Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

Brabers, A.; Moors, Ellen H.M.; Weely, Sonja van; Vrueh, Remco L. A. de

doi:10.1186/1750-1172-6-59

Cited by 10 publications

(6 citation statements)

References 28 publications

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“…Marketing authorization for the first orphan drug may indicate feasible development for future drugs for the same rare disease. Arguments rejecting claims of market monopolies commonly attribute the occurrence of a single orphan drug for a single rare disease on the small market size, with an inability to attract competition [ 18 ].…”

Section: Resultsmentioning

confidence: 99%

“…However, the efficacy of marketing exclusivity in promoting patient access orphan medicines is not apparent [ 3 , 42 ]. The formation of ‘mini-monopolies’ is a major concern [ 3 , 4 , 18 , 27 , 29 , 31 , 36 , 44 ].…”

Section: Discussionmentioning

confidence: 99%

“…Seven of 35 countries (Canada, Germany, Sweden, US, Switzerland, Denmark and Greece) have dedicated co-payments protection programs that provide financial support once an annual amount of co-payment is exceeded to protect against the risk of excessive out-of-pocket expenditure. Interestingly, countries including, but not limited to, Australia, Italy and the Netherlands have special programmes reimbursing orphan drugs (namely, Australia’s Life-saving drug program, Italy’s 5% Agenzia italiana del Farmaco (AIFA) fund for reimbursing orphan drugs not yet marketed, and the Dutch Policy Rule for Expensive Hospital and Orphan Drugs that supports hospitals financially for prescribing orphan drugs).that are separate to their national drug coverage programs for non-orphan drugs [ 3 , 4 , 10 , 15 , 18 , 37 ].…”

Section: Discussionmentioning

confidence: 99%

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Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

2015

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ObjectiveTo review existing regulations and policies utilised by countries to enable patient access to orphan drugs.MethodsA review of the literature (1998 to 2014) was performed to identify relevant, peer-reviewed articles. Using content analysis, we synthesised regulations and policies for access to orphan drugs by type and by country.ResultsFifty seven articles and 35 countries were included in this review. Six broad categories of regulation and policy instruments were identified: national orphan drug policies, orphan drug designation, marketing authorization, incentives, marketing exclusivity, and pricing and reimbursement. The availability of orphan drugs depends on individual country’s legislation and regulations including national orphan drug policies, orphan drug designation, marketing authorization, marketing exclusivity and incentives such as tax credits to ensure research, development and marketing. The majority of countries (27/35) had in place orphan drug legislation. Access to orphan drugs depends on individual country’s pricing and reimbursement policies, which varied widely between countries. High prices and insufficient evidence often limit orphan drugs from meeting the traditional health technology assessment criteria, especially cost-effectiveness, which may influence access.ConclusionsOverall many countries have implemented a combination of legislations, regulations and policies for orphan drugs in the last two decades. While these may enable the availability and access to orphan drugs, there are critical differences between countries in terms of range and types of legislations, regulations and policies implemented. Importantly, China and India, two of the largest countries by population size, both lack national legislation for orphan medicines and rare diseases, which could have substantial negative impacts on their patient populations with rare diseases.

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Section: Resultsmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

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Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

2015

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“…An in-depth evaluation of the impact of the American Orphan Drug Act from 1985 to 2017 showed that there is little relation between market incentives and drug approvals [ 67 ]. Factors such as market size, type of disease, clinical evidence, and the age of onset of symptoms are predictors for the development of other orphan drugs for the same rare indication [ 68 ]. In addition, generic manufacturers may believe that orphan drugs fail to provide a large enough return on investment, resulting in a lack of competition and making the additional period of exclusivity for the orphan drug nonessential, even when it exceeds the duration of the patent [ 67 ].…”

Section: Discussionmentioning

confidence: 99%

Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab

et al. 2021

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Background This study examines the dynamics of the eculizumab patenting, orphan designation, and marketing authorization process in different countries and regulatory systems and analyzes drug revenues since its first marketing authorization. Methods A retrospective case study was conducted. Multiple information sources were used to: determine the status of eculizumab patents; examine the designation of orphan drug status by US, European, Japanese, and Brazilian regulatory authorities to determine registration status and approved clinical indications; estimate the prevalence of associated clinical conditions; investigate the history of the drug manufacturer, Alexion Pharmaceuticals, Inc., and its financialized business model; and examine global eculizumab sales revenues since its first marketing authorization. Results Our search yielded 32 patent families divided into 98 applications. The first patent granted was filed in 1995 by Alexion Pharmaceuticals, Inc. in the US. Eculizumab has always been as an orphan drug, except in the Brazilian regulatory agency. All clinical indications approved thus far refer to rare diseases (e.g., paroxysmal nocturnal hemoglobinuria syndrome, atypical hemolytic-uremic syndrome, refractory and generalized myasthenia gravis, and neuromyelitis optica spectrum disorder). Alexion’s revenues amounted to more than US$25 billion between 2007 and 2019, showing a growing trend. Eculizumab led sales from the beginning, being the only product in the company’s portfolio until 2015. In 2019, the drug accounted for 79.1% of all revenues. Discussion Our findings show that a strategy focused on obtaining orphan drug designation, expanding therapeutic indications and the geographic range of marketing approvals, extending monopoly periods, and prioritizing public procurement niches has enhanced revenues and helped the company achieve leadership in a highly specific and profitable market.

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“…In addition, there is circumstantial evidence which suggests that availability of a (potential) therapeutic modality for a specific rare disease appears to encourage rather than discourage additional orphan drug development for the same rare disorder [ 22 ]. As such crossing the translational chasm between research and development not only represents the development of a potential life-saving or quality of life-improving therapy for rare disease patients, but may also have an important spin-off effect.…”

Section: Discussionmentioning

confidence: 99%

Drug development for exceptionally rare metabolic diseases: challenging but not impossible

Putzeist

Mantel‐Teeuwisse

Wied

et al. 2013

Orphanet J Rare Dis

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BackgroundWe studied to what extent the level of scientific knowledge on exceptionally rare metabolic inherited diseases and their potential orphan medicinal products is associated with sponsors deciding to apply for an orphan designation at the US Food and Drug Administration (FDA) or the European Medicines Agency (EMA).MethodsAll metabolic diseases with a genetic cause and prevalence of less than 10 patients per 1 million of the population were selected from the ‘Orphanet database of Rare diseases’. The outcome of interest was the application for an orphan designation at FDA or EMA. The level of publicly available knowledge of the disease and drug candidate before an orphan designation application was defined as whether the physiological function corresponding with the pathologic gene and initiation of the pathophysiological pathway was known, whether an appropriate animal study was identified for the disease, whether preclinical proof of concept was ascertained and the availability of data in humans. Other determinants included in the study were metabolic disease class, the prevalence of the disease, prognosis and time of first description of the disease in the literature. Univariate relative risks (RRs) and 95% confidence intervals (CIs) of an orphan designation application were calculated for each of these determinants. In addition, a multivariate Cox regression analysis was conducted (Forward LR).ResultsIn total, 166 rare metabolic genetic diseases were identified and included in the analysis. For only 42 (25%) of the diseases an orphan designation application was submitted at either FDA or EMA before January 2012. The multivariate analysis identified preclinical proof of concept of a potential medicinal product as major knowledge related determinant associated with an orphan designation application (RRadj 3.9, 95% CI 1.9-8.3) and confirmed that prevalence of the disease is also associated with filing an application for an orphan designation (RRadj 2.8, 95% CI 1.4-5.4).ConclusionFor only one out of four known exceptionally rare metabolic inherited diseases sponsors applied for an orphan designation at FDA or EMA. These applications were found to be associated with the prevalence of the rare disease and the level of available scientific knowledge on the proof of concept linking possible drug candidates to the disease of interest.

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Does market exclusivity hinder the development of Follow-on Orphan Medicinal Products in Europe?

Cited by 10 publications

References 28 publications

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

Access to Orphan Drugs: A Comprehensive Review of Legislations, Regulations and Policies in 35 Countries

Dynamics of patents, orphan drug designation, licensing, and revenues from drugs for rare diseases: The market expansion of eculizumab

Drug development for exceptionally rare metabolic diseases: challenging but not impossible

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