Article Commentary
A different animal? Identifying the features of health technology assessment for developers of medical technologies
- Janet Bouttell, Andrew Briggs, Neil Hawkins
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- Published online by Cambridge University Press:
- 24 June 2020, pp. 285-291
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Health technology assessment (HTA) conducted to inform developers of health technologies (development-focused HTA, DF-HTA) has a number of distinct features when compared to HTA conducted to inform usage decisions (use-focused HTA). To conduct effective DF-HTA, it is important that analysts are aware of its distinct features as analyses are often not published. We set out a framework of ten features, drawn from the literature and our own experience: a target audience of developers and investors; an underlying user objective to maximize return on investment; a broad range of decisions to inform; wide decision space; reduced evidence available; earlier timing of analysis; fluid business model; constrained resources for analysis; a positive stance of analysis; and a “consumer”-specific burden of proof. This paper presents a framework of ten features of DF-HTA intended to initiate debate as well as provide an introduction for analysts unfamiliar with the field.
Beyond the numbers: a critique of quantitative multi-criteria decision analysis
- Michael J. DiStefano, Carleigh B. Krubiner
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- Published online by Cambridge University Press:
- 01 July 2020, pp. 292-296
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When setting priorities for health, there is broad agreement that a range of social values and ethical principles beyond clinical and cost-effectiveness matter, but exactly how health technology assessment (HTA) should account for a broader set of criteria remains an area of ongoing debate. In light of this, we welcome a recent review paper by Baltussen et al. evaluating the potential of different multi-criteria decision analysis (MCDA) approaches to enable HTA agencies to incorporate a broader set of values in their appraisals. The authors describe three approaches to MCDA—qualitative MCDA, quantitative MCDA, and MCDA with decision rules—laying out their relative advantages and disadvantages and providing recommendations for how they can best be implemented. While we endorse many of the authors' assessments and conclusions, including the critical role of deliberation in any MCDA approach and the undertaking of qualitative MCDA at a minimum, we take a stronger position regarding the flaws of quantitative MCDA and strongly caution against it. We find quantitative MCDA antithetical to at least two of the ways MCDA is intended to improve HTA recommendations: (i) enhancing quality and (ii) promoting transparency. Quantitative MCDA may mask the complex tradeoffs that exist within and between decision criteria and remain generally inaccessible to those who are not well-versed in its technical methods of appraisal. We advocate for a predominantly qualitative approach to MCDA appraisal centered around deliberation and supplemented with decision aids to help account for health opportunity costs.
Valuing Alzheimer's disease drugs: a health technology assessment perspective on outcomes
- Annette Bauer, Raphael Wittenberg, Amanda Ly, Anders Gustavsson, Christin Bexelius, Claire Tochel, Martin Knapp, Mia Nelson, Catherine Sudlow
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- Published online by Cambridge University Press:
- 27 August 2020, pp. 297-303
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Objectives
Due to the nature of Alzheimer's disease (AD), health technology assessment (HTA) agencies might face considerable challenges in choosing appropriate outcomes and outcome measures for drugs that treat the condition. This study sought to understand which outcomes informed previous HTAs, to explore possible reasons for prioritizations, and derive potential implications for future assessments of AD drugs.
MethodWe conducted a literature review of studies that analyzed decisions made in HTAs (across disease areas) in three European countries: England, Germany, and The Netherlands. We then conducted case studies of technology assessments conducted for AD drugs in these countries.
ResultsOverall, outcomes measured using clinical scales dominated decisions or recommendations about whether to fund AD drugs, or price negotiations. HTA processes did not always allow the inclusion of outcomes relevant to people with AD, their carers, and families. Processes did not include early discussion and agreement on what would constitute appropriate outcome measures and cut-off points for effects.
ConclusionsWe conclude that in order to ensure that future AD drugs are valued appropriately and timely, early agreement with various stakeholders about outcomes, outcome measures, and cut-offs is important.
Method
Towards greater impact in health technology assessment: horizon scanning for new and emerging technologies in Singapore
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- Wan Qing Wong, Liang Lin, Hong Ju, Kwong Ng
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- 22 June 2020, pp. 304-310
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Objectives
To alert policy makers early about emerging health technologies that could significantly impact the healthcare system at the clinical, financial and organizational levels, the Agency for Care Effectiveness (ACE) in Singapore established a horizon scanning system (HSS) in 2019. This paper describes the development of the ACE HSS and showcases its application with cell and gene therapy products as the first example.
MethodsA literature review of existing HSS methods, including the processes of the EuroScan International Network and other overseas horizon scanning agencies, was done to inform the development of our horizon scanning framework. The framework was first applied to the new and emerging cell and gene therapies.
ResultsIdentification sources, filtration and prioritization criteria, and horizon scanning outputs for the HSS were developed in alignment to international best practices, with recommendations for technology uptake represented by a traffic light system. For the first horizon scanning exercise on cell and gene therapies, forty therapies passed the filtration step, of which eight were prioritized for further assessment. The few early reports developed were used to inform and prepare the healthcare system for their potential introduction, particularly in terms of the need to develop health and funding policies.
ConclusionsEarly assessment of prioritized topics has provided support for strategic efforts within the Ministry of Health. Given that ACE's horizon scanning program is still in its infancy, the framework will continue to evolve to ensure relevance to our stakeholders so that it remains fit for purpose for our healthcare system.
Assessment of technical errors and validation processes in economic models submitted by the company for NICE technology appraisals
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- Demi Radeva, Gareth Hopkin, Elias Mossialos, John Borrill, Leeza Osipenko, Huseyin Naci
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- Published online by Cambridge University Press:
- 03 July 2020, pp. 311-316
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Background
Economic models play a central role in the decision-making process of the National Institute for Health and Care Excellence (NICE). Inadequate validation methods allow for errors to be included in economic models. These errors may alter the final recommendations and have a significant impact on outcomes for stakeholders.
ObjectiveTo describe the patterns of technical errors found in NICE submissions and to provide an insight into the validation exercises carried out by the companies prior to submission.
MethodsAll forty-one single technology appraisals (STAs) completed in 2017 by NICE were reviewed and all were on medicines. The frequency of errors and information on their type, magnitude, and impact was extracted from publicly available NICE documentation along with the details of model validation methods used.
ResultsTwo STAs (5 percent) had no reported errors, nineteen (46 percent) had between one and four errors, sixteen (39 percent) had between five and nine errors, and four (10 percent) had more than ten errors. The most common errors were transcription errors (29 percent), logic errors (29 percent), and computational errors (25 percent). All STAs went through at least one type of validation. Moreover, errors that were notable enough were reported in the final appraisal document (FAD) in eight (20 percent) of the STAs assessed but each of these eight STAs received positive recommendations.
ConclusionsTechnical errors are common in the economic models submitted to NICE. Some errors were considered important enough to be reported in the FAD. Improvements are needed in the model development process to ensure technical errors are kept to a minimum.
Projecting effectiveness after ending a randomized controlled trial: a two-state Markov microsimulation model
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- Fei Yuan, Shrikant I. Bangdiwala, Wesley Tong, Andre Lamy
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- Published online by Cambridge University Press:
- 03 August 2020, pp. 317-324
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Objective
To investigate the behavior of restricted mean survival time (RMST) and designs of a two-state Markov microsimulation model through a 2 × 4 × 2 full factorial experiment.
MethodBy projecting patient-wise 15-year-post-trial survival, we estimated life-year-gained between an intervention and a control group using data from the Cardiovascular Outcomes for People Using Anticoagulation Strategies Study (COMPASS). Projections considered either in-trial events or post-trial medications. They were compared based on three factors: (i) choice of probability of death, (ii) lengths of cycle, and (iii) usage of half-a-cycle age correction. Three-way analysis of variance and post-hoc Tukey's Honest Significant Difference test compared means among factors.
ResultsWhen both in-trial events and post-trial study medications were considered, monthly, quarterly, or semiannually were not different from one other in projected life-year-gained. However, the annual one was different from the others: mean and 95 percent confidence interval 252.2 (190.5–313.9) days monthly, 251.8 (192.0–311.6) quarterly, 249.1 (189.7–308.5) semiannually, and 240.8 (178.5–303.1) annually. The other two factors also impacted life-year-gained: background probability (269.1 [260.3–277.9] days projected with REACH-based-probabilities, 227.7 [212.6–242.8] with a USA life table); half-a-cycle age correction (245.5 [199.0–292] with correction and 251.4 [209.1–293.7] without correction). When not considering post-trial medications, only the choice of probability of death appeared to impact life-year-gained.
ConclusionFor a large trial or cohort, to optimally project life-year-gained, one should consider using (i) annual projections, (ii) life table probabilities, (iii) in-trial events, and (iv) post-trial medication use.
Exploring the identification, validation, and categorization of costs and benefits of education in mental health: The PECUNIA project
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- Irina Pokhilenko, Luca M.M. Janssen, Silvia M.A.A. Evers, Ruben M.W.A. Drost, Judit Simon, Hans-Helmut König, Valentin Brodszky, Luis Salvador-Carulla, A-La Park, William W. Hollingworth, Aggie T.G. Paulus
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- Published online by Cambridge University Press:
- 28 May 2020, pp. 325-331
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Background
Mental health problems can lead to costs and benefits in other sectors (e.g. in the education sector) in addition to the healthcare sector. These related costs and benefits are known as intersectoral costs and benefits (ICBs). Although some ICBs within the education sector have been identified previously, little is known about their extensiveness and transferability, which is crucial for their inclusion in health economics research.
ObjectivesThe aim of this study was to identify ICBs in the education sector, to validate the list of ICBs in a broader European context, and to categorize the ICBs using mental health as a case study.
MethodsPreviously identified ICBs in the education sector were used as a basis for this study. Additional ICBs were extracted from peer-reviewed literature in PubMed and grey literature from six European countries. A comprehensive list of unique items was developed based on the identified ICBs. The list was validated by surveying an international group of educational experts. The survey results were used to finalize the list, which was categorized according to the care atom.
ResultsAdditional ICBs in the education sector were retrieved from ninety-six sources. Fourteen experts from six European countries assessed the list for completeness, clarity, and relevance. The final list contained twenty-four ICBs categorized into input, throughput, and output.
ConclusionBy providing a comprehensive list of ICBs in the education sector, this study laid further foundations for the inclusion of important societal costs in health economics research in the broader European context.
Benchmarking health technology assessment agencies—methodological challenges and recommendations
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- Ting Wang, Iga Lipska, Neil McAuslane, Lawrence Liberti, Anke Hövels, Hubert Leufkens
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- 08 September 2020, pp. 332-348
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Objectives
The objectives of the study were to establish a benchmarking tool to collect metrics to enable increased clarity regarding the differences and similarities across health technology assessment (HTA) agencies, to assess performance within and across HTA agencies, identify areas in the HTA processes in which time is spent and to enable ongoing performance improvement.
MethodsCommon steps and milestones in the HTA process were identified for meaningful benchmarking among agencies. A benchmarking tool consisting of eighty-six questions providing information on HTA agency organizational aspects and information on individual new medicine review timelines and outcomes was developed with the input of HTA agencies and validated in a pilot study. Data on 109 HTA reviews from five HTA agencies were analyzed to demonstrate the utility of this tool.
ResultsThis study developed an HTA benchmarking methodology, comparative metrics showed considerable differences among the median timelines from assessment and appraisal to final HTA recommendation for the five agencies included in this analysis; these results were interpreted in conjunction with agency characteristics.
ConclusionsIt is feasible to find consensus among HTA agencies regarding the common milestones of the review process to map jurisdiction-specific processes against agreed metrics. Data on characteristics of agencies such as their scope and remit enabled results to be interpreted in the appropriate local context. This benchmarking tool has promising potential utility to improve the transparency of the review process and to facilitate both quality assurance and performance improvement in HTA agencies.
Assessment
Evaluation of 3D printing costs in surgery: a systematic review
- Carole Serrano, Sarah Fontenay, Hélène van den Brink, Judith Pineau, Patrice Prognon, Nicolas Martelli
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- Published online by Cambridge University Press:
- 03 June 2020, pp. 349-355
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Objectives
The use of three-dimensional (3D) printing in surgery is expanding and there is a focus on comprehensively evaluating the clinical impact of this technology. However, although additional costs are one of the main limitations to its use, little is known about its economic impact. The purpose of this systematic review is to identify the costs associated with its use and highlight the first quantitative data available.
MethodsA systematic literature review was conducted in the PubMed and Embase databases and in the National Health Service Economic Evaluation Database (NHS EED) at the University of York. Studies that reported an assessment of the costs associated with the use of 3D printing for surgical application and published between 2009 and 2019, in English or French, were included.
ResultsNine studies were included in our review. Nine types of costs were identified, the three main ones being printing material costs (n = 6), staff costs (n = 3), and operating room costs (n = 3). The printing cost ranged from less than U.S. dollars (USD) 1 to USD 146 (in USD 2019 values) depending on the criteria used to calculate this cost. Three studies evaluated the potential savings generated by the use of 3D printing technology in surgery, based on operating time reduction.
ConclusionThis literature review highlights the lack of reliable economic data on 3D printing technology. Nevertheless, this review makes it possible to identify expenditures or items that should be considered in order to carry out more robust studies.
Measuring patient satisfaction with video consultation: a systematic review of assessment tools and their measurement properties
- Esther Z. Barsom, Ewout van Hees, Willem A. Bemelman, Marlies P. Schijven
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- Published online by Cambridge University Press:
- 23 June 2020, pp. 356-362
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Background
Video consultation (VC) is considered promising in delivering healthcare closer to the patient and improving patient satisfaction. Indeed, providing care-at-distance via VC is believed to be promising for some situations and patients, serving their needs without associated concomitant costs. In order to assess implementation and perceived benefits, patient satisfaction is frequently measured. Measuring patient satisfaction with VC in healthcare is often performed using quantitative and qualitative outcome analysis. As studies employ different surveys, pooling of data on the topic is troublesome. This systematic review critically appraises, summarizes, and compares available questionnaires in order to identify the most suitable questionnaire for qualitative outcome research using VC in clinical outpatient care.
MethodsPubMed, Embase, and Cochrane were searched for relevant articles using predefined inclusion criteria. Methodological quality appraisal of yielded questionnaires to assess VC was performed using the validated COSMIN guideline.
ResultsThis systematic search identified twelve studies that used ten different patient satisfaction questionnaires. The overall quality of nine questionnaires was rated as “inadequate” to “doubtful” according to the COSMIN criteria. None of the questionnaires retrieved completed a robust validation process for the purpose of use.
Conclusion and recommendationsAlthough high-quality studies on measurement properties of these questionnaires are scarce, the questionnaire developed by Mekhjian has the highest methodological quality achieving validity on internal consistency and the use of a large sample size. Moreover, this questionnaire can be used across healthcare settings. This finding may be instrumental in further studies measuring patient satisfaction with VC.
Effectiveness, efficacy, and safety of wearable cardioverter-defibrillators in the treatment of sudden cardiac arrest – Results from a health technology assessment
- Pamela Aidelsburger, Janine Seyed-Ghaemi, Christian Guinin, Andreas Fach
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- Published online by Cambridge University Press:
- 30 June 2020, pp. 363-371
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Objectives
To assess the effectiveness, efficacy, and safety of a wearable cardioverter-defibrillator (WCD) in adult persons with high risk for sudden cardiac arrest and for which an implantable cardioverter is currently not applicable.
MethodsWe performed a systematic literature search in Medline, Embase, Cochrane Library, and CRD-databases. Study selection was performed by two reviewers independently. Data were presented quantitatively; due to heterogeneity of studies no meta-analysis was performed.
ResultsOne randomized-controlled trial (RCT), one non-randomized comparative trial, and forty-four non-comparative trials were included. The RCT reported an overall mortality of 3.1 percent in the WCD group versus 4.9 percent in controls (relative risk [RR]: .64; 95 percent confidence interval [CI], .43–.98, p = .04), but no significant effect on arrhythmia-related mortality. The RR for arrhythmia-related mortality amounted to .67 (95 percent CI, .37–1.21, p = .18) as assessed in the RCT. Appropriate shocks were observed in 1.3 percent of patients in both comparative studies, and inappropriate shocks in .6 percent of patients in the RCT. Termination of ventricular tachycardia (VT) or ventricular fibrillation (VF) was successful in 75 to 100 percent of appropriate shocks in all studies. Adverse events assessed in the RCT showed a lower incidence of shortness of breath (38.8 percent vs. 45.3 percent; p = .004), higher incidence of rash at any location (15.3 percent vs. 7.1 percent; p < .001), and higher incidence of itching at any location (17.2 percent vs. 6.4 percent; p < .001) for WCD.
ConclusionsAvailable evidence demonstrates that the WCD detects and terminates VT/VF events reliably and shows a high rate of appropriate shocks in mixed patient populations. Data of large registries confirm that the WCD is a safe intervention.
Review of real-world evidence studies in type 2 diabetes mellitus: Lack of good practices
- Véronique Lambert-Obry, Jean-Philippe Lafrance, Michelle Savoie, Sandrine Henri, Jean Lachaine
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- Published online by Cambridge University Press:
- 24 June 2020, pp. 372-379
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Objectives
Unlike randomized controlled trials, lack of methodological rigor is a concern about real-world evidence (RWE) studies. The objective of this study was to characterize methodological practices of studies collecting pharmacoeconomic data in a real-world setting for the management of type 2 diabetes mellitus (T2DM).
MethodsA systematic literature review was performed using the PICO framework: population consisted of T2DM patients, interventions and comparators were any intervention for T2DM care or absence of intervention, and outcomes were resource utilization, productivity loss or utility. Only RWE studies were included, defined as studies that were not clinical trials and that collected de novo data (no retrospective analysis).
ResultsThe literature search identified 1,158 potentially relevant studies, among which sixty were included in the literature review. Many studies showed a lack of transparency by not mentioning the source for outcome and exposure measurement, source for patient selection, number of study sites, recruitment duration, sample size calculation, sampling method, missing data, approbation by an ethics committee, obtaining patient's consent, conflicts of interest, and funding. A significant proportion of studies had poor quality scores and was at high risk of bias.
ConclusionsRWE from T2DM studies lacks transparency and credibility. There is a need for good procedural practices that can increase confidence in RWE studies. Standardized methodologies specifically adapted for RWE studies collecting pharmacoeconomic data for the management of T2DM could help future reimbursement decision making in this major public health problem.
Quality of economic evaluations of ventricular assist devices: A systematic review
- Sarah Fontenay, Lionel Catarino, Soumeya Snoussi, Hélène van den Brink, Judith Pineau, Patrice Prognon, Nicolas Martelli
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- Published online by Cambridge University Press:
- 18 June 2020, pp. 380-387
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Objective
Because of a lack of suitable heart donors, alternatives to transplantation are required. These alternatives can have high costs. The aim of this study was to perform a systematic review of cost-effectiveness studies of ventricular assist devices (VADs) and to assess the level of evidence of relevant studies. The purpose was not to present economic findings.
MethodsA systematic review was performed using four electronic databases to identify health economic evaluation studies dealing with VADs. The methodological quality and reporting quality of the studies was assessed using three different tools, the Drummond, Cooper, and CHEERS (Consolidated Health Economic Evaluation Reporting Standards) checklists.
ResultsOf the 1,258 publications identified, thirteen articles were included in this review. Twelve studies were cost–utility analyses and one was a cost-effectiveness analysis. According to the Cooper hierarchy scale, the quality of the data used was heterogeneous. The level of evidence used for clinical effect sizes, safety data, and baseline clinical data was of poor quality. In contrast, cost data were of high quality in most studies. Quality of reporting varied between studies, with an average score of 17.4 (range 15–19) according to the CHEERS checklist.
ConclusionThe current study shows that the quality of clinical data used in economic evaluations of VADs is rather poor in general. This is a concern that deserves greater attention in the process of health technology assessment of medical devices.
Real-world evidence use in assessments of cancer drugs by NICE
- Ash Bullement, Tanja Podkonjak, Mark J. Robinson, Eugene Benson, Ross Selby, Anthony J. Hatswell, Gemma E. Shields
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- Published online by Cambridge University Press:
- 10 July 2020, pp. 388-394
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Objective
To establish how real-world evidence (RWE) has been used to inform single technology appraisals (STAs) of cancer drugs conducted by the National Institute for Health and Care Excellence (NICE).
MethodsSTAs published by NICE from April 2011 to October 2018 that evaluated cancer treatments were reviewed. Information regarding the use of RWE to directly inform the company-submitted cost-effectiveness analysis was extracted and categorized by topic. Summary statistics were used to describe emergent themes, and a narrative summary was provided for key case studies.
ResultsMaterials for a total of 113 relevant STAs were identified and analyzed, of which nearly all (96 percent) included some form of RWE within the company-submitted cost-effectiveness analysis. The most common categories of RWE use concerned the health-related quality of life of patients (71 percent), costs (46 percent), and medical resource utilization (40 percent). While sources of RWE were routinely criticized as part of the appraisal process, we identified only two cases where the use of RWE was overtly rejected; hence, in the majority of cases, RWE was accepted in cancer drug submissions to NICE.
DiscussionRWE has been used extensively in cancer submissions to NICE. Key criticisms of RWE in submissions to NICE are seldom regarding the use of RWE in general; instead, these are typically concerned with specific data sources and the applicability of these to the decision problem. Within an appropriate context, RWE constitutes an extremely valuable source of information to inform decision making; yet the development of best practice guidelines may improve current reporting standards.
Collaboration applications for mixed home care — A systematic review of evaluations and outcomes
- Madeleine Renyi, Ulrike Lindwedel-Reime, Lisa Blattert, Frank Teuteberg, Christophe Kunze
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- Published online by Cambridge University Press:
- 20 July 2020, pp. 395-403
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Objectives
Mixed home care, in which informal and professional actors work closely together, contributes significantly to ensuring home care up to old age. In this context, collaboration applications can considerably enhance the interactions among caregivers. However, although much research is conducted on need and requirement analyses of such applications, little is known about their introduction and use in care models. The purpose of this contribution is to identify studies that evaluate collaboration applications for mixed home care and compare their outcomes.
MethodsTo identify literature on mixed home care collaboration applications (mHCA) and their evaluation, a systematic literature review was conducted in five bibliographic databases covering the years 2008 through 2019. The results were supplemented by a search in the meta-database Google Scholar. The evaluation approaches of the studies were analyzed and results compared by using the NASSS framework. Finally, a context concretized model was derived which summarizes interrelations.
ResultsTwelve qualitative studies evaluating eleven applications could be identified. They report on increased competency in self-management, psychological relatedness, involvement, and understanding. However, most studies conclude that large scale platform tests are still needed to prove significant changes in care processes, communication, or organization.
ConclusionAmong other things, their implementation is rather difficult due to the specifics of the target group. To enable a more targeted and successful implementation, it might be helpful to classify care networks beforehand and assess their communication behavior and needs. To prove the added value of mHCAs standardized assessment tools should be used.
Impact of rarity on Canadian oncology health technology assessment and funding
- James Keech, Wei Fang Dai, Maureen Trudeau, Rebecca E. Mercer, Rohini Naipaul, Frances C. Wright, Sarah E. Ferguson, Gail Darling, Scott Gavura, Andrea Eisen, C. Tom Kouroukis, Jaclyn Beca, Kelvin K.W. Chan
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- Published online by Cambridge University Press:
- 11 August 2020, pp. 404-409
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Objectives
The pan-Canadian Oncology Drug Review (pCODR) evaluates new cancer drugs for public funding recommendations. While pCODR's deliberative framework evaluates overall clinical benefit and includes considerations for exceptional circumstances, rarity of indication is not explicitly addressed. Given the high unmet need that typically accompanies these indications, we explored the impact of rarity on oncology HTA recommendations and funding decisions.
MethodsWe examined pCODR submissions with final recommendations from 2012 to 2017. Incidence rates were calculated using pCODR recommendation reports and statistics from the Canadian Cancer Society. Indications were classified as rare if the incidence rate was lower than 1/100,000 diagnoses, a definition referenced by the Canadian Agency for Drugs and Technologies in Health. Each pCODR final report was examined for the funding recommendation/justification, level of supporting evidence (presence of a randomized control trial [RCT]), and time to funding (if applicable).
ResultsOf the ninety-six pCODR reviews examined, 16.6 percent were classified as rare indications per above criteria. While the frequency of positive funding recommendations were similar between rare and nonrare indication (78.6 vs. 75 percent), rare indications were less likely to be presented with evidence from RCT (50 vs. 90 percent). The average time to funding did not differ significantly across provinces.
ConclusionRare indications appear to be associated with weaker clinical evidence. There appears to be no association between rarity, positive funding recommendations, and time to funding. Further work will evaluate factors associated with positive recommendations and the real-world utilization of funded treatments for rare indications.
Evaluation of precision medicine assessment reports of the Belgian healthcare payer to inform reimbursement decisions
- Laurenz Govaerts, Anouk Waeytens, Walter Van Dyck, Steven Simoens, Isabelle Huys
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- Published online by Cambridge University Press:
- 07 September 2020, pp. 410-417
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Introduction
Precision medicines rely on companion diagnostics to identify patient subgroups eligible for receiving the pharmaceutical product. Until recently, the Belgian public health payer, RIZIV-INAMI, assessed precision medicines and companion diagnostics separately for reimbursement decisions. As both components are considered co-dependent technologies, their assessment should be conducted jointly from a health technology assessment (HTA) perspective. As of July 2019, a novel procedure was implemented accommodating for this joint assessment practice. The aim of this research was to formulate recommendations to improve the assessment in the novel procedure.
MethodsThis study evaluated the precision medicine assessment reports of RIZIV-INAMI of the last 5 years under the former assessment procedure. The HTA framework for co-dependent technologies developed by Merlin et al. for the Australian healthcare system was used as a reference standard in this evaluation. Criteria were scored as either present or not present.
ResultsThirteen assessment reports were evaluated. Varying scores between reports were obtained for the domain establishing the co-dependent relationship between diagnostic and pharmaceutical. Domains evaluating the clinical utility of the biomarker and the cost-effectiveness performed poorly, whereas the budget impact and the transfer of trial data to the local setting performed well.
RecommendationsBased on these results we recommend three amendments for the novel procedure. (i) The implementation of the linked evidence approach when direct evidence of clinical utility is not present, (ii) incorporation of a bias assessment tool, and (iii) further specify guidelines for submission and assessment to decrease the variability of reported evidence between assessment reports.
Exploring the identification, validation, and categorization of the cost and benefits of criminal justice in mental health: the PECUNIA project
- Luca M. M. Janssen, Irina Pokhilenko, Silvia M. A. A. Evers, Aggie T. G. Paulus, Judit Simon, Hans-Helmut König, Valentin Brodszky, Luis Salvador-Carulla, A-La Park, W. Hollingworth, Ruben M. W. A. Drost, on behalf of the PECUNIA group
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- Published online by Cambridge University Press:
- 27 July 2020, pp. 418-425
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Background
Mental health disorders and their treatments produce significant costs and benefits in both healthcare and non-healthcare sectors. The latter are often referred to as intersectoral costs and benefits (ICBs). Little is known about healthcare-related ICBs in the criminal justice sector and how to include these in health economics research.
ObjectivesThe triple aim of this study is (i) to identify healthcare-related ICBs in the criminal justice sector, (ii) to validate the list of healthcare-related ICBs in the criminal justice sector on a European level by sector-specific experts, and (iii) to classify the identified ICBs.
MethodsA scientific literature search in PubMed and an additional grey literature search, carried out in six European countries, were used to retrieve ICBs. In order to validate the international applicability of the ICBs, a survey was conducted with an international group of experts from the criminal justice sector. The list of criminal justice ICBs was categorized according to the PECUNIA conceptual framework.
ResultsThe full-text analysis of forty-five peer-reviewed journal articles and eleven grey literature sources resulted in a draft list of items. Input from the expert survey resulted in a final list of fourteen unique criminal justice ICBs, categorized according to the care atom.
ConclusionThis study laid further foundations for the inclusion of important societal costs of mental health-related interventions within the criminal justice sector. More research is needed to facilitate the further and increased inclusion of ICBs in health economics research.
Policy
NICE's evaluations of medicines authorized by EMA with conditional marketing authorization or under exceptional circumstances
- Pilar Pinilla-Dominguez, Huseyin Naci, Leeza Osipenko, Elias Mossialos
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- Published online by Cambridge University Press:
- 25 June 2020, pp. 426-433
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Objectives
To investigate the impact of the uncertainty stemming from products with European conditional marketing authorization (CMA) or authorization in exceptional circumstances (AEC) on the National Institute for Health and Care Excellence's (NICE) recommendations.
MethodsProducts which received CMA/AEC by European Medicines Agency (EMA) up to 1 December 2016 were identified and matched with corresponding NICE decisions issued by August 2017, the status of which was then traced to August 2019. We assessed whether the conversion of CMA to full marketing authorization triggered a review of a NICE decision. The odds of a recommendation carrying a commercial arrangement for products with and without CMA/AEC were calculated.
ResultsFifty-four products were granted CMA/AEC by EMA. NICE conducted thirty evaluations of products with CMA/AEC. Twelve products were recommended by NICE by August 2017 and fourteen by August 2019. All recommendations had an associated commercial arrangement. The odds of carrying a commercial arrangement were higher for products with CMA/AEC compared to those with full authorization. Conversions from conditional to full authorization among products not recommended by NICE did not trigger an appraisal review.
ConclusionsUncertainty, stemming from the lack of robust clinical data of products authorized with CMA/AEC, has a substantial impact on HTA recommendations, frequently requiring risk mitigation mechanisms such as commercial and data collection arrangements. Further analyses should be conducted to assess whether the benefits of early access strategies outweigh the risks for patients and the healthcare system.
Can multi-criteria decision analysis (MCDA) be implemented into real-world drug decision-making processes? A Canadian provincial experience
- Tracey-Lea Laba, Bashir Jiwani, Robert Crossland, Craig Mitton
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- Published online by Cambridge University Press:
- 07 August 2020, pp. 434-439
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Objective
To describe the implementation of multi-criteria decision analysis (MCDA) into a Canadian public drug reimbursement decision-making process, identifying the aspects of the MCDA approach, and the context that promoted uptake.
MethodsNarrative summary of case study describing the how, when, and why of implementing MCDA.
ResultsFaced with a fixed budget, a pipeline of expensive but potentially valuable drugs, and potential delays to drug decision making, the Ministry of Health (i.e., decision makers) and its independent expert advisory committee (IAB) sought alternative values-based decision processes. MCDA was considered highly compatible with current processes, but the ability as a stand-alone intervention to address issues of opportunity cost was unclear. The IAB nevertheless collaboratively voted to implement an externally developed MCDA with support from decision makers. After several months of engagement and piloting, implementation was rapid and leveraged strong pre-existing formal and informal communication networks. The IAB as a whole rates new submissions which serves as an input into the deliberative process.
ConclusionsMCDA can be a highly adaptable approach that can be implemented into a functioning drug reimbursement setting when facilitated by (i) a truly limited budget; (ii) a shared vision for change by end-users and decision makers; (iii) using pre-existing deliberative processes; and (iv) viewing the approach as a decision framework rather than the decision (when appropriate). Given the current limitations of MCDA, implementing an academically imperfect tool first and evaluating later reflects a practical solution to real-time fiscal constraints and impending delays to drug approvals that may be faced by decision makers.