Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval

Harada, Kenji; Toriyabe, Kazuki; Ono, Shunsuke

doi:10.1002/jcph.1501

Cited by 7 publications

(5 citation statements)

References 21 publications

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“…Regardless, the next decade may see a large increase in rare disease drug approvals, if this complementary relationship between designations and approvals persists. Projecting when these future approvals may occur, and in what disease areas we may see them, is an important area of future study [ 27 ].…”

Section: Discussionmentioning

confidence: 99%

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Miller

Fermaglich

Maynard

2021

Orphanet J Rare Dis

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Background Orphan drug designations are a useful proxy to investigate trends in rare disease drug development. Drug developers must receive a designation before they are eligible for the economic incentives of the Orphan Drug Act in the United States. We created a database of all orphan drugs designated between 1983 and 2019 that included numerous drug characteristics, including therapeutic area. In addition, we constructed a “broad disease” categorization of designations as an alternative to therapeutic area, based on disease etiology and age of onset rather than organ system. By looking at the pattern of orphan drug designations over the past four decades, this analysis studied the impact of the evolving rare disease drug development landscape and considers the future of rare disease therapies over the coming decades. Results Between 1983 and 2019, a total of 5099 drugs and biologics received orphan drug designation. Designations more than doubled between the 1980s and 1990s, almost doubled between the 1990s and 2000s, and almost tripled in number between the 2000s and 2010s. The top three therapeutic areas represented in the orphan drug designations were: oncology (1910, 37%), neurology (674, 13%), and infectious diseases (436, 9%). The broad disease categorization found that the proportion of designations for pediatric-onset diseases has increased in the most recent decade to 27%. Conclusions Analysis of the last four decades of orphan drug designation indicates seismic shifts have occurred in the rare disease drug development space. The number of designations granted more than quadrupled between the 1990s and 2010s. While these substantial increases led to growth in the absolute number of designations within all therapeutic areas (bar one) and broad disease categories, the relative proportions have seen considerable change over time. In the most recent decade, there have been notable increases in the proportion of drugs in oncology, pediatric-onset diseases, and neurologic disorders. The dramatic rise in overall orphan designations over the past four decades suggests we may continue to see an upward trajectory in designations leading to an increased number of approvals for drugs and biologics designed specifically for diagnosing, preventing, and treating rare diseases in the coming decades.

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Section: Discussionmentioning

confidence: 99%

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Miller

Fermaglich

Maynard

2021

Orphanet J Rare Dis

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“…ATC A is one of the therapeutic areas that include fatal and orphan/rare diseases. 17 The trend from mass to niche markets was also confirmed for this disease area. The cost calculation method and the innovativeness and usefulness premiums in the comparative method are considered to be applied to the drugs that are expected to be clinically meaningful.…”

Section: Discussionmentioning

confidence: 63%

Analysis of new molecular entity trends from 2006 to 2015 in Japan

Shibata

Fukumoto

Ozaki

et al. 2020

Journal of Generic Medicines

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Japan is one of the largest pharmaceutical markets in the world, and as such, pharmaceutical companies are intensively conducting research and development of new drugs in Japan. However, information on Japanese pharmaceutical market profiles is limited. In this context, we elucidated the market characteristics and trends of new molecular entities approved between 2006 and 2015 in Japan. Among antineoplastic and immunomodulating agents, the highest number was first approved in the United States, and for nervous system drugs, the highest number was first approved in Europe. The alimentary tract and metabolism and the musculoskeletal system were the therapeutic areas for which the most drugs were approved for the first time in Japan. Most of the musculoskeletal system drugs were developed by Japanese companies, and most of the antineoplastic and immunomodulating agents were developed outside of Japan. Using various performance indexes, such as market forecasts and significance of clinical benefits, we determined that the number of new molecular entities and market size increased and the number of drugs with significant clinical benefits decreased. These results suggest that new molecular entity innovation by Japanese companies has been decreasing and the government’s comparative method for pricing does not work well in Japan.

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“…Consequently, data pertaining to safety and efficacy harvested during the clinical trial phase of orphan drugs constitute a crucial element in the benefit-risk assessment within pharmacovigilance for rare diseases. Presently, a plethora of orphan drugs are under the clinical trial phase, while only a minority have successfully navigated to marketing approval [ 33 ]. The probing of the efficacy and safety of orphan drugs during the clinical trial phase has emerged as a focal research point within this domain.…”

Section: Main Research Hot Spots In the Field Of Pharmacovigilance Fo...mentioning

confidence: 99%

Pharmacovigilance for rare diseases: a bibliometrics and knowledge-map analysis based on web of science

Xu,

Li,

et al. 2023

Orphanet J Rare Dis

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Objectives The aims of this paper is to search and explore publications in the field of pharmacovigilance for rare diseases and to visualize general information, research hotspots, frontiers and future trends in the field using the bibliometric tool CiteSpace to provide evidence-based evidence for scholars. Methods We searched the Web of Science Core Collection (WoSCC) for studies related to pharmacovigilance for rare diseases, spanning January 1, 1997-October 25, 2022. CiteSpace software was utilized to discuss countries/regions, institutions, authors, journals, and keywords. Results After screening, a total of 599 valid publications were included in this study, with a significant upward trend in the number of publications. These studies were from 68 countries/regions with the United States and the United Kingdom making the largest contributions to the field. 4,806 research scholars from 493 institutions conducted studies on pharmacovigilance for rare diseases. Harvard University and University of California were the top two productive institutions in the research field. He Dian of the Affiliated Hospital of Guizhou Medical University and Peter G.M. Mol of the University of Groningen, The Netherlands, were the two most prolific researchers. The Cochrane Database of Systematic Reviews and the New England Journal of Medicine were the journals with the highest number of articles and co-citation frequency respectively. Clinical trial, therapy and adverse event were the top three most cited keywords. Conclusions Based on keywords co-occurrence analysis, four research topics were identified: orphan drug clinical trials, postmarketing ADR surveillance for orphan drugs, rare diseases and orphan drug management, and diagnosis and treatment of rare diseases. Immune-related adverse reactions and benefit-risk assessment of enzyme replacement therapy were at the forefront of research in this field. Treatment outcomes, early diagnosis and natural history studies of rare diseases may become hotspots for future research.

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Survey of Japanese Orphan Drug Program: Factors Related to Successful Marketing Approval

Cited by 7 publications

References 21 publications

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Using four decades of FDA orphan drug designations to describe trends in rare disease drug development: substantial growth seen in development of drugs for rare oncologic, neurologic, and pediatric-onset diseases

Analysis of new molecular entity trends from 2006 to 2015 in Japan

Pharmacovigilance for rare diseases: a bibliometrics and knowledge-map analysis based on web of science

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