According to the Food and Drug Administration (FDA), 35 drugs were approved in the past 12 months, as of this writing, surpassing the number approved in 2009 by two.1 The recently passed agents include important medical advances in the fields of hepatitis C, where two new agents were approved, and systemic lupus erythematosus (SLE), where belimumab was the first new lupus agent to be approved in 50 years. Innovative processes were used to approve these new medications while not compromising drug safety. Additionally, 24 of the agents were approved in the United States before approval in any other country. Ten of the new agents are for orphan conditions, some are for cancers, and some were approved with genetic tests using principles of personalized medicine. Further, 34 of the 35 were approved on or before their targeted Prescription Drug User Fee Act (PDUFA) review dates. Prospects about drug approvals in 2011 were anticipated to be few, but this does not seem to have happened.
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Explore This IssueJanuary 2012
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In a research letter published online in the Archives of Internal Medicine, Powers and Cook stated that approximately half (48%) of the potential “drug safety signals” about adverse events listed on the FDA’s website from 2008 through 2010 resulted in labeling changes. However, most (62%) of these “signals” were listed in an “updated Warnings and Precautions section” without guidance as to what action should be taken.2 Four Risk Evaluation and Mitigation Strategy (REMS) and one drug withdrawal were also identified. These “signals” included serious risks and new safety information that were identified through the Adverse Event Reporting System (AERS). It is a significant challenge for the FDA and for drug safety scientists to promptly identify safety signals and efficiently and promptly evaluate them so that the interval of uncertainty between the time a safety signal is identified and when it is resolved is minimized.3 The FDA is not the only group evaluating drug safety signals: The Institute for Safe Medical Practices, also known as ISMP, also screens the AERS database and publishes their own quarterly report. The FDA is currently exploring ways to enhance and analyze drug safety and surveillance.
As reported in The Rheumatologist‘s September 2011 issue (“Drug Updates,”), over-the-counter (OTC) as well as prescription labeling of acetaminophen are undergoing changes to reduce the risk of toxicity.4 The United Kingdom is also experiencing similar problems. Significant hepatotoxicity and renal toxicity have been reported in patients associated with too much acetaminophen (paracetamol), according to a recently published study.5,6 The study’s authors report acetaminophen as the main cause of acute liver failure. Staggered acetaminophen dosing, rather than a one-time ingestion, and delayed presentation to a hospital are common reasons for fatalities and liver transplantation. When patients were asked why they repeatedly ingested more than the recommended dose of acetaminophen, pain relief was often cited as their reason (58%). Since this is a worldwide problem, patients that ingest acetaminophen for pain management should be adequately counseled by their pharmacist and/or physician to prevent toxicity while getting the greatest benefit from it. Those patients with problematic dosing should be closely monitored for multiorgan failure.
Apremilast, an investigational oral phosphodiesterase-4 inhibitor, was recently shown to have a similar benefit in patients with both psoriatic arthritis and ankylosing spondylitis (AS), when used along with methotrexate (MTX).7 In a 12-week, placebo-controlled study of patients with psoriatic arthritis, 47% of patients taking apremilast 20 mg twice daily plus MTX achieved an American College of Rheumatology 20 (ACR20), compared with 41% of patients who received apremilast monotherapy (20 mg twice daily). Additionally, 37% of patients treated with apremilast 40 mg once daily plus MTX achieved an ACR20 response, versus 35% of patients who received apremilast monotherapy at the same dose. No treatment differences were considered statistically significant. It is believed that apremilast interrupts the inflammatory cascade in immune cells acting as a pluripotent immunomodulator. For AS, patients received 10, 20, or 30 mg of apremilast twice daily and were compared with placebo-treated patients. After treatment for 85 days, apremilast-treated patients had decreased Bath Ankylosing Spondylitis Activity Index (BASAI) scores of more than 1.5 points, compared with a decrease of approximately 0.75 points for placebo-treated patients (compared with baseline). The differences were not statistically significant. Additionally, there was a mean change from baseline for receptor activator of nuclear factor kB Ligand (RANKL) for apremilast-treated patients versus placebo-treated patients (73.2 versus 108.2; P=0.016). Apremilast represents a promising new oral biologic agent for both of these conditions.