Role of Patients and Advocates in Cancer Therapeutics Development

Abstract

Advocates who are highly motivated, active, and well educated about the disease landscape play a pivotal role in the success of drug development and approval. While qualitative and quantitative evidence of advocate impact in pediatric cancer drug development is sparse, the importance of advocate input is nevertheless championed by all stakeholders and is demonstrated by the growing publication rate on this subject. The field of drug development for children with cancer has many challenges relating to scientific, ethical, logistical, economic, and regulatory issues. Driving progress in this acute disease area requires a multi-stakeholder approach. Putting unmet medical needs of patients first, early in the development process, can lead to focused priorities and better decisions on research design and potentially avoid costly late-stage failures. To drive faster and increased access to innovative therapies, challenges and opportunities are discussed. Distinct efforts of research advocates to impact the research and drug development process and the impact of policy advocates on the legislative and regulatory environment are discussed. Recommendations are made to maximize opportunities for all stakeholders to attain their shared goal: evidence-based medicines reaching as many children and young people with cancer as possible.