Featured Research

from universities, journals, and other organizations

Study examines reasons for delay, denial of new drugs by FDA

Date:
January 21, 2014
Source:
The JAMA Network Journals
Summary:
Several potentially preventable deficiencies, including failure to select optimal drug doses and suitable outcome measures for a study, account for significant delays in the approval of new drugs by the Food and Drug Administration.

Several potentially preventable deficiencies, including failure to select optimal drug doses and suitable outcome measures for a study, accounted for significant delays in the approval of new drugs by the Food and Drug Administration (FDA), according to a study in the January 22/29 issue of JAMA.

Related Articles


"The road from medical product discovery to marketing is typically long and costly. The interval between initial clinical testing and product approval has been estimated to average 8 years and only 1 in 6 drugs entering clinical trials ultimately obtains U.S. Food and Drug Administration approval," according to background information in the article. Many drugs do not receive approval not because they are unsafe or ineffective, but because the information supplied is unsatisfactory to make that determination. Delays and failures that occur late in development affect the availability of innovative new drugs and increase the costs of drug development.

Leonard V. Sacks, of the U.S. Food and Drug Administration, Silver Spring, Md., and colleagues reviewed marketing applications for all new molecular entities (NMEs; active ingredients never before marketed in the United States in any form) first submitted to the FDA between 2000 and 2012. Using FDA correspondence and reviews, the researchers investigated the scientific and regulatory reasons approval of NMEs were delayed or denied.

Of the 302 identified NME applications, 151 (50 percent) were approved when first submitted and 222 (73.5 percent) eventually achieved marketing approval. Of the 151 first-cycle failures, 71 (47.0 percent) eventually obtained approval in a median (midpoint) of 435 days following the first unsuccessful submission.

Among the reasons for failure to initially receive approval:

  • Uncertainty about the optimal dose to maximize efficacy and to minimize safety risks;
  • Populations that were studied did not reflect the populations likely to use the drug;
  • End points used in clinical trials were unsatisfactory;
  • Inconsistent results for multiple predefined end points in clinical studies;
  • Inconsistencies in efficacy for portions of the study population.

There were 20 drugs (13.2 percent) that despite showing superiority to placebo were considered to have inadequate efficacy compared with the standard of care.

The frequency of safety deficiencies was similar among never-approved drugs compared with those with delayed approval. However, efficacy deficiencies were significantly more frequent among the never-approved drugs than among those with delayed approvals. Among the 48 drugs with initial efficacy concerns alone, only 31.3 percent were eventually approved compared with 61.5 percent of the 39 drugs with safety concerns alone.

"Failures late in drug development are costly, often involving the commitment of many study participants and personnel. It is advantageous to identify products that fail as early as possible in the development process to avoid these issues. For those drugs that require resubmission before approval is obtained, delays are taxing on the industry and regulators, and patients may have to wait for access to promising, and sometimes lifesaving, new treatments," the authors write.

"Our findings may be helpful to clinicians and policy makers in interpreting the extensive literature reporting the design and outcome of clinical trials, which in turn may have an effect on practice. For drug developers and clinical investigators, our findings suggest areas of deficiencies in new drug applications in which strategies for drug development could be improved. Early and frequent dialogue between the FDA and drug sponsors addressing critical aspects of study design (including the selection of study populations, study end points, and drug doses) has the potential to reduce delays in the approval of new drugs."


Story Source:

The above story is based on materials provided by The JAMA Network Journals. Note: Materials may be edited for content and length.


Journal Reference:

  1. Leonard V. Sacks, Hala H. Shamsuddin, Yuliya I. Yasinskaya, Khaled Bouri, Michael L. Lanthier, Rachel E. Sherman. Scientific and Regulatory Reasons for Delay and Denial of FDA Approval of Initial Applications for New Drugs, 2000-2012. JAMA, 2014; 311 (4): 378 DOI: 10.1001/jama.2013.282542

Cite This Page:

The JAMA Network Journals. "Study examines reasons for delay, denial of new drugs by FDA." ScienceDaily. ScienceDaily, 21 January 2014. <www.sciencedaily.com/releases/2014/01/140121164750.htm>.
The JAMA Network Journals. (2014, January 21). Study examines reasons for delay, denial of new drugs by FDA. ScienceDaily. Retrieved December 19, 2014 from www.sciencedaily.com/releases/2014/01/140121164750.htm
The JAMA Network Journals. "Study examines reasons for delay, denial of new drugs by FDA." ScienceDaily. www.sciencedaily.com/releases/2014/01/140121164750.htm (accessed December 19, 2014).

Share This


More From ScienceDaily



More Health & Medicine News

Friday, December 19, 2014

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

The Best Tips to Curb Holiday Carbs

The Best Tips to Curb Holiday Carbs

Buzz60 (Dec. 19, 2014) It's hard to resist those delicious but fattening carbs we all crave during the winter months, but there are some ways to stay satisfied without consuming the extra calories. Vanessa Freeman (@VanessaFreeTV) has the details. Video provided by Buzz60
Powered by NewsLook.com
Kids Die While Under Protective Services

Kids Die While Under Protective Services

AP (Dec. 18, 2014) As part of a six-month investigation of child maltreatment deaths, the AP found that hundreds of deaths from horrific abuse and neglect could have been prevented. AP's Haven Daley reports. (Dec. 18) Video provided by AP
Powered by NewsLook.com
Dads-To-Be Also Experience Hormone Changes During Pregnancy

Dads-To-Be Also Experience Hormone Changes During Pregnancy

Newsy (Dec. 18, 2014) A study from University of Michigan researchers found that expectant fathers see a decrease in testosterone as the baby's birth draws near. Video provided by Newsy
Powered by NewsLook.com
Prenatal Exposure To Pollution Might Increase Autism Risk

Prenatal Exposure To Pollution Might Increase Autism Risk

Newsy (Dec. 18, 2014) Harvard researchers found children whose mothers were exposed to high pollution levels in the third trimester were twice as likely to develop autism. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins