The toll of cancer on children, especially those living in low-resource countries, is staggering. Each year, an estimated 400,000 children and adolescents worldwide develop cancer,1 and despite improved survival rates, the global 5-year net childhood cancer survival rate is only 37.4%,2 making cancer a leading cause of childhood mortality.1 Children in low- and middle-income countries bear the greatest burden of mortality from a variety of cancers, including leukemias; lymphomas; and solid tumors such as brain cancer, neuroblastoma, and Wilms tumor, with fewer than 30% surviving the disease compared with more than 80% in high-income countries. A confluence of factors contributes to the large disparity in survival outcomes in these children, including an inability to receive an accurate diagnosis, lack of access to treatment, treatment abandonment, death from treatment toxicity, and disease relapse.1
Carlos Rodriguez-Galindo, MD
To increase survival rates in children living in low-resource countries, in 2018, St. Jude Children’s Research Hospital partnered with the World Health Organization (WHO) to launch the Global Initiative for Childhood Cancer. This initiative provides leadership and technical assistance to governments, supporting their efforts in building and sustaining high-quality childhood programs, with the goal of achieving at least 60% survival for all children with cancer by 2030.
In December 2021, to remove one of the obstacles preventing pediatric cancer care from reaching children in low- and middle-income countries and improve their cure rates, St. Jude and the WHO launched the Global Platform for Access to Childhood Cancer Medicines (https://global.stjude.org/en-us/medicines.html). The platform will provide an uninterrupted supply of childhood cancer chemotherapeutics on the WHO’s Model List of Essential Medicines for Children at no cost to 12 low- and middle-income countries in the initial 2-year phase of the program; then the platform will scale up to provide drugs to between 40 and 50 countries over the next 5 years, potentially reaching more than 120,000 children. The center of operations for the platform will be housed at the WHO. At the time of publication, the specific countries participating in the initial phase were still being decided.
According to Carlos Rodriguez-Galindo, MD, Executive Vice President and Chair, Department of Global Pediatric Medicine at St. Jude Children’s Research Hospital and Director of St. Jude Global, the 6-year, $200 million investment to establish the Global Platform for Access to Childhood Cancer Medicines is the largest financial commitment to a global effort to procure childhood cancer medicines.
“Access to cancer medications is a major issue no matter the country. In the United States, drug shortages are a recurring problem, and once you start seeing these problems in the United States in childhood cancers, you can imagine how big the problem is in limited-resource countries,” said Dr. Rodriguez-Galindo. “As we started developing the Global Platform for Access to Childhood Cancer Medicines, we realized that unless we address drug access in a global systematic way, we will never be able to make the progress we want in improving survival outcomes in childhood cancers.”
In an extended interview with The ASCO Post, Dr. Rodriguez-Galindo talked about the challenges of launching the Global Platform for Access to Childhood Cancer Medicines and its potential to save the lives of thousands of children with cancer. He also addressed the difficulty of treating children with cancer in conflict countries, including Ukraine.
Providing Care for 120,000 Children With Cancer
What criteria did you use to determine which 12 countries would participate in the initial phase of the Global Platform for Access to Childhood Cancer Medicines?
For this pilot phase of the program, we chose two countries from each of the six World Health Organization regions, including the African Region, Eastern Mediterranean Region, South-East Asia Region, Region of the Americas, Western Pacific Region, and European Region. It will take us a couple of years to optimize the platform, and we hope that by the fifth or sixth year, we will reach out to between 40 and 50 countries to participate in the program.
The process for selecting those additional countries is now being developed with the WHO and other partners, including the International Society of Pediatric Oncology and Childhood Cancer International. The idea is to select countries with low-income economies, defined by the World Bank as those with a gross national income per capita of $1,045 or less; and lower-middle-income economies, defined as those with a gross national income per capita between $1,046 and $4,095.3 The countries also have to have governments that are willing to be part of this initiative and have systems in place to deliver cancer care.
We think that by the time we get into the fourth, fifth, and sixth years of the program, we will be able to provide medicines for between 40,000 and 50,000 children per year, reaching close to 120,000 pediatric patients with cancer over the next 6 years.
Paying for the Program
St. Jude is investing $200 million to develop this platform. How will you pay for the program after its initial phase is in operation?
Right now, we are completely subsidizing the program to make sure this global platform is possible and can be developed. The financial package we are putting together will support the purchase and storage of drugs at no cost to the countries involved, with additional support for tracking, warehousing, and distributing the medicines to cancer care facilities.
As we scale up the platform, bring more countries on board, and the volume of patients increases, we will think about developing a hybrid financial model in which some countries may be responsible for purchasing these medicines at a discount. Some countries on the higher spectrum of gross national income may choose to purchase from this platform because it guarantees drug quality and supply, it’s a one-stop shop, and may only need to be partially subsidized. Other countries may need to be fully subsidized.
Please talk more about how the medicines will be distributed and prioritized. How will you monitor progress to ensure that the medicines are going to children with cancer?
Setting up a good tracking system for proper monitoring is critical to the success of the program. We are creating an entire infrastructure that will follow the process from the time a drug is shipped to a country, to its storage and distribution. Countries that participate in the program will have to agree to have a tracking and auditing system in place and will also be selected based on their capacity to provide pediatric oncology services. We will assist countries with the selection of medicines and develop treatment standards.
A third component of the platform is engaging with a procurement agent to manage the engagement with the manufacturers and provide the logistics for the distribution of the drugs.
Once its development is complete, the platform will be able to forecast the amount of medicines a country needs, and the procurement agency will take care of all the logistics, including shipping and distribution, to make sure the medicines are used in the context of providing good outcomes for children with cancer.
Increasing Survival Outcomes for Children With Cancer
Can you project how many young lives might be saved with this platform?
I would say right now that, globally, about 400,000 children develop cancer each year. Only about half of these children get a diagnosis. Of those children who are able to access care, between 60% and 70%, if not more, do not receive the full course of treatment due to a lack of medications.
We think that if by 2027, the end of this initial phase, we are providing drugs to about 50,000 children per year, which is close to 70% of the children with cancer in low- and lower-middle-income countries, we will have a major impact on increasing survival rates in these children. If in the succeeding years of the program we can increase that rate to between 70,000 and 100,000 children per year, that will account for 50% of all the children diagnosed with cancer around the world, improving outcomes and saving the lives of many children.
Reducing Cancer Care Inequities in Low-Resource Countries
According to a WHO Noncommunicable Disease Country Capacity Survey published in 2020, only 29% of low-income countries report that cancer medicines are generally available to their citizens, compared with 96% of high-income countries.4 Why is there such a high discrepancy in the availability of medicines between low- and high-income countries? How will this new global platform overcome the problems of geopolitical upheaval, strife, and political corruption in these countries?
This is why the problem of accessing cancer medications in low- and middle-income countries is so difficult to resolve—otherwise it would have been resolved a long time ago. The reasons for the lack of drug availability are multifactorial.
First, there is the lack of actual availability of the drugs to these countries due to the instability of the manufacturing process. We are talking about providing essential medicines that are off-patent and mostly in the generics market. That generics market is very fragmented and generates low profits for manufacturers. Because most of these drugs are for children alone, the volume is very small, reducing company profit margins even more. So, there isn’t much incentive to sell drugs to low- and middle-income countries, which opens the market to less reputable generics manufacturers to fill the void.
Second, governments or hospitals do not have the proper capacity for accurate forecasting, and shortages are common. Finally, there is the added complication of the lengthy and complex process for drug registration at a national level, which many small drug companies may not be able to afford when the market is so small. With so many vulnerable points in the supply chain, there is a risk of less expensive substandard medications being prioritized by many governments and hospitals, which can result in worse outcomes.
Next is the added problem of political strife, graft, and corruption within countries. Taken together, these problems create a very unstable market, which makes drugs either unavailable or unaffordable for the majority of children with cancer in the world.
We need to have a drug access platform that can adjust to all of these factors, provide an accurate forecast at both global and country levels, and stabilize the market and the supply chain of quality drugs. And this is what we are trying to do by creating the Global Platform for Access to Childhood Cancer Medicines.
This platform is a good model for improving care for children with cancer in limited-resource settings. If successful, it could be expanded to other components in the cancer control continuum, from diagnostics to therapeutics.
Adapting a Humanity-in-Crisis Model in Cancer Care
Please talk about the difficulty of treating children with cancer in conflict countries and in treating refugees with cancer.
We have some experience with providing financial aid to Syrian refugees with cancer living in Lebanon and Jordan, for example, but we do not have good solutions to address this complex problem. One way to address these situations is through the creation of regional networks and humanitarian platforms, which allow these patients to receive cancer care in a center of excellence.
For Ukraine, we created the SAFER Ukraine program (https://global.stjude.org/en-us/saferukraine.html) in collaboration with multiple partners in Europe; it has facilitated the safe evacuation of more than 500 children with cancer from Ukraine to Poland and their transfer to centers in Europe and North America. However, this model may be more difficult to scale up in lower-resource settings, where the regional networks are less developed.
Delivering cancer care in times of manmade or natural disasters is complex, and international, multisectoral efforts are critical to its success.
DISCLOSURE: Dr. Rodriguez-Galindo reported no conflicts of interest.
1. World Health Organization: Childhood Cancer: Key Facts. Available at www.who.int/news-room/fact-sheets/detail/cancer-in-children. Accessed May 4, 2022.
2. Ward ZJ, Yeh JM, Bhakta N, et al: Global childhood cancer survival estimates and priority-setting: A simulation-based analysis. Lancet Oncol 20:972-983, 2019.
3. The World Bank: World Bank Country and Lending Groups. Available at https://datahelpdesk.worldbank.org/knowledgebase/articles/906519-world-bank-country-and-lending-groups. Accessed May 4, 2022.
4. World Health Organization: NCD Country Capacity Survey. Available at www.who.int/teams/ncds/surveillance/monitoring-capacity/ncdccs. Accessed May 4, 2022.