Using multicriteria decision analysis during drug development to predict reimbursement decisions

Abstract

BackgroundPharmaceutical companies design clinical development programs to generate the data that they believe will support reimbursement for the experimental compound.ObjectiveThe objective of the study was to present a process for using multicriteria decision analysis (MCDA) by a pharmaceutical company to estimate the probability of a positive recommendation for reimbursement for a new drug given drug and environmental attributes.MethodsThe MCDA process included 1) selection of decisions makers who were representative of those making reimbursement decisions in a specific country; 2) two pre-workshop questionnaires to identify the most important attributes and their relative importance for a positive recommendation for a new drug; 3) a 1-day workshop during which participants undertook three tasks: i) they agreed on a final list of decision attributes and their importance weights, ii) they developed level descriptions for these attributes and mapped each attribute level to a value function, and iii) they developed profiles for hypothetical products ‘just likely to be reimbursed’; and 4) use of the data from the workshop to develop a prediction algorithm based on a logistic regression analysis. The MCDA process is illustrated using case studies for three countries, the United Kingdom, Germany, and Spain. The extent to which the prediction algorithms for each country captured the decision processes for the workshop participants in our case studies was tested using a post-meeting questionnaire that asked the participants to make recommendations for a set of hypothetical products.ResultsThe data collected in the case study workshops resulted in a prediction algorithm: 1) for the United Kingdom, the probability of a positive recommendation for different ranges of cost-effectiveness ratios; 2) for Spain, the probability of a positive recommendation at the national and regional levels; and 3) for Germany, the probability of a determination of clinical benefit. The results from the post-meeting questionnaire revealed a high predictive value for the algorithm developed using MCDA.ConclusionsPrediction algorithms developed using MCDA could be used by pharmaceutical companies when designing their clinical development programs to estimate the likelihood of a favourable reimbursement recommendation for different product profiles and for different positions in the treatment pathway.

A Multiple Stakeholder Multicriteria Decision Analysis in Diabetic Macular Edema Management: The MULTIDEX-EMD Study

“…Other applications of MCDA include the development of an assessment framework for rare diseases at a regional level in Catalonia [ 22 ] and the development of a decision-support tool for drug evaluation in a pharmacy and therapeutics committee setting at the hospital level [ 20 ]. MCDA was also used to establish an algorithm for predicting price and reimbursement decisions at national and regional levels [ 27 ].…”

“…In our country, Spain, only a few MCDAs have been published in the field of healthcare [20][21][22][23][24][25][26][27][28], although the number of healthcare-related publications that incorporate MCDA is increasing in Spain and globally.…”

“…Some studies have used the EVIDEM methodology to develop the analysis [20,[22][23][24][25]28]. In those studies, the methods used to prioritize the criteria were fundamentally compositional, such as direct classification or the analytic hierarchical process [22][23][24][25][26][27][28]. Either a single-stakeholder perspective was used, such as that of patients [20,21,26] or evaluation agencies [22,27], or a multidisciplinary perspective was employed, which included patients, physicians, and other stakeholders [23][24][25]28].…”

International Experience in Therapeutic Value and Value-Based Pricing: A Rapid Review of the Literature

“…The VBP policy in the United Kingdom was officially described in 2009 in the Pharmaceutical Price Regulation Scheme (PPRS) program update. 21,22 During this process, the Department of Health and the Association of the British Pharmaceutical Industry (ABPI) helped to establish 2 measures that better reflected the Table 2. Description of the entities involved in the process of drugs pricing, negotiation, and reimbursement in the 6 reference countries.…”

Stakeholder opinions on value in healthcare

“…The literature does not provide a consistent and universal definition of value in healthcare 13. Published definitions range from those valuing outcomes, including health, in monetary terms14 15 to those taking into account healthcare system policy objectives, which represent societal rather than individual preferences for states of health,16 using constructed measures of health outcomes such as the quality-adjusted life-year (QALY) 17. The value of healthcare products under the latter approach may include broader aspects such as the importance placed on a health state by patients or society, the social and psychological aspects of living with an illness or using a technology, and ethical implications 18.…”