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Table 2 Mean scores, standard deviations and main comments from study participants for each criteria of the MCDA framework

From: Determining the value contribution of selexipag for the treatment of pulmonary arterial hypertension (PAH) in Spain using reflective multi-criteria decision analysis (MCDA)

MCDA framework criteria Mean score ± Standard Deviation Main comments from participants
Disease severity 4.5 ± 0.5 on a scale of 0 to 5 • “The impact of PAH on patient’s health (in the most severe cases) and quality of life and family environment (even in less advanced cases or responders to treatment) is severe, given the irreversible nature of the process, often known by patients / family, which leads to a pessimistic view about it.”
• “Interference, often serious, when performing normal daily activities”
Unmet needs 4.1 ± 0.8 on a scale of 0 to 5 • “More effective and selective drugs are needed that reduce mortality and disease progression in clinical trials and in real life.
Greater comfort than prostacyclines, so an oral treatment is welcome”
• “Currently available drugs that act on the prostacyclin route are uncomfortable, and they require time and training from patients and caregivers and interfere with the proceeding of a normal daily activity.”
Comparative efficacy/effectiveness 2.3 ± 1.9 on a scale of −5 to 5 • “Although there is no data available for all the variables in the studies conducted with iloprost that allow comparison with selexipag, it seems that better results are seen corresponding to “death by any event.” First Event. However, I believe that the impossibility of comparing more extensively with other alternatives limits the interpretation of results.”
• “The results presented favour the GRIPHON study of selexipag. Not only for the inclusion of a greater number of patients (up to five times more) but also for the design and choice of the primary endpoints. The designation of morbi-mortality parameters (progression, death, hospitalization, need for IV therapy, ...) make the trial more appropriate to the actual practice, with greater statistical power and conforms to the guidelines designated in the PAH symposium at Dana Point 2007.”
Comparative safety/tolerability 0.1 ± 2.1 on a scale of − 5 to 5 • “I think that the lower proportion of patients who died due to “any cause”, who had to interrupt the medication and who had potentially more severe undesirable effects (syncope) in the selexipag group, could be an element to be considered in the choice of selexipag with respect to iloprost.”
• “It seems that iloprost has fewer adverse effects than what is mentioned, although similar at a general level.”
Comparative patient-perceived health/patient reported outcomes (PRO) 2.4 ± 1.8 on a scale of − 5 to 5 • “Since there is no data regarding the groups treated with iloprost, even assuming that these are not studies that by their design allow direct comparison, it is not possible for me to clearly opt for any, except for the aspects related to the administration of the drugs (posology, manipulation of the inhalation device...), which evidently can suppose an interference in the quality of life of the patient derived from the greater complexity in the case of iloprost.”
• “The limitation imposed by the inhalation of iloprost up to 9 times a day is important, uncomfortable and makes the patient self-conscious”
• “An oral dosage twice a day clearly improves the quality of life and the autonomy of the patient.”
Type of preventive benefit 3.0 ± 1.1 on a scale of 0 to 5 • “The reduction of mortality due to any event, and the need for hospitalization, especially taking into account the ease of administration with respect to prostanoids, confers a high therapeutic value in my opinion.”
• “It does not prevent the disease, although, it stabilises and delays the appearance of new events of morbidity and mortality in relation to the disease.”
Type of therapeutic benefit 3.0 ± 0.8 on a scale of 0 to 5 • “The improvement in the time of progression of the disease and the increased convenience of administration are, in my opinion, favourable criteria to selexipag, especially in relation to prostanoids (aerosolized or parenteral), although the lack of effect on mortality continues to limit its therapeutic efficacy.”
• “It does not cure, it stabilizes and probably helps to chronify the disease.”
Comparative cost of intervention −1.6 ± 1.6 on a scale of − 5 to 5 • “The annual cost is substantially higher in the case of selexipag, which undoubtedly hinders its acceptance by the paying entities, although the convenience in its administration, with favorable repercussion on the quality of life of the patient and caregivers, as well as improvement in the evolutionary course of the disease (with reduced costs related to, for example, the need for hospitalization) should be arguments to be considered in the negotiation with the health authorities”
• “The economic cost of selexipag is 10% more than that of inhaled iloprost, which does not seem excessive, considering the ease of application.”
Comparative other medical costs 2.3 ± 2.0 on a scale of − 5 to 5 • “The main benefit of selexipag in terms of costs lies in its potential effect on the reduction of indirect costs, such as the need for hospitalizations, emergency visits, or other specific techniques or treatments. Although these costs are difficult to quantify for hospital pharmacies, they are one of the main problems in chronic diseases.”
• “The reason for the assigned score is purely estimative, since there are no cost-efficiency studies, neither with selexipag nor with any other therapeutic alternative.”
• “The economic impact of a drug is measured not only by its initial cost or investment, but by its ability to save in other aspects, so, a priori, selexipag seems to have a better impact on costs.”
Comparative other non-medical costs 2.1 ± 1.9 on a scale of − 5 to 5 • “The ease of administration / therapeutic compliance with selexipag could represent significant economic savings in the occupational and social sphere of patients.”
• “Difficult to estimate the impact on productivity, since the majority of patient candidates for selexipag or iloprost have a recognized legal incapacity status.”
Quality of evidence 4.0 ± 1.2; on a scale of 0 to 5 • “I believe that it strictly meets the quality criteria required for a clinical trial, especially given the difficulties of including patients as it is a low prevalence disease.”
• “I believe that the Griphon study is one of the best designed, with the largest number of patients included, with a broad spectrum of basic treatments (similar to the usual practice) and with one of the longest follow-up periods to date”
• “The scientific evidence provided by the Griphon study is now unquestionable, well above the existing data for many other medications and marks a new paradigm within clinical trials in pulmonary hypertension.”
Expert consensus/clinical practice guidelines (CPG) 3.4 ± 1.1, on a scale of 0 to 5 • “The current guideline recommendations place selexipag in both functional class II and III of the WHO, with a high level of evidence (IA). However, no distinction is made with respect to other oral drugs (endothelin antagonists or phosphodiesterase inhibitors).”
• “The real distinction is made with respect to analogous drugs (prostacyclines), with an introduction in earlier stages (functional class II), as well as a higher level of evidence.”
• “In the clinical guidelines of clinical practice, selexipag is recommended with the same degree of indication (degree I) as the rest of oral treatments with grade B evidence, similar to riociguat or macitentan.”