New cancer immunotherapy drugs rapidly reach patients after approval

The majority of patients eligible for cancer immunotherapy drugs known as checkpoint inhibitors received treatment within a few months of FDA approval, according to a new Yale-led study. The finding suggests that cancer immunotherapies are adopted at a much quicker pace than is typical for newly approved medical treatments, the researchers said.

However, patients receiving the therapies are older than those in the clinical trials used to evaluate them, pointing to a disconnect between research and practice that should be addressed, they noted.

The study was published in JAMA Oncology.

Immune checkpoint inhibitors have transformed cancer care, leading to dramatic responses in patients with advanced cancers who previously had very limited chances for long-term survival. To evaluate the speed with which these drugs are adopted after FDA approval, the Yale team collaborated with researchers from Flatiron Health, an oncology software and services company that develops real-world datasets from a national network of oncology clinics and academic research centers.

The researchers found that it took less than four months after approval for the majority of eligible patients to receive treatment with at least one of the drugs. The patients included individuals treated for melanoma, non-small cell lung cancer (NSCLC), and renal cell carcinoma, a type of kidney cancer.

While this rapid rate of adoption is encouraging, the research team said, they also found that the majority of patients treated with checkpoint inhibitors in practice were 65 and older, unlike patients treated in clinical trials, whose median ages ranged from the late 50s to early 60s. It's unclear whether patients in practice will respond in the same way as clinical trial patients. The recognition that patients in clinical trials differ from those treated in routine clinical practice highlights the importance of studying these real-world populations, the researchers said.

"There are many reasons to be excited about the rapid adoption of these new treatments," said Jeremy O'Connor, M.D., first author and a postdoctoral fellow at Yale School of Medicine (YSM). "But it can be risky for rapid adoption to be based on early findings from trials because drugs might be shown in later trials to be less effective or even harmful. This is why it's so important for the FDA to use strong evidence for its approvals and for trials to test drugs among patients who are similar to those being treated in everyday practice."

The study results also concern the researchers because new therapies are being approved when questions remain about their risks and benefits.

"Our findings underscore the importance of a thorough FDA review of new cancer therapies," said Cary Gross, M.D., senior author, YSM professor of medicine, and a member of Yale Cancer Center. "We found that the standard of care can change virtually overnight, when a new drug is brought to market. But because these initial FDA approvals are frequently based on studies that may be small, have no comparison arm, or are restricted to younger and healthier patients, it is essential that we think of FDA approval as one step in the continuing evaluation of whether new drugs are indeed beneficial in the larger population."

Jeremy M O'Connor, Kristen L Fessele, Jean Steiner, Kathi Seidl-Rathkopf, Kenneth R Carson, Nathan C Nussbaum, Emily S Yin, Kerin B Adelson, Carolyn J Presley, Anne C Chiang, Joseph S Ross, Amy P Abernethy, Cary P Gross.
Speed of Adoption of Immune Checkpoint Inhibitors of Programmed Cell Death 1 Protein and Comparison of Patient Ages in Clinical Practice vs Pivotal Clinical Trials.
JAMA Oncol. 2018; :e180798. doi: 10.1001/jamaoncol.2018.0798.

Most Popular Now

Chemists characterize the fatal fungus among us

Life-threatening fungal infections affect more than two million people worldwide. Effective antifungal medications are very limited. Until now, one of the major challenge...

FDA approves first cancer drug through new oncolog…

The U.S. Food and Drug Administration today approved Kisqali (ribociclib) in combination with an aromatase inhibitor for the treatment of pre/perimenopausal or postmenopa...

Discovery of kidney cancer driver could lead to ne…

University of North Carolina Lineberger Comprehensive Cancer Center scientists have uncovered a potential therapeutic target for kidney cancers that have a common genetic...

The immune system: T cells are built for speed

Without T cells, we could not survive. They are a key component of our immune system and have highly sensitive receptors on their surface that can detect pathogens. The e...

Pfizer initiates pivotal Phase 3 program for inves…

Pfizer Inc. (NYSE:PFE) and Spark Therapeutics (NASDAQ:ONCE) announced today that Pfizer initiated a Phase 3 open-label, multi-center, lead-in study (NCT03587116) to evalu...

FDA approves first targeted treatment for patients…

The U.S. Food and Drug Administration today approved Tibsovo (ivosidenib) tablets for the treatment of adult patients with relapsed or refractory acute myeloid leukemia (...

Sanofi and REVOLUTION Medicines launch global part…

Sanofi and REVOLUTION Medicines, Inc. today announced an exclusive worldwide partnership to develop and commercialize targeted therapies, based on the biology of the cell...

Novartis renews drug donation of Egaten® (triclabe…

Novartis reaffirms its commitment to the fight against liver fluke (fascioliasis), signing a renewed memorandum of understanding with the World Health Organization (WHO) ...

Women and older people under-represented in drug t…

Trying to determine how best to treat a patient, doctors often look to randomized clinical trials to guide their choice of what drug to prescribe. One of the most common ...

New findings suggest allergic responses may protec…

The components of the immune system that trigger allergic reactions may also help protect the skin against cancer, suggest new findings. The research, led by Imperial Col...

FDA grants Breakthrough Therapy Designation for Ro…

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tecentriq® (atezolizumab) i...

Novartis marks a new era for migraine patients wit…

Novartis announced today that the European Commission (EC) approved Aimovig® (erenumab) for the prevention of migraine in adults experiencing four or more migraine days p...