Comment on: “Can the EVIDEM Framework Tackle Issues Raised by Evaluating Treatments for Rare Diseases: Analysis of Issues and Policies, and Context-Specific Adaptation”

Multi-Criteria Decision Analysis (MCDA) Models in Health Technology Assessment of Orphan Drugs—a Systematic Literature Review. Next Steps in Methodology Development?

“…Recently, a number of authors have expressed critical views on the current MCDA frameworks and pointed out several flaws and drawbacks of the used design and scoring methodology, as well as proposing new models or alterations to correct these faults ( 8 , 59 , 60 ). These critiques and the fact that new MCDA models or improvements to existing ones are continuously being developed, shows that the MCDA approach in healthcare has not yet reached maturity or general consensus.…”

“…A study on the practical applicability of MCDA in Canada found that the quantification of evidence and interpretation of the aggregate MCDA score was “challenging” and that comparing/ranking interventions would require a better grasp of the underlying methodology ( 63 ). Gandjour ( 60 ) commented on the EVIDEM framework and expressed that the model in its current state is an “intermediate” solution and several improvements should be made, including an independent ethical justification and stronger theoretical foundation, especially for the use of individual preferences ( 60 ).…”

Can the Consideration of Societal Costs Change the Recommendation of Economic Evaluations in the Field of Rare Diseases? An Empirical Analysis

“…In this regard, it should be noted that, given the results usually achieved by the economic evaluations of health interventions in rare diseases, an appeal has been made to the use of other methods that complement or even substitute the previous ones, and that help either to expressly include elements that should be present in the decision making about financing and prices, or to introduce innovative reimbursement schemes to reconcile the patients' access to treatments with the requirements of good value for money 16,[61][62][63][64][65] and affordability. However, it should be noted that, first, the substitution of some of the proposed methods instead of the usual economic evaluations is not exempt from criticism either, 66,67 and second, that although theoretically there are convincing arguments to justify a special societal consideration for treatments used on rare diseases, there is no solid empirical evidence that "rarity" is a basic element that is valued more intensely by citizens than other factors that also affect this type of treatment, such as the severity of the disease, the potential gain in health, or the absence of alternative treatments. 20,[68][69][70][71][72] Given this framework of complexity and uncertainty, comparing the results achieved from the societal perspective with those from the perspective of the healthcare payer could be of potential utility in the decision-making process.…”