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Objective This research assesses the impact of an outcome-based payment arrangement (OBA) linking complete remission (CR) to survival as a means of maintaining cost-effectiveness for a chimeric antigen receptor T cell (CAR-T) ther...
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Objective This research assesses the impact of an outcome-based payment arrangement (OBA) linking complete remission (CR) to survival as a means of maintaining cost-effectiveness for a chimeric antigen receptor T cell (CAR-T) therapy in young patients with acute lymphoblastic leukemia (ALL). Methods A partitioned survival model (PSM) was used to model the cost-effectiveness of tisagenlecleucel versus blinatumomab in ALL from the Australian healthcare system perspective. A decision tree modeled different OBAs by funneling patients into a series of PSMs based on response. Outcomes were informed by individual patient data, while costs followed Australian treatment practices. Costs and quality-adjusted life years (QALYs) were combined to calculate a single incremental cost-effectiveness ratio (ICER), reported in US dollars (2022) at a discount rate of 5% on costs and outcomes. Results For the base case, incremental costs and benefit were $379,595 and 4.27 QALYs, giving an ICER of $88,979. The ICER was most sensitive to discount rate ($57,660-$75,081), "cure point" ($62,718-$116,206) and extrapolation method ($76,018-$94,049). OBAs had a modest effect on the ICER when response rates varied. A responder-only payment was the most effective arrangement for maintaining the ICER ($88,249-$89,434), although this option was associated with the greatest financial uncertainty. A split payment arrangement (payment on infusion followed by payment on response) reduced variability in the ICER ($82,650-$99,154) compared with a single, upfront payment ($77,599-$107,273). Conclusion OBAs had a modest impact on reducing cost-effectiveness uncertainty. The value of OBAs should be weighed against the additional resources needed to administer such arrangements, and importantly overall cost to government.
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Access to highly expensive and innovative medicine is a challenge. Managed entry agreements/ arrangements (MEAs) consist of various forms of confidential agreements between pharmaceutical manufacturers and payers (hospitals, socia...
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Access to highly expensive and innovative medicine is a challenge. Managed entry agreements/ arrangements (MEAs) consist of various forms of confidential agreements between pharmaceutical manufacturers and payers (hospitals, social insurance), which are mainly negotiated when there is uncertainty of the actual clinical benefit of the medicines, but high public expenditures are required. Continued and expanding use of these schemes requires a greater understanding of payer perceptions, experience, and orientation towards their current and future use. This literature review aimed to identify the stakeholders’ concerns with regards to MEAs. A total of 20 articles were identified between 2007 and 2018. MEAs are mostly studied in the first world countries, whereas limited documentation is available for other nations. The reasons for rejection of proposed MEAs could possibly be related to existing low-cost effective treatments, lack of trust between stakeholders, high administrative burden and if the authorities believe they are funding the substantial proportion of the drug’s development cost. Lack of well-designed and easy-to-use data collection methods for MEAs has an impact on the evidence generation. Most articles highlighted the lack of transparency of the agreements which may not benefit other health systems, especially for systems that are dependent on international referencing price. Stakeholders also identified the lack of expertise in assessing the pharmacoeconomic and health outcomes data and challenges in assessing risks up-front due to the complexity of the real-world data. Lack of transparency in most health systems are also a hindrance to further understanding and confidence in the MEAs. However, the views of the stakeholders may also evolve with experience. Though there are challenges in the designing and implementation of MEAs, close networking, improved human capital development especially in the area of health technology assessment and health economics would be able to close the gap in the implementation.
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? 2023 Fellowship of Postgraduate MedicineObjectives: This paper illustrates the results of a research aimed at investigating the opinions collected from selected European payers (HTA organisations, authorities/committees assessin...
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? 2023 Fellowship of Postgraduate MedicineObjectives: This paper illustrates the results of a research aimed at investigating the opinions collected from selected European payers (HTA organisations, authorities/committees assessing, appraising and negotiating drug prices) and experts (researchers/consultants identified through LinkedIn groups) on drug price regulation, managed entry agreements, transparency and HTA advice. Methods: Expert and payer opinions were gathered through a structured questionnaire, validated by three potential respondents and self-administered online between July and November 2021. Results: Respondents totalled 39 (response rate 29%). The response rate was higher among the experts than the payers. Respondents mostly agreed that price regulation should award drug value through a multiple criteria approach (21 respondents) or cost-effectiveness evidence (14). For most respondents the added therapeutic value and the comparative safety profile should be the main drivers of a premium price. A quite high proportion of respondents supported the use of cost-effectiveness, and suggest relying on the perspective of the health care system. Most respondents expect larger diffusion of outcome-based and financial-based managed entry agreements in the future. Finally, respondents advocated for higher transparency of the negotiation process rather than net price transparency, and expressed the belief that HTA advice could be useful in reaching consensus on the level of unmet need, the comparators to consider, and the dimension of the target population. Conclusions: Despite the limited number of respondents, the paper provides very interesting exploratory insights into much-debated topics related to drug price regulation. The opinions of European payers and experts are very useful for future regulation of drug pricing in Europe. Public interest summary: Our research aimed at gathering the opinions of payers and experts on drug price regulation. The main findings are that pricing should reflect the value of medicines, that a premium price should be awarded only to those drugs that provide for an added therapeutic value and/o a better safety profile, even if other value dimensions (patient preferences and organisational impact) should be not disregarded. Experts and payers expect a larger role of managed entry agreements in the future, despite they may impose an important administrative burden. Finally, transparency of price negotiation is prioritized compared to net price transparency.
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The process of introducing a new health technology into a healthcare system is characterized by uncertainty and risk for those involved-pharmaceutical companies, payers, patients and the government. In view of the accelerated intr...
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The process of introducing a new health technology into a healthcare system is characterized by uncertainty and risk for those involved-pharmaceutical companies, payers, patients and the government. In view of the accelerated introduction of new technologies in recent years, mechanisms to reduce uncertainty are of growing interest. One example is the Managed Entry Agreement (MEA), which we explore using a mechanism design approach. We make use of the Israeli experience, in which pharmaceutical companies and health plans (i.e., payers) negotiate over the introduction of new technologies into the national Health Services Basket (HSB) with the Ministry of Health acting as a mediator. We use the framework of bargaining within a mechanism design framework to show that in the process of negotiation the parties, the pharmaceutical company (PC) and the health plan (HP), have independent private valuations and that a situation of common knowledge that gains from MEA exists is rare. Adding a mediator (i.e., the MEA team) to the mechanism, as in a direct-revelation mechanism, reduces the level of uncertainty for both sides (i.e., the PC and the HP), thus making it possible to meet the budget constraint while increasing value for patients and enhancing ex-post efficiency.
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Slovakia has adopted an amendment to Act No. 363/2011, regulating, among other things, drug reimbursement and is undergoing a significant change in the availability of innovative treatments for patients. High expectations are asso...
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Slovakia has adopted an amendment to Act No. 363/2011, regulating, among other things, drug reimbursement and is undergoing a significant change in the availability of innovative treatments for patients. High expectations are associated with arrangements related to performance-based managed entry agreements. Opinions and positions towards this change appear to be inconsistent, and for the further application of the law in practice and when setting up the main implementation processes, it is necessary to understand the positions and opinions of the individual actors who are involved in the PB-MEA process. The interviews were conducted in the period from 20 May to 15 August 2022 around the same time as the finalisation of the amendment to Act No. 363/2011 and its adoption. A roughly one-hour open interview was conducted on a sample of 12 stakeholders in the following groups: representatives of the Ministry of Health, health-care providers, pharmaceutical companies and others, including a health insurance company. The main objective was to qualitatively describe the perception of this topic by key stakeholders in Slovakia. The responses were analysed using MAXQDATA 2022 software to obtain codes associated with key expressions. We identified three main strong top categories of expressions that strongly dominated the pro-management interviews with stakeholders: legislation, opportunities and threats. Ambiguity and insufficient coverage of the new law, improved availability of medicinal products and threats associated with data, IT systems and potentially unfavourable new reimbursement schemes were identified as key topics of each of the said top categories, respectively. Among individual sets of respondents, there is frequent consensus on both opportunities and threats in the area of implementing process changes in PB-MEA. For the successful implementation of the law in practice, some basic threats need to be removed, among which in particular is insufficient data infrastructure.
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Background: Traditional drug payment schemes in Catalonia are generally based on the negotiation of fixed prices; however, disadvantages arise in the case of innovative therapies. Risk sharing agreements distribute potential healt...
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Background: Traditional drug payment schemes in Catalonia are generally based on the negotiation of fixed prices; however, disadvantages arise in the case of innovative therapies. Risk sharing agreements distribute potential health and economic uncertainties and high prices on access across the interested parts. Objectives: To identify, characterize and analyze current publicly available agreement reports signed by the Catalan Health Service and different pharmaceutical companies evaluating the current market access scene for new drugs in Catalonia. Methods: A database of agreements implemented between 2013 and 2018 was developed by using publicly available data. Data analysis was performed in a descriptive way, presenting summaries in datasheets. Results: A total of 7 managed entry agreements were analyzed. Two extensions regarding previous agreements were also taken into account. The main involved disease area is oncology (57%) and the most common length is 1 year, whereas the longest is 3 years. Conclusions: Managed entry agreements are gaining popularity and are viewed as positive schemes by stakeholders, payers and health services, leading to a general increase of accords during the last years. However, there are hardly any studies regarding the impact of RSA post-implementation, a field of great relevance regarding health policies.
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Background: Traditional drug payment schemes in Catalonia are generally based on the negotiation of fixed prices; however, disadvantages arise in the case of innovative therapies. Risk sharing agreements distribute potential healt...
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Background: Traditional drug payment schemes in Catalonia are generally based on the negotiation of fixed prices; however, disadvantages arise in the case of innovative therapies. Risk sharing agreements distribute potential health and economic uncertainties and high prices on access across the interested parts. Objectives: To identify, characterize and analyze current publicly available agreement reports signed by the Catalan Health Service and different pharmaceutical companies evaluating the current market access scene for new drugs in Catalonia. Methods: A database of agreements implemented between 2013 and 2018 was developed by using publicly available data. Data analysis was performed in a descriptive way, presenting summaries in datasheets. Results: A total of 7 managed entry agreements were analyzed. Two extensions regarding previous agreements were also taken into account. The main involved disease area is oncology (57%) and the most common length is 1 year, whereas the longest is 3 years. Conclusions: Managed entry agreements are gaining popularity and are viewed as positive schemes by stakeholders, payers and health services, leading to a general increase of accords during the last years. However, there are hardly any studies regarding the impact of RSA post-implementation, a field of great relevance regarding health policies.
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Background: Healthcare payers are increasingly adopting managed entry agreements (MEAs) between themselves and manufacturers, to overcome the challenge of sustaining access in an era of innovative and high-cost medicines. This stu...
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Background: Healthcare payers are increasingly adopting managed entry agreements (MEAs) between themselves and manufacturers, to overcome the challenge of sustaining access in an era of innovative and high-cost medicines. This study aims to investigate current MEA activity in Saudi Arabia and explore the challenges encountered when implementing such agreements. Methods: An explanatory sequential mixed-methods approach was used. Quantitative data on MEAs were collected, followed by qualitative semi-structured interviews with different stakeholders. Results: Our questionnaire garnered responses from 18 pharmaceutical companies from 3 different continents and identified 25 agreements in Saudi Arabia since 2010. Financial-based agreements were more prevalent than outcomes-based agreements at 44% versus 32%, respectively. Stakeholders showed positive attitudes toward MEAs, valuing their benefits in facilitating market access for both costly and innovative medicines. The main challenges included data availability, administrative and financial burden, lack of expertise, confidentiality, and lack of clear regulations. Conclusions: Despite clear implementation challenges, a growing tendency toward MEAs exists in Saudi Arabia because of the potential benefits they bring to patients, healthcare providers, payers, and manufacturers. It is believed that the newly established health technology assessment center in Saudi Arabia will bring more clarity and shape the concept of MEAs in the country.
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There has been an increasing demand to reimburse high-cost medicines, through public health insurance schemes in Thailand.A mixed method approach was employed. First, a rapid review of select high-income countries was conducted, f...
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There has been an increasing demand to reimburse high-cost medicines, through public health insurance schemes in Thailand.A mixed method approach was employed. First, a rapid review of select high-income countries was conducted, followed by expert consultations and an in-depth review of three countries: Australia, England and Republic of Korea to understand reimbursement mechanisms of high-cost medicines. In Thailand, current pathways for reimbursing high-cost medicines reviewed, the potential opportunity cost estimated, and stakeholder consultations were conducted to identify context specific considerations.High-income countries reviewed have implemented a variety of pathways and mechanisms for reimbursing high-cost medicines under specific eligibility criteria, listing processes, varying cost-effectiveness thresholds and special funding arrangements. In Thailand, high-cost medicines that do not offer good value-for-money are excluded from the reimbursement process. A framework for reimbursing high-cost medicines that are not cost-effective at the current willingness-to-pay threshold was proposed for Thailand. Under this framework, specific criteria are proposed to determine their eligibility for reimbursement such life-saving nature, treatment of conditions with no alternative treatment options, and affordability.High-cost medicines may become eligible for reimbursement through alternative mechanisms based on specific criteria which depend on each context. The application of HTA methods and processes is important in guiding these decisions to support sustainable access to affordable healthcare in pursuit of Universal Health Coverage (UHC).
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Background Withdrawal of conditional regulatory approval or subsidization of new medicines when subsequent evidence does not confirm early trial results may not be well understood or accepted by the public. Objectives We present a...
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Background Withdrawal of conditional regulatory approval or subsidization of new medicines when subsequent evidence does not confirm early trial results may not be well understood or accepted by the public. Objectives We present a case study of the US Food and Drug Administration (FDA)’s decision to withdraw the indication of bevacizumab for the treatment of advanced breast cancer and include an analysis of the reactions of stakeholders with a view to identifying opportunities for improving risk management for new medicines with conditional approval or funding. Methods We drew on a range of information sources, including FDA documents, medical journals and media reports, to describe the evidentiary basis of the FDA decisions. We analysed the reactions and perspectives of the stakeholders. Results In 2008 bevacizumab was granted conditional approval for treatment of advanced breast cancer by the FDA pending submission of supplementary satisfactory evidence. In 2011 the FDA decision to withdraw the indication was met with a hostile reaction from many clinicians and cancer survivors. There were different interpretations of the therapeutic value of bevacizumab with strong beliefs among cancer survivors that the medicine was effective and potential harm was manageable. High expectations of the public may have been encouraged by overly positive media reports and limited understanding by the public of the complexity of the scientific evaluation of new medicines and of the regulatory processes. Conclusions Improving understanding and acceptance of approval or coverage schemes conditional to evidence development may require the development of risk management plans by regulatory and funding institutions. They may include a range of strategies such as requirements for formal patient acknowledgment of the conditional availability of the medicine, ‘black-triangle’ equivalent labels that identify full approval is based on pending evidence, and ongoing communication with the media, public and health professionals.
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