I believe that it was Plavac who few days back mentioned a potential large market for Leronlimab: off-label indications.
This becomes now a reality that cannot be ignored after the basket announcement; Let’s have a look at this (italics are copy-and-paste from relevant literature):
What is off-label drug use?
In the United States new drugs are tested in clinical trials (research studies) before they are approved by the US Food and Drug Administration (FDA) for use in the general public. The clinical trials are done to show that the drug:
• Works to treat a certain medical condition
• Works the way it’s expected to
• Is safe when used as directed
When the FDA is satisfied that the drug works and is safe, it and the maker of the drug create the drug label. This is not an actual label that sticks to a bottle, but a report of very specific information about the drug. The FDA must approve this report, which is made available to all health professionals who prescribe or sell the drug.
The drug label gives information about the drug, including the specific medical condition(s) it’s approved for (called the indication(s) for use), the doses to be used, and how it’s to be given. When a drug is used in a way that is different from that described in the FDA-approved drug label, it’s said to be an “off-label” use. This can mean that the drug is:
• Used for a different disease or medical condition
• Given in a different way (such as by a different route)
• Given in a different dose than in the approved label
For example, when a chemotherapy drug is approved for treating one type of cancer but is used to treat a different cancer, it’s off-label use. The same is true if a drug is approved to treat a type of cancer at a specific stage (extent of spread), but it is used to treat a cancer at a different stage.
Off-label is also called non-approved or unapproved use of a drug.
So, what is the potential for this market ?? A systematic literature search was performed in PubMed as per the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines from 1975 to 2016. Studies assessing the prevalence of off-label use of anticancer drugs were included. Data synthesis: Of the 199 eligible papers retrieved, 23 studies were included in this systematic literature review. Off-label drug use in inpatients ranged from 18% to 41%. Among adult patients with cancer, 13%-71% received a minimum of one off-label chemotherapy. The main reasons for off-label drug use were 'drug unapproved for specific tumor' and 'modified drug applications'. Among adults, metastatic cancers and palliative care patients received the most off-label drugs. The off-label drug use unsupported by standard treatment guidelines or drug compendia was in the range of 7%-31%. Conclusion: Off-label drug use in cancer therapy is commonly practiced but outcomes could vary significantly.
All this, only for oncology. The HIV/AIDS community is also known to use extensively off-label drugs: “A 2008 study found that 8 out of 10 cancer doctors surveyed had prescribed drugs off-label. Off-label drug use is also well-documented and very common in certain other settings, such as in pediatrics and HIV/AIDS care.”
So, we are talking here of a potentially HUGE market. The main point here is that it will spread rapidly as soon as: the FDA approves Lero for one indication, and Lero is understood for the medical community at large to be a SAFE drug with myriad of indications in both, cancer and HIV.
What about the market in dollars? Well, I will not dare to start calculating it at this point as if will be difficult for only one drug, impossible for many, however I am sure we understand the implications of having a drug out there that can be safely prescribed for a basket of immunological ailments.
This becomes now a reality that cannot be ignored after the basket announcement; Let’s have a look at this (italics are copy-and-paste from relevant literature):
What is off-label drug use?
In the United States new drugs are tested in clinical trials (research studies) before they are approved by the US Food and Drug Administration (FDA) for use in the general public. The clinical trials are done to show that the drug:
• Works to treat a certain medical condition
• Works the way it’s expected to
• Is safe when used as directed
When the FDA is satisfied that the drug works and is safe, it and the maker of the drug create the drug label. This is not an actual label that sticks to a bottle, but a report of very specific information about the drug. The FDA must approve this report, which is made available to all health professionals who prescribe or sell the drug.
The drug label gives information about the drug, including the specific medical condition(s) it’s approved for (called the indication(s) for use), the doses to be used, and how it’s to be given. When a drug is used in a way that is different from that described in the FDA-approved drug label, it’s said to be an “off-label” use. This can mean that the drug is:
• Used for a different disease or medical condition
• Given in a different way (such as by a different route)
• Given in a different dose than in the approved label
For example, when a chemotherapy drug is approved for treating one type of cancer but is used to treat a different cancer, it’s off-label use. The same is true if a drug is approved to treat a type of cancer at a specific stage (extent of spread), but it is used to treat a cancer at a different stage.
Off-label is also called non-approved or unapproved use of a drug.
So, what is the potential for this market ?? A systematic literature search was performed in PubMed as per the Preferred Reporting Items for Systematic Reviews and Meta-Analyses guidelines from 1975 to 2016. Studies assessing the prevalence of off-label use of anticancer drugs were included. Data synthesis: Of the 199 eligible papers retrieved, 23 studies were included in this systematic literature review. Off-label drug use in inpatients ranged from 18% to 41%. Among adult patients with cancer, 13%-71% received a minimum of one off-label chemotherapy. The main reasons for off-label drug use were 'drug unapproved for specific tumor' and 'modified drug applications'. Among adults, metastatic cancers and palliative care patients received the most off-label drugs. The off-label drug use unsupported by standard treatment guidelines or drug compendia was in the range of 7%-31%. Conclusion: Off-label drug use in cancer therapy is commonly practiced but outcomes could vary significantly.
All this, only for oncology. The HIV/AIDS community is also known to use extensively off-label drugs: “A 2008 study found that 8 out of 10 cancer doctors surveyed had prescribed drugs off-label. Off-label drug use is also well-documented and very common in certain other settings, such as in pediatrics and HIV/AIDS care.”
So, we are talking here of a potentially HUGE market. The main point here is that it will spread rapidly as soon as: the FDA approves Lero for one indication, and Lero is understood for the medical community at large to be a SAFE drug with myriad of indications in both, cancer and HIV.
What about the market in dollars? Well, I will not dare to start calculating it at this point as if will be difficult for only one drug, impossible for many, however I am sure we understand the implications of having a drug out there that can be safely prescribed for a basket of immunological ailments.
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