Addressing Unmet Needs in Patients Undergoing Treatment for Rare Lymphomas

Collaborative efforts between the FDA, industry, and investigators are seeking to more effectively disseminate novel therapies to patients in more rural areas, or those receiving care in community practices, according to Neha Mehta-Shah, MD, MSCI.

CancerNetwork® spoke with Mehta-Shah, associate professor in the John T. Milliken Department of Medicine in the Division of Oncology at the Washington University Medical School in St. Louis, MO, about how clinical practice is working to actively address unmet needs existing for patients with rare lymphomas.

She began by expressing that unmet needs exist among therapies that can be administered with curative intent, particularly in bolstering their cure rate, minimizing toxicity and late-term effects of chemotherapy, and enhancing the quality of life for patients. She further stated that understanding patient outcomes through the inclusion of patient-reported outcomes was an unmet need that could help ascertain outcomes of therapy through systematic measures of self-report data.

Mehta-Shah further highlighted the Lymphoma Epidemiology and Outcomes (LEO) consortium, which she explained is attempting to better understand the impact of treatment for and diagnosis of a rare lymphoma on quality of life, among other factors.

Additionally, Mehta-Shah underscored a challenge associated with expedited FDA approvals, which may result in withdrawals. One such withdrawal, romidepsin (Istodax) for relapsed/refractory T-cell lymphoma, she claimed was difficult, given that the therapy has no active comparator.

Transcript:

Some of the needs that we need to address are things about rare lymphomas that we can cure, [such as] primary mediastinal B-cell lymphoma, gray zone lymphoma, Hodgkin lymphoma, diffuse large B-cell lymphoma. These are all lymphomas where the purpose of the treatment is [curative], but we know our current therapies do not cure 100% of the patients.

There is room for improvement there, across the spectrum, but also room to improve with regards to minimizing long-term toxicity, minimizing late-term effects of chemotherapy, monitoring the quality of life, and better understanding the patient experience through patient-reported outcomes, remains an unmet need. There is work being done nationally to do that, [many] efforts to look at survivorship, rates of fertility, impacts of neuropathy, and measure these systematically using novel patient-reported outcome tools that are being developed specific to these diseases to better understand the outcomes of therapy.

Some of this is being done by the LEO consortium––the Lymphoma Epidemiology and Outcomes consortium––to better understand the impact on quality of life, financially, socially, and emotionally of having a lymphoma diagnosis on those respective components. There are other rare lymphomas where there is not great therapy for the median survival for patients who have relapsed/refractory T-cell lymphomas––only 6 to 10 months. There is a lot of room to improve on treatments for patients.

In the current era, the FDA has pushed for randomized phase 3 studies to lead to approval, which has led to the withdrawal of drugs like romidepsin’s label in T-cell lymphoma, making it even more challenging to pursue phase 3 clinical trials in this space because there is not a great comparator. It is hard to design studies that patients and doctors want to be involved in, where patients could be randomly assigned to treatment that is not likely to be effective, although that is the current standard treatment. These regulatory impacts––which we understand why the FDA chose to make these decisions––do have downstream effects on the ability to develop clinical trials for patients with these rare diseases. There will be a continued collaboration between the FDA, industry, clinical investigators, and patients to bring these promising drugs to patients in local areas and not just at academic centers through clinical trials.

Reference

Bristol Myers Squibb statement on Istodax® (romidepsin) relapsed/refractory peripheral T-cell lymphoma U.S. indication. New release. Bristol Myers Squibb. August 2, 2021. Accessed April 10, 2025.