Welcome to Study of the Week from Patient Worthy. In this segment, we select a study we posted about from the previous week that we think is of particular interest or importance and go more in-depth. In this story we will talk about the details of the study and explain why it’s important, who will be impacted, and more.
If you read our short form research stories and find yourself wanting to learn more, you’ve come to the right place.
This week’s study is…
Therapeutic value of first versus supplemental indications of drugs in US and Europe (2011-20): retrospective cohort study
We previously published about this research in a story titled “Study: Recent Drug Approvals Offer Less Therapeutic Value” which can be found here. The study was originally published in the British Medical Journal. You can read the full text of the study here.
What Happened?
As new drugs gain approval in the US and Europe, they often see expanded use for multiple conditions or diseases. These expanded approvals are dubbed supplemental indications. The goal of this study was to assess the real-world value of drugs that had been approved from 2011-2020. The research team’s approach for reaching this goal was to assess the therapeutic value of the first indications versus the supplemental indications for the drugs that were approved during those years.
This study relied on publicly accessible data, which found 124 first indications from the US Food and Drug Administration (FDA) and 88 from the European Medicines Agency (EMA). Meanwhile, there were 334 supplemental indications from the FDA and 215 from the EMA. 48% of drugs in both regions had one supplemental indication; in the EU, 23% carried two, 13% carried three, and 17% had even more. As for the US, 20% carried two, 14% carried three, and 18% had at least four. 58% of these indications in both regions were for cancer treatment.
For the FDA approved therapies, 41% carried high therapeutic value ratings for first approval indications. 34% of supplemental indications carried high value ratings. As for Europe, 47% carried high therapeutic value ratings and 36% of supplemental indications did. Generally, the more supplemental indications a therapy had, the less likely that the subsequent indication held a high value rating.
The team concluded that less than half of the approvals that had been given a value rating during the timeframe studies carried high therapeutic value. The researchers emphasized that patients and physicians both need to be informed that new therapies aren’t necessarily far and away superior to older ones—and that therapeutic value should also be reflected during drug pricing.
Weaknesses of the study include the fact that therapeutic value ratings weren’t available for all drugs, particularly those approved by the FDA that didn’t get EMA approval later. Country specific assumptions and factors could also influence the findings.
Why Does it Matter?
When a new drug is introduced to the market, even for a disease that already has approved therapies, press releases and drug marketing often portray the new treatment as a major breakthrough or innovation in the treatment of the disease. While there are real instances where this is true, the results of this study suggest that it’s not happening as often as is portrayed in the media.
At the end of the day, perhaps the results here shouldn’t be too surprising. After all, we live in a system where the principal goal of drug development is bringing a profitable product to market. While pharmaceuticals companies are not willing to admit this openly, other considerations, such as real-world benefits to patients’ lives, are secondary.
It shouldn’t be up to patients to figure out of a new treatment will provide greater benefit than an older one. While there is some value to simply having multiple treatment options, particularly if a patient doesn’t respond to the first line treatment, the industry risks creating redundancy and confusion by introducing new therapies that aren’t superior to old ones.
