Studies for approval of new drugs have insufficient patients to evaluate safety
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 rare diseases. On average, medicines for chronic use (for example, asthma medications) were studied in more patients (2338) than those for intermediate use such as anti-cancer drugs (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 new medicines, 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."
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
Journal reference:
PLoS Medicine
Provided by
Public Library of Science
-
Risks of increased access to over-the-counter medicines may outweigh benefits
Mar 28, 2008 |
not rated yet |
0
-
New drugs should be compared with existing treatments before approval, say experts
Sep 07, 2011 |
not rated yet |
0
-
In drug-approval race, US FDA ahead of Canada, Europe
May 16, 2012 |
not rated yet |
0
-
Medical societies respond to the FDA's safety announcement on the use of Actos
Jun 16, 2011 |
not rated yet |
0
-
Medicine agency: Roche did not report side effects
Jun 22, 2012 |
not rated yet |
0
-
Motion perception revisited: High Phi effect challenges established motion perception assumptions
Apr 23, 2013 |
3 / 5 (2) |
2
-
Anything you can do I can do better: Neuromolecular foundations of the superiority illusion (Update)
Apr 02, 2013 |
4.5 / 5 (11) |
5
-
The visual system as economist: Neural resource allocation in visual adaptation
Mar 30, 2013 |
5 / 5 (2) |
9
-
Separate lives: Neuronal and organismal lifespans decoupled
Mar 27, 2013 |
4.9 / 5 (8) |
0
-
Sizing things up: The evolutionary neurobiology of scale invariance
Feb 28, 2013 |
4.8 / 5 (10) |
14
-
Why is zone 1 in liver more prone to ischemic injury?
May 23, 2013
-
How can there be villous adenoma in colon, if there are no villi there
May 22, 2013
-
How can there be a term called "intestinal metaplasia" of stomach
May 21, 2013
-
Pressure-volume curve: Elastic Recoil Pressure don't make sense
May 18, 2013
-
If you became brain-dead, would you want them to pull the plug?
May 17, 2013
-
MRI bill question
May 15, 2013
- More from Physics Forums - Medical Sciences
More news stories
Feds fight morning-after pill age ruling in NY
(AP)—Department of Justice lawyers have again asked a federal appeals court in New York to delay lifting age restrictions and prescription requirements on an emergency contraceptive popularly known as the morning-after ...
Medications
20 hours ago |
not rated yet |
0
Merck ends development of Parkinson's disease drug
(AP)—Merck & Co. says it is ending development of an experimental Parkinson's disease drug because the drug wasn't working.
Medications
May 23, 2013 |
1 / 5 (1) |
0
J&J expects 10-plus new drug applications by 2017
(AP)—Johnson & Johnson is developing what could eventually be game-changing treatments for depression and pain, and it's aiming to apply for approval of more than 10 new medicines by 2017, executives said Thursday during ...
Medications
May 23, 2013 |
5 / 5 (1) |
0
Experts favor US approval of Merck sleeping pill (Update)
An independent panel of experts on Wednesday recommended US approval of a new Merck sleeping pill called suvorexant, but expressed concerns over the highest dosage and risks of drowsy daytime driving.
Medications
May 22, 2013 |
not rated yet |
0
Glaxo, US partnering to develop new antibiotics
GlaxoSmithKline PLC says it's starting an unusual collaboration with the U.S. government to develop several antibiotics for both bioterrorism threats and bacterial infections resistant to current medicines.
Medications
May 22, 2013 |
not rated yet |
0
First drug to improve heart failure mortality in over a decade
Coenzyme Q10 decreases all cause mortality by half, according to the results of a multicentre randomised double blind trial presented today at Heart Failure 2013 congress. It is the first drug to improve heart failure mortality ...
Heart failure accelerates male 'menopause'
Heart failure accelerates the aging process and brings on early andropausal syndrome (AS), according to research presented today at the Heart Failure Congress 2013. AS, also referred to as male 'menopause', was four times ...
Seniors more likely to crash when driving with pet, study finds
(HealthDay)—Animals make great companions for senior citizens, but elderly people who always drive with a pet in the car are far more likely to crash than those who never drive with a pet, researchers have ...
New immune system discovered
(Medical Xpress)—A research team, led by Jeremy Barr, a biology post-doctoral fellow, unveils a new immune system that protects humans and animals from infection.
Brain can be trained in compassion, study shows
Until now, little was scientifically known about the human potential to cultivate compassion—the emotional state of caring for people who are suffering in a way that motivates altruistic behavior.
Death highest in heart failure patients admitted in January, on Friday, and overnight
Mortality and length of stay are highest in heart failure patients admitted in January, on Friday, and overnight, according to research presented today at the Heart Failure Congress 2013. The analysis of nearly 1 million ...