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 for money of rare disease treatments (RDTs). This research examines whether supplemental appraisal processes for RDTs are needed, and if so, what form should these take. 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 countries with 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 account for rarity. 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 HTA process. They apply to ultra-RDTs and/or RDTs, and are often combined. 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, which is likely to lead to accepting high prices. 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 allow for more adapted and consistent decision-making. Many of these processes are new and countries continue to adjust as they gain experience. These results do not outline what an ideal process for RDTs looks like, but identify some key features that can be implemented to facilitate this process.

Examining the impact of different country processes for appraising rare disease treatments: a case study analysis

“…This analysis of public HTA reports illustrates how standard and supplemental appraisal processes for RDTs are implemented. Although in these case studies both RDTs were generally accepted for full or conditional reimbursement, making it difficult to conclude how much of a difference supplemental processes make, they do reflect the finding by Nicod et al (11) that supplemental RDT processes have a number of different mechanisms/more formal criteria than standard processes, which can enable RDT specificities to be accounted for in the decision-making process. This includes, for instance, differing evidence requirements for RDTs, being more lenient around the quality of evidence and accepting higher uncertainty, broader considerations, and a higher WTP threshold.…”

“…Our previous work characterized the features included in these separate or adapted appraisal processes for RDTs, referred to from now as "supplemental." Supplemental processes vary across countries and include features such as different requirements for clinical and/or economic evidence, more lenience around evidence quality, greater disease-specific input from patient and clinical experts, additional considerations of value, different decision rules, different WTP thresholds, or conditional approval (Supplementary Figure 1) (11).…”

“…In some countries, standard appraisal processes are used. In others, standard appraisal processes are adapted in order to better deal with some of the common challenges encountered with RDTs or ultra-RDTs, and others have entirely separate appraisal processes for RDTs or ultra-RDTs (10;11). Our previous work characterized the features included in these separate or adapted appraisal processes for RDTs, referred to from now as “supplemental.” Supplemental processes vary across countries and include features such as different requirements for clinical and/or economic evidence, more lenience around evidence quality, greater disease-specific input from patient and clinical experts, additional considerations of value, different decision rules, different WTP thresholds, or conditional approval (Supplementary Figure 1) (11).…”

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

“…Thirty-two of 37 countries (86%) were included in the study. Vignettes for each of these were created [ 16 ]. The five non-respondent countries excluded from the study were: Australia, Croatia, Cyprus, Luxembourg, and Wales.…”

Essential List of Medicinal Products for Rare Diseases – Recommendations from the IRDiRC Rare Disease Treatment Access Working Group

“…Some are grounded in "high evidential uncertainty" in extending clinical trial data to real-world outcomes. This is highly problematic in countries that apply "traditional" health technology assessment (HTA) or valuebased assessment (VBA) methodology to RD therapies compared to those jurisdictions that use supplemental processes with greater exibilities that treat RD treatments differently [16]. The EUM4all initiative dealt with a broad range of medicines with high impact in LMICs, but we propose that the procedure could pro tably be applied to RD medicines.…”