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A practical approach to communicating benefit-risk decisions of medicines to stakeholders.

ABSTRACT: PURPOSE: The importance of a framework for a systematic structured assessment of the benefits and risks has been established, but in addition, it is necessary that the benefit-risk decisions and the processes to derive those decisions are documented and communicated to various stakeholders for accountability. Hence there is now a need to find appropriate tools to enhance communication between regulators and other stakeholders, in a manner that would uphold transparency, consistency and standards. METHODS: A retrospective, non-comparative study was conducted to determine the applicability and practicality of a summary template in documenting benefit-risk assessment and communicating benefit-risk balance and conclusions for reviewers to other stakeholders. The benefit-risk (BR) Summary Template and its User Manual was evaluated by 12 reviewers within a regulatory agency in Singapore, the Health Sciences Authority (HSA). RESULTS: The BR Summary Template was found to be adequate in documenting benefits, risks, relevant summaries and conclusions, while the User Manual was useful in guiding the reviewer in completing the template. The BR Summary Template was also considered a useful tool for communicating benefit-risk decisions to a variety of stakeholders. CONCLUSIONS: The use of a template may be of value for the communicating benefit-risk assessment of medicines to stakeholders.

SUBMITTER: Leong J

PROVIDER: S-EPMC4463867 | biostudies-other | 2015

REPOSITORIES: biostudies-other

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A practical approach to communicating benefit-risk decisions of medicines to stakeholders.

Leong James J Walker Stuart S Salek Sam S

Frontiers in pharmacology 20150611

<h4>Purpose</h4>The importance of a framework for a systematic structured assessment of the benefits and risks has been established, but in addition, it is necessary that the benefit-risk decisions and the processes to derive those decisions are documented and communicated to various stakeholders for accountability. Hence there is now a need to find appropriate tools to enhance communication between regulators and other stakeholders, in a manner that would uphold transparency, consistency and st ...[more]

PMID: 26124720

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Project description:BackgroundThe importance of reporting research evidence to stakeholders in ways that balance complexity and usability is well-documented. However, guidance for how to accomplish this is less clear. We describe a method of developing and visualising dimension-specific scores for organisational context (context rank method). We explore perspectives of leaders in long-term care nursing homes (NHs) on two methods for reporting organisational context data: context rank method and our traditionally presented binary method-more/less favourable context.MethodsWe used a multimethod design. First, we used survey data from 4065 healthcare aides on 290 care units from 91 NHs to calculate quartiles for each of the 10 Alberta Context Tool (ACT) dimension scores, aggregated at the care unit level based on the overall sample distribution of these scores. This ordinal variable was then summed across ACT scores. Context rank scores were assessed for associations with outcomes for NH staff and for quality of care (healthcare aides' instrumental and conceptual research use, job satisfaction, rushed care, care left undone) using regression analyses. Second, we used a qualitative descriptive approach to elicit NH leaders' perspectives on whether the methods were understandable, meaningful, relevant, and useful. With 16 leaders, we conducted focus groups between December 2017 and June 2018: one in Nova Scotia, one in Prince Edward Island, and one in Ontario, Canada. Data were analysed using content analysis.ResultsComposite scores generated using the context rank method had positive associations with healthcare aides' instrumental research use (p < .0067) and conceptual research use and job satisfaction (p < .0001). Associations were negative between context rank summary scores and rushed care and care left undone (p < .0001). Overall, leaders indicated that data presented by both methods had value. They liked the binary method as a starting point but appreciated the greater level of detail in the context rank method.ConclusionsWe recommend careful selection of either the binary or context rank method based on purpose and audience. If a simple, high-level overview is the goal, the binary method has value. If improvement is the goal, the context rank method will give leaders more actionable details.

Project description:BackgroundRare disease patients and caregivers face uncommon, serious, debilitating conditions often characterised by poor prognosis and limited treatment options. This study aimed to explore what they consider of value when choosing between hypothetical therapeutic options and to quantify both their benefit-risk preferences and the influence of disease context.MethodsA mixed-methods survey with patients and caregivers was conducted in the United Kingdom across a range of rare diseases. Discrete-choice experiments that compared hypothetical treatment profiles of benefits and risks were used to measure respondent preferences across a set of seven attributes related to health outcomes, safety, and process of care. Bespoke questions on current disease management and the joint use of the 12-item WHODAS 2.0 questionnaire and of two Likert scales capturing self- and proxy-assessed disease-induced threat to life and impairment were implemented to describe disease context. Additionally, qualitative insights on the definitions of value and risk were collected from respondents.ResultsFinal study sample included 721 patients and 152 informal caregivers, across 52 rare diseases. When choosing between hypothetical novel treatments for rare diseases, respondents attributed most importance to drug response, risk of serious side effects, and the ability to conduct usual activities while on treatment. In contrast, attributes related to treatment modalities were the least important. Respondents expressed a willingness to accept risks in hopes of finding some benefit, such as a higher chance of drug response or greater health improvement potential. Increasing disease severity, impairment or disability, and the lack of effective therapeutic options were shown to raise significantly the willingness to gain benefit through increased risk.ConclusionsThis is the first study performing a quantitative discrete choice experiment amongst patients and caregivers across 52 rare conditions. It enables a more detailed understanding of the relationship between disease context, treatment attributes and the degree of risk respondents are willing to take to gain a specific degree of benefit. Researchers of novel therapeutics for rare diseases should be encouraged to invest in preference elicitation studies to generate rigorous patient evidence and specific regulatory guidance should be issued to acknowledge their importance and their use in marketing authorisations.

Project description:Background and aimsCommunicating information about the risks and benefits of benzodiazepines so that it is meaningful to the patient has not been previously described. This study aims to determine patient preferences regarding information received before initiating a benzodiazepine.MethodsAn online survey was distributed through social media and advertisements to Canadians ≥18 years old over a 6-month period (May-Oct 2022) to collect participant's rating of importance of statements and factors about the risk and benefits of benzodiazepines before initiating treatment using a 10-point Likert-type scale. Treatment preferences based on efficacy and risk information were also elicited. The survey was developed and pilot-tested in collaboration with an advisory committee of individuals with lived and living experience with benzodiazepine use.ResultsThirty-seven participants responded to the survey (mean age 30 years old, 81.1% identified as female). The majority of respondents had a history of anxiety (83.8%) or insomnia (32.4%), and 10 (27.0%) respondents had used a benzodiazepine. Patient counseling related to withdrawal symptoms of benzodiazepines, risk of harm in combination with other sedating agents, risk of physical and psychological dependence, and risk of effects on cognition were rated high in the importance of receiving this information before starting a benzodiazepine relative to efficacy endpoints, such as improvement in sleep parameters. When provided with information about the chance of efficacy and risk of harm, 100% would have selected cognitive behavioral therapy as the best treatment option. The most frequently reported source of medication information where patients have sought information was from the internet (25.0%), followed by doctors (21.9%) and pharmacists (18.8%).ConclusionsThis study identified patient important factors and statements viewed as important to communicate before initiating a benzodiazepine. The findings of this survey study will help inform decision-making when considering treatment options for managing anxiety or insomnia.

Project description:BackgroundWhile different ways of presenting treatment effects can affect health care decisions, little is known about which presentations best help people make decisions consistent with their own values. We compared six summary statistics for communicating coronary heart disease (CHD) risk reduction with statins: relative risk reduction and five absolute summary measures-absolute risk reduction, number needed to treat, event rates, tablets needed to take, and natural frequencies.Methods and findingsWe conducted a randomized trial to determine which presentation resulted in choices most consistent with participants' values. We recruited adult volunteers who participated through an interactive Web site. Participants rated the relative importance of outcomes using visual analogue scales (VAS). We then randomized participants to one of the six summary statistics and asked them to choose whether to take statins based on this information. We calculated a relative importance score (RIS) by subtracting the VAS scores for the downsides of taking statins from the VAS score for CHD. We used logistic regression to determine the association between participants' RIS and their choice. 2,978 participants completed the study. Relative risk reduction resulted in a 21% higher probability of choosing to take statins over all values of RIS compared to the absolute summary statistics. This corresponds to a number needed to treat (NNT) of 5; i.e., for every five participants shown the relative risk reduction one additional participant chose to take statins, compared to the other summary statistics. There were no significant differences among the absolute summary statistics in the association between RIS and participants' decisions whether to take statins. Natural frequencies were best understood (86% reported they understood them well or very well), and participants were most satisfied with this information.ConclusionsPresenting the benefits of taking statins as a relative risk reduction increases the likelihood of people accepting treatment compared to presenting absolute summary statistics, independent of the relative importance they attach to the consequences. Natural frequencies may be the most suitable summary statistic for presenting treatment effects, based on self-reported preference, understanding of and satisfaction with the information, and confidence in the decision.Clinical trials registrationISRCTN85194921.

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A practical approach to communicating benefit-risk decisions of medicines to stakeholders.

Publications

A practical approach to communicating benefit-risk decisions of medicines to stakeholders.

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