Multi-Criteria Decision Analysis to Value Orphan Medicines

Abstract

The purpose of this research is to identify the attributes to include in an orphan medicinal product (OMP) value framework, determine their relative importance via a multi-criteria decision analysis (MCDA) process, and test whether an MCDA approach can practically support decision making. MethodsThe project included literature searches on the natural history and burden of 40 rare diseases and how payers assess treatment value as well as three workshops. Workshops also were held: the first with GlaxoSmithKline managers working on OMPs, the second with EU clinical and health economics experts, and the third with representatives of rare diseases patient groups in the European Union. Participants refined the attributes, weighted them, scored two case study OMPs in terms of those attributes, and tested the sensitivity of the overall ratings to changes in weights and scores. ResultsEight non-monetary attributes were agreed: four concern the disease being treated and four the treatment itself. Workshop participants agreed consensus weights for the attributes. The patient group representatives and the clinical and health economics experts both attributed about half of the weight to attributes reflecting the disease being treated, and half to attributes of the treatment. Patient group representatives gave greater weight than the experts to patients' quality of daily life and less weight to clinical factors. The weighted attributes were readily applied by workshop participants to two example OMPs and yielded distinct ratings of their respective values. ConclusionsAn OMP value framework with agreed attributes and weights is a viable proposition using an MCDA approach, and could improve clarity and transparency in decision making about the value of OMPs.

Application of multicriteria decision analysis in health care: a systematic review and bibliometric analysis

“…The application of MCDA has been considered by several public and private health-care organizations and agencies including the US Agency for Healthcare Research and Quality’s (AHRQ), 31 the Canadian Agency for Drugs and Technologies in Health (CADTH), 32 the UK Department of Health, 33 the National Institute for Health and Care Excellence (NICE) in England and Wales, 34 the UK Office for Health Economics (OHE), 35 the German Institute for Quality and Efficiency in HealthCare (IQWiG), 36 the International Health Policy Programme (IHPP) and the Health Intervention and Technology Assessment Programme (HITAP) in Thailand. 37,38 These organizations/agencies have used and proposed MCDA as an approach to: incorporate stakeholder preferences in comparative effectiveness research (AHRQ), 31 assess new health technologies, 32 prioritize investment in public health interventions, 33 assess orphan drugs, 35 support benefit risk assessment and weigh the multiple endpoints considered in the assessment of quality and efficiency in health care, 36 develop universal coverage health benefit package.…”

“…37,38 These organizations/agencies have used and proposed MCDA as an approach to: incorporate stakeholder preferences in comparative effectiveness research (AHRQ), 31 assess new health technologies, 32 prioritize investment in public health interventions, 33 assess orphan drugs, 35 support benefit risk assessment and weigh the multiple endpoints considered in the assessment of quality and efficiency in health care, 36 develop universal coverage health benefit package. 37 …”

Orphan drugs policies: a suitable case for treatment

“…However, to the extent that orphan drugs are regarded as being 'special', it may not make sense to use cost-effectiveness alone as a basis for pricing. Other approaches such as multi-criteria decision-making (MCDA) have been proposed [17]. MCDA enables decision-makers to explicitly trade off various factors against each other, such as the seriousness of the condition and the lack of suitable treatment alternatives, alongside costeffectiveness.…”

Health technologies for rare diseases: does conventional HTA still apply?

“…In fact, multicriteria decision analysis provides a framework that is consistent with HTA, given that the definition of HTA points to multicriteria decision analysis by stating that a health technology needs to be assessed against multiple criteria in order to pronounce a judgment about the value of the health technology. Several authors have advocated the application of multicriteria decision analysis to health technologies for rare diseases [8,9]. For instance, Hughes-Wilson et al argued that standard HTA methods need to be tailored to consider the specificities of orphan drugs and that multicriteria decision analysis could serve as a tool to inform healthcare payer the coverage decisions of health technologies for rare diseases.…”

“…The Office of Health Economics used this approach to provide a value framework for health technologies for rare diseases [9]. Workshop participants identified eight nonmonetary criteria (four about the disease and four about the health technology) and carried out a weighted assessment of two health technologies for rare diseases against these criteria.…”