Rescuing Drugs That are Discontinued in Adult Oncology Development for the Benefit of Children and Adolescents With...

Better therapies for childhood cancer remain an unmet need to improve the dismal prognosis of certain malignancies and to reduce the burden of toxicity. Rescuing discontinued or shelved drugs for children, adolescents, and young adults is a strategy to identify new uses for approved or investigational medicines outside the scope of their original medical indication. Our proposed multistakeholder consensus focuses on the development of innovative, patent-protected targeted agents, sourced from previously shelved or discontinued programs that have the potential to provide significant benefit to underserved patient populations, with unmet medical needs. There are several challenges to continuing/rescuing drugs for pediatric oncology development, which include the lack of information for decision making, corporate strategy considerations underlying the decision to invest in pediatric development, and the contracting and technology transfer complexities required to enable divestment and subsequent development. The multistakeholder approach for drug development has the advantage of conveying a consensus among academia, patient advocates, and importantly industry itself. We propose three areas of action to facilitate rescuing potentially beneficial drugs for children and adolescents with cancer: (i) initiatives to provide information to companies considering developing these drugs and a standards framework; (ii) incentives both in Europe and in the United States to encourage companies to develop pediatric-only drugs, with the reform of the EU Pharmaceutical Legislation posing an important opportunity; and (iii) communication of the issues to all stakeholders. Ultimately, this will benefit children and adolescents with cancer.