Only approximately half of drugs that are newly approved by the US FDA include data which compares the new medication with existing alternatives – known as comparative effectiveness (efficacy) data – thus hindering a doctor’s ability to make the best treatment decisions, researchers from Brigham and Women’s Hospital and Harvard Medical School reported in JAMA (Journal of the American Medical Association).

In fact, the authors add that almost one-third of newly FDA-approved medications have no such information at all.

The US Congress assigned $1.1 billion for comparative effectiveness research. The Institute of Medicine defines this type of research as “generation and synthesis of evidence that compares the benefits and harms of alternative methods to prevent, diagnose, treat, and monitor a clinical condition or to improve the delivery of care.”

Information comparing the efficacy of drugs is useful for doctors when deciding on treatment options.

The authors explained:

“Comparative effectiveness is taking on an increasingly important role in U.S. health care, yet little is known about the availability of comparative efficacy data for drugs at the time of their approval in the United States.”

Nikolas H. Goldberg, and team set out to find out what proportion of newly approved medications included comparative efficacy data, how long it took for that data to become available, and whether certain medical areas varied in the inclusion of such data.

They determined whether effectiveness studies were head-to-head active controlled trials, and also whether those trial results were available in the packaging.

They identified 197 eligible approved New Molecular Entities (NMEs) between 2000 and 2010. 51% (100) of them had comparative effectiveness data available when they went onto the market. After they excluded orphan products and other drugs for which no alternative treatments existed anyway, the percentage of drugs with comparative effectiveness data stood at 70% – or nearly one third without such data.

The percentage of NMEs with comparative efficacy data (excluding orphan drugs and those with no alternative therapies anyway) varied from 50% to 92%, depending on the year.

The availability of comparative efficacy data varied according to therapeutic area, as seen below (therapeutic areas with comparative efficacy data):

  • Diabetes mellitus – 89%
  • Infectious diseases – 73%
  • Hormones and contraceptives – 33%
  • Cancer – 35%

After excluding drugs for which no alternative treatment existed anyway, as well as orphan medications, the difference between therapeutic areas was much less, the authors wrote. They also found that medications that received priority review designations had a much smaller chance of having any comparative effectiveness data.

The authors concluded:

“In conclusion, we found that publicly available documents include results of at least 1 head-to-head trial with an approved alternative for approximately half of all newly approved NMEs. Strategies are needed to enhance the accessibility of, and ultimately the use of, this information, particularly in the early marketing experience, when comparative effectiveness data from other sources are scarce or nonexistent.”

“Availability of Comparative Efficacy Data at the Time of Drug Approval in the United States”
Nikolas H. Goldberg; Sebastian Schneeweiss, MD, ScD; Mary K. Kowal, BA; Joshua J. Gagne, PharmD, MS
JAMA. 2011;305(17):1786-1789. doi: 10.1001/jama.2011.539

Written by Christian Nordqvist