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Benefits versus profits: has the orphan drug act gone too far?
... Negative consequences of the ODA Although many authors consider the ODA and similar pieces of legislation as successful policies for promoting drug R&D, negative consequences of the Act were raised shortly after its implementation. Five main issues arose before the 2000s and are here summarized from Arno et al., 1995;Asbury, 1991Asbury, , 1992Clissold, 1995;Garber, 1994;Haffner, 1991;Shulman & Manocchia, 1997. ...
This article aims to synthesize the existing literature on the implementation of public policies to incentivize the development of treatments for rare diseases, (diseases with very low prevalence and therefore with low commercial interest) otherwise known as orphan drugs. The implementation of these incentives in the United States (US), Japan, and in the European Union (EU) seems to be related to a substantial increase in treatments for these diseases, and has influenced the way the pharmaceutical research & development (R&D) system operates beyond this policy area. Despite the success of the Orphan Drug model, the academic literature also highlights the negative implications that these public policies have on affordability and access to orphan drugs, as well as on the prioritization of certain disease rare areas over others.
The synthesis focuses mostly on the United States’ Orphan Drug Act (ODA) as a model for subsequent policies in other regions and countries. It starts with a historical overview of the creation of the term “rare diseases”, continues with a summary of the evidence available on the US ODA’s positive and negative impacts, and provides a summary of the different proposals to reform these incentives in light of the negative outcomes described. Finally, it describes some key aspects of the Japanese and European policies, as well as some of the challenges captured in the literature related to their impact in Low- and Middle-Income Countries (LMICs).
... Negative consequences of the ODA Although many authors consider the ODA and similar pieces of legislation as successful policies for promoting drug R&D, negative consequences of the Act were raised shortly after its implementation. Five main issues arose before the 2000s and are here summarized from Arno et al., 1995;Asbury, 1991Asbury, , 1992Clissold, 1995;Garber, 1994;Haffner, 1991;Shulman & Manocchia, 1997. First, the market exclusivity clause has been portrayed as the major contributor to the success of the ODA, but also as a major driver of unaffordable prices, as the monopolies allow companies to set high prices without competition. ...
This article aims to synthesize the existing literature on the implementation of public policies to incentivize the development of treatments for rare diseases, (diseases with very low prevalence and therefore with low commercial interest) otherwise known as orphan drugs. The implementation of these incentives in the United States (US), Japan, and in the European Union (EU) seems to be related to a substantial increase in treatments for these diseases, and has influenced the way the pharmaceutical research & development (R&D) system operates beyond this policy area. Despite the success of the Orphan Drug model, the academic literature also highlights the negative implications that these public policies have on affordability and access to orphan drugs, as well as on the prioritization of certain disease rare areas over others.
The synthesis focuses mostly on the United States’ Orphan Drug Act (ODA) as a model for subsequent policies in other regions and countries. It starts with a historical overview of the creation of the term “rare diseases”, continues with a summary of the evidence available on the US ODA’s positive and negative impacts, and provides a summary of the different proposals to reform these incentives in light of the negative outcomes described. Finally, it describes some key aspects of the Japanese and European policies, as well as some of the challenges captured in the literature related to their impact in Low- and Middle-Income Countries (LMICs).
... Although many authors consider the ODA and similar pieces of legislation as successful policies for promoting drug R&D, negative consequences of the Act were raised shortly after its implementation. Five main issues arose before the 2000s and are here summarized from Arno et al., 1995;Asbury, 1991Asbury, , 1992Clissold, 1995;Garber, 1994;Haffner, 1991;Shulman & Manocchia, 1997. First, the market exclusivity clause has been portrayed as the major contributor to the success of the ODA, but also as a major driver of unaffordable prices, as the monopolies allow companies to set high prices without competition. ...
This article aims to synthesize the existing literature on the implementation of public policies to incentivize the development of treatments for rare diseases (which are diseases with very low prevalence and therefore with low commercial interest) otherwise known as orphan drugs. The implementation of these incentives in the 1980s in the United States (US), later in Japan, and in the European Union (EU) seems to be related to a substantial increase in treatments for these diseases, and has influenced the way the pharmaceutical research & development (R&D) system operates beyond this area. In spite of this success, the academic literature also highlights the negative implications that these public policies have on affordability and access to orphan drugs, as well as on the prioritization of certain rare disease areas over others.
The synthesis focuses mostly on the United States’ Orphan Drug Act (ODA) as a model for subsequent policies in other regions and countries. It starts with a historical overview of the creation of the term “rare diseases”, continues with a summary of the evidence available on the US ODA’s positive and negative impacts, and provides a summary of the different proposals to reform these incentives in light of the negative outcomes described. Finally, it describes some key aspects of the Japanese and European policies, as well as some of the challenges captured in the literature related to their impact in Low- and Middle-Income Countries (LMICs).
... They argue that the lack of price controls coupled with vague definitions of orphan diseases encourage an inflationary market. 40 While financial aid programs are available to help rare disease sufferers purchase treatments that are otherwise unobtainable due to cost, it is not uncommon to hear stories of patients having to sell their homes and spend their life-savings simply to qualify for government or corporate assistance programs. 41 This lack of price control defeats the purpose of the legislation which is to ensure that those patients that need life-saving or life-enhancing products have access to them. ...
The United States Orphan Drug Act passed in 1983 provides four provisions to spur the development of medications for conditions that might otherwise have been abandoned. The Office of Orphan Drug Product Development was established to provide assistance in protocol development, and to administer a grants program. A tax credit incentive also was enacted but the most significant incentive has been the market exclusivity clause. Despite such measures, the act in the wake of technological and economic developments should continually be reexamined to enhance efficiency in development of orphan products, to ensure accessibility of these products to patients, and to minimize economic abuses by developers.
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