Spending For Orphan Indications Among Top-Selling Orphan Drugs Approved To Treat Common Diseases

Chua, Kao-Ping; Kimmel, Lauren; Conti, Rena M.

doi:10.1377/hlthaff.2020.01442

Cited by 20 publications

(17 citation statements)

References 11 publications

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“…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 ]. Further still, these policies are susceptible to manipulation, for example via “partial” orphan strategies whereby a drug is first approved for an orphan indication then for a common one but without an associated adjustment to the price [ 9 , 11 – 13 ].…”

Section: Introductionmentioning

confidence: 99%

“…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 ]. Further still, these policies are susceptible to manipulation, for example via “partial” orphan strategies whereby a drug is first approved for an orphan indication then for a common one but without an associated adjustment to the price [ 9 , 11 – 13 ]. Both arguments are reasonable, but neither is complete without also considering the value that orphan drugs generate for patients with rare diseases and the broader society.…”

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

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

Postma

Noone²,

Rozenbaum

et al. 2022

Orphanet J Rare Dis

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Conventional cost-effectiveness analysis—i.e., assessing pharmaceuticals through a cost per quality-adjusted life year (QALY) framework—originated from a societal commitment to maximize population health given limited resources. This "extra-welfarist" approach has produced pricing and reimbursement systems that are not well- aligned with the unique considerations of orphan drugs. This framework has been slow to evolve along with our increased understanding of the impact of rare diseases, which in turn has complicated the assessment of orphan drugs meant to treat rare diseases. Herein, we (i) discuss the limitations of conventional cost-effectiveness analysis as applied to assessing access to, as well as the pricing and reimbursement of, orphan drugs, (ii) critically appraise alternative and supplemental approaches, and (iii) offer insights on plausible steps forward.

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Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

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

Postma

Noone²,

Rozenbaum

et al. 2022

Orphanet J Rare Dis

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“…The clinical front has witnessed the development and release of multiple contemporary therapeutic approaches for the management of MD. The high developmental cost associated with orphan drugs to treat rare metabolic disorders [ 36 , 37 ] and their limited usage [ 38 ] across the world [ 39 ] due to the high cost [ 40 , 41 , 42 , 43 ] has led to the proposition of novel approaches such as the development of personalized therapeutics using RNA interference (RNAi)-based platforms [ 44 ]. Concerns regarding the inherent instability of the RNA molecule and difficulties in the industrial manufacture initially resulted in the idea of employing messenger RNA (mRNA) as a therapeutic molecule with skepticism.…”

Section: Introductionmentioning

confidence: 99%

Current Understanding on the Genetic Basis of Key Metabolic Disorders: A Review

Rodrigues

Yong

Bhuiyan

et al. 2022

Biology

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Advances in data acquisition via high resolution genomic, transcriptomic, proteomic and metabolomic platforms have driven the discovery of the underlying factors associated with metabolic disorders (MD) and led to interventions that target the underlying genetic causes as well as lifestyle changes and dietary regulation. The review focuses on fourteen of the most widely studied inherited MD, which are familial hypercholesterolemia, Gaucher disease, Hunter syndrome, Krabbe disease, Maple syrup urine disease, Metachromatic leukodystrophy, Mitochondrial encephalopathy lactic acidosis stroke-like episodes (MELAS), Niemann-Pick disease, Phenylketonuria (PKU), Porphyria, Tay-Sachs disease, Wilson’s disease, Familial hypertriglyceridemia (F-HTG) and Galactosemia based on genome wide association studies, epigenetic factors, transcript regulation, post-translational genetic modifications and biomarker discovery through metabolomic studies. We will delve into the current approaches being undertaken to analyze metadata using bioinformatic approaches and the emerging interventions using genome editing platforms as applied to animal models.

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“…Despite the rapid increase in the number of new orphan drugs over the last 10 years [11], many important therapy areas remain underserved. Indeed, some of these designations are for drugs that are predominantly used to treat non-orphan conditions [12]. Furthermore, a signi cant proportion of new orphan indications granted between 2015 and 2019 were for rare cancer treatments [13].…”

Section: Introductionmentioning

confidence: 99%

Evaluation of the Societal Burden of Rare Diseases in the United States

Cioffi

Andreu

Karam

et al. 2021

Preprint

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BackgroundMost patients with rare diseases have no effective treatment or cure available to them. However, scarcity of data and disease complexity mean the full extent of the patient, family and social burden of rare diseases remains undocumented. Understanding the cost drivers and the economic impact that a lack of treatment poses is critical for highlighting the unmet need to inform future investments and policymaking.MethodsWe selected five priority therapeutic areas (TAs; metabolic, neurological, congenital, hematological and immunological) encompassing 227 well documented rare diseases. Discussions with patients and physicians identified the 24 most relevant (highest unmet need) diseases within the five TAs. We assessed direct (costs associated with medical care), indirect (costs related to productivity losses) and mortality (costs associated with loss of life) costs. We also assessed these costs for 24 chronic mass-market (MM) diseases. Based on the overall cost burden per patient per year (PPPY) for the rare diseases, a scenario analysis was conducted to assess the average cost if treatments were not available. Lastly, the findings from the initial 24 rare diseases were extrapolated to 227 rare diseases in the five TAs. ResultsThe average cost burden of the 24 diseases analyzed ranged from $121 000 to $334 000 PPPY compared with $26 000 PPPY for MM diseases. Averaging across selected rare diseases, lack of treatment was associated with a 21.2% increase in total costs, from $198 000 to $240 000 PPPY. When these results were extrapolated to 227 rare diseases belonging to the five TAs, similar results were obtained: the average cost of rare diseases was approximately 10 times higher ($266 000 PPPY vs $26 000 PPPY) than for the MM diseases. ConclusionsOur findings demonstrate that rare diseases impose substantial economic burden, which remains high even when treatments are available. However, the cost composition shifts towards medical care and away from other types of burden. To the extent that new treatments provide clinical benefit for patients and their families, these shifts in burden are likely productive. Accelerated progress in the development of diagnostic methods, treatments and updated regulatory frameworks for rare diseases are recommended.

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Spending For Orphan Indications Among Top-Selling Orphan Drugs Approved To Treat Common Diseases

Cited by 20 publications

References 11 publications

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

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

Current Understanding on the Genetic Basis of Key Metabolic Disorders: A Review

Evaluation of the Societal Burden of Rare Diseases in the United States

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