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 crosscountry 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.

How to Value Orphan Drugs? A Review of European Value Assessment Frameworks

“…Previous research has focused on the identification, description or legitimacy of OMP VAFs implemented by decision-making bodies in Europe ( Nicod et al, 2020 ; Zelei et al, 2016 ; Bourke et al, 2018 ; Picavet et al, 2014b ; Szegedi et al, 2018 ; Nicod et al, 2017 ; Hughes et al, 2005 ; Hughes et al, 2007 ; Wagner et al, 2016 ; Annemans et al, 2017 ; Sussex et al, 2013b ; Schey et al, 2017 ; Towse and Garau 2018 ; Garrison et al, 2018 ; Drummond and Towse 2014 ; Simoens 2012 ; Simoens et al, 2012 ). Our study adds to the literature by providing an extensive and integrated overview of the strengths and weaknesses of OMP VAFs that are cited in the literature.…”

“…Previously, researchers in the field of health policy have mainly focused on the appraisal criteria in the context of OMPs ( Zelei et al, 2016 ; Bourke et al, 2018 ; Picavet, et al, 2014a ; Szegedi et al, 2018 ; Nicod et al, 2017 ), arguments in favor or against a special reimbursement status for OMPs ( Simoens et al, 2012 ; Hughes et al, 2005 ; Picavet, et al, 2012b ), proposals concerning existing or conceptual VAFs tailored to the needs of OMPs ( Sussex et al, 2013b ; Wagner et al, 2016 ; Annemans et al, 2017 ; Schey et al, 2017 ), or discuss ethical, social, or other features of specific VAFs for OMPs ( Drummond and Towse 2014 ; Simoens 2014 ; Schlander et al, 2016 ; Towse and Garau 2018 ). Most recently, a study was published by Nicod et al mapping the different VAFs that jurisdictions have implemented ( Nicod et al, 2020 ). As of yet, however, no publication exists that provides an overview of the strengths and weaknesses that are associated with each of these VAFs.…”

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 value-based assessment (VBA) methodology to RD therapies compared to those jurisdictions that use supplemental processes with greater flexibilities that treat RD treatments differently [ 23 ].…”

Assessing the value of orphan drugs using conventional cost-effectiveness analysis: Is it fit for purpose?

“…Indeed, preference for this ideal over pure utilitarianism (in the way that it is defined by health economists) has been routinely demonstrated in preference research [ 39 ]. Many would agree that persons with severe rare diseases are either in greater need than the general public or, at least, should not have societal benefits curtailed for them simply because of the low prevalence of their disease [ 3 , 40 , 41 ].…”