This is the subject of a new paper Drummond et al. (2022)When a medical device receives regulatory approval, the payer can make one of three coverage decisions: (1) Approval, (2) Denial, or (3) Coverage Evidence Development (CED). Note that all countries approve medical devices based on safety and efficacy, but only the UK, Netherlands and Belgium also rely on cost-effectiveness models to inform medical device reimbursement decisions. If you choose CED, there are two types:
- (3a): Approved in Research Only (OIR). This means that the device is only approved for use in patients in post-registration confirmation studies (PRS), or
- (3b) Approved by Research Only (OWR). This means that the device is approved for use in all eligible patients provided that the manufacturer performs one or more PRS.
Implementation of CED may be based on 4 stages of discussion, such as Garrison et al. (2013) (i) Evaluate the desirability of the CED Program, (ii) Design the Program, (iii) Implement the Program, and (iv) Evaluate the Program. The figure below summarizes this approach, and I briefly summarize Drummond et al.’s key considerations at each stage. 2022 paper.
- assess the desirability: Usually occurs during the reimbursement process. For example, “In France, a request to conduct a post-registration study (PRS) (LPPR) can be issued for any technology for which the manufacturer has identified relevant evidence gaps during the initial request for registration in the List of Reimbursable Products and Procedures. The application for PBRSA is made by The Council for Evaluation of Medical Devices and Health Technologies (CNEDiMTS) clearly states that, if accepted by the Ministry of Health, the renewal of registration for the LPPR (after approximately 3 years) is conditional on the provision of new evidence from the applicant.” The desirability of developing coverage around evidence is often lacking A formal assessment of the information; for example, no formal “information value” analysis is conducted. However, demand for CEDs may be influenced by the requirements of key stakeholders, such as clinical groups, hospitals or manufacturers. A appears Green et al. (2016) A checklist of questions is provided to determine the potential need for an Administrative Entry Agreement (MEA), the level of uncertainty involved, and whether the uncertainty can be resolved at a reasonable cost by the MEA. “In some countries (e.g. UK, France, Germany and Belgium) a deliberative approach is adopted based on pre-specified criteria, while in others (e.g. Spain, Netherlands and Switzerland) quantitative tools or checklists are used to select and prioritize options .”
- Design CED solutions. Key decisions here include the type of conditional approval (ie, OWR or OIR), the research question of interest, the research method (ie, RCT vs. observation), and the duration of the study. With regard to research questions, key topics can include not only confirmation of clinical efficacy, but also (i) durability of efficacy effects, (ii) efficacy in subgroups of patients, (iii) effects of learning effects on performance, (iv) real-world Absorption rates and conditions of use, (eg, including potential off-label use; patient compliance rates). While RCTs are often preferred, for medical devices RCTs are often difficult to implement in practice (eg, blinding may not be possible, patients may not want to participate in a “control” group once a treatment is approved) and may be unethical. Another question is whether studies should prespecify target outcomes. While this makes sense in a clinical trial setting, standards of care may evolve over time, making fixed, pre-specified standards difficult to implement in practice. Most studies supporting CED last 2 to 5 years, but Drummond et al. (2015) noted that plans longer than 3 years may be distracted from the top priorities of policymakers. The Appointment-by-Evaluation (CtE) scheme in England usually has a fixed term of 2 years.
- Implement the CED program. A key question is who will fund these studies. In France, manufacturers fund data collection and analysis for post-registration studies (PRS), so about 15% of devices evaluated for inclusion in the list of products and procedures for reimbursement (LPPR) require PRS. On the other hand, the CED schemes in England and Germany are publicly funded and centrally administered, so the number of PRSs for devices was only 5 (UK) and 10 (Germany) during the most recent 5-year period. PRS design becomes complicated when new equipment enters the market or a new generation of the same product enters the market.
- Evaluate CED Programs. Reviewing CED retrospectively helps (i) to better design future CED programs for other products, and (ii) to make any decisions based on the results of the CED program (eg, should it be repaid in perpetuity?; if so, the price is How much?). The authors note that “many devices are used in hospital settings and funded as part of bundled payments (eg, DRG payments) for procedures that use the device.” Therefore, if the CED shows that the new device is not only safe and effective, but also value for money, DRG payments may need to be adjusted more frequently.
What are the results of most CEDs?
In the European program, which ended in 2014 to 2019, the results led to final reimbursement decisions, most of which resulted in unlimited and unconditional reimbursement of equipment.
What advice do Drummund and co-authors have? They made 6 specific recommendations:
- Define the purpose of the CED scenario in terms of the uncertainty to be addressed
- Apply VOI where feasible, or at least apply VOI principles
- Reflects the nature of uncertainty in research design
- Balancing scientific and practical considerations when determining the length of a CED program
- Identify early decisions to be made at the end of the CED program
- Provide good reasons for deviating from common CED principles
appendix.
Five elements of good practice for performance-based drug risk-sharing agreements:
… (i) develop strategies to guide the use of PBRSA for medicines; (ii) ensure they are only used when the benefits of additional evidence outweigh the costs of negotiating and enforcing the agreement; (iii) clearly identify the uncertainty, to ensure data sources and study design are appropriate to address uncertainty; (iv) implement a governance framework that ensures transparency of the process and allows payers to take action based on additional evidence, including withdrawal from agreements and possible revocation of contingent insurance; (v) Ensure minimal transparency of content, limiting confidentiality to potentially commercially sensitive parts of the agreement (especially price).
