Budget impact analysis and payment approaches for orphan drugs: A multidisciplinary perspective
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| Titel: | Budget impact analysis and payment approaches for orphan drugs: A multidisciplinary perspective |
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| Autoren: | Abdallah, Khadidja |
| Weitere Verfasser: | Simoens, Steven, Claes, Kathleen, Huys, Isabelle |
| Verlagsinformationen: | 2023. |
| Publikationsjahr: | 2023 |
| Beschreibung: | The EU Orphan Regulation has led to a surge in available orphan drugs, as pharmaceutical companies prioritize expanding their portfolios with specialized treatments. However, the process of estimating affordability and regulating financial resource allocation through BIA has been reported to be suboptimal, often lacking applicability on intricacies of orphan drugs. Additionally, governments have expressed concerns about the sustainability of financing orphan drugs and are actively seeking the most appropriate payment approaches to ensure sustainable healthcare spending. Therefore, the primary aim of this PhD project was to offer evidence-based, practical, and scientifically-driven methodological guidance to relevant stakeholders on optimizing approaches for the reimbursement and payment of orphan drugs. Part I of this project addresses BIA of orphan drugs. Through a systematic review, Chapter 1 evaluates the methodological quality of BIAs for orphan drugs and provided guidelines for improvement. It was observed that recommendations on payer's perspective, periodic reporting over a short-term horizon and omitting discounting, were commonly adhered to. In contrast, standards for parameters on population dynamics, integration of costs other than drug acquisition were often overlooked. We concluded that current BIAs for orphan drugs were simplistic and inconsistent with internationally published guidelines. Chapter 2 employs a mixed-methods approach centered around SCD. Chapter 2.1. presents a hypothetical BIA for adoption of crizanlizumab, voxelotor, and CTX001 by the Belgian healthcare payer. We estimated a five-year cumulative budget impact of €39,924,682, with 87% of the total attributed to drug acquisition costs. CTX001 accounted for the largest share (€35,063,194), while crizanlizumab (€2,261,927) and voxelotor (€2,599,561) constituted comparatively smaller portions. Our findings suggest that Belgian policymakers may consider exploring innovative payment models, such as outcome-based or risk-sharing agreements, to ensure sustainable coverage for these treatments. In Chapter 2.2. French-Belgian and British stakeholder groups assessed the value of well-established hydroxyurea and HSCT compared to newer treatments crizanlizumab, voxelotor and CTX001 using MCDA. Hydroxyurea received the highest overall value ranking, while CTX001 received the lowest rating from both groups. Voxelotor, crizanlizumab, and HSCT had variable ranking in both groups. Although the current treatment was most valued, our study also emphasized the need for improved therapies for patients with SCD and underscored the importance of engaging diverse stakeholders in decision-making. Part II focuses on various payment approaches for orphan drugs. In Chapter 3 we evaluated implications of adopting private insurance or an insulated fund for financing orphan drugs and early access though compassionate use or off-label use in Belgium by consulting a diverse group of experts. Our findings suggest that rather than establishing alternative systems, it may be more effective to re-evaluate and refine existing financing models by addressing current shortcomings pertaining data collection, transparency, collaboration, administration and legal responsibilities. In Chapter 4 we conducted a comparative analysis of facial prices, coverage decisions, economic evaluations, BIAs and MEAs of Kalydeco®, Orkambi® and Symkevi® across twelve European countries. Belgium and Switzerland exhibited the lowest list prices, while Spain and Poland had the highest. Across all countries, the treatments were generally deemed not to be cost-effective. The annual budget impact of CFTR modulators varied, with most countries considering it to be high. Nevertheless, ten of the evaluated countries fully reimbursed all modulators through MEAs, which were implemented to address financial and clinical uncertainties. However, we captured discrepancies concerning pricing, reimbursement and timely access to CFTR modulators. In Chapter 5 we introduced a communication roadmap for the disinvestment of orphan drugs under conditional reimbursement, drawing on input from Belgian stakeholders. We emphasized the importance of considerate communication towards patients and the public and underscored confidentiality, transparency, collaboration, and cohesiveness as vital for effective communication of disinvestment decisions. Our roadmap could assist policymakers in effectively communicating disinvestment decisions, ultimately benefiting society as a whole. Finally, we formulated six key recommendations for policy- and decision-makers: 1. Improve data collection at national and EU level 2. Shift from reference pricing to collective price negotiations 3. Holistically define 'Unmet Medical Need' to ensure accessibility to orphan drugs for those underserved 4. Provide a BIA template and validate it to enhance accurate forecasting of affordability 5. Educate stakeholders, including clinical and patient experts, to assess orphan drugs comprehensively 6. Dare to disinvest high-cost and ineffective orphan drugs after conditional reimbursement |
| Publikationsart: | Doctoral thesis |
| Sprache: | English |
| Zugangs-URL: | https://lirias.kuleuven.be/handle/20.500.12942/732839 |
| Dokumentencode: | edsair.od......1131..fea1e60a4b051ddce11c8bfcec2c602c |
| Datenbank: | OpenAIRE |
Nájsť tento článok vo Web of Science | Abstract: | The EU Orphan Regulation has led to a surge in available orphan drugs, as pharmaceutical companies prioritize expanding their portfolios with specialized treatments. However, the process of estimating affordability and regulating financial resource allocation through BIA has been reported to be suboptimal, often lacking applicability on intricacies of orphan drugs. Additionally, governments have expressed concerns about the sustainability of financing orphan drugs and are actively seeking the most appropriate payment approaches to ensure sustainable healthcare spending. Therefore, the primary aim of this PhD project was to offer evidence-based, practical, and scientifically-driven methodological guidance to relevant stakeholders on optimizing approaches for the reimbursement and payment of orphan drugs. Part I of this project addresses BIA of orphan drugs. Through a systematic review, Chapter 1 evaluates the methodological quality of BIAs for orphan drugs and provided guidelines for improvement. It was observed that recommendations on payer's perspective, periodic reporting over a short-term horizon and omitting discounting, were commonly adhered to. In contrast, standards for parameters on population dynamics, integration of costs other than drug acquisition were often overlooked. We concluded that current BIAs for orphan drugs were simplistic and inconsistent with internationally published guidelines. Chapter 2 employs a mixed-methods approach centered around SCD. Chapter 2.1. presents a hypothetical BIA for adoption of crizanlizumab, voxelotor, and CTX001 by the Belgian healthcare payer. We estimated a five-year cumulative budget impact of €39,924,682, with 87% of the total attributed to drug acquisition costs. CTX001 accounted for the largest share (€35,063,194), while crizanlizumab (€2,261,927) and voxelotor (€2,599,561) constituted comparatively smaller portions. Our findings suggest that Belgian policymakers may consider exploring innovative payment models, such as outcome-based or risk-sharing agreements, to ensure sustainable coverage for these treatments. In Chapter 2.2. French-Belgian and British stakeholder groups assessed the value of well-established hydroxyurea and HSCT compared to newer treatments crizanlizumab, voxelotor and CTX001 using MCDA. Hydroxyurea received the highest overall value ranking, while CTX001 received the lowest rating from both groups. Voxelotor, crizanlizumab, and HSCT had variable ranking in both groups. Although the current treatment was most valued, our study also emphasized the need for improved therapies for patients with SCD and underscored the importance of engaging diverse stakeholders in decision-making. Part II focuses on various payment approaches for orphan drugs. In Chapter 3 we evaluated implications of adopting private insurance or an insulated fund for financing orphan drugs and early access though compassionate use or off-label use in Belgium by consulting a diverse group of experts. Our findings suggest that rather than establishing alternative systems, it may be more effective to re-evaluate and refine existing financing models by addressing current shortcomings pertaining data collection, transparency, collaboration, administration and legal responsibilities. In Chapter 4 we conducted a comparative analysis of facial prices, coverage decisions, economic evaluations, BIAs and MEAs of Kalydeco®, Orkambi® and Symkevi® across twelve European countries. Belgium and Switzerland exhibited the lowest list prices, while Spain and Poland had the highest. Across all countries, the treatments were generally deemed not to be cost-effective. The annual budget impact of CFTR modulators varied, with most countries considering it to be high. Nevertheless, ten of the evaluated countries fully reimbursed all modulators through MEAs, which were implemented to address financial and clinical uncertainties. However, we captured discrepancies concerning pricing, reimbursement and timely access to CFTR modulators. In Chapter 5 we introduced a communication roadmap for the disinvestment of orphan drugs under conditional reimbursement, drawing on input from Belgian stakeholders. We emphasized the importance of considerate communication towards patients and the public and underscored confidentiality, transparency, collaboration, and cohesiveness as vital for effective communication of disinvestment decisions. Our roadmap could assist policymakers in effectively communicating disinvestment decisions, ultimately benefiting society as a whole. Finally, we formulated six key recommendations for policy- and decision-makers: 1. Improve data collection at national and EU level 2. Shift from reference pricing to collective price negotiations 3. Holistically define 'Unmet Medical Need' to ensure accessibility to orphan drugs for those underserved 4. Provide a BIA template and validate it to enhance accurate forecasting of affordability 5. Educate stakeholders, including clinical and patient experts, to assess orphan drugs comprehensively 6. Dare to disinvest high-cost and ineffective orphan drugs after conditional reimbursement |
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