Studies for approval of new drugs have insufficient patients to evaluate safety

March 19, 2013

For medicines intended for chronic use, the number of patients studied before regulatory approval is insufficient to properly evaluate safety and long-term efficacy, requiring the need for new legislation, according to a study by European researchers published in this week's PLOS Medicine.

Current European guidelines specify that in order to fully evaluate the safety of medicines being developed for chronic (long-term) treatment of non-life threatening diseases, at least 1000 patients must take the new drug and that 300 and 100 patients must use the drug for 6 and 12 months, respectively, before approval by the European Medicines Agency.

In an analysis led by Ruben Duijnhoven from Utrecht University, the authors used information from the European Commission about 200 medicines approved between 2000 and 2010 to investigate whether the number of patients included were in compliance with the International Conference on Harmonisation E1 guidelines.

The authors found that the average number of patients studied before approval was 1708 for standard medicines and 438 for orphan medicines, medicines used to treat . On average, medicines for chronic use (for example, ) were studied in more patients (2338) than those for intermediate use such as anti- (878) or short-term use such as antibiotics (1315). The safety and efficacy of chronic use was studied in fewer than 1000 patients for at least 6 and 12 months in 46.4% and 58.3% of , respectively. Finally, the authors found that among the 84 medicines intended for chronic use, 69 were studied in at least 300 patients for 6 months and 67 were studied in at least 100 patients for 12 months.

The authors say: "For medicines intended for chronic use, the number of patients studied before marketing is insufficient to evaluate safety and long-term efficacy. Both safety and efficacy require continued study after approval."

They conclude: "In light of new scientific and legislative tools to monitor benefits and risks in clinical use, discussion of the long-term exposure requirements for approval of medicines, particularly for medicines intended for chronic use, seems warranted."

The authors add: "Such a discussion should involve healthcare providers, patients, and academia, as well as industry and regulators, and should include debate on the level of acceptable uncertainty, especially for adverse events and the long-term outcomes for chronic medication."

Explore further: New drugs should be compared with existing treatments before approval, say experts

More information: Duijnhoven RG, Straus SMJM, Raine JM, de Boer A, Hoes AW, et al. (2013) Number of Patients Studied Prior to Approval of New Medicines: A Database Analysis. PLoS Med 10(3): e1001407. doi:10.1371/journal.pmed.1001407

Related Stories

New drugs should be compared with existing treatments before approval, say experts

September 7, 2011
Manufacturers should have to show how their drugs compare to existing treatments before approval to help ensure that the most beneficial and safest therapies reach patients and that limited healthcare resources are invested ...

In drug-approval race, US FDA ahead of Canada, Europe

May 16, 2012
The U.S. Food and Drug Administration (FDA) generally approves drug therapies faster and earlier than its counterparts in Canada and Europe, according to a new study by Yale School of Medicine researchers. The study counters ...

Medical societies respond to the FDA's safety announcement on the use of Actos

June 16, 2011
Diabetes leaders today are responding to the announcement made by the U.S. Food and Drug Administration (FDA) yesterday that the use of the diabetes medication Actos (pioglitazone) for more than one year may be associated ...

Medicine agency: Roche did not report side effects

June 22, 2012
(AP) — The European Medicines Agency is investigating "deficiencies" in the way drug maker Roche reported possible side effects in patients in the U.S., though it said there was no evidence patients are at risk.

Recommended for you

Study suggests ending opioid epidemic will take years

July 20, 2017
The question of how to stem the nation's opioid epidemic now has a major detailed response. A new study chaired by University of Virginia School of Law Professor Richard Bonnie provides extensive recommendations for curbing ...

Team-based model reduces prescription opioid use among patients with chronic pain by 40 percent

July 17, 2017
A new, team-based, primary care model is decreasing prescription opioid use among patients with chronic pain by 40 percent, according to a new study out of Boston Medical Center's Grayken Center for Addiction Medicine, which ...

Private clinics' peddling of unproven stem cell treatments is unsafe and unethical

July 7, 2017
Stem cell science is an area of medical research that continues to offer great promise. But as this week's paper in Science Translational Medicine highlights, a growing number of clinics around the globe, including in Australia, ...

Popular heartburn drugs linked to higher death risk

July 4, 2017
Popular heartburn drugs called proton pump inhibitors (PPIs) have been linked to a variety of health problems, including serious kidney damage, bone fractures and dementia. Now, a new study from Washington University School ...

Most reproductive-age women using opioids also use another substance

June 30, 2017
The majority of reproductive-age and pregnant women who use opioids for non-medical purposes also use at least one other substance, ranging from nicotine or alcohol to cocaine, according to a University of Pittsburgh Graduate ...

At-risk chronic pain patients taper opioids successfully with psychological tools

June 28, 2017
Psychological support and new coping skills are helping patients at high risk of developing chronic pain and long-term, high-dose opioid use taper their opioids and rebuild their lives with activities that are meaningful ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.