Subscribe to Newsletter
Discovery & Development Clinical Trials

Efficacy vs Effectiveness

Traditional efficacy trials are not enough to guarantee that a drug will work in the more diverse population seen in the clinic, according to researchers who evaluated the effectiveness of an inhaled drug combination for chronic obstructive pulmonary disease (COPD) in everyday clinical practice (1).

“Efficacy studies are limited in their usefulness to clinicians as they are often restricted in their inclusion criteria, meaning that they show what the drugs can do in a controlled setting but not necessarily what they can do in the real world,” says Jørgen Vestbo, first author and professor of respiratory medicine at the University of Manchester, UK.

In fact, the authors suggest that fewer than 10 percent of COPD patients would normally be eligible for efficacy trials, since they typically exclude anyone with a coexisting condition. The investigators carried out a randomized study in patients under the care of general practitioners, without the frequency, monitoring or strict eligibility criteria of a controlled trial, to allow for the variation in adherence, dosing frequency, and inhaler technique seen in unsupervised patients.

Rather than efficacy under ideal conditions, the trial assessed the real-world effectiveness of an inhaled combination of fluticasone furoate and vilanterol. The results showed that a broad population of COPD patients benefitted from the inhalant combination, without a significantly greater risk of adverse effects. The authors argue in their paper that incorporating effectiveness trials as a standard component of the translational process would provide much clearer evidence on which to base clinical decisions.

“It’s not a question of either/or,” says Vestbo, “Efficacy studies are still needed; however, effectiveness studies are also required to ensure that the drugs have the expected effects in the real world.”

Receive content, products, events as well as relevant industry updates from The Medicine Maker and its sponsors.
Stay up to date with our other newsletters and sponsors information, tailored specifically to the fields you are interested in

When you click “Subscribe” we will email you a link, which you must click to verify the email address above and activate your subscription. If you do not receive this email, please contact us at [email protected].
If you wish to unsubscribe, you can update your preferences at any point.

  1. J Vestbo et al, “Effectiveness of fluticasone furoate–vilanterol for COPD in clinical practice”, N Engl J Med, [Epub ahead of print] (2016). PMID: 27593504.
About the Author
William Aryitey

My fascination with science, gaming, and writing led to my studying biology at university, while simultaneously working as an online games journalist. After university, I travelled across Europe, working on a novel and developing a game, before finding my way to Texere. As Associate Editor, I’m evolving my loves of science and writing, while continuing to pursue my passion for gaming and creative writing in a personal capacity.

Register to The Medicine Maker

Register to access our FREE online portfolio, request the magazine in print and manage your preferences.

You will benefit from:
  • Unlimited access to ALL articles
  • News, interviews & opinions from leading industry experts
  • Receive print (and PDF) copies of The Medicine Maker magazine