The Food and Drug Administration Safety and Innovation Act (FDASIA), which was signed on July 9, 2012, introduced and described a new designation – Breakthrough Therapy Designation. According to FDA, “a breakthrough therapy is a drug:
- Intended alone or in combination with one or more other drugs to treat a serious or life threatening disease or condition and
- Preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.”
A breakthrough is “any significant or sudden advance, development, achievement, or increase, as in scientific knowledge or diplomacy that removed a barrier to progress.” FDA uses the word to refer to a part of the review process. Since this designation does not necessarily state or confirm that the drug in question is effective or will help patients live longer, referring to the product as a “breakthrough therapy” could potentially be misleading to the public.
“Breakthrough designation is based on preliminary evidence, which can include changes in surrogate markers of disease, such as laboratory measurements, that do not always translate into meaningful clinical benefit.” According to the Agency, there have been 267 requests for breakthrough therapy designation; 95 of which have been granted. Although some of the products that have been granted breakthrough designation may actually be “breakthroughs” it is not likely that the majority of them are.
A recent study surveyed roughly 600 Americans over the age of 18, asking them to read various “descriptions” of a hypothetical drug which were based on the language used in actual FDA press releases. Of the descriptions, one contained only “facts” about the hypothetical drug, while the other two contained additional information and referred to the product as either “promising” or as a “breakthrough” therapy.
Not surprisingly, the results from the study showed that the inclusion of terms such as “promising” or “breakthrough” tend to result in “higher ratings of the drug’s effectiveness and perceptions of the strength of supporting evidence.” For more details on the study, view the full report here.
The question many people are having, is should FDA be publishing press releases with terms that are more neutral? Would the inclusion of less impactful terms help consumers make more accurate and informed judgements about these products? While only time will show how this plays out, it will certainly be interesting to see the end results.
Do you have a product that may be eligible to receive breakthrough designation? We can help determine if your drug is eligible for this designation or others. To learn more about how we can help you, email us here.