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  1. Article

    Open Access

    Issues, Challenges and Opportunities for Economic Evaluations of Orphan Drugs in Rare Diseases: An Umbrella Review

    There are significant challenges when obtaining clinical and economic evidence for health technology assessments of rare diseases. Many of them have been highlighted in previous systematic reviews but they hav...

    Tobias Sydendal Grand, Shijie Ren, James Hall, Daniel Oudin Åström in PharmacoEconomics (2024)

  2. Article

    Open Access

    Cost Effectiveness of Difelikefalin Compared to Standard Care for Treating Chronic Kidney Disease Associated Pruritus (CKD-aP) in People with Kidney Failure Receiving Haemodialysis

    Chronic kidney disease-associated pruritus (CKD-aP) is associated with an increased risk of depression, poor sleep and reduced health-related quality of life. Two phase III studies (KALM-1 and KALM-2) of difel...

    Praveen Thokala, Pann Ei Hnynn Si, Monica Hernandez Alava in PharmacoEconomics (2023)

  3. Article

    Living Health Technology Assessment: Issues, Challenges and Opportunities

    Health technology assessments (HTAs) are typically performed as one-off evaluations and can potentially become out-of-date due to the availability of new data, new comparators, or other factors. Recently, livi...

    Praveen Thokala, Tushar Srivastava, Robert Smith, Shijie Ren in PharmacoEconomics (2023)

  4. Article

    Open Access

    ENTIMOS: A Discrete Event Simulation Model for Maximising Efficiency of Infusion Suites in Centres Treating Multiple Sclerosis Patients

    Improved multiple sclerosis (MS) diagnosis and increased availability of intravenous disease-modifying treatments can lead to overburdening of infusion centres. This study was focused on develo** a decision-...

    Kristyna Lacinova, Praveen Thokala in Applied Health Economics and Health Policy (2022)

  5. Article

    Open Access

    Cost effectiveness of nusinersen for patients with infantile-onset spinal muscular atrophy in US

    Patients with infantile-onset spinal muscular atrophy (SMA), a rare, genetic neuromuscular disease, do not achieve key motor function milestones (e.g., sitting) and have short life expectancy in the absence of...

    Praveen Thokala, Matt Stevenson in Cost Effectiveness and Resource Allocation (2020)

  6. No Access

    Article

    Multi-criteria Decision Analysis Software in Healthcare Priority Setting: A Systematic Review

    The objectives of this systematic review were to identify studies using Multi-Criteria Decision Analysis (MCDA) software tools to support health prioritisation processes and describe the technical capabilities...

    Alexander Moreno-Calderón, Thai S. Tong, Praveen Thokala in PharmacoEconomics (2020)

  7. No Access

    Article

    Transparency in Health Economic Modeling: Options, Issues and Potential Solutions

    Economic models are increasingly being used by health economists to assess the value of health technologies and inform healthcare decision making. However, most published economic models represent a kind of bl...

    Eric Q. Wu, Zheng-Yi Zhou, Jipan **e, Cinzia Metallo, Praveen Thokala in PharmacoEconomics (2019)

  8. No Access

    Article

    Ibrutinib for Treating Waldenström’s Macroglobulinaemia: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

    As part of its Single Technology Appraisal (STA) process, the UK National Institute for Health and Care Excellence (NICE) invited the manufacturer of ibrutinib (Janssen) to submit evidence on the clinical and ...

    Paul Tappenden, Christopher Carroll, John Stevens, Emma Simpson in PharmacoEconomics (2019)

  9. Article

    Open Access

    Stakeholder involvement in Multi-Criteria Decision Analysis

    This brief perspective highlights the importance of decision maker buy-in and ownership through stakeholder engagement in the co-construction of the multi-criteria decision analysis (MCDA) model. A brief histo...

    Praveen Thokala, Guruprasad Madhavan in Cost Effectiveness and Resource Allocation (2018)

  10. Article

    Open Access

    Incorporating MCDA into HTA: challenges and potential solutions, with a focus on lower income settings

    Multicriteria decision analysis (MCDA) has the potential to bring more structure and transparency to health technology assessment (HTA). The objective of this paper is to highlight key methodological and pract...

    Kevin Marsh, Praveen Thokala in Cost Effectiveness and Resource Allocation (2018)

  11. No Access

    Article

    Cost-Effectiveness Thresholds: the Past, the Present and the Future

    Cost-effectiveness (CE) thresholds are being discussed more frequently and there have been many new developments in this area; however, there is a lack of understanding about what thresholds mean and their imp...

    Praveen Thokala, Jessica Ochalek, Ashley A. Leech, Thaison Tong in PharmacoEconomics (2018)

  12. No Access

    Article

    Pertuzumab for the Neoadjuvant Treatment of Early-Stage HER2-Positive Breast Cancer: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

    As part of its single technology appraisal process, the National Institute for Health and Care Excellence invited the manufacturer of pertuzumab (Perjeta®; Roche Products Limited) to submit evidence of its clinic...

    Hazel Squires, Abdullah Pandor, Praveen Thokala, John W. Stevens in PharmacoEconomics (2018)

  13. No Access

    Article

    Discrete Event Simulation-Based Resource Modelling in Health Technology Assessment

    The objective of this article was to conduct a systematic review of published research on the use of discrete event simulation (DES) for resource modelling (RM) in health technology assessment (HTA). RM is bro...

    Syed Salleh, Praveen Thokala, Alan Brennan, Ruby Hughes, Simon Dixon in PharmacoEconomics (2017)

  14. No Access

    Article

    Simulation Modelling in Healthcare: An Umbrella Review of Systematic Literature Reviews

    Numerous studies examine simulation modelling in healthcare. These studies present a bewildering array of simulation techniques and applications, making it challenging to characterise the literature.

    Syed Salleh, Praveen Thokala, Alan Brennan, Ruby Hughes, Andrew Booth in PharmacoEconomics (2017)

  15. No Access

    Book

  16. No Access

    Chapter

    Introduction

    Multi-criteria decision analysis (MCDA) has the potential to support better healthcare decision making. But a number of challenges need to be overcome before it can achieve its potential. These are both techni...

    Kevin Marsh, Mireille Goetghebeur MEng PhD in Multi-Criteria Decision Analysis to Suppor… (2017)

  17. No Access

    Chapter

    MCDA for Resource Allocation at a Local Level: An Application in the UK

    Resource allocation at a local level involves making difficult decisions about investment and, particularly in a time of economic stringency, about disinvestment. These complex decisions are influenced by a nu...

    Brian Reddy, Praveen Thokala in Multi-Criteria Decision Analysis to Suppor… (2017)

  18. No Access

    Chapter

    Incorporating Preferences and Priorities into MCDA: Selecting an Appropriate Scoring and Weighting Technique

    A key component of many multi-criteria decision analyses (MCDAs) is the elicitation of stakeholder preferences in the form or scores and weights. A challenge to the MCDA practitioner is that there is little gu...

    Kevin Marsh, Praveen Thokala in Multi-Criteria Decision Analysis to Suppor… (2017)

  19. No Access

    Article

    Olaparib for Maintenance Treatment of BRCA 1 or 2 Mutated, Relapsed, Platinum-Sensitive Ovarian, Fallopian Tube and Peritoneal Cancer in People Whose Relapsed Disease has Responded to Platinum-Based Chemotherapy: An Evidence Review Group Perspective of a NICE Single Technology Appraisal

    As part of its Single Technology Appraisal process, the National Institute for Health and Care Excellence (NICE) invited the manufacturer of olaparib (AstraZeneca) to submit evidence on the clinical and cost e...

    Paul Tappenden, Sue Harnan, Shijie Ren, Praveen Thokala, Ruth Wong in PharmacoEconomics (2017)

  20. No Access

    Article

    Sipuleucel-T for the Treatment of Metastatic Hormone-Relapsed Prostate Cancer: A NICE Single Technology Appraisal; An Evidence Review Group Perspective

    The National Institute for Health and Care Excellence (NICE) invited Dendreon, the company manufacturing sipuleucel-T, to submit evidence for the clinical and cost effectiveness of sipuleucel-T for asymptomati...

    Emma L. Simpson, Sarah Davis, Praveen Thokala, Penny R. Breeze in PharmacoEconomics (2015)

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