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Off-label use of drugs is not prohibited and originates from the idea that physicians should, based on their professional experience, be allowed to prescribe drugs not yet approved with the intention-to-treat. In the US, the requirement for off-label use of drugs is that the drug is on the market, i.e. approved by the FDA for some other indications (mainly due to safety reasons). Also, the law prohibits companies to promote off-label use. Anyway, the off-label use is widely practised and there are no reasons to believe that this will change in the nearest future.
Belinostat is currently being tested in a phase II clinical trial in combination with carboplatin and paclitaxel (the BelCaP combination) and the objective is to demonstrate improved efficacy in comparison to carboplatin and paclitaxel (CaP) when used to treat patients suffering from carcinoma of unknown primary (CUP). Based on previous study data there are strong reasons to believe that upcoming data, which are to emerge in H2 2011, will show that the BelCaP combination is more efficient than CaP alone. Today, it do not exist any effective treatment for CUP and CaP is the standard treatment for patients within the US.
CUP is a serious mid-size disease with a poor prognosis affecting ~83000 persons within the US, Japanand 5 major European countries annually. Global sales of belinostat for this indication have been estimated to reach 0.5-0.6 BUSD by Topotarget.
Because a new drug application (NDA) will be submitted to the FDA by the end of this year or early 2012 for belinostat in the treatment of peripheral t-cell lymphoma (PTCL), it is likely that this drug will be approved by the end of 2012 by the FDA [because of a generous special protocol assessment, SPA]. So, if the ongoing phase II data for CUP turn up to be as good as expected by Topotarget, off-label use may be initiated as soon as late 2012 or 2013.
To determine whether off-label sales of belinostat is a realistic option or merely a far-fetched one, I wrote to the principal investigators in the ongoing CUP study and asked them if they believe that physicians within the US will generally prescribe off-label use of belinostat with the intention-to-treat patients suffering from CUP provided that 1) the BelCap combination shows significantly better efficacy than CaP alone and 2) if the side-effects and toxicity of this combination can be viewed as mild. Although I chose to not reveal the names, I can assure you that they have an extensive experience in treating patients suffering from CUP and are widely recognized in this field.
Answers (3 received):
“If the phase II trial (now completed accrual) shows a benefit for patients receiving chemotherapy + belinostat, oncologists in the USwould be interested in using this combination for treatment of their patients with CUP. However, since belinostat is not currently approved for any indication in the US, use would not be possible. If it gets approved for T cell lymphoma or something else, a positive CUP study would lead to off-label use for that indication. “
“The key to adoption of "Off-Label" use of a treatment regimen in the USis listing of the treatment by a 'Compendium' recognized by Medicare and other 3rd party payers for reimbursement. The Carboplatin-Paclitaxel combination currently enjoys such compendium recognition. If the BelCaP regimen is demonstrated to have significant activity, without significant added toxicity, is published in a recognized peer-reviewed medical journal, and is recognized by a compendium publication, such as the NCCN, then it would find broad use in the US.”
"The main problem with off-label use in the United States is cost and coverage of the medication. If the data shows improved efficacy and no increased toxicity physicians will be willing to use it for their patients. Most insurance companies now require pre-certification for chemotherapy approval, and if the drug is off-label it may be denied. In these instances other pharma companies had establish patient access programs that cover or provide the drug."
Because also a minor market penetration in the off-label use of belinostat [for CUP] may imply significant contribution to the cash-flow from 2013 and onwards, this information should be viewed as very interesting for a potential investor in Topotarget. It remains however to estimate the numbers but because this task involves the issues of insurance policies, market penetration and most important how good upcoming data will really be, I leave that to others to do.
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