Evaluating The Impact Of The Orphan Drug Act’s Seven-Year Market Exclusivity Period

2018 ◽  
Vol 37 (5) ◽  
pp. 732-737 ◽  
Author(s):  
Ameet Sarpatwari ◽  
Reed F. Beall ◽  
Abdurrahman Abdurrob ◽  
Mengdong He ◽  
Aaron S. Kesselheim
Keyword(s):  
Orphan Drug ◽  
Market Exclusivity ◽  
The Impact

Prescribing originality: investigating the impact of original knowledge on patent quality in the pharmaceutical sector

2021 ◽  
Vol 10 (1) ◽  
pp. 78-97
Author(s):  
Kristie Briggs
Keyword(s):  
Market Value ◽  
Pharmaceutical Sector ◽  
Market Exclusivity ◽  
Optimal Length ◽  
Content Type ◽  
Patent Quality ◽  
Extant Literature ◽  
New Drug ◽  
The Impact ◽  
The Relationship

PurposeThis paper examines the relationship between the originality of a pharmaceutical innovation and its patent quality. Greater patent quality has been shown in the extant literature to enhance market value, which better enables firms to recoup research and development (R&D) expenditures incurred during the innovation process. Understanding how originality improves patent quality can assist policymakers, when determining the optimal length of pharmaceutical patent protection and/or market exclusivity.Design/methodology/approachThe relationship between originality and patent quality is empirically investigated using a tobit, as well as a zero-inflated negative binomial, estimation approach to account for prevalence of patents receiving zero forward citations. Moderating effects of joint innovation, innovation by a university researcher and innovation by an established innovator on originality are also considered.FindingsThere is a robust and positive relationship between patent originality and quality in the pharmaceutical sector. This relationship is positively moderated by joint patent ownership with a university. As such, innovators that target originality in new drug development (especially those collaborating with universities) should, according to extant literature, see greater increases in their market value.Originality/valuePolicymakers can use information on the originality of a new drug to discern the optimal length of market exclusivity needed to enable the innovator to recoup expenditures related to R&D. Better predictions of the timing for which firms can recoup R&D expenditures will equip policymakers with knowledge about the appropriate timing to introduce competition into the market, which is critical to reducing the price of pharmaceuticals to consumers.

scholarly journals Excluding Orphan Drugs from the 340B Drug Discount Program: the Impact on 18 Critical Access Hospitals

2012 ◽  
Vol 3 (1) ◽  
Author(s):  
Madeline Carpinelli Wallack ◽  
Todd Sorensen
Keyword(s):  
Federal Government ◽  
Orphan Drug ◽  
Orphan Drugs ◽  
Safety Net ◽  
Original Research ◽  
Critical Access Hospitals ◽  
Patient Protection ◽  
High Prevalence ◽  
Safety Net Providers ◽  
The Impact

Purpose: The 340B Drug Pricing Program is a federal program designed to reduce the amount that safety net providers spend on outpatient drugs. The Patient Protection and Affordable Health Care Act of 2010 extended eligibility for 340B to critical access hospitals (CAHs) for all drugs except those designated as "orphan." Because this policy is unprecedented, this study quantifies the gross financial impact that this exemption has on a group of CAHs. Methods: Drug spending for 2010 from 18 CAHs in Minnesota and Wisconsin are reviewed to identify the prevalence of orphan drug purchases and to calculate the price differentials between the 340B price and the hospitals' current cost. Results: The 18 CAHs' purchases of orphan drugs comprise an average of 44% of the total annual drug budgets, but only 5% of units purchased, thus representing a very high proportion of their expenditures. In the aggregate, the 18 hospitals would have saved $3.1 million ($171,000 average per hospital) had purchases of drugs with orphan designations been made at the 340B price. Because CAH claims for Medicare are reimbursed on a cost-basis, the Federal government is losing an opportunity for savings. Conclusion: The high prevalence of orphan drug use and considerable potential for cost reduction through the 340B program demonstrate the loss of benefit to the hospitals, Federal government and the states.   Type: Original Research

scholarly journals PMU49 NEGOTIATION OUTCOMES IN ITALY: WHAT’S THE IMPACT OF ORPHAN DRUG DESIGNATION?

2019 ◽  
Vol 22 ◽  
pp. S716-S717
Author(s):  
A. Enrici ◽  
D. Lidonnici ◽  
E. Lanati
Keyword(s):  
Orphan Drug ◽  
Orphan Drug Designation ◽  
The Impact

Transporters involved in renal excretion of N-carbamoylglutamate, an orphan drug to treat inborn n-acetylglutamate synthase deficiency

2014 ◽  
Vol 307 (12) ◽  
pp. F1373-F1379 ◽  
Author(s):  
Elisabeth Schwob ◽  
Yohannes Hagos ◽  
Gerhard Burckhardt ◽  
Birgitta C. Burckhardt
Keyword(s):  
Orphan Drug ◽  
Organic Anion ◽  
Xenopus Laevis Oocytes ◽  
Human Embryonic Kidney ◽  
Estrone Sulfate ◽  
Anion Transporters ◽  
293 Cells ◽  
Acetylglutamate Synthase ◽  
Inborn Defects ◽  
The Impact

Inborn defects in N-acetylglutamate (NAG) synthase (NAGS) cause a reduction of NAG, an essential cofactor for the initiation of the urea cycle. As a consequence, blood ammonium concentrations are elevated, leading to severe neurological disorders. The orphan drug N-carbamoylglutamate (NCG; Carbaglu), efficiently overcomes NAGS deficiency. However, not much is known about the transporters involved in the uptake, distribution, and elimination of the divalent organic anion NCG. Organic anion-transporting polypeptides (OATPs) as well as organic anion transporters (OATs) working in cooperation with sodium dicarboxylate cotransporter 3 (NaDC3) accept a wide variety of structurally unrelated drugs. To test for possible interactions with OATPs and OATs, the impact of NCG on these transporters in stably transfected human embryonic kidney-293 cells was measured. The two-electrode voltage-clamp technique was used to monitor NCG-mediated currents in Xenopus laevis oocytes that expressed NaDC3. Neither OATPs nor OAT2 and OAT3 interacted with NCG, but OAT1 transported NCG. In addition, NCG was identified as a high-affinity substrate of NaDC3. Preincubation of OAT4-transfected human embryonic kidney-293 cells with NCG showed an increased uptake of estrone sulfate, the reference substrate of OAT4, indicating efflux of NCG by OAT4. In summary, NaDC3 and, to a lesser extent, OAT1 are likely to be responsible for the uptake of NCG from the blood. Efflux of NCG across the luminal membrane into the tubular lumen probably occurs by OAT4 completing renal secretion of this drug.

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