‘Mapping’ Health State Utility Values from Non-preference-Based Measures: A Systematic Literature Review in Rare Diseases

Meregaglia, Michela; Whittal, Amanda; Nicod, Elena; Drummond, Michael

doi:10.1007/s40273-020-00897-4

Cited by 14 publications

(12 citation statements)

References 79 publications

(223 reference statements)

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“…The range of HSUVs derived in diseases with similar characteristics can be used as a benchmark to validate results of such new RD-specific tools [ 43 ] Probably not feasible: This approach is valuable, but developing such preference-based algorithms for additional PROMs would require significant time and resources Need for recognition of the limited number of preference-based disease-specific PROMs, which makes it very difficult to derive HSUVs in RDs Creating new disease-specific PROMs Advantages: Can be well-tailored to disease; high possibility of capturing meaningful outcomes PROMs are time and resource intensive to create; it cannot realistically be done for every RD and manifestation [ 5 , 23 , 36 , 44 ] It is important to use innovative and flexible PROM strategies for RDs, for instance, computer-assisted technology can ease the process by streamlining responses, reducing the burden on patients and allowing multi-site data collection [ 11 , 13 , 15 , 24 ] Feasible with additional challenges: Creating new PROMs requires significant time and resources, particularly for RD populations Need for recognition that innovative and flexible PROM strategies is required The natural history of most RDs is poorly understood, making it hard to identify concepts of interest [ 14 ] All available sources of information should be used to understand the natural history of an RD [ 11 ] Incorporate patient voice early and throughout process of PROM development [ 5 , 7 , 46 , 47 ] Focus on the most common symptoms and impact that seem to be most important to patients [ 49 ] Feasible: The effort required to gather and use all sources of possible information is valuable, as the lack of understanding of the natural history is a key challenge Probably feasible: Incorporation of the patient voice requires willingness, participation and time commitments of stakeholders Need acceptance of a variety of sources of information Effective approaches to developing PROMs are not always clear [ 38 ] Take into account existing development guidance (e.g. FDA) and examples of PROM development [ 38 , 50 ] Feasible: Referring to and following any available high-quality guidance is only a matter of takin...…”

Section: Resultsmentioning

confidence: 99%

“…EQ-5D) are often preferred by HTA bodies. The ‘mapping’ technique can potentially allow the conversion of disease-specific PROM responses onto HSUVs derived from generic PROMs, but the lack of concordance between disease-specific and generic PROMs means it is complicated to conduct a mapping exercise in practice, and their degree of ‘overlap’ should be assessed in advance using proper correlation tests [ 31 , 50 ].…”

Section: Resultsmentioning

confidence: 99%

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The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Whittal

Meregaglia

Nicod

2021

Patient

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Background Patient-reported outcome measures (PROMs) are used in health technology assessment (HTA) to measure patient experiences with disease and treatment, allowing a deeper understanding of treatment impact beyond clinical endpoints. Developing and administering PROMs for rare diseases poses unique challenges because of small patient populations, disease heterogeneity, lack of natural history knowledge, and short-term studies. Objective This research aims to identify key factors to consider when using different types of PROMs in HTA for rare disease treatments (RDTs). Methods A scoping review of scientific and grey literature was conducted, with no date or publication type restrictions. Information on the advantages of and the challenges and potential solutions when using different types of PROMs for RDTs, including psychometric properties, was extracted and synthesized. Results Of 79 records from PubMed, 32 were included, plus 12 records from the grey literature. PROMs for rare diseases face potential data collection and psychometric challenges resulting from small patient populations and disease heterogeneity. Generic PROMs are comparable across diseases but not sensitive to disease specificities. Disease-specific instruments are sensitive but do not exist for many rare diseases and rarely provide the utility values required by some HTA bodies. Creating new PROMs is time and resource intensive. Potential solutions include pooling data (multi-site/international data collection), using computer-assisted technology, or using generic and disease-specific PROMs in a complementary way. Conclusions PROMs are relevant in HTA for RDTs but pose a number of difficulties. A deeper understanding of the potential advantages of and the challenges and potential solutions for each can help manage these difficulties.

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Whittal

Meregaglia

Nicod

2021

Patient

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“…However, there was little evidence of patient involvement in the OBMEA case studies. A few of the OBMEAs indicated the plan to collect patient-reported outcomes, which are considered particularly important in rare diseases [ 19 , 20 ]. However, there is often no suitable patient-reported outcome for a rare disease (as was the case for nusinersen in England) and thus other mechanisms are needed to capture patients’ perspectives.…”

Section: Discussionmentioning

confidence: 99%

Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

et al. 2021

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Background and Objective Enthusiasm for the use of outcomes-based managed entry agreements (OBMEAs) to manage uncertainties apparent at the time of appraisal/pricing and reimbursement of new medicines has waned over the past decade, as challenges in establishment, implementation and re-appraisal have been identified. With the recent advent of innovative treatments for rare diseases that have uncertainties in the clinical evidence base, but which could meet a high unmet need, there has been renewed interest in the potential of OBMEAs. The objective of this research was to review the implementation of OBMEAs for two case studies across countries in the European Union, Australia and Canada, to identify good practices that could inform development of tools to support implementation of OBMEAs. Methods To investigate how OBMEAs are being implemented with rare disease treatments, we collected information from health technology assessment/payer experts in countries that had implemented OBMEAs for either nusinersen in spinal muscular atrophy or tisagenlecleucel in two cancer indications. Operational characteristics of the OBMEAs that were publicly available were documented. Then, the experts discussed issues in implementing these OBMEAs and specific approaches taken to overcome challenges. Results The OBMEAs identified were based on individual outcomes to ensure appropriate use, manage continuation of treatment and in two cases linked to payment schedules, or they were population based, coverage with evidence development. For nusinersen, population-based OBMEAs are documented in Belgium, England and the Netherlands and individual-based schemes in Bulgaria, Ireland, Italy and Lithuania. For tisagenlecleucel, there were population-based schemes in Australia, Belgium, England and France and individual-based schemes in Italy and Spain. Comparison of the OBMEA constructs showed some clear published frameworks and clarity of the uncertainties to be addressed that were similar across countries. Agreements were generally made between the marketing authorisation holder and the payer with involvement of expert physicians. Only England and the Netherlands involved patients. Italy used its long-established, national, web-based, treatment-specific data collection system linked to reimbursement and Spain has just developed such a national treatment registry system. Other countries relied on a variety of data collection systems (including clinical registries) and administrative data. Durations of agreements varied for these treatments as did processes for interim reporting. The processes to ensure data quality, completeness and sufficiency for re-analysis after coverage with evidence development were not always clear, neither were analysis plans. Conclusions These case studies have shown that important information about the constructs of OBMEAs for rare disease treatments are publicly available, and for some jurisdictions, interim re...

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“…Since societal valuations still need to be linked to measurements from patients who are experiencing the conditions and treatments, utility scores may be attached to diseasespecific questionnaires. This may be a relatively objective and sensitive approach, but tends to overestimate utility in poor health states [76]. Instead, generic questionnaires have been developed that span a wide range of health and QoL domains, and for which societal valuations are available to calculate utility scores [77][78][79][80].…”

Section: Macro-level Of Decision-makingmentioning

confidence: 99%

Implications of response shift for micro-, meso-, and macro-level healthcare decision-making using results of patient-reported outcome measures

et al. 2021

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Purpose Results of patient-reported outcome measures (PROMs) are increasingly used to inform healthcare decision-making. Research has shown that response shift can impact PROM results. As part of an international collaboration, our goal is to provide a framework regarding the implications of response shift at the level of patient care (micro), healthcare institute (meso), and healthcare policy (macro). Methods Empirical evidence of response shift that can influence patients’ self-reported health and preferences provided the foundation for development of the framework. Measurement validity theory, hermeneutic philosophy, and micro-, meso-, and macro-level healthcare decision-making informed our theoretical analysis. Results At the micro-level, patients’ self-reported health needs to be interpreted via dialogue with the clinician to avoid misinterpretation of PROM data due to response shift. It is also important to consider the potential impact of response shift on study results, when these are used to support decisions. At the meso-level, individual-level data should be examined for response shift before aggregating PROM data for decision-making related to quality improvement, performance monitoring, and accreditation. At the macro-level, critical reflection on the conceptualization of health is required to know whether response shift needs to be controlled for when PROM data are used to inform healthcare coverage. Conclusion Given empirical evidence of response shift, there is a critical need for guidelines and knowledge translation to avoid potential misinterpretations of PROM results and consequential biases in decision-making. Our framework with guiding questions provides a structure for developing strategies to address potential impacts of response shift at micro-, meso-, and macro-levels.

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‘Mapping’ Health State Utility Values from Non-preference-Based Measures: A Systematic Literature Review in Rare Diseases

Cited by 14 publications

References 79 publications

The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

The Use of Patient-Reported Outcome Measures in Rare Diseases and Implications for Health Technology Assessment

Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and Tisagenlecleucel

Implications of response shift for micro-, meso-, and macro-level healthcare decision-making using results of patient-reported outcome measures

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