Profitable and Nonprofitable Drugs

Woert, Van; Melvin, Holly

doi:10.1056/nejm197804202981608

Cited by 19 publications

(4 citation statements)

References 5 publications

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“…Concerns about the lack of economic viability of pharmaceutical R&D in certain areas started emerging in the late 1960s in the US. According to several authors, the implementation of the Kefauver-Harris Drug Amendments in 1962 – which increased regulatory standards for drug safety and efficacy – led to an increase in development and regulatory costs for the industry ( Arno et al ., 1995 ; Asbury, 1981 , 1992 ; Cheung et al ., 2004 ; Haffner et al ., 2002 ; Huyard, 2009 ; Loughnot, 2005 ; Mikami, 2017 ; Van Woert, 1978 ).…”

Section: Resultsmentioning

confidence: 99%

“…There were two main consequences related to this shift: first, the creation of a group of treatments known to be effective but not compliant with the new regulation, named “homeless” or “orphan” treatments, as their development was not commercially attractive for the industry ( Cheung et al ., 2004 ; Huyard, 2009 ; Loughnot, 2005 ; Mikami, 2017 ; Provost, 1968 ; Van Woert, 1978 ). Second, the abandonment of the development of “service drugs” (treatments with public health relevance, but with small profitability prospects) as a regularized practice in the industry.…”

Section: Resultsmentioning

confidence: 99%

“…Second, the abandonment of the development of “service drugs” (treatments with public health relevance, but with small profitability prospects) as a regularized practice in the industry. This affected drugs treating diseases with low prevalence ( Asbury, 1981 , 1991 , 1992 ; Huyard, 2009 ; Mikami, 2017 ; Provost, 1968 ; Van Woert, 1978 ), but also those treating larger target populations ( Arno et al ., 1995 ; Asbury, 1991 ; Haffner, 1991 ; Huyard, 2009 ), such as vaccines and “drugs for developing nations” ( Asbury, 1981 ; Huyard, 2009 ).…”

Section: Resultsmentioning

confidence: 99%

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Pharmaceutical policy and innovation for rare diseases: A narrative review

Alonso Ruiz,

Large,

Moon

et al. 2023

F1000Res

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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).

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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

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Pharmaceutical policy and innovation for rare diseases: A narrative review

Alonso Ruiz,

Large,

Moon

et al. 2023

F1000Res

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“…A new cardiovascular drug must be effective, a new drug must be suitable for short-term clinical trial, a new drug must be well tolerated and safe for chronic treatment, a new drug must be convenient to use, and a new drug should even be inexpensive. VanWoert [1978] summarized the factors involved in new drug discovery as including the basic scientific discoveries that justify the preliminary synthesis and testing of a new compound, the need for a drug in a particular disease, the scientific aptitude of a company's research staff, and the anticipated market for the drug.…”

mentioning

confidence: 99%

Meeting the challenge of cardiovascular new drug discovery

Smith

1984

Drug Development Research

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The Orphan Drug Act

Asbury¹

1991

JAMA

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The 1983 Orphan Drug Act sought to increase market incentives and decrease regulatory barriers for products used to treat rare ("orphan") diseases. Major provisions included market exclusivity, tax credits, and regulatory process clarifications. This analysis compares pre- and post-Act industry and government data to examine changes associated with the law. While industry sponsored 34 marketed and 24 experimental orphan drugs in the 17 years prior to the Act, it has sponsored 39 of 42 marketed orphan products in the 7 years since the Act. An additional 301 experimental products have orphan designation. While 25 of 40 marketed orphan products reportedly had annual sales of less than $1 million, product sales for three conditions are more than $100 million annually. This prompted changes in the law, passed by Congress in 1990, but vetoed. Overall, the law has been associated with an increase in orphan product development. The law's costs and benefits to companies, patients, and the public should be examined if future changes are proposed.

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Profitable and Nonprofitable Drugs

Cited by 19 publications

References 5 publications

Pharmaceutical policy and innovation for rare diseases: A narrative review

Pharmaceutical policy and innovation for rare diseases: A narrative review

Meeting the challenge of cardiovascular new drug discovery

The Orphan Drug Act

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