Cancer. A diagnosis no doctor wants to deliver, and no patient wants to hear. As devastating as the diagnosis is for an adult, it’s infinitely more tragic and terrifying when the patient is your child. Yet nearly 12,000 American parents hear that nightmare diagnosis every year. I was one of them.
While technical and medical advances mean pediatric cancer is not always a death sentence, carrying your child through cancer treatments is always unimaginable. Physicians offer patients and their families great hope with treatment protocols to prolong and improve patients’ quality of life. Sometimes they can even cure the disease. But treatment protocols are far from simple, and depend on the type and stage of cancer, possible side effects, and the patient’s overall health. Whether facing surgery, radiation, and/or chemotherapeutic and immunotherapeutic medications, the struggle to maintain any sort of normalcy in the child’s and the family’s lives is a huge challenge.[1] Treatments are given in cycles, with their duration, frequency, and number precisely regulated. Eventually families become accustomed to the new rhythm of their days, weeks, months, and years, living on pure hope that all will go as planned.
There’s hope but there’s also a catch
Imagine pinning your hope and your child’s future on a potentially life-saving treatment protocol, only to hear that one or more of the medicines isn’t available when and where it’s needed. That’s what happened to our family when we were told the life-saving chemotherapy my daughter needed was unavailable because there was a shortage. I soon learned that every day, for a host of reasons, medicines aren’t immediately available to the people that need them. A patient fighting for their life—or a parent fighting for their child’s life-- doesn’t have the luxury of time to wait. Though the problem is worse in lower- and middle-income countries, drug shortages occur around the globe. Over the past decade, despite tremendous efforts, drug shortages in the U.S. have occurred with greater frequency and are lasting longer.[2]
Drug shortages can range from problematic inconveniences to dire emergencies. If there are multiple treatment options for a patient’s condition, a drug shortage can sometimes be overcome by alternative treatments substituted for the one that’s in short supply. But for other drugs, there may be few, or even only a single manufacturer. Sadly, this is the case for some pediatric oncology drugs, which means families are sometimes told they cannot have access to a treatment needed to save their child’s life. The most vulnerable patients with the smallest voices are sometimes being left behind.
Even more infuriating is a related situation that occurs all too often: the drug is available, but not at the place and time a patient may need it. The quantity and location of some drugs is poorly tracked due, in part, to the size and complexity of the global medicine supply chain. Sometimes there are even legal implications to the sharing of information about where a drug may be located. Information gaps can make finding critical medicines like looking for the proverbial needle in a haystack. Yet, when potentially life-saving treatments exist, all patients deserve access to these critical drugs that can save their lives. Doctors must have access to the tools they need to treat patients when and where the patients need them.
Advocates for the most vulnerable
Angels for Change is a non-profit organization dedicated to increasing awareness of the life-saving drug shortage crisis and building support to create solutions that will help solve drug shortages and enable equitable patient access to the medicines they need. I founded Angels for Change when I was told that there was a shortage of the oncology medicine that my daughter needed, and her treatment would have to be postponed. I’m glad to be able to say that we were able to find the drug and get it with only a 2 week delay in my daughter’s treatment. Without our direct involvement that delay could have been 2 months or more. To say that it was a monumental task would be like saying that the ocean is a little damp. But it is a task I undertook for my child, and one I continue to undertake, through Angels for Change, for other families when needed.
Our experience wasn’t an anomaly. Over the past decade, eight of the ten drugs used to treat a single type of childhood leukemia have been temporarily unavailable. At the height of the shortage, over 80% of oncologists surveyed reported that they couldn’t prescribe their preferred chemotherapeutic due to shortages. Over 40% reported delaying patient treatment while trying to track down a drug in short supply.[3] I’m proud to say that since our founding in October of 2019, Angels for Change has helped over 40 families locate and obtain pediatric oncology medicines that were supposedly unavailable. But so much more needs to be done. It can’t end there. While our help was significant to each individual facing a shortage, by the time I found out about each shortage, the patient’s treatment plan was already disrupted. We want to eradicate these patient-level disruptions. To do so we must start focusing our efforts further up the supply chain.
Why do drug shortages happen?
What causes drug shortages? According to a report from the inter-agency Drug Shortage Task Force, led by the US FDA, root causes of drug shortages include a lack of incentives offered to manufacturers for making less-profitable drugs, lack of recognition and reward for manufacturers that focus on continuous improvement and early detection of supply chain issues, and logistical and regulatory challenges that hamper recovery from disruptions.[4]
The complexity and global nature of the medicine supply chain means that economics is one of the many variables that can potentially put drugs at risk of shortages. Others include a limited number of manufacturers, limited geographic diversity, natural disasters, quality issues, and changes in regional political climates.
Information and communication – or lack thereof – is another contributing factor. The medicine supply chain is an intricate labyrinth. Raw materials and finished drug products come from all parts of the globe. They are transported by planes, trains, ships and trucks to manufacturers, packaging facilities, warehouses, distributors, retailers, hospitals, pharmacies, and other stops along the way before they reach physicians and, ultimately, patients. It’s easy to imagine information getting lost in multiple inventory tracking and management systems that may not communicate with each other.
What can be done?
But when a child’s life is at stake, none of these reasons for drug shortages seem acceptable—or unsolvable. Surely there is more that can be done. We need ways to know how much of a given drug is available and where it is. An early warning system that lets us know there’s an imminent risk of a shortage, so steps can be taken to mitigate or avoid it altogether. Such a system would aggregate and analyze many kinds of data sets associated with factors that could impact the supply of a drug. It would characterize and quantify a medicine’s vulnerability, building a holistic view of the supply chain that can highlight risk. With this kind of advanced warning, manufacturers, and other links in the supply chain could take action to help prevent the shortage from occurring.
Angels for Change is engaging with stakeholders in different parts of the healthcare system to foster multi-disciplinary approaches that will be needed to help end drug shortages for pediatric oncology medicines. Industry leaders at the Children’s Hospital Association, McKesson Corporation, Phlow Corporation, United States Pharmacopeia (USP), and Vizient Inc. are working with us to bring us closer to solutions to this crisis. As founder of Angels for Change, I believe that we are all members of this health system and it will take all of us together to end this healthcare crisis. I believe in the goodness of people and the power of minds working together to solve problems.
One of our current projects focuses on the various factors that contribute to shortages of pediatric oncology medicines. We’ve leveraged USP’s Medicine Supply Map to help us identify and evaluate some of these contributing factors.
The Medicine Supply Map analyzes information from disparate data sources and extracts relevant insights about potential risk and resilience of the upstream pharmaceutical supply chain. As more data sets are included, a more comprehensive understanding of the drug supply is generated
With more comprehensive information, organizations across the medicine supply chain can respond to potential risk sooner, and even better, prevent shortages before they happen. We would like to eradicate the patient level conversations that took place in that hospital room for us in April of 2019. No patient should hear the words “We don’t have the drugs to save you,” and no doctor should have to say those words.
The report from the Drug Shortage Task Force recommends that solutions to help end drug shortages include creating a shared understanding of the impact of shortages on patients, incentivizing manufacturers to invest in quality management maturity for their facilities and promoting sustainable private sector contracts to ensure a reliable supply of important medicines.[5]
Solving the crisis of drug shortages will ultimately require a global effort to make eradication of drug shortages a priority and dedicate the time and resources that are needed to solve it. It won’t happen overnight, but it can and must happen. Enduring solutions will come from collaboration.
The first step in solving any problem is identifying and understanding it. The work we are doing, and tools like the Medicine Supply Map may help us make this possible.
[1] https://www.cancer.net/cancer-types/childhood-cancer/types-treatment
[2] https://www.statnews.com/2019/03/19/drug-shortages-jeopardize-children-cancer/
[3] https://www.nejm.org/doi/full/10.1056/NEJMc1307379
[4] https://www.fda.gov/media/131130/download
[5] https://www.fda.gov/drugs/drug-shortages/report-drug-shortages-root-causes-and-potential-solutions