With the explosion of new cancer drugs, combinations, targeted agents, and immune therapies, one question remains: What is the impact of these new treatments on the quality of life (QOL) for patients that receive them? The answer is, we do not know.
To address the question, a study reported in JAMA (Arciero, V. JAMA Network, Open, 2021; 4(2)) examined 214 oncology drug indications approved by the FDA between 2006 and 2017 for their impact on the QOL of cancer patients.
They found that QOL data were reported in only 40% of registration clinical trials. More concerning was that improvements beyond minimal clinically meaningful differences (MCID) were observed in only 6% of these FDA-approved indications.
Despite significant advances in our understanding of cancer biology and the development of numerous new drugs, the impact of these drugs on long-term survival remains limited. As a result, much of the care delivered is considered palliative, which means that it is delivered in an attempt to make cancer patients feel better. The fact that QOL data was neither a driving factor nor even included in many clinical studies is disappointing.
The American Society of Clinical Oncology has established a framework for the assessment of QOL in the form of a score. One of the best determinants of a drug's impact on QOL is the minimal clinically important difference (MCID) which is the smallest amount of improvement for a new treatment over the standard treatment that actually affects a patient’s life.
The authors show that of 71 solid tumor drugs approved by the FDA between 2002 and 2014, the average improvements in progression-free survival and overall survival were a rather meager 2.5 months and 2.1 months, respectively. With such small survival differences, quality rather than quantity of life would seem to be the obvious target yet the authors note "Our studies suggest that systemic oncology therapies are often approved by regulatory agencies without evidence to demonstrate quality of life improvement."
Cancer chemotherapies are among the most expensive drugs in use today. The average cancer drug approved since 2012 costs over $10,000 per month. If these costs are not associated with improved QOL then we may need to reassess resource allocation in this field.
The implications of this study are several. First, the approval process for drugs in oncology has relied on endpoints like response rate and progression-free survival, which doesn’t always translate into overall survival. Secondly, in the absence of meaningful survival advantages, the drug toxicities that are associated with these responses and progression-free survival benefits must be carefully considered. If we, unfortunately, cannot cure many cancer patients then there must be a much greater focus on these patient’s comfort and wellbeing.
The authors make a compelling point when they argue that it is not just the quantity of extended life measured in days, weeks, or months but more importantly the quality of life associated with the use of these new cancer treatments.
As always, I appreciate your thoughts and comments.
Dr. Robert Nagourney has been internationally recognized as a pioneer in cancer research and personalized cancer treatment for over 20 years. He is a TEDx speaker, author of the book Outliving Cancer, a practicing oncologist, and triple board-certified in Internal Medicine, Medical Oncology, and Hematology helping cancer patients from around the world at his Nagourney Cancer Institute in Long Beach, California. For more info go to NagourneyCancerInstitute.com