Rare diseases in healthcare priority setting: should rarity matter?

Abstract

Rare diseases pose a particular priority setting problem. The UK gives rare diseases special priority in healthcare priority setting. Effectively, the National Health Service is willing to pay much more to gain a quality-adjusted life-year related to a very rare disease than one related to a more common condition. But should rare diseases receive priority in the allocation of scarce healthcare resources? This article develops and evaluates four arguments in favour of such a priority. These pertain to public values, luck egalitarian distributive justice the epistemic difficulties of obtaining knowledge about rare diseases and the incentives created by a higher willingness to pay. The first is at odds with our knowledge regarding popular opinion. The three other arguments may provide a reason to fund rare diseases generously. However, they are either overinclusive because they would also justify funding for many non-rare diseases or underinclusive in the sense of justifying priority for only some rare diseases. The arguments thus fail to provide a justification that tracks rareness as such.

How Can We Optimize the Value Assessment and Appraisal of Orphan Drugs for Reimbursement Purposes? A Qualitative Interview Study Across European Countries

“…For instance, participants generally believed that ethical considerations such as disease severity and unmet need should play a significant role when considering an orphan drug’s reimbursement. However, consistent with existing literature, the trade-off between ethical and economic considerations remains debatable ( McCabe 2010 ; Henderson et al, 2020 ; Picavet et al, 2013 ; Albertsen 2021 ; McCabe et al, 2005 ; Drummond and Towse 2014 ). In addition, our results remained inconclusive as to whether disease rarity legitimizes the implementation of a separate framework for (ultra-)orphan drugs or to what extent this criterion warrants more flexibility when assessing these treatments.…”

“…Whereas previous research in this area has focused on the reimbursement frameworks for orphan drugs in general – their identification, description and legitimacy – ( Nicod et al, 2020a ; Zelei et al, 2016 ; Bourke et al, 2018 ; Picavet et al, 2014b ; Szegedi et al, 2018 ; Nicod et al, 2019 ; Hughes et al, 2005 ; Hughes et al, 2007 ; Wagner et al, 2016 ; Annemans et al, 2017 ; Drummond and Towse 2014 ; Schey et al, 2017 ; Simoens 2012 ; Simoens et al, 2012 ; Degtiar 2017 ; Rosenberg-Yunger et al, 2011 ; Denis et al, 2010 ; Albertsen 2021 ; Schey et al, 2020 ) the present study investigated how experts perceive the reimbursement (value assessment and appraisal) process for orphan drugs in their country and how it can be optimised. In this regard, it has identified the positive attributes, barriers and external factors that impact the process.…”

Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study

“…Rare or ‘orphan’ disorders such as EB previously attracted little attention from biopharmaceutical companies; developing and bringing new therapies to market was considered financially unviable while benefitting relatively few individuals. However, arguments for developing new therapies for rare diseases may be more complex than a straight cost/benefit analysis 10 . Increased attention is focussing on the economics of delivering lifelong care with some governments and regulatory authorities offering financial frameworks to support development of new therapies 11–15 …”

“…However, arguments for developing new therapies for rare diseases may be more complex than a straight cost/benefit analysis. 10 Increased attention is focussing on the economics of delivering lifelong care with some governments and regulatory authorities offering financial frameworks to support development of new therapies. [11][12][13][14][15] Costs of illness studies are important in defining healthcare and research priorities for governments and biopharmaceutical companies.…”

Comparison of Rare and Common Diseases in the Setting of Healthcare Priorities: Evidence of Social Preferences Based on a Systematic Review

“…1,2 The potential social preference for rare disease health resource allocation has generated considerable debate among scholars, healthcare providers and policymakers, arguing around the rationality and plausibility behind the egalitarian viewpoint. 3,4 Some perspectives argue for the equitable distribution of resources across rare and common diseases, focusing on the severity of the condition as the driving factor. 5 This approach holds that treatments for severe rare diseases should not be disregarded when determining funding priorities.…”