Are supplemental appraisal/reimbursement processes needed for rare disease treatments? An international comparison of country approaches

Abstract

Background: There is increasing recognition that conventional appraisal approaches may be unsuitable for assessing the value rare disease treatments (RDTs). This research examines what supplemental appraisal/reimbursement processes for RDTs are used internationally and how they can be characterised. A qualitative research design was used that included (1) documentation of country appraisal/reimbursement processes for RDTs via questionnaires, desk research and iterative interactions with country experts to produce country vignettes, and (2) a cross-country analysis of these processes to identify and characterise features in supplemental processes for RDTs, and compare them to countries without supplemental processes.

Results: Thirty-two of the 37 invited countries participated in this research. Forty-one percent (13/32) use supplemental processes for RDTs. Their level of integration within standard processes ranged from low to high, characterised by whether they are separate or partially separate from the standard process, adapted or accelerated standard processes, or standard processes that may be applied to RDTs. They are characterised by features implemented throughout the appraisal process. These features are mechanisms that allow application of different standards to assess the value of the medicine, support to the appraisal/decision-making process, overcome the issues of lack of cost-effectiveness, or exempt from part of/the full appraisal/reimbursement process. They increase the likelihood of reimbursement by adjusting and/or foregoing part of the assessment process, or accepting to pay more for the same added benefit as for common conditions. A large proportion of countries with standard processes include one or more of these features (formally or informally) or are discussing potential changes in their systems.

Conclusions: Results suggest revealed preferences to treat RDTs differently than conventional medicines. Some of the challenges around uncertainty and high price remain, but supplemental process features can support decision-making that is more flexible and consistent. Many of these processes are new and countries continue to adjust as they gain experience.

Keywords: Access to treatments; Appraisal; Health technology assessment; Orphan medicine; Rare disease treatment; Reimbursement; Supplemental processes; Ultra-orphan medicine.

Fig. 1

Responder characteristics (n = 33)

Fig. 2

Classification of supplemental processes by level of integration and applicability to rare versus ultra-rare. This figure provides an overview of the study countries that have supplemental processes for the routine use of rare and/or ultra-rare disease treatments in a defined patient population within a health service. * Scotland and Slovakia have two different supplemental pathways for rare and ultra-rare disease treatments respectively, which are differentiated here. ** Rare disease treatment with orphan designation from European Medicines Agency (“Orphan Medicinal Product”, OMP); ultra-rare disease treatment defined by individual country definitions, often alongside other criteria. RDT: rare disease treatment; OMP: orphan medicinal product; P&R: pricing and reimbursement process

Fig. 3

Features included in supplemental processes for rare diseases across the HTA process. Illustrates the (unique and/or different) features of supplemental processes to standard ones. They have been categorised according to their occurrence throughout the HTA process. RDT: rare disease treatment; WTP: willingness-to-pay