Oral medications are being increasingly used to treat patients with lymphoma and chronic lymphocytic leukemia (CLL). Five oral drugs have been approved over the past three years for eight different indications in lymphoma and CLL. Many patients report numerous advantages of oral therapies, including greater convenience and the ability to receive treatment in close proximity to their home. However,disadvantages such as complex treatment regimens, added caregiver responsibilities for symptom management and financial burden are also reported. As these agents are administered outside of a medical setting, the reasons behind patient nonadherence is difficult to accurately measure, which creates an impediment to the development of related adherence interventions and tools. In October 2017, the Lymphoma Research Foundation (LRF) convened the Adherence and Oral Therapies in Lymphoma and CLL Workshop as a next step identified in the 2015 workshop Oral Therapies in Lymphoma: Opportunities and Challenges in Research and Treatment.
The 2017 workshop was convened with a diverse faculty to explore encourage dialogue among experts in the field on areas of future investigation, including an overview of the epidemiology and types of nonadherence; the need to research polypharmacy (use of multiple drugs simultaneously), adherence and collaborative healthcare models; and the experience of clinicians and researchers working in chronic myeloid leukemia (CML) over the past decade following the introduction of oral tyrosine kinase inhibitors. The workshop co-chairs, Jonathan Friedberg,MD, MMSc, of the University of Rochester Wilmot Cancer Institute and Michael E. Williams, MD, ScM, of the University of Virginia Cancer Center, together with the Workshop Steering Committee – Christopher Flowers, MD, of Winship Cancer Institute, Emory University, John P. Leonard, MD, of Weill Cornell Medicine and Sonali M. Smith, MD of The University of Chicagoalso provided their insights from their work in lymphoma.
Five core themes emerged over the course of the October 2017
- Patients on oral therapy regimens generally receive less supervision than those on intravenous therapies and have less interaction with their healthcare team.
- Patients utilizing medications for other health conditions may experience issues including drug-drug interactions, or have additional difficulty adhering to complex regimens or drug administration schedules.
- Currently, there are no standardized methods for assessing, defining, or measuring adherence, and how adherence affects clinical outcomes and disease control in patients with lymphoma is unknown.
- Based on experience with long-term treatment of other malignancies with oral therapies like CML, some side effects may not emerge until after years on the regimen.
- Because of the drug costs and often long duration of treatment required, oral therapies may lead to financial burden or financial toxicity due to high copays. The type of insurance coverage the patient has may be a contributor to poor adherence.
Recommendations included the development of new models of patient management specific to oral therapies; greater patient education about the importance of taking their medication as prescribed; research on the relationship between patients’ level of adherence to oral therapies and disease outcomes in patients with lymphoma and CLL; and the creation of patient registries to capture both short-term and long-term adverse events to capture toxicities that may be bothersome or that may emerge late during treatment.
LRF plans publication of a white paper followed by a proceedings document to help inform decision-making and research within the public, private, and nonprofit sectors. The latter opinion piece will be a call to action emphasizing the need for further research into the impact of adherence and its relationship to patient outcomes.