Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review

Abdallah, Khadidja; Huys, Isabelle; Claes, Kathleen; Simoens, Steven

doi:10.3389/fphar.2021.630949

Cited by 13 publications

(12 citation statements)

References 113 publications

(76 reference statements)

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Model validation and sensitivity analysis should be carried out to ensure the robustness of the BIA research. A sensitivity analysis is essential to investigate the influence of assumptions on structural aspects or variable inputs of the BIAs (37). Moreover, sensitivity analysis allows a more comprehensive prediction of budget impact.…”

Section: Discussionmentioning

confidence: 99%

Budget Impact Analysis of Diabetes Drugs: A Systematic Literature Review

Luo

Ruan

Yao

et al. 2021

Front. Public Health

View full text Add to dashboard Cite

Background: Budget impact analysis (BIA) is an economic assessment that estimates the financial consequences of adopting a new intervention. BIA is used to make informed reimbursement decisions, as a supplement to cost-effectiveness analyses (CEAs).Objectives: We systematically reviewed BIA studies associated with anti-diabetic drugs and assessed the extent to which international BIA guidelines were followed in these studies.Methods: We conducted a literature search on PubMed, Web of Science, Econlit, Medline, China National Knowledge Infrastructure (CNKI), Wanfang Data knowledge Service platform from database inception to June 30, 2021. ISPOR good practice guidelines were used as a methodological standard for assessing BIAs. We extracted and compared the study characteristics outlined by the ISPOR BIA Task Force to evaluate the guideline compliance of the included BIA.Results: A total of eighteen studies on the BIA for anti-diabetic drugs were identified. More than half studies were from developed countries. Seventeen studies were based on model and one study was based on real-world data. Overall, analysis considered a payer perspective, reported potential budget impacts over 1–5 years. Assumptions were mainly made about target population size, market share uptake of new interventions, and scope of cost. The data used for analysis varied among studies and was rarely justified. Model validation and sensitivity analysis were lacking in the current BIA studies. Rebate analysis was conducted in a few studies to explore the price discount that was required for new interventions to demonstrate cost equivalence to comparators.Conclusion: Existing studies evaluating budget impact for anti-diabetic drugs vary greatly in methodology, some of which showed low compliance to good practice guidelines. In order for the BIA to be useful for assisting in health plan decision-making, it is important for future studies to optimize compliance to national or ISPOR good practice guidelines on BIA. Model validation and sensitivity analysis should also be improved in future BIA studies. Continued improvement of BIA using real-world data is necessary to ensure high-quality analyses and to provide reliable results.

show abstract

Section: Discussionmentioning

confidence: 99%

Budget Impact Analysis of Diabetes Drugs: A Systematic Literature Review

Luo

Ruan

Yao

et al. 2021

Front. Public Health

View full text Add to dashboard Cite

show abstract

“…Lack of transparency due to confidentiality also prevents insight into the actual budget impact. Assuring the methodological quality of future BIA could allow a more in-depth analysis and better informing of decision-makers on affordability ( Abdallah et al, 2021 ).…”

Section: Discussionmentioning

confidence: 99%

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

et al. 2021

Self Cite

View full text Add to dashboard Cite

Objectives: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators, Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor), have substantially improved patients’ lives yet significantly burden healthcare budgets. This analysis aims to compare pricing and reimbursement of aforementioned cystic fibrosis medicines, across European countries.Methods: Clinical trial registries, national databases, health technology assessment reports and grey literature of Austria, Belgium, Denmark, France, Germany, Ireland, Poland, Spain, Sweden, Switzerland, Netherlands, the United Kingdom were consulted. Publicly available prices, reimbursement statuses, economic evaluations, budget impact analyses and managed entry agreements of CFTR modulators were examined. Results: In Belgium, lowest list prices were observed for Kalydeco® (ivacaftor) and Symkevi® (tezacaftor/ivacaftor) at €417 per defined daily dose (DDD) and €372 per average daily dose (ADD), respectively. Whereas, Switzerland had the lowest price for Orkambi® (lumacaftor/ivacaftor) listed at €309 per DDD. Spain had the highest prices for Kalydeco® (ivacaftor) and Symkevi® (tezacaftor/ivacaftor) at €850 per DDD and €761 per ADD, whereas Orkambi® (lumacaftor/ivacaftor) was most expensive in Poland at €983 per DDD. However, list prices were subject to confidential discounts and likely varied from actual costs. In all countries, these treatments were deemed not to be cost-effective. The annual budget impact of the CFTR modulators varied between countries and depended on factors such as local product prices, size of target population, scope of costs and discounting. However, all modulators were fully reimbursed in ten of the evaluated countries except for Sweden and Poland that, respectively, granted reimbursement to one and none of the therapies. Managed entry agreements were confidential but commonly adopted to address financial uncertainties.Conclusion: Discrepancies concerning prices, reimbursement and access were detected for Kalydeco® (ivacaftor), Orkambi® (lumacaftor/ivacaftor) and Symkevi® (tezacaftor/ivacaftor) across European countries.

show abstract

“…These rare disease registries can also be an instrumental source of data on cost and patient numbers in the context of budget impact analyses of orphan medicines. In Belgium (as in other countries), budget impact analyses of orphan medicines tend to be of low quality (Abdallah et al, 2021). More attention needs to be paid to conducting and reporting budget impact analyses for (orphan) medicines that adhere to good practice guidelines, such as those set up by the International Society for Pharmacoeconomics and Outcomes Research (Abdallah et al, 2021).…”

Section: Sustaining Innovation and Access To Orphan Medicinesmentioning

confidence: 99%

How to balance valuable innovation with affordable access to medicines in Belgium?

et al. 2022

Self Cite

View full text Add to dashboard Cite

Background: Countries are struggling to provide affordable access to medicines while supporting the market entry of innovative, expensive products. This Perspective aims to discuss challenges and avenues for balancing health care system objectives of access, affordability and innovation related to medicines in Belgium (and in other countries).Methods: This Perspective focuses on the R&D, regulatory approval and market access phases, with particular attention to oncology medicines, precision medicines, orphan medicines, advanced therapies, repurposed medicines, generics and biosimilars. The authors conducted a narrative review of the peer-reviewed literature, of the grey literature (such as policy documents and reports of consultancy agencies), and of their own research.Results: Health care stakeholders need to consider various initiatives for balancing innovation with access to medicines, which relate to clinical and non-clinical outcomes (e.g. supporting the conduct of pragmatic clinical trials, treatment optimisation and patient preference studies, optimising the use of real-world evidence in market access decision making), value assessment (e.g. increasing the transparency of the reimbursement system and criteria, tailoring the design of managed entry agreements to specific types of uncertainty), affordability (e.g. harnessing the role of generics and biosimilars in encouraging price competition, maximising opportunities for personalising and repurposing medicines) and access mechanisms (e.g. promoting collaboration and early dialogue between stakeholders including patients). Conclusion:Although there is no silver bullet that can balance valuable innovation with affordable access to medicines, (Belgian) policy and decision makers should continue to explore initiatives that exploit the potential of both the on-patent and off-patent pharmaceutical markets.

show abstract

Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review

Cited by 13 publications

References 113 publications

Budget Impact Analysis of Diabetes Drugs: A Systematic Literature Review

Budget Impact Analysis of Diabetes Drugs: A Systematic Literature Review

A Comparative Analysis of Pricing and Reimbursement of Cystic Fibrosis Transmembrane Conductance Regulator Modulators in Europe

How to balance valuable innovation with affordable access to medicines in Belgium?

Contact Info

Product

Resources

About