Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives

Chachoua, L; Dabbous, Monique; François, Clément; Deroanne, Claude; Aballéa, Samuel; Toumi, Mondher

doi:10.3389/fmed.2020.543046

Cited by 23 publications

(24 citation statements)

References 48 publications

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“…HTA professionals are looking to more "scientific" ways to provide patients' perspectives, as with syntheses of qualitative studies to provide patient evidence (36). Patient preference studies are now being extended beyond economic studies to build clinical trial evidence for an intervention or technology (37)(38)(39)(40). Uptake in the USA has been slow (41,42).…”

Section: Are We Seeing Changes In Approach?mentioning

confidence: 99%

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Wale¹,

Chandler

Collyar³

et al. 2022

Front. Med. Technol.

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Health technology assessment (HTA) is intended to determine the value of health technologies and, once a technology is recommended for funding, bridge clinical research and practice. Understanding the values and beliefs expressed by patients and health professionals can help guide this knowledge transfer and work toward managing the expectations of end users. We gathered patient and patient group leader experiences to gain insights into the roles that patients and patient advocacy groups are playing. We argue that through partnerships and co-creation between HTA professionals, researchers and patient advocates we can strengthen the HTA process and better align with service delivery where person-centered care and shared decision making are key elements. Patient experiences and knowledge are important to the democratization of evidence and the legitimacy of HTAs. Patient preference studies are used to balance benefits with potential harms of technologies, and patient-reported outcomes (PROs) can measure what matters to patients over time. A change in culture in HTA bodies is occurring and with further transformative thinking patients can be involved in every step of the HTA process. Patients have a right to be involved in HTAs, with patients' values central to HTA deliberations on a technology and where patients can provide valuable insights to inform HTA decision-making; and in ensuring that HTA methodologies evolve. By evaluating the implementation of HTA recommendations we can determine how HTA benefits patients and their communities. Our shared commitment can positively effect the common good and provide benefits to individual patients and their communities.

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Section: Are We Seeing Changes In Approach?mentioning

confidence: 99%

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Wale¹,

Chandler

Collyar³

et al. 2022

Front. Med. Technol.

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“…In response to this, several research projects, such as the IMI PREFER project, have been initiated by drug developers, academic researchers, as well as HTA bodies and regulatory agencies. Such projects aim to investigate how patient preference studies could inform decision-making, and how such studies could be designed to meet methodological requirements of stakeholders involved in these decisions ( 27 – 29 ).…”

Section: Introductionmentioning

confidence: 99%

“…As qualitative methods provide in-depth and meaningful information from patients, their use is recommended for the development of attributes and levels. Qualitative methods with patients may reduce the potential for misspecification of attributes through overreliance on the views of experts and researchers ( 27 , 28 ). In doing so, using qualitative research for the development of attributes and levels may improve the validity of subsequent quantitative preference surveys.…”

Section: Introductionmentioning

confidence: 99%

Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

et al. 2021

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Background: Investigational and marketed drugs for the treatment of multiple myeloma (MM) are associated with a range of characteristics and uncertainties regarding long term side-effects and efficacy. This raises questions about what matters most to patients living with this disease. This study aimed to understand which characteristics MM patients find most important, and hence should be included as attributes and levels in a subsequent quantitative preference survey among MM patients.Methods: This qualitative study involved: (i) a scoping literature review, (ii) discussions with MM patients (n = 24) in Belgium, Finland, Romania, and Spain using Nominal Group Technique, (iii) a qualitative thematic analysis including multi-stakeholder discussions.Results: MM patients voiced significant expectations and hopes that treatments would extend their lives and reduce their cancer signs and symptoms. Participants however raised concerns about life-threatening side-effects that could cause permanent organ damage. Bone fractures and debilitating neuropathic effects (such as chronic tingling sensations) were highlighted as major issues reducing patients' independence and mobility. Patients discussed the negative impact of the following symptoms and side-effects on their daily activities: thinking problems, increased susceptibility to infections, reduced energy, pain, emotional problems, and vision problems. MM patients were concerned with uncertainties regarding the durability of positive treatment outcomes, and the cause, severity, and duration of their symptoms and side-effects. Patients feared short-term positive treatment responses complicated by permanent, severe side-effects and symptoms.Conclusions: This study gained an in-depth understanding of the treatment and disease-related characteristics and types of attribute levels (severity, duration) that are most important to MM patients. Results from this study argue in favor of MM drug development and individual treatment decision-making that focuses not only on extending patients' lives but also on addressing those symptoms and side-effects that significantly impact MM patients' quality of life. This study underscores a need for transparent communication toward MM patients about MM treatment outcomes and uncertainties regarding their long-term efficacy and safety. Finally, this study may help drug developers and decision-makers understand which treatment outcomes and uncertainties are most important to MM patients and therefore should be incorporated in MM drug development, evaluation, and clinical practice.

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“…Patient involvement in HTAs can complement the clinical and economic evidence that HTA bodies evaluate by providing unique patient insights [ 3 , 15 ]. Indeed, health authorities may be able to make more efficient and effective decisions when it comes to pricing and reimbursement when an understanding of the real-world benefits and harms of treatments are integrated with traditional evidence [ 3 ]. However, in our study, the impact of patient input on the assessment process was rated as low by patients in both South Korea and New Zealand and average in Taiwan.…”

Section: Discussionmentioning

confidence: 99%

“…It is thought that HTA can be strengthened by including the patient perspective, with some countries having taken significant steps to involve patients in their HTA processes [ 2 ] and incorporate their perspectives into decision-making [ 3 , 4 ].…”

Section: Introductionmentioning

confidence: 99%

Patient advocacy group involvement in health technology assessments: an observational study

Single¹,

Cabrera²,

Fifer³

et al. 2021

Res Involv Engagem

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Background In some jurisdictions, patients and patient groups may be invited to provide input when Health Technology Assessment (HTA) is used to inform decisions about which medicines should be subsidised or funded. This input can help frame the evidence from a patient perspective, address uncertainties in the evidence and interpret it for the local setting. However, there is currently no evidence linking patient involvement with positive reimbursement decisions. Aim We aimed to understand the expectations of patient involvement in the reimbursement process, especially among cancer patient advocacy groups (PAGs) in New Zealand (Aotearoa), South Korea and Taiwan. Methods We developed an online survey to help understand the role that cancer PAGs play in reimbursement processes and identify knowledge gaps about the processes that might impact the efforts of PAGs. The survey elicited the views of staff and patients affiliated with PAGs (n = 43) on current practices and how the assessment and reimbursement of new cancer drugs might be improved. Results There was variability in knowledge of the HTA assessment processes and in experience of being involved in them. Those with HTA experience were more likely to have confidence in the process. Those who had not been involved tended to have little awareness of, or frustration with, decision-making processes. Most identified cost, finances and economic assessments as key considerations in current processes. Some respondents had clear ideas about how their knowledge and involvement could improve processes to determine the value of new medicines. However, for many, a lack of information about the basis for decision making and opportunities to be involved was a barrier to identifying process improvement. Conclusions HTA is implemented primarily in countries seeking to have fair and equitable processes for funding medicines. PAGs often recognise the financial challenges of funding new medicines and share the desire for procedural fairness. The connection PAGs make between patient involvement and improved access to new medicines may be based on the belief they can add information to the evidence base, help solve problems, ensure fairness through transparency and/or influence the culture towards increased access to medicines they value.

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Use of Patient Preference Information in Benefit–Risk Assessment, Health Technology Assessment, and Pricing and Reimbursement Decisions: A Systematic Literature Review of Attempts and Initiatives

Cited by 23 publications

References 48 publications

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Can We Afford to Exclude Patients Throughout Health Technology Assessment?

Patient Preferences for Multiple Myeloma Treatments: A Multinational Qualitative Study

Patient advocacy group involvement in health technology assessments: an observational study

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