Mcda Approach To Ranking Rare Diseases In Russia: Preliminary Results

Abstract

A539of 16 criteria. Methods: 85 experts were interviewed to estimate the importance of each criterion in the decision-making on financing MT for rare diseases. We used 10-point scale, where 10 points mean major importance to the priority indicator, and 1point means minor importance. Mean estimates were calculated using descriptive statistics, then means were normalized. Results: Respondents were 41 years on average (ranging from 23 to 64 years), and included 20 public servants, 16 health administrators, 32 practitioners, and 14 researchers. 44 respondents had a scientific degree. The most important criteria were characteristics of treatment -"Effect of treatment on quality of life" and "Effect of treatment on life expectancy" with 1 points each. The least important criteria were both characteristics of the disease -"Cognitive disorders as manifestations of the disease and "Additional burden on the daily lives of care-givers" with 0.28 and 0.1 respectively. ConClusions: Characteristics of treatment turned out to be more important for respondents than characteristics of disease, therefore characteristics of treatment should be given consideration when evaluating rare diseases to determine priority financing.

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

“…Sixteen publications focused mostly on defining the most appropriate model criteria [Wagner et al ( 25 ) and Wagner et al ( 43 )], Hughes-Wilson et al ( 33 ), Kolasa et al ( 34 ), Iskrov et al ( 35 ), Trip et al ( 36 ), Sussex et al ( 37 ), Schey et al ( 42 ), Fedyaeva et al ( 38 ), Paulden et al ( 8 ), Palaska and Hutchings ( 31 ), and Piniazkho et al ( 40 )] but in 5 it did not lead to the creation of a defined MCDA model [Schlander et al ( 52 ), Zhang et al ( 53 ), Nemeth and Piniazhko ( 54 ), Korchagina et al ( 55 ), and Hutching et al ( 56 )].…”

A Review of Rare Disease Policies and Orphan Drug Reimbursement Systems in 12 Eurasian Countries

“…Researchers in the rare disease area are also looking into the use of MCDA, which has resulted in a list of scientific publications and MCDA model designs, but full consensus on MCDA is still lacking and further research is needed to support implementation in (rare disease) HTA (94,(101)(102)(103)(104)(105)(106)(107)(108)(109)(110)(111)(112)(113)(114)(115)(116).…”

Healthcare decision‐making applications using multicriteria decision analysis: A scoping review

“…Assessing the value of treatment options was the objective of seven of them. The other papers used the MCDA process to assess criteria in setting priorities (Fedyaeva, Omelyanovsky, Rebrova, Khan, & Petrovskaya, ), assessing quality care (Dionne, Mitton, Macdonald, Miller, & Brennan, ), assessing drug harms (Nutt et al, ), evaluating regulatory decision‐making (Maro et al, ), improving outpatient services (Kuo et al, ), selecting waste water technologies (Lu et al, ), supporting HTA decision‐making processes (Kwon, Park, Byun, & Lee, ; Wilson, Sussex, Macleod, & Fordham, ), assessing a suitable model (Vasconcelos, Almeida, & Nobre, ), and evaluating the performance of medical devices (Lee et al, ).…”

“…When workshops (a primary source of information) were the only source of information with which to build the model ( n = 6), the number of criteria was relatively small (from four to nine) because the process was iterative, hence, time consuming. Researchers also chose to review the literature first (Nutt et al, ; Fedyaeva et al, ; Mussen, Salek, & Walker, ; Kaslow et al, ) and then organized workshops to complete the list of criteria. When both primary and secondary sources of information were chosen, then the models counted between 15 and 20 criteria.…”