“Clinicians are not alone in taking notice, according to Niesha Griffith, MS, RPh, a pharmacist at the James Cancer Hospital of The Ohio State University in Columbus.
“Multiple patients at her center have requested these drugs for off-label use, and offered to pay upfront and out-of-pocket for the expensive therapies, she said. Such offers were rare before the advent of cancer immunotherapy, but now occur regularly.”
The gist: The drug docetaxel was widely used to treat patients with metastatic prostate cancer before phase III clinical trials showed it was better than standard treatment. This is significant for a few reasons. First, it shows that many doctors are willing to prescribe promising drugs “off-label,” before the U.S. Food and Drug Administration (FDA) has approved them for treating certain conditions. It also shows that a drug that is used for one type of cancer is more likely to be prescribed off-label for other types of cancer. Some are concerned that off-label prescriptions undermine the clinical trial process and put some patients at risk by giving them drugs that may not turn out to be better.
‘Docetaxel was being widely used by patients with metastatic prostate cancer before phase III evidence that it was more effective than standard-of-care for patients with castration-resistant prostate cancer, according to an analysis of Medicare claims from before and after the trial results and approval of docetaxel by the U.S. Food and Drug Administration. The uptake of new treatments, or diffusion, “prior to definitive evidence indicates the prevalence of off-label chemotherapy use,” wrote Unger et al in the Journal of the National Cancer Institute…
“Rapid and widespread use of docetaxel was not observed among all study participants receiving chemotherapy for metastatic prostate cancer. Statistically significant docetaxel uptake (P < .01) was seen in patients older than 65 years of age, blacks, patients in lower income areas, and those who experienced poverty. This observation ‘presents opportunities to improve the uptake of proven new therapies in subpopulations,’ the authors wrote, adding that direct-to-consumer advertising ‘could be a useful tool.’
Another potential method cited for encouraging more rapid adoption of new treatments was ‘enhancing communication channels among physicians, especially between key opinion leaders and their colleagues,’ the authors noted. ‘One factor that has been repeatedly identified to increase adoption rates is attendance at scientific symposia, which serve as forums for disseminating information about new treatments.’ ”
The gist: A new clinical trial will help patients get access to personalized treatments. Sometimes, doctors can match a patient to a targeted drug that is likely to work, based on genetic mutations found in the patient’s tumor(s). Different types of cancer can sometimes have the same tumor mutations, so they could potentially be treated with the same drugs. In this clinical trial, all patients’ tumors will be molecularly tested to determine which targeted drugs might work best. These drugs may have been developed for a different type of cancer, so they will be prescribed “off-label”. A drug that is prescribed off-label has already been approved by the U.S. Food and Drug Administration for a different cancer type, so doctors already know safe dosages and side effects to look out for. This clinical trial’s unique approach will help researchers better understand how well different off-label treatments work.
“ASCO plans to launch a first-ever study that will offer cancer patients access to molecularly-targeted cancer drugs and collect ‘real-world’ data on clinical outcomes to help oncologists learn the best uses of these drugs outside of approved indications, ASCO Chief Medical Officer Richard L. Schilsky, MD, FACP, FASCO, reported today at a cancer research conference held in Washington, DC.
“ ‘One of the major challenges to implementing personalized medicine is the lack of information about the risks and benefits of targeted drugs that are used off label to treat patients whose tumor harbors a genomic abnormality,’ Dr. Schilsky said at the Friends of Cancer Research-Brookings Institution Conference on Clinical Cancer Research. ‘Other difficulties are lack of access to these agents for patients and interpretation by oncologists of the complex genomic test results. This ASCO-led clinical trial will address both challenges.’
“The ASCO project, called the Targeted Agent and Profiling Utilization Registry (TAPUR) Study, will be the first clinical trial ever led by the society in its 50-year history.”
The gist: New research looking into tumor mutations in small cell lung cancer (SCLC) and neuroendocrine tumors (NET) may open up new drug options to treat these conditions. Drugs called targeted therapies have been developed to treat people with tumors that have certain genetic mutations. Several targeted therapies are available for people with non-small cell lung cancer (NSCLC). But so far, targeted therapies have not been very useful in SCLC. Now, researchers have found that some SCLC and NET tumors may share some tumor mutations with NSCLC tumors. Theoretically, a patient with SCLC or NET could ask their oncologist for molecular testing to see whether their tumor(s) could potentially be treated off-label with an existing NSCLC drug. To learn more about SCLC treatment, see our Need to Know blog.
“Analysis of 607 small cell lung cancer (SCLC) lung tumors and neuroendocrine tumors (NET) identified common molecular markers among both groups that could reveal new therapeutic targets for patients with similar types of lung cancer, according to research presented today at the 2014 Chicago Multidisciplinary Symposium in Thoracic Oncology. The Symposium is sponsored by the American Society of Clinical Oncology (ASCO), the American Society for Radiation Oncology (ASTRO), the International Association for the Study of Lung Cancer (IASLC) and The University of Chicago Medicine.
“This study examined the clinical specimens of 607 total cases of SCLC tumors (375) and lung NET (232), which included carcinoid, atypical carcinoid and large-cell neuroendocrine tumors. Biomarker testing was achieved through a combination of DNA sequencing (Next-Generation Sequencing (NGS) or Sanger-based); immunohistochemistry (IHC) to identify which proteins are present; and in situ hybridization (ISH) testing, a form of gene amplification, to determine if any of the markers that can cause cancer cells to grow or to become resistant to treatment are present…
” ‘Even cancers that appear to be very similar can be dramatically different at the molecular level, and these differences may reflect unique vulnerabilities that could positively impact therapeutic options and decisions,’ said Stephen V. Liu, MD, senior study author and Assistant Professor of Medicine in the Division of Hematology/Oncology at Georgetown University’s Lombardi Comprehensive Cancer Center in Washington, DC. ‘We are pleased that this research confirms these rarer subtypes; it calls for additional investigation on a larger scale. Once confirmed, molecular profiling of small cell tumors and NET could become standard, as it is currently for non-small cell lung cancers, which will be especially important as more molecularly targeted chemotherapy agents are developed.’ “