MCDA in Rare Diseases: A Tool for Accurate and Fair Treatment Evaluation
MCDA in the field of rare diseases: a tool for accurate and fair evaluation of therapy
On October 22, the Orphan Consortium of the CIS, together with the National Scientific Center for Healthcare Development named after Salidat Kairbekova (Kazakhstan) held a webinar on the application of multicriteria decision analysis (MCDA) in health technology assessment.
The event was held as part of the working group of the Consortium on Drug Supply and Health Technology Assessment.
International Experience
The speakers noted that countries use two main approaches to assessing orphan drugs:
This approach carries the risk of ineffective spending. To reduce this cost, additional assessment tools, real-world clinical data collection, and managed access agreements are needed.
Russian Experience
Vlada Konstantinovna Fedyaeva (NAEREZ) presented the MCDA model for orphan drugs developed in 2016.
More than 200 experts participated in the study. Weighting coefficients were determined for key criteria:
Although the model was not implemented in practice, it allowed specialists to master the MCDA methodology and laid the foundation for future adaptations of the tool in Russia.
Kazakhstan's Experience
Adlet Berikbolovich Tabarov, Deputy Chairman of the Board of the National Scientific Center for Healthcare Development, said that MCDA is already being used in Kazakhstan, both in hospital technology assessments and in developing a list of high-tech medical care. He also presented draft amendments to the Order of the Ministry of Health of the Republic of Kazakhstan "Rules for Conducting HTA and Their Application," which provide for the assessment of all drugs for inclusion in the reimbursement system using MCDA. The tool includes the following criteria:
The assessment results are advisory in nature, helping expert commissions formulate more transparent and balanced decisions, including for Orphan drugs.
The participants agreed to continue studying international experience with MCDA and explore the possibility of creating a unified tool for prioritizing rare diseases for CIS countries. This approach could improve the validity of decisions in drug supply.
A recording of the webinar can be viewed on the website.
On October 22, the Orphan Consortium of the CIS, together with the National Scientific Center for Healthcare Development named after Salidat Kairbekova (Kazakhstan) held a webinar on the application of multicriteria decision analysis (MCDA) in health technology assessment.
The event was held as part of the working group of the Consortium on Drug Supply and Health Technology Assessment.
International Experience
The speakers noted that countries use two main approaches to assessing orphan drugs:
- creating special procedures for rare diseases;
- including orphan drugs in the general HTA system, with more lenient evidence requirements and a higher willingness to pay threshold.
This approach carries the risk of ineffective spending. To reduce this cost, additional assessment tools, real-world clinical data collection, and managed access agreements are needed.
Russian Experience
Vlada Konstantinovna Fedyaeva (NAEREZ) presented the MCDA model for orphan drugs developed in 2016.
More than 200 experts participated in the study. Weighting coefficients were determined for key criteria:
- existence of effective treatment options,
- life expectancy without treatment,
- age of disease onset,
- burden on caregivers,
- impact of therapy on life expectancy and quality of life,
- frequency of severe and other adverse events.
Although the model was not implemented in practice, it allowed specialists to master the MCDA methodology and laid the foundation for future adaptations of the tool in Russia.
Kazakhstan's Experience
Adlet Berikbolovich Tabarov, Deputy Chairman of the Board of the National Scientific Center for Healthcare Development, said that MCDA is already being used in Kazakhstan, both in hospital technology assessments and in developing a list of high-tech medical care. He also presented draft amendments to the Order of the Ministry of Health of the Republic of Kazakhstan "Rules for Conducting HTA and Their Application," which provide for the assessment of all drugs for inclusion in the reimbursement system using MCDA. The tool includes the following criteria:
- novelty and innovation,
- clinical efficacy and safety,
- economic effectiveness and budget impact,
- social significance, ethical aspects, demand, and return on investment.
The assessment results are advisory in nature, helping expert commissions formulate more transparent and balanced decisions, including for Orphan drugs.
The participants agreed to continue studying international experience with MCDA and explore the possibility of creating a unified tool for prioritizing rare diseases for CIS countries. This approach could improve the validity of decisions in drug supply.
A recording of the webinar can be viewed on the website.
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