Auquier,&nbsp;K Baumstarck,&nbsp;L Boyer","doi":"10.1080/20016689.2021.1885789","DOIUrl":"https://doi.org/10.1080/20016689.2021.1885789","url":null,"abstract":"<p><p><b>Background</b>: The objective of this study was to develop a conceptual framework to define a domain map describing the experience of patients with severe mental illnesses (SMIs) on the quality of mental health care. <b>Methods</b>: This study used an exploratory qualitative approach to examine the subjective experience of adult patients (18-65 years old) with SMIs, including schizophrenia (SZ), bipolar disorder (BD) and major depressive disorder (MDD). Participants were selected using a purposeful sampling method. Semistructured interviews were conducted with 37 psychiatric inpatients and outpatients recruited from the largest public hospital in southeastern France. Transcripts were subjected to an inductive analysis by using two complementary approaches (thematic analysis and computerized text analysis) to identify themes and subthemes. <b>Results</b>: Our analysis generated a conceptual model composed of 7 main themes, ranked from most important to least important as follows: interpersonal relationships, care environment, drug therapy, access and care coordination, respect and dignity, information and psychological care. The interpersonal relationships theme was divided into 3 subthemes: patient-staff relationships, relations with other patients and involvement of family and friends. All themes were spontaneously raised by respondents. <b>Conclusion</b>: This work provides a conceptual framework that will inform the subsequent development of a patient-reported experience measure to monitor and improve the performance of the mental health care system in France. The findings showed that patients with SMIs place an emphasis on the interpersonal component, which is one of the important predictors of therapeutic alliance. <b>Trial registration</b>: NCT02491866.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1885789"},"PeriodicalIF":0.0,"publicationDate":"2021-02-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2021.1885789","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"25445409","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":"Economic evaluation of adverse events of dabrafenib plus trametinib versus nivolumab in patients with advanced BRAF-mutant cutaneous melanoma for adjuvant therapy in Germany.","authors":"S Wahler, A Müller, C Koll, P Seyed-Abbaszadeh, J M Von Der Schulenburg","doi":"10.1080/20016689.2020.1861804","DOIUrl":"10.1080/20016689.2020.1861804","url":null,"abstract":"<p><p><b>Background:</b> Adjuvant treatment options have become the standard therapy for stage III and IV resectable cutaneous melanoma. Two recent studies led to the registration of dabrafenib and trametinib as targeted therapies for BRAF-mutated melanoma, and of immunotherapy with nivolumab irrespective of BRAF-mutation status. Both therapies have different spectrums of adverse events. <b>Objective:</b> To estimate the financial impact of side effects from the perspective of the German statutory sick funds to compare both therapeutic options and to relate the burden to the overall costs of the treatment.</p><p><strong>Study design and setting: </strong>Thirty-six adverse event categories for the combination of dabrafenib and trametinib ('combi treatment') and for nivolumab were extracted from the original publications of the studies named COMBI-AD and CheckMate 238.</p><p><strong>Patients and intervention: </strong>For all event categories a diagnosis and therapy recommendation were determined according to current national or international guidelines or from leading German textbooks.</p><p><strong>Main outcome measure: </strong>The resulting diagnostic steps, treatments, and therapies were evaluated with unit costs based on the German fee schedule for ambulatory physicians, the German G-DRG scheme, and the German drug price list.</p><p><strong>Results: </strong>The number of events with nivolumab per one hundred treatments amounted to 3.8 mandatory hospitalizations, 3.5 emergency care events and 0.8 life-threatening events. For the combi treatment, the respective number of events per one hundred treatments was 2.7, 1.8, and 0.5. The overall cost burden was calculated as €899 for nivolumab and €861 for combi-treatment.</p><p><strong>Conclusion: </strong>The treatment of adverse events resulting from adjuvant melanoma therapy showed comparable costs for both therapies.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1861804"},"PeriodicalIF":0.0,"publicationDate":"2020-12-28","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/db/3c/ZJMA_9_1861804.PMC7781974.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38827813","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 algorithm to generate correlated input-parameters to be used in probabilistic sensitivity analyses.","authors":"Mohamed Neine,&nbsp;Desmond Curran","doi":"10.1080/20016689.2020.1857052","DOIUrl":"https://doi.org/10.1080/20016689.2020.1857052","url":null,"abstract":"<p><p><b>Background</b>: Assessment of uncertainty in cost-effectiveness analyses (CEAs) is paramount for decision-making. Probabilistic sensitivity analysis (PSA) estimates uncertainty by varying all input parameters simultaneously within predefined ranges; however, PSA often ignores correlations between parameters. <b>Objective</b>: To implement an efficient algorithm that integrates parameter correlation in PSA. <b>Study design</b>: An algorithm based on Cholesky decomposition was developed to generate multivariate non-normal parameter distributions for the age-dependent incidence of herpes zoster (HZ). The algorithm was implemented in an HZ CEA model and evaluated for gamma and beta distributions. The incremental cost-effectiveness ratio (ICER) and the probability of being cost-effective at a given ICER threshold were calculated for different levels of correlation. Five thousand Monte Carlo simulations were carried out. <b>Results</b>: Correlation coefficients between parameters sampled from the distribution generated by the algorithm matched the desired correlations for both distribution functions. With correlations set to 0.0, 0.5, and 0.9, 90% of the simulations showed ICERs below $25,000, $33,000, and $38,000 per quality-adjusted life-year (QALY), respectively, varying incidence only; and below $38,000, $48,000, and $58,000 per QALY, respectively, varying most parameters. <b>Conclusion</b>: Parameter correlation may impact the uncertainty of CEA results. We implemented an efficient method for generating correlated non-normal distributions for use in PSA.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1857052"},"PeriodicalIF":0.0,"publicationDate":"2020-12-15","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1857052","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38784819","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":"The impact of patent expiry on drug prices: insights from the Dutch market.","authors":"Simon van der Schans,&nbsp;Gert T Vondeling,&nbsp;Qi Cao,&nbsp;Simon van der Pol,&nbsp;Sipke Visser,&nbsp;Maarten J Postma,&nbsp;Mark H Rozenbaum","doi":"10.1080/20016689.2020.1849984","DOIUrl":"https://doi.org/10.1080/20016689.2020.1849984","url":null,"abstract":"<p><p><b>Background</b>: Currently literature on the impact of patent expiry on drug prices is lacking. <b>Objective</b>: To determine the impact of patent expiration and generic entry on drug prices in the Netherlands. <b>Methods</b>: Prescription and price data from 1999 up to and including December 2016 were collected from two national databases. The overall price ratio of drugs prices up to 48 months after patent expiration was compared to the price in the month before expiry. Sub-analyses were performed to provide insights in generic uptake, length of market exclusivity and price development for originators and generics separately. <b>Results</b>: In total 250 drugs faced patent expiration during the study period. Forty-eight months after patent expiration the median price ratio decreased to 0.59 (IQR = 0.23-0.86) compared to the month prior patent expiry. Major differences in price developments were observed depending on the level of revenue prior to patent expiration and the time of patent expiration with ratios ranging from 0.08 (IQR = 0.07-0.16) to 0.81 (IQR = 0.62-0.97). Prior to patent expiry, the price decreased by 2.3% annually while having market exclusivity for 11.3 years on average. <b>Conclusion</b>: This study showed that the median drug price after patent expiration decreased by 41% after 4 years. The results of this study can be used to provide more reliable estimates on drug prices over its lifecycle and can be implemented in economic evaluations to inform the cost-effectiveness and long-term budget impact of new drugs.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"9 1","pages":"1849984"},"PeriodicalIF":0.0,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://sci-hub-pdf.com/10.1080/20016689.2020.1849984","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38704802","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 of orphan drugs in oncology and rare diseases.","authors":"Mark Nuijten, Stefano Capri","doi":"10.1080/20016689.2020.1838191","DOIUrl":"10.1080/20016689.2020.1838191","url":null,"abstract":"<p><p><b>Objective:</b> The objective of this paper is to determine an upper price limit for an orphan drug by taken a broader perspective and, including also other monetary and non-monetary values for the society. <b>Methods:</b> This model is based on the expected free cash flows and the required minimum rate of return for the investor. In addition we calculated an innovation premium resulting from cost savings due to the substitution effect and the monetary gain in QALYs of a new medicine. We selected Spinraza®, a first in class drug with only best supportive care as comparator, and Perjeta®, a first in class drug with already an actual treatment as comparator. <b>Results</b>: The results show that Spinraza® leads to an innovation premium of € 78,966 and Perjeta® shows an innovation premium of € 4,388, because there were no cost savings. The analyses show the outcomes are sensitive to discount rate for QALYs. <b>Conclusion:</b> The break-even price from only an investor perspective may not reflect the value of drug from a broader perspective. This study shows drug prices based on an innovation premium may be more representative of the actual value of innovation for the society.</p>","PeriodicalId":73811,"journal":{"name":"Journal of market access & health policy","volume":"8 1","pages":"1838191"},"PeriodicalIF":0.0,"publicationDate":"2020-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://ftp.ncbi.nlm.nih.gov/pub/pmc/oa_pdf/1e/79/ZJMA_8_1838191.PMC7717868.pdf","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"38367393","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}