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The Well-Framed Question of Efficacy

To best inform the decision to take the new agent, the right question of efficacy would be:

1. In patients with elevated cholesterol who are most similar to us,

2. is the ‘New Agent’ more effective than

3. the standard recommended dose for a particular statin therapy with respect to

4. heart attacks, stroke and death.”

If you frame your question this way, you will focus your search to find information that will be most relevant and meaningful to you and your doctor. You are more likely to find the right answer to the right question.

The 5 Key Outcomes

Even though you may have clearly framed your question on efficacy, you still need to ask more questions. There are four other outcomes you need to consider separately. The mnemonic “STEPC” helps you remember all the outcomes that are needed to assess with your doctor whether one therapy is likely to be one “STEPC” better than another:

Safety: These are serious and usually infrequent complications like death, seizures or liver failure;
Tolerability: These are usually more common nuisance side effects like nausea or dry mouth;
Efficacy: as discussed above;
Price: Your out-of-pocket costs. Your health plan will also consider whether the drug is cost-effective;
Convenience: Frequency of daily use? Is it oral, topical or injected, easy to use, etc? These factors affect whether you are likely to take the therapy as prescribed.

The Well-Framed Question of Safety

Unlike questions of efficacy where you compare an intervention against a best available standard, for questions of safety you want to know if the intervention is as safe as placebo (“doing nothing”).

Thus an important and appropriately framed question of safety in our scenario would be:

1. In patients with an elevated cholesterol who are most similar to us,

2. is the New Agent safe with respect to

4. liver failure or other serious complications.

All 5 Key Questions

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The Well-Framed Question of Efficacy

The 5 Key Outcomes

The Well-Framed Question of Safety