What Are IDEAs Made Of: Efficacy

Mike Rea

IDEA Pharma

Here’s the thing about Efficacy. It doesn’t exist. It is an abstract concept, a shorthand, a broad clustering of things that a product does. It is a descriptor under whose vagueness all kinds of disasters can happen.

If you threw your hands up and protested every time that you saw Efficacy with that capital letter, you’d be a long way to being hailed a strategic guru.

There is only one time in a Market Research presentation when the agency should be allowed to get away with capitalising ‘efficacy’, and that is when they show the slide that shows “Efficacy: what doctors meant when they said they want more of it…”

Unfortunately, that is wishful thinking. An overwhelming majority of primary research still comes back reporting that doctors want more Efficacy, Safety and Tolerability, and ideally at lower cost. At that point, the researchers had heard enough, and turned to PowerPoint for its handy paperclip and its HelpMe function: “I see that you’re creating a primary research report… Would you like to use the Efficacy, Safety, Price template?”

Imagine telling a salesman that you want a car. “What kind of car?” “One with more, well.., car-ness. One that does what cars do, but better…” Or, imagine presuming that people buy new mp3 players because they want to hear their mp3s better.


“Even when you have dutifully informed your audience that doctors want more Efficacy, they should still be sitting there saying, ‘well, d’uh… Which dimension, exactly?'”

Even when you have dutifully informed your audience that doctors want more Efficacy, they should still be sitting there saying, ‘well, d’uh… Which dimension, exactly?’ If we take something as simple as obesity, all of the following parameters could be included in a review of Efficacy: degree of average weight loss at a certain timepoint (the usual understanding), effect on satiety, effect on ‘food addiction’, durability of response, effect beyond withdrawal, amplification of lifestyle change, effect on visceral fat, effect on mood, effect on fat distribution, responder rate… Each respondent in a piece of Market Research study on obesity may have meant one or more of those dimensions when they chose ‘Efficacy’. It isn’t enough to think that we’re talking about the same thing – why not go one step further, and find out exactly?

(It is to be assumed that, having read this far, you understand that the same principle applies to Tolerability and Safety…)

This is all unfortunate, for it is in the detail that efficacy becomes beautiful, and strategic opportunity becomes possible. Consider the simple switch that enabled Lipitor to gain advantage: moving the market from measurement of outcomes to measurement of a lab test as a measure of efficacy.

The problem comes when we all assume we’re talking about the same thing when we use the word (unfortunately, English doesn’t allow a way to hear whether we mentally capitalised that ‘e’ or not…). The Development folks hear that physicians want more Efficacy and think, ‘well, that’s fine… Let’s go looking for something, anything, in the studies that fits under that banner…” Which is fine, if you’ve an unlimited budget, and you’re happy to believe that the drug is the best molecule in anyone’s pipeline. These days, companies who want to sharpen their Development and Marketing have to do better.


“These days, companies who want to sharpen their Development and Marketing have to do better.”

Even worse, you end up with a view that your product, from all the products out there, offers the perfect ‘balance’ of Efficacy and Tolerability. Consider an anticancer drug for a moment. Efficacy is often taken to mean Overall Survival, or Progression-Free Survival (when Overall Survival is considered to be a ‘better kind of efficacy’, you’d imagine that the point of this article was already made…). Those are perfectly reasonable things to measure. However, there are many other ways to evaluate the efficacy of an anticancer agent: visible effect on tumour regression, effect on symptoms, effect in different lines, in different stages, in different risk patients. All of those dimensions are running through the minds of oncologists, and then a marketer goes and claims that their drug offers ‘the perfect balance’… Little wonder if that doesn’t come across as more than a little thoughtless.

The problem of that capital E is manifest in any review of ‘Unmet Need’. (The same rule applies to those capital letters…) Because we can all agree that Efficacy is a shorthand for a granular set of (often conflicting) things that a drug might do, any review of unmet need must respect that granularity or be rendered pointless, a waste of Energy, Enthusiasm and Effort…

About the author:

Mike Rea is a Principal with IDEA Pharma, who enjoys taking a look outside the industry to learn how it can think differently. For direct enquiries he can be contacted on mike.rea@ideapharma.com and for more information on IDEA Pharma please see http://www.ideapharma.com/what/default.htm.

Is there too much confusion over the term ‘efficacy’?