The influence of the inclusion of social cost in the review of the drug classics on the economic evaluation results of rare diseases

Author: Lucia

Rare diseases (RDs), also known as "orphan diseases", refer to those diseases with extremely low incidence or prevalence

Rare diseases are mostly chronic, serious, and usually have degenerative and fatal characteristics

Rare diseases are generally complicated in diagnosis, lack of effective treatment, and need to cooperate with multidisciplinary follow-up, comprehensive management, and family support

On December 5, 2020, researchers from the University of Castilla-La Mancha in Spain wrote an article on Value in Health, which focused on the use of the whole society and the health system to diagnose rare diseases, drug treatments and other medical treatments.

For the selection of drugs for the treatment of rare diseases, the article uses the list of orphan drugs listed on the European Medicines Agency (EMA) website in May 2018, and selects 104 active substances that EMA believes are authorized orphan drugs

The author uses the orphan drug list, as well as "costs", "cost analysis", "cost-effectiveness", "cost-utility", "cost-benefit", "economic evaluation", "economic analysis", "QALY", "quality -Adjusted life years" and other economic evaluation keywords, a systematic review of the literature included in the Medline and Tufts University Cost Effectiveness Research Registry from the beginning of 2000 to November 2018, and a total of 2306 unique items were retrieved Record

Source: Value in Health

In this study, direct medical costs include all medical-related costs; social costs mainly involve the cost of informal care (provided by non-professional nursing staff), as well as the labor time of patients and their families due to illness, disability or death.

Analyzing the related economic evaluation literature of rare diseases from the perspective of society as a whole, it was found that among 14 included literatures, 12 considered productivity loss, and most of them used the human capital method for calculation

The included 14 documents contain 26 sets of economic evaluation programs.

Source: Value in Health

In most of the studies carried out from the perspective of the whole society, no significant changes have been found in the economic evaluation results due to the high cost of treatment or the low quality-adjusted life obtained

At the end of the article, it is pointed out that there is a considerable degree of uncertainty in the pricing and reimbursement decisions of rare disease treatments, and it faces many challenges such as high prices and small sample sizes included in trials and studies

In addition, when carrying out relevant economic evaluations of rare diseases, it is necessary to conduct multi-angle research from the whole society and the health system to increase the number of publications of this type of literature

Reference materials:

[1]Can the consideration of societal costs change the recommendation of economic evaluations in the field of rare diseases? An empirical analysis (Source: Value in Health)