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.

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.…”

“…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).…”

Reflexión estratégica sobre financiación y acceso a las terapias dirigidas a enfermedades raras en España

“…Desde 2007, Italia utiliza un algoritmo específico para evaluar los medicamentos innovadores, que incluyen los dirigidos a EERR, que considera la eficacia clínica, la relación coste-efectividad, el impacto presupuestario y el grado de innovación. Para EERR, evalúan también la existencia de terapias alternativas y la extensión del efecto terapéutico, pese a que la calidad de la evidencia sea baja 41 . Una vez que la terapia haya demostrado ser innovadora y su reembolso sea aceptado, se financia con presupuestos nacionales con cargo al Fondo para medicamentos innovadores o al Fondo para medicamentos oncológicos innovadores, según corresponda 42 .…”