Fair pricing of “old” orphan drugs: considerations for Canada’s orphan drug policy

Roberts, Eve A.; Herder, Matthew; Hollis, Aidan

doi:10.1503/cmaj.140308

Cited by 10 publications

(4 citation statements)

References 17 publications

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“…As Roberts stated in his paper, it is common experience that generic ODs are rarely produced and, however, their prices remain exceptionally high. 20 Other analyses about RD drugs' expenditures have been performed, but often focusing only on ODs and especially on the ones with ongoing orphan designation. They usually miss other high cost drugs without orphan designation or with an expired one.…”

Section: Discussionmentioning

confidence: 99%

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High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

Manea¹,

Pozza²,

Minichiello³

et al. 2023

Health Serv Manage Res

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Background: in the field of rare diseases (RDs) most of the European studies on budget impact analysis of drugs that have been conducted often lay on theoretical assumptions and focus only on Orphan drugs (ODs). Objectives: we aimed to estimate the budget impact of specific drugs for non-oncological RDs, both ODs and non-ODs, using real-world data about patients residing in Veneto Region (Italy) and to describe its expenditure structure and dynamics. Methods: a population-based multi-source observational study was conducted using data from Regional administrative databases; an ad-hoc drugs’ list specific for RDs including both ODs and non-ODs and classifying them by ATC codes has been created. Results: In 2019, the total expenditure for drugs specific for RDs was EUR 97.2 million (6.6% of the total Regional budget). The RD drug list included 58 ATC codes, of which 15 ATC had an annual budget impact over EUR 1 million (“blockbuster drugs”). The most expensive treatment was a non-OD drug (Coagulation factor VIII). The two most represented therapeutical areas were the metabolic and the hematological ones. Conclusions: Cost analyses on RD high-cost drugs expenditure should consider any specific RD drug, not only ODs. Expenditure dynamics for RD drugs are peculiar showing “blockbuster drugs”. Some therapeutical areas seem to be lacking in the drug research field.

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Section: Discussionmentioning

confidence: 99%

“…As Roberts stated in his paper, it is common experience that generic ODs are rarely produced and, however, their prices remain exceptionally high. 20…”

Section: Discussionmentioning

confidence: 99%

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

Manea¹,

Pozza²,

Minichiello³

et al. 2023

Health Serv Manage Res

View full text Add to dashboard Cite

show abstract

“…22 Finally, any orphan drug policy with a goal of making drugs more affordable should not only aim to publicly subsidize the cost of these medications but should also focus on reducing the prices that companies charge. 23…”

Section: Discussionmentioning

confidence: 99%

Does an Orphan Drug Policy Make a Difference in Access? A Comparison of Canada and Australia

Lexchin

Moroz

2019

Int J Health Serv

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Canada has been discussing whether to implement an orphan drug policy for more than 25 years. Recently, the federal government announced funding for orphan drugs starting in 2022, and the Canadian Senate has recommended that the country develop an orphan drug policy. This paper uses a list of orphan drugs approved by the United States Food and Drug Administration between 2008 and 2017, inclusive. It then compares Canada, which has no orphan drug policy, and Australia, which has had such a policy since 1997. There was no difference between the countries in the proportion of orphan drugs approved, the time drugs spent in the regulatory review process, and any delay in marketing the drugs in the respective countries compared to the United States. Both Canada and Australia approved virtually all of the drugs that offered a moderate to significant therapeutic improvement. If Canada hopes to provide faster access to orphan drugs, especially those that are therapeutically innovative, it will need to develop a policy that is significantly different from that in Australia.

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“…10 Other examples where value-based pricing of known active substances caused public outcry are chenodeoxycholic acid in Europe and trientine in Canada. 11,12 Considerations about what would be a fair price in such cases are frequently hampered by lack of transparency and background information on costs that justify the commercial price. 13 Cost-based pricing may provide a suitable alternative for these cases of repurposed orphan drugs.…”

Section: Introductionmentioning

confidence: 99%

Cost-Based Price Calculation of Mexiletine for Nondystrophic Myotonia

et al. 2021

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Objectives: Mexiletine is a long-known drug used for the treatment of arrhythmias and repurposed in the 1980s for patients with nondystrophic myotonia (NDM). Recently, the price of mexiletine in Europe increased significantly after registration as an orphan drug for NDM. This led to international discussions on affordability and willingness to reimburse mexiletine in the absence of background information that would justify such a price. Our objective was to calculate a cost-based price for mexiletine for adult patients with NDM based on detailed information on development costs.Methods: We calculated a fair price based on a cost-based pricing model for commercial mexiletine to treat adults with NDM using a recent European drug-pricing model as a framework to include actual costs incurred. Three scenarios were applied: 1 with minimum estimated costs, 1 with maximum estimated costs, and 1 with costs as if mexiletine was innovative.Results: The calculated fair price of mexiletine per patient per year (PPPY) is V452 for the minimum scenario and V1996 for the maximum scenario. By using hypothetical R&D costs used for innovative drugs, the price would be V6685 PPPY. In Europe, the list price of mexiletine ranges from V30 707-60 730 PPPY, based on 600 mg daily. Conclusions:The current list price for mexiletine in Europe is manifold higher than any scenario of the cost-based models. Accounting for the reduced costs for clinical development in a repurposing scenario, the cost-based pricing model provides a fair commercial price range, which can be used as benchmark for pricing negotiations and/or reimbursement decisions.

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Fair pricing of “old” orphan drugs: considerations for Canada’s orphan drug policy

Cited by 10 publications

References 17 publications

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

High-cost drugs for rare diseases: their expenditure and value based on a regional area-based study

Does an Orphan Drug Policy Make a Difference in Access? A Comparison of Canada and Australia

Cost-Based Price Calculation of Mexiletine for Nondystrophic Myotonia

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