Global Platform for Access to Childhood Cancer Medicines

It is estimated that approximately 400,000 children develop cancer each year, approximately only half of whom are diagnosed. Close to 90% of them live in low- and middle-income countries (LMICs), where survival rates are less than 30%.

The Global Platform for Access to Childhood Cancer Medicines is a joint effort by St. Jude and WHO to end disparities in access to safe and effective medicines to treat children with cancer. Working with many collaborators worldwide, this platform will offer a comprehensive solution that forecasts the needs of individual countries and provides the mechanism through which quality and safe medicines are procured and distributed. The platform will provide an uninterrupted supply of quality-assured cancer medicines to treat children in LMICs. The development and implementation of this platform will follow a phased approach.

The Global Platform for Access to Childhood Cancer Medicines brings St. Jude, WHO, governments, the pharmaceutical industry, non-governmental organizations, and nonprofit sector decisionmakers together in a truly collaborative spirit to co-design a platform that will be sustainable. It is unique in its size and scope, catalyzed by a $200 million commitment from St. Jude that will restructure the market to better respond to the needs of children with cancer and their providers. We believe this platform not only will transform access to childhood cancer medicines, but also will serve as a model for the broader global health community in how we might work together to address global health challenges in access to care for non-communicable diseases.

Many hospitals treating children with cancer across the world struggle to consistently access quality-assured, effective, and affordable medicines for their patients. Although essential medicine availability is a global challenge, an analysis of the market has shown hospitals in LMICs face the most severe consequences pertaining to cancer medicines. Children with cancer may lack access to essential curative treatments or have prolonged interruptions in therapy thus increasing their risk of dying.

St. Jude provided a six-year, $200 million investment to support the development and launch of the platform. This is the largest financial commitment for a global childhood cancer medicines project to date. St. Jude is committed to the success of this important endeavor. Moving forward, stakeholders may choose to contribute additional funds to ensure its success and/or collaborate to bring on additional partners and funding sources. In addition, St. Jude will provide technical support, including expertise on global pediatric oncology.

During the pilot phase in year one, we anticipate that approximately 5,000 children will benefit from an increased access to childhood cancer medicines through the platform that offers end-to-end support. The goal is to double this number each subsequent year to reach approximately 50,000 children per year by year six. In total, we expect that approximately 120,000 children will have benefitted from the platform’s operations by the end of 2027.

By the end of the first six years, we estimate that approximately 25% of all children with cancer in the world will benefit from this platform, including close to 70% of those in LMICs.

St. Jude approved the development of the platform in October 2021. St. Jude and WHO are currently defining/refining the processes that will enable the platform to achieve the expected impact of 25% of all children with cancer benefiting worldwide. This includes establishing an Administrative Operational Unit that will manage the platform, engaging a procurement agency, creating a governance structure, and establishing a country selection process. Over the coming months, more information about the operational model will be confirmed and shared with the public.

During development and launch, the platform will be focused on procuring and distributing medicines that have been categorized by WHO as “Essential Medicines” and are cancer medicines (or anti-neoplastics). WHO defines Essential Medicines as those that satisfy the priority health care needs of a population. They are selected with due regard to disease prevalence and public health relevance, evidence of efficacy and safety and comparative cost-effectiveness. WHO updates a list of Essential Medicines every two years. The first list of Essential Medicines for children was published in 2007, and the most recent list published in 2021.

The need for safe and effective medicines is global. In the Development Phase, the focus will be on LMICs and determined by strict criteria for participation.

The implementation of the Global Platform will follow three phases:

1. Development Phase (from October 2021 to June 2022), which will focus on the establishment of the Administrative Operations Unit (AOU), the governance structure, and contracting with a procurement agency. 
2. Pilot Phase (from July 2022 to June 2024), which will include the procurement and distribution of childhood cancer medicines for approximately 12 countries representing the six WHO regions and covering the treatment of approximately 12,000 children per year. 
3. Growth Phase (from July 2024 to June 2027), wherein, by the end of 2027, the Global Platform will provide medications to up to 50,000 children per year and will have provided medications for an estimated 120,000 children in total.

An experienced procurement agent with whom St. Jude and WHO will engage will develop agreements with manufacturers that meet a quality or regulatory threshold. The platform also will have mechanisms to monitor for sub-standard or falsified medicines.

• St. Jude and WHO will co-chair the steering committee as founding and permanent members, with the steering committee serving to set strategic directions, structure, scope and roadmap of the Platform while overseeing activities of the Administrative Operational Unit and define eligibility of participating countries.
• St. Jude and WHO will establish and staff an Administrative Operational Unit that manages the Platform.
• St. Jude and WHO will create governance structure and technical committees of the Platform.
• St. Jude and WHO will choose a procurement agency that will identify best value for money and act in the best interest of the partners and of recipient entities in countries.
• St. Jude will provide technical support, including expertise on global pediatric oncology, input on eligible countries and medicines to be made available, and organize stakeholder meetings.
• Based on agreements between partners, WHO will manage relationships with global partners and provide support to the platform’s governance bodies; facilitate dialogue with governments by designing the plan for participating, orienting stakeholders and implementation; host the administrative operational unit; and enable the link between it and the WHO Global Initiative for Childhood Cancer that ensures medicines are delivered as part of a broader strategy to build capacity in childhood cancer care.
• St. Jude has committed to fund the Administrative Operational Unit through FY27 and will have the option to continue funding for six-year increments moving forward.

Following the initial development and pilot phases, different levels of engagement will exist in which external stakeholders can get involved. St. Jude and WHO will share these opportunities when available.

• ASH Journal - The Drug Shortage Dilemma
• New York Times - Faced With a Drug Shortfall, Doctors Scramble to Treat Children With Cancer
• STAT News - Drug shortages jeopardize the lives of children with cancer