Limitations and Challenges of Conducting Budget Impact Analyses in Rare Diseases: A Case Study of Alpha-1 Antitrypsin Deficiency

Rueda, Juan David; Sieluk, Jan; Sandhaus, Robert A.; Mullins, C. Daniel

doi:10.1016/j.vhri.2020.04.003

Cited by 5 publications

(4 citation statements)

References 29 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Even the costs' structure should be examined in a wider way, assessing not only the cost of the drugs per se, but the costs of the integrated care plans and, at the same time, the avoided costs, health and social ones, direct and indirect ones, of a failed integration of the patient. 25 This work is a sectorial cost-analysis and does not consider clinical and quality of life outcomes. For its characteristics has the limit of not being able to show the advantages of therapeutical options but only to verify the financial burden of existing treatments, and need to be developed and deepened with future research.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

Manea¹,

Pozza²,

Minichiello³

et al. 2023

Health Serv Manage Res

View full text Add to dashboard Cite

Background: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). Objectives: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. Methods: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs’ list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. Results: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million (“blockbuster drugs”). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. Conclusions: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing “blockbuster drugs”. Some therapeutical areas seem to be lacking in the drug research field.

show abstract

Section: Discussionmentioning

confidence: 99%

“…Even the costs’ structure should be examined in a wider way, assessing not only the cost of the drugs per se, but the costs of the integrated care plans and, at the same time, the avoided costs, health and social ones, direct and indirect ones, of a failed integration of the patient. 25…”

Section: Discussionmentioning

confidence: 99%

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

Manea¹,

Pozza²,

Minichiello³

et al. 2023

Health Serv Manage Res

View full text Add to dashboard Cite

show abstract

“…IV-AAT therapy carries significant cost implications to patients themselves (if the treatment is self-funded), the insurer or the health system that covers the cost of the treatment. Multiple studies have assessed the financial implications of augmentation therapy [52][53][54][55][56][57] as well as the cost of comorbidities associated with AATD [58]. IV-AAT costs equate to approximately USD 80 000 per patient annually [52].…”

Section: Implications Of Adherence To the 11 µM Threshold As A Determ...mentioning

confidence: 99%

“…Furthermore, in real-world practice, clinicians understandably feel responsible for instituting an appropriate management plan for individuals presenting with abnormal AAT genotypes. To this end, a 2020 study examining claims databases of privately insured and Medicare Advantage enrolees found that implementation of an AATD disease management programme resulted in a reduction in cost in the management of patients with AATD over a 5-year period, attributable to a reduction in the number of exacerbations, emergency-room and specialist visits [56]. While the study did not compare AATD by genotypes specifically, it would stand that individuals with moderate genotypes may in the first instance be better served by the implementation of a disease management programme rather than resorting to IV-AAT.…”

Section: Implications Of Adherence To the 11 µM Threshold As A Determ...mentioning

confidence: 99%

Alpha-1 antitrypsin deficiency: clarifying the role of the putative protective threshold

et al. 2021

View full text Add to dashboard Cite

AATD is the only readily identifiable monogenic cause of COPD. To date the only condition-specific treatment for AATD-associated COPD is weekly administration of intravenous purified pooled human AAT (IV-AAT). Uncertainties regarding which AATD genotypes should benefit from IV-AAT persist. IV-AAT is costly and involves weekly administration of a plasma product. Much of the risk stratification has been centred around the long-accepted hypothesis of a “putative protective threshold” of 11 µM (0.57 g·L−1) in serum. This hypothesis has become central to the paradigm of AATD care, though its derivation and accuracy for defining risk of disease remain unclear.We review the literature and examine the association between the 11 µM threshold and clinical outcomes to provide context and insight into the issues surrounding this topic.We found no data which demonstrates an increased risk of COPD dependent on the 11 µM threshold. Moreover, an abundance of recent clinical data examining this threshold refutes the hypothesis. Conversely, the use of 11 µM as a treatment target in appropriate ZZ individuals is supported by clinical evidence, although more refined dosing regimens are being explored.Continued use of the 11 µM threshold as a determinant of clinical risk is questionable, perpetuates inappropriate AAT-augmentation practices, may drive increased healthcare expenditure and should not be used as an indicator for commencing treatment.Genotype represents a more proven indicator of risk, with ZZ and rare ZZ-equivalent genotypes independently associated with COPD. New and better risk assessment models are needed to provide individuals diagnosed with AATD with reliable risk estimation and optimised treatment goals.

show abstract

Budget impact analyses for treatment of heart failure. A systematic review

Teimourizad,

Jafari,

Esmaeilzadeh

2024

Heart Fail Rev

View full text Add to dashboard Cite

Limitations and Challenges of Conducting Budget Impact Analyses in Rare Diseases: A Case Study of Alpha-1 Antitrypsin Deficiency

Cited by 5 publications

References 29 publications

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

Alpha-1 antitrypsin deficiency: clarifying the role of the putative protective threshold

Budget impact analyses for treatment of heart failure. A systematic review

Contact Info

Product

Resources

About