Standardizing the Benefit-Risk Assessment of New Medicines

@article{Liberti2012StandardizingTB,
  title={Standardizing the Benefit-Risk Assessment of New Medicines},
  author={Lawrence Liberti and James Neil McAuslane and Stuart R Walker},
  journal={Pharmaceutical Medicine},
  year={2012},
  volume={25},
  pages={139-146}
}
Pharmaceutical scientists and healthcare professionals participating in the development of new medicines bring important practical perspectives to the evaluation of benefits and risks. Within pharmaceutical companies, these specialists can serve to inform the benefit-risk (BR) assessment process, particularly in the latter stages of drug development. However, pharmaceutical scientists may not be aware of the current state of activity in the ongoing efforts to standardize the BR assessment… 

The Potential Role of Individual-Level Benefit-Risk Assessment in Treatment Decision Making: A DIA Study Endpoints Community Workstream

TLDR
Various approaches as to how this information could be collected, including via patient-reported outcome measures, open-ended questioning, and stated-preference methods are presented, and the utility of this information for various stakeholders is discussed.

The Emergence of Regulatory Science in Pharmaceutical Medicine

TLDR
It is concluded that the emerging field of regulatory science is likely to influence the future shaping and implementation of laws and regulations in pharmaceutical medicine.

Balancing benefit and risk of medicines: a systematic review and classification of available methodologies

TLDR
This work systematically collected, appraised and classified available benefit–risk methodologies to facilitate and inform their future use.

Multi-criteria decision analysis for health technology assessment: addressing methodological challenges to improve the state of the art

TLDR
A critical review of published studies on MCDA in the context of HTA is provided by assessing their methodological quality and summarising methodological challenges, highlighting the need for advancement in robust methodologies, procedures and tools to improve methodological quality ofMCDA in HTA studies.

The Cost of Paying Attention: Cognitive Resource Scarcity and Investor Activity Around FDA Announcements

TLDR
A sample of FDA resolutions over a period from 2006 to 2014, consisting of 133 cases, brings about an interesting picture of price adjustments following the FDA announcements, where the FDA has not approved the drug application, as well as in the cases of approval, stock prices tended to decline after the announcement.

The Cost of Paying Attention : Cognitive Resource Scarcity and Formative Event Processing

Investigating limited attention in financial markets has threefold merit: in real markets, investors may learn from experience; market prices may erase individual violations of rationality; investors

References

SHOWING 1-10 OF 26 REFERENCES

Relative efficacy of drugs: an emerging issue between regulatory agencies and third-party payers

TLDR
The current political background to the RE debate is described and the scientific and methodological challenges as they relate to RE assessment are presented.

Benefit-Risk Appraisal of Medicines: A Systematic Approach to Decision-making

TLDR
This work focuses on the development of a benefit-risk framework for regulatory review of new medicines, and the development and application of a Benefit-risk Assessment Model Based on Multi-Criteria Decision Analysis.

Measuring Benefit and Balancing Risk: Strategies for the Benefit–Risk Assessment of New Medicines in a Risk‐Averse Environment

TLDR
The framework identifies the essential elements that both regulators and companies should consider throughout the development, review, and postmarketing experience of new medicines in order to apply a consistent approach toward the assessment of BR balance.

Refining the Benefit–Risk Framework for the Assessment of Medicines: Valuing and Weighting Benefit and Risk Parameters

TLDR
This forum focused on identifying challenges and approaches to the assignment of values and weightings using a case study applied to a hypothetical medicinal product.

A Multiattribute Model for Evaluating the Benefit-Risk Profiles of Treatment Alternatives

TLDR
A representational model based on multicriteria decision analysis that can incorporate both evaluative judgments from different perspectives and quantitative data to inform tradeoffs between multiple benefit and multiple risk elements in a logically consistent and transparent manner is proposed.

Balancing early market access to new drugs with the need for benefit/risk data: a mounting dilemma

TLDR
The current demands by stakeholders and the scientific and regulatory issues at stake are summarized, existing and emerging regulatory approaches are described, and future directions are speculated on, such as evolution of the current regulatory model from a one-off marketing authorization to a life-cycle approach.

Development of a Framework for Enhancing the Transparency, Reproducibility and Communication of the Benefit–Risk Balance of Medicines

TLDR
The Benefit Risk Action Team framework is a set of processes and tools for selecting, organizing, summarizing, and interpreting data that is relevant to decisions based on benefit–risk assessments that provides a standardized yet flexible platform for incorporating study outcomes and preference weights.

Benefit-risk methodology project: work package 2 report: applicability of current tools and processes for regulatory benefit-risk assessment

TLDR
The Lynd-O’Brien study dealt with just two drugs, and single risk and benefit criteria, but an opportunity exists for combining these two approaches to assist regulators in simultaneously looking at the effects of both uncertainty and multiple objectives with no loss of data.

Measures for Conducting Comparative Benefit: Risk Assessment

In this article, we review quantitative measures that have been proposed to conduct comparative benefit:risk assessment. Comparative benefit:risk assessment could be conducted at the societal level

A scale for assessing the severity of diseases and adverse drug reactions; Application to drug benefit and risk

TLDR
It is the opinion that this type of scale is preferable to nonnumerical descriptions of severity such as “mild” or “serious,” since, when combined with data on frequency of occurrence, a numerical scale permits a determination of expectation of both benefit and risk.