A rare cancer diagnosis comes with uncertainty that leads to anxiety and frustration for patients, doctors, and scientists. While experts do their best to recommend therapies for these cancers, we face many challenges when diagnosing and treating them.
World Cancer Day is an initiative of the Union for International Cancer Control (UICC), the largest and oldest international cancer organization dedicated to taking the lead in convening, capacity building and advocacy initiatives that unite the cancer community to reduce the global cancer burden, promote greater equity, and integrate cancer control into the world health and development agenda. Through investing in research and innovation, there have been many extraordinary breakthroughs in medicine and research, especially for rare diseases. Tools like gene therapy and decentralized trials—which help lessen the burden on patients who have to travel far distances to sites—help patients get the care they need.
In light of the cooperation that helped us create the COVID-19 vaccine in record time, we’re seeing new methods and more collaboration in the research field. At PRA, we commit to helping patients get the resources they need for better, more accessible rare cancer treatment. Let’s look at some of the struggles that come with rare cancer diagnoses and how we’re working to improve treatment therapies.
How Rare Are Rare Cancers?
With 158 types of rare cancer, they’re more common than you might think. While each one affects no more than six in 100,000 people, the combined diagnoses mean they make up a significant portion of cancer cases. Between 25 percent and 47 percent of cancer patients have a rare cancer, depending on which counting criteria is used.
However, all of these people don’t have the same rare cancer, which makes treatment more challenging. Consider, too, that one-third of those people with rare cancers have an especially rare cancer, which means there may be very little information available about it.
What Challenges Do We Face in Rare Cancer Diagnosis and Treatment?
When it comes to patient challenges, people with rare cancers often have less access to helpful treatment and therapies. With less research on these types of cancer, there is less support for patients dealing with them. Plus, no standard treatment exists for most rare cancers, as researchers don’t have enough information to support drug development.
Consequently, rare cancers have lower survival rates as a result of late or incorrect diagnoses. Fewer doctors and researchers know what to look for in rare cancers or how to treat them. Even less knowledge exists in rare pediatric cancers, as few scientists focus on Very Rare Tumors (VRTs).
When it comes to research, laboratories have trouble conducting clinical trials due to issues like:
- Lack of patient availability
- Lack of qualifications from patients with rare cancers
- Not enough interest from the oncology field
Rare cancer patients often have trouble traveling for treatment. They may lack the necessary access to resources and treatments that might improve their condition. Researchers also have trouble getting enough tumor tissue to study the cancer, making it harder to create new drugs.
When patients receive a diagnosis, it can be difficult to proceed when different doctors have varying ideas about treating rare cancers. Doctors can’t tell patients what to expect with a rare cancer because they simply do not know. They have a limited ability to answer patient questions due to minimal training in treating specific rare cancers.
What Are We Doing to Improve Rare Cancer Treatment?
The good news is that cancer research is progressing at an incredible rate. As we continue to discover cancer subtypes and advance oncology research, we have developed new therapies.
Genomic medicine, a new form of cancer therapy, uses tumor tissue or blood samples to create new treatments. Genomic testing identifies biomarkers that can lead to a better understanding of what treatments may help a specific rare cancer.
This testing can lead to more personalized treatment plans, which may improve a patient’s prognosis. As a result, we can gain information about which treatments may work best for other patients. Genomic medicine also helps prevent harm to healthy cells and reduces treatment side effects.Learn about how gene therapy is revolutionizing treatments for rare diseases.
Along with new therapies, more experts around the world have begun collaborating after the success of the COVID-19 vaccine development. Organizations are coming together to focus on treating rare cancers, too.
Target Rare Cancer Knowledge (TRACK) has formed to support genomic testing and precision medicine to recommend better-personalized treatment and maintain focus on rare cancers. The PARTNER project is also creating a VRT registry in places that don’t already have one. Having that information available gives more people access to resources and therapies they may not have otherwise.
What Needs to Happen to Support Better Treatment?
We need more research and understanding of rare cancers. That means more clinical trials and better study methods. It also means more samples to establish rare cancer progression patterns. Above all, we need collaboration and communication between experts. When we work together, we can establish a wider knowledge base of how rare cancers affect people. That leads to more research into treatment options and a better understanding of the right direction when it comes to studying these diseases.
“I find clinical research to still be rather archaic, and I believe we can do much better,” says Scott Schliebner, Senior Vice President, Scientific Affairs and Head, Center for Rare Diseases. “I am inspired to look for smarter, better ways to engage patients, to ensure clinical research can be a legitimate care option, and to design clinical trials that are feasible and realistic for all patients. I want to be disruptive and help push our industry forward.”
For more information on how our patient-centric approach and remote technology has made a difference in cancer research, connect with our Oncology team.
The time and effort spent by the patient and their loved ones in managing a rare disease is time better spent on living a fuller life. We dedicate our time to rare disease research so they don’t have to.
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