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.

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.

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.

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.

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

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. A review of official submission guidelines and analysis of comments in HTA submissions that have used different ITC methodologies. 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.