Considerations and Regulatory Challenges for Innovative Medicines in Expedited Approval Programs: Breakthrough Therapy and Sakigake Designation

Kajiwara, Eiji; Shikano, Mayumi

doi:10.1007/s43441-019-00019-z

Cited by 4 publications

(2 citation statements)

References 5 publications

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“…Among the drugs priced using the latter, premium rewards are added to some, such as for innovation and/or usefulness. These include orphan disease drugs and drugs for pediatric use and/or sakigake (i.e., accelerated approval of drugs designated as breakthrough therapies and that address unmet medical needs) [ 19 , 20 ]. Furthermore, revisions may include price adjustment for consistency with overseas prices, inter-specification adjustments, and different dosage forms [ 21 ].…”

Section: Discussionmentioning

confidence: 99%

Relationships between developmental strategies for additional indications and price revisions for anticancer drugs in Japan

Maeda

Okabe

Sakakura

et al. 2021

BMC Health Serv Res

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Background The relationships between developmental strategies for additional indications and drug price revisions have not been thoroughly studied. Here, we investigated the price revisions for anticancer drugs approved in Japan. Methods The study was based on published information on anticancer drugs approved between January 2009 and March 2020 in Japan. We investigated the relationships between the pharmacological and regulatory characteristics of anticancer drugs and occurrence/non-occurrence of the Japanese National Health Insurance (NHI) price revisions. Results Eighty-one new anticancer drugs were given NHI price listings during the survey. On April 1, 2020, the prices of 23 anticancer drugs had been revised from the initial pricing, the prices were reduced for 21 drugs (91.3%). Several parameters showed the relationships between drug characteristics and NHI price revisions. The achievement of additional indications and compound type were identified as explanatory factors for these relationships. Additional indication profiles were defined to assess the relationships between the methods for additional indication achievement and price revisions. When the type of additional indication was “Expansion”, the percentage of drugs received NHI price revisions was the highest (P<0.001). Conclusions NHI price revision was significantly related to the achievement of additional indications and compound type. The strategy for additional indications was found to affect the occurrence/non-occurrence of NHI price revisions.

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Section: Discussionmentioning

confidence: 99%

Relationships between developmental strategies for additional indications and price revisions for anticancer drugs in Japan

Maeda

Okabe

Sakakura

et al. 2021

BMC Health Serv Res

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“…19 This new regulatory scheme is intended to encourage the conduct of R&D on new therapeutic products in Japan efficiently, especially in areas on which global pharmaceutical companies have been focusing. [20][21][22][23][24] When regulatory approval is obtained in Japan ahead of other countries, a premium based on the SAKIGAKE review designation is also applied. 19 This is expected to encourage increased R&D by Japanese pharmaceutical companies, especially in therapeutic areas where contributions have been made mainly by global pharmaceutical companies, resulting in the Japanese pharmaceutical market becoming more comparable to the global market.…”

Section: Discussionmentioning

confidence: 99%

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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The Role of Regulatory Agencies in Alzheimer’s Disease Drug Development

Sampaio¹,

Sathe²

2022

Alzheimer's Disease Drug Development

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Considerations and Regulatory Challenges for Innovative Medicines in Expedited Approval Programs: Breakthrough Therapy and Sakigake Designation

Cited by 4 publications

References 5 publications

Relationships between developmental strategies for additional indications and price revisions for anticancer drugs in Japan

Relationships between developmental strategies for additional indications and price revisions for anticancer drugs in Japan

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

The Role of Regulatory Agencies in Alzheimer’s Disease Drug Development

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