A multi-stakeholder multicriteria decision analysis for the reimbursement of orphan drugs (FinMHU-MCDA study)

Abstract

Background Patient access to orphan medicinal products (OMPs) is limited and varies between countries, reimbursement decisions on OMPs are complex, and there is a need for more transparent processes to know which criteria should be considered to inform these decisions. This study aimed to determine the most relevant criteria for the reimbursement of OMPs in Spain, from a multi-stakeholder perspective, and using multicriteria decision analysis (MCDA). Methods An MCDA was developed in 3 phases and included 28 stakeholders closely related to the field of rare diseases (6 physicians, 5 hospital pharmacists, 7 health economists, 4 patient representatives and 6 members from national and regional health authorities). Initially [phase A], a bibliographic review was conducted to identify the potential reimbursement criteria. Then, a reduced advisory board (8 members) proposed, selected, and defined the final list of criteria that could be relevant for reimbursement. A discrete choice experiment (DCE) [phase B] was developed to determine the relevance and relative importance weight of such criteria according to the stakeholders’ preferences by choosing between pairs of hypothetical financing scenarios. A multinomial logit model was fitted to analyze the DCE responses. Finally [phase C], the advisory board review the results using a deliberative process. Results Thirteen criteria were selected, related to 4 dimensions: patient population, disease, treatment, and economic evaluation. Nine criteria were deemed relevant for decision-making and associated with a higher relative importance: Health-related quality of life (HRQL) (23.53%), treatment efficacy (14.64%), availability of treatment alternatives (13.51%), disease severity (12.62%), avoided costs (11.21%), age of target population (7.75%), safety (seriousness of adverse events) (4.72%), quality of evidence (3.82%) and size of target population (3.12%). The remaining criteria had a < 3% relative importance: economic burden of disease (2.50%), cost of treatment (1.73%), cost-effectiveness (0.83%) and safety (frequency of adverse events) (0.03%). Conclusion The reimbursement of OMPs in Spain should be determined by its effect on patient’s HRQL, the extent of its therapeutic benefit from efficacy and the availability of other therapeutic options. Furthermore, the severity of the rare disease should also influence the decision along with the potential of the treatment to avoid associated costs.

Early Access to Medicines: Use of Multicriteria Decision Analysis (MCDA) as a Decision Tool in Catalonia (Spain)

“…MCDA has been used in a diverse range of health care decisions and levels [18,32]. At the macro level, it has been mostly used by HTA bodies to rank and set priorities among therapeutic alternatives and inform reimbursement decisions [20][21][22][23]25,33], and it has also has been proposed for the benefit-risk assessment to support marketing…”

“…MCDA has been used in a diverse range of health care decisions and levels [ 18 , 32 ]. At the macro level, it has been mostly used by HTA bodies to rank and set priorities among therapeutic alternatives and inform reimbursement decisions [ 20 , 21 , 22 , 23 , 25 , 33 ], and it has also has been proposed for the benefit–risk assessment to support marketing authorisation [ 34 ]. At lower decision levels, MCDA has been used to inform hospital decision making to incorporate medicines into the hospital formulary [ 24 ] and also to support shared decision making involving the patient’s voice [ 35 ].…”

“…Multicriteria decision analysis (MCDA) enables a holistic assessment by a systematic, transparent and explicit consideration of multiple aspects beyond the traditional criteria used by HTA [ 18 , 19 , 20 ]. This methodology has been used to support different types of decisions and at different levels and contexts: to set priorities by HTA among alternatives at the national level or by PTC in the hospital setting, to support decision making about pricing and reimbursement or to assist clinical decisions in specific areas such as oncology or orphan drugs [ 21 , 22 , 23 , 24 , 25 , 26 , 27 ]. The Evidence and Value: impact on decision making (EVIDEM) is a reflective MCDA approach that provides an adaptable framework that has been used in a variety of healthcare settings to support the deliberative process.…”

A Cross-Sectional Study of Price and Affordability of Drugs for Rare Diseases in Shandong Province, China

“…Previous studies have shown that using health technology assessment (HTA) approaches typically results in rare disease drugs not being cost-effective [ 38 , 39 , 40 ]. The multi-criteria decision analysis (MCDA) framework as a tool for assessing the value of rare disease drugs has improved the technical and ethical quality of decisions regarding prioritization, coverage, and reimbursement of rare diseases [ 41 , 42 , 43 ].…”

ODs with a positive TPR conclusion, not subject to a conditional approval, and approved without requiring a PASS would be more likely to be reimbursed in Spain

“…Despite their low prevalence, they are life-threatening or chronically debilitating conditions with a high burden and very often limited level of awareness [ 1 , 4 ]. RDs have a high impact on patients, their families, healthcare systems and even society in general, and are characterized by pain, disability, significant organ damage, and high mortality rates [ 5 ]. Although their prevalence is low, RDs are numerous and heterogeneous [ 6 ].…”