Busting 5 common myths around access to unlicensed medicines

There are some common misconceptions or myths regarding providing access to unlicensed medicines that I hear on a regular basis in my global business development role at Clinigen. Here are a few I most frequently hear and thought it would be worth sharing.

Myth #1: “Access to an unlicensed medicine only occurs during the early stages of a medicine’s life”

Reality: Access to an unlicensed medicine can actually happen at any time during the medicine’s lifecycle. Initial demand is driven at the early stages of a medicine’s life, prior to first global approval or alongside early commercial roll-out, with Health Care Professionals (HCPs) trying to gain access in countries where it is not yet licensed. However, the medicine may remain unavailable or unlicensed for many years in some countries where there are significant launch delays or indeed no commercial activities planned at all. In a number of cases we see medicines that were first launched 10-15 years ago still being accessed as an unlicensed medicine in various parts of the world providing a vital lifeline to patients. It is therefore important that controlled and compliant ways to address this need are in place throughout a medicine’s lifecycle.

Myth #2: “Unlicensed medicines are only made available for life threatening or fast progressing diseases”

Reality: Of course the history of unlicensed medicines is closely linked to fast progressing life threatening diseases, however, we also see HCPs wanting to gain access to unlicensed medicines for many other disease areas. For example, a large amount of unlicensed medicines are for the treatment of slow progressing chronic inflammatory or neurological conditions where there might be a ‘treatment window of opportunity’ that can be addressed by an unlicensed medicine. The overriding factor here is not the disease type, but whether the treating physician feels an unlicensed medicine is more appropriate to treat their patient than licensed alternatives, whether the treatment is life saving, life extending or life enhancing.

Myth #3: “Pharma companies need to review and approve every patient that gains access to their unlicensed medicine”

Reality: Sure – if the medicine is very ‘early’ in its lifecycle then the pharma company are going to want to have control over the type of patient being treated with their medicine via a set of specific eligibility criteria and may want to approve some patients on a case-by-case basis – typically those who do not meet all the criteria. However this is the pharma company’s decision, not a mandate. As the medicine moves through the life cycle and starts to become commercially available in some markets, these eligibility criteria tend to be relaxed to the point where eventually, they are removed entirely, and it is purely the treating physician that deems whether the medicine is appropriate for their patient.

Myth #4: “You cannot collect real world data when providing access to an unlicensed medicine”

Reality: You can collect data when providing access to an unlicensed medicine and in fact there is a view that if you are providing access to an unlicensed medicine then you should be capturing some data. Moreover, the type of data that can be collected can be more than top-level access data and actually fall into the ‘real world’ bracket including clinical data that help identify how the medicine is working in the real world. Once again the earlier the medicine is in the life cycle the more data that is typically collected which could form the missing piece of the jigsaw when trying to demonstrate the value of a medicine to payors and HCPs.

Myth #5: “We can’t tell HCPs that you are making an unlicensed medicine available”

Reality: It is important that if HCPs are proactively seeking information about access to an unlicensed medicine that this information is both easy to find and helpful. There is no benefit to having a compliant and controlled route to supply an unlicensed medicine if this information cannot be easily accessed and there are many, compliant ways to ensure communication does not become a barrier to access. In fact the 21st Century Cures Act actually mandates industry to provide information about their stance on providing access to unlicensed medicines in a publically available place such as their website.