An evaluation of managed access agreements in England based on stakeholder experience.

OBJECTIVES: The objective of this research was to evaluate managed access policy in England, drawing upon the expertise of a range of stakeholders involved in its implementation. METHODS: Seven focus groups were conducted with payer and health technology assessment representatives, clinicians, and representatives from industry and patient/carer organizations within England. Transcripts were analyzed using framework analysis to identify stakeholders' views on the successes and challenges of managed access policy. RESULTS: Stakeholders discussed the many aims of managed access within the National Health Service in England, and how competing aims had affected decision making. While stakeholders highlighted a number of priorities within eligibility criteria for managed access agreements (MAAs), stakeholders agreed that strict eligibility criteria would be challenging to implement due to the highly variable nature of innovative technologies and their indications. Participants highlighted challenges faced with implementing MAAs, including evidence generation, supporting patients during and after the end of MAAs, and agreeing and reinforcing contractual agreements with industry. CONCLUSIONS: Managed access is one strategy that can be used by payers to resolve uncertainty for innovative technologies that present challenges for reimbursement and can also deliver earlier access to promising technologies for patients. However, participants cautioned that managed access is not a "silver bullet," and there is a need for greater clarity about the aims of managed access and how these should be prioritized in decision making. Discussions between key stakeholders involved in managed access identified challenges with implementing MAAs and these experiences should be used to inform future managed access policy.

Early access schemes for innovative health technologies: the views of international stakeholders.

OBJECTIVES: Early access schemes (EASs) are approaches used by payers to balance and facilitate earlier patient access to innovative health technologies while evidence generation is ongoing. Schemes require investment from payers and are associated with significant risk since not all technologies will be routinely reimbursed. The purpose of this study was to gain the perspectives of policy experts about the key challenges for EASs and potential solutions for their optimal design and implementation. METHODS: Two virtual workshops were convened including (i) UK-based policy experts (England, Wales, and Scotland) and (ii) representatives from multiple healthcare systems (England, France, Sweden, Canada, Poland, and Norway). Participants were encouraged to share their experiences with EASs in their healthcare system and highlight key challenges for policy makers. Discussions were transcribed and analyzed using framework analysis. RESULTS: Participants agreed that EASs have value when targeted toward innovative technologies with the potential for significant clinical benefit in an area of high unmet need. Participants discussed potential solutions to the challenges faced by payers implementing EASs, including defining eligibility criteria, supporting evidence generation, and approaches to reimbursement. CONCLUSIONS: Participants agreed that EASs are one possible solution for their healthcare systems and have the potential to deliver significant clinical value to patients. However, widespread adoption of EASs is limited due to concerns about the risks for patients and healthcare budgets, further solutions are needed to deliver EASs for targeted therapies.

An Analysis of Uncertainties and Data Collection Agreements in the Cancer Drugs Fund.

BACKGROUND: Managed Access Agreements (MAAs) are a commercial arrangement that provide patients earlier access to innovative health technologies while uncertainties in the evidence base are resolved through data collection. In the UK, data collection agreements (DCAs) outline the evidence that will be collected during the MAA period and are intended to resolve uncertainties in the clinical- and cost-effectiveness of a technology sufficient for the National Institute of Health and Care Excellence (NICE) committee to make a final decision on reimbursement. OBJECTIVE: The aim of this study was to identify the primary uncertainties leading to a recommendation for entry to the Cancer Drugs Fund (CDF) and evaluate how the corresponding DCAs attempt to address these. METHODS: A database of MAAs agreed within the CDF was compiled with coverage between July 2016 and December 2020 (the time during which evidence generation was routinely collected within the CDF up until the time of analysis). Uncertainties in the evidence base for technologies entering the CDF were analysed alongside the outcomes planned for data collection during the MAA. These data provide an overview of the key uncertainties surrounding health technologies in the CDF on entry and the types of evidence targeted by DCAs. RESULTS: In the assessment of 39 Cancer Drugs Fund (CDF) cases, NICE committees identified a total of 108 key uncertainties in cost-effectiveness estimates. Overall survival was the most commonly identified uncertainty, followed by generalisability of the evidence to the target population. DCAs specified a range of outcomes relevant to understanding the clinical effectiveness of the technology, though fewer than half (43.6%) of the DCAs addressed all the key uncertainties identified by the NICE committee. CONCLUSION: The analysis indicated that data collection within the CDF is not sufficient to resolve all the uncertainties identified by the NICE committee, meaning that other approaches will be needed at re-appraisal to ensure that the NICE committee can reach a final decision on reimbursement.

Payer and Implementation Challenges with Advanced Therapy Medicinal Products (ATMPs).

Advanced therapy medicinal products (ATMPs) are a dynamic and current topic for healthcare systems, with new products progressing to market at an increasing rate. ATMPs highlight the growing gap between payer and regulator requirements; the limited evidence base combined with pressure to implement rapidly is exacerbating the clinical and financial uncertainties associated with these products. There are a number of key uncertainties with ATMPs related to implementation and healthcare planning-these uncertainties at the time of evaluation have the ability to change the value proposition of products. ATMPs also have the potential to reduce the amount of net health gain available to healthcare systems, and evaluators should consider the opportunity cost when seeking to accelerate access to one-off therapies with a limited clinical evidence base. Therefore, ATMPs have the potential to transform clinical care pathways, but implementation challenges and application of key health economic principles may highlight the requirement to exercise caution.

Analysis of indirect treatment comparisons in national health technology assessments and requirements for industry submissions.

Aim: To determine the preferred methodologies of health technology assessment (HTA) agencies across Europe, Canada and Australia to ascertain acceptance of indirect treatment comparisons (ITC) as a source of comparative evidence. Method: A review of official submission guidelines and analysis of comments in HTA submissions that have used different ITC methodologies. Conclusion: ITC is generally accepted as a technique that allows demonstration of noninferiority to a comparator provided the chosen methodology and underlying assumptions are clear and justified. However, HTA agencies are more likely to closely scrutinize submitted data and evaluate statistical significance of results when superiority is claimed. In addition, the HTA agencies in scope tended to be cautious and only accept ITC data as support for similarity of treatments.