Public spending on orphan medicines: a review of the literature

Abstract

Background and objective Little is known about how much public payers spend on orphan medicines. This study aimed at identifying information on orphan medicine expenditure incurred by public payers that was published in literature globally and at possibly synthesising their shares as portion of the total pharmaceutical expenditure. Methods A literature review was undertaken using Medline, the Orphanet Journal of Rare Diseases and Google Scholar. Titles and abstracts were screened, and full texts of potentially qualifying studies were reviewed for inclusion. Included articles were analysed, and bibliometric parameters as well as public expenditure data on orphan medicines were retrieved. Results Six hundred forty three articles excluding duplicates were identified. After screening of the abstracts and a review of the full texts, 13 articles qualified for in-depth analysis. The 13 selected articles on orphan pharmaceutical expenditure were published between 2010 and 2018. Survey periods varied between 1 year and 12 years. One publication included 22 countries but the majority of the studies were related to a single country. Expenditure data was available in five of the 13 articles, and eight articles used ‘expenditure proxies’ such as sales data. Spending data had been sourced from public institutions (4 studies), private providers (5 studies) and a combination of both (3 studies, no information on data source in 1 study). In all included studies, secondary data were analysed. Reported expenditure shares for orphan medicines in relation to total pharmaceutical spend was frequently below 3%. Countries with higher shares included the USA, Canada, the Netherlands and Bulgaria—the latter reporting spending on orphan medicines as high as 9%. Conclusions A low number of studies that informed about pharmaceutical spending on orphan medicines was published, thereof only a few explicitly analysed expenditure data of public payers. A conclusive synthesis of public spending on orphan medicines is a challenge given to the diversity in methodologies to measure expenditure. There is a need for further research to survey primary data of public spending for orphan medicines, based on a sound methodology to measure these data and to compare them internationally.

Initiating and Defining a Sustainable Project on the Example of Rare Disease Therapy

“…Operational solutions in the form of public fundraising for treatment of individual patients (out of pocket) only emphasize the weakness of the system, besides, they are possible in the case of one-off, often spectacular aid actions, and not in the case of chronic treatment (Baumbusch et al, 2018;Gombocz and Vogler, 2020). A chance for changes in this area is the Medical Fund established in 2020 with a target annual budget of PLN 4.2 billion, which will finance early and conditional access to drug technologies with high clinical value and access to drug technologies with a high level of innovation (Act of October 7, 2020 on the Medical Fund, Journal of Laws of October 26, 2020, item 1875).…”

Methodological Quality Assessment of Budget Impact Analyses for Orphan Drugs: A Systematic Review

“…In contrast, annual health expenditures of orphan drugs in Europe are generally low (Schlander et al, 2018), with a maximum per capita spending of €20.23 in France (Hutchings et al, 2014), and are forecasted to stabilize below the market growth rate at 4-5% of total pharmaceutical expenditures (Schey et al, 2011). However, a recent publication could not refute a possible slignificant impact of orphan drugs on future pharmaceutical budgets (Gombocz and Vogler, 2020).…”

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

“…On the one hand, some argue that pro-orphan drug policies address important unmet needs among persons who might otherwise experience barriers to timely and effective care and who tend to be younger and have more severe health issues [ 2 , 5 , 6 ]. They cite low associated per-capita spending [ 7 ] and the small share of pharmaceutical budgets attributable to orphan drugs [ 8 ] as evidence that the budgetary impact of orphan drugs is mitigated by the low prevalence of their indications. On the other hand, society bears the cost of subsidizing orphan drug development even though the acquisition costs of orphan drugs have outpaced increases in drug spending for common indications [ 3 , 9 , 10 ].…”