Frequently Asked Questions


How we create the DrugFactsBox™ and how to use one to help you learn about drugs

 

Q:  Where do you get your information?  

A: All of our data comes from the FDA’s review of the drug, the FDA-approved package insert, or other high-quality systematic reviews.

 

Q: Are you conducting these trials and making these conclusions?

A: No,  we use information that the drug company submitted to the FDA when they wanted to get the drug approved for this use. The analysis and these conclusions are the FDA’s – we make it easier for you to access this valuable information so you can answer questions like “Is this drug worth it?”

 

Q: What do you mean by “main” trials?

A: When we say “main” trials, we are referring to trials that the FDA considered “pivotal,” or crucial, in establishing that the drug works and that its benefits outweigh its harms. These are often - but not always - the largest and/or longest of the trials that the FDA reviews when evaluating a drug for approval.

 

Q: What do you mean by a “placebo-controlled” trial?

A: To understand the true effect of a drug, you need to compare it to something that isn’t changing – this is called a control. In a placebo-controlled trial, people randomly get assigned to take either the drug being tested, or to take a placebo.  A placebo looks like the drug the others are taking, but often has no medication in it. Sometimes these are called “sugar pills.” In a placebo-controlled trial, we can understand how the drug can help compared to taking no medication at all. In these trials, the drug company is asking: Does this drug help compared to taking nothing?

 

Q: What do you mean by an “active-controlled” trial?

A: In an active-controlled trial, some people receive the drug being tested, while others receive a different drug used to treat the same condition. These trials are often more meaningful than placebo-controlled trials because they compare the effects of two or more drugs used for the same condition, rather than comparing the effect of the drug to doing nothing.  In these trials, the drug company often asks two questions: Is this drug as good as other drugs already on the market? (If the answer is yes, then the drug has met what is called “non-inferiority” because the new drug is not inferior – not worse – than drugs already on the market.) Is this drug better than other drugs on the market? (If the answer is yes, then the drug has “superiority”, which means it has more benefit and sometimes even fewerharms than other drugs on the market.

 

Q: How can I tell if a drug works, and if so, can I tell how much?

A: Each DrugFactsBox’s Trials tab contains Clinical Trials data that are organized by the specific groups of people that the drug was tested in.  This is important because a drug might not have the same effect on people of different ages, severity of condition, or prior treatment history. In the Benefits section under each trial you will find the results. “Outcomes” are the concepts or key questions that the FDA wants to know about a drug. For example, some of these key questions might be: Does the drug relieve depression? Do asthma symptoms get worse? Outcomes are displayed in the blue column in the Results section. The FDA answers those questions by looking at the different ways the drug company tried to answer them.  We call these metrics and they are in the grey columns in the Results section. For example, metrics might be average depression scores on a scale of 0 [no depression] to 52 [severe depression], number of puffs of an inhaler used per day, or percentage of people who had a stroke. Whatever the outcome, you can get a sense of the drug’s effect by comparing the number in the drug group to the number in the control group.

 

Q: What do you mean by “DRUG helped?”

A: When we say “DRUG helped” in the context of a placebo-controlled trial, we mean that the difference between the outcome measured in the drug group and the outcome measured in the placebo group is “statistically significant”- that is, we can be 95% sure that the difference between the drug’s effect and the placebo’s effect is not due to chance.  A drug’s effect tends to be statistically significant if the measured difference and the population size are both sufficiently large.

 

Q: What do you mean by “DRUG did NOT help?”

A: When we say “DRUG did NOT help” in the context of a placebo-controlled trial, we mean that the difference between the outcome measured in the drug group and the outcome measured in the placebo group is NOT “statistically significant”- that is, we cannot be 95% sure that the difference between the drug’s effect and the placebo’s effect is not due to chance. A drug’s effect tends to not meet statistical significance if the measured difference and/or the population size are not large enough.

 

Q: What do you mean by “No FDA comment?”

A: When we say “No FDA comment” in the context of a placebo- or active-controlled trial, we mean that the FDA did not report whether or not the difference between the outcome measured in the drug group and the outcome measured in the control group is “statistically significant.” Often, the FDA will not report on statistical significance for “secondary” outcomes, because these are not considered crucial for establishing that the drug works.  Likewise, the FDA will sometimes leave out a report on statistical significance for the difference between the outcome measured in the drug group and the outcome measured in one of the “secondary” control groups because this comparison is not considered crucial for establishing that the drug works.

 

Q: What do you mean by “DRUG better?”

A: When we say “DRUG better” in the context of an active-controlled superiority trial, we mean that the difference between the outcome measured in the test drug group and the outcome measured in the control drug group is “statistically significant”- that is, we can be 95% sure that the difference between the test drug’s effect and the control drug’s effect is not due to chance- and favors the test drug.  A drug’s effect tends to be statistically significant if the measured difference and the population size are both sufficiently large.

 

Q: What do you mean by “DRUG worse?”

A: When we say “DRUG worse” in the context of an active-controlled superiority trial, we mean that the difference between the outcome measured in the test drug group and the outcome measured in the control drug group is “statistically significant”- that is, we can be 95% sure that the difference between the test drug’s effect and the control drug’s effect is not due to chance - and favors the control drug.  A drug’s effect tends to be statistically significant if the measured difference and the population size are both sufficiently large.

 

Q: What do you mean by “DRUG NOT better?”

A: When we say “DRUG NOT better” in the context of an active-controlled superiority trial, we mean that the difference between the outcome measured in the test drug group and the outcome measured in the control drug group is NOT “statistically significant”- that is, we cannot be 95% sure that the difference between the test drug’s effect and the control drug’s effect is not due to chance. A drug’s effect tends to not meet statistical significance if the measured difference and/or the population size are not large enough.

 

Q: What do you mean by “DRUG NOT worse?”

A: When we say “DRUG NOT worse” in the context of an active-controlled non-inferiority trial, we mean that the FDA concluded that the test drug’s effect on the measured outcome was not any worse than the control drug’s effect on the measured outcome.  Usually the FDA will set a boundary value for the difference between the test drug’s effect on the outcome and the control drug’s effect on the outcome, and as long as the difference does not exceed that limit, the test drug is determined to be “non-inferior” or not any worse than the control drug. Sometimes, superiority and non-inferiority will be tested in the same trials, with or without multiple controls. What does this mean for you? It means a new drug may work as well as the current existing drugs already available.

 

Q: What is a Black Box Warning and why should I pay attention to these?

A: Black Box Warning is the FDA’s most serious side effect alert. It’s called “Black Box” because on the FDA-approved package insert (the tissue paper pamphlet that comes with your drug), this information is presented in a black outlined box. Pay attention to which drugs have black box warnings as these are often side effects that are potentially life-threatening.  If you have choice between two drugs that work equally well but one has a black box warning, you might want to choose the drug without the black box warning.  

 

Q: What do you mean by “serious” side effects?

A: When we say “serious” side effects, we are referring to side effects that the FDA has required the pharmaceutical company to include in the “Warnings and Precautions” section of the FDA-approved package insert.  A package insert  comes with every  prescription.  Generally, if you experience any of a drug’s serious side effects, you should immediately notify your doctor and seek further medical attention. Sometimes, the rates at which these side effects occurred in clinical trials are not reported in the Warnings and Precautions section of the package insert but are taken from the FDA’s review.

 

Q: What do you mean by “symptom” side effects?

A: When we say “symptom” side effects, we are referring to side effects that the FDA has required the pharmaceutical company to include in the “Adverse Reactions” section of the FDA-approved package insert that comes with a prescription, along with other side effects which may not appear in the package insert but apply to the population described in the Informulary indication.  Like serious side effects, some symptom side effects may not have rates reported in the Adverse Reactions section of the package insert but are taken from the FDA’s review. Generally, these side effects have occurred at a rate that is some number greater in the test drug group than in the control group. These side effects can be attributed to the test drug rather than to chance or some other element of the trial’s design.

 

Q: What do you mean by “Extra X% of people had SIDE EFFECT from DRUG?”

A: When we say “Extra X% of people had SIDE EFFECT from DRUG,” we are reporting the difference between the rate of the side effect in the drug group and the rate of the side effect in the placebo group. Often, the rate of a side effect in the drug group will be the same as the rate of the side effect in the placebo group. These side effects are more likely to be caused by chance or some other element of trial design than the actual drug.

 

Q: What do you mean by “More people had SIDE EFFECT with DRUG?”

A: When we say “More people had SIDE EFFECT with DRUG,” we are referring to a side effect that the FDA explicitly states occurred at a higher rate in the test drug group than in the control drug group. Conversely, when we say “Fewer people had SIDE EFFECT with DRUG,” we are referring to a side effect that the FDA explicitly states occurred at a lower rate in the test drug group than in the control drug group.

 

Q: What do you mean by “Seen with both drugs?”

A: When we say “Seen with both drugs,” we are referring to a side effect that is reported in both the test drug group and the control drug group, but the FDA has not explicitly stated which group had a higher rate of the side effect.

 

Q: What do you mean by “Not a side effect at this dose but seen with higher/other doses?”

A: When we say “Not a side effect at this dose but seen with higher/other doses,” we are referring to side effects that are highlighted by the FDA because they occurred at a higher rate than the control group at one dose, but not the dose for which we are reporting data. We point these out in case your doctor changes your dose so you get the full picture of possible side effects.  

 

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