Hurdles in pediatric oncology drug development and their countermeasures: From the perspective of pharmaceutical companies
2023 Volume 60 Issue 5 Pages 341-345
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2023 Volume 60 Issue 5 Pages 341-345
In recent years, several measures have been implemented to promote the development of pediatric drugs, generating increasing momentum and expectations. However, pharmaceutical companies still face numerous challenges; continuing collaborations with government agencies, academia, and patient groups is deemed necessary to develop solutions. Current data reveal that over 70% of pediatric drugs approved overseas remain unapproved and approximately half of the domestically unapproved drugs are yet to begin development in Japan. In the initial stages of new drug development (from the creation of new drug seeds to the establishment of Proof-of-Concept in early clinical development), emerging companies in the West have led many cases; the concept of national borders in new drug development is gradually disappearing, with domestically completed cases becoming rare.
In the field of pediatric cancer drug development, this trend is more pronounced. To consider measures to promote pediatric cancer drug development, implementing policies that encourage even emerging overseas companies to show enthusiasm for development in Japan is necessary, without being bound by the concept of “Japanese companies/Japan-originated new drug development.”
In particular, a transparent development scheme for pediatric drugs, a regulatory design that enables rapid marketing, and a guarantee of returns commensurate with investment and business continuity are necessary. For this, the issuance of guidance, regulatory systems not bound by existing mechanisms, and financial assistance (reduction of development costs, drug price allowances, tax incentives, etc.) are anticipated.
In this paper, we share several ideas discussed by the members of the National Council on Pediatric Cancer Measures, which includes Japanese and overseas pharmaceutical companies. We aim to contribute to enhancing access to pediatric cancer drugs in Japan and encourage all stakeholders to continue to cooperate and take action.
