e-Repositori UPF :: Online Master in Health Economics and Pharmacoeconomics

Recent Submissions

To tender or not to tender: a literature review and policy guidelines on tendering for outpatient generic pharmaceuticals
(2022) Nieto Rueda, Jorge
Background: Despite progress in recent years, generic drugs markets are not developing its full potential, particularly in countries with more intense price regulation. Tendering is an alternative and indirect price setting scheme with great potential in achieving more efficient prices. However, its development has been scarce and controversial in outpatient settings. Objectives: To analyse whether and how tendering for outpatient generic pharmaceuticals in an EU country system could be a beneficial policy and to provide orientations on its key variables to policy-makers. Methods and search strategy: A literature review on outpatient generic tenders was conducted between 2011 and 2021 using PubMed and google scholar. The same search terms as a 2016 review by the WHO were used. In addition, aggregate and pool procurement of pharmaceuticals studies -not limited to outpatient settings- and public procurement and competition policy studies and documents from leading institution and scholars are also included in the analysis. Results: 12 studies from the literature review met the inclusion criteria. Tendering was overall a beneficial policy, with great potential for price reductions and for enhancing transparency. However, it is a complex policy interacting with a wide set of factors that should be considered: a) the legal, organisational and political framework; b) market and competition dynamics; c) stakeholders, as resistance is expected and are key on the policy implementation; d) tender design and implementation monitoring. Conclusion: Tendering is a powerful instrument but a complex one. A multidisciplinary approach should be followed while designing it. Risks of implementation gaps are substantial. There is no one-size-fits-all solution. However, balancing short-and long-term outcomes, favouring multiwinner schemes, ensuring a capable organisation and an appropriate framework, proper management of stakeholders and strengthening planning and implementation monitoring will be determinant for the success of a tender programme.
Cost Effectiveness of Use of Urine Based Lateral-Flow Lipoarabinomannan Test in Diagnosis of Tuberculosis in HIV-positive Patients in Namibia
(2021) Vukile Sithole, Edwin Gorden
Background: Namibia is only notifying 62% of the estimated TB cases. Xpert MTB/RIF (Xpert) is considered the gold standard for tuberculosis (TB) diagnosis. The urine lipoarabinomannan (LF-LAM) assay has been recommended to provide rapid TB diagnosis in HIV positive patients to improve case finding. Objectives: To evaluate the cost-effectiveness of LF-LAM when used in combination with Xpert compared to Xpert only for TB diagnosis in an HIV-positive population in Namibia. Method: This was a cost effectiveness study from a health system perspective. A model based on the decision tree was utilized to compare the two strategies for TB diagnosis. A cohort of 1000 patients was assumed. The primary outcomes were the costs and the DALYs averted for each algorithm. The incremental cost-effectiveness ratio (ICER) was determined. Results: The combination of Xpert and LF-LAM compared to Xpert testing alone was very highly cost-effective at ICER of $601 per DALY averted. The willingness to pay (WTP) threshold was the annual GDP per capita ($9,383.20) of Namibia. The cost-effectiveness was most sensitive to the probability of treatment success on first line TB regimen, probability of a positive test for both algorithms as well as the cost of ART but still remained below the WTP threshold. Conclusion: The addition of urine LF-LAM to Xpert test for TB diagnosis in HIV infected individuals is very highly cost effective when compared to Xpert alone. Even after variation of the parameters the ICER remains within WTP in the one-way sensitivity analysis.
Cost-effectiveness Analysis of Continuous subcutaneous insulin infusion compared with multiple daily injections in adult patients with type 1 and type 2 Diabetes mellitus: a systematic review
(2021) Sadek, Mina
Background: Insulin replacement therapy is the cornerstone for the treatment of type 1 and 2 diabetes mellitus. Two of the most used methods are continuous subcutaneous insulin infusion and multiple daily injections. This systematic review aims to find, can the continuous subcutaneous insulin infusion be considered cost-effective compared to the multiple daily injections? Methods: A systematic search was conducted in August 2021 that focused on the published literature comparing the cost-effectiveness of continuous subcutaneous insulin infusion with multiple daily injections. Searched databases included Pubmed, Scopus, Cochrane, and UPF finder. Results: A total of 621 publications were identified, of which 21 were analyzed. Results were significantly dependant on the study design and method used. Most of the studies based on Modelling reported that continuous subcutaneous insulin infusion is considered cost-effective compared to multiple daily injections with an average Incremental cost effectiveness ratio of US $37,717/QALY gained, with an Adjusted Incremental cost effectiveness ratio (to the year 2021) calculated as US $51,016/QALY gained. However, Randomized-controlled trials reported opposite results. Conclusion: Continuous subcutaneous insulin infusion is proven cost-effective compared to Multiple daily injections, especially in patients with high mean HbA1C, and can thus be considered a valuable therapy option in adult patients with type 1 diabetes mellitus. More evidence is needed in patients with type 2 diabetes mellitus treatment, but it would most probably be cost-effective, especially in patients with difficulty reaching optimal HbA1C levels using Multiple daily injections.
Cost-effectiveness and cost-utility study of hypercholesterolemia treatment in Spain: evolocumab added to standard of care vs standard of care.
(2022) Imedio Cerezo, Beatriz
Background. Cardiovascular diseases have a significant economic burden on healthcare systems. Effective treatments for hypercholesterolemia are available, with statins being currently the standard of care. A PCSK9 inhibitor, evolocumab, has also shown efficacy in reducing cardiovascular events in these patients. This study evaluates the cost-effectiveness and cost-utility of evolocumab with standard of care vs standard of care alone from the perspective of the Spanish National Health System (sNHS).Methods.A 20 years Markov model, based on a Markov chain developed including estimates of effectiveness, progression-free life years, quality-adjusted life years and costs. Monthly cycles were used to fit the disease course. Outcome measures were avoided cardiovascular events, life years and quality-adjusted life years (QALYs). Finally, a Monte Carlo simulation to manage uncertainty was employed and an acceptability curve was presented.Results. The incremental cost-effectiveness and incremental utility cost ratios per patient obtained were 59.895,20 €per life year free of progression and 289.677,66 €per QALY gained, respectively. The ratios obtained for the standard of care and for evolocumab added to the standard of care in that order were 191.73€and 7,238.04€per progression-free life year and 141.04€and 5,916.71€per quality-adjusted life year gained.Conclusions. In light of these results, and assuming a reference threshold of €30,000/QALY for the sNHS, adding evolocumab to standard treatment is not cost-effective when compared to standard treatment alone. However, these results should be interpreted with caution, as evolocumab may still provide cost effective value in some specific subgroups of patients. Even if no explicit cost-effectiveness threshold is used in Spain to decide funding and/or pricing levels, this work may contribute to inform decision-making in our setting.
Cost-effectiveness and costutility analysis of Deprexis®, a digital therapy intended for the treatment of mild to severe depression, in Catalonia
(2021) Rothe Muñoz, Rainer Mariano
Depression is an illness that has a strong social and economic impact, and that has seen its prevalence increased during the COVID-19 pandemic. As with many sectors of healthcare, the increased costs of treatment and the scarce availability of professionals lead to long waiting lists, generating a need for digital innovation in the management of depression. Deprexis® is a digital therapy that has been recently approved for its reimbursement under prescription in Germany and that has been studied for its efficacy in the treatment of depression as a complement of care-as-usual, as a partial replacement of care-as-usual (CAU) and as a tool to be used in patients in waiting list. In this study the incremental cost-effectiveness ratio (ICER) and incremental cost-utility ratios (ICUR) of the introduction of Deprexis® as a complement of CAU for the treatment of depression in the region of Catalonia and from the perspective of both, society, and that of the National Health Service, were estimated using a Markov model. It was found that, when compared with CAU, the introduction of Deprexis® incurred an ICER of 3,866 € per clinically improved individual over a one-year time horizon and a ICUR of 125,498 € per quality adjusted life year from the perspective of the National Health service, and 2,248 € per clinically improved individual and 72,970 € per QALY from the societal perspective
A cost-effectiveness analysis in a Norwegian setting: Introducing genotyping to patients with ACS treated with PCI before prescribing antiplatelet therapy
(2021) Politi, Angeliki Louiza
The purpose of this study is to conclude as to whether it is cost-effective to introduce genotyping in the Norwegian clinical practice in the case of acute coronary syndrome, as a guide of pharmacotherapy decision making after a percutaneous coronary intervention.
Appraisal of NICE’s cost-effectiveness thresholds for assessing orphan drugs
(2021) Nan, Ruodan
A rare disease is one that affects less than 1 in 2,000 in general population, and they are often chronic and life-threatening. Due to the high cost to develop and small market for orphan drugs, historically manufacturers are reluctantly to explore the rare disease therapeutic areas. There is highly unmet need from patients with rare diseases. As the pharmaceutical expenditure rises, governments around the world are increasingly under pressure to contain the costs on drug reimbursement. Meanwhile patients with rare diseases need to be ensured for access to orphan drugs, and the industry needs to be incentivized to innovate. Health technology assessment and reimbursement bodies set out measures and policies for orphan drugs to be given market access. In this literature review, the landscape of health technology appraisals for orphan drugs in England UK is studied. The process for an orphan drug to gain marketing authorisation in the European Union (EU), the methods and criteria that used by National Institute for Health and Care Excellence (NICE) are reviewed. All orphan drugs with marketing authorisations from European Medicine Agency (EMA) are searched from the agency’s online database, whereas each of the authorised orphan drug is searched in NICE’s database for any technology appraisals and recommendations made for the orphan drug. The search results are then analysed to find factors that may influence NICE’s decisions on whether the drug should be considered cost-effective. Based on the analyses, suggestions are made for NICE to potentially improve its way of assessing orphan drugs.
Valuing health related quality of life in adolescents from their own perspective: a potential EQ-5D-Y-3L value set for Spain
(2021) Estévez Carrillo, Anabel
There is a growing interest in measuring youth preferences for decision making. The EQ-5D-Y-3L is generic preference-based instrument developed by the EuroQol Group that is appropriate for children and adolescents. Recent evidence demonstrates the need of specific value sets for the EQ-5D-Y-3L. Our study analyses the validity of DCE responses in adolescents and explores a potential methodology to obtain an adolescents’ value set in Spain from their own perspective.
Tisagenlecleucel (chimeric antigen receptor T-cells targeting CD19) for patients with relapsed/refractory B-cell acute lymphoblastic leukemia: asystematic review and cost-utility analysis from a Spanish perspective
(2020) Delgado González, Julio
Patients with relapsed/refractory (R/R) B-cell acute lymphoblastic leukemia (ALL) have a poor prognosis with conventional therapies. In the summer of 2018, the European Medicines Agency approved tisagenlecleucel (tisa-cel, Kymriah®, Novartis), for the treatment of these patients based on a single-arm phase 2 international clinical trial with a short follow-up. The price tag for this product is €320,000 but under a pay-for-performance scheme in which the health care provider pays €192,000 upfront and the other €128,000 if the patient is alive and in remission 18 months later. The main objective of this study was to perform a costutility analysis of tisa-cel from a Spanish point of view. Tisa-cel resulted in a longer life expectancy (9.89 LYs) than the other two therapies evaluated (6.45 and 1.94 LYs for blinatumomab and clofarabine, respectively). The difference in relapse-free survival (RFS) led to 3.75 additional QALYs for patients receiving tisa-cel compared to blinatumomab at a cost of €55,779/QALY, and 7,15 additional QALYs compared to clofarabine at a cost of €35,787/QALY. The Markov process revealed very similar results: the difference in RFS resulted in 3.67 additional QALYs for tisa-cel compared to blinatumomab at a cost of €57,368/QALY (95% confidence interval [CI]: 52,779-61,956), and 7.3 additional QALYs compared to clofarabine at a cost of €35,504/QALY (95% CI: 35,153-35,854). One-way sensitivity analyses showed that the factor that had the greatest impact on the cost-effectiveness of tisa-cel (over blinatumomab) was its cost. In conclusion, tisa-cel could be considered costeffective in the treatment of patients with R/R pediatric ALL when the WTP threshold is set at €50,000/QALY (or higher), but many uncertainties remain (lack of comparative data, short follow-up).
Cost-effectiveness of first-line treatment options and treatment sequencing in advanced renal cell carcinoma
(2020) Carson, Brittany
Continued cancer research allows for the development of novel and potentially superior treatments and management approaches. The recent identification of the role of the immune system in cancer has led to the development of agents targeting the anti-tumor immune response, bringing increased survival and improved patient quality of life. However, these agents, known as immune checkpoint inhibitors (ICIs), also bring additional costs associated with the drug itself and management of immune-associated safety events. Despite the approval of three ICI-based combinations for the first-line treatment of advanced renal cell carcinoma (aRCC), the question remains as to which of the three options is the most cost effective and whether these new agents are more cost-effective overall for society. Further, given that most patients progress on first-line therapy, there is motivation in clinical practice to save ICIs as second-line salvage therapy, retaining the current non-ICI first-line standard of care. A Markov model was created comparing the costeffectiveness of these 3 ICI-based options and the current first-line standard of care in the context of multi-line treatment. The model included direct medical costs, utilities associated with progression-free survival and progressive disease, and commonly used second-line treatment options for patients who progressed on the first-line treatment options. Incremental cost-effectiveness ratios (ICERs) were calculated for all 4 first-line treatment options assessing cost (US dollars) per quality-adjusted life year (QALY). Avelumab + axitinib was the more cost effective first-line option and ICI-based combination. Only nivolumab + ipilimumab exceeded the selected willing-to-pay threshold of $100,000 USD/QALY. In summary, ICI-based combinations, in particular avelumab + axitinib, are also cost-effective first-line options in the treatment of aRCC.
Cost-effectiveness análisis ofmicro-dystrophin: a novel gene therapy for duchenne muscular dystrophy
(2020) Antolin Fontes, Beatriz
Cost-effectiveness analysis of different anticoagulant classes in cancer associated thrombosis (CAT) : a differential approach accounting thrombotic and bleeding risk, applied in the Greek health care environment
(2019) Sarigiannidis, Ioannis
Background: Guidelines recommend that Cancer-associated thrombosis (CAT) should be treated with low-molecular weight heparins (LMWH), which prevent more effectively the recurrence of Venous Thrombo-Embolism (VTE) than Vitamin K Antagonists (VKA). New Direct Oral Anti-coagulants (DOAC) may be more convenient and have been recently tested against LMWH in CAT patients. Objective: Assess the cost effectiveness of the different anticoagulant classes in CAT in the Greek Healthcare (HC) setting from the hospital perspective, accounting different bleeding/thrombotic Risk groups (High/Low) defined by presence of chemotherapy/metastasis. Methods: Markov chain modeling accounting VTE, Major Bleeding (MB), clinically relevant non-major bleeding (CRB) and death as major states, incorporating additional mortality risk by VTE and MB. All comparisons were made versus (vs) VKA as base. All costs were derived in the context of the Greek HC system and included direct pharmacological costs, Diagnosis Related Group (DRG) costs for the management of VTE, MB, CRB and secondary support costs of VKA (laboratory testing and HC professionals consultation visits). Results: For Low Risk CAT patients DOAC were more cost effective in all analyzed endpoints (VTE, MB, CRB) including Life Years Gained (LYG) and total Utility with a favorable estimated Incremental Cost-Effectiveness Ratio (ICER) of less than €300 per LYG or per Quality Adjusted Life Year (QALY) gained vs VKA. In High Risk CAT patients DOACs were dominated by VKA in QALY, LYG, MB and CRB with negative ICER values, making LMWH acceptably cost effective option (ICER of €15.000 per LYG and €22.000 per QALY gained vs DOAC). Conclusions: Risk stratification for CAT patients might prove useful achieving higher cost effectiveness from the different anticoagulant classes. For the Greek HC environment, DOAC seem more cost effective for the Low Risk CAT patients, while LMWH deem preferred for High Risk CAT patients offering acceptable cost-utility gains.
Following the 2001 Abuja Declaration of committing 15 percent government expenditure on health, is Africa making progress towards universal health coverage?
(2019) Sadr-Azodi, Nahad
Background: In 2001, 46 African Union nations signed the Abuja Declaration, committing 15 percent of their public expenditures to health. Objectives: In this paper, I was motivated to determine if government spending on health could significantly predict the African countries’ progress towards universal health coverage service coverage (UHC-sc). Methods: The nature of the term paper was primarily of qualitative descriptive research with limited inferential analysis. I explored the research question around the relations between domestic resources and progress towards UHC in Africa in two phases: literature review and secondary data analysis and interpretation. Results: In 2018, only three countries have reached or exceeded the Abuja Declaration target, and no country has achieved the universal health coverage service coverage index threshold value of 80. The findings show that government health spending, particularly absolute per capita expenditure, does explain improvements in UHC-sc. Therefore, I rejected the primary and secondary null hypotheses indicating that linear coefficient is not equal to zero at the 5 percent p-value. Moreover, I determined that absolute expenditure per person has a stronger correlation and explanation for progress towards UHC-sc compared with government health spending as a proportion of government expenditures in African countries. Discussion and conclusions: In moving forward, African leaders should institutionalize their commitments to attaining universal health coverage (UHC) through legislations and prioritize health by striving towards the Abuja Declaration target while ensuring a minimum per capita spending required for provision of basic health services. Given the wide variations in performance despite same level spending, African countries should concurrently address allocative and technical efficiencies and strengthen financial management systems. Finally, at least in the short to medium terms, international and donor communities should support the African countries’ path towards UHC with sufficient development assistance for health to bridge the expected domestic shortfalls.

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